2020 Pharm Exec 50 United

LEADERSHIP TECHNOLOGY FINANCE STRATEGY CRISIS PLAYBOOK QUANTUM LEAPS TIME FOR DIALOGUE THE SOCIAL MANDATE Pharmaceutical ® MASTER THE SCIENCE OF SUCCESS Executive

2020 PHARM EXEC 50 UNITED

FRONTDELIVERING HOPE IN A PANDEMIC

June 2020 VOLUME 40, NUMBER 6 WWW.PHARMEXEC.COM Now and Next: A COVID-19 Access and Patient Engagement Update

On-demand COVID-19 is front of mind for everybody. Many voices in webinar the industry — including IQVIA — are releasing their own Aired, May 28, 2020 reports, updates, and estimates for what COVID-19 means for our businesses and clients. In this webinar, we will bring View now for free! many of these dynamics together and lay the groundwork www.pharmexec.com/pe_p/ patient_engagement for pharma’s response to these uncertain times.

THREE KEY TAKEAWAYS:

Luke Greenwalt • The impact of shelter-in-place protocols on Vice President, US Market Access pharmaceutical demand and the underlying drivers Center of Excellence, IQVIA • Shifting channel dynamics and the impact on MODERATOR: manufacturer margin Lisa Henderson • Geographic variations for impact and recovery Editorial Director, Pharmaceutical Executive

Presented by:

Sponsored by:

Adaptive Leadership Through the Pandemic THIS MONTH IS OUR ANNUAL PHARMA 50 ISSUE, where we list, with our data partner EvaluatePharma, the top 50 companies according to drug sales for the past year. This is a big deal and, generally, is the all-time, most-read article on our website each year. Pharmaceutical company success by brand revenue is a milestone, LISA HENDERSON don’t get me wrong, and this year it will still garner massive amounts of attention. Editor-in-Chief [email protected] But it’s June. 2020. As we creep out of the pandemic. So this month, we also address our industry in regard to agile leadership and how the COVID-19 crisis should be Follow Lisa on Twitter: influencing the ways executives lead their companies and the industry now—and on @trialsonline the other side.

he last time I heard Lieutenant General (Ret) Rick new eye toward changing the balance of work-from-home and Lynch, US Army, speak was at an “Adapt or Die” office schedules or reducing their commercial real estate footprint. meeting held by Bob Jansen, CEO of Zensights and They are doing exactly what Lynch suggests: visualize the future, a Pharmaceutical Executive Editorial Advisory Board what it looks like, and how you get there. Tmember, over two years ago (see http://www.pharmexec.com/ The final stage is preparation. Once the crisis has passed and pharmas-image-fix-lessons-field). the smoke has cleared, said Lynch, you have to prepare to preclude At these meetings, Jansen brings together pharma and health- it from happening again. “Capture your lessons learned and put care executives to learn what leadership looks like from inside them into practice. What worked, what didn’t work,” he advised. those industries, as well as outside from Lynch’s formidable mil- “Because it will happen again.” itary experience and masterful Part of Lynch and Jansen’s viewpoint. I had the opportu- mission, established when they nity to listen again to Lynch in first met, was figuring out how late May, when Jansen brought Capture your lessons learned pharma could repair its sagging together a large group—virtu- reputation and public percep- ally, of course—to discuss lead- and put them into practice. tion, much like the military ership through the COVID-19 Because it will happen again. achieved post-Vietnam. The health crisis. answer was adaptive leadership. Lynch began with the current Right now, many pharma lead- state of affairs in our lives…it’s ers sense this is a time that could volatile, uncertain, and ambiguous. The task for agile and adap- change the public’s negative image of “big pharma.” See our tive leaders is to respond appropriately in three manageable phases. Senior Editor Elaine Quilici’s article on page 8, where she touches The first phase is survival, while sustaining some level of forward on the emerging sentiments around just that. Our Finance Col- movement; the second is recovery; and the third, preparation. umnist Barbara Ryan also addresses the reputation issue on page For the first phase, Lynch stressed to the leaders to look for the 11, asking pharma and biotech leaders to take the reins. opportunities and not the obstacles. “Master the technology. We Lynch said, “There is a great opportunity to be the heroes of all are on Zoom now. Understand the technology and take steps the world, with the right therapy, the right vaccine. But everyone to use it better,” he said. Lynch noted that the crisis is also an oppor- is looking at you with suspect. Take advantage of this moment, tunity to learn about other views (read a book, suggests Lynch) or but you must do it with integrity and transparency.” take time to think, since you aren’t going from meeting to meeting. He noted that the public is closely watching how pharma invests And, most of all, spend this found time with your families. in its resources. Is it appropriate or not? Are companies saying one Recovery is the next stage, and one that many have started. thing, but doing another? Are you not only doing the right things, Some CEOs present at a recent roundtable that Pharmaceutical Exec- but doing things right? utive hosted on behalf of our parent company, MJH Life Sciences Now is the time to check-in and ensure your vision, leadership, (https://bit.ly/3guzL0Z), are looking at their operations with a and moral compass are aligned. 4 4 Executive Profile INSIDE THE ISSUE News & Analysis Washington Report FDA’s Emergency 06 Drug Use Process Under Scrutiny Jill Wechsler, Washington chakisatelier - stock.adobe.com Correspondent Cover Feature Global Report The 2020 The Specter of 14 Pharm Exec 50 07 Compulsory Licensing Haunts Vaccine Debate Michael Christel, Managing Editor Reflector, Brussels Correspondent Pharm Exec’s 20th annual listing of the top global biopharma companies by Rx sales highlights brand surges, new ranking battles, Strategy & Tactics and M&A-sparked upswings, but in the shadow of COVID-19, a peek behind the Leadership numbers tells a different story. How to Prepare for Delivering Hope 08 Times of Crisis 18 in a Pandemic Elaine Quilici, Senior Editor Elaine Quilici, Senior Editor Pharma is boosting outreach and its image, Technology as the industry faces a defining period in its quest to sway the tide of public perception. Eyeing Quantum Leaps Executive Profile 10 for Drug Discovery Market Access Julian Upton, European and Online Rare Insights: Editor Unlocking Clues Launch Strategies 28 26 for 2020 and Beyond Finance to Hereditary Diseases By Bart Lombardi Will Biopharma Leaders Julian Upton, European and Online Editor 11 Please Take the Stage! Pharm Exec speaks with Arndt Rolfs, CEO Insights By Barbara Ryan and founder of CENTOGENE, who discusses his company’s rise from a small From the Editor Strategy genetic diagnostics startup to a global Adaptive Leadership organization focused on advancing the Better Social-Value understanding of the pathophysiology of Through the Pandemic 03 12 Sharing with Investors rare hereditary diseases. Lisa Henderson, Editor-in-Chief By Sandor Schoichet Patient Engagement Brand Insights Digital Engagement with COVID-19: Meeting Keep in Touch! Physicians & Other HCPs Scan here with your smartphone to sign up 32 the Needs of Patients 09 for weekly newsletters with Chronic Conditions By Robert Adler By Rebecca Cotton Back Page Pharma companies are in a unique position to support and educate at-risk populations How to Thank Frontline through patient services hubs. 34 Doctors Post-COVID-19 By Daniel S. Fitzgerald

PHARMACEUTICAL EXECUTIVE VOLUME 40, NUMBER 6 (Print ISSN 0279-6570, Digital ISSN: 2150-735X) is published monthly by MultiMedia Healthcare LLC, 2 Clarke Dr. Suite 100 Cranbury, NJ 08512. Subscription rates: $73.50 (1 year), $131.25 (2 years) in the United States and Possessions; $94.50 (1 year), $152.25 (2 years) in Canada and Mexico; $141.75 (1 year), $261.45 (2 years) in all other countries. Price includes air-expedited service. Periodicals postage paid at Cranbury, NJ and additional mailing offices. POSTMASTER: Please send address changes to PHARMACEUTICAL EXECUTIVE, P.O. Box 457, Cranbury, NJ 08512-0457. Canadian G.S.T. Number: r-12421 3133rt001, Publications mail agreements NO. 40612608. Return Undeliverable Canadian Addresses to: IMEX Global Solutions, P. O. Box 25542, London, ON N6C 6B2, Canada. Printed in the USA. VOLUME 40, NUMBER 6 5 5

JUNE 2020 PHARMACEUTICAL EXECUTIVE

Pharm Exec Webcasts Pharm Exec Podcasts Twitter Talk

Using Big Data to Build Excellent Commercial Planning for Pharma’s New Normal As of November 27, 2020, Pratap Khedkar, managing principal of ZS Forecasts #pharmaceutical dispensers will no Associates, talks about pharma’s scenario planning bit.ly/2SovtxK amid the COVID-19 pandemic. longer be allowed to receive drugs into bit.ly/3chkPQA Powering HCP Promotional Precision and inventory that are not serialized with a 2D ROI Using AI & Predictive Analytics: How to Killing Cancer Through Direct Injection #barcode. #DSCSA deadline. Use AI & ML with Patient-level Data Lew Bender, founder of Intensity Therapeutics, GS1 US, @GS1_US discusses the technology that allows the company’s “Key Regulatory Deadlines Loom for Drug bit.ly/3dgyMj2 treatments to be injected directly into solid tumors. Dispensers and Wholesalers” bit.ly/3chC1pd bit.ly/2XlqWPT

Pharmaceutical Executive’s 2020 Editorial Advisory Board is a distinguished group of thought leaders with expertise in various facets of pharmaceutical research, business, strategy, and marketing. EAB members suggest feature subjects relevant to the industry, review article manuscripts, participate in and help sponsor events, and answer questions from staff as they arise.

MURRAY L. AITKEN JAMES J. GALEOTA, JR. (JAY) BOB JANSEN BARBARA RYAN Senior Vice President, IQVIA, President and CEO, Inheris Principal Partner, Zensights LLC Founder, Barbara Ryan Advisors Executive Director, IQVIA Institute for Biopharma Human Data Science KENNETH KAITIN SANJIV SHARMA STEVE GIRLING Director & Professor, Center for the Chief Commercial Officer, HLS INDRANIL BAGCHI, PhD President, IPSOS Healthcare North Study of Drug Development, Tufts Therapeutics Senior Vice President and Head, America University Global Value Access, Novartis TERESE WALDRON ADELE GULFO CARRIE LIASKOS Director, Executive MBA Programs, FREDERIC BOUCHESEICHE Chief of Commercial Development, Vice President, Market Engagement, St. Joseph’s University Chief Operating Officer, Focus ROIVANT Sciences Syneos Health Reports Ltd. PETER YOUNG NICOLE HEBBERT CHANDRA RAMANATHAN President, Young & Partners LES FUNTLEYDER Senior Vice President, Head of Patient Vice President and Head, East Coast Portfolio Manager, Esquared Asset Services, UBC Innovation Center, Bayer US Management MICHELE HOLCOMB AL REICHEG JOHN FUREY Head, Strategy & Corporate CEO, Sea Change Healthcare CEO, Imvax, Inc. Development, Cardinal Health

GROUP PUBLISHER TEL [732] 346.3002 MJH LIFE SCIENCES TM Todd Baker [email protected] EDITOR-IN-CHIEF TEL [732] 346.3080 Chairman and Founder Mike Hennessy Sr Lisa Henderson [email protected] President and CEO Mike Hennessy Jr MANAGING EDITOR TEL [732] 346.3022 Michael Christel [email protected] Vice Chairman Jack Lepping EUROPEAN & ONLINE EDITOR TEL 011 44 [208] 956.2660 Julian Upton [email protected] Chief Financial Officer Neil Glasser, CPA/CFE SENIOR EDITOR TEL [609] 250.4651 Executive Vice President, Operations Tom Tolvé Elaine Quilici [email protected] Executive Vice President, Global Medical Affairs & Joe Petroziello ASSISTANT EDITOR TEL [732] 346.3025 Miranda Schmalfuhs [email protected] Corporate Development ASSISTANT EDITOR TEL [732] 346.3079 Senior Vice President, Content Silas Inman Andrew Studna [email protected] Senior Vice President, I.T. & Enterprise Systems John Moricone EDITORIAL OFFICES TEL [732] 596.0276 485 Route 1 South, Building F, Suite 210 FAX [732] 647.1235 Senior Vice President, Audience Generation & Product Fulfillment Joy Puzzo Iselin, NJ 08830 www.pharmexec.com SENIOR ART DIRECTOR TEL [609] 716.7777 Vice President, Human Resources & Administration Shari Lundenberg Marie Maresco [email protected] Vice President, Business Intelligence Chris Hennessy GRAPHIC DESIGNER TEL [609] 716.7777 Maya Hariharan [email protected] Executive Creative Director, Creative Services Jeff Brown WASHINGTON CORRESPONDENT Jill Wechsler [email protected] ©2020 MultiMedia Pharma Sciences LLC All rights reserved. No part of this publication may be reproduced or SENIOR DIRECTOR, DIGITAL MEDIA TEL [732] 346.3028 transmitted in any form or by any means, electronic or mechanical including by photocopy, recording, or information Michael Kushner [email protected] storage and retrieval, without permission in writing from the publisher. Authorization to photocopy items for internal/ MANAGING EDITOR, SPECIAL PROJECTS TEL [732] 346.3033 educational or personal use, or the internal/educational or personal use of specific clients is granted by MultiMedia Kaylynn Chiarello-Ebner [email protected] Pharma Sciences LLC for libraries and other users registered with the Copyright Clearance Center, 222 Rosewood Dr. DIGITAL PRODUCTION MANAGER TEL [732] 346.3081 Danvers, MA 01923, 978-750-8400 fax 978-646-8700 or visit http://www.copyright.com online. Periodicals Postage Sabina Advani [email protected] paid in Trenton NJ 08650 and at additional mailing offices. For uses beyond those listed above, please direct your PROJECT MANAGER, DIGITAL MEDIA TEL [732] 346.3021 written request to Permission Dept. Alexa Rockenstein, [email protected]. Vania Oliveira [email protected] MultiMedia Pharma Sciences LLC provides certain customer contact data (such as customers’ names, addresses, SALES MANAGER–MIDWEST, SOUTHWEST, WEST COAST TEL [609] 874.8311 Margie Rogers [email protected] phone numbers, and e-mail addresses) to third parties who wish to promote relevant products, services, and other opportunities that may be of interest to you. If you do not want MultiMedia Pharma Sciences LLC to make your contact SALES DIRECTOR TEL +44 (0) 7852.142.284 information available to third parties for marketing purposes, simply email [email protected] and a customer Wayne Blow [email protected] service representative will assist you in removing your name from MultiMedia Pharma Sciences LLC lists. REGIONAL SALES DIRECTOR TEL [609] 819.5209 Vahé Akay [email protected] Pharmaceutical Executive does not verify any claims or other information appearing in any of the advertisements VP/MANAGING DIRECTOR, PHARM/SCIENCE GROUP TEL [732] 346.3012 contained in the publication, and cannot take responsibility for any losses or other damages incurred by readers in Dave Esola [email protected] reliance of such content. AUDIENCE DEVELOPMENT MANAGER TEL [201] 391.2359 Pharmaceutical Executive welcomes unsolicited articles, manuscripts, photographs, illustrations, and other Christine Shappell [email protected] materials, but cannot be held responsible for their safekeeping or return. C.A.S.T. DATA AND LIST INFORMATION TEL [218] 740.6431 Melissa Stillwell [email protected] To subscribe, email [email protected]

REPRINTS For reprint requests, contact Todd Baker at +1 (732) 346-3002, [email protected] WWW.PHARMEXEC.COM 6 6 Washington Report PHARMACEUTICAL EXECUTIVE JUNE 2020

FDA’s Emergency Use Process

JILL WECHSLER Under Scrutiny is Pharmaceutical Executive’s Washington Correspondent. She can be reached at As authorizations skyrocket, review standards face questions [email protected]

nder pressure to expand public access to new drugs, Stockpile (SNS) but only for distribution to health professionals diagnostic tests, and other medical products needed treating hospitalized patients with confirmed COVID-19. to detect and treat patients struck by COVID-19, Despite such limitations, FDA continued to draw criticism for FDA has issued more than 100 Emergency Use acceding to political pressure from the White House to enable UAuthorizations (EUAs) since early February, compared to less than broad use of these drugs. But in an interview posted on STAT 75 over the 10 previous years. While this activity reflects the imper- April 24, Center for Drug Evaluation and Research (CDER) Direc- ative for fast action by federal agencies and manufacturers to tor Janet Woodcock explained that permitting access to some three address the pandemic, it also generates questions about the vetting million pills donated by Bayer and others augmented short supplies of these requests and how well FDA can follow up with sufficient that undermined approved treatment for lupus and rheumatoid tracking of the safety and efficacy of these products. And some arthritis, and that FDA tested the donated drug, which came from observers fear that political interference in the process may erode uninspected manufacturing facilities in India, extensively for qual- confidence in the scientific basis for FDA regulatory decisions. ity. Woodcock maintained that the EUA does not undermine FDA FDA may issue an EUA to use an unapproved drug or medical review standards, and that the agency clearly stated such emer- product, or an approved drug for additional uses, in an emergency gency use does not represent approval of the drug for a new use. when there are no adequate, approved, and available alternatives. Woodcock is supported by subsequent revelations about the Most of this year’s EUAs are for diagnostics and medical products political infighting leading up to this EUA. The lengthy whistle- needed to augment national resources for testing individuals for blower complaint filed May 5 by Rick Bright, former director of contamination. Some EUAs facilitate use of innovative technology, HHS’s Biomedical Advanced Research and Development Author- as seen in recent authorizations for a new saliva-based COVID-19 ity (BARDA), details efforts by Bright and others to prevent wide- test that would permit wider at-home sample collection and for spread use of hydroxychloroquine as a supposed preventive and the first antigen test, which promises to be less costly and more cure for COVID-19. Bright recounts how HHS officials initially rapid in detecting the virus. Some EUAs involve new respirator pressured BARDA to establish a nationwide expanded access inves- cleaning systems and innovative protective personal equipment. tigational new drug (IND) protocol for chloroquine, which would Only two EUAs this year are for therapeutics. FDA issued one make the drug available outside the hospital setting and without on May 1 to Gilead Sciences to permit limited use of the investi- close physician supervision, but that Woodcock provided support gational drug remdesivir to treat very ill COVID-19 patients. Here for an EUA from FDA with restricted uses. the emergency authorization was approved after a clinical trial In response to these developments, Sens. Elizabeth Warren provided evidence of some effectiveness in highly critical situations. (D-Mass) and Patty Murray (D-Wash) seek to examine more closely As expected, the EUA limits the drug’s use to hospitalized patients FDA’s role in enabling rapid emergency use of drugs, biologicals, with severe COVID-19 disease, while clinical testing continues. and devices and the agency’s ability to track adverse events and More controversial is FDA’s approval of an EUA to permit the other outcomes related to such authorizations. The legislators sent use of certain widely available malaria drugs to treat COVID a letter to FDA Commissioner Stephen Hahn on May 6 request- patients, despite concerns from medical authorities about no data ing such information. supporting effectiveness against the coronavirus and the risk of Meanwhile, FDA faces challenges in continuing to evaluate hun- serious adverse effects on individuals with certain heart conditions. dreds of EUA requests. The agency website on EUAs notes that Even so, FDA issued an EUA for chloroquine and hydroxychloro- some 385 diagnostic test developers plan to submit EUA requests quine on March 28 to permit use of drugs in the Strategic National for tests to detect the coronavirus. WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Global Report 7 7

The Specter of Compulsory Licensing

REFLECTOR is Pharmaceutical Executive’s Haunts Vaccine Debate correspondent in Brussels Industry prospects at mercy of public discourse, policy shifts

aul Hudson’s observation in mid-May that you get the about the pharmaceutical industry, already caricatured by some vaccines that you pay for could hardly have come at a campaigners as readying itself for predatory exploitation of the worse time for a pharmaceutical industry struggling pandemic. to hold the line against patent rights erosion. At any Since then, determination to avoid the richest bidder winning Pother time, the Sanofi CEO’s reminder that those who fund the prize of the first vaccine has dominated public discourse. Euro- research and development are entitled to expect a return might pean Commission Vice President Margaritis Schinas told the Euro- have passed without much comment. It would have been seen pean Parliament just days later that “bold steps together are perhaps as no more than a statement of an unfortunate, uncom- needed” to deliver a vaccine that will be accessible and affordable fortable, but unavoidable fact of life. and equitable worldwide. That debate featured calls from across But not at this particular moment, with the chronic European the political spectrum for easy universal access, and for policy concerns about access to medicines inflamed by acute anxiety over changes to make that happen. The obstacle that IP might present who will get treated with an eventual vaccine against COVID- to such universal access has become a central element in the 19—and who will not. So Hudson was denounced left, right, and broader European discussions, with growing enthusiasm for com- center—quite literally. European accusations of high treason pulsory licensing across swathes of the population that until a few greeted the suggestion by the boss of a largely European company weeks ago were blissfully ignorant of the minutiae of the interna- that it would be the US that deserved first claim on a vaccine pro- tional trade agreements on patents. duced with a hefty dose of US money. Europeans are not only The most remarkable feature of this conversion to compulsory fearful of their own vulnerability in the face of coronavirus; they licensing has been a switch in the position of the center-right are also loud in their insistence that everyone in the whole world European People’s Party (EPP), the largest political group in the should benefit from a vaccine. Parliament. “It is legally possible to go for so-called forced or com- French President Emmanuel Macron, who has been at the pulsory licencing. This makes it possible for member states to use forefront of calls for universal access to any vaccine, summoned the recipe for the vaccine production without formal consent of the errant boss of what is France’s biggest hitter in the interna- the original patent holder,” announced the EPP’s principal health tional drug market. Hudson attempted to contextualize his con- mouthpiece, German physician Peter Liese, with the full backing troversial remarks by telling everyone who would listen,“We need of his group. Until now, the EPP has been a staunch defender of to get this vaccine to everybody,” promising not to stop “until the interests of the pharmaceutical industry—notably in attempt- everyone gets what they need around the world.” But his recan- ing (unsuccessfully) last year to resist weakening of EU protection tation came too late. The wheels of policy had already started to against generic products in Europe. The shift presents the Euro- grind away at intellectual property before he shot to pariah prom- pean drug industry with a new problem at a sensitive moment. inence, and his intervention did little more than cause them to The official position from the main European drug lobby, the spin a little faster. European Federation of Pharmaceutical Industries and Associa- What Hudson did do was to provide an easy target, as a bad tions (EFPIA), has been to endorse the principle of wide access to guy clearly identified and prominently displayed for all to see and a vaccine—but it has conspicuously avoided detailed discussion vilify, in a complex debate that was underway in Europe—and of how this should be achieved. Now, with compulsory licensing international bodies such as WHO and even the UN—over dis- firmly on the table, coupled with the defection of its main ally in tributing and paying for a vaccine. The debate had been driven EU policymaking, EFPIA may find it can no longer dodge some in large part by altruism or a sense of social responsibility and difficult questions about just how far it is ready to break the habit solidarity, but Hudson’s remarks unleashed a wave of resentment of a lifetime on the scale that COVID-19 will require. WWW.PHARMEXEC.COM 8 8 Leadership PHARMACEUTICAL EXECUTIVE JUNE 2020

Pivotal Moments: How Leaders Can Prepare

ELAINE QUILICI for the Unexpected is Pharm Exec’s Senior Editor. She can be reached at equilici@ Balancing bold and pragmatic approaches during times of crisis mjhlifesciences.com

lexibility, adaptability, and open-mindedness are all “Confirming an enterprise’s purpose and reminding every team important concepts when it comes to running a busi- member of the ‘why’ is essential to rallying people around what ness. You never know when a curve ball will catch you is needed to rise to this challenge,” says Krueger. “Leaders in this by surprise. But when that curve ball is big enough to industry will need purpose-led leadership as a core competency Fchange your entire game—say a global pandemic—these qualities in their success profile going forward, otherwise they’re not going become critical. Sure, leaders can draw on their experiences for to be able to drive success.” possible direction, but in unprecedented times, there is no proven Another way to be prepared for a crisis is to invest in continu- course of action to follow. There is no playbook to rely on. ing learning and education so that when the need arises, you will “Having an open mind and being agile and flexible are almost have a broader base of knowledge to leverage decisions. table stakes in situations like these,” says Dana M. Krueger, man- “Even if you don’t have an immediate application for an aging director and global healthcare sector leader at Russell insight, that continuous curiosity—whether it’s learning content Reynolds Associates. “But what makes a leader really stand out or improving your own leadership or whatever it is—if it’s part is being decisive and willing to take risks. That means not only of your repertoire, that will at some point in your career become are you open for input more broadly, you’re also keeping the lon- relevant and helpful,” says Krueger. ger term in mind, you’re grounding things in purpose, and you’re As much as leaders need to focus on business matters and willing to make a decision that might be wrong, but you can always values, there is another element that can’t be ignored during a change course as necessary.” crisis—people. Reaching out to individuals to understand their Though it may seem risky, bold decision-making can help cut needs and responding to those needs with empathy can provide through the ambiguity of novel situations. It’s easy to become a clearer picture on how to lead them. paralyzed by the amount of everchanging information coming Creating open lines of communication is paramount. While across your desk, but swift choices keep you moving. While there it’s common to hear about the need for frequent and clear com- is potential for mistakes when acting quickly, the key is to know munication during trying times, it’s not just about sending mes- when to pivot. It’s okay to try a different, riskier approach, as long sages. Leaders need to be open to receiving messages. A time of as you recognize where the new direction can lead you. crisis can be an opportunity to seek and incorporate feedback from “That really instills confidence in an organization, in a team, employees into the decision-making process. and energizes people around a common goal,” says Krueger. “Great leaders change their approach to communication in While being bold to address short-term priorities is important, times of crisis by making it more two-way than it might [be] in a you need to strike a balance with thinking toward the future. Even- typical scenario,” says Krueger. “If they engage earnestly with tually, the crisis will subside, and it’s what you’re prepared to do those around them, have the humility to ask questions, and listen at the other end that will carry you forward. This is a great time to a wide range of opinions, they often create not only a sense of to tap into your company’s core values and mission statement. togetherness, but they also often are able to envision and ultimately Weighing options against what is fundamental to the company rally the broader organization around what is the path forward.” can help guide decisions. Of course, emerging from a crisis is not a one-person job, and The pharma industry is unique in that it has a clear purpose: one of the most effective ways to prepare for the unexpected is to to help patients. Though that critical value driver has always been prepare others to be leaders. “The best leaders step up individu- there, many companies are now demonstrating that commitment, ally during turbulent times, but they also provide others the latitude not only by racing to develop treatments for COVID-19, but by to lead,” says Krueger. “In turbulent times, the strongest leader- donating time and goods to people on the frontlines (see story on ship often comes from those without senior titles. Don’t stand in page 18). the way, and let emerging stars shine.” WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Brand Insights - Pharma Marketing 9 9

Digital Engagement With Physicians and

ROBERT ADLER Brand Insights Contributor, Other Healthcare Founding Strategic Partner, CultHealth Professionals Brand Insights - Thought Leadership from Marketers I Paid Program Earning purposeful attention online

henever you hear “tried-and-true” used to Maintaining knowledge and knowing you can apply that describe a strategy, tactic, workshop, or any knowledge in diverse clinical scenarios to the broad range of other fundamental step required to achieve patient psychographic segments found in a typical practice market domination—ask the speaker what require a commitment to lifelong learning that grows and main- yearW it is. Case studies and experience have a place, but when tains clinical confidence. The need to update credentials and they’re used to justify outdated thinking, that’s not cool. One translate science into patient-centric actions changes weekly. area where spoiled milk is rebranded as cheese way too often is The half-life of information alone is measured in days and hours; online, non-personal engagement, and everyone should be both- from one patient encounter to another. ered by that because this channel is now as essential for success When a new product campaign or so-called disease-state edu- as any other. If you are not lapping your competition with share cation program is presented online, didactically, informationally, of attention and truly deep engagement online, you can forget as if a sales rep, or MSL, is pointing to bullets, the very strong about exceeding expectations and dominating your market. possibility exists that applied clinical confidence will be eroded Physicians and other healthcare professionals (HCPs) live in as often as it is enhanced. a world with many distractions. Some are urgent and important, Delivering against KPIs with declining HCP–rep encounters many are not. They all steal time from the essential task of con- has been a challenge to the industry model for information tinuing professional development. exchange. In a post-COVID-19 personal-selling environment, Hoping to provide a shortcut to behavior change, industry we may look upon access to 50% of HCPs with envy. Online offers content as information. Access to information is not formats, based on corollaries to adult learning theory that enough. Information is quick and easy to produce and consume; uniquely apply to HCPs, can change these access trends and and it mirrors traditional formats such as detail aids and reprint propel the rapid behavior and clinical belief changes that HCPs carriers. The problem is, it doesn’t change behavior very well are trained to form and assimilate into practice. because information is not knowledge. Industry can earn the purposeful online attention of HCPs HCPs change behavior quickly when knowledge, rather than by offering programs that recognize the following: information, is transferred in ways that provide clinical context. • Behaviors and beliefs are directly linked to confidence. Why? Because they “know that they know,” which raises clini- • The ability to apply knowledge consistently, flexibly, and in cal confidence, solidifying new therapeutic beliefs and behaviors. diverse situations raises confidence. HCPs learned how to learn via Socratic dialogue; at the foot of • Knowing needs rather than names makes communications patients’ beds, grand rounds, and morbidity and mortality con- “personal,” and non-personal selling needs to be very personal? ferences—challenging environments where belief systems are • Disease-state education is for professors, and innovation-in- tested in real time, and information without immediate applica- context education is for industry. tion context is irrelevant. The mantra, “see one, do one, teach • Mechanistic actions are not outcomes, and mechanistic out- one,” summarizes the expectation that the very next patient (not comes are what matter. the patient six months from now) deserves the best knowledge • Awareness, trial, and usage are not representative of true belief we have. systems. WWW.PHARMEXEC.COM 10 10 Technology PHARMACEUTICAL EXECUTIVE JUNE 2020

Accelerating Drug Discovery with

Supercomputers JULIAN UPTON is Pharm Exec’s European and Their use in COVID-19 fight paves way for future leaps Online Editor. He can be reached at [email protected]

rtificial intelligence and computing companies THE QUANTUM EQUATION around the world are currently applying their pow- If this drug discovery model currently seems not just beyond the erful technology to help scientists better understand capabilities of the individual, but also beyond that of many pharma and, hopefully, conquer COVID-19. Among them, companies, Laino reminds us that all the individual components IBMA Research announced in April that it was making multiple mentioned “already exist in pharma R&D.” It is IBM’s integration resources available free to researchers, including a cloud-based of these technologies that serves to accelerate their power. Laino AI research resource that has been trained on a corpus of more says that the quantum computing component will have an impact than 45,000 scientific papers contained in the COVID-19 Open within this decade. “We will use quantum computing as the systems Research Dataset (CORD-19) and the IBM Functional Genom- increase in their capabilities,” he explains, “for example, in their ics Platform, a cloud-based repository and research tool built to application to the calculation of properties of possible targets.” He discover the molecular features in viral and bacterial genomes. adds, “We are also exploring the possibility of using quantum for IBM Research has also played a leading role (in collaboration some algorithms for designing retrosynthesis. This is a very com- with the White House Office of Science and Technology Policy, plex problem; you have to explore a very complex graph of chem- the US Department of Energy, and others) in launching the US ical reactions, and that’s where quantum technologies can actually COVID-19 High Performance Computing Consortium, which make an incredible impact compared with classical computing.” is making available to scientists more than 38 US-based super- While, for some, quantum computing may conjure up thoughts computers with “more than 400 petaflops of computing power.” of science fiction, Laino emphasizes that “the quantum computer is actually closer than people think.” He says, “You can easily have BEYOND HUMAN ENDEAVOR access to a quantum computer in 2020; in fact, it’s been possible “High-performance computing systems allow researchers to run to access a quantum computer since May 2016. The only constraint very large numbers of calculations in epidemiology, bioinformat- is that they work slightly differently to a traditional computer. Map- ics, and molecular modeling, experiments that would take years to ping the quantum architecture to the problem that you have to complete if worked by hand, or months if handled on slower, tra- solve is part of the equation.” IBM’s largest quantum computing ditional computing platforms,” wrote Dario Gil, director of IBM installation is a 53-qubit system on which “you can run molecules Research, on the IBM Research blog (April 3). Dr. Teodoro Laino, of a decent size,” says Laino, “but this isn’t yet comparable with manager of accelerated discovery, IBM Research Europe, explained what you can do on a high-performance computing installation.” IBM’s work in this area further. “Accelerating drug discovery does The situation is changing quickly, however. Laino believes it’s not come from the fact that you can extract data—you need to just a matter of years before “we will really see the gain from using complement the data with physical and mathematical modeling, quantum computers.” There are currently 18 quantum comput- using high-performance computing, quantum, and AI,” he told ers in existence; IBM is installing another one in Germany at the Pharm Exec. “Once the data extracted from documents has been end of this year, followed by one in Japan. As part of the IBM Q augmented and completed, these data can be used to create gen- Network, 110 companies can currently access these systems. erative models, which can generate hypotheses. Following that is As well as being hopeful that the release of its resources will help validation and testing.” IBM Research is now driving chemical labs to speed up the global effort against COVID-19, IBM’s Dario Gil completely on a system with no human intervention. Says Laino, wrote in April that the move is another step toward an age “where “We’re relieving people from tedious programming tasks by having multi-disciplinary scientists and clinicians work together to rapidly AI self-program the automation hardware for testing. It is a self- and effectively create next-generation therapeutics, aided by novel sustained, completely closed loop of accelerated drug discovery.” AI-powered technologies.” WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Finance 11 11

Will the Biopharma Leaders Please

BARBARA RYAN is Founder, Barbara Ryan Take the Stage! Advisors, and a member of Pharm Exec’s Editorial Now is the time, the world is listening Advisory Board

ver the past month, and year to date, biotech stocks of the 24/7 news world we live in. The outrageous price of a pill have massively outperformed the markets, financ- is an easy campaign slogan sure to curry favor with voters. ings are on a tear, money continues to pour into Today, people the world over are confused, they are scared, the space, and M&A is alive and well. The Nasdaq our ecosystem was shamefully ill-prepared for the pandemic and OBiotech ETF is currently trading above its late 2019 and early even worse, people are getting conflicting messages and guidance 2020 highs. In theory, the group has more room to run. and the entire crisis has spiraled into a political food fight. All eyes are on biopharma for the therapeutics and vaccines DON’T wear a mask, DO wear a mask, stay indoors, go out- key to ending the COVID-19 side, the list goes on and on. pandemic, saving lives, and What we need now are reopening our economy. What we need now are informed informed leaders with deep According to a running tally knowledge and understanding of by Fundstrat, there are currently leaders with deep knowledge the science behind this pan- 10 vaccines in clinical develop- demic; that would be the leaders ment and 114 in preclinical eval- and understanding of the of our very own biopharma uation. In addition, there are industry. likely 100 therapeutics in devel- science behind this pandemic We are all in this together, so opment as the industry rapidly I challenge our leaders to com- pivots programs to redirect their municate, now, about important efforts to fighting the coronavirus. All of this is great news and information that can save people’s lives, put their hearts and minds gives much hope to a world literally stopped in its tracks, riddled at ease, and be the voice of reason in the midst of this storm. with fear and confusion. Please initiate the dialogue on: News of progress across these efforts, including most recently » What we know. Moderna’s vaccine, are literally driving the markets as they dic- » What we don’t know. tate the terms of the economy. The world is rooting for our prog- » What does it take to develop therapeutics and vaccines? ress with all it has. This is great news. » What are appropriate best practices people should be Over my 40-year career in this space, it has always saddened following? me that the reputation of an industry that does so much good The biopharma enterprise has never had a greater opportu- through saving lives is so poor. The fact is, ours is a very complex nity to put its best foot forward and show the world how hard it industry, is not well understood, and our profitability is usually is to be successful in our industry—but how powerful it is when under attack. The innovations at Apple and the commensurate we are. profits are heralded, yet the successes of biopharma are often Let’s leave this pandemic with a much better reputation than viewed as shameful. It is well-accepted that successful athletes, when we went into it. actors, and companies should be handsomely rewarded. However, Take it upon yourselves to be the light to lead us out of the when a drug company is successful, its profits are often publicly darkness. shamed and the company, or industry, accused of mercenary greed. In the words of Margaret Mead, “Never doubt that a small In all candor, our leaders have done a poor job telling our group of thoughtful, concerned citizens can change the world. story; it is not easily condensed into a sound bite, the vernacular Indeed it is the only thing that ever has.” WWW.PHARMEXEC.COM 12 12 Sustainability PHARMACEUTICAL EXECUTIVE JUNE 2020

Better ESG Communication

SANDOR SCHOICHET with Investors Director, Meridian Management Consultants, and Co-Founder Guidance addresses struggles in reporting non-financial performance of the Biopharma Sustainability Roundtable. He can be reached at [email protected]

etter communication between biopharma companies Last month, I introduced the 12 highest priority ESG topics and investors is vital. Our industry is not only about jointly identified by the biopharma and investor participants. Now making money; it’s about reducing suffering and let’s look at how the guidance is structured, and how it’s being improving the quality of life. However, unlike financial used to improve biopharma-investor communications. Bperformance reporting, which has had hundreds of years for gen- What makes the guidance different from prior efforts? Focus erally accepted accounting principles (GAAP) to mature, perfor- on the biopharma sector, a collaborative development process mance tracking and disclosure for environmental, social, and gov- that included both companies and investors, emphasis on best ernance (ESG) topics is still evolving. Today’s ESG reporting practices rather than normative demands, and building on the landscape is defined by complex and sometimes contradictory foundation of prior work by SASB (the Sustainability Accounting disclosure demands that are expensive and time-consuming to Standards Board) and TCFD (the Task Force on Climate-related address, yet still incomplete in meeting the needs of key stakehold- Financial Disclosures). The result is a shared model that helps ers, especially investors. An emerging industry of third-party anal- biopharma companies tell their own sustainability stories in a ysis, rankings, and ratings seeks to fill the gap, but is based mostly way that connects ESG performance to long-term strategy and on proprietary algorithms that produce non-comparable results. value-creation while improving comparability across the sector. The lack of an effective and efficient model for communicat- The guidance structures ESG disclosures around five core ele- ing non-financial performance information between biopharma ments, using a two-tiered approach. The elements include Gover- companies and investors was repeatedly identified as a major frus- nance, Strategy and Objectives, Risks, Opportunities, and Metrics tration by both groups in discussions held by the Biopharma Sus- and Performance. The first tier discusses how sustainability is man- tainability Roundtable (BSRT) over the past few years. The BSRT aged strategically at the corporate level, while the second takes the is a sector-focused network of senior sustainability executives that discussion to a deeper level, addressing the same core elements in I founded eight years ago and co-lead, along with my colleagues relation to each high-priority topic. This reflects the fact that both Thomas Scheiwiller, Myrto Kontaxi, and Maggie Kohn. strategy and robust topical information are important to investors. We launched the Biopharma Investor ESG Communications Biopharma companies have started using the guidance as a Initiative in mid-2018 to help address this frustration. The Initia- tool for engaging sustainability and investor relations functions tive enabled a direct dialog between biopharma sustainability and in reviewing their existing ESG communications for completeness investor relations executives on the one hand, and their investment and focus, ensuring that the information investors need is easily counterparts on the other, the biopharma sector and ESG analysts available. Some companies have already found the guidance help- from major investors. The resulting Biopharma Investor ESG Commu- ful in coordinating their COVID-19-related communications. nications Guidance was published at the end of April (read the release Investors are finding the guidance to be a valuable for struc- at https://bit.ly/3cwjXaY; contact me to request a free copy). turing stewardship engagements with portfolio companies, espe- The guidance was developed by a working group sponsored by cially companies only starting to define a sustainability strategy. Amgen, Bristol Myers Squibb, Merck & Co. (MSD outside the For larger organizations, it’s critical that investors see that sus- US and Canada), with active participation by Johnson & Johnson tainability is part of a coherent strategic framework with Board- and Novartis. The High Meadows Institute, a think tank and pol- level oversight, not buried in functional silos. icy institute focused on strengthening the role of business leader- It’s on all of us not only to step up our efforts to address social ship in creating a sustainable society, joined as a strategic partner, challenges in medicine and healthcare, but also to communicate our bringing key investors into the conversation. efforts and outcomes more effectively to investors and society. For nearly two decades, MGM Resorts has led the industry in Planet” also reaffirms our commitments to our guests, employees social impact investments that benefit employees, shareholders and and partners; to the communities we call home; and to the planet we communities. We have done so because we have long believed that must protect for future generations. Now, more than ever, is the time modern corporations have the responsibility to affect the world’s to focus on these goals. most pressing economic, environmental and social issues. Believing that greener business is better business is why MGM “Focused on What Matters: Embracing Humanity and Protecting Resorts is a leader in sustainable meetings and events. From earning the Planet” asserts our values as a Company and our commitment LEED certifications to holding the prestigious 5 Keys award at five as a community partner. It defines our environmental sustainability properties, MGM Resorts is constantly working to improve on con- and social impact strategy in four critical areas: Fostering Diversity serving natural resources. and Inclusion, Investing in Community, Caring for One Another and The 28-acre solar array that sits atop the Mandalay Bay Con- Protecting the Planet. In each of these areas, we have adopted goals vention Center supplies 25% of the power demand for the entire against which we will chart our progress, from today to 2025 and campus which includes Mandalay Bay, Delano Las Vegas and Four beyond. In 2018, we joined the world’s leading Seasons, and their dedicated recycling facility sorts 60 tons of trash a companies and aligned our social impact day, recycling 81% of convention waste on average. and sustainability priorities to the Through these, and other efforts, MGM Resorts’ meetings and United Nations Sustainable De- conventions programs reduce the consumption of the planet’s limited velopment Goals (UNSDGs), resources through innovation, education and efficiency. selecting 10 UNSDGs For MGM Resorts, a rigorous approach to acting responsibly is which range from striving in our DNA; it is a driver of strategy and a mechanism of lasting to reduce poverty and change. We are immensely proud of what we have achieved to boost hunger to promoting the quality of life in our communities, reduce our environmental gender equality and footprint and provide a safe and healthy work environment where stimulating economic employees can develop and succeed. Our intent: to lead with a bold growth. “Focused vision, ambitious goals and expanded, data-driven methods to mea- on What Matters: sure our progress. Embracing Humanity We’re always better together. Visit mgmresorts.com to plan the and Protecting the meetings and special events that impact lives for the better. WWW.PHARMEXEC.COM 14 14 Pharma 50 PHARMACEUTICAL EXECUTIVE JUNE 2020

2020 PHARM EXEC50

Brand surges, new ranking battles, and M&A-sparked upswings highlight Pharm Exec’s annual listing of the top global biopharma players, but in the shadow of COVID-19, a peek behind the numbers also tells another story, one that reinforces the industry’s wider mission

By Michael Christel

ith much of the world’s attention today naturally to generate outputs and sustain investments that ultimately helps focused on the COVID-19 global crisis—and turn new science and promising innovation into approved prod- for governments, the healthcare and medical ucts or important treatment modifications. Pfizer’s announcement communities, and others, fixed squarely on crit- early this month to commit $500 million to a series of clinical-stage icalW response, resource, and research efforts to end the pandemic— biotechs—and allow access to Pfizer resources in research, clinical it may seem an odd juxtaposition to highlight our annual listing development, and manufacturing—is one such example. of the top 50 biopharma sales producers. Or could it be that this backdrop actually offers very real con- RAISING THEIR GAME text or perspective to this year’s ledger, our 20th? Perhaps never Shaped from the most recent full-year drug revenue tallies, COVID more apparent than today is the scope and force that has long been has little influence financially in our latest Pharma 50 rankings. required to fight not just diseases like cancer or crippling chronic The top 10 positions, however, did experience their biggest shake- conditions, but unforeseen viral scourges such as COVID-19; up in several years, impacted in part by a pair of mega mergers. Ebola, Swine flu, and HIV/AIDS before; and the next pandemic Roche, with an increase of 8.3% in Rx sales, moved up one spot to come. After all, many of the companies that made our list are to end Pfizer’s four-year reign atop the leaderboard. Avastin, on the front lines in combating the present crisis through avenues accounting for 15% of the company’s total pharmaceutical reve- such as treatment, outreach, education, and research (see story on nue, remains Roche’s top seller, but like Herceptin and Rituxan, page 18)—and will likely be for the next one. Some have launched faces biosimilar competition in the years ahead. As reinforcements their own initiatives, many are working side by side (e.g., the emerge, key sales drivers for Roche include multiple sclerosis drug COVID-19 Therapeutics Accelerator consortium, composed of Ocrevus, hemophilia medicine Hemlibra, and cancer treatments 12 organizations in our Pharma 50), and others are partnering Tecentriq and Perjeta. In a rarity for the Pharma 50 in recent his- with government (e.g., Operation Warp Speed, the US Depart- tory, the same company swept first place in drug revenue and R&D ment of Health and Human Services’ pact with AstraZeneca to spend, with Roche totaling $10.3 billion in the latter, the only orga- expedite the development and manufacturing of a coronavirus nization to eclipse billion-dollar double-digits in R&D investment. vaccine developed by Oxford University). The data for the Pharma 50 listing was again provided in part- And as yearly prescription drug revenue and R&D spending nership with life sciences market intelligence firm Evaluate Ltd (see totals illustrate, it is the capability of these companies, collectively, the explainer on how the listings were calculated on facing page). WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Pharma 50 15

Novartis also inched up a spot from our previous rankings, numbers for its nerve pain drug Lyrica, which has several com- jumping from third to second behind a 6% rise in drug sales. In peting generic versions. Sales of breast cancer treatment Ibrance January, the big pharma completed its $9.7 billion acquisition of rose 20.5% last year and Pfizer, which completed its $11.4 billion The Medicines Company, adding inclisiran, a potential first-in- acquisition of cancer-focused Array BioPharma in July 2019, class cholesterol-lowering therapy (works by harnessing the body’s anticipates continued growth from other brands like Eliquis, Xel- natural mechanisms for RNA silencing). Novartis’ heart failure janz, Xtandi, and Inlyta, and from recent and expected launches drug Entresto grew 74% in the fourth quarter of 2019 to $1.7 such as Vyndaqel/Vyndamax, Braftovi, Mektovi, and oncology billion for the year; it totaled $569 million in the first quarter of biosimilars. Merck Rx sales increased 9.5%, spurred by a spike 2020. And gene therapy Zolgensma, launched to much attention of 54.5%, to $11.1 billion, for cancer immunotherapy Keytruda, last June for spinal muscular atrophy, has steadily gained market as the drug continues to collect approvals for new indications. momentum, as payers haven’t shied away from backing the one- BMS zoomed from 11th to fifth, as our list factors in consensus dose treatment despite its hefty price tag. Rx sales estimates for Celgene, officially acquired for $74 billion Rounding out the top five Pharma 50 placeholders are Pfizer, in November after the deal was announced last January. Celgene’s Merck & Co., and Bristol Myers Squibb. Pfizer dropped two spots, multiple myeloma stalwart Revlimid leads the combined charge, as Rx sales decreased 3.6%, due in part to sinking fourth-quarter posting almost $11 billion in sales, while BMS’ Eliquis is next, with

2019 2019 2019 Rx Sales* R&D Spend* Top-selling Drugs* Avastin 7.118 1 Roche $48.247 $10.293 Rituxan 6.518 BASEL, SWITZERLAND [ROCHE.COM] Herceptin 6.078 Cosentyx 3.551 Novartis $46.085 $8.386 Gilenya 3.223 2 BASEL, SWITZERLAND [NOVARTIS.COM] Lucentis 2.086 Prevnar 13 5.847 Pfizer $43.662 $7.988 Ibrance 4.961 3 NEW YORK, NEW YORK [PFIZER.COM] Lyrica 3.321 Keytruda 11.084 Merck & Co. $40.903 $8.730 Gardasil 3.737 4 KENILWORTH, NEW JERSEY [MERCK.COM] Januvia 3.482 Revlimid 10.970 Bristol Myers Squibb** $40.689 $9.381 Eliquis 7.929 5 NEW YORK, NEW YORK [BMS.COM] Opdivo 7.204 Stelara 6.381 Johnson & Johnson $40.083 $8.834 Remicade 4.086 6 NEW BRUNSWICK, NEW JERSEY [JNJ.COM] Darzalex 2.998 Lantus 3.372 Sanofi $34.924 $6.071 Dupixent 2.322 7 PARIS, FRANCE [SANOFI.COM] Pentacel 2.178 Humira 19.169 AbbVie $32.351 $4.989 Imbruvica 3.830 8 NORTH CHICAGO, ILLINOIS [ABBVIE.COM] Mavyret 2.893 Triumeq 3.255 GlaxoSmithKline $31.288 $5.541 Shingrix 2.311 9 BRENTFORD, ENGLAND [GSK.COM] Advair 2.209 Entyvio 3.182 Ta ke d a $29.247 $4.432 Vyvanse 2.520 10 OSAKA, JAPAN [TAKEDA.COM] Gammagard Liquid 2.151

Source: EvaluatePharma® May 2020, Evaluate Ltd, www.evaluate.com *numbers USD in billions How the listings were compiled: 2019 Rx Sales and R&D Spend analyses were provided by life ® **2019 data for BMS is composed of its reported financial figures, plus Evaluate’sbroker-derived science market intelligence firm Evaluate Ltd via its EvaluatePharma service, www.evaluate.com. consensus estimates for Celgene’s WW Rx Sales and R&D Spend for January to November 20, 2019. Pharm Exec would like to thank EvaluatePharma for assisting in the development of this year’s Pharma 50 listing. PLEASE NOTE: 2019 figures represent prescription pharmaceutical sales from Specific values are as follows: the named company only, and exclude revenues from royalties, co-promotions, etc., as well as sales BMS 2019 WW Rx Sales: $25.174 billion; 2019 R&D Spend: $5.891 billion from non-prescription pharmaceuticals. Figures in green are Evaluate Consensus Estimates, and are included where actual reported sales data was not available at the time of generation. Evaluate’s Sales Celgene 2019 WW Rx Sales (estimated): $15.515 billion; 2019 R&D Spend (estimated): $3.490 billion and R&D Spend figures represent the fiscal year that ended in 2019. For most American and European companies, that means the year ending December 31, 2019. For many Japanese companies, the fiscal year ending March 31, 2020, was used. Historic averages were used in the conversion of companies’ native currency to USD. WWW.PHARMEXEC.COM 16 16 Pharma 50 PHARMACEUTICAL EXECUTIVE JUNE 2020

a 23% year-over-year increase. COVID-19 demand for the blood made perhaps before the full extent was understood. Nevertheless, thinner, which BMS manufactures in partnership with Pfizer, drove M&A uncertainty aside, it seems big pharma remains relatively up its revenue 37%, to $2.6 billion, in the first quarter of 2020. bullish revenue-wise for the rest of 2020, amid the coronavirus Together, BMS and Celgene spent an estimated $9.4 billion in (and with the US presidential election looming in November). R&D last year, ranking second in this metric, with Johnson & Evaluate Vantage notes that, as of mid-May, Merck was the only Johnson and Merck manning third and fourth, respectively. Earlier company that cut full-year guidance (by $1.7 billion). Along with this year, Brand Finance, the independent branded business valu- BMS, Novartis, GSK, and Lilly were among those reporting sig- ation consultancy, named J&J its 2020 most valuable pharma com- nificantly increased demand in 2020. pany, due to J&J’s strong R&D complement, notably for investment Outside the top 10, noteworthy observations for the rest of the in antimicrobials. J&J was one of the first to announce the devel- Pharma 50 field include Japan-based Astellas Pharma moving up opment of a vaccine for coronavirus during the outbreak in China. four spots to 19th, the additions of four other Asian companies to As anticipated, Takeda has muscled into the Pharma 50 top our list (Meiji Holdings, Japan, 27th; Yunnan Baiyao Group, 10 as well, moving from 16th to 10th behind the integration of China, 37th; Shanghai Pharmaceuticals Holding, China, 48th; Shire. The Japanese drugmaker’s Rx revenue rose 67.8% in its Aurobindo Pharma, India, 50th), and Regeneron Pharmaceuti- first full year following the $62 billion acquisition. cals’ climb from 38th to 31st, behind an 18% surge in Rx sales While AbbVie (ranked eighth) finally closed its $62 billion pur- (the New York biotech also upped its R&D spend by 19%, ranking chase of Allergan (ranked 18th) last month, likely elevating AbbVie 17th in that metric). into the top-five mix in the coming year or two, COVID-19 has stalled the completion of several healthcare-related M&As struck RESEARCH MIX before the pandemic. Evaluate Vantage reports that more recent Not surprisingly, the COVID-19 pandemic—and resulting health- transactions, like Gilead’s acquisition of cancer biotech Forty care restrictions and state and country quarantine measures—has Seven in March, while likely not as threatened by the crisis, were caused significant disruption to new and ongoing clinical develop-

2019 2019 2019 Rx Sales* R&D Spend* Top-selling Drugs* Tagrisso 3.189 11 AstraZeneca $23.207 $5.320 Symbicort Turbuhaler 2.495 LONDON, ENGLAND [ASTRAZENECA.COM] Brilinta 1.581 Enbrel 5.226 12 Amgen $22.204 $4.027 Neulasta 3.221 THOUSAND OAKS, CALIFORNIA [AMGEN.COM] Prolia 2.672 Biktarvy 4.738 13 Gilead Sciences $21.703 $4.059 Genvoya 3.931 FOSTER CITY, CALIFORNIA [GILEAD.COM] Truvada 2.813 Trulicity 4.128 14 Eli Lilly $20.085 $5.595 Humalog 2.821 INDIANAPOLIS, INDIANA [LILLY.COM] Alimta 2.116 Xarelto 4.028 15 Bayer $18.610 $3.081 Eylea 2.792 LEVERKUSEN, GERMANY [BAYER.COM] Mirena 1.369 Victoza 3.289 16 Novo Nordisk $18.296 $2.132 NovoRapid 2.708 BAGSVÆRD, DENMARK [NOVONORDISK.COM] Ozempic 1.685 Jardiance 2.409 17 Boehringer Ingelheim $15.629 $3.038 Spiriva 2.304 INGELHEIM, GERMANY [BOEHRINGER-INGELHEIM.COM] Tradjenta 1.745 Botox 2.128 18 Allergan $15.153 $1.709 Botox Cosmetic 1.663 IRVINE, CALIFORNIA [ALLERGAN.COM] Juvederm Voluma 1.244 Xtandi 3.587 19 Astellas Pharma $11.444 $1.976 Myrbetriq 1.496 TOKYO, JAPAN [ASTELLAS.COM] Prograf 1.470 Tecfidera 4.433 20 Biogen $11.380 $2.281 Spinraza 2.097 CAMBRIDGE, MASSACHUSETTS [BIOGEN.COM] Tysabri 1.892

Source: EvaluatePharma® May 2020, Evaluate Ltd, www.evaluate.com *numbers USD in billions WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Pharma 50 17

ment studies for non-COVID-related diseases. Research for COVID-19 Pipeline COVID therapies and vaccines, meanwhile, has been fast and Market Status Prophylaxis Treatment Total furious, with many projects up against demanding timelines. Phase III clinical trial data, released early this month, for Gilead’s exper- FDA Approved 1 1 imental treatment, remdesivir, which has been used under FDA’s Phase III 1 16 17 emergency use authorization, did reveal efficacy. Results found the drug more effective in patients who received a five-day regi- Phase II 2 15 17 men than those who took remdesivir for twice as long or those on the standard of care. Full clinical data, measuring other critical Phase I 3 9 12 endpoints, has not been disclosed yet. Preclinical 25 45 70 There is research underway for more than 175 vaccine candi- dates across a wide range of technologies. Moderna Therapeutics’ Research Project 14 27 41 mRNA-1273 may be the highest-profile prospect at the moment, Source: EvaluatePharma® May 2020, Evaluate Ltd but big pharma, of course, is heavily involved in these activities, Unique products in development by life sciences companies/ on the development and manufacturing sides. Along with AZ and collaborators. Product in development as a prophylaxis and J&J, other notable vaccine projects have been launched by Merck, treatment are counted in both columns. Pfizer, Sanofi, and GSK. Regeneron is developing an “antibody cocktail” to treat coro- 200 US commercial and hospital labs in the coming weeks. See navirus, and Roche’s COVID-19 antibody test, which was given the sidebar on page 24 for a look at how the public is better grasp- emergency use authorization last month, may be live at more than ing biopharma’s drug development efforts during these times.

2019 2019 2019 Rx Sales* R&D Spend* Top-selling Drugs* Fluticasone Propionate; Salmeterol Xinafoate 592 21 Mylan Glatiramer Acetate 503 CANONSBURG, PENNSYLVANIA [MYLAN.COM] $11.200 $518 Levothyroxine sodium 333

Teva Pharmaceutical Copaxone 1.513 22 $11.090 $1.010 Bendeka 496 Industries Methylphenidate Hydrochloride 412 PETACH TIKVA, ISRAEL [TEVAPHARM.COM] Privigen 2.837 23 CSL $8.951 $857 Hizentra 1.252 MELBOURNE, AUSTRALIA [CSL.COM.AU] Kcentra 690 Savaysa 1.418 24 Daiichi Sankyo $7.942 $1.817 Nexium 734 TOKYO, JAPAN [DAIICHISANKYO.COM] Injectafer 477 Rebif 1.425 25 Merck KGaA $7.516 $1.865 Glucophage 981 DARMSTADT, GERMANY [MERCKGROUP.COM] Erbitux 975 Abilify MAINTENA 934 26 Otsuka Holdings $6.711 $1.882 Samsca 841 TOKYO, JAPAN [OTSUKA.COM] Rexulti 824 Vancomycin Hydrochloride 88 27 Meiji Holdings Remeron 82 TOKYO, JAPAN [MEIJI.COM] $6.353 $149 Piperacillin Sodium; Tazobactam Sodium 82 Xifaxan 550 1.452 28 Bausch Health Companies $5.295 $471 Ocuvite 313 LAVAL, CANADA [BAUSCHHEALTH.COM] Wellbutrin XL 269 Cimzia 1.917 29 UCB $5.102 $1.424 Vimpat 1.434 BRUSSELS, BELGIUM [UCB.COM] Keppra 664 Soliris 3.946 30 Alexion Pharmaceuticals $4.990 $783 Strensiq 593 CHESHIRE, CONNECTICUT [ALXN.COM] Ultomiris 339

Source: EvaluatePharma® May 2020, Evaluate Ltd, www.evaluate.com *USD in billions, lower numbers in millions WWW.PHARMEXEC.COM 18 18 Pharma 50 PHARMACEUTICAL EXECUTIVE JUNE 2020

Delivering Hope in a Pandemic Pharma boosts outreach and image amid COVID-19, as the industry faces a defining period in its quest to sway the tide of public perception

By Elaine Quilici

ast August, a Gallup Poll placed pharma at an all-time view of the industry than they did before the pandemic began. reputation low. Consumers ranked it 25th out of 25 Between April 11 and 13, that number increased to 40%, where industries, edging out the federal government for last it remained for Harris’ mid-May poll. place. It was the industry’s most abysmal finish since This upward trend seems natural due to pharma’s demonstrated Lthe annual poll began in 2001. With pricing issues and the opioid dedication to finding treatments and a vaccine (see sidebar on page epidemic plaguing the industry, many wondered what it would 24). But there’s something else that may be contributing to the take to turn things around. No one could have imagined it would uptick: The public is finally recognizing pharma has a heart. be a pandemic. About eight months later, The Harris Poll shed a ray of sun- WHAT’S IN A NAME? shine through the clouds of coronavirus when its research indi- “This is a trend that we have also seen in our continuous tracking cated pharma’s reputation was on the rise. Between March 21 of major pharma companies,” says Sven Klingemann, PhD, direc- and 23, the poll revealed 33% of Americans had a more positive tor at the RepTrak Company (formerly Reputation Institute), a data

2019 2019 2019 Rx Sales* R&D Spend* Top-selling Drugs* Eylea 4.644 31 Regeneron Pharmaceuticals $4.834 $2.607 Libtayo 176 TARRYTOWN, NEW YORK [REGENERON.COM] Arcalyst 15 Lenvima 1.029 32 Eisai $4.703 $1.380 Humira 544 TOKYO, JAPAN [EISAI.COM] Halaven 380

Sun Pharmaceutical Absorica 158 33 $4.658 $302 Ciprofloxacin Hydrochloride 153 Industries Atorvastatin Calcium 134 MUMBAI, INDIA [SUNPHARMA.COM] Diamicron 561 34 Les Laboratoires Servier $4.658 N/A Daflon 500 499 NEUILLY-SUR-SEINE, FRANCE [SERVIER.COM] Aceon 378 Drospirenone; 35 Abbott Laboratories Ethinyl Estradiol 26 ABBOTT PARK, ILLINOIS [ABBOTT.COM] $4.486 $185 Ademetionine 23 Choline Alfoscerate 15 Gamunex-C 1.225 36 Grifols $4.471 $309 Prolastin-C 851 BARCELONA, SPAIN [GRIFOLS.COM] Flebogamma 758

No disclosed product sales or available 37 Yunnan Baiyao Group estimates KUNMING, CHINA [YUNNANBAIYAO.COM.CN] $4.284 $25

38 Fresenius Kabi Heparin Sodium (FRES) 79 BAD HOMBURG, GERMANY [FRESENIUS-KABI.COM] $4.224 $568 Symdeko 1.418 39 Vertex Pharmaceuticals $4.161 $1.396 Orkambi 1.332 BOSTON, MASSACHUSETTS [VRTX.COM] Kalydeco 991 Actemra 383 40 Chugai Pharmaceutical $3.996 $937 Edirol 337 TOYKO, JAPAN [CHUGAI-PHARM.CO.JP] Hemlibra 231

Source: EvaluatePharma® May 2020, Evaluate Ltd, www.evaluate.com *USD in billions, lower numbers in millions WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Pharma 50 19

and insights firm helping business leaders understand how their the shake-up coronavirus has caused the world, that caring attitude company is perceived. “When we look at the biggest drivers of now has been able to organically float to the surface. positive reputational change for the pharma industry in the US, we “I don’t think we’ve changed,” says Tiffany Benjamin, senior see that increases in positive perceptions around corporate respon- director of corporate social responsibility and global health for sibility, for example, positive societal and workplace contributions Eli Lilly and Company and president of Lilly Foundation. “I think as well as transparent and ethical behavior, have risen to the top.” that this has given the public a window into what we do every day. The RepTrak Company’s data also indicate that the public’s And now that people can really see all of our many different emotional connection with pharma companies has increased at efforts, they’re impressed.” twice the rate compared to how they measure pharma companies on their perceived actions in domains such as innovation, products INVESTING IN OUTREACH and services, corporate responsibility, and leadership. When it comes to contributions, the greatest impact pharma has “While we have not measured the specific had outside of R&D has been through monetary donations to impact of select contribution efforts, we various organizations that aim to alleviate the impact of COVID- know that citizenship is currently the second 19 on communities, whether through food pantries, the supply most important driver of reputation for the of personal protective equipment (PPE), or education campaigns. pharma industry,” says Klingemann. Lilly is one company that helped to start a COVID-specific What makes pharma different than other fund along with other investors. The newly created Indianapolis industries is that it has always had an intrin- e-Learning Fund recently announced $1.5 million in grants to Sven Klingemann sic dedication to improving lives. As part of Marion County schools to address urgent gaps in e-learning for

2019 2019 2019 Rx Sales* R&D Spend* Top-selling Drugs* Sumitomo Dainippon Latuda 1.744 41 $3.623 $823 Brovana 302 Pharma Aptiom 211 OSAKA, JAPAN [DS-PHARMA.COM] Focus V 385 42 Sino Biopharmaceutical $3.373 $347 Runzhong 368 HONG KONG [SINOBIOPHARM.COM] Tianqingganmei 261 Aitan 336 43 Jiangsu Hengrui Medicine Sevoflurane 287 LIANYUNGANG, JIANGSU PROVINCE, CHINA [HRS.COM.CN] $3.321 $518 Dexmedetomidine Hydrochloride 233 Opdivo 858 44 Ono Pharmaceutical $3.266 $653 Januvia 242 OSAKA, JAPAN [ONO.CO.JP/ENG] Orencia SC 182 Bystolic 340 45 Menarini $3.233 N/A Enantyum 162 FLORENCE, ITALY [MENARINI.COM] Uloric 114 Vasostrict 523 46 Endo International $2.887 $131 Xiaflex 328 DUBLIN, IRELAND [ENDO.COM] Adrenalin Injection 179 Somatuline 1.155 47 Ipsen $2.884 $435 Trelstar 456 PARIS, FRANCE [IPSEN.COM] Dysport 435

Shanghai Pharmaceuticals Tanshinone IIA 195 48 $2.875 $195 Shenmai Injection (SHANPH) 138 Holding Hydroxychloroquine Sulfate 113 SHANGHAI, CHINA [SPHCHINA.COM] Remicade 495 49 Mitsubishi Tanabe Pharma $2.822 $783 Simponi 418 OSAKA, JAPAN [MT-PHARMA.CO.JP] Radicava 249 Ertapenem Sodium 80 Hydrochlorothiazide; Losartan 50 Aurobindo Pharma Potassium 60 HYDERABAD, INDIA [AUROBINDO.COM] $2.787 $115 Folotyn 51 Source: EvaluatePharma® May 2020, Evaluate Ltd, www.evaluate.com *USD in billions, lower numbers in millions INNOVATION AND PERSEVERANCE: BRINGING A NEW DELIVERY METHOD TO MARKET

Pharmaceutical Executive: With so many TRT options our clinical sales specialists in the field because so many Innovation and Perseverance: available, why did you investigate a new format? healthcare providers’ offices were either closed or were severely limiting the number of patients they were seeing. Dudley: The short answer is there was a void in the market. Bringing a New Delivery The available products at the time were deep muscle Clarus is not a huge pharmaceutical company that can just injectables, a newer subcutaneous injectable, transdermal throw massive resources at these problems. So, we looked A Q&A Method to Market patches, an initial gel product that I co-invented and quickly and carefully at how we could pivot to interacting brought to market, other similar gels, and a nasal product. with healthcare professionals from a virtual perspective. What was clearly missing was an oral TRT option. The only We quickly retooled promotional materials so they could be oral TRT ever approved by the FDA before our product was used in virtual presentations. We went through immense amounts of new training with our sales professionals he market for testosterone replacement products has expanded rapidly over the past methyltestosterone, which can be toxic to the liver and is about these new technologies. When the dust settles, it couple of decades, with patients having access to numerous options from injectables rarely used. will show that we have handled this current COVID crisis to patches to gels. “When you look at what’s available, it almost screams, ‘Where’s the T Pharmaceutical Executive: How has the TRT amazingly well and maintained some forward momentum oral option?” says Robert E. Dudley, PhD, chairman, chief executive officer and president landscape evolved since you’ve developed the first in some pretty clever and ingenious ways when I think a at Clarus Therapeutics, Inc. testosterone gel? Robert E. Dudley, PhD lot of companies, particularly bigger ones, cannot move as Chairman, Chief Executive Officer In 2019, the FDA approved a testosterone replacement therapy from Clarus Therapeutics, Dudley: First and foremost is the availability of delivery quickly. I’m really, really proud of the team. and President methods that were more convenient than the deep muscle Clarus Therapeutics, Inc. Inc. that is designed for oral delivery to avoid certain administration challenges that exist with other therapies. Here, Dudley speaks with Pharmaceutical Executive about the long injectable products that made up the market before the road to developing and commercializing JATENZO® (testosterone undecanoate) capsules, late 1990s. CIII, the first and only FDA-approved oral softgel testosterone replacement therapy for the The second thing that occurred more broadly in the scope treatment of men with certain forms of hypogonadism. He also talks about what it has of men’s health was the approval of Viagra. This product been like to launch a new product amid the COVID-19 pandemic. “ What was clearly missing was an increased awareness of low testosterone as a real medical condition with real symptoms, helped men open up about oral TRT option. The only oral TRT Pharmaceutical Executive: How did you which is a testament to why no one else their issues, and provided an opportunity for people become involved in men’s health as a has managed to do what we’ve been able to begin looking at therapies to address it. The market ever approved by the FDA before to do. dramatically expanded in the early 2000s, and continued focus for your career? our product can be toxic to the The first obstacle was that you cannot to grow quite steadily, growing considerably around 2009– Dudley: I trained in pharmacology and give testosterone orally by itself. It is 2013. Arguably then, too many men were being prescribed toxicology at the University of Kansas liver and is rarely used. metabolized too efficiently by the liver, so testosterone and that raised concerns with FDA that it was Medical School and my background we needed to have a testosterone prodrug. not being used as properly as it should be. is in drug development. And then, This led to a second obstacle: testosterone quite serendipitously, I joined a small Around 2013–2014, the FDA raised the possibility that prodrugs are very lipophilic, and thus are entrepreneurial company as head of R&D, there could be increased cardiovascular risk associated very insoluble, difficult to formulate, and with testosterone products, particularly in older men. The ” where I made my first foray into the world of challenging to keep in solution. In fact, we Pharmaceutical Executive: What sets Clarus testosterone replacement with a sublingual agency began to look more closely at testosterone products looked at 80 different formulations before Therapeutics apart from other companies in the testosterone product. in development and tightened the regulations on them. we ultimately settled on one that we took men’s health space? So, by the time our oral testosterone product was up for From there, I went on to develop a into the clinic and used in the approved Dudley: I founded Clarus for the sole purpose of developing approval, FDA’s regulatory requirements had tightened up testosterone gel product, the first gel oral testosterone. men’s health products, starting with an oral testosterone product that was approved and marketed pretty significantly. We not only had to address the average A third obstacle was the pharmacokinetics. replacement product. It has been my passion and my drive. in the United States. In 2004, I founded peak concentrations I mentioned earlier, but we also had Transdermal patches and the gels produce to show very strong evidence that if a healthcare provider I love the entrepreneurial environment of a smaller company. Clarus Therapeutics, Inc. to take my fairly flat testosterone levels in the blood, decided to adjust the dose, it would result in a testosterone I excel in providing leadership in that kind of environment. experience and develop a safe and effective but the oral prodrug creates peak-and- level in the normal range. oral testosterone prodrug, which received Pharmaceutical Executive: How would you describe valley levels of testosterone in the blood, FDA approval in 2019. That was the first time that we are aware of that these your leadership style? like any oral drug. You have to get to the strict criteria were applied to testosterone products of any right average testosterone concentration in Dudley: I’m a planner and all about execution. I’m not afraid Pharmaceutical Executive: What kind. It was just another example of the evolution within the normal range without excessive peaks, of challenges. I thank my parents for raising me never to challenges did you face in your the agency and the requirements that we needed to meet. which is a challenge. quit. No matter what the challenges are, you simply have to quest to create an oral testosterone In the end, we were able to provide FDA with all of the replacement therapy (TRT)? find your way around them. That upbringing and my ability These were our three biggest challenges, information it needed and received approval. to bring together and empower teams and individuals Dudley: We faced several challenges. It which, along with a few others, consumed Pharmaceutical Executive: How did the COVID-19 within the organization are hallmarks of my leadership. I a lot of our time and effort as we brought took the greater part of 16 years to develop crisis affect your plans to go to market? hire people that are really great at what they do, and my the product forward. and receive FDA approval on an oral TRT, job is to support them. Dudley: After overcoming so many obstacles to get our product to market, one could not have imagined that we I’m very keen on transparency, integrity, and being honest. would be dealing with the COVID-19 pandemic, which We are in a very important business of treating, in our SPONSORED BY surfaced four to five weeks after we launched our product. case, men. All of us in the healthcare field have a real moral We never fathomed that we would suddenly have to pull back obligation to be men and women of high integrity. INNOVATION AND PERSEVERANCE: BRINGING A NEW DELIVERY METHOD TO MARKET

Pharmaceutical Executive: With so many TRT options our clinical sales specialists in the field because so many Innovation and Perseverance: available, why did you investigate a new format? healthcare providers’ offices were either closed or were severely limiting the number of patients they were seeing. Dudley: The short answer is there was a void in the market. Bringing a New Delivery The available products at the time were deep muscle Clarus is not a huge pharmaceutical company that can just injectables, a newer subcutaneous injectable, transdermal throw massive resources at these problems. So, we looked A Q&A Method to Market patches, an initial gel product that I co-invented and quickly and carefully at how we could pivot to interacting brought to market, other similar gels, and a nasal product. with healthcare professionals from a virtual perspective. What was clearly missing was an oral TRT option. The only We quickly retooled promotional materials so they could be oral TRT ever approved by the FDA before our product was used in virtual presentations. We went through immense amounts of new training with our sales professionals he market for testosterone replacement products has expanded rapidly over the past methyltestosterone, which can be toxic to the liver and is about these new technologies. When the dust settles, it couple of decades, with patients having access to numerous options from injectables rarely used. will show that we have handled this current COVID crisis to patches to gels. “When you look at what’s available, it almost screams, ‘Where’s the T Pharmaceutical Executive: How has the TRT amazingly well and maintained some forward momentum oral option?” says Robert E. Dudley, PhD, chairman, chief executive officer and president landscape evolved since you’ve developed the first in some pretty clever and ingenious ways when I think a at Clarus Therapeutics, Inc. testosterone gel? Robert E. Dudley, PhD lot of companies, particularly bigger ones, cannot move as Chairman, Chief Executive Officer In 2019, the FDA approved a testosterone replacement therapy from Clarus Therapeutics, Dudley: First and foremost is the availability of delivery quickly. I’m really, really proud of the team. and President methods that were more convenient than the deep muscle Clarus Therapeutics, Inc. Inc. that is designed for oral delivery to avoid certain administration challenges that exist with other therapies. Here, Dudley speaks with Pharmaceutical Executive about the long injectable products that made up the market before the road to developing and commercializing JATENZO® (testosterone undecanoate) capsules, late 1990s. CIII, the first and only FDA-approved oral softgel testosterone replacement therapy for the The second thing that occurred more broadly in the scope treatment of men with certain forms of hypogonadism. He also talks about what it has of men’s health was the approval of Viagra. This product been like to launch a new product amid the COVID-19 pandemic. “ What was clearly missing was an increased awareness of low testosterone as a real medical condition with real symptoms, helped men open up about oral TRT option. The only oral TRT Pharmaceutical Executive: How did you which is a testament to why no one else their issues, and provided an opportunity for people become involved in men’s health as a has managed to do what we’ve been able to begin looking at therapies to address it. The market ever approved by the FDA before to do. dramatically expanded in the early 2000s, and continued focus for your career? our product can be toxic to the The first obstacle was that you cannot to grow quite steadily, growing considerably around 2009– Dudley: I trained in pharmacology and give testosterone orally by itself. It is 2013. Arguably then, too many men were being prescribed toxicology at the University of Kansas liver and is rarely used. metabolized too efficiently by the liver, so testosterone and that raised concerns with FDA that it was Medical School and my background we needed to have a testosterone prodrug. not being used as properly as it should be. is in drug development. And then, This led to a second obstacle: testosterone quite serendipitously, I joined a small Around 2013–2014, the FDA raised the possibility that prodrugs are very lipophilic, and thus are entrepreneurial company as head of R&D, there could be increased cardiovascular risk associated very insoluble, difficult to formulate, and with testosterone products, particularly in older men. The ” where I made my first foray into the world of challenging to keep in solution. In fact, we Pharmaceutical Executive: What sets Clarus testosterone replacement with a sublingual agency began to look more closely at testosterone products looked at 80 different formulations before Therapeutics apart from other companies in the testosterone product. in development and tightened the regulations on them. we ultimately settled on one that we took men’s health space? So, by the time our oral testosterone product was up for From there, I went on to develop a into the clinic and used in the approved Dudley: I founded Clarus for the sole purpose of developing approval, FDA’s regulatory requirements had tightened up testosterone gel product, the first gel oral testosterone. men’s health products, starting with an oral testosterone product that was approved and marketed pretty significantly. We not only had to address the average A third obstacle was the pharmacokinetics. replacement product. It has been my passion and my drive. in the United States. In 2004, I founded peak concentrations I mentioned earlier, but we also had Transdermal patches and the gels produce to show very strong evidence that if a healthcare provider I love the entrepreneurial environment of a smaller company. Clarus Therapeutics, Inc. to take my fairly flat testosterone levels in the blood, decided to adjust the dose, it would result in a testosterone I excel in providing leadership in that kind of environment. experience and develop a safe and effective but the oral prodrug creates peak-and- level in the normal range. oral testosterone prodrug, which received Pharmaceutical Executive: How would you describe valley levels of testosterone in the blood, FDA approval in 2019. That was the first time that we are aware of that these your leadership style? like any oral drug. You have to get to the strict criteria were applied to testosterone products of any right average testosterone concentration in Dudley: I’m a planner and all about execution. I’m not afraid Pharmaceutical Executive: What kind. It was just another example of the evolution within the normal range without excessive peaks, of challenges. I thank my parents for raising me never to challenges did you face in your the agency and the requirements that we needed to meet. which is a challenge. quit. No matter what the challenges are, you simply have to quest to create an oral testosterone In the end, we were able to provide FDA with all of the replacement therapy (TRT)? find your way around them. That upbringing and my ability These were our three biggest challenges, information it needed and received approval. to bring together and empower teams and individuals Dudley: We faced several challenges. It which, along with a few others, consumed Pharmaceutical Executive: How did the COVID-19 within the organization are hallmarks of my leadership. I a lot of our time and effort as we brought took the greater part of 16 years to develop crisis affect your plans to go to market? hire people that are really great at what they do, and my the product forward. and receive FDA approval on an oral TRT, job is to support them. Dudley: After overcoming so many obstacles to get our product to market, one could not have imagined that we I’m very keen on transparency, integrity, and being honest. would be dealing with the COVID-19 pandemic, which We are in a very important business of treating, in our SPONSORED BY surfaced four to five weeks after we launched our product. case, men. All of us in the healthcare field have a real moral We never fathomed that we would suddenly have to pull back obligation to be men and women of high integrity. WWW.PHARMEXEC.COM 22 22 Pharma 50 PHARMACEUTICAL EXECUTIVE JUNE 2020

underserved students and their families. This includes providing teered their time as part of a joint effort with Merck & Co. and internet connectivity and laptops to students, and a statewide Pfizer, which enables medically licensed employees to aid in the e-learning lab currently in development, which will provide coronavirus fight while earning their regular pay. e-learning resources and best practices to educators. For those unqualified to make such a hands-on contribution, Novartis’ Swiss headquarters announced a $20 million fund the company is encouraging remote volunteerism. to respond to coronavirus outreach and is taking requests for help “We’ve always had a volunteerism site, but within that, we’ve from around the world. A separate program known as the Novar- created a page that’s specific to opportunities to volunteer tis US Foundation focuses specifically on the US. When the foun- remotely,” says Benjamin. “You can make masks from your home dation announced a new $5 million relief fund specifically for or cards for seniors, or [get] information on donation drives— COVID-19 on April 3, it received nearly 240 requests. ways to still socially distance but also individually make an impact Novartis has funded 19 programs to date with 11 pending, on your community.” donating a total of almost $4 million. Lilly has also partnered with the governor of Indiana, Eric The first two grants were to Americares and Direct Relief to Holcomb, along with the Indianapolis mayor, Joe Hogsett, in a support safety net clinics, hospitals or clinics that take all patients social media initiative called #INthistogether. The online cam- regardless of their ability to pay. Another grant went toward paign helps raise awareness about social distancing to help flatten developing COVID Check, a free public digital health service the curve in Indiana. aimed at helping people who have or are concerned they might AstraZeneca is another company working hard to make life have COVID-19. better. In addition to providing financial donations that support Another grant supported the New Jersey Primary Care Asso- relief efforts, it has partnered with the World Economic Forum’s ciation, which made it possible for them to launch five new tele- COVID Action Platform to donate nine million face masks to health programs for community health centers in the state. support healthcare workers around the world. Employees have “I’ve received a lot of positive comments also provided refurbished phones to patients and staff in medical about what the foundation is doing,” says facilities so that they can speak to their loved ones who they are Thomas Kendris, US country president at unable to be with. Novartis. “But this kind of activity, the kind Anticipating that their workforce would want to help during of support that our associates do in the com- the current pandemic, AstraZeneca updated its Global Volun- munity, it’s not new. There are people through- teering Policy during COVID-19. Employees trained as health- out the company who have devoted their time care professionals (HCPs) can now take paid leave for up to four Thomas Kendris and volunteered to lots of causes and local weeks to work on the front lines. All employees are also being groups [even before the pandemic].” offered a week’s paid leave in addition to their local volunteer The recent experience with Novartis US Foundation’s COVID- paid leave to participate in general community 19 fund has demonstrated to Kendris that there is always more volunteering activities. that can be done. Before the pandemic, he says it was difficult to “Many of our employees have already connect with small groups that might not have known about the taken up this opportunity to make a positive available funds Novartis had to offer or didn’t have the resources impact in their local community,” says Fiona to pursue them. Cicconi, executive vice president of human “The pandemic has had an unintended consequence that it resources at AstraZeneca. “The positive effect Fiona Cicconi has put our foundation into contact with dozens and dozens of on employee morale is also evident. The pride groups that we had not been in contact with before that we can I have seen across the globe is really special and is helping us to help,” he says. “There is tremendous upside in terms of Novartis do even more as a company to contribute to helping the community through our foundation. I think it’s really the efforts to fight COVID-19.” good moving forward.” This sense of employee pride isn’t just good for the community; it’s also good for COMMUNITY SERVICE the company, says Dr. Anita Gupta, senior Since March, Lilly has been partnering with the Indiana State vice president of medical strategy and gov- Department of Health with FDA support to accelerate COVID- ernment affairs at Heron Therapeutics. 19 testing in Indiana. The company launched a drive-through “When they have an authentic purpose, Anita Gupta testing facility at its corporate headquarters in downtown India- employees are more likely to drive forward a positive culture, and napolis where they have completed more than 60,000 tests. Many with an evolved vision and a growth mindset, they are more likely of the on-site workers are Lilly medical professionals who volun- to have a higher performance and impact overall,” she says. “An WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Pharma 50 23

Lilly employees volunteering at a drive-through testing site for front-line responders in Indianapolis. organization often defines its purpose when a major crisis occurs, by subject-matter experts from across the organization, they pro- such as COVID-19, which ultimately will drive value for the orga- vide advice and tips on how to navigate both professional and nization and the broader global society.” personal challenges. The toolkits were so well received that the company has made them available externally for other organiza- EYE ON EMPLOYEES tions to use. In addition to donating 130 million doses of hydroxychloroquine Employee programs and resources such as this also help to and providing in-kind donations to hospitals, including two ven- boost morale. In AstraZeneca’s latest biannual employee engage- tilators, thousands of masks, 10,000 caps, 10,000 gloves, and ment survey, for example, 86% of employees reported they would more than 1,000 gowns and coveralls, Novartis is taking a direct recommend the company as a great place to work compared to approach to help support all families touched by the virus, start- a pharma norm of 80%. ing with its own employees. The company has extended a global commitment of 12 days OPPORTUNITY KNOCKS paid leave in addition to vacation and time off. It is providing Whether it’s through grants, donations, or support of local com- childcare assistance to manufacturing and laboratory employees munities and employees, people are taking notice of all the good and offering a variety of online homeschooling resources. pharma is doing, and they’re seeing the industry in a new light. Employees also have access to an app that provides advice, strat- What might this mean? Well, it could be the opportunity of a egies, and support in areas such as nutrition, movement, mind- lifetime to turn things around. The key will be holding onto this fulness, and recovery. image moving forward. “We also try to stay connected, not just for meetings that we One way pharma might try to maintain its improved reputa- have to have to run the business, but to just check on each other, tion is by getting the word out more. According to RepTrak, make sure people are okay— if someone who’s alone is doing media analysis of the COVID-19 crisis shows that, relatively okay—and getting them help if they need it,” says Kendris. speaking, pharma has not had the same media coverage as other AstraZeneca compiled two toolkits—one for employees and industries. This suggests that the industry could benefit from step- one for leaders—to help them through the pandemic. Created ping up its efforts to publicize its contributions. WWW.PHARMEXEC.COM 24 24 Pharma 50 PHARMACEUTICAL EXECUTIVE JUNE 2020

“The expectation is that of increased education and commu- While the future is uncertain, now is the time for pharma to nication—ideally by company CEOs—of the COVID-19 crisis regain some trust and redefine itself as a force for positive change. about implications for customers, the Fortunately, it looks like many industry community, employees, and supply CEOs are aware of this. In a recent chain personnel,” says Klingemann. This is where pharma closed virtual roundtable focused on “This is where pharma companies need COVID-19, organized by Pharmaceutical to work on achieving a better commu- companies need to work Executive, a number of industry CEOs nication and education breakthrough.” expressed their opinions with com- This is also the time to build a “rep- on achieving a better ments such as: “This is an existential utational buffer,” Klingemann says, that moment for the industry,” “I think this can mitigate some of the negative communication and will be our finest hour,” and “I hope impacts of any future crisis. This good- education breakthrough that we will take this opportunity as we will capital can serve to remind the pub- go forward not to let it go back to where lic of pharma’s track record as a trust- we were before this pandemic.” worthy partner who has the public’s best interests in mind, and Pharma has been given another chance, and it sounds like it it can act as insurance moving forward. isn’t about to waste it.

Spotlight on Science Gives Public New Perspective

The public may appreciate seeing pharma’s softer side as a demonstrates it better than this situation where they’re result of the industry’s pandemic outreach efforts. But it is also hearing news reports that don’t come from the companies getting some new perspectives on what exactly goes into drug themselves,” says Kendris. “It’s the media covering the work development. The industry’s improving reputation suggests that the companies are doing. And I think people are probably people are gaining a better understanding and newfound appreciating that more now than ever before.” respect for what pharma actually does. In fact, in a Pew Research Center study conducted April 29 “When you look at the polls, the public is really counting on to May 5, 83% of US adults said there will probably or the government and the industry during this time of crisis, and definitely be an effective treatment or cure for COVID-19 in the the biopharmaceutical industry can play and is playing a really next 12 months. It also indicated that 73% of respondents felt important role,” said Bristol Myers Squibb Chairman and CEO there will probably or definitely be a vaccine to prevent Giovanni Caforio during CNBC’s Healthy Returns summit last coronavirus in the next year. Around 72% of those surveyed month. “That’s who we are and this is what we do.” said they would get a coronavirus vaccine if one were Two aspects of biopharma that have come to the forefront available. as a result of the COVID-19 pandemic are how important the The Pew Research Center study found that 83% of adults discovery of potential new vaccines and therapies is and how expect another coronavirus outbreak within the year, and 69% difficult it is to develop them. expect the focus on the disease to delay progress on other As government turns to the pharma industry not just for treatments for non-COVID-related conditions. therapies or a vaccine but for tests and data to evaluate these “With its new-found reputational credit, the pharma industry treatments, people are receiving an education. They’re will certainly get the benefit of the doubt as the search for learning about the complexities of clinical trials and how treatment continues,” says Sven Klingemann, director of different tests can yield different results. They’re realizing the RepTrak. “That should buy it some time. [But] it is critical that painstaking process from discovery to clinical trials to they continue to reinforce their actions around corporate regulatory approval and manufacturing, and why it traditionally responsibility as a foundation that will endure outside of major takes 12 to 18 months to develop a vaccine. health crises.” “I think that’s not something that people had top of mind This certainly is not a time for pharma to rest on its laurels. before this,” says Thomas Kendris, US country president at It’s time to continue putting the focus on the power of science Novartis. “I think they’re now probably more aware than ever and promoting that idea in a very public way. that the pharmaceutical industry does play an important role in “I hope that this crisis could reset the perception about the finding solutions to a crisis like this.” valuable position that the industry is bringing to society,” said Though there is no vaccine yet for the novel coronavirus Pfizer Chairman and CEO Albert Bourla during the CNBC and many therapies remain unproven, people have taken summit. “It’s a great opportunity to be able to set it where it notice that the industry has moved quickly and is working should be. I know that reputation is earned in drops, but you tirelessly to get there. And much of the information is coming can lose it in buckets. And I hope that we will earn it drop from a neutral third party, the media, giving more weight to after drop.” the message. “We can say what we do, but I think nothing — Elaine Quilici Secondary Data First: Discover how routinely collected healthcare data can be used to generate reliable evidence faster to address regulator, payer, and provider needs.

On-demand webinar Secondary data sourced from routinely collected healthcare Aired June 10, 2020 information is playing a decisive role in stakeholder decision making. Join us to learn how this data is accessed today, Regisiter now for free! which database is right for your purposes, and the key steps www.pharmexec.com/pe_p/ you need to consider to optimize your studies from planning drug_lifecycle to execution. Discover more about:

• Identifying the typical research questions across the drug Massoud Toussi, MD, PhD, MBA lifecycle Senior Principal, Real World Solutions, Paris, France, IQVIA • Understanding what secondary data sources are fit for which purpose Nikita Jeswani Principal, Real World Solutions, London, UK, • Accessing clinically rich secondary data in rare diseases IQVIA and oncology through novel solutions

MODERATOR: • Generating the right evidence for regulators, payers, Elaine Quilici providers, and patients Senior Editor, Pharmaceutical Executive

We will share tangible examples of how multi-country database studies leveraging secondary data have helped answer key stakeholder questions throughout the product lifecycle. Presented by:

Sponsored by: CONTACT US www.iqvia.com

For technical questions about this webinar,

Product Launch Strategies Adapting Approaches in 2020 and Beyond BART LOMBARDI With new access levers emerging, positioning products will require a shift Founder and Managing form the traditional launch playbook Director, Clarion

he ecosystem surrounding biopharmaceuticals is blocks diminish the traditional opportunity for healthcare provid- changing in transformative ways along numerous ers (HCPs) to trial a new pharmacy benefit product and begin to dimensions. Those include therapeutic innovation, establish early physician demand, hindering uptake in the critical use of predictive analytics and artificial intelligence first six months of launch. More broadly, the bar has been raised T(AI), the increasing role of patients and caregivers, market access to provide compelling evidence of product differentiation and restrictions, pricing pressure, and new manufacturing and distri- value to secure coverage. As one panelist suggested, “good prod- bution channels and players, to name just a few. The challenge ucts can still get strong market access, it just takes a lot of dedi- for pharmaceutical and biotechnology companies—also dealing cated effort, years in advance, with significant collaboration across today with uncertain business headwinds from the COVID-19 clinical, medical, and commercial teams.” pandemic, which is impacting planning and decision-making—is 2) Downward pressure on pricing by policymakers (and to recognize how these factors affect new product introduction in politicians), health plans, and benefit managers. The uncertainty 2020 and beyond, and proac- and potentially sweeping tively adapt commercialization impact of policy changes is strategies. In the years to come, challenging to biopharma those biopharma industry lead- Uncertainty on which legislative companies. One executive sur- ers who integrate deep customer vey respondent pointed out, insights, leverage new technolo- or regulatory actions will be taken “uncertainty on which legisla- gies to identify and engage cus- tive or regulatory actions will tomers, focus on delivering value, creates many different scenarios be taken creates many differ- and continue to anticipate the to consider and monitor, ent scenarios to consider and impact of evolving market and monitor, particularly for fore- healthcare dynamics will be the particularly for forecasting casting.” The focus of media best positioned to deliver poten- attention on this issue is often tially successful new product around limiting price increases launches. over time. Such restrictions This article offers insights from two complementary sources: would put pressure on biopharma organizations to get the price a panel moderated at CBI’s 5th Annual Bio/Pharmaceutical right at launch. That, in turn, means getting ahead of potential Product Commercialization and Launch Roadmap Conference pricing criticisms with robust rationale and supporting evidence. in late October 2019 and Clarion’s second annual BioNetwork One-time, curative therapies (e.g., cell and gene therapies) are Survey of senior US biopharmaceutical executives. navigating this situation today. 3) Entry of novel distribution channels was the trend THREE TRENDS SHAPING TOMORROW’S most often cited as transformative by executives in Clarion’s sur- GO-TO-MARKET STRATEGIES vey. On one end of the spectrum, there are advanced therapies What do US biopharma executives think will be the most impact- like cell and gene medicines driving the need for new logistical ful trends affecting successful new product commercialization this solutions (e.g., chain of identity, chain of custody) in a nascent year and beyond? supply chain that is completely outside traditional drug distribu- 1) Increasing market access restrictions, including US tion channels. On the other, there are new players leveraging payer adoption of new-to-market blocks, growing exclusion lists, digital technology and the consumerization of healthcare to higher patient out-of-pocket costs, and challenging reimburse- potentially revolutionize the way most drugs are sold. For instance, ment arrangements. Market access challenges can change launch Amazon, one such potential player, would not be just another dynamics in fundamental ways. For example, new-to-market wholesale channel to sell into, but represents a radically different WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Market Access 27

consumer-centric, e-commerce platform that is dominating the Across stakeholder groups, the nature of information being broader consumer marketplace. In competitive markets, Amazon communicated is also increasingly complex, owing to more could enable unprecedented consumer engagement in prescrip- nuanced science and new data sources. To this end, medical affairs tion medication feature and price comparisons and provide a is emerging as a critical strategic leader of scientific and data- seamless acquisition process integrated into a platform that con- focused engagement across stakeholders, leveraging the scientific sumers shop through every day. and clinical expertise of those leaders in this area. Biopharma organizations are wise to invest in medical affairs ADAPTING COMMERCIALIZATION capabilities that enable optimal experiences and outcomes for all STRATEGIES TO NEW MARKET REALITIES stakeholders. What strategic levers are most important to succeeding in this • Sophistication in customer segmentation. Health- evolving landscape? Forward-thinking companies in the industry care entities no longer accept a one-size-fits-all approach, but, already have and continue to evolve their approach. Many aspects fortunately, we have also seen the advent of the data and engage- of “traditional” launch playbooks (e.g., sales force scale, advertis- ment channels required to more robustly characterize customer ing and promotional (A&P) spending, broad marketing cam- archetypes and their unique needs, as well as tailor content and paigns, share-of-voice measure) are being replaced by greater engagement accordingly. Artificial intelligence (AI)-based focus on sales force sophistication, real-world evidence (RWE), approaches are being applied throughout the entire value chain high quality, direct patient engagement, and innovative payer from drug discovery to patient-finding to commercial marketing. contracts and access programs. This sophistication relies on Among the specific levers our advanced analytical and compu- panelists and survey respondents tational capabilities, areas where viewed as being most important Phase I is not too early to be biopharma has formerly lagged include the following. other industries and is catching up. • Demonstrating product working on market access Drug manufacturers should iden- value. Biopharmas need to get tify the data capabilities that are ahead of access barriers by gen- most critical to success, given their erating and communicating evidence of the differentiated clini- specific product portfolio and company strategy, and invest to build cal and economic value of their new therapies. Value evidence or acquire the right enabling talent and infrastructure. generation needs to start early, with medical and commercial • Multifaceted customer-facing field teams.The ongo- input into clinical study design, even earlier than pivotal studies. ing shifts in stakeholder dynamics and the types of new drugs Engaging with payers and other stakeholders also needs to start entering the market require companies to think more critically early (i.e., years, not months before launch) to educate on the about the structure and makeup of their field force to ensure disease and unmet needs as well as understand what value means customer needs are met. Increasingly, this means engaging cus- to each stakeholder. As one panelists suggested, “Phase I is not tomers through a team of personnel with specialized capabilities. too early to be working on market access.” Moreover, it doesn’t HCPs and their staff are being engaged not only by sales repre- stop at launch—increasingly there is need to continue demon- sentatives and medical science liaisons (MSLs), but also commer- strating value through real-world data. cial thought leader liaisons, field reimbursement specialists, and • Meaningful engagement with the expanded set of clinical educators. For payers, many companies now employ influencers and decision-makers. Our survey respondents medical outcomes specialists as well as traditional commercial and panelists acknowledged that key opinion leader (KOL) engage- account managers, and patients and their caregivers may interact ment (traditionally focused on HCP KOLs) has always been and with patient educators, case managers, reimbursement support continues to be a key factor in successful product introduction. specialists, and patient advocacy engagement personnel. These However, the reasons to engage KOLs—to gather insights, edu- field teams need to be highly coordinated to maximize the impact cate, and secure advocacy—are increasingly critical across a of their collective efforts. broader set of stakeholders, including payers, regulators, and, Although the industry is changing in fundamental ways, bio- notably, patients, caregivers, and advocacy groups. Patients are pharma leaders can get ahead of the curve to succeed in the new empowered by increased access to information and are actively ecosystem. In 2020 and beyond, this means focusing on deliver- taking a greater role in healthcare decisions. To be successful, bio- ing value backed by data, tailored and meaningful engagements pharma companies need to meaningfully engage patients/care- with a diverse range of more distinctly defined stakeholders, and givers and advocacy groups to understand their needs at a funda- continuing to monitor the rapidly evolving healthcare space for mental level and communicate information in highly tailored ways. new trends that will impact success moving forward. WWW.PHARMEXEC.COM 28 28 Executive Profile PHARMACEUTICAL EXECUTIVE JUNE 2020

Rare Insights Neurologist Arndt Rolfs, CEO and founder of CENTOGENE, discusses how he grew the genetic diagnostics company from small startup to a global organization focused on advancing the understanding of the pathophysiology of rare hereditary diseases to inform future drug development

By Julian Upton WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Executive Profile 29

ENTOGENE, headquartered in Rostock, medicine have. We are somewhat hampered by the Germany, is a rare disease company focused complexity of the brain. on transforming clinical, genetic, and bio- But in the last couple of decades, we have become chemical data into medical solutions for very well educated about the topic and about how we Cpatients. With operations also in Berlin, Germany, and can diagnose diseases. Cambridge, MA, the company is guided on the prin- I have a strong background in the clinical under- ciple that a detailed, global understanding of the standing of rare hereditary diseases. I was active for genetic basis and the clinical phenotype of rare hered- several years in the Max Planck Institute for Molecular itary diseases will unlock the ability to target rare dis- Genetics, where the first PCR (polymerase chain reac- eases and provide critical knowledge that will facilitate tion) and non-radioactive sequencing in Europe was orphan drug development. implemented. With that technical experience, I founded The company’s data repository, said to be the world’s my first two biotech companies in 2000. largest repository for genetic information on rare hered- To cut a long story short, it was too early to open a itary diseases, combines clinical data and phenotype, biotech in Europe, and at that time some of the regula- genotype, proteomics, and metabolomics data to aid in tory and governance topics had not been properly the development of new treatments. addressed. But it was part of the learning curve and, CENTOGENE was founded in 2006 by Dr. Arndt based on that experience, I went on to found CENTO- Rolfs, a neurologist with long clinical experience in GENE in 2006. I went about starting CENTOGENE rare hereditary diseases. Rolfs received his official in a completely different way. I decided not to bring license to practice medicine from the Universities of any investors on board; I wanted to carry my own Mainz and Vienna in 1985 and was granted a tenure weight and rely on my own expertise and my knowl- track professorship for clinical neurology in 1997. He edge to develop the company and create solutions for worked at the Department of Neurology and served the rare disease challenges to come. as head of the Laboratory for Neurochemistry at the Free University of Berlin, at the Max Planck Institute PE: What sort of trajectory has the company had? Is it some- for Molecular Genetics, and at the psychiatric clinic thing that took off quickly or has it been a longer process to of the University Hospital Rudolf-Virchow in Berlin. get where you are today? Since 1998, he has been the head of the neurobio- Rolfs: You can always do things faster, but for us it logical research laboratory and vice director of the has been a stepwise process. With rare hereditary dis- neurological clinic and outpatients department at the eases, you cannot focus on a small geographical area, University of Rostock. In 2008, Rolfs was appointed on one country or one continent, because the epide- director of the Albrecht-Kossel-Institute for Neurore- miology, the clinical manifestation, the genetics, the generation at the University of Rostock. He has an ethnicity, and the diseases themselves are completely extensive track record in medical and scientific publi- different. Because we are sometimes talking about cations and acts as a consultant for international bio- ultra-rare diseases, with perhaps just over 50 patients tech and industrial companies. in some cases, it’s clear that whatever you do in that Taking time out from his busy schedule, Rolfs sat environment has to be a global activity from the outset, down with Pharm Exec recently to talk about how he and has to be based on a global understanding of the grew CENTOGENE from small startup to a world- complexity of the data. wide company with nearly 500 employees, and his Pharma companies are very interested in orphan vision for the treatment of rare hereditary diseases drugs, of course, but they face significant problems in going forward. understanding rare events or rare diseases. What, for example, is the natural history of the disease? What PE: After a long and distinguished career in medicine and aca- are the manifestations? What mutations, what genetic demia, what prompted you to found CENTOGENE in 2006? defects are driving the disease? All of these topics are Arndt Rolfs: I’ve spent 30 years in molecular medi- extremely difficult for pharma partners to address cine; I’m a neurologist, a neurogeneticist and a psy- properly. But you have to start with the first step. It’s chiatrist, and I guess it’s to do with a characteristic of about getting access to the data, collecting the data, neurologists—we don’t have those therapeutic topics, standardizing the documentation. We have more than for example, like our colleagues in oncology or internal a million patients in our registry, so how can you stan- WWW.PHARMEXEC.COM 30 30 Executive Profile PHARMACEUTICAL EXECUTIVE JUNE 2020

dardize the documentation? You have to solve one problem at We are using what we call the CentoCard, which has contributed a time. to a lot of the company’s success. It’s a simple piece of paper; During the first phase of the company, the first six to seven when you put a drop of blood on the paper, and let it dry, the years, we were constantly trying to find a solution to the next sample stays stable for at least 13 years. Because of the drying challenge. In the second phase, we took all of that expertise and process, you can just put it in an envelope and ship it anywhere knowledge, those new solutions, to our pharma partners. Now in the world; it doesn’t require dry ice or a cooling device, which we are moving into the third phase, where we have what is prob- would typically increase the transportation costs massively. But ably the largest data bank in the world focused on rare hereditary whenever you perform mass spectrometry on the blood sample, diseases. The question now is: How can we create the biggest you are generating a huge spectrum of different peaks, typically impact from those huge data sets? What additional information 150,000 to 250,000 peaks per run. Very quickly that small sam- can we get out of the exome or the genome? How do we get ple of blood from an individual will generate about 12 to 14 more basic science information out of the existing data? terabytes of data. So we use AI, which can tell us if the pattern of the marker is parallel over time or in the disease. That AI is PE: What for you were the leadership challenges of growing the company? a combination of what is already out there in the market— Rolfs: Leadership for me means that you have to really live for because there’s no need to reinvent the wheel if it has already what you are doing. There is no compromise. But, of course, it’s been done—and the core expertise of our own AI team. always a team effort; success is based on good team interaction. CENTOGENE is a highly interdisciplinary company. From an PE: What does the next five years hold for Centogene? outside perspective, it may look like we are primarily focused on Rolfs: As an industry, we can lose approximately 10 to 11 years diagnostics, but actually, from the outset, CENTOGENE has from the first proof-of-concept experiments to the time when a been an IT-based company. Bioinformatics, big data, and AI are drug is released into the market. That time period is something generating the knowledge on one side; and on the clinical envi- we truly believe we can expedite with the knowledge we are gen- ronment side, it’s about how to generate a deeper insights in erating of around 5,500 different rare diseases. understanding of the pathophysiology of the different diseases. We are now starting to position CENTOGENE more as a So, these characteristics—leadership, teamwork, a thirst for the direct partner to pharmaceutical companies to allow them to topic, a highly interdisciplinary and international structure—have speed up the development of new drugs. How can we do that? helped to turn the company into a success story. Because we are so close to the patients. We have a detailed under- From the very beginning, we have tried to recruit very interna- standing, like a fingerprint, of patients worldwide from the knowl- tional colleagues. Bioinformaticians, edge gained from genomics, pro- applied IT specialists, human genet- teomics, and metalobomics. icists, biotechnologists, AI experts— We’re running huge prospective these are super-specialist roles. It’s How can we create the longitudinal studies with more than clear you can’t just focus on the Ger- biggest impact from those 22,000 patients globally to get a man market or the European mar- better understanding of growth kets. About 70% of our colleagues data sets? What additional trends and also to validate our new come from outside Europe; we now biomarkers. These are generating have more than 50 different nation- information can we get out of deep insights into the public phys- alities on board. At first, identifying iology of different diseases, which and hiring the right people was def- the exome or the genome? enables us also to start supplemen- initely a challenge, but the simple tary programs for treatment devel- answer is the more successful you opment in specific indications. are, the easier it becomes. Success makes you sexy. I started with In the next five years, then, I would like to see CENTOGENE two colleagues and now we have just under 500 people in the offering a holistic solution under the rare-disease umbrella. company. This has to start with a detailed understanding of the patients and the individual disease. It then moves into an understanding PE: With increasing amounts of AI power being deployed in the industry, of how the disease is changing over time and combines all the are you developing your own AI systems and platforms? Is the technology knowledge and expertise together. moving quick enough for what you are trying to do? Ideally, we then end up with the development of a new drug Rolfs: A few years ago, we decided to use biomarker programs and new treatment concepts, which we could then develop fur- we were running for proof of concept for our AI technologies. ther with our pharmaceutical partners. Powering HCP Promotional Precision and ROI Using AI & Predictive Analytics A practical overview of how to use AI & ML with patient-level data to improve HCP targeting, call planning and messaging

Live webinar IQVIA is proud to join the global effort to proactively fight COVID-19. In helping life science companies to plan and Thursday, June 18, 2020 at 11:00 EDT manage through this pandemic, we are committed to helping customers adapt to the disruption in HCP engagement that some marketing and sales teams have experienced, especially Regisiter now for free! around personal promotion. This webinar addresses how www.pharmexec.com/pe_p/ pharma and biotech companies can leverage patient-level data HCP_promotion and machine learning (ML) to interact with HCP customers more effectively and efficiently, delivering the right message to the right customer at the right time. Steve Laux Principal, Predictive Analytics, IQVIA Key Takeaways:

Stephanie Roy • Learn how ML predictive alerts can help your sales force Principal, Predictive Analytics precisely engage physicians, especially with the greater Center of Excellence, IQVIA need for remote personal detailing

• Improve HCP targeting by anticipating the near-term MODERATOR: therapy needs Lisa Henderson Editorial Director, • How predictive alerts can be used to improve call planning Pharmaceutical Executive • Deliver your brand’s message when your customer is ready to listen — when they have a patient that can benefit from Presented by: your therapy

This is a non-technical webinar for pharma/biotech marketing and sales professionals. It provides practical guidance on how and when to use AI-fueled predictive analytics to optimize HCP engagement. Sponsored by:

COVID & Chronic Disease REBECCA COTTON Executive Director of Data Analytics and Meeting the Needs of Vulnerable Patients Intelligence, Cardinal Health Sonexus Pharma companies are in a unique position to support and educate Access and Patient at-risk populations through patient services hubs Support

n times of a public health crisis, the cracks in the system care visits4 as patients and clinical staff fear unnecessary exposure that leave many patients vulnerable can become even more to COVID-19 and even a lack of comfort with the current stan- apparent. While our country continues to grapple with the dard of telemedicine. As most non-critical care appointments uncertainty of COVID-19 and its implications on our cur- move to a virtual format5, elderly patients or those who have lim- Irent healthcare system, patients with chronic illnesses are at a ited access to technology may face additional barriers to care. particular risk of declining health. Pharmaceutical companies, The current requirements for social distancing are not only result- with the support of patient hubs, are well-positioned to identify ing in delays in diagnosis and subsequent treatment, but also dif- patients who are most at risk of experiencing a disruption in their ficulties with adherence to current therapies. therapy. With the proper technology and capabilities in place, Without the same level of interaction with their doctors and pharma companies can recognize potential barriers to care and other healthcare providers, some patients may be more likely to address the needs of their patient populations through additional fall off medical therapy if they’re experiencing challenges, such support and assistance offerings. as medication side effects.

SOCIAL AND ECONOMIC CHANGES ARE ANALYTICS CAN HELP IDENTIFY PUTTING MORE PATIENTS AT RISK PATIENTS MOST AT RISK The first step in meeting patient needs is to identify those who are Pharma companies may be in a unique position to support at-risk or may be at risk of falling off their medical therapy. It is important patients with chronic illnesses at this unusual time, particularly if to consider how the sudden changes of a public health crisis may they already have patient support programs in place with a patient impact patients’ lives and pres- services hub. Today, many patient hubs collect and aggregate vast ent new challenges or barriers sets of patient information, from to care. Our country is cur- the more traditional sources of rently in the midst of record- As patient hubs increasingly adopt enrollment and insurance data, high unemployment rates with to newer sources, including millions of people filing for AI technology, these tools may be patient call data. unemployment1, which has When analyzed properly, this resulted in many patients los- useful in helping pharma quickly information enables patient ing health insurance. The identify and address issues hubs to flag early warning signs growing income gap created when a patient is at risk of fall- by COVID-19 is expected to related to therapy management ing off therapy. leave each uninsured patient Performing electronic bene- with up to $74,310 in hospital fits verification can quickly iden- bills2 should they contract the tify patients who have experi- virus. While Medicaid offers an alternative option for many fac- enced changes in health insurance coverage. While this is typically ing unemployment, in states that did not expand Medicaid, 40% done during reverification season en masse, this technology could will likely remain uninsured.3 Patients on medications for chronic be used now to identify patients who may be in need of additional illnesses may face difficult decisions about whether to continue assistance due to the pandemic. medical therapy if their incomes have decreased or their health As patient hubs increasingly adopt artificial intelligence tech- coverage has changed. nology, these tools may be useful in helping pharma companies At the same time, patients may also face limited access to to quickly identify and address issues related to therapy manage- healthcare providers. Across the US, most healthcare facilities are ment. For example, natural language processing technology can seeing a decline in elective surgeries, procedures, and preventive be used to identify specific patient concerns or challenges by ana- WWW.PHARMEXEC.COM JUNE 2020 PHARMACEUTICAL EXECUTIVE Customer Engagement 33

lyzing recorded interactions between patients, payers, and clini- usually registered nurses (RNs) who work on behalf of pharma cians for key terms and adjacencies. In response to the pandemic, companies and can offer guidance to patients who have questions these tools have been adapted to capture coronavirus-related or concerns about their prescribed treatment. Clinical nurse edu- words and phrases to identify patients who are using language cators, available to patients by phone, can be an additional that indicates financial hardship or barriers to care. resource to reinforce the therapy instructions that patients have This data can be used to help drug manufacturers plan addi- received from their physicians, and to answer medication-related tional patient support and education tailored to the needs of their questions. Nurse educators can also alert recently unemployed patients. patients to the existence of manufacturers’ patient assistance programs for potential additional financial support or free drug pro- CONSIDER NEW AND EXPANDED gram options. These resources provide supplemental understand- PROGRAMS TO SUPPORT PATIENTS ing for patients undergoing therapy and have been shown to In addition to identifying patients who may be at risk, pharma increase adherence to treatment. organizations also have an opportunity to provide broader support at a time when more patients may need help accessing and LOOK TO THE FUTURE managing their chronic conditions. A few specific steps for con- As our country and healthcare system continue to evolve with sideration are as follows. the current public health crisis, pharma companies have an • Expand patient assistance programs. Many pharma unprecedented opportunity to support continuity of care for companies offer patient assistance programs (PAPs), which pro- patients with chronic diseases and help prevent disease regres- vide financial assistance to help qualifying patients get access to sion. The critical factors for making these patient programs effec- free or reduced-cost medications. Most PAP programs base eli- tive are having the data and analytics to identify the patients most gibility at least partially on income, with the majority of programs at risk, and the expertise and technology to deliver personalized requiring proof-of-income6 documents such as tax returns. As support to patients. Patient hubs are uniquely qualified to provide our country navigates through the largest “layoff spree” in its remote, patient-specific support to help patients from end to end history7, pharma sponsors of PAPs have an opportunity to re- through their medical treatment journeys. evaluate income verification processes and consider broadening the parameters to meet the needs of their changing patient pop- References ulation right now. In addition, drug companies may want to 1. Chaney, S., and Morath, E. (April 2, 2020). Record 6.6 Million Americans consider, for appropriate products, extending their dispenses of Sought Unemployment Benefits Last Week. https://www.wsj.com/articles/ free medicine from 30-day to 60- or 90-day to maintain greater another-3-1-million-americans-likely-sought-unemployment-benefits-last- continuity of therapy. week-11585819800 • Consider establishing a “bridge” program. Due to 2. The Projected Economic Impact of the COVID-19 Pandemic on the US Healthcare System. (March 25, 2020). https://s3.amazonaws.com/media2. the significant increase in unemployment since the start of the fairhealth.org/brief/asset/COVID-19 — The Projected Economic Impact of pandemic, a record number of patients may be finding themselves the COVID-19 Pandemic on the US Healthcare System.pdf without health insurance, at least for the short term. Interrup- 3. Garrett, B., and Gangopadhyaya, A. (May 4, 2020). How the COVID-19 tions in health coverage can often lead to disruptions in therapy, Recession Could Affect Health Insurance Coverage. https://www.rwjf.org/en/ particularly for patients taking higher-cost specialty medications. library/research/2020/05/how-the-covid-19-recession-could-affect-health- insurance-coverage.html?cid=xem_other_unpd_ini:quick strike_dte:20200504_ A “bridge” assistance program can help provide continuity of des:quick strike care and prevent disease regression by delivering temporary help 4. Norvell, K., and O’Donnell, J. (April 2, 2020). Thousands of US medical work- with the cost of medication during a gap in coverage. A bridge ers furloughed, laid off as routine patient visits drop during coronavirus pandemic. program may be particularly helpful for patients during the https://www.usatoday.com/story/news/health/2020/04/02/coronavirus-pandemic-jobs-us-health-care-workers-furloughed-laid-off/5102320002/ COVID-19 pandemic, as many people are likely to be rehired once businesses reopen. 5. Galewitz, P. (March 27, 2020). Telemedicine Surges, Fueled By Coronavirus Fears And Shift In Payment Rules. https://khn.org/news/telemedicine-surges- • Evaluate how clinical nurse educators can support fueled-by-coronavirus-fears-and-shift-in-payment-rules/ care. Some patients may be having fewer interactions with their 6. Choudhry, N. K., Mulcahy, A. W., Johnson, K., Weinberg, M., Colla, C. H., healthcare providers due to social distancing guidelines in place. Mainor, A. J., Starner, C. I. (May 1, 2009,). Drug Company–Sponsored Patient While there is no substitute for medical guidance from a physi- Assistance Programs: A Viable Safety Net? https://www.healthaffairs.org/ doi/10.1377/hlthaff.28.3.827 cian, a pharma-sponsored clinical nurse educator team working in a patient services hub may play a complementary role in sup- 7. Lambert, L. (April 16, 2020). How each industry is fueling the U.S. unemployment rate in one chart. https://fortune.com/2020/04/15/unemployment-rate- porting patients with management of therapy during this unusual claims-benefits-coronavirus-layoffs-hospitality-retail-transport-manufactur- time. Clinical nurse educators are trained medical professionals, ing-us/ WWW.PHARMEXEC.COM 34 34 Back Page PHARMACEUTICAL EXECUTIVE JUNE 2020

How to Thank a Physician Post-COVID-19 The appreciation is nice, but advancing their voice—and acting on DANIEL S. FITZGERALD is CEO and President of their experiences—is what HCPs want the most, reports find InCrowd

he novel coronavirus has shown physicians at their 1) Pharma leaders must listen closely to doctors. Hear finest. They are worthy of every bit of praise, respect, how they are coping with evolving pandemic challenges and how and thanks being given them now. Yet, physicians they are continuing to adapt to a future care delivery environment. could use something more powerful than thanks. To ensure their valuable feedback continues, the industry must TDoctors want to be heard and need us to act on their voice. align to meet physicians where they are now and will be tomorrow. InCrowd was early to capture and bring forward the authen- 2) We must adjust to new point-of-care realities. The tic voice of healthcare professionals (HCPs). Between January data suggests that telehealth is here to stay, with 77% of frontline- and May, the company performed six waves of COVID-19 phy- treating physicians in mid-April saying they expect telehealth use sician research, two among high-need US patients, and one with to remain after COVID-19, and 69% of high-needs patients nurses. In quantifying US physician perceptions, InCrowd reports reporting in May they are using telehealth services to meet with examined sentiment on the healthcare system’s readiness for the their physicians. Clinicians wearing PPE around patients (29%), pandemic and respondents’ personal experience with it, includ- increased phone and front-of-office triage, and one-swab and drive ing their thoughts on reopening. through testing will make the care process different. The pandemic is affecting physicians with greater intensity 3) We must rethink demands on their time. Respondents and personal risk, combined with a need to change their delivery to InCrowd’s COVID-19 reports emphasized the importance of and care models. Shortages of personal protective equipment conducting insights gathering in a non-intrusive way, with 97% (PPE) have meant that 70% of frontline-treating physicians in preferring short online mobile surveys that are suitable for on-the- March and 66% in April were concerned for their personal safety, go response. Given the virtual world of the past few months—par- while 78% were concerned for the safety of loved ones in both ticularly as doctors and patients transition to telehealth—expecta- months. Even now, one in 10 doctors report living separately from tions for technology to mitigate physicians’ demands will likely his or her family to ensure at-risk loved ones are protected from increase post-pandemic. exposure to illness. 4) We must support physicians seeking balance. It seems For many, rescheduled procedures and appointments have hit like only yesterday that the healthcare system was concerned over their business hard. More than 40% of frontline-treating physi- physician burnout—at nearly 80% among PCPs as of mid-2019. cians surveyed by InCrowd in both April and May indicate fear As physicians juggle harsh personal/family safety and business for the loss of their jobs. considerations against a duty to provide care, verbatim remarks Yet, despite this backdrop, 97% of the 263 frontline-treating suggest increased responsibilities at work due to resource, funding, physician respondents in March said they wanted to continue and staff shortages as acuity increases, may create even more chal- receiving and participating in market research microsurveys. lenges to achieving work/life balance. Fortunately, COVID-19 Respondents included emergency room and critical care special- data show 15% of frontline-treating physicians plan to spend more ists, primary care physicians (PCPs), and pediatricians—groups time with family and friends after the crisis subsides, the second- that arguably face the greatest time demands and stress from most cited change to their personal life, behind more hygiene and COVID-19. When asked why, at a time when so much was being handwashing. demanded of them, multiple responses reiterated one ER doc- Physician feedback is vital to the drug development and com- tor’s plaintive remark: “I want my voice heard.” mercialization process. By accommodating the physician’s new InCrowd’s COVID-19 physician data offers the four following normal, industry professionals can better assure an ongoing dia- calls to action for the pharmaceutical industry: logue with those most critical to their mission. Introducing

As a quarterly supplement to Urology Times®, the number one publication in Urology, Urologists in Cancer Care™ focusses on core content areas important to your brand. Surround your message with trends at the forefront of urology, like prostate, kidney, and bladder cancer, broken down by leaders in the field. Position your brand as a thought leader by aligning your message with respected key opinion leaders and expert commentary. Reach thousands of engaged urologists, and deliver strategic, integrated communications today!

For additional information on sponsorships through Urologists in Cancer Care™, UrologyTimes.com, and Urology Times®, contact:

Bill Markowitz Paul Barchitta Associate Publisher National Accounts Associate (732) 346-3083 (718) 354-6178 [email protected] [email protected] WE’RE ALWAYS BETTER TOGETHER.

Each day at MGM Resorts, we take the well-being of our guests seriously while creating a brighter tomorrow for all. We continue to be focused on what matters by embracing humanity and protecting our planet. Through generous contributions to the MGM Resorts Foundation and critical product donations, we have increased our support for those in need in our communities. Our Gold LEED Certified buildings, Stay Well™ rooms featuring the latest in wellness technology and our dedication to renewable energy put us at the forefront of sustainability. We can’t wait to make plans with you now for a bigger, better, more responsible tomorrow.

Partner with us at MGMResorts.com to plan the meetings and special events that impact lives for the better.