Centerwatch Weekly, August 19, 2019

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CenterWatch August 19, 2019 Weekly Industry Briefs…2

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Drug & Device Pipeline News…7 Industry Seeks More Detail from FDA Guidance Nine drugs and devices have entered a new on Trial Diversity trial phase this week. By Colin Stoecker Roche/Genentech wrote, such as those JobWatch…8 he FDA’s draft guidance on broaden- with other conditions, unique racial ing eligibility criteria and increasing characteristics and varying age and diversity in clinical trials needs more body profile. T CLINICAL TRIAL RISK definition and depth, according to drugmak- In addition to sponsors, CROs and aca- AND PERFORMANCE ers, clinical trial organizations and patients demic research centers, more than half of MANAGEMENT SUMMIT responding to the agency’s call for com- the comments were from individuals, many ments on the document. representing the EndBrainCancer Initiative, A common theme in the 90 separate a patient advocacy organization. The draft comments on the guidance was the lack of guidance, Enhancing the Diversity of Clini- SEPT. 4–5, 2019 REGISTER TODAY clarity on how to determine a representative cal Trial Populations, was issued in June PHILADELPHIA participant sample. Several commenters (CenterWatch, June 10, 2019). asked the FDA to develop a statistical model The FDA needs to “more clearly eluci- NEW WHITE PAPERS AVAILABLE or metric to help sponsors design trials date the scientific reasons for its focus on An Actionable Approach An Actionable Approach to Decentralized Clinical Trials to and set eligibility criteria that result in an diversity,” the Brigham and Women’s Hos- Grow Enrollment and Retention to Decentralized adequate patient sample. pital’s Multi-Regional Clinical Trials Center Clinical Trials to Grow Enrollment and Retention Broadening eligibility could open tri- (MRCT) said. From Symphony Clinical Research als to more medically complex patients, see Industry Seeks on page 4 »

Melissa Hawking Symphony Clinical Research June 2019 LEARN MORE

Ask the Expert: Sub-Investigator Compensation centerwatch.com/whitepapers Answer: his monthly feature presents a variety of The Sunshine law in Florida requires that questions from clinical trial profession- 1) meetings of boards or commissions must als with answers from WCG Clinical’s ex- be open to the public; 2) reasonable notice T The CRC’s Guide to Coordinating pert staff. To ask a question of WCG’s experts, of such meetings must be given; and 3) min- The CRC’s Guide Clinical Research to Coordinating Clinical Research click here: https://bit.ly/2XB9F6R. utes of the meeting must be taken. There is Stay informed and be Question: no mention of payments to investigators or prepared to respond to industry changes. We are planning on using a sub-inves- sub-investigators. FOURTH EDITION

Sandra “SAM” Sather, MS, CCRC tigator to support a clinical trial at our site. However, under the Centers for Medi- ORDER TODAY What do we need to include in the contract care & Medicaid Services Open Payments to ensure that we are compliant with the Program, there is a national disclosure Florida Sunshine law and the Stark law? requirement by making the financial Should we explicitly state in the contract relationships between applicable manufac- May–June 2018 42 CE program information 43 Issues in research management 52 SMART IRB Learn about best practices 55 CE post-test that we are compensating the sub-investi- turers, such as pharmaceutical companies Emerging trends in clinical research: The need for change By John W. Mitchell, MS to e ectively manage and o understand the opportunity o ered don’t know what we don’t know.” Learning Objectives/Outcomes: by emerging trends in the clinical trial In recent issues, Research Practitioner has 1. Explain the need for change in clinical research. research sector, consider insight from explored individual key emerging trends in T 2. List key drivers of transformation in clinical trials. a business author and a businessman/politi- clinical research, including patient centricity, 3. Describe examples of clinical trial research trends in cian. In his groundbreaking book, Good eMobile advances, and precision medicine. action. to Great: Why Some Companies Make the As stand-alone trends, these innovations 4. Discuss why the clinical trial sector will make the execute clinical trials with Leap…And Others Don’t,” Jim Collins wrote: hold great promise and excitement. jump from trends to practice. “Good is the enemy of great. And that is But what insight is gained when all emerg- one of the key reasons why we have so little ing trends are considered in totality? What gator for services and that we are not paying and healthcare providers, available to the that becomes great. We don’t have great do these opportunities say about the eld of ese seem to be fair questions. In a Feb- schools, principally because we have good clinical research? As Rumsfeld alluded, what ruary 2017 CenterWatch article about survey schools. We don’t have great government, might we not know that we don’t know? Are results on the use of electronic-sourced principally because we have good govern- current advancements in clinical research, as data, Hugo Stephenson, executive chairman the Research Practitioner. ment.” Collins asserted, relevant enough to power of DrugDev and a physician investigator, In a 2002 Department of Defense brieng, (great) the sector beyond the status quo observed: “I’m surprised and disappointed Secretary Donald Rumsfeld noted that one (good), given the rapidly evolving medical there hasn’t been much improvement in the of the biggest threats to security was driven reality of interconnectivity, articial intel- entire clinical trial process, including the by factors that fell under the category of “we ligence (AI), and the expectation of patients? for subject referrals? What is a good com- public. Under the Open Payments pro- see Emerging trends on page 44 Caution for NIH's single IRB policy for multi-site research By Sue Coons, MA » Earn ANCC contact hours Learning Objectives/Outcomes: s of Jan. 25, 2018, research institu- It does not apply to career development, 1. List the details that local IRBs may know about their tions had to comply with the National research training, or fellowship awards. study populations as opposed to a single IRB. Institutes of Health’s (NIH’s) Final However, some institutions continue to 2. Describe NIH’s reasoning for going to a single IRB A policy for multi-site research. NIH Policy on the Use of a Single Institutional be concerned about their ability to adhere 3. Discuss the impact of using a single IRB on a study’s Review Board for Multi-Site Research is to the policy and the cost of doing so. IRB direct costs. policy established the expectation that a executives say it is wise to be cautious. Some 4. Explain the concern about NIH’s history of choosing pensation for a sub-investigator? — Staff at gram, you are required to report only the single institutional review board (sIRB) of institutions could spend millions of dollars the lowest-cost provider. » Maintain nursing certi cation record would be used in the ethical review to upgrade their IRB infrastructure, while of non-exempt human subjects research some IRBs already have many of the policy protocols funded by NIH that are carried out requirements in place. In addition, one IRB show that the agency could revise its policy at more than one site in the United States. executive worries that past NIH actions see Single IRB on page 49 research site see Ask the Expert on page 5 » © 2018 CenterWatch. Duplication or sharing of this publication is strictly prohibited. Volume 19, Number 3 » Subscriptions at $197 DOWNLOAD A SAMPLE Volume 23, Issue 31. © 2019 CenterWatch. All rights reserved. WCG | CWWeekly August 19, 2019 2 of 8 Industry Briefs

FDA Proposes Endpoints for IQVIA Patient Portal Provides from real-world evidence. By adding Compre- Gastroparesis Trials Real-World Trial Results hend’s Clinical Intelligence Platform, Saama The FDA has named five acceptable end- IQVIA’s new patient portal will give trial will increase its support of CROs. Under Saama, points for trials of gastroparesis treatments in participants a window into the clinical research Comprehend will partner with Rockville, a draft guidance released last week. process by giving them access to their lab Maryland-based CRO DP Clinical to deploy its The agency says that five core signs and results and other study-related information. centralized trial monitoring software tool. symptoms of the disease that prevents Participants in trials that use the Patient proper emptying of the stomach should be Portal are offered access when they are re- Signant Program Supports used as primary endpoints: nausea, vomit- cruited. Throughout the trial, they receive visit CRO-Sponsor Communication ing, fullness after a meal, early satiety and and medication reminders, disease education Signant Health’s new partner program abdominal pain. and travel support services. teams with CROs to help them improve com- Sponsors should provide a primary end- The Patient Portal is part of IQVIA’s suite of munication with and service to sponsors on all point definition and a method for measur- trial support tools but also can be used as a aspects of clinical trial management. ing each of the five signs and symptoms stand-alone solution. The program will provide support on described in the guidance. For example, the protocol design and optimization, data quality agency recommends that all signs be given a FDA Collaboration Pulls RWE in oversight, patient engagement, eConsent and rating of severity, except for vomiting, which Clinical Oncology Trials clinical supply management. Signant also will should be measured in terms of the number Syapse and the FDA’s Oncology Center for offer its partners exclusive commercial terms, of times it occurs over a 24-hour period. Excellence are collaborating on ways to better business development support, joint marketing, The trial’s primary efficacy assessment use real-world evidence (RWE) to support the clinical and scientific expertise, product training should be based on changes in patient- agency’s regulatory process. and dedicated resources for governance. reported signs and symptom scores. The collaboration will focus on regulatory The stomach’s emptying time should not questions about testing and treatment pat- Boehringer-Ingelheim Forms be used as a primary efficacy endpoint, the terns, dosing and safety, and outcomes, and Virtual Research Center with agency advises, “because changes in gastric will investigate multiple sources of RWE in addi- MD Anderson emptying time are not associated with the tion to electronic health records and registries. Boehringer-Ingelheim has partnered with changes in the clinically important signs and Syapse’s oncology researchers from its the University of Texas MD Anderson Cancer symptoms” of the disease; however, it may be Learning Health Network will participate in Center to conduct research on therapies for a used as a secondary endpoint. the effort. variety of cancers. The comment period closes September 21. Through the virtual research center, BI and Read the draft guidance here: https://bit. Saama Builds on Cloud-Based AI Anderson will share data and analysis on stud- ly/2TxnXjt. with Comprehend Acquisition ies of products in the pharma giant’s pipeline Data analytics company Saama Technolo- from the research stage through clinical trials. VirTrial Launches Training and gies has purchased cloud-based AI company The center will be staffed by investigators Certification Program Comprehend Systems to support its clinical from both partners. Telehealth platform provider VirTrial has analytics platform. The partnership will focus on inves- launched a program to train research sites to Saama has been using an AI-driven clinical tigation of BI’s lung and gastrointestinal conduct hybrid in-person clinical trials. data analytics platform to draw clinical insights cancer candidates. The platform — which offers video, text and email messaging — helps sites CenterWatch Weekly coordinate and conduct virtual visits as well (ISSN 1528-5731) as manage communication among trial sub- CenterWatch Main and Editorial Offices Leslie Ramsey Editorial Director jects, investigators and trial staff. Research 300 N. Washington St., Suite 200, Falls Church, VA 22046 Stephanie Akers Production [email protected] / [email protected] sites that complete VirTrial’s training will Russell Titsch Business Development Director Permission requests can be emailed to [email protected]. receive certification and be placed on a list © 2019 CenterWatch. All rights reserved. No part of this publication of “Virtual Trial Capable” sites that the com- may be distributed or reproduced in any form or by any means Advertising packages and reprints are available: without the express written consent of the publisher. Email [email protected] or call (703) 538-7651. pany will share with sponsors and CROs.

This feature highlights changes in clinical scientist in oncology biomarker develop- M2Gen research organizations’ personnel. ment at Genentech. M2Gen has announced the appointment of Helge Bastian as president and chief AmerisourceBergen Imara executive officer. Bastian was formerly vice Leslie Donato has been named executive Imara has appointed Eleanor Lisbon as vice president and general manager of synthetic vice president and chief strategy officer at Am- president of clinical development, Farah biology at Thermo Fisher Scientific. erisourceBergen. Donato was previously vice Natoli as president of program management, president of strategy at Bayer Pharmaceuticals. Dora Rau as vice president of quality assur- OneOme ance, Anita Treohan as vice president of de- Patrick McIntyre has been appointed chief Aperiomics velopment operations, and Frank Waligora executive officer at OneOme. McIntyre was Aperiomics has named Alexander Valencia as vice president of technical operations. previously president and founder of Portage chief clinical officer. Valencia previously Lisbon formerly served as the senior Health Insights. served as the molecular laboratory director medical director within the hematology at Cincinnati Children’s Hospital Medical and oncology division of the data sciences, Ritter Pharmaceuticals Center and PerkinElmer Genomics. safety and regulatory team at IQVIA. Natoli Anthony Lembo has been appointed to the served as the executive director of program medical advisory board at Ritter Pharmaceu- Arvinas and alliance leadership and humanitar- ticals. Lembo is a director of the GI mobility Ronald Peck has been appointed as the first ian aid program at Bioverativ. Rau served and functional bowel disorder center at Beth chief medical officer at Arvinas. Peck was as the vice president of global quality at Israel Deaconess Medical Center and a profes- most recently senior vice president of clini- Sucampo Pharmaceuticals. Treohan served sor of medicine at Harvard Medical School. cal research at Tesaro. as the senior director of portfolio and program management at Axcella Health. Springworks Theraputics AveXis Waligora previously served as the vice Springworks Theraputics has named Frank Avexis has named Page Bouchard chief sci- president of chemistry, manufacturing and Perier as chief financial officer. Perier most ence officer. Bouchard previously served as controls at Curis. recently served as the chief financial officer global head of preclinical safety for parent of Forest Laboratories. company Novartis Institutes. Immunovant Robert Zeldin has been appointed chief Strongbridge Biopharma Cerevel Theraputics medical officer at Immunovant. Zeldin was Strongbridge Biopharma has named Richard Cerevel Theraputics has named Orly Mishan previously the chief medical officer at Ac- Kollender as chief operating officer and as chief business officer. Mishan most recently celeron Pharma. Robert Lutz as chief financial officer. Kol- served as principal of Bain Capital Life Sciences. lender most recently served as principal and Isofol partner of Quaker Partners Management, a Curis Isofol has named Roger Tell chief medical of- healthcare investment firm, and Lutz worked Reinhard von Roemeling has been named ficer. Tell came from Aprea Therapeutics where in management at Shire Pharmecuticals. senior vice president of clinical development, he was vice president of clinical development. and Christine Guertin has been named vice Todos Medical president of regulatory affairs and quality iTeos Theraputics SA Todos Medical has named Paula Trzepacz assurance. Roemeling was previously global Matthew Call has been appointed chief chief medical officer of breakthrough diag- head of research and development at Huya operating officer at iTeos Theraputics SA. nostics. Trzepacz served previously as a senior Bioscience International, and Guertin was the Call was most recently chief operating of- medical fellow in neurosciences at Eli Lilly & Co. head of regulatory affairs at Synlogic and di- ficer at Endocyte. rector of regulatory affairs at Array BioPharma. Western Oncolytics Kite Pharma Helena Chaye has been named chief execu- Foundation Medicine Christi Shaw has been appointed chief tive officer of Western Oncolytics. Chaye was Priti Hedge has been named chief science executive officer at Kite Pharma. Shaw was most recently chief business officer and part officer at Foundation Medicine. Hedge was previously senior vice president of Eli Lilly & of the strategic planning and development most recently senior director and principal Co. and president of Lilly Bio-Medicines. team at SillaJen Biotherapeutics. © 2019 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW2331 WCG | CWWeekly August 19, 2019 4 of 8 Features

Industry Seeks PhRMA joined MRCT in calling for the continued from page 1 inclusion of real-world evidence (RWE) and Novartis’s comment sounded a similar real-world data (RWD) to better understand note, saying that “enrolling these diverse The FDA needs to “more effects on diverse populations beyond subjects in clinical trials before much is clearly elucidate the study groups. “PhRMA believes that RWE/ known about the drug could jeopardize scientific reasons for its RWD could potentially complement and patient safety.” support the findings of a clinical trial in the Several commenters raised the issue focus on diversity.” broader patient population and enhance of adding pediatric components to a trial. the diversity of patient population studies,” “Under what criteria would it be appropri- —Comment from Multi-Regional the group wrote. ate to include children in a confirmatory Clinical Trials Center of Brigham and MRCT also noted that the guidance phase 3 trial?” MRCT asked. “Should chil- Women’s Hospital mentions only drugs trials, pointing out dren be analyzed as a separate subgroup that the topic of diversity applies to medi- or as part of the overall population?” cal devices as well as other types of biologi- Clinical Research Pathways (CRP) cal products. lamented the fact that “there is no direct and minority groups. The guidance should Read the comments here: https://bit. mention of race and ethnicity” in the draft provide direction specific to involving ly/33DwIx1. guidance, leaving it to the industry to guess African Americans, Latinos and Asians, Read the draft guidance here: https:// what the FDA means by underrepresented CRP said. bit.ly/2Z0jr2n.

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Ask the Expert must be within fair market value. Some of continued from page 1 the considerations that help determine if names of principal investigators and not payments are considered fair market value sub-investigators. The report may include Under the Open Payments include: up to five principal investigators for each program, you are required to }} Is the payment usual and customary research payment reported. fees for similar services; report only the names of The Stark law prohibits physicians from }} Does the payment reflect the local referring patients to receive “designated principal investigators and not jurisdiction in which the services are health services” payable by Medicare or sub-investigators. performed; and Medicaid from entities with which the }} Are the fees defensible when judged physician or an immediate family mem- against other fees paid to investigators ber has a financial relationship, unless an working on the same study? exception applies. Financial relationships financial interests as stated above and does Please be advised that one of the critical include both ownership/investment inter- not make such referrals, he/she is likely to components in scrutinizing payments to ests and compensation arrangements. The be compliant. investigators and sub-investigators is the Stark law is a strict liability statute, which Unfortunately, we cannot comment on division of labor. Paying an investigator and means proof of specific intent to violate the compensation for this sub-investigator. sub-investigator the same amount for the the law is not required. Therefore, so long However, it should be noted that all pay- same services is not acceptable. — Marco as the sub-investigator does not have any ments for services related to clinical trials Capasso, Chief Legal Officer, WCG Clinical

Unique content, critical analysis & expert market research Subscribe to the industry’s leading global news source. September 2018 A CenterWatch Publication Volume 25, Issue 09 Start-up Costs Can Be an Uphill Slog in Need of Change

By John W Mitchell Download a sample issue! tients per study 10 years ago, and today the and wearables are used by the different rustration about clinical trial start-up number is less than three,” says Jeff King- sponsors. When each sponsor uses their costs is not unlike Sisyphus’InReview dilemma. sley, CEO at IACT Health. His company own networks and devices for patient-re-In this issue operates 13 locations in Georgia and a few ported outcomes, it adds to site workload www.centerwatch.com/promotions FA host of long-standing expense and 3-4 In Review inefficiency realities continues to create outside the state in a network of about 100 and time. Regulatory Update such uphill headwinds for the clinical trial clinical specialists. “The protocols are lon- “We have no ability to standardize tech- sector. According to someRegulatory sources, such ger, and there are more Update procedures per pa- nology,” Kingsley says “We have to do so5 Action Items problems even threaten the viability of the tient per day. So, you’re doing loads more many trials with so many sponsors, andPart 4: How to Identify Candidate Fraudulence Early inThe Recruitment Cycle work, but you’re only paid when you put they have their own decision-making… sector. Sites complain theyFDA incur Signs more Off over- on Treatment for through Therapy designation in the U.S. Its By Angela Roberts patients in trials. The averages speak for » In-depth analytical reports on key trends head costs driven by regulatoryRare, documen-Adrenal Gland Tumors license is held by ProgenicsOur industry Pharmaceuticals. suffers from adoptive pho- themselves — you put fewer patients in tri- Top Considerations for Using Digital tation, antiquated data collection and the bia (standardization between sponsors andHealth Devices in ClinicalTrials demands of precision medicineThe to FDA name has a approvedals today.” the injectable sites).” By Vicki Gashwiler offering insights for your strategies and practices According to Kingsley, Japanthe only Greenlights way to Parkinson’s Trial few — all without increaseddrug compensation Azedra for rare cancers of the adre- As an example, he cites the advantagesRisk-based of Quality Management: continue this financial model is to partici- from sponsors and CROs.nal A recentglands study — the first ever non-surgical In the first trial ofsites its adoptingkind, Kyoto electronic University platforms suchThe as New Normal pate in three times as many concurrent tri- By Brion Regan supports this concern. Researcherstherapy OK’d at the for these tumors. scientists have won eSource. approval Using from Japanesesuch a platform could save als. However, he says it’s far more efficient Tufts Center for the Study Azedraof Drug (iobenguaneDevel- I131) is a radiother- regulators to test adultbillions stem compared cells as a possible to the aggregate cost 10-11of Pipeline News » Key regulatory updates to keep organizations from an overhead standpoint to have one opment found that the studyapy start-updrug that phase attacks tumors with a high, treatment for Parkinson’smonitors disease. flying to sites to review sourceFDA Actions trial that places eight patients, rather than of five to six months for specificallyclinical trials targeted has dose. It’s designed to Induced pluriopotentdocuments. stem cells Clinically, (iPS) an electronic plat- three trials that place eight patients. remained unchanged for treat the past adults decade. and children (12 and older) are derived from skinform or bloodalso prevents cells and errors such as entering informed of the latest FDA initiatives, changes Also, at a time when technology is con- The study also concludedwith that inoperable CROs mak- locally advanced or metas- induced back into ana bloodembryonic-like pressure incorrectly plu- or performing quering inefficiencies in other industries, ing investments in technologytic cancers are getting called phenochromocytoma ripotent state that cana patient divide procedure into more out of order from the technology is compounding problems in and requirements trials done faster. None ofand this paraganglioma. bodes well stem cells or becometest any protocol. type of cell in the The CenterWatch Monthly the clinical trial sector. In each of the three ISSN 1556-3367 for smaller, independent players.Pheochromocytoma forms inside and body, leading to a potentially“We’re unlimitedtrying to convince the industry studies he cites above, it’s likely that three it’s a huge benefit, but the sites can’t afford “Sites are doing more workparaganglioma for [fewer] grows outside the adrenal source of any type of human cell needed Editorial Director LisaStein different electronic health records, tablets patients. We used to enrollgland(s). about eight pa- Production Renee Breau for therapeutic purposes. They’re consid-see Start-up Costs on page 6 » Subscriptions start at $399 Both tumor types release hormones that ered promising for regenerative research © 2018 CenterWatch, LLC. All rights reserved. can cause symptoms including high blood because they can become different hu- No part of this publication may be distributed or pressure, rapid heartbeat and anxiety. reproduced in any form or by any means without the Status Quo is Not a Viable Option:man cells and, also, avoid controversy sur- express written consent of the publisher. Permission University of Pennsylvania research- rounding stem cells from human embryos. requests can be obtained via fax at (617) 948-5101 or A Host of ersChallenges gave 68 patients in a trial Strainat least one ClinicalResearchers plan Trialto transplant SiteiPS cells Viabilityemailed at [email protected]. Single-user annual subscriptions start at $399. therapeutic dose of Azedra. The results: 25 into the brains of Parkinson’s patients in By John W Mitchell SUBSCRIBE TODAY! store.centerwatch.com [email protected] who receivedgish at least payments, one dose contract and viability,(617)the hope preferred they 948-5100 will help repair or replace For inquiries on multi-reader and corporate Research, which operates 19 trial and prima-subscription rates and article reprints: 32 percent of those who received two doses site status, interconnectivitydamaged and something nerve cells. ry This care issites the in first Canada. trial Tel: (617) 948-5100 linical trial site finances are a problem saw their blood pressureless tangible drop enough — a lack to of considerationof its kind to in use the iPS cells.According to recent data, 80 percent Email:of sites [email protected] that keeps getting bigger. While there’s cut their hypertensionrelationship. meds in half. Scientists say they’reprefer cautiously monthly optimis- payments — with 59 percentFor inquiriesin- regarding your subscription: agreement improvements are on the C “This is a true breakthrough.Payments, whichUntil to- have traditionallytic after testing ar- the dicating process thatin quarterlymonkeys, payments have a nega-CenterWatch Customer Service horizon — and at least one sponsor is work- Tel: (866) 219-3440, Fax: (617) 948-5101 day, there were no anti-tumorrived quarterly, therapies are often nowwho even showed later. improvementtive impact and on didn’t trials. de- Given a choice, they said © 2019 CenterWatch. Duplication or sharing of this publication is strictly prohibited. ing to shake things up — there’s also a lot of CWW2331Email: [email protected] available for patientsAnd with sites these are tumorshiring extra staffvelop to chase brain down tumors —they’d a much-feared choose studies po- that reimburse monthly. frustration about the slow pace of reinven- For inquiries regarding advertising: who were not candidatespayments— for surgery,” and medical said doctorstential to side keep effect. up “We learned very quickly that we had to tion. A host of longstanding challenges are Advertising Sales Representative principal investigatorwith Daniel the regulatory Pryma, an requirements, says Karri have a floating line-of-credit...[or] we wouldTel: (617) 948-5100, Email: [email protected] intensifying. These issues range from slug- associate professor ofVenn, Radiology president and of Ra- research at LMC Mann see FDA: Focus on Hardest to Site Viability on pageSend 7 address changes to: diation Oncology. CenterWatch Customer Service “This therapy© 2018 CenterWatch. not only controls Duplication the or sharingtu- of thisTreat publication in Cohort is strictly prohibited.Cancer Trials 10Winthrop Square, Fifth Floor, Boston, MA 02110 mors but also the debilitating symptoms CenterWatch Main and Editorial Ofﬁces: The FDA says sponsors considering Tel: (617) 948-5100 Fax: (617) 948-5101 caused by their excess hormone produc- adaptive studies for early phase cancer Email: [email protected] tion, meaning it provides dual benefit treatments should focus on patients with to patients,” added Pryma, also chief of no other options. Penn’s Nuclear Medicine and Clinical Mo- The agency notes that so-called expan- lecular Imaging. sion cohort trials — which start out rela- Reportedsideeffectsincludelowwhiteblood tively small but expand if a potential treat- cell and platelet counts, fatigue and anemia. ment shows promise--can be a way to get a The FDA gave Azedra an Orphan Drug treatment to market more quickly. designation, Fast Track status and Break- see Regulatory Update on page 4

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Company Drug/Device Medical Condition Status Sponsor Contact Coordination CPI-200 advanced tumors Phase 1 trial initiated enrolling 27 coordinationpharma.com Pharmaceuticals Inc. subjects Rimo Therapeutics RiMO-301 advanced tumors Phase 1 trial initiated enrolling 18 rimorx.com Inc. subjects CStone BLU-667 RET-altered non-small cell lung Phase 1 trial initiated cstonepharma.com Pharmaceuticals cancer (NSCLC), medullary thyroid cancer (MTC) and other advanced solid tumors Bio-Thera Solutions, BAT2506 rheumatoid arthritis, psoriatic Phase 1 trial initiated enrolling bio-thera.com Ltd. arthritis, ankylosing spondylitis (AS) 182 healthy volunteers and ulcerative colitis Mallinckrodt plc MNK-6106 hepatic (liver) cirrhosis and hepatic Phase 2a trial initiated enrolling mallinckrodt.com (L-ornithine encephalopathy (HE) 48 adult male and non-pregnant phenylacetate) female subjects with hepatic cirrhosis who have a history of at least two or more documented episodes of HE in the last 12 months – with one in the last six months – and who present with hyperammonemia at the time of screening Kedrion Biopharma 10 percent primary immunodeficiency Phase 3 trial initiated enrolling 50 kedrion.com intravenous diseases (PI) subjects at 16 sites in the U.S. immunoglobulin binx health binx io (first-of-kind, chlamydia (CT) and gonorrhea (NG) 510(k) clearance granted by the mybinxhealth.com highly sensitive and FDA specific, molecular point-of-care (POC) diagnostic testing platform) Regeneron EYLEA (aflibercept) multiple retinal diseases, Approval granted by the FDA regeneron.com Pharmaceuticals, Injection 2 mg, including wet age-related macular Inc. single-dose, degeneration and diabetic eye sterilized prefilled diseases syringe TB Alliance Pretomanid Tablets Extensively Drug-Resistant Approval granted by the FDA tballiance.org in combination with Tuberculosis (XDR-TB) and bedaquiline and treatment intolerant/non- linezolid responsive Multidrug-Resistant (MDR) TB

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Assistant Manager of Donor Services Clinical Research Scientist SEPTEMBER 27-29, 2019 Knoxville, TN San Francisco, CA Society of Clinical Research Associates 2019 Annual Vice President CMC Operations Clinical Research Coordinator - NIH Conference Galveston, TX Bethesda, MD San Antonio, TX For the 28th year, SOCRA will Contracts Advisor Clinical Study Coordinator Assistant I welcome clinical research profes- South San Francisco, CA Torrance, CA sionals from across the world. Scientific Database Manager - NIH Associate Clinical Laboratory Scientist I This three-day conference will Bethesda, MD Pittsburgh, PA offer current information and tools, best practices and training Patient Registration Specialist (Dental Clinical Lab Specialist to assure that you’re up-to-date Receptionist) St Louis, MO and compliant in your clinical Detroit, MI Clinical Lab Manager research practice. Chemist I San Leandro, CA Nashville, TN OCTOBER 23-25, 2019 Quality Systems Manager- Pharmaceuticals/ FDA Inspections Summit Associate Scientist Biotechnology Bethesda, MD Worcester, MA Torrance, CA Over the past 13 years thousands of industry professionals have [ VIEW ALL KELLY SERVICES JOBS ] attended the FDA Inspections Summit and benefited from the unmatched presentations and panel discussions led by FDA of- More Jobs ficials and industry experts.

Clinical Trial Project Coordinator Content Director OCTOBER 27-30, 2019 Pacira BioSciences WIRB-Copernicus Group (WCG) MAGI Clinical Research Parsippany, NJ Falls Church, VA Conference 2019 West Total Feasibility Associate Manager Clinical Project Manager Las Vegas, NV WIRB-Copernicus Group (WCG) WIRB-Copernicus Group (WCG) The conference includes 100+ ses- Myrtle Point, OR Bala Cynwyd, PA sions and workshops in six tracks that offer practical information Operations Coordinator Manager, Clinical Programming on clinical operations & project WIRB-Copernicus Group (WCG) WIRB-Copernicus Group (WCG) management, site management, Santee, CA Bala Cynwyd, PA contracts, budgets & billing, Project Specialist Director, Human Capital Management regulatory compliance and special WIRB-Copernicus Group (WCG) WIRB-Copernicus Group (WCG) topics. Earn 23.5+ CME, CNE, CLE, Bala Cynwyd, PA Munich, ND CCB and other credits.

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