US FDA Commissioner Nominee on Fast Track with Nov. 20 Hearing

Vol. 81 / No. 45 November 11, 2019

nation of replacement in almost 15 years. US FDA Commissioner Nominee (See chart below.) Among the potential nominees considered were Ned Sharpless, then acting com- On Fast Track With Nov. 20 Hearing missioner and now back at NCI, and Brett M. NIELSEN HOBBS [email protected] Giroir, the Health and Human Services assistant secretary for health who is now tephen Hahn’s nomination to lead November, seven months after the pre- acting commissioner, as part of the Fed- the US Food and Drug Administra- vious confirmed commissioner, Scott eral Vacancies Reform Act shuffle that the S tion was a long time coming, but it Gottlieb, left the post. (Also see “Stephen administration performed on Friday. (Also appears that Senate leadership is hoping Hahn For US FDA Commissioner: Will Aca- see “Giroir Becomes Acting US FDA Commis- that his time as nominee will be pleasantly demic Experience Suffice For Confirma- sioner, Giving Agency Experienced Political short, with a hearing in the Health Com- tion?” p. 4.) Hand” - Pink Sheet, 1 Nov, 2019.) mittee now scheduled for 20 November. The lengthy process to pick Hahn result- Tapping the runner-up as the de facto Sen. Lamar Alexander, R-TN, an- ed in the longest gap between departure interim commissioner isn’t unusual (think nounced on 6 November that he was of a confirmed commissioner and nomi- CONTINUED ON PAGE 4 supporting the nomination. “I had an excellent meeting with Dr. Wait Times For New Commissioners Hahn and believe he is well-qualified Hahn was the longest gap between the resignation of the previous commissioner to lead the FDA. As an oncologist, Dr. and nomination in over a decade. Hahn knows firsthand the importance

of bringing new, lifesaving drugs and David Kessler 9.8 .53 months devices quickly through the regulatory process as well as the need for innova- Jane Henney 15.86 3.93 months

tion for patients with debilitating and Mark McClellan* 20.2 20.2 months chronic pain. As a successful chief executive, he will bring a guiding hand to an Lester Crawford 10.63 5.13 months agency tasked with protecting the pub- Andrew von Eschenbach 5.67 8.73 months lic’s health,” Alexander, who chairs the 1.73 health committee, said in a statement. Margaret Hamburg* 2.13 months “This is a crucial perspective at a time Robert Califf 5.37 5.3 months when FDA is implementing the 21st Scott Gottlieb* 1.7 Century Cures Act, regulating tobacco 1.7 months and vaping products, addressing the Stephen Hahn 7

opioid crisis, and protecting our nation’s 0 2 4 6 8 10 12 14 16 18 20 food supply.” Months Hahn, currently the chief medical executive at the MD Anderson Cancer Time Between Resignation of Previous Commissioner and Nomination Center, was nominated by President Time from Nomination to Confirmation Trump to be FDA commissioner on 1 Note: * indicates nomination following presidential inauguration

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exclusive online content inside: COVER US FDA Commissioner Nominee On Fast Track HIV PrEP Patent Dispute Escalates As HHS Files Suit Against Gilead With Nov. 20 Hearing https://pink.pharmaintelligence.informa.com/PS141154 Trump administration wants the HIV drug maker to license HHS US FDA patents related to pre-exposure prophylaxis. Lawsuit follows 5 Stephen Hahn For US FDA Commissioner: Gilead’s request in August for the USPTO’s PTAB to conduct an inter partes review of the four-year-old patents. Will Academic Experience Suffice For Confirmation? 7 US FDA Expands ‘Cures’ Hiring Authority To Fill Variety Rx Pricing, Rebating Should Be Back To Business Of Open Positions As Usual In 2020, PBMs Say https://pink.pharmaintelligence.informa.com/PS141153 Political focus of drug pricing debate has shifted away from UK NICE pharmacy benefit managers, CVS Health’s Merlo suggests. 9 Everyone Wants A Piece Of The UK’s NICE

Gene And Cell Therapies In Asia: New Korean Law DRUG REVIEWS To Change Landscape Of Cutting Edge Biologics https://pink.pharmaintelligence.informa.com/PS141155 10 EMA Accepts First Diabetes Drug Onto PRIME South Korea will undergo many changes as it gears up for a new 12 China Alzheimer’s Approval Raises Hope law on cutting edge regenerative medicine and biologics, which But Also Questions will be implemented next year. Until then, further discussions will be needed to set details of the rules that can step up the industry’s competitiveness and bring the country more in line SUPPLY CHAIN with global standards. 11 UK Adds MMR Vaccine To Export Ban List

Another Ebola Vaccine Among New Filings At EMA https://pink.pharmaintelligence.informa.com/PS141156 ADVISORY COMMITTEES The European Medicines Agency has received 11 new marketing 14 Recent And Upcoming FDA Advisory Committee Meetings authorization applications, including three for orphan products from Vertex, MYR Pharmaceuticals and AstraZeneca.

ONLINE ONLY!  join the conversation FDA PERFORMANCE We are tweeting, liking and sharing the latest industry TRACKER news and insights from our global team of editors and analysts — join us! Regularly updated information about new submissions, pending applications and FDA actions, online-only interactive content at your fingertips 24/7 at @PharmaPinksheet pink.pharmaintelligence.informa.com/product- reviews-and-approvals/fda-performance-tracker pink.pharmaintelligence.informa.com November 11, 2019 | Pink Sheet | 3 US FDA

Time To Senate Hearings For FDA Nominees CONTINUED FROM PAGE 1 Joshua Sharfstein or Lester Crawford) For the last three commissioners, time to hearing corresponded with time and putting Giroir at FDA could actually to confirmation. help speed Hahn’s nomination along. Democrats have expressed concern about Girior’s policy work on abortion 54 Margaret Hamburg and on reproductive issues, so they may 64 be disinclined to try to hold up Hahn’s confirmation since it would mean keep- 63 ing Girior at the head of the agency. Robert Califf 159 Hahn doesn’t appear to have any industry conflicts that have held up previous

26 nominees. (Also see “Hahn May Not Run Scott Gottlieb 56 Into Questions About Industry Ties Like His Predecessors” - Pink Sheet, 1 Nov, 2019.) But senators could always try to hold Stephen Hahn 19 up the nomination because of concerns they have about policy issues within the 0 20 40 60 80 100 120 140 160 FDA’s orbit, be they drug pricing, vaping, or CBD. So while a quickly scheduled Days from Nomination to Hearing Days from Nomination to Confirmation Scheduled hearing hearing is a good sign, it’s no guarantee of a smooth confirmation. Hamburg and Califf had the disadvantage of needing to go through the Senate Still, a long wait for a nominee fol- while it was controlled by the opposition party. lowed by a quickly scheduled hearing could signal a quick confirmation. Mark FDA Commissioner Confirmation Votes McClellan’s 2002 nomination was the longest waited for at 20.2 months, but Gottlieb got the fewest. the second-quickest time from nomination to confirmation, 22 days, with a David Kessler 100 hearing coming after just 12 days – while the Senate was controlled by Democrats. Jane Henney 100 (See top chart left.)

Mark McClellan 100 Quick doesn’t always mean pleasant, though. While McClellan passed the Sen- Lester Crawford 78 16 ate by voice vote, Scott Gottlieb, who had the third-quickest nomination at 56 days Andrew von Eschenbach 80 11 and got a hearing in 26 days, ended up with the fewest votes of any FDA nomi- Margaret Hamburg 100 nee ever, a tally that probably reflected

Robert Califf 89 4 resistance politics at the beginning of the Trump administration as much as any Scott Gottlieb 57 42 concerns with him in particular. (See bot-

0 10 20 30 40 50 60 70 80 90 100 tom chart left.)

Yes No Passed by Voice Vote Passed by Unanimous Consent Published online 6 November 2019

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4 | Pink Sheet | November 11, 2019 © Informa UK Ltd 2019 US FDA Stephen Hahn For US FDA Commissioner: Will Academic Experience Suffice For Confirmation? DERRICK GINGERY [email protected]

ne of Stephen Hahn’s best as- eral administrative experience. Kessler sets as the nominee for commis- served from November 1990 until late O sioner of the US Food and Drug February 1997. Administration may also be his biggest Hahn was a senior investigator at the cause for concern. NCI for about a year, but since July 1995 From an administrative perspective, Hahn he has practiced medicine in the private is a government neophyte. The chief medi- sector at the University of Pennsylvania cal executive of MD Anderson Cancer Cen- and MD Anderson Cancer Center. ter has never held a significant leadership Like Hahn, Kessler also came to FDA position at a government agency, let alone from an academic hospital setting. He the FDA. That makes him a government out- was the medical director at the Hospital sider, which Republicans in the Senate and of the Albert Einstein College of Medi- other circles may find valuable. cine prior to becoming the first Senate- However, the lack of knowledge of the confirmed commissioner. FDA and inner workings of the federal But Kessler also had contacts on Capitol government may raise questions among Hill, having been a consultant for the Senate Photo credit: University of Texas MD Anderson Cancer Center Cancer Anderson MD Texas University of credit: Photo Senators about his ability to run such a large Committee on Labor and Human Resources Stephen Hahn, an executive at MD bureaucracy, which could slow implementa- for four years. There he worked for then-US Anderson Cancer Center, was nominated tion of his agenda should he be confirmed. Sen. Orrin Hatch, R-Utah, who was ranking to be US FDA Commissioner. President Trump announced his inten- member of the committee handling the tion to nominate Hahn for commissioner nomination. (Also see “FDA Commissioner- on 1 November. Adm. Brett Giroir, HHS as- Designate Kessler May Face Senate Confir- icine may help him avoid questions about sistant secretary for health, was tapped by mation Hearing As Early As Week Of Oct. 15 If industry ties, which stakeholders have HHS Secretary Alex Azar to be acting FDA Sen. Hatch Has His Way; Kessler Brings Off-Job raised with his predecessors. commissioner. (See box for more coverage Training” - Pink Sheet, 15 Oct, 1990.) of the nomination.) Kessler said in an interview with the Pink MD ANDERSON EXPERIENCE Giroir will replace Norman Sharpless, Sheet that the academic medicine back- HELPFUL, BUT IT’S NOT FDA who became acting commis- ground, as well as his Hill and other Nancy Myers, a former special assistant and sioner after the resignation of experience, prepared him for the strategic advisor in the FDA Office of the Scott Gottlieb, the last confirmed FDA role and he was completely Commissioner who now heads Catalyst commissioner. (Also see “Gottlieb’s comfortable upon taking over. Healthcare Consulting, said in an interview Parting Words For US FDA: ‘Be CLICK “I had studied the agency for that the wide depth and bredth of the FDA’s Willing To Push’ For Major Policy Visit our website for a decade,” Kessler said. “I don’t purview will require some time to fully grasp. Changes” - Pink Sheet, 5 Apr, 2019.) related stories available think there was anything they In Hahn’s case, and to a certain extent only online. Sharpless will return to once threw at me where I said ‘excuse Sharpless’ case as well, the learning curve Giroir Becomes Acting again run the National Cancer US FDA Commissioner, me’ and had to go in the other may be longer than it was for former com- Institute. Giving Agency room and look it up.” missioner Scott Gottlieb, who at one point Experienced Political Hahn was a commander in Hand Kessler also said running a hos- was FDA deputy commissioner for medical the US Public Health Service for https://bit.ly/32sjkdB pital was good training for head- and scientific affairs before returning to Hahn May Not Run six years and during that time Into Questions About ing the FDA because both direct- the agency to take the top job. served one year as chief of the Industry Ties Like His ly impact people’s lives. Sharpless, who came to the agency af- Predecessors National Cancer Institute De- https://bit.ly/2WUfPvg “These are very complicated ter a stint as NCI director, indicated several partment of Prostate Cancer Gottlieb On Being institutions with thousands of times in speeches that he was surprised at Clinic. But he would be the first Senate-Confirmed employees,” he said. “You wake the depth and breadth of the FDA’s man- US FDA Commissioner: confirmed commissioner since It’s Certainly An up and are not quite sure what is date. (Also see “Sharpless Calls For A Nim- David Kessler to enter the po- Advantage going to happen today.” ble, Flexible, More Efficient US FDA” - Pink https://bit.ly/2NtlJR4 sition with no substantial fed- Hahn’s career in academic med- Sheet, 2 May, 2019.) pink.pharmaintelligence.informa.com November 11, 2019 | Pink Sheet | 5 US FDA

Stephen Hahn Donated To GOP And Dems... Myers said much of Hahn’s immediate duties will involve continuing implemen- DATE AMOUNT RECIPIENT tation of blueprints that already are set up and moving forward. She also indicated 12 October 2012 $206 Mitt Romney (R) the senior leaders at FDA are proficient at briefing new and acting commissioners. 25 August 2008 $250 American Society/Therapeutic Radiology However, Myers said that while Hahn 29 September 2008 $250 Republican National Committee (R) will have knowledgeable people around him, his lack of in-depth understanding of 29 September 2008 $250 John P. Murtha (D) some FDA issues may slow some activities. “I think you learn it over time,” she said. 15 December 2009 $500 Robert A. Brady (D) “When a center director caught Scott [Got- tlieb] in the hallway and needed a quick 13 July 2010 $500 American Society/Therapeutic Radiology decision, he could do it right then.” 21 November 2011 $500 American Society/Therapeutic Radiology Comparisons to Gottlieb may be unfair. He is considered among the best com- 20 October 2012 $500 Mitt Romney (R) missioners in recent memory, not only for the issues he had the agency tackle, 20 October 2012 $500 American Society/Therapeutic Radiology but for his ability to communicate them 12 September 2014 $500 American Society/Therapeutic Radiology to the public. (Also see “Gottlieb’s Greatest Strength As US FDA Commissioner? Stabil- 27 June 2015 $500 American Society for Radiation Oncology ity” - Pink Sheet, 27 Mar, 2019.) Attorney Mark Mansour, a partner at 24 September 2016 $500 American Society for Radiation Oncology Locke Lord, said Hahn’s experience at MD Anderson should help his confirmation. 27 January 2017 $500 American Society for Radiation Oncology The Houston-based center is a large and 21 March 2017 $1,000 New Pioneers PAC (R) influential institution in US health care. MD Anderson projected a $4.8bn bud- Total $6,456 get in 2018-2019 fiscal cycle and had more than 14,500 full-time-equivalent employees at the end of its 2019 third ...While Norman Sharpless Supported Only Dems quarter, according to hospital documents. That is somewhat less than the FDA’s FTE DATE AMOUNT RECIPIENT count, 17,607 in FY 2019, but both organi- 31 March 2008 $250 Kay R. Hagan (D) zations are similar in size. Salaries will not be similar. Hahn was 26 June 2008 $750 Kay R. Hagan (D) paid $933,600 by MD Anderson, according to data as of 30 August compiled 27 August 2008 $250 Larry Kissell (D) by the Texas Tribune. Gottlieb made 9 September 2008 $250 Barack Obama (D) $155,500 in fiscal year 2017, his first year as FDA commissioner. 29 September 2008 $1,000 Kay R. Hagan (D) HAHN THE GOP, AND 3 October 2008 $250 Larry Kissell (D) DEMOCRATIC, DONOR Hahn also likely built up some good will 26 April 2010 $250 Cal Cunningham (D) among Republican Senators, despite 5 August 2012 $500 Barack Obama (D) working largely outside of Washington, as a donor to GOP campaigns and other 2 September 2014 $2,000 Kay R. Hagan (D) groups. Sharpless, who had expressed in- terested in becoming the permanent com- 26 October 2014 $600 Kay R. Hagan (D) missioner, has donated to only Democrats, Total $6,100 which Republicans likely noticed. The Center for Responsive Politics’ Source: Center for Responsive Politics Open Secrets database OpenSecrets.org database of political do-

6 | Pink Sheet | November 11, 2019 © Informa UK Ltd 2019 US FDA nations listed more than $6,400 given by at the University of Pennsylvania. (See table.) In picking Hahn, the Administration Hahn to candidates and other organiza- Sharpless made multiple donations to Kay went against the recommendation of five tions between 2008 and 2017. Hagan’s successful campaign to become US former commissioners, as well as many Most of the money, $4,750, went to what Senator from North Carolina in 2008 and patient advocates and others. They wrote were classified as political action commit- her failed re-election bid in 2014. He also to the White House recently asking that tees, including the American Society for gave to President Obama’s campaign in Sharpless be nominated as the permanent Radiation Oncology and American Society 2008 and 2012. commissioner. (Also see “Former Commis- for Therapeutic Radiology and Oncology. Sharpless also gave to Cal Cunningham sioners Push For Sharpless Nomination, In 2017, Hahn also gave to New Pioneers in 2010, who was looking to run against Confirmation” - Pink Sheet, 4 Sep, 2019.) PAC, a political action committee affiliated incumbent US Sen. Richard Burr, R-NC, that Congress has many issues swirling at the with US Rep. Greg Walden, R-OR, who is year, but lost the Democratic primary. Burr moment, which may distract it from acting the ranking member on the House Energy retained the seat and remains a member on Hahn’s nomination, such as the federal and Commerce Committee. of the Senate Health, Education, Labor and budget, which remains under a continuing Hahn also gave money to the Republi- Pensions Committee, which handles FDA resolution that is set to expire soon, and can National Committee in 2008 and the commissioner confirmations. Sharpless also the potential impeachment of President presidential campaign of US Sen. Mitt gave to Larry Kissell in 2008, who ousted a Trump by the House. Romney, R-UT, in 2012. Republican incumbent for a North Carolina The hyper-partisan attitude in Congress But Hahn also supported Democratic can- US House of Representatives seat that year. today, further fueled by the 2020 presiden- didates. In 2008, he gave to the campaign of The donations likely damaged Sharpless’ tial election, also could lead to a hold on then US Rep. John Murtha, D-PA, who was prospects of gaining the commissioner the nomination on the Senate floor, which running for what turned out to be his final nomination, given that Republicans control would further delay proceedings, even if term in office. In 2009, Hahn gave to the the Senate. (Also see “US FDA Commissioner there is enough support for confirmation. campaign of US Rep. Robert Brady, D-PA. Transition: Sharpless Audition Is Ending – Will Both were made while Hahn was working He Get The Part?” - Pink Sheet, 5 Sep, 2019.) Published online 1 November 2019

US FDA Expands ‘Cures’ Hiring Authority To Fill Variety Of Open Positions

KATE RAWSON [email protected]

he US Food and Drug Administra- tion has made approximately 80 T appointments under the “21st Cen- tury Cures” hiring authority through an expanded use of the alternative pay system. FDA is now using the “Cures” hiring authority to fill a broader range of positions levels than originally envisioned when the law was enacted at the end of 2016. While the initial Cures appointments filled leadership positions within the Centers, the agency has now expanded its focus to include key technical positions in the review CDER has not been able to keep pace teams where recruitment and retention can be challenging, such as regulatory with hiring deadlines under PDUFA – health counsel and consumer safety offi- even as the “modernization” of the cers, FDA said. Office of New Drugs is opening up The expanded use of the “Cures” personnel authority comes as FDA is facing ongo- many new positions to fill. ing recruiting challenges. In particular, the pink.pharmaintelligence.informa.com November 11, 2019 | Pink Sheet | 7 US FDA

Center for Drug Evaluation and Research staffed” and said FDA would be aggres- outside the agency: CDER Deputy Direc- has not been able to keep pace with hir- sively hiring from inside and outside the tor for Operations Patrizia Cavazzoni and ing deadlines under the Prescription Drug agency to fill those positions. Ellen Flannery, deputy center director for User Fee Act – even as the “modernization” “You’re going to see a lot of recruiting policy in the Center for Devices and Ra- of the Office of New Drugs is opening up going on. We’ll be doing some heavy ex- diological Health. many new positions to fill. ternal recruiting, as well as bringing up Cavazzoni is seen as a potential suc- CDER did not meet its fiscal year 2018 promising members within the organiza- cessor to Woodcock, should she decide hiring goal of 45 employees until 22 June tion and putting them in positions of more to retire. (Also see “New CDER No. 2 May 2019, according to FDA data. The center responsibility,” Woodcock said. “It’ll be a Impact FDA’s Safety Monitoring Aspira- also missed its FY 2019 hiring goal of 60 mixture. But OND is very low on staff … tions” - Pink Sheet, 17 Apr, 2018.) employees, with 40 (67% to goal) hired as and we hope to use the Cures authority to of September 28, 2019. (A “hire” is defined rapidly fill in those gaps.” HIRING ‘PILOT’ CUTS TIME BY 70% as an individual who has been confirmed Alongside the “Cures” authority is a re- for a full-time position. While some hires A FOCUS ON EXTERNAL HIRES vamped hiring system. A CDER/CBER are recruited from outside, they can also be The 80 positions already filled under pilot program launched last year with a a current employee who is changing posi- “Cures” demonstrates that the hiring goal of reducing the amount of time it tions within the agency.) process is helping to fill vacancies, albeit takes to recruit and bring onboard new The revamped Office of New Drugs will perhaps not as fast as FDA would like. employees by removing bottlenecks add to the number of open positions, with the addition of two offices and eight review divisions. Most of the leadership posts will The 80 positions already filled under “Cures” be filled internally through promotions, so demonstrates that the hiring process is helping to fill the hiring crunch mostly will be in “back- filling” the review-level positions that open vacancies, albeit perhaps not as fast as FDA would like. up as a result. CDER Director Janet Woodcock spoke The agency continues to struggle fill- in the HR process. (Also see “US FDA Hir- to the hiring challenges during the Previ- ing certain positions in emerging areas, ing For Senior Staff Vacancies Boosted By sion Policy/Friends of Cancer Research Bio- such as gene therapy in the Center for Cures Act, Pilot Program – Gottlieb” - Pink pharma Congress on 7 October. Woodcock Biologics Evaluation and Research, and Sheet, 7 May, 2018.) acknowledged that OND is “very under- 3-D printing and software-based device The pilot was delayed in part due to a products in the Center for Devices and damaging US Office of Personnel Man- Radiological Health. agement report in December 2018 that The five-week partial government detailed multiple irregularities in the shutdown that end in January was also FDA’s hiring process. (Also see “US FDA a setback. (Also see “Cures Act Hiring Au- Moves To Fix Hiring Process Threatened LET’S GET thority Expansion Could Help Recruitment By Personnel Agency” - Pink Sheet, 21 Woes, US FDA’s Sharpless Says“ - Pink Oct, 2019.) Sheet, 19 Jun, 2019.) Under the “Reimagined Hiring” pilot, SOCIAL FDA says a priority will be using the FDA has reduced the timeline from open- We are tweeting, liking and “Cures” authority for external hires. One ing up a position to the “tentative offer” sharing the latest industry important caveat is that while the law stage to an average of 40 days, which the news and insights from our granted FDA the authority to bypass the agency says is more than a 70% reduc- global team of editors and traditional government pay scale and of- tion from baseline. To date in the hiring fer an annual salary of up to $400,000 for pilot, 213 offers have been made and 154 analysts, join us! certain positions, it does not come with have been accepted, FDA said. additional funding. Centers must make The hiring pilot, which is overseen their “Cures” hires within existing bud- by FDA Acting Associate Commissioner gets – and even then, the salaries cannot for Scientific and Clinical Recruitment be expected to compete with the private Melanie Keller, has also deployed a new sector. (Also see “The Freeze Thaws: US “talent tracker” that monitors positions FDA Allowed To Hire Staff For Cures, User through the hiring process. A report on @PharmaPinksheet Fee Activity” - Pink Sheet, 22 Mar, 2017.) the program is due in 2020. FDA’s first hires under the “Cures” authority were leadership positions from Published online 4 November 2019

8 | Pink Sheet | November 11, 2019 © Informa UK Ltd 2019 UK NICE Everyone Wants A Piece Of The UK’s NICE NEENA BRIZMOHUN [email protected]

ngland’s heath technology assessment (HTA) body, NICE, has relaunched its international division for sharing its expertise “The outcomes of the original division E with overseas government agencies and organizations after were widely recognised worldwide receiving a growing number of enquiries for advice from health and social care bodies abroad. by different international and NICE International, the not-for-profit division the HTA body first formed in 2008 and later partly transferred to the University of Lon- national organisations.” – NICE don’s Imperial College, has been fully reactivated to offer countries abroad a more comprehensive advisory service and intensive support with implementing evidence-based decision making. Since transferring a part of the division’s team that focused on supporting low- and middle-income countries to Imperial College in 2016, NICE continued to offer an international knowledge transfer service in the form of speaking engagements, seminars and support with using NICE content. It has been working with countries such as Denmark, India, Japan, and South Korea, and more recently with several countries in Latin America. Interest in and demand for a consultancy service provision has now prompted the relaunch of NICE International, a spokesperson for the HTA body told the Pink Sheet. FEES The international division aims to provide support in developing NICE International operates on a fee for service basis. It can obtain systems to: improve health and social care evidence-based decision funding directly from the client country and can also access fund- making; embed HTA to allocate resources in a cost effective, transpar- ing from other public and charitable sources. ent and equitable way; and improve the quality of care and reduce NICE said that clients can learn about its products, methods variation of access. and processes through its bespoke knowledge transfer seminars Noting that the development of clinical guidelines can be expen- hosted at its offices, in the client country or via web conference, sive and time consuming, NICE said the division would also work with or arrange for an international speaking engagement. “The oppor- clients to adapt guidelines for England for their health care systems. tunity to publish, translate or adapt our NICE content overseas is available using our NICE content re-use and adaptation services.” WIDE RECOGNITION For a more detailed engagement, NICE International is offer- “The outcomes of the original division were widely recognised ing consultancy services to provide “more intensive support with worldwide by different international and national organisations,” implementing evidence-based decision making. Services include according to spokesperson. technical and institutional capability training, support with imple- “The current division has been working and delivering collab- menting new methods and programmes and contextualising NICE orative work with different international organisations including guidelines to local settings.” countries such as Denmark, India, Japan, South Korea amongst oth- Regarding who will deal with the increase in requests for advice, ers, and has also had recent engagements with several countries NICE’s spokesperson said: “We have a dedicated team within the in Latin America. NICE is also collaborating in the FCO [Foreign and NICE Scientific Advice team, a well-established fee for service ad- Commonwealth Office] funded Better Health Programme Prosper- vice programme, but people from the entire institute will partici- ity Fund, which aims to increase life expectancy, improve productiv- pate in the delivery of services.” ity and deliver economic growth in 8 partner countries. NICE… will The team that had moved to Imperial College in 2016 did so contribute in the project based on its expertise in health technology because its work had evolved to focus on supporting low- and assessment, guidelines and standards development.” middle-income countries and it was expected to benefit from the The HTA body said it had been receiving a growing number of experience and knowledge of the Institute of Global Health In- enquiries from overseas health and social care bodies. novation based at the college. “We will still work with the team at The division has engaged with health and social care bodies Imperial College and will collaborate in different projects when op- from over 30 different countries, according to the spokesperson. portunities arise,” the spokesperson said. “They have been based in countries like China, Japan, Switzerland, Latin America countries and Indonesia, amongst others.” Published online 6 November 2019

pink.pharmaintelligence.informa.com November 11, 2019 | Pink Sheet | 9 REVIEW PATHWAYS EMA Accepts First Diabetes Drug Onto PRIME NEENA BRIZMOHUN [email protected]

rovention Bio has become the first company to get an inves- years in children and adults considered to be at high risk. tigational treatment for diabetes accepted onto the Europe- Commenting on the PRIME designation, the company said it P an Medicines Agency’s PRIME (priority medicines) scheme, was pleased the EMA had recognized “the transformative poten- which aims to get drugs for unmet medical needs to patients faster. tial of PRV-031.” The company’s anti-CD3 monoclonal antibody, PRV-031 (tepli- Provention has also received breakthrough therapy designa- zumab), is a potentially transformative treatment for type 1 diabe- tion (BTD) for the product from the US Food and Drug Admin- tes (T1D) that could prevent or delay the disease in individuals at istration; BTD is similar to PRIME. In September the company risk of developing the condition. suggested that it might be able to submit a biologics license ap- The product is one of three investigational treatments that plication for the product to the FDA in the fourth quarter of 2020 have recently won a place on the three-and-a-half-year-old PRIME for the at-risk indication. scheme. The other two are Bayer’s factor VIII gene therapy candi- date for treating hemophilia A, BAY2599023 (DTX201), and Milt- SECOND HEMOPHILIA A GENE THERAPY enyi Biotec’s MB-CART2019.1, a CAR T-cell therapy for relapsed and Bayer’s BAY2599023 involves a recombinant adeno-associated refractory diffuse large B-cell lymphoma (DLBCL). virus vector based on the AAV serotype hu37 containing a single stranded DNA genome encoding a form of human FVIII. To date, 60 products The gene therapy is being tested in a Phase I/II trial to de- termine the safety and tolerability of the factor VIII gene trans- have made it onto the PRIME scheme, fer treatment in patients with severe hemophilia A. It is being co- developed with Ultragenyx Pharmaceutical. and 190 have been rejected. BAY2599023 is the second gene therapy to get accepted on PRIME for the treatment of hemophilia A. BioMarin Pharmaceuti- cal received PRIME designation for Valrox (valoctocogene roxapar- vovec) in early 2017. BioMarin recently learnt that the CHMP had PRIME offers developers enhanced scientific and regulatory granted an accelerated assessment for its planned EU marketing support from the EMA to help optimize their development plans, authorization application (MAA), which is expected to be submit- as well as the likelihood of having their product reviewed under ted later this year. (Also see “EU Fast-Track Success For BioMarin’s Val- the EU’s accelerated assessment procedure when it is filed for rox But Not For Amarin’s Vascepa” - Pink Sheet, 25 Oct, 2019.) regulatory review. Acceptance on the scheme means that the applicant has met ANOTHER CAR-T FOR DLBCL PRIME’s tough entry criteria and managed to convince the EMA Miltenyi Biotec’s CAR T-cell therapy MB-CART2019.1 was granted that its product has the potential to benefit patients with an unmet PRIME designation for the treatment of patients with relapsed and medical need, based on early clinical data. refractory DLBCL after frontline therapy and who are ineligible for To date, 60 products have made it onto the scheme, and 190 autologous stem cell transplantation. have been rejected. Teplizumab, BAY2599023 and MB-CART2019.1 According to the ClinicalTrials.gov website, Miltenyi Biotec, were accepted onto PRIME during the latest monthly meeting of which is headquartered in Germany, is collaborating with ICON on a the EMA’s drug evaluation committee, the CHMP, which took place Phase I/II safety, dose finding and feasibility trial of MB-CART2019.1 on 14-17 October. Another three applications for PRIME were also in patients with relapsed or resistant CD20 and CD19 Positive B-cell considered at the meeting but they were rejected. non-Hodgkin lymphoma. MB-CART2019.1 is one of several treatments that have been ac- A FIRST FOR TYPE 1 DIABETES cepted onto PRIME for DLBCL. One of these products, Gilead Sci- Provention’s teplizumab is the first diabetes treatment to get ac- ences/Kite’s Yescarta (axicabtagene ciloleucel), has already gone cepted onto PRIME. Earlier this year the product also became the on to receive EU marketing authorization. first immune modulator to show a delay in the clinical onset of type As for the applications for PRIME that failed to make the grade 1 diabetes. (Also see “T1D Silver Bullet? Provention Bio Soars On Hopes in October, these were for investigational products for previously- Teplizumab Is Type 1 Diabetes Game Changer“ - Scrip, 10 Jun, 2019.) treated non-small cell lung cancer, for mild cognitive impairment Its acceptance onto PRIME was based on clinical data from due to Alzheimer’s disease (AD) and mild AD dementia, and for the Provention’s At-Risk study. The data showed that a single 14-day prevention of graft versus host disease. course of the product significantly delayed the onset of T1D, compared with placebo, by a clinically relevant median of at least two Published online 4 November 2019

10 | Pink Sheet | November 11, 2019 © Informa UK Ltd 2019 SUPPLY CHAIN

UK Adds MMR Vaccine To Export Ban List

IAN SCHOFIELD [email protected]

he UK government has added another four medicines to its list of products that cannot be parallel exported to the EU, T bringing the total to 31. The drugs added on 5 November are the measles/mumps/ rubella (MMR) vaccine, pneumococcal polysaccharide vaccine, alogliptin/metformin and misoprostol. Four other products – al- prostadil, beclometasone diproprionate, norethisterone and ranitidine – were placed on the list on 17 October. Parallel traders are unhappy with the government’s action in drawing up the list, saying it should instead be targeting what they claim are the real causes of shortages, such as drug manufacturing problems and quality concerns. According to the Department of Health and Social Care, the aim of the list, which was first published on 3 October, is to “stop some medicine wholesalers from taking advantage of the present circumstances by parallel exporting” and potentially causing or aggravating supply problems. (Also see “UK Bans Parallel Export Of 24 Drugs As Brexit Nears” - Pink Sheet, 3 Oct, 2019.) ject to a precautionary recall following the detection of possible A product will be added to the list if it is required to meet the needs contamination with N-nitrosodimethylamine (NDMA), an impurity of UK patients, is subject to or at risk of parallel export, and export of with genotoxic and carcinogenic potential. the drug is contributing, or might contribute, to a shortage in the UK. All UK stock of ranitidine has been quarantined and only very Distributors that do not abide by the new rules could incur sanc- limited supplies are available for patients in whom alternative tions imposed by the regulatory agency, the MHRA. These include treatments are not appropriate, according to a Supply Disruption immediate suspension of the distributor’s licence or the suspension Alert issued last month by the DHSC. of supply of certain products under the licence, or a 28-day notice The alert includes a table of alternative medicines, including the proposing to vary the licence to restrict or prevent export activity. proton pump inhibitors omeprazole, lansoprazole, pantoprazole, It has been suggested that the government could use the list to esomeprazole, rabeprazole, and the H2-antagonists nizatidine, fa- help mitigate supply problems caused by a possible no-deal Brexit motidine and cimetidine. and to prevent the selling-off of stockpiles built up by companies The MHRA is continuing to evaluate the situation alongside in advance of the Brexit deadline, although the DHSC has denied the European Medicines Agency, which recently produced a list this is the intention. of potential sources of nitrosamine contamination to help drug The DHSC said the MMR vaccine had been added to the list be- companies evaluate their products for the possible presence of the cause an increase in global demand for the product risked affect- impurity. (Also see “EMA Identifies Potential Sources Of Nitrosamine ing private patients and those who are not eligible to receive the Contamination” - Pink Sheet, 21 Oct, 2019.) vaccine under the National Health Service. “This amounts to less than 1% of the total number of MMR vaccines in the country,” the PARALLEL TRADERS NOT HAPPY department noted, adding that there is no supply issue affecting The EAEPC, the association that represents European distributors the NHS immunization program itself. involved in parallel trade, has attacked the UK initiative. Vaccines for non-NHS groups such as private patients are pur- The association agrees that placing temporary, transparent and chased at local level through wholesalers by private pharmacies objective restrictions based on EU defined criteria where genuine and others, the DHSC said. Wholesalers do not have access to na- shortages occur could be helpful in averting shortages. However, it tionally procured stock, which is held centrally by Public Health takes issue with what it calls “the complete failure” of the government England for the national immunization program, and can only par- to be transparent in its action and on the actual causes of shortages. allel export locally procured vaccines intended for the private mar- EAEPC secretary general Kaspar Ernest said the products on the ket and for use outside the national immunization programme, the UK list had not been exported “and therefore most obviously we department noted. “Doses of locally procured MMR vaccines are at cannot be the cause of the shortage.” He said the parallel trade sec- risk of being parallel exported.” tor had “found the level of transparency unsatisfactory.” Another product on the list, ranitidine (GlaxoSmithKline’s Zantac “Since the products on the list are actually not really big export and generic versions), is in extremely short supply after it was sub- products (most not exported at all), I think it is fair to say this is pink.pharmaintelligence.informa.com November 11, 2019 | Pink Sheet | 11 SUPPLY CHAIN DRUG REVIEWS mainly motivated by the need to show political action rather than a real belief it will have any impact whatsoever on supply,” Ernest told the Pink Sheet. He suggested the government should instead be targeting the role of pharmaceutical companies and what he claims is their failure to meet supply obligations. “Exports are a handy excuse, and ministers can show political action quickly, but the root causes are in no way addressed,” he said. “You need to look to France for more proper actions. France for example do not stop with a requirement for notifying shortages for manufacturers. They furthermore oblige them to make a prevention/response plan and to follow it. Economic sanctions may follow if these plans are not made/followed.” He said that shortage reporting requirements on manufacturers should be strengthened in the UK. “I would refer to the HMA[Heads of Medicines Agencies]/EMA guidance on detection and notification of shortages, including the definition in there. This guidance should be transposed into legislation.” The China Alzheimer’s much awaited guidance, which was published in the summer, includes a proposed template for notifying shortages (Also see Approval Raises Hope “Defining & Reporting Drug Shortages in the EU - New Guidance “ - Pink Sheet, 4 Jul, 2019.) But Also Questions “Exports are a handy excuse, BRIAN YANG [email protected] and ministers can show political ailed as the first novel drug for Alzheimer’s disease in 17 years, the approval of Green Valley Pharmaceutical’s action quickly, but the root causes H GV-971 in China initially had many celebrating, but the euphoria is proving short-lived. are in no way addressed.” A novel oligosaccharide derived from brown algae, GV-971 – Kasper Ernest, EAEPC (sodium oligomannate) gained approval for the mild-to-mod- erate form of the disease, announced the National Medical Products Administration (NMPA) on 2 November. The country’s regulatory agency granted the therapy a conditional approval, Practical action also needs to be taken at EU level, according saying it “improves cognitive function” in patients with this to Ernest. “I do believe that most member states on their own are form of Alzheimer’s. in a weak position to force transformations in the global supply Developed by China Maine University in Qingdao and li- chain of the pharmaceutical industry. It is adamantly clear that censed to Shanghai-based Green Valley Pharmaceutical, GV- the EU needs to act together on this point.” 971 is being hailed as a domestic innovation champion. The approval is based on a Phase III trial that enrolled 818 patients LEGAL ASPECTS across 34 sites in China. Bans on the export of selected products are permitted under Ar- In this program, GV-971 showed statistically significant im- ticle 81 of EU Directive 2001/83/EC if the aim is to ensure patients provement against the primary endpoint of ADAS (Alzheimer’s have the drugs they need. More general prohibitions, though, Disease Assessment Scale)-Cog12 (p<0.0001). The mean differ- can lead to trouble. ence between GV-971 versus placebo in ADAS-Cog12 Score at Last month, the Belgian Constitutional Court overturned a 36 weeks was 2.54, announced the Shanghai firm during the general ban on parallel exports that the government had in- 11th Clinical Trials on Alzheimer’s Disease Conference (CTAD), troduced as a way of preventing drug shortages. The court said held on 25 October in Barcelona, Spain. the ban contravened EU rules and had not achieved its objec- Published research show that the drug is thought to work tive because only a very small percentage of medicines currently by remodeling gut microbiota and to suppress phenylalanine unavailable had in fact been exported. (Also see “Belgian Court and isoleucine accumulation and related neuroinflammation. Overturns Parallel Export Ban” - Pink Sheet, 25 Oct, 2019.) Gut dysbiosis during the progression of the disease leads to the peripheral accumulation of phenylalanine and isoleucine, Published online 6 November 2019 stimulating the proliferation of T helper cells. Brain infiltration

12 | Pink Sheet | November 11, 2019 © Informa UK Ltd 2019 DRUG REVIEWS of these is associated with M1 microglia activation, contributing jectable traditional Chinese medicine with $700m in annual to the neuroinflammation associated with Alzheimer’s. sales. The company in the past gained notoriety for exaggerat- ing its the effects of its ganoderma lucidum-derived products, MIXED DATA, MORE NEEDED? although the company has said its committed to R&D to meet However, the company acknowledged that there was a non-sig- patient needs. nificant trend for improvement against another more elaborate Even in the US, the prospect for GV-971 to gain an approval is scale endpoint, CIBIC (Clinician’s Interview-Based Impression not totally out of reach. Given the large unmet need, Alzheim- of Change Plus Caregiver Input)-plus (p=0.059) and that there er’s may see more novel approvals, particularly as the Shanghai were no statistically significant differences in terms of ADCS- firm is planning to conduct a global Phase III study, said Spicer. ADL (Activities of Daily Living) or NPI (Neuropsychiatric Inven- tory- Questionnaire). BIG PHARMA FAILURES AN OPPORTUNITY? While medical experts were encouraged by the signs of Alzheimer’s and the neurodegenerative space in general have seen improved cognition, they cautioned that further studies are many large companies exit after late-stage failures while some needed to classify and confirm efficacy. “Based on its suggested smaller firms have thrived. mechanisms of action and promising cognitive effects, GV-971 could help diversify the portfolio of treatments for this terrible disease,” said Banner Alzheimer’s Institute executive director Eric Reiman. However, “Additional studies are needed to further clarify and confirm its promising biological and clinical effects,” he added. Developed by China Maine As conditions for the approval, the NMPA has required Green Valley to conduct continued studies. “NMPA requires the ap- University in Qingdao and licensed plicant to continue to study pharmacological mechanisms and to Shanghai-based Green Valley long-term efficacy and safety post-market, and to provide time- ly analysis of the oligosaccharide,” it stated. Pharmaceutical, GV-971 is being hailed

CONTRASTING TRIAL DESIGN, AND USE? as a domestic innovation champion. Given that the Chinese approval came just two weeks after US firm Biogen Inc. said it would revitalize its Alzheimer’s antibody therapy Just a week ago, US biotech giant Amgen Inc. announced that it aducanumab after reanalyzing data from the EMERGE study and would withdraw from most degenerative neurology R&D and then would seek US approval, many are wondering if the Chinese nod invested $2.7bn in China’s BeiGene Ltd. to ramp up its oncology for GV-971 may be an attempt to take a lead in the sector. presence. (Also see “Amgen Joins China Oncology Market Race With Analysts appear to disagree. “The conditional Chinese approval $2.7bn BeiGene Stake” - Scrip, 1 Nov, 2019.) is based on GV-971’s Phase III pivotal study results that were not Other big pharma firms from Pfizer Inc. to GlaxoSmithKline only significant, but also appear to be clinically meaningful, both PLC have also scaled back their investments in the space, a situ- of which have been nearly impossible criteria to meet in pivotal Al- ation that leaves lesser-known players in a market like China and zheimer’s disease studies,” noted Datamonitor Healthcare analyst smaller biotechs to move up. Pamela Spicer. Unlike oncology, neurodegenerative areas incl Alzheimer’s Unlike Biogen and aducanumab, Green Valley enrolled fewer pa- have fewer major players and less competition, allowing faster tients and had a shorter study time, which may lead to limited use, development and lower initial investment, noted Spicer. “The Al- noted the analyst. “Phase III trials for drugs that target the underly- zheimer’s drug development landscape may have shifted to ar- ing disease, like aducanumab, typically have one-to-two year treat- eas where there may be faster development timelines, and there- ment durations and target over 1,000 patients. In contrast, GV-971’s fore less initial investment…with smaller biotechs now leading pivotal Chinese trial randomized 818 patients with a nine-month the way in that arena.” treatment duration, signaling that labeling for this drug might be China also has some other advantages. It is facing a large aging limited to use as a symptomatic treatment rather than being able population and increasing numbers of dementia patients; Data- to slow the course of disease progression.” monitor estimates that Asia had the largest number of prevalent The EMERGE study enrolled 1,638 patients, double the figure in the cases in 2018 (55.2 million), and medical researchers are eager to GV-971 trial, and ran for 78 weeks compared to 36 weeks for GV-971. become primary investigators for clinical studies. But given GV-971‘s encouraging clinical results and safety pro- The GV-971 study researchers included Shifu Xiao of Shanghai file, the novel treatment will potentially make it to the world stage, Jiaotong University Hospital and Xinyu Zhang of Beijing Union although physician familiarity with existing therapies despite sub- Hospital, two leading hospitals in China. optimal tolerability could pose hurdles, added the analyst. Founded in 1997, Green Valley markets Salvianolate, an in- Published online 4 November 2019 pink.pharmaintelligence.informa.com November 11, 2019 | Pink Sheet | 13 ADVISORY COMMITTEES

Recent And Upcoming FDA Advisory Committee Meetings

TOPICS ADVISORY COMMITTEE DATE

Vaccines and Related Development of chikungunya vaccines Nov. 8 iological Products

Boehringer Ingelheim’s Jardiance (empagliflozin) as an adjunct to insulin therapy to Endocrinologic and Nov. 13 improve glycemic control in adults with type 1 diabetes mellitus Metabolic Drugs

Amarin’s Vascepa (icosapent ethyl) to reduce the risk of cardiovascular events, as an Endocrinologic and adjunct to statin therapy, in adults with elevated triglyceride levels (135 mg/dL or Nov. 14 Metabolic Drugs greater) and other risk factors for CV disease

Correvio International Sarl’s vernakalant for rapid conversion of recent onset atrial Cardiovascular and Dec. 10 fibrillation to sinus rhythm Renal Drugs

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