REGULATORY UPDATE LEGISLATION MANUFACTURING QUALITY Brexit: EMA Could Lose Up To Half Its US FDA Getting More Money Up Front FDA’s Revised Quality Metrics Program: Staff If It Has To Move, p. 10 Under Cures Bill Revisions, p. 5 Voluntary Now, Mandatory Later, p. 19 Pharma intelligence Pinkpink.pharmamedtechbi.comSheetVol. 78 / No. 49 December 5, 2016 informa costs, both Tom and Seema will play an in- Trump’s HHS, CMS Leadership Choices tegral role in leading that effort.” Price, a physician and current chairman of the House Budget Committee, has been Underscore Focus On ACA Reform a vocal critic of the ACA. He has sponsored CATHY KELLY [email protected] legislation (HR 2300) that would repeal the law completely and then establish a resident-elect Donald Trump’s choices series of programs designed to broaden to lead HHS and the Centers for Medi- insurance coverage, such as refundable tax Pcare and Medicaid Services demon- credits and high-risk insurance pools. The strate his focus on implementing alterna- legislation would also seek to lower costs tives to the Affordable Care Act – and not through medical liability reforms and fur- just repealing it. ther eliminating fraud and abuse in Medi- The Trump transition team announced care and Medicaid. Nov. 29 that Rep. Tom Price, R-Ga., will be Price’s bill is not generally expected nominated as HHS secretary and a health to serve as the template for a Trump-en- care policy consultant with ties to incom- dorsed attack on the ACA but it shows his ing Vice President Mike Pence, Seema Ver- interest and engagement in developing ma, will be named CMS administrator. alternatives to the law. Given Republican control of the Senate, Tom Price One likely starting point is expected to confirmation of the two appointments be legislation passed by both chambers seems assured. Sen. Orrin Hatch, R-Utah, of Congress through the budget reconcili- head of the Senate Finance Committee, ation process in December 2015. That bill which will hold hearings on the confirma- focuses on repealing key provisions of the tion, announced his support for the nomi- ACA related to the coverage expansion, nees in a Nov. 29 statement. HHS Secretary- such as federal subsidies and the individu- “Congressman Price has been selected al mandate. It would also eliminate the an- for this crucial HHS post because of his designee Price has nual market share fee paid by biopharma experience as a respected physician and manufacturers that was established by the deep understanding of the health law and strongly criticized law, a plus for industry. its flaws,” he said. “Verma’s strong health the CMS Innovation Price’s selection also signals a couple policy background will benefit the reform of other positive developments for drug effort at CMS, a mammoth agency whose Center and its firms. He has strongly criticized some responsibilities ballooned under President of the recent payment demonstration Obama ad his health law. As Republicans Medicare Part B projects developed by the CMS Center chart the course to repeal and replace the drug payment for Medicare and Medicaid Innovation, health law with patient-centered reforms particularly the Medicare Part B drug that increase access to care and reduce demonstration. CONTINUED ON PAGE 4 BROUGHT TO YOU BY THE EDITORS OF SCRIP REGULATORY AFFAIRS, THE RPM REPORT, TAN SHEET, GOLD SHEET, PINK SHEET DAILY AND PINK SHEET Maximize Your Reimbursement Potential The balance of power behind the prescribing decision is changing: payers are ever more in charge. That means that insight into how payers make decisions – how they evaluate drugs, one against another – will be crucial to any successful drug launch. 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See the payer perspective. 6 10 16 exclusive online content inside: COVER Trump’s HHS, CMS Leadership Choices Underscore UK To Ratify Unified Patent Court Deal Despite Focus On ACA Reform Uncertainty Over Its Future Participation http://bit.ly/2ghb7BN LEGISLATION The UK government has announced that it will proceed 5 US FDA Getting More Money Up Front Under Cures with preparations for ratifying Europe’s Unified Patent Court Bill Revisions agreement, despite uncertainty over whether the UK will be able 6 Cures Bill Authorizes ‘Intercenter Institutes,’ to remain part of the UPC system once it leaves the EU. But Will US FDA Create Them? 8 21st Century Cures Revisions Tell FDA To Highlight ‘Patient Gottlieb Joins HHS Transition Team; Will Next Stop Be FDA? Experience Data’ http://bit.ly/2gheng9 Former US FDA, CMS official has been among those thought to REGULATORY UPDATE be in the running for Trump’s FDA commissioner nomination. 10 Brexit: EMA Could Lose Up To Half Its Staff If It Has To Move 11 EMA’s PRIME to Speed Sage’s Plans For Postpartum Sunscreen Industry Asks FDA For Flexibility Despite Depression Guidances’ Rigidity 12 Japan’s PMDA Hits Markers On Path To Key http://bit.ly/2gOztpK Performance Targets FDA latest sunscreen guidances incorporate little industry 17 ‘Regulatory Risk’: A New Factor In Risk/Benefit Equation input and are met with frustration and hope among industry At US FDA? stakeholders. The PASS Coalition and the Natural Products Association weigh in on future cooperation between FDA and GENERIC DRUGS industry under SIA. 14 FDA’s ANDA Approvals Amicus Eyes Broad Future Label For Migalastat After CLINICAL TRIALS Huge Filing Setback 15 New French Clinical Trial Rules Focus on Phase I http://bit.ly/2g0PEPE 16 Second French Trial Tragedy Verdict: No Danger Signs US FDA recommends company start a new study of oral Fabry In Lower Dose Triallists disease drug, to be designed using the agency’s irritable bowel syndrome guidance for measuring efficacy in diarrhea. MANUFACTURING QUALITY 19 FDA’s Revised Quality Metrics Program: Voluntary Now, Podcast: The Price is Right – HHS Transition And Next Mandatory Later FDA Commissioner? http://bit.ly/2gJaSQp CONSUMER DRUGS Listen in for perspectives on the nomination of Tom Price as HHS 22 OTC Monograph Application Reviews Get Deadlines, secretary and other policy developments evolving since the US Not GRASE Flexibility elections. DRUG REVIEW PROFILE 25 Gilead’s Epclusa: FDA Sought To Push Efficacy Bar Higher Podcast: ACA Repeal And The Drug Industry In Hepatitis C http://bit.ly/2fP4oCB 28 Harvoni Approval Led To Narrowing Of Epclusa’s Listen in to this podcast on risk and opportunities for biopharma Breakthrough Status and possible scenarios for repeal of the US health care reform law and development of potential new provisions, once the new AdvISORY COMMITTEES Congress convenes and Donald Trump takes office as president. 30 Harvoni Approval Led To Narrowing Of Epclusa’s Breakthrough Status pink.pharmamedtechbi.com December 5, 2016 | Pink Sheet | 3 US ELECTION 2016 CONTINUED FROM COVER spent more than 10 years in Congress and experiment. Prospects that the Part B demo is also currently a member of the House would be implemented under his regime Ways and Means Committee and its Sub- now seem non-existent. committee on Health. Price has also spoken out in opposition of the Medicare Independent Payment Advisory CMS ADMINISTRATOR CHOICE Board, which was established by the ACA to Trump’s choice for BRINGS MEDIcaID EXPERTISE develop cost cutting proposals that could be Trump’s choice for CMS administrator, Seema Verma, is president and founder of fast-tracked through Congress. IPAB has long CMS administrator, SVC Consultants, a health care consultancy been in the cross-hairs for biopharma compa- Seema Verma, has based in Indianapolis, Indiana. She brings nies because it has been expected to focus its experience with Medicaid reforms in a cost cutting efforts on drug spending. experience with number of states to her new role, signaling Price’s selection does not follow the re- Medicaid reforms in a the Trump administration’s likely focus as it cent pattern of former state governors approaches entitlement reform. being named to head the department number of states. Verma is considered the architect of but it is not without precedent (see box). the Healthy Indiana Plan, the nation’s first Former President Ronald Reagan named consumer-directed Medicaid program, es- two former members of Congress to head tablished in 2007 under Governor Mitch HHS during his administration – Richard Daniels and extended under Governor Schweiker and Margaret Heckler. Price has Mike Pence. The program requires Med- icaid enrollees to contribute to their own healthcare expenses through a mecha- A HISTORY OF HHS SECRETARIES nism modeled on health savings accounts. She and her company have also devel- • Sylvia Burwell, serving from 2014 to the present under oped Medicaid reform programs in Iowa, President Obama; was previously director of the Office of Ohio and Kentucky and helped design Management and Budget. Tennessee’s Medicaid expansion proposal. Verma also served as the state of Indiana’s • Kathleen Sebelius served from 2009-2014 under President Obama; health reform lead following passage of was previously governor of Kansas. the ACA and worked with public health agencies and state insurers as they pre- • Mike Leavitt served from 2005-2009 under President George W. pared to implement the law.