2016 Roundtable Discussion

Progress Toward Reauthorization of the Prescription Drug and Medical Device User Fee Programs to Benefit Older Adults

Overview

Every five years, the U.S. Food and Drug Administration (FDA) must seek reauthorization of its user fee programs. The core purpose of user fees is to fund direct review activities for drugs, medical devices, generic and biosimilar products. User fees enable the FDA to maintain appropriate staffing levels and processes across centers to ensure the timely evaluation of medical products. The reauthorization process begins with direct negations between the FDA and representatives from regulated industry. FDA meets in parallel with the patient and consumer stakeholders to seek additional input.

The current PDUFA V and MDUFA III programs will expire in September of 2017. FDA began negotiations with industry and consultation with the stakeholder community in September of 2015. The Alliance for Aging Research (Alliance) has been the only patient stakeholder from the aging community consistently advocating before the FDA for enhancements to the user fee programs that would improve the development of treatments and medical devices intended for use by people 65 and older. Major areas of focus for the PDUFA VI and MDUFA IV discussions include: administrative improvements to strengthen the agency’s workforce; enhancing initiatives that support integration of patient perspectives into medical product development and review; increasing transparency of benefit/risk determinations; increasing efficiency and predictability in pre-market approval pathways; and exploring the use of post-market data in regulatory decision-making.

Convened by the Alliance, this expert roundtable will provide an opportunity for leaders from FDA’s Center for Drug Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH) to reflect on progress that has been made to reauthorize the prescription drug and medical device user fee programs in advance of the 2017 deadline. Speakers will also share their thoughts on what PDUFA VI and MDUFA IV mean for the future of FDA’s workforce, key initiatives to promote regulatory science, and any specific impacts the reauthorization may have on innovations for older adult patients.

Susan Peschin, MHS (Moderator)

Susan Peschin, MHS, is president and CEO at the Alliance for Aging Research. The Alliance for Aging Research is dedicated to accelerating the pace of scientific discoveries and their application to vastly improve the universal human experience of aging and health. For more than 30 years, the Alliance has guided efforts to substantially increase funding and focus for aging at the NIH, FDA, AHRQ, and CDC; built influential coalitions to guide groundbreaking regulatory improvements for age-related diseases; and created award-winning, high-impact educational materials to improve the health and well-being of older adults and their family caregivers.

Since 2012, Ms. Peschin has been a driving force in the growth and success of the organization. Under her leadership, the Alliance has remained strong in these priorities and continued to build on its reputation as a forward-thinking public policy and health education organization. As president and CEO, she leads the strategic development and implementation of Alliance initiatives, as well as manages Board governance and organizational operations. As a thought leader on many aging-related issues, she has led the Alliance in efforts to: boost older adult immunization rates; increase NIH Alzheimer’s disease and aging research funding; raise awareness of geriatric cardiac issues; develop a senior patient and family caregiver network through a PCORI engagement grant; and co-organize a first-ever NIH geroscience summit. She participates in major industry and policy symposiums around the country each year, and has published opinion pieces in news outlets nationwide.

Ms. Peschin currently serves on the Board of Heart Valve Voice U.S. and the National Council on Patient Information and Education; on the World Economic Forum’s Council on Human Enhancement; on the National Advisory Council for the National Institute on Aging at the NIH; on the Steering Committee for the Coalition for Imaging & Bioengineering Research; and as a member of the Lifespan Domain Task Force for the Clinical & Translational Science Award program, funded by NCATS at the NIH.

Ms. Peschin earned a B.A. in Sociology from Brandeis University, and a M.H.S. degree in Health Policy from the Johns Hopkins University Bloomberg School of Public Health.

Janet Woodcock, MD (Panelist)

Janet Woodcock is the director of the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration (FDA). The center makes sure that safe and effective drugs are available to improve the health of people in the United States.

Dr. Woodcock and her center: evaluate prescription and over the counter drugs before they can be sold and oversee their testing in clinical trials; provide health care professionals and patients the information they need to use medicines wisely; ensure that drugs, both brand-name and generic, work correctly and that their health benefits outweigh their known risks; and take action against unapproved, contaminated, or fraudulent drugs that are marketed illegally.

“New drugs—and new uses for older drugs—save lives, reduce suffering, and improve the quality of life for millions of Americans,” says Dr. Woodcock. “I am continually challenged to make sure that FDA’s regulatory process remains the world’s gold standard for drug approval and safety.”

Dr. Woodcock has led many of FDA’s drug initiatives. She introduced the concept of risk management in 2000 as a new approach to drug safety. Since 2002, she has led the “Pharmaceutical Quality for the 21st Century Initiative,” FDA’s highly successful effort to modernize drug manufacturing and its regulation. In 2004, she introduced FDA’s “Critical Path” Initiative, which is designed to move medical discoveries from the laboratory to consumers more efficiently.

Most recently, Dr. Woodcock launched the “Safety First” and “Safe Use” initiatives designed to improve drug safety management within and outside FDA, respectively.

Dr. Woodcock previously served as FDA’s deputy commissioner and chief medical officer. She also led CDER as director from 1994–2005. Prior to joining CDER, Dr. Woodcock oversaw approval of the first biotechnology-based treatments for multiple sclerosis and cystic fibrosis in her position as director of the Office of Therapeutics Research and Review in FDA’s Center for Biologics Evaluation and Research (CBER).

Dr. Woodcock received her medical degree from Northwestern University Medical School, and her undergraduate degree from Bucknell University. She has held teaching appointments at Pennsylvania State University and the University of California at San Francisco. She joined FDA in 1986.

Jeff Shuren, MD, JD (Panelist)

Jeffrey E. Shuren became the director of the Center for Devices and Radiological Health at the Food and Drug Administration (FDA) in January 2010. He previously served as Acting Center Director, beginning in September 2009. The center is responsible for assuring the safety, effectiveness, and quality of medical devices; assuring the safety of radiation-emitting products (such as cell phones and microwave ovens); and fostering device innovation.

“Our center experts and programs help get safe and effective technology to patients and health care professionals on a daily basis,” says Dr. Shuren. “Rapid technological advances enable us to approve such innovations as a diagnostic test for the H1N1 influenza virus, an expandable prosthetic rib for children with abnormal growth conditions, and a test that can help detect ovarian cancer.”

Dr. Shuren received his B.S. and M.D. degrees from Northwestern University under its Honors Program in Medical Education. He completed his medical internship at Beth Israel Hospital in Boston, his neurology residency at Tufts New England Medical Center, and a fellowship in behavioral neurology and neuropsychology at the University of Florida. He received his J.D. from the University of Michigan.

Dr. Shuren has held various policy and planning positions within FDA from 1998 to 2009, including acting deputy commissioner for policy, planning, and budget; associate commissioner for policy and planning; special counsel to the principal deputy commissioner; assistant commissioner for policy; and medical officer in the Office of Policy.

Dr. Shuren has served in a leadership role at FDA or on behalf of the agency on numerous initiatives, including: reauthorization of the Medical Device User Fee Act, which dramatically shortens review times for device applications; creation of the Sentinel Initiative, which works toward a national electronic system for monitoring medical product safety; development of FDA’s Pandemic Influenza Preparedness Strategic Plan; development of FDA’s Counterfeit Drug Task Force Report; development of the Interagency Food Safety Working Report to the President; implementation of FDA provisions of the Medicare Prescription Drug Improvement and Modernization Act; and, development and implementation of the Interagency Import Safety Working Group’s Report to the President: Action Plan for Import Safety.

From 1999 to 2000, Dr. Shuren served as a detailee on Senator Edward Kennedy’s staff on the Senate Health, Education, Labor, and Pensions Committee. From 1998 to 2003, he also was a staff volunteer in the National Institutes of Health’s Cognitive Neuroscience Section where he supervised and designed clinical studies on human reasoning.

As director of the Division of Items and Devices, Coverage and Analysis Group at the Centers for Medicare and Medicaid Services, Dr. Shuren oversaw the development of Medicare national coverage determinations for drugs, biologics, and non-implantable devices.

Janet Jenkins-Showalter (Panelist)

Janet Jenkins-Showalter is Head of US Regulatory Policy at Genentech, a member of the Roche group, in Washington DC. She is responsible for leading the DC Pharmaceutical Development Regulatory (PDR) policy function, which she built when she joined Roche in 2003. In her current role, she is responsible for the PDUFA VI reauthorization program (Roche was a member of the PDUFA VI industry negotiating team and led the industry effort on Drug Development Tools) and addressing legislative changes, particularly 21st Century Cures and the Innovation Act. She directed a number of internal policy initiatives and worked with external stakeholders in activities that led to important changes in the way that new therapies for unmet medical need are approved, including the breakthrough therapy designation enacted under PDUFA V and the CDRH Expedited Approval Pathway. She works extensively with BIO and other external organizations and stakeholders on a variety of policy hot topics with the goal of ensuring that regulatory pathways are developed to keep pace with scientific advances. These topics include Targeted Therapies/Personalized and Precision Medicine, Patient Reported Outcomes, Companion Diagnostics, Pediatrics, Rare Diseases, Innovative Trial Design, and Real World Evidence.

Prior to this position, she served as the Director of the International Scientific Activities and Standards Staff, Office of International Programs, Office of the Commissioner, FDA, personally managing the ICH Program, with oversight responsibilities for other harmonization programs, including the veterinary medicine harmonization program (VICH), the device global harmonization program (GHTF), Codex Alimentarius, and a number of standards development programs (e.g., ANSI, AAMI, ASTM). She began her career at FDA’s Center for Devices and Radiological Health and was responsible for directing CDRH’s policy analysis, legislative, and Chief Engineer activities prior to serving in the Commissioner’s Office of International Programs in 1992. She received the FDA Award of Merit in 1991 and 1998, represented PhRMA on the ICH MedDRA Management Board, and was a Senior Executive Fellow at Harvard’s Kennedy School of Government.

Andrew Emmett (Pfizer)

Andrew J. Emmett is Senior Director for Pfizer’s FDA Liaison Office in Silver Spring, Maryland. Andrew serves as the primary point of contact between the company and the FDA on regulatory policy and intelligence issues in the Washington DC area and interfaces with trade associations and FDA stakeholder groups. In addition to deepening relationships with key stakeholders, Andrew develops Pfizer positioning and advances specific policy priorities to enhance the regulatory environment for biomedical innovation.

Prior to Pfizer, Andrew spent nearly 14 year with the Biotechnology Industry Organization (BIO) where he was Managing Director for Science and Regulatory Affairs. Andrew led BIO’s engagement in the 2007 and 2012 reauthorizations of the Prescription Drug User Fee Act (PDUFA) and implementation of related FDA modernization legislation, including the FDA Safety and Innovation Act of 2012 (FDASIA). As head of scientific and regulatory affairs for BIO, he coordinated BIO activities regarding drug and biologic research and development, pharmacovigilance and benefit/risk management, regulatory science, pediatrics, manufacturing, biosimilars, anti-counterfeiting, health information technology, and FDA funding.

Attended Cornell University and holds a Master’s of Public Health in healthcare policy from George Washington University. He earned his Regulatory Affairs Certification (RAC) through the Regulatory Affairs Professional Society (RAPS).