Benefit-Risk Advisory Council
To guide FasterCures’ efforts to expand opportunities for patient perspectives to shape product development and influence regulatory decisions so that products patients value advance more rapidly from bench to bedside.
The nature of service on the Benefit Risk Advisory Council is advisory, voluntary and participatory. The Council has no policy-making authority. Members are selected for their expertise and membership is not related to sponsorship; participants receive no compensation. There is an expectation for active involvement and mutual benefit from service.
FasterCures Benefit-Risk Program
Assessing benefits and risks is core to medical product development, regulation, and healthcare decision-making. The tradeoffs between desired benefits and tolerable risks may look quite different whether you’re a patient, a physician, a regulator, or a drug/device developer. They also might change over time.
Recent legislation has opened a new dialogue between patients, pharmaceutical and device developers/manufacturers, and regulatory gatekeepers about expected benefits and tolerable risks. FasterCures is working to expand opportunities for patient perspectives to shape product development and influence regulatory decisions so that products patients value advance more rapidly from bench to bedside.
Marc Boutin, JD
National Health Council
Marc Boutin is the executive vice president and chief operating officer of the National Health Council (NHC), an organization that provides a united voice for the more than 133 million people with chronic diseases and disabilities and their family caregivers. In addition to overseeing financial management and operations at NHC, Marc builds consensus among member patient advocacy organizations to propose and support legislation and regulations that address the collective needs of patients and their family caregivers. He also provides guidance to patient organizations on corporate structure, government relations, fundraising, and outreach. He is a member of the International Alliance of Patients’ Organizations Governing Board, Community Health Charities Board of Directors, PCORI Advisory Panel on Patient Engagement, and the North America Advisory Board to the Drug Information Association.
“To understand patients you must truly engage them. And when people with chronic conditions are involved in a meaningful way throughout the drug development continuum, we greatly increase the probability of producing the kinds of drugs patients want and need.”
John Bridges, PhD
Johns Hopkins Bloomberg School of Public Health
John F P Bridges, PhD, associate professor within the Center for Health Services and Outcomes Research at the Johns Hopkins Bloomberg School of Public Health, is an international leader in the application of both qualitative and quantitative stated-preference methods, especially as they pertain to benefit-risk analysis. He has completed several seminal benefit-risk preference studies, especially in the area of rare diseases, and has played a leading role in the development of consensus-based standards for stated-preference methods. In recent years, John has advocated for patients and caregivers to have a greater role in medical research and for the incorporation of preference data in regulatory decisions.
“It is increasingly important to involve patients and caregivers in the assessment of the benefits and risks of medical technologies. While it is vital to involve patients and caregivers in decisions, we also need to empower them with scientific, and generalizable, data on treatment preferences. Stated-preference methods can be valuable tools in determining meaningful benefit, risk tolerance, and preference heterogeneity, and are vital in fostering patient-centered drug development.”
Andrew Emmett, MPH
Biotechnology Industry Organization (BIO)
Andrew J. Emmett is Managing Director for Science and Regulatory Affairs at the Biotechnology Industry Organization (BIO). In his role, Andrew develops and implements strategic BIO responses to scientific and regulatory issues that affect the ability of BIO's human healthcare focused companies to research and develop new medicines and biotechnology therapies, and to bring these products to market. Specifically, Andrew leads BIO’s efforts relating to the implementation of the Prescription Drug User Fee Act (PDUFA V) and issues relating to drug and biologic research and development, biotherapeutics manufacturing and quality, and post-market surveillance.
“For entrepreneurial biotechnology companies researching the next generation of modern medicines, the patient voice is crucial. A key element of the biomedical innovation ecosystem is a patient-centric and science-driven regulatory framework that carefully balances benefits and risks in the context of disease severity and unmet medical need.”
Andrea is the President and Chairman of LUNGevity and a member of the Board of Directors. In her role as President of LUNGevity, Andrea is responsible for setting and executing the strategic direction of the organization and its science programs. Andrea came to LUNGevity through the merger with Protect Your Lungs, an organization she and her family started to fund lung cancer research following her mother’s death from lung cancer in 2008.
Before the merger, Andrea founded and built Protect Your Lungs, an organization dedicated to funding research into the early detection of lung cancer. Andrea was instrumental in building PYL’s Scientific Advisory Board and establishing its funding process, both of which followed her to the new LUNGevity organization.
In her for-profit career, Andrea has a wealth of management experience. Andrea was the Vice President of Strategy and Growth of Decision Lens, Inc. a company she helped launch in January 2005. Prior to joining Decision Lens, Andrea held a variety of management positions at Johnson & Johnson, including Director of Investor Relations, Manager Corporate Mergers & Acquisitions, and Plant Controller. She also spent several years at McNeil Consumer Products, a J&J subsidiary, in marketing and mergers & acquisitions. Prior to her time at J&J, Andrea worked for Lehman Brothers and Coopers & Lybrand in New York City in both Mergers and Acquisitions and as a CPA.
Andrea received her BS in Economics from Wharton with concentrations in Accounting, Decision Sciences, and Finance. She received her MBA from Wharton with concentrations in Finance and Latin American Studies. She served on Washington, DC’s Kennedy Center National Committee of Performing Arts and on the Board of ARCS (Achievement Rewards for College Scientists) of Metro DC. She has also served on the Executive Committee of the Board of Directors for DC Metro Boys and Girls Club and has worked with the Ronald McDonald House and the Philadelphia Museum of Art.
Parent Project Muscular Dystrophy
Pat Furlong is the founding president and CEO of Parent Project Muscular Dystrophy (PPMD), the nation’s largest nonprofit organization focused solely on ending Duchenne muscular dystrophy (Duchenne). When doctors diagnosed sons Christopher and Patrick with Duchenne in 1984, Pat didn’t accept “there’s no hope and little help” as an answer. She immersed herself in Duchenne, working to understand its pathology, the extent of research investments and mechanisms for optimal care. Her sons lost their battle with Duchenne in their teenage years, but she continues to fight—in their honor and for all families affected by Duchenne. Pat is considered one of the foremost authorities on Duchenne in the world.
“Patient focused drug development validates the voice of the patient as critical to the review process. It acknowledges the need to gather input from patients and their caregivers in order to create a more complete assessment of the benefit risk equation, encouraging predictability and increased flexibility within the review process. The clock is ticking for patients who need and deserve access to promising therapies.”
Sascha Haverfield, PhD
Pharmaceutical Research and Manufacturers for America (PhRMA)
Sascha Haverfield is the Vice President for Scientific and Regulatory Affairs at PhRMA. He leads PhRMA's activities on global regulatory policy issues including the implementation and reauthorization of the Prescription Drug User Fee Act (PDUFA). He previously worked in the biopharmaceutical and biotechnology industry in drug discovery, translational medicine and regulatory affairs with a focus on global drug development issues. Sascha completed his undergraduate studies in biological sciences at the University of Marburg, Germany and received a doctorate in genetics and cell biology from the University of Oxford, UK.
“A thorough understanding and integration of the patient perspective on benefits and risks throughout the drug development and regulatory review processes have the potential to significantly advance the biopharmaceutical industry’s efforts to bring to patients innovative medicines that extend and improve the quality of their lives.”
Paul Huckle, PhD, MPharm, RPh
Dr. Paul Huckle is Chief Regulatory Officer and Senior Vice President of Global Regulatory Affairs and Global Quality and Compliance at GlaxoSmithKline. He is responsible for worldwide filing of new submissions, and support for existing licenses for the GSK portfolio. His role impacts all projects developed or licensed by GSK; and supports the acquisition and divestment of established products as part of the company diversification strategy. Before entering the regulatory field, Paul worked for ten years in pharmaceutical development with responsibility for leading the development of prescription and OTC products for both US and non-US markets. Prior to joining industry he was a Teaching Fellow at The School of Pharmacy, University of London. He holds an Honours degree in Pharmacy and a Ph.D. in Pharmaceutics from the University of London, and is a registered Pharmacist.
“The current focus by key stakeholders on patient focused drug development and benefit-risk assessment provides a real opportunity to bring important new medicines more swiftly to patients in need.”
Bennett Levitan, MD, PhD
Janssen R&D and Johnson & Johnson
Bennett Levitan, MD-PhD is Director, Epidemiology at Janssen R&D. He is responsible for enabling teams to develop benefit-risk assessments for FDA, EMA and other health authorities. He has over 20 years’ experience in decision analysis, modeling and simulation. Bennett co-led technical development of the PhRMA Benefit Risk Action Team (BRAT) Framework for drug benefit-risk assessment. He serves on several PhRMA committees that influence policy in this field and is a member of the Next Steps Working Group, the Centre for Innovation in Regulatory Science Benefit-risk Task Force, the ISPE Benefit Risk Assessment Special Interest Group and the Medical Device Innovation Consortium Patient Center Benefit/Risk Project Steering Committee. Bennett received his B.Sc. (Electrical Engineering) from Columbia University in New York and his M.D.-Ph.D. (Bioengineering) from the University of Pennsylvania.
“It is the patient whose health and life are on the line, and understanding how patients weigh the benefits and risks of medical treatments is crucial to providing patients with treatments they value. Patient-focused drug and device development provides an essential means to assess patient views and have these views influence medical benefit-risk assessment and regulatory review.”
Robert Metcalf, PhD
Eli Lilly & Co.
Robert Metcalf is Vice President, Global Regulatory Affairs - U.S. He is responsible for interactions with the FDA supporting new drug development and marketed products, including U.S. product labeling, advertising and promotion, and regulatory policy. Robert also has responsibility for global label management, global submission management, and global Chemistry, Manufacturing and Control for Lilly. He completed his Ph.D. in Pharmacology and Toxicology at Queen’s University in Canada. His previous roles at Lilly have included Global Project Management, Regulatory Affairs, Health Outcomes, and Quality in Canada, Project Management and Pharmaceutical Development in Japan, Executive Director -- Global Patient Safety, and Vice President -- Global Ethics and Compliance.
“Both the FDA and companies are more eager than ever to hear from patients and understand their views. I am excited about the promise of the patient-focused drug development initiative as another means to help ensure fully-informed benefit/risk assessments that will ultimately speed safe and effective medicines to patients in need through more predictable, balanced and timely regulatory decisions.”
Robert J. Meyer, MD
University of Virginia, School of Medicine
Robert Meyer, M.D., is a pulmonary physician who is currently the director of the Virginia Center for Translational and Regulatory Sciences at the University of Virginia’s School of Medicine, as well as Associate Professor of Public Health Sciences. Previously, Robert was head of Global Regulatory Strategy, Policy and Safety at Merck & Co., from 2008-2013. During that time, Robert chaired the Regulatory Affairs Coordinating Committee for Pharmaceutical Research and Manufacturers of America (PhRMA). Prior to Merck, he was the Director of the Office of Drug Evaluation II within the Center for Drugs at FDA (2002 – 2007).
“For FDA to do its job properly, it must have a robust understanding of how patients perceive the burden of their own diseases and how they would weigh alleviation of their disease’s impact vs. the potential risks of any new treatments. I am pleased to be a part of efforts to provide that understanding.”
Joseph S. Ross, MD, MHS
Yale University School of Medicine
Joseph S. Ross, MD, MHS, is an Assistant Professor of Medicine (General Medicine) and of Public Health (Health Policy and Management) at the Yale University School of Medicine, a member of the Center for Outcomes Research and Evaluation at the Yale-New Haven Hospital, and an Assistant Director of the Robert Wood Johnson Foundation’s Clinical Scholars program at Yale. He is an expert at performance measure development and on the translation of clinical research into practice, using health policy research methods to examine the use and delivery of higher quality care and to better understand issues related to pharmaceutical and medical device evidence development and post-market surveillance. Joe has published more than 130 articles in peer-reviewed journals and is currently an Associate Editor at JAMA Internal Medicine.
“Patient-centered benefit-risk assessments for pharmaceuticals, biologics, and medical devices will create opportunities for manufacturers to develop and physicians to communicate clear, actionable information to patients, allowing them to make fully informed decisions about whether or not to use these medical products, improving clinical care.”
National Organization for Rare Disorders (NORD)
Peter Saltonstall is President and Chief Executive Officer of the National Organization for Rare Disorders (NORD). He has over 30 years of healthcare experience in both for-profit and not-for-profit environments, as well as extensive federal and commercial market knowledge. Peter has held senior positions within a number of major academic medical centers and organizations, including Harvard's Brigham and Women's Hospital, Tufts-New England Medical Center and St. Elizabeth's Medical Center of Boston. Throughout his career, he has developed a broad understanding of complex healthcare issues and demonstrates continuing commitment to improving the healthcare system to benefit patients.
Joseph V. Selby, MD, MPH
Patient-Centered Outcomes Research Institute (PCORI)
Joe V. Selby, M.D., M.P.H., is the first Executive Director of the Patient-Centered Outcomes Research Institute (PCORI). A family physician, clinical epidemiologist and health services researcher, he has dedicated his career to patient care, clinical research and administration. Joe joined PCORI from Kaiser Permanente, Northern California. He has authored more than 220 peer-reviewed articles. Joe was elected to membership in the Institute of Medicine in 2009. He served as a commissioned officer in the Public Health Service with the National Health Services Corp from 1976-1983 and received the Commissioned Officer's Award in 1981.
“PCORI applauds this effort to bring the patient perspective to benefit-risk assessment. It fits squarely with our mission of building evidence in support of decision-making about prevention, diagnosis and treatment options that aligns with patients’ needs and preferences. One-sized approaches rarely fit all persons well. There is great room for improvement.”
Anthony Yanni, MD, MBA
Sanofi-Genzyme Global R&D
Anthony J. Yanni, MD, MBA currently serves as Head, Patient Value and Strategy for the Sanofi-Genzyme R&D Center. In this role, Anthony leads a team in the evaluation of early assets in the Sanofi-Genzyme portfolio highlighting medical value and patient unmet need from very early in the lifecycle of a potential product. He currently co-leads a Sanofi Initiative: “Patient Engagement in The R&D Process.” Previously, Dr. Yanni served as Senior Director, Medical Value and Strategy and as Director, Clinical Development at Sanofi Pasteur where he worked on vaccine development as well as evaluations of external products considered for acquisition. Prior to joining the Sanofi group, Anthony practiced clinical medicine for 12 years and served as Chief Medical Officer for a hospital system in Pennsylvania. He holds an MD degree from Hahnemann University School of Medicine and an MBA from The University of Massachusetts.
“Successful drug development will depend heavily on the methods developed by industry and industry partners to integrate patients into the process from very early in the developmental lifecycle. Understanding patient need and creating vehicles internally to make actionable this important input will be the critical step in developing products more in line with patient need.”