Confirmed Speaker Faculty for EA3, January 2020
Paul Aliu, PharmD Global Head Medical Governance, Chief Medical Office, Novartis Pharmaceuticals
Chris Austin, MD Director, National Center for Advancing Translational Sciences, NIH
Georges van Baelen, MScPh Founder and President of Arctiryon, Inc.
Behtash Bahador, MSc Associate Director, Center for Information and Study on Clinical Research Participation (CISCRP)
Amy Barone, MD Medical Officer, Oncology Center of Excellence, FDA
Naomi Lopez Bauman Director of Healthcare Policy, Goldwater Institute
Chris Beardmore CEO, Anova LLC
Stuart Bell, PhD Vice President, Consulting, Inceptua Medicines Access
Amar Bhat, PhD Interim Executive Director, Reagan-Udall Foundation, FDA
Marc Boutin Chief Executive Officer, National Health Council
Suanna Bruinooge, MPH Director of Research Strategy, CENTRA, ASCO
Connie Coulomb, MBA Managing Partner, Coulomb Strategy Consulting LLC
Anne Cropp, PharmD Chief Scientific Officer, Early Access Care, LLC
Jonathan Darrow, S.J.D. Faculty Member, Harvard Medical School
Khrystal K. Davis Founder & President of Texas Rare Alliance, Texas Newborn Screening Advisory Committee
David Farber, JD Senior Partner, King & Spalding
Anthony Fauci, MD Director, National Institute of Allergy and Infectious Diseases (NIAID) 1984-Present
Karen Frascello Director, Global Medical Affairs, Early Access Programs, Alnylam Pharmaceuticals
Pat Furlong Founding President and CEO, Parent Project Muscular Dystrophy (PPMD)
Christina Hartman, MPH Senior Director of Advocacy, The Assistance Fund
William Hoos, MBA, MS Chief Commercial Officer, xCures
Peter Barton Hutt, JD Senior Counsel, Covington & Burling LLP
Richard Klein Former Director, Patient Liaison Program, FDA
Michael Kurilla, MD, PhD Director, Clinical and Translational Science Awards (CTSA) Program, NCATS, NIH
John Lagus Managing Director of Business Development, Tanner Pharma Group
Jeff Leider President, X2, “Let Them Be Little” Foundation
Hilary Marston, MD, MPH Medical Officer, National Institute of Allergy and Infectious Diseases (NIAID), NIH
Alexander Natz Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
Edmund Pezalla, MD, MPH Former National Medical Director and VP, Aetna (2007 – 2016); CEO of Enlightenment BioConsult
Peter Pitts President, Center for Medicine in the Public Interest
Tobias Polak, MSc Department of Health Technology Assessment, Erasmus MC
Jess Rabourn, CFA Executive Producer, Expanded Access Summit | CEO, WideTrial, Inc.
David Schwicker Principal and Founder, ORPHA Strategy Consulting
Erika Segear, PhD, RAC Associate Director of Regulatory Affairs, Duke University School of Medicine
Andrew Shuman, MD, FACS Chief of Clinical Ethics Services, University of Michigan Medical School
Marjorie Speers, PhD Executive Director, Clinical Research Pathways
Douglas Sproule, MD Vice President, Spinal Muscular Atrophy Therapeutic Area Head, AveXis
Steve Usdin Senior Editor, BioCentury
Robert Walker, MD Director, Division of Clinical Development, Biomedical Advanced Research and Development Authority (BARDA), HHS
Kevin Weatherwax Managing Director, Michigan Institute for Clinical and Health Research, University of Michigan Health System
Janet Woodcock, MD Director, Center for Drug Evaluation and Research (CDER), FDA
(In Memory) Frank Young, MD, PhD Former FDA Commissioner (1984-1989)
He has worked in the pharmaceutical industry for over 15 years in multiple roles in drug development, medical affairs and program management covering therapeutic areas such as oncology, rheumatology, transplantation, infectious and tropical diseases. Prior to joining the pharmaceutical industry he trained and practiced as a clinical pharmacist.
He previously served as an industry representative on the WHO/Roll-Back Malaria (RBM) case management working group, managed alliances and partnership agreements (Public-Private & Private-Private) with multilateral organizations, NGOs, academic/research institutes; and participated in various round-table and stakeholder discussions on Global Health issues.
Chris Austin, MD
Christopher P. Austin has served as director of the National Center for Advancing Translational Sciences at the National Institutes of Health since 2012. Prior to this role, he was NCATS’ scientific director, focusing on translating basic science discoveries into new treatments and technologies to improve the efficiency of therapeutic/diagnostic development. He founded several initiatives, including the NIH Chemical Genomics Center, the Therapeutics for Rare and Neglected Diseases program, and the Toxicology in the 21st Century program.
Before joining NIH in 2002, he led genomic-based target discovery, pharmacogenomic, and neuropsychiatric drug-development programs at Merck. From 2016 to 2018, he served as chair of the International Rare Disease Research Consortium (IRDiRC); Dr. Austin is also a member of National Academy of Medicine.
He earned an A.B. from Princeton University, an M.D. from Harvard Medical School, and completed training in internal medicine and neurology at Massachusetts General Hospital.
Georges van Baelen, MScPh
He was a key person in the management and oversight of the OPTIME trial before becoming a Director of Cardiology at United Health, where he participated in publication planning and authoring of several articles on heart disease and its therapies. As Global Director of Pharmacovigilance at AXA, Mr. van Baelen built globally integrated Pharmacovigilance Systems in Europe, Latin America and Australia, while overseeing Global Medical Information Services for various biotechnology and pharmaceutical companies.
Mr. van Baelen is currently Founder and President of Arctiryon, Inc., headquartered in Chicago, USA, and travels extensively throughout Latin America, advising and assisting US and EU-based clients with the pharmaceutical regulations of Central and South American countries, as well as many European Union members. He resides part-time in Brazil and part-time in the United States.
Behtash Bahador, MSc
Since 2014, he has collaborated with a range of stakeholder groups to establish and implement patient- and public-centric initiatives across the life-cycle of drug and treatment development. This has included supporting the development of regulatory and cross-disciplinary best practice guidelines, operationalizing key elements of evidence-based public health programming into research engagement activities, and always keeping the needs of patients, participants and the public at the forefront of his work.
Amy Barone, MD
As a medical officer, she is responsible for the review and regulatory oversight of expanded access programs, numerous investigational new drug applications (INDs), new drug applications (NDAs), biologics license applications (BLAs), and single patient INDs.
She is very involved in the FDA effort to move the field of oncology drug development for rare diseases forward.
Naomi Lopez Bauman
She has twenty-five years’ experience in policy at both the federal and state levels and has previously served at organizations including the Pacific Research Institute and the Cato Institute. Lopez Bauman holds a B.A. in economics from Trinity University and an M.A. in government from The Johns Hopkins University.
While at the University of Maryland at Baltimore (UMAB), the University of California at Los Angeles (UCLA) and the United States Army Medical Research Institute of Infectious Disease (USAMRIID) he worked to improve human subject protection systems, served as an advisory to streamline research processes, drafted policies and procedures and developed training and education programs to improve systems. This included work with all stakeholders on issues related to ethical and regulatory application of International Conference on Harmonization Guidelines for Good Clinical Practice. While with USAMRIID, Mr. Beardmore authored a position paper proposing innovative approaches to developing investigational bio-warfare protective products for force health protection.
These experiences led Mr. Beardmore to recognize the serious need to improve systems and permit ethical research to be conducted more efficiently. In 2004 he took on the role of Chief Operating Officer and Co-Founder of Premiere Onocology, located in Santa Monica, CA, Scottsdale, AZ, and San Diego, CA; offering patients access to early phase oncology clinical trials in one of the first community treatment settings.
In 2009, Mr. Beardmore secured financing and launched Translational Research Management, LLC. The company created a 120 site community oncology network to speed development of innovative products for cancer patients. The network was connected to thousands of ancillary support providers and conducted hundreds of cancer clinical trials for the biopharmaceutical industry.
Mr. Beardmore is a frequent presenter on these issues, and was invited by the Institute of Medicine to participate in a workshop envisioning a transformed clinical trials enterprise in the United States.
Stuart Bell, PhD
Prior to Inceptua, Stuart set up the Consulting Dept. at Idis/Clinigen, pioneering the development of global corporate strategies on pre-approval access and developing the first pre-approval-specific EDC for real-world data collection. He has formerly held roles as: Principal, Real-World Evidence at IQVIA, Director, Informatics Initiative, UK Dept. of Health, Consultant to the European Association of Neuro-Oncology and Director of Communications for the European Cancer Organization.
Amar Bhat, PhD
Dr. Bhat started his career at the NIH’s Fogarty International Center and later joined the Office of the Secretary of the U.S. Department of Health and Human Services as Director of the Office of Asia and the Pacific. He subsequently left government and served in a variety of executive positions in the biopharmaceutical industry.
Dr. Bhat has a Ph.D. from the George Washington University in Public Policy, a Master’s in Public Policy from Duke University, and a Bachelor’s in Chemistry, also from Duke University.
Marc Boutin, JD
Marc M. Boutin, JD, is the Chief Executive Officer of the National Health Council. He has been a leading voice for greater patient involvement at every stage of the health care continuum, starting with the development of new drugs, to regulatory oversight of health care delivery, to shared decision-making at the point of care.
Under his leadership, the National Health Council has convened a broad range of stakeholders to create and effectively implement pragmatic strategies and public policy that address diverse issues, such as enhancing patient engagement, advancing the development of new treatments, and developing a better health delivery system to meet the needs of people with chronic conditions.
Boutin has a long history of board and committee service. Currently, he serves as a member of the Patient-Centered Outcomes Research Institute (PCORI) Patient Engagement Advisory Panel, FasterCures Benefit-Risk Advisory Council, and the Medical Device Innovation Consortium (MDIC) Patient-Centered Benefit-Risk Steering Committee.
Boutin has been actively involved in patient advocacy organization management, health advocacy, and both federal and state policy throughout his career. He is a founding member of the international Patient-Focused Medicine Development consortium and has served on the Governing Board of the International Alliance of Patients’ Organizations as a member and treasurer. He is also a former member of the Partnership to Fight Chronic Disease Board of Directors, the Humana Cares Clinical Advisory Board, the eHealth Initiative Leadership Council, Community Health Charities Board of Directors, Healthcare Systems Research Collaboratory, and the North America Advisory Board to the Drug Information Association.
Suanna Bruinooge, MPH
Prior to joining ASCO, Suanna worked for seven and a half years in the U.S. House of Representatives, working for Congresswoman Nancy Johnson (R-CT) and Congressman Vernon Ehlers (R-MI). Ms. Bruinooge earned a Master’s of Public Health in Health Policy at The George Washington University’s Milken Institute School of Public Health in 2015.
Connie Coulomb, MBA
With more than 20 years’ experience in biopharma, Mrs. Coulomb has held a variety of roles in the areas of strategy, business development, marketing, sales, market research and market access in large, medium and small biopharma companies, including Merck, Amgen, Biogen, Gilead, and Onyx.
Mrs. Coulomb has extensive international experience, having served as Executive Director of Commercial Operations at Amgen, overseeing a 35-country division that included Canada, Mexico, Latin America, the Middle East, Turkey, Australia and Africa. Previously at Onyx, she was responsible for the launch of Kyprolis in the Americas markets. At Merck & Co., she served as the commercial lead of the Oncology business for Puerto Rico, Central America and the Caribbean.
Connie has worked across several therapeutic areas including oncology, hematology, CNS, cardio-metabolic, osteoporosis, vaccines, autoimmune diseases, HIV, pain management, generics and biosimilars.
A native of Argentina, Connie received degrees in Business Administration and Accounting from the University of Buenos Aires, graduating with honors as Magna Cum Laude. She later completed her MBA at Stanford University.
Mrs. Coulomb is one of the co-authors of The Global Guide to Compassionate Use Programs.
Anne Cropp, PharmD
Prior to founding Early Access Care, Anne was Vice President at Pfizer Inc. Anne has extensive experience in pharmaceutical and biopharmaceutical drug development. She has built high-performing, diverse leadership teams supporting pipeline products from Phase 1 through NDA/MAA submission. Her expertise spans drugs, biologics and devices.
Anne is a globally recognized expert in Expanded Access US and ex-US and led the development and execution of the industry’s first physician-entry request portal for a global pharmaceutical company. As a life long member of the scientific research community, Anne’s passion has always been to bring solutions to advance patient care.
Anne began as a pharmacist meeting the needs of hospitalized patients. She certified as a clinical pharmacologist and completed two post-doctoral research fellowships in cardiovascular clinical pharmacology. While at the NHLBI of the NIH she first experienced Expanded Access. Motivated by a desire to help patients, she founded Early Access Care.
Jonathan Darrow, S.J.D.
After admission to the bar in 2001, Dr. Darrow worked on emerging company issues in the Silicon Valley and on pharmaceutical litigation matters at a large private firm in Washington, DC. He has since served on the faculties of four universities, and has supported various global heath projects at the Geneva campuses of the World Trade Organization, the World Health Organization, and the World Intellectual Property Organization. He is a co-author of Cyberlaw: Management & Entrepreneurship (2015) and The Legal and Ethical Environment of Business (2d ed. forthcoming 2018).
His scholarship on health policy and intellectual property has appeared in the British Medical Journal, the New England Journal of Medicine; the Journal of Law, Medicine & Ethics; the Stanford Technology Law Review; the Yale Journal of Health Policy Law & Ethics, and the Harvard Journal of Law & Technology, among many others.
Khrystal K. Davis
In May of 2016, Khrystal advocated alongside the FAST Movement (Families for the Acceleration of Spinal Muscular Atrophy Treatments) in a meeting with top FDA representatives for access to Spinraza, an SMA treatment in clinical trials at the time. Together with other FAST members, she asked the FDA to stop placebo trials, provide a means of access for the weakest SMA patients, accelerate the approval, and approve the treatment for all SMA patients regardless of age or type of SMA. An Expanded Access Program for SMA Type 1 patients commenced August 12, 2016, and the FDA approved Spinraza for children and adults with SMA on December 23, 2016.
Khrystal holds a Juris Doctorate from Stetson University College of Law and is certified in Clinical Trial Design and Interpretation by Johns Hopkins University and Drug Discovery by University of California San Diego.
David Farber, JD
A leading national expert on Medicare matters, he works regularly on Capitol Hill and with key regulatory agencies, particularly the Centers for Medicare & Medicaid Services and U.S. Food and Drug Administration, on legislative and regulatory advocacy and counseling matters.
His regulatory experience complements and augments the firm’s FDA regulatory, life sciences and healthcare teams, all of which are recognized as leading practices by Chambers USA.
David regularly speaks before national audiences on expanded access, health care reimbursement, secondary payer, and CMS and FDA regulatory matters.
Anthony Fauci, MD
Anthony S. Fauci, M.D. is director of the National Institute of Allergy and Infectious Diseases (NIAID) at the U.S. National Institutes of Health, where he oversees an extensive research portfolio focused on infectious and immune-mediated diseases. He serves as one of the key advisors to the White House and Department of Health and Human Services on global HIV/AIDS issues, and on initiatives to bolster medical and public health preparedness against emerging infectious disease threats.
He is the winner of numerous prestigious awards including the Presidential Medal of Freedom, the National Medal of Science and the Lasker Award for Public Service.
Karen has over 26 years of experience in the biopharma industry, in a variety of commercial and strategic consulting roles. She started her career as a pharmaceutical sales representative and was promoted into increasing levels of responsibility eventually having managerial oversight for primary care and specialty sales teams across the US for Pfizer and AstraZeneca.
Karen moved into the global early access area in 2010 first with Clinigen Group, then Caligor Coghlan Pharma Services. During this time, she was responsible for helping biotech and pharma companies establish strategies and implementation of early access programs for their innovative medicines, with a special focus on orphan drugs and oncology. In 2019, she joined Alnylam Pharmaceuticals to lead the strategy and operational execution of early access programs and compassionate use across their pipeline.
Karen graduated with honors and holds a BS in Biology from the University of Akron. She has been a contributor to several patient-facing publications which center around obtaining early access to medicines as well as a frequent presenter at rare disease industry conferences over the past 9 years.
Pat Furlong is the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest nonprofit organization in the United States solely focused on Duchenne muscular dystrophy (Duchenne). Its mission is to improve the treatment, quality of life, and long-term outlook for all individuals affected by Duchenne through research, advocacy, education, and compassion.
Pat graduated from Mt. St. Joseph College in Cincinnati, Ohio with a BS in Nursing. She attended Graduate School at Ohio State University. While attending Ohio State, Pat spent most of her time in the Medical Intensive Care Unit.
After marrying Dr. Tom Furlong, Pat ran the Renal Dialysis Unit and Patient Education Center at Akron General Hospital. In 1994, Pat, together with other parents of young men with Duchenne, founded PPMD to change the course of Duchenne and, ultimately, to find a cure. Today, Pat continues to lead the organization and is considered one of the foremost authorities on Duchenne in the world.
Christina Hartman, MPH
She is experienced in elevating the voices of patients, parents, scientists and clinicians to have a positive impact on health and nutrition policy. Christina worked with staff and member leadership at the American College of Cardiology to develop an agenda for improving cardiovascular health outcomes. At the Pew Charitable Trusts, she worked with a broad range of partners to advance legislative goals that incentivize the development of new antibiotic drugs.
Prior to that, she served as an analyst at the Centers for Disease Control and Prevention in Atlanta and in the Office of the Secretary for the U.S. Department of Health and Human Services (HHS) in Washington, D.C. Christina served as Project Officer for a Cooperative Agreement between HHS and the World Health Organization.
She has also worked in the food and beverage industry, including at the Beer Institute, where she pursued outcomes favorable to industry on a wide range of domestic and international public health and policy issues.
Christina was attracted to the opportunity to build and lead an advocacy team at the EveryLife Foundation in Washington, DC based on her own experience with the diagnostic odyssey with her youngest daughter, Charlotte.
William Hoos, MBA, MS
They assembled a boundary-pushing team with extensive experience in AI, health tech, and Pharma, and with the expertise, creativity, and persistence needed to solve the biggest problems in oncology. Mika Newton, a 20+ year veteran of the life sciences industry, is xCures’ CEO.
Through its precision oncology platform, xCures provides patients, and their physicians with the best individualized treatment options and services including access via reimbursement, clinical trials, expanded-access support and outcomes generation. By tightly integrating cancer research and clinical care, xCures enables bio-pharmas to slash the time and cost of developing drugs, physicians to make better treatment decisions, and patients to achieve superior outcomes.
Willy earned an MBA from Georgia Institute of Technology. He holds a BS Chemistry from Wake Forest University and MS Organic Chemistry from the University of California, Los Angeles.
Peter Barton Hutt, JD
Mr. Hutt served as Chief Counsel for the Food and Drug Administration during 1971- 1975. He is the co-author of the casebook used to teach Food and Drug Law throughout the country, and has published more than 175 book chapters and articles on food and drug law and health policy. Beginning in 1994 he has taught a full course on this subject each year during Winter Term at Harvard Law School and in 1998 he taught the same course during Spring Term at Stanford Law School.
Mr. Hutt has been a member of the Institute of Medicine (now the National Academy of Medicine) since it was founded in 1971. He serves on academic, philanthropic, and venture capital advisory boards, and the boards of startup biotechnology companies. He served on the Working Group on Innovation in Drug Development and Evaluation of President Obama’s Council of Advisors on Science Technology, the Panel on the Administrative Restructuring of the National Institutes of Health, the Working Group to Review Regulatory Activities Within the Division of AIDS of the National Institute of Allergy and Infectious Diseases, and the FDA Science Board Subcommittee to review the agency’s science needs.
Mr. Klein has worked in various capacities at FDA providing him with a well-rounded understanding of the regulatory issues that affect patients. He participated in the development of revised expanded access regulations and guidelines, and led the creation of the FDA expanded access website, played an active role in the development of the streamlined application for individual patient access, the exemption from full board IRB review for Individual Patient IND expanded access, and the Expanded Access Navigator. He also worked to create an expanded access full-board IRB waiver for Individual INDs to facilitate and speed the review process.
Prior to working in patient engagement, he helped to develop policies and regulations for the protection of human research subjects, and provided guidance for institutional review boards (IRBs).
Michael Kurilla, MD, PhD
Director of the Division of Clinical Innovation, NCATS-NIH
Michael Kurilla is the director of the Division of Clinical Innovation at NCATS (National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)). In this capacity, he oversees the Clinical and Translational Science Awards (CTSA) Program, which supports innovative solutions to advance the efficiency, quality and impact of translational science, with the ultimate goal of getting more treatments to more patients more quickly.
Prior to joining NCATS, Kurilla served as the director of the Office of Biodefense Research Resources and Translational Research within the National Institute of Allergy and Infectious Diseases (NIAID), where he focused on translational efforts toward infectious disease product development, including vaccines, therapeutics and diagnostics, with emphasis on biodefense and emerging infectious disease threats.
Prior to joining NIAID in 2003, Kurilla was an associate director for infectious diseases at Wyeth. He also worked in antimicrobials at DuPont and on clinical microbiology and molecular pathology at the University of Virginia Health Sciences Center.
Kurilla received his M.D. and his Ph.D. in microbiology and immunology from Duke University. He was a postdoctoral research fellow at Harvard Medical School and completed a residency in pathology at Brigham and Women’s Hospital. He received a B.S. in chemistry from the California Institute of Technology.
Before joining Tanner Pharma, John was most recently the Head of Managed Access at Clinigen Group. Over the past 13 years, he has had extensive experience with design, implementation and execution of over 200 global access programs spanning small to large companies, and a wide array of therapeutic areas including oncology, hematology, infectious diseases, and rare diseases.
John originally began his career in pharma as a statistician at 3M Pharmaceuticals, and then joined Orphan Medical in 2004 to oversee the development and FDA approval of Antizol. He joined Idis in 2006 to lead expansion of their pharma service business in the US. John has a BA in mathematics from St Olaf College, and an MS in statistics from Iowa State University.
Mr. Leider has been on numerous television segments and countless newspaper articles with the hopes of educating people about the Rare Disease community. Visiting the NJ State Capital and Washington, DC, Jeff has made strides in the MPS World. With his support, The Let Them Be Little Act, providing testing of MPSI and MPSII in newborns, was signed into NJ law.
Mr. Leider was integral in persuading the FDA and Shire Pharmaceuticals to open a clinical trial, giving nine MPSII children a chance to receive experimental drugs to halt disease progression. After implementation, Jeff became a patient representative for the FDA.
Jeff has been a guest speaker at the Capital and continues to moderate conferences throughout the country. He serves as Board of Director of Rare New Jersey, The Hunters Syndrome Research Coalition, member of MPS Society and many local organizations. In 2014, Mr. Leider was awarded The Abby for Advocacy-State Level at the Rare Voice Awards in Washington, DC.
Hilary Marston, MD, MPH
From 2008 to 2013, he was Head of the Brussels Office of Bundesverband der Pharmazeutischen Industrie e.V. (BPI). Before, he has been a lawyer at Sträter Law Firm in Germany with a special focus on managed entry agreements and licensing of pharmaceuticals.
Dr. Natz has also worked in the field of competition law with the European Commission and in the pharmaceutical industry. As a research assistant at Duke University (USA) he has dealt with international pharmaceutical law. His doctorate was supervised by the former judge at the European Court of Justice, Prof. Dr. Dr. Ulrich Everling.
Edmund Pezalla, MD, MPH
Former National Medical Director and VP, Aetna (2007 – 2016); CEO of Enlightenment BioConsult
Dr. Pezalla served for nine years as VP for Pharmaceutical Policy and Strategy for Aetna, and four years as VP of Clinical Services for RxSolutions. In these roles he developed programs for drug evaluation, creation of clinical policy and formulary inclusion. Dr. Pezalla also served as the leading executive for Aetna on public policy issues related to drug approval, drug pricing and regulation. He is a member of the MIT Center for Biomedical Innovation NEWDIGS project and was a member of the inaugural class of Scholars-in-Residence at the Duke-Margolis Center for Health
Dr. Pezalla received his BS and MD degrees with honors from Georgetown University and holds an MPH from the University of California at Berkeley. He was a Health Services Research Fellow and doctoral student at the University of Michigan
Schools of Medicine and Public Health.
Dr. Pezalla has published on a range of topics related to regulatory affairs/adaptive licensing and innovative healthcare financing models. Most recently Dr. Pezalla published a paper with Dr. Sam Nusbaum in JMCP on health plan member retention related to disease severity. And Dr. Pezalla recently presented at the MIT Paying for Cures conference in Washington, DC and the Pharma Pricing and Market Access Summit in Amsterdam.
In 2010, he was named by Modern Healthcare magazine as one of the 300 “most powerful people in American healthcare.” He is a Visiting Lecturer at the École Supérieure des Sciences Économiques et Commerciales (Paris and Singapore), and has served as an adjunct professor at Indiana University’s School of Public and Environmental Affairs and Butler University.
Peter is a widely published author and commentator on matters of FDA policy and process, healthcare technology assessment and reimbursement issues, real-world evidence, off label-communications, pharmacovigilance, patient-focused drug development, GMP standards, drug safety, personalized medicine, clinical trial transparency, and drug importation. He serves on the boards of several policy institutes and foundations.
Tobias Polak, MSc
His research topics cover causal inference, dynamic borrowing and, in particular, the use of data of expanded access programs.
Jess Rabourn, CFA
He is a member of the American Academy of Neurology and was lead author of the Expanded Access section of the submitted draft for FDA’s Guidance on ALS Drug Development. Prior to this work, Jess spent 15 years in the investment management industry at firms in San Francisco and New York. He earned the Chartered Financial Analyst designation in 2004.
David’s experience includes rare oncology and haematology (ALL, AML, MM, CD30+ HL/NHL, CTCL, ALK+/EGFR+ NSCLC, breast cancer), metabolic and endocrine diseases (MPS, Graves’, acromegaly), neurology (CIDP, RTT, MG, narcolepsy, Friedrich’s ataxia), ophthalmic disorders (LCA), hepatic conditions (PSC), and rare immunological diseases (AMR, GvHD).
He is the author and co-author of numerous peer-reviewed publications, a rare disease, orphan drugs and gene therapies trainer, and speaker at international meetings (ISPOR, RAPS EU Congress, Orphan Drugs Development and Commercialization).
Erika Segear, PhD, RAC
Dr. Segear also co-leads Duke’s Expanded Access Committee and led the development of a resource that assists physicians at Duke University in obtaining regulatory approvals for use of investigational drugs under FDA’s expanded access program.
Finally, she oversees and manages the implementation of new educational, operational, and collaborative initiatives within ORAQ. Prior to joining ORAQ, Dr. Segear conducted her graduate research studies at Duke, where she earned a PhD in Molecular Cancer Biology with a certificate in Cell and Molecular Biology. During her graduate training, she was the recipient of a Ruth L. Kirschstein-NRSA Predoctoral Fellowship and a Robert J. Fitzgerald Scholar Award.
Andrew Shuman, MD, FACS
Dr. Shuman completed fellowships in head and neck surgical oncology in the Department of Surgery at Memorial Sloan-Kettering Cancer Center, and in medical ethics at Weill Medical College of Cornell University. He completed his residency in the Department of Otolaryngology – Head and Neck Surgery at the University of Michigan.
He is a graduate of the University of Michigan Medical School, and also graduated from the University of Michigan’s College of Literature, Science and the Arts. His research explores the ethics of medication usage and drug shortages, as well as precision head and neck oncology.
Marjorie Speers, PhD
Previously, beginning in 2001, Dr. Speers was the inaugural president and CEO of the Association for the Accreditation of Human Research Protection Programs (AAHRPP) until her retirement in 2013. From 1999-2001, she was the Acting Executive Director of the National Bioethics Advisory Commission, where she oversaw the Commission’s work and report on the oversight system for research involving human participants.
Prior to that, she was the Deputy Associate Director for Science at the Centers for Disease Control and Prevention (CDC) where she oversaw all domestic and international research involving human participants. Dr. Speers was on the faculty at the University of Texas Medical Branch prior to joining CDC. As an epidemiologist, her research focused on risk factors for cardiovascular disease and cancer prevention.
Douglas Sproule, MD
Before transitioning into industry, Douglas was an Assistant Professor in Neurology at Columbia University Medical Center, New York, where he completed fellowship training in pediatric neurology and neuromuscular neurology. He is board-certified in neurology with special qualification in child neurology and in neuromuscular medicine by the American Board of Psychiatry and Neurology.
He has participated as principal investigator for numerous industry and government-sponsored clinical trials and has written or contributed to more than 30 publications in the area of neuromuscular disorders, including Spinal Muscular Atrophy and Duchenne Muscular Dystrophy
Steve’s reporting about biotechnology and biomedical policy has been cited in The Economist, The Wall Street Journal, the Washington Post, New Scientist and other publications.
In 2012, the FDA Alumni Association named Steve the Harvey W. Wiley Lecturer, making him the first journalist to receive the Wiley Award. His book, “Engineering Communism: How Two Americans Spied for Stalin and Founded the Soviet Silicon Valley,” was published in 2005 by Yale University Press.
Robert Walker, MD
Prior to joining BARDA, Dr. Walker was Chief Medical Officer at Nuron Biotech in Exton, PA. His other prior work in clinical development includes directing tuberculosis vaccine clinical trials at Aeras, and positions of increasing responsibility at MedImmune, Inc., where he oversaw the late-stage clinical development and U.S. marketing approval of FluMist intranasal influenza vaccine, and where he managed the respiratory virus vaccines and inflammatory and pulmonary diseases portfolios, international expansion activities, and phase 4/epidemiology studies.
Before working in the biotech industry, Dr. Walker was a clinical investigator at the National Institute of Allergy and Infectious Diseases and an attending physician in Critical Care Medicine at the National Institutes of Health Clinical Center. Dr. Walker earned his medical degree from the University of Maryland School of Medicine, and received his internal medicine training at George Washington University and his subspecialty training in pulmonary and critical care medicine at the University of Pennsylvania.
Kevin is the Managing Director of the Michigan Institute for Clinical & Health Research (MICHR) and Adjunct Associate Clinical Professor in the College of Pharmacy. He also serves as Co-chair of the Michigan Medicine Expanded Access Oversight Committee to develop and enhance processes and infrastructure to support treating clinicians interested in providing access to investigational therapies/agents to patients with no alternatives.
Kevin has been instrumental in the development of institutional policy and processes as they relate to FDA-regulated research at the University of Michigan. Kevin is Co-principal investigator with Dr. George Mashour on a NIH U01 award promoting a national network for the FDA’s Expanded Access program.
In the role of Managing Director, Kevin is the administrative lead and is responsible for operational, fiscal, and executive oversight. He works closely with Dr. Mashour and the MICHR leadership team to build the organization, shape strategy, and implement programs and resources to serve the research enterprise.
Janet Woodcock, MD
Dr. Woodcock first joined CDER in 1994. For three years, from 2005 until 2008, she served FDA’s Commissioner, holding several positions, including as Deputy Commissioner and Chief Medical Officer, Deputy Commissioner for Operations, and Chief Operating Officer. Her responsibilities involved oversight of various aspects of scientific and medical regulatory operations.
Before joining CDER, Dr. Woodcock served as Director, Office of Therapeutics Research and Review, and Acting Deputy Director in FDA’s Center for Biologics Evaluation and Research. Dr. Woodcock received her M.D. from Northwestern Medical School and completed further training and held teaching appointments at the Pennsylvania State University and the University of California in San Francisco. She joined FDA in 1986.
Frank Young, MD, PhD
Dr. Frank Young was a previous Commissioner of the U.S. Food and Drug Administration (FDA), and had served as Deputy Assistant Secretary of the U.S. Department of Health and Human Services.
He was most recently Executive Vice President for Clinical, Regulatory Affairs, and Quality and had served in a leadership capacity with multiple other life sciences companies. Previous to his appointment to lead the FDA, Dr. Young was Dean of the School of Medicine and Dentistry and Vice President for Health Affairs at the University of Rochester.
While serving at the U.S. Department of Health Human Services, Dr. Young received the Inspector General’s Award for Outstanding Integrity, and the Surgeon General’s Exemplary Service Medal from the U.S. Public Health Service. Dr. Young was a member of the National Academy of Medicine. In 2015, he received the Distinguished Scientist Award from the American College of Toxicology.
Dr. Young contributed to more than 200 scientific publications in microbiology, biotechnology and pathology.