Our Speaker Faculty
Ax-S Pharma brings together world leaders from industry, medicine, and policy centers to provide members an unparalleled range of information and perspective on the integration of research and healthcare.
Amy Abernethy, MD, PhD
Principal Deputy Commissioner and Acting Chief Information Officer, FDA
Amy P. Abernethy, M.D., Ph.D. is an oncologist and internationally recognized clinical data expert and clinical researcher. As the Principal Deputy Commissioner of Food and Drugs, Dr. Abernethy helps oversee FDA’s day-to-day functioning and directs special and high-priority cross-cutting initiatives that impact the regulation of drugs, medical devices, tobacco and food. As acting Chief Information Officer, she oversees FDA’s data and technical vision, and its execution. She has held multiple executive roles at Flatiron Health and was professor of medicine at Duke University School of Medicine, where she ran the Center for Learning Health Care and the Duke Cancer Care Research Program. Dr. Abernethy received her M.D. at Duke University, where she did her internal medicine residency, served as chief resident, and completed her hematology/oncology fellowship. She received her Ph.D. from Flinders University, her B.A. from the University of Pennsylvania and is boarded in palliative medicine.
Paul Aliu, PhD
Global Head Medical Governance, Chief Medical Office, Novartis Pharmaceuticals
Paul Aliu is the Global Head Medical Governance within the cross-divisional Chief Medical Office at Novartis with responsibility for the oversight, processes, training and systems for compassionate use/expanded access and post-registration medical programs (e.g. Investigator initiated trials, non-interventional studies, registries and Phase IV studies).
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
Director, National Center for Advancing Translational Sciences, NIH
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
Founder and President of Arctiryon, Inc.
Georges van Baelen, MScPh studied Medical and Pharmaceutical Sciences at the “Katholieke Universiteit Leuven KUL” and the “Université Catholique de Louvain UCL”, in Belgium and did doctoral work in Toxicology and Advanced Pharmacology at the Joint Graduate Program at Rutgers University / Robert Wood Johnson Medical School UMDNJ in New Jersey, USA. He also obtained certification in Latin American Pharmacovigilance Practices from the University of Antioquia in Medellín, Colombia.
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
Associate Director, Center for Information and Study on Clinical Research Participation (CISCRP)
Amy Barone, MD
Medical Officer, FDA Office of Hematology and Oncology Products
Dr. Amy Barone is a pediatric oncologist who joined the US Food and Drug Administration (FDA) in January 2014 as a medical officer for the CNS, Pediatric Solid Tumors, and Rare Cancers.
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
Director of Healthcare Policy, Goldwater Institute
Naomi Lopez Bauman is the director of healthcare policy at the Goldwater Institute. She directs and engages in research and strategy in healthcare issues including Right to Try, off-label communications, state insurance markets, the Affordable Care Act, Medicaid, pharmaceutical drug pricing, and healthcare innovation.
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.
CEO & Co-Founder, Anova
Mr. Beardmore is an experienced and qualified executive with start-up, regulatory, project management and operational experience across the healthcare industry. He began his career in regulatory affairs, influenced by leading human subject and animal subject protection committees at large academic institutions.
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
VP Consulting, Inceptua Medicines Access
Stuart has more than 20 years of healthcare and pharma consulting experience, with a particular focus in unlicensed medicines and pre-approval access. He is one of the senior management team responsible for establishing Inceptua Medicines Access and is responsible for Inceptua’s consulting engagements, covering strategy and policy, real-world evidence, communications and market access.
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
Interim Executive Director, Reagan-Udall Foundation, FDA
Dr. Bhat is the Interim Executive Director of the Reagan-Udall Foundation for the FDA, a non-profit organization created by Congress to advance the mission of the Food and Drug Administration. Dr. Bhat joined the Foundation in 2018 as the Director of Business Planning and Programs, with a portfolio focused on developing new activities and strategic planning.
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
Chief Executive Officer, National Health Council
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
Director of Research Strategy, CENTRA, American Society of Clinical Oncology (ASCO)
Suanna Bruinooge, MPH, is the Division Director of Research Strategy and Operations in ASCO’s Center for Research and Analytics (CENTRA). CENTRA generates, integrates, analyzes, and shares oncology data to foster innovation in research and patient care and help develop and evaluate ASCO’s policy positions. CENTRA develops and implements ASCO’s research agenda, including the Targeted Agent Profiling and Utilization (TAPUR) clinical trial and projects to advance clinical trial design and methodology. CENTRA also staffs ASCO’s Cancer Research Committee, Research Community Forum, and Workforce Advisory Group.
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
Managing Partner, Coulomb Strategy Consulting LLC
Connie Coulomb is Managing Partner at Coulomb Strategy Consulting LLC, a firm dedicated to helping companies advance their strategy and turn exciting research into successful products that can benefit patients’ lives.
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
Chief Scientific Officer, Early Access Care, LLC
Anne Cropp, BSc, Pharm.D. BCAP serves as the Chief Scientific Officer for Early Access Care, providing consultative, functional and operational support to biopharmaceutical companies for Expanded Access / Compassionate Use of investigational drugs.
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.
Faculty, Harvard Medical School; Associate Scientist, Women and Brigham’s Hospital
Jonathan Darrow joined the faculty of Harvard Medical School and the Program on Regulation, Therapeutics, and Law (PORTAL) in 2016, following service as Senior Law Clerk to a judge on the United States Court of Appeals for the Federal Circuit, the court that decides all patent appeals involving pharmaceuticals, biologics, and medical devices. He holds degrees in genetics, law, and business from Cornell, Duke, and Boston College, respectively, as well as a research doctorate in pharmaceutical policy and intellectual property theory from Harvard, where he also completed the LL.M. program.
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
Founder & President of Texas Rare Alliance, Texas Newborn Screening Advisory Committee
Khrystal joined the rare disease community in 2011 when her newborn son, Hunter, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, the leading genetic cause of mortality in children under the age of two. SMA Type 1, often described as ALS in babies, robs the ability to move, swallow, and ultimately breathe. Khrystal is the author of Hunt for a Cure: An Unexpected Adventure to Save a Life.
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
Senior Partner, King & Spalding
David Farber is a partner in the FDA and Life Sciences practice at King & Spalding, resident in the firm’s Washington, D.C. office, where he maintains a health care practice focused on government advocacy and regulatory counseling.
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
Director, National Institute of Allergy and Infectious Diseases (NIAID), NIH
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.
Director, Global Medical Affairs, Early Access Programs, Alnylam Pharmaceuticals
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.
Founding President and CEO, Parent Project Muscular Dystrophy (PPMD)
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.
Lee Gehrke, PhD
Professor, Institute for Medical Engineering and Science, MIT
Lee Gehrke is the Hermann L.F. von Helmholtz Professor in the Institute for Medical Engineering and Science at M.I.T. and Professor of Microbiology at Harvard Medical School. Professor Gehrke’s research has focused on positive-sense single-stranded RNA viruses with particular focus in flaviviruses, hemorrhagic fever viruses, translation-level regulation of gene expression, and the design and construction of rapid point-of-care diagnostic devices. Professor Gehrke’s collaborative work with engineers and materials scientists led to the development of rapid diagnostics to detect and distinguish the four dengue serotypes, as well as Zika virus and Chikungunya virus. He is co-founder of e25Bio where he currently serves on the scientific advisory board.
Christina Hartman, MPH
Senior Director of Advocacy, The Assistance Fund
Christina Hartman, MPH, is a DC-based policy and advocacy expert with a background in building alliances. Christina leads policy and advocacy efforts to advance the development of treatment and diagnostic opportunities for rare disease patients at the EveryLife Foundation.
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
Chief Commercial Officer, xCures
xCures is a health technology and services company founded by Marty Tenenbaum, a renowned computer scientist, Internet entrepreneur and cancer survivor and Jeff Shrager, a Stanford professor and expert in applying high-performance cloud computing and machine learning in biological science.
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
Senior Counsel, Covington & Burling LLP
Peter Barton Hutt is a Senior Counsel in the Washington, D.C. law firm of Covington & Burling LLP specializing in food and drug law. He graduated from Yale College and Harvard Law School and obtained a Master of Laws degree in Food and Drug Law from NYU Law School.
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.
Michael Joyner, MD
Faculty Member, Mayo Clinic
Dr. Michael Joyner is a professor of anesthesiology and clinical researcher at the Mayo Clinic, where his laboratory has been funded by the National Institutes of Health since 1993. Dr. Joyner has served as principal investigator in several US Government sponsored programs including the Expanded Access program for COVID-19 convalescent plasma for the treatment of individuals with severe or life-threatening disease.
Former Director, FDA, Office of Health and Constituent Affairs
Richard Klein recently left FDA after more than 41 years with the agency. He served as director of the FDA’s Patient Liaison Program in the agency’s Office of Health and Constituent Affairs, the primary agency interface with patients and patient advocate communities. He interacted extensively with outside communities and within the agency’s scientific and policy offices to advocate for patient interests, and facilitate patient engagement. He actively addressed issues and concerns of patients in a variety of areas, including treatment access to unapproved drugs, product safety, and clinical trial design.
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.
Managing Director of Business Development, Tanner Pharma Group
John is currently the Managing Director of Business Development for the Tanner Pharma Group, Charlotte, NC. John has worked in the rare disease or expanded access space for over 25 years in various development, operational, and commercial roles.
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.
President, X2 Foundation
Jeff Leider’s passion for advocacy began when his two sons were diagnosed with MPSII/Hunters Syndrome. Since diagnosis, he has worked tirelessly not only for his children but all children with Rare Diseases, creating both Jason & Justin’s Journey for his sons and the Let Them Be Little X2 Foundation for rare disease families.
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.
Founder, Clinical Innovation Partners
Craig Lipset is a recognized leader at the forefront of innovation in clinical research and medicine development. He is the founder of Clinical Innovation Partners, working as an advisor and board member with pharma, tech and venture capital to bring vision and driving action at the intersection of research, digital solutions, and patient engagement. Craig was the Head of Clinical Innovation and Venture Partner at Pfizer, on the founding Operations Committee for TransCelerate Biopharma, and on the founding management teams for two successful startup ventures.
Craig is Adjunct Assistant Professor in Health Informatics at Rutgers University, and Adjunct Instructor in the Center for Health + Technology at University of Rochester. He serves on the Board of Directors for the Foundation for Sarcoidosis Research and the MedStar Health Research Institute, as well as on the Editorial Board for Therapeutic Innovation & Regulatory Science.
Craig has been listed among the PharmaVOICE most inspiring people in the life sciences (Red Jacket hall-of-fame), Pharmaceutical Executive’s Emerging Leaders, CenterWatch Top 20 Innovators in Clinical Trials, and the AlleyWatch Who’s Who in eHealth.
Peter Marks MD PhD
Director, Center for Biologics Evaluation and Research, FDA
Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016.
Hilary Marston, MD, MPH
Medical Officer, National Institute of Allergy and Infectious Diseases (NIAID), NIH
Hilary Marston, MD, MPH is a Medical Officer and Policy Advisor for Global Health focusing on emerging infectious disease preparedness and response. In this role, she coordinates the National Institute of Allergy and Infectious Diseases’ response to outbreaks including Zika in the Americas and Ebola in West Africa and the Democratic Republic of the Congo. Dr. Marston trained in internal medicine at Brigham & Women’s Hospital, during which time she worked with Partners in Health and the Clinton Health Access Initiative. Before her medical training, Dr. Marston worked for McKinsey & Company and at the Bill & Melinda Gates Foundation as a Program Officer and Special Assistant to the Co-Chair.
Secretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (www.eucope.org) in Brussels and advises innovative pharmaceutical and biotech companies, including start-ups, in regulatory and pricing & reimbursement matters from the EU law and German law perspective.
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 is Founder and CEO of Enlightenment Bioconsult, LLC, a strategic payer consultancy advising biopharmaceutical firms on access, technology assessment, and drug evaluation in the US market. His clients include established pharmaceutical firms and emerging companies with innovative therapies.
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.
President, Center for Medicine in the Public Interest (CMPI)
Peter Pitts is President of the Center for Medicine in the Public Interest and Chief Regulatory Officer for Adherent Health. A former member of the United States Senior Executive Service (SES), Peter was FDA’s Associate Commissioner for External Relations, serving as senior communications and policy adviser to the Commissioner. He supervised FDA’s Office of Public Affairs, Office of the Ombudsman, Office of Special Health Issues, Office of Executive Secretariat, and Advisory Committee Oversight and Management.
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
Department of Health Technology Assessment, Erasmus MC
Tobias Polak holds a BSc and Msc (cum laude) in Econometrics from Erasmus University of Rotterdam and currently is a PhD Candidate at the Department of Biostatistics and the Department of Health Technology Assessment of the Erasmus MC.
His research topics cover causal inference, dynamic borrowing and, in particular, the use of data of expanded access programs.
Jess Rabourn, CFA
Founder and CEO, WideTrial
Jess Rabourn is the founder and CEO of WideTrial, an early-stage company that serves as a third-party sponsor of multi-site, large-group Expanded Access programs. Since 2010 he has been a nationally recognized speaker on pre-approval access and, through his consulting company and non-profit projects, has provided substantial education and policy support on the topic.
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.
Principal and Founder, ORPHA Strategy Consulting
David is a sought-after expert for accelerating marketing authorization, time to launch, early patient and market access in the European environment of orphan and advanced therapy medicinal products.
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
Associate Director of Regulatory Affairs, Duke University School of Medicine
Erika Segear, PhD, RAC is the Associate Director of Regulatory Affairs in the Office of Regulatory Affairs and Quality (ORAQ) within the Duke University School of Medicine. In this role, Dr. Segear works with a team of Regulatory Affairs professionals who serve as a no cost resource to the clinical research community at Duke University. She is responsible for providing guidance and support to Duke investigators in various aspects of regulatory affairs, including regulatory strategy development, preclinical testing, product manufacturing, FDA meetings, regulatory submissions, and regulatory education.
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
Chief of Ethics Service, University of Michigan Medical School
Andrew G. Shuman, MD FACS is chief of the Clinical Ethics Service in the Center for Bioethics and Social Sciences in Medicine at the University of Michigan. He is also an Assistant Professor in the Department of Otolaryngology – Head and Neck Surgery at the University of Michigan Medical School, and Chief of the ENT Section of the Surgery Service at the VA Ann Arbor Health System.
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
Executive Director, Clinical Research Pathways
Dr. Marjorie Speers is Executive Director of Clinical Research Pathways (formerly WCG Foundation), a public charity, since 2015. Clinical Research Pathways promotes compassion, inclusion and education in research. Clinical Research Pathways helps physicians and institutional review boards streamline the Food and Drug Administration’s expanded access process to make it easier for desperately ill patients to try experimental medicines.
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
Vice President, Spinal Muscular Atrophy Therapeutic Area Head, AveXis
Douglas M. Sproule is currently Vice President, Spinal Muscular Atrophy Therapeutic Area Head at AveXis, a role he has held since February 2016. Prior to this, he was Global Medical Affairs Director supporting the Pompe and Fabry disease franchises at Sanofi Genzyme.
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
CEO, NRG Collective Ltd
Rebecca is Co-founder of the dedicated rare disease publication RARE Revolution Magazine, the youth programme #RAREYouthRevolution and the International charity Teddington Trust – an organisation, founded in 2012, supporting those affected by the Ultra-Rare Disease Xeroderma Pigmentosum.
Passionate about appropriate and accessible resources Rebecca and co-founder Nicola Miller have dedicated the last eight years to creating award winning patient education materials and to provide a platform that educates, signposts, and connects the whole rare disease community.
Senior Editor, BioCentury
Steve Usdin has been Washington Editor of BioCentury since 1993, and has spent the past 25 years in the nation’s capital covering political and policy issues affecting the life sciences sector. He also is BioCentury Senior Editor responsible for coverage of social issues involving biotechnology, as well as the former host of BioCentury This Week, BioCentury’s public affairs television program.
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
Director, Division of Clinical Development, Biomedical Advanced Research and Development Authority (BARDA), HHS
Dr. Walker is the Director of the Division of Clinical Development (DCD) and (Acting) Chief Medical Officer in the Biomedical Advanced Research and Development Authority (BARDA) in the Office of the Assistant Secretary for Preparedness and Response (ASPR). The DCD manages the Clinical Studies Network and is responsible for oversight of the coordination, execution, and analysis of interventional clinical studies funded by BARDA intended to support licensure and Emergency Use Authorization of medical countermeasures. Dr. Walker joined BARDA in 2014, and oversees a diverse portfolio of vaccines, therapeutics, and devices in clinical development for potential use in public health emergencies.
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.
Managing Director, Michigan Institute for Clinical and Health Research, University of Michigan Medical System
Co-Chair, Expanded Access Oversight Committee, Michigan Medicine; Adjunct Associate Clinical Professor, College of Pharmacy
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
Director, Center for Drug Evaluation and Research, FDA
Janet Woodcock is Director of the Center for Drug Evaluation and Research (CDER), at the Food and Drug Administration (FDA). In 2015, Dr. Woodcock also assumed the role of Acting Director of CDER’s newly formed Office of Pharmaceutical Quality, (OPQ).
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
Executive Vice President Clinical & Reg Affairs, TissueTech Inc.; Former FDA Commissioner, 1984-1989
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.