The meeting was moderated by Saira Sultan, Connect 4 Strategies. Attendees included representatives of leading innovators in the rare and ultra-rare space. A brief introductory discussion framed up the between orphan and ‘orphan blockbuster’ from a business perspective and the lack of such a distinction in the current policy landscape, particularly given the increased awareness of “blockbuster orphan” drugs.
SPEAKER BIOS
Gayatri R. Rao, M.D., J.D.
Director, Office of Orphan Products Development, Food and Drug Administration
Gayatri R. Rao, is the Director for the Office of Orphan Products Development (OOPD) at FDA. The Office’s mission is to advance the development of promising drugs, biologics, devices, and medical foods for rare diseases. As Director, she oversees a number of programs created to promote the development of such rare disease products, including multiple designation programs – like orphan drug, rare pediatric disease, and humanitarian use device – as well as several multi-million dollar grant programs. In addition, she serves as FDA’s lead in coordinating cross-Agency efforts on rare disease issues, is actively engaged in a number of internal and external collaborations, and oversees the Office’s extensive outreach efforts to patients, sponsors, and other stakeholders.
Lynne Yao, M.D.
Director, Division of Pediatric and Maternal Health, Office of New Drugs, CDER, FDA
Lynne Yao, M.D., is the Director, Division of Pediatric and Maternal Health in the Office of New Drugs, Center for Drug Evaluation and Research. She has held this position since 2012. The Division of Pediatric and Maternal Health oversees quality initiatives which promote and necessitate the study of drug and biological products in the pediatric population; and improve pregnancy and lactation-related information in product labeling. Dr. Yao started at FDA as a Medical Officer and primary reviewer on the Inborn Errors of Metabolism team in the Division of Gastroenterology and Inborn Errors Products (DGIEP) in 2008, and was a team leader in DGIEP from 2009-2012. Dr. Yao graduated from the George Washington University School of Medicine, completed residency in Pediatrics at Walter Reed Army Medical Center, and fellowship in Pediatric Nephrology at the Georgetown University Children’s Medical Center. Dr. Yao is board certified in both Pediatrics and Pediatric Nephrology.
John Alexander, M.D.
Deputy Director, Division of Pediatric and Maternal Health, Office of New Drugs, CDER, FDA
Dr. John Alexander is currently the Deputy Director of the Division of Pediatric and Maternal Health in the Office of New Drugs at the Center for Drug Evaluation and Research. He is a pediatrician with more than 20 years of experience in drug regulation at FDA, including review of drugs for orphan diseases.
Craig Burns, PhD MS
Vice President of Research, The Center for Policy & Research, America’s Health Insurance Plans
Craig Burns oversees research studies at The Center for Policy & Research at America’s Health Insurance Plans, where he is currently Vice President of Research. Much of his research at AHIP focuses on pharmaceutical related issues. Prior to joining AHIP, Craig spent 13 years working in research and development in the biotechnology and pharmaceutical industries, mainly in the oncology and autoimmune disease areas. Craig worked for six years for Sanofi-Aventis as a Project Director where he led four, multi-disciplinary, international, oncology drug development teams and was responsible for all aspects of a drug’s development from the start of development until regulatory approvals in the EU, US, and Japan. Prior to joining Sanofi, Craig worked for four years as an R&D Project Manager for Medimmune, Inc., developing monoclonal antibodies in the oncology, autoimmune disease, and infectious disease areas. Craig began his career in drug development as a Regulatory Affairs Associate working in the US Regulatory Affairs group of British Biotech, plc. Craig received his Bachelor’s degree from The University of Tulsa in Analytical Chemistry; his Master’s degree from Oklahoma State University in Toxicology; and his PhD from the University of Oklahoma in Health Economics.
Stuart Portman, MPH
Senior Healthcare Legislative Assistant, Senator Orrin Hatch (R-UT)
Stuart Portman serves as the Senior Healthcare Legislative Assistant for Senator Orrin G. Hatch. In this role, he provides policy recommendations on a variety of health matters ranging from disease research and pharmaceutical development to health information technology and health coverage for persons with disabilities, among others. Stuart also serves as deputy to the Medicaid adviser for Chairman Hatch, linking the Utah perspective to broader policy debates and working collaboratively on pressing Medicaid issues across the country. Stuart received his Master of Public Health degree specializing in health policy from the Milken Institute School of Public Health at The George Washington University.