On Monday, October 7, 2019, Friends of Cancer Research (Friends) and Prevision Policy hosted the fifth annual BioPharma Congress. This year’s event focused on gene therapies, approval standards, new payment models, drug pricing, and the future of the U.S. Food & Drug Administration (FDA). Featured speakers included Assistant to the President and Director of the Domestic Policy Council, Joe Grogan, Acting FDA Commissioner Ned Sharpless, and former FDA Commissioner Scott Gottlieb. The event brought together regulators, industry, patient advocacy groups, and congressional staff as well as payment and reimbursement organizations.
This year’s BioPharma Congress was split into 8 sessions covering a diverse range of vital health policy topics. The morning featured a keynote conversation featuring Former FDA Commissioner Scott Gottlieb as he discussed the past, present, and future of the FDA. The second panel focused on a lively discussion of drug pricing and impact on patient care, including Friends Board Member David Mitchell, President and Founder of Patients for Affordable Drugs, who noted that continuous innovation must be paired with affordability. White House Domestic Policy Council Director Joe Grogan followed the panel up with a conversation around the administration’s drug pricing proposals, saying that it wanted to first and foremost give certainty to patients with any legislation to address the issues behind cost.
Friends’ Founder & Chairperson, Ellen Sigal, then moderated a morning panel with FDA center directors including Dr. Janet Woodcock (CDER), Dr. Peter Marks (CBER), and Dr. Richard Pazdur (OCE). The directors covered a wide breadth of topics including gene therapy, how the FDA Oncology Center of Excellence has instigated a cultural change at FDA, and the need to fix the clinical trial system in the U.S.
Acting FDA Commissioner Ned Sharpless presented the lunch keynote at this year’s BioPharma Congress with Friends Vice President of Public Affairs, Ryan Hohman, introducing him. Sharpless discussed the agency’s challenges and the initiatives they are undertaking to address them. He mentioned the importance of accelerated approval pathways such as the FDA’s Breakthrough Therapy designation, and their role in helping the FDA adapt to changing science and innovation. The Acting Commissioner also added that the FDA is looking intently at operationalizing and validating the use of real-world evidence (RWE), noting Friends recent conference and leading work on RWE.
In the afternoon, the first panel addressed a new era of personal and individualized drug development, bringing together regulators and innovators. Lilly Oncology’s Symantha Melemed noted that it is important to pay attention to the evolution of real-world data sets in drug development, adding that the data will inform how we construct trials and development programs in the future. The following panel set the stage for how drug pricing issues intersect with insurance and payment reform, featuring former HHS Senior Advisor John O’Brien. The panel emphasized how the Medicare and Medicaid population impact payment systems and coverage for innovations such as CAR-T and cell therapies. The final panel, moderated by Prevision Policy’s Kate Rawson, featured FDA’s rising stars across the agency’s medical product portfolio as they discussed their own experiences witnessing changes within their own disease areas, the FDA structure, and as they plan for the future of medical product and regulatory innovation.