AMCP Partnership Forum Develops Solutions to Address Evidence Gaps in the Expedited Review Process

Alexandria, Va., December 9, 2021 – Last week, nearly 40 experts representing payers, pharmacy benefit managers, integrated delivery systems, patient advocates, academicians, and other key stakeholders in the managed care setting convened for AMCP’s Partnership Forum Addressing Evidence Gaps in the Expedited Review Process: Payer Perspectives. The purpose of the forum was to identify payer needs and current barriers for therapies that are approved through the Food and Drug Administration’s  (FDA) expedited pathways. The group also worked to identify potential solutions to address evidence gaps and reduce the financial uncertainty associated with these products.

Forum participants discussed the need and impact of the expedited pathways to advance the development and review of innovations for serious medical conditions. However, forum participants emphasized that accelerated regulatory approval does not necessarily equate to accelerated coverage and reimbursement. Additional outcomes from the discussions included that education regarding these pathways is needed,  surrogate endpoints should be meaningful, and enhanced communication is desired to build trust between regulators and payers.

After deliberating on potential solutions to address existing evidence gaps, reduce financial uncertainty, and shorten the time between approval and coverage decisions, participants came up with the following recommendations:

• Support innovation: Innovation in the development of drugs and other therapies is critical to all stakeholders, and meaningful endpoints should be considered during the approval process to aid coverage determinations.
• Clarify terminology: The surrogate endpoint definition that serves   as the basis for accelerated approval needs to be refined, codified, and shared.
• Correlate surrogate endpoints with meaningful outcomes: Surrogate endpoints need to map or connect to meaningful outcomes, increasing confidence in clinical benefits and providing greater certainty for payment and reimbursement.
• Develop an evidence ecosystem: An evidence ecosystem with both enhanced data and analytic capabilities can deliver information more readily on the long-term safety, effectiveness, and durability of treatment benefits projected at approval.
• Advance potential policy solutions: Policies and practices should be implemented to reduce the financial uncertainty associated with drugs approved using the accelerated pathway.

“Today, more than 50% of novel drugs are approved by the FDA through expedited development and review programs,”  said AMCP CEO Susan Cantrell, RPh, CAE. “As more companies use these methods   for approval, it is vital that the evidence gaps are addressed in order to ensure positive health outcomes and better patient care. The participants at the Partnership Forum laid a foundation that will increase shared knowledge and reduce the timetable and barriers to appropriate access of   life-saving drugs and treatments.”

All findings and recommendations from the AMCP Partnership Forum Addressing Evidence Gaps in the Expedited Review Process: Payer Perspectives will be published in an upcoming issue of Journal of Managed Care + Specialty Pharmacy. In addition, AMCP will host a webinar in 2022 reporting the findings and recommendations. The event was sponsored by Gilead, Janssen, Pfizer, PhRMA,  Seagen, Takeda, and uniQure.

About AMCP

AMCP is the professional association leading the way to help patients get the medications they need at a cost they can afford. AMCP’s diverse membership of pharmacists, physicians, nurses, biopharmaceutical professionals, and other stakeholders leverage their specialized expertise in clinical evidence and economics to optimize medication benefit design and population health management and help patients access cost-effective and safe medications and other drug therapies. AMCP members improve the lives of nearly 300 million Americans served by private and public health plans, pharmacy benefit management firms, and emerging care models. Visit www.amcp.org.