Addressing Evidence Gaps in the FDA Accelerated Approval: Payer Perspectives

To support balancing patient access with payer needs related to the U.S. Food and Drug Administration (FDA) Accelerated Approval (AA) pathway program, AMCP convened key stakeholders in managed care from health plans, pharmacy benefit managers, integrated delivery systems, patient advocacy organizations, research and policy organizations, academia, and biopharmaceutical manufacturers. These participants were asked to:

1) identify gaps between FDA AA requirements and treatment outcomes valued by payers,

2) explore opportunities for an evidence ecosystem to address these gaps, and

3) evaluate and prioritize policy options to facilitate communication of payer needs, reduce financial uncertainty, or improve the time between FDA approval or clearance and payer coverage decisions.

Participants felt it was important to continue to support innovation in drug development and patient access through the FDA expedited programs while also recognizing payer needs to aid coverage determinations and increase stakeholder trust in the AA review process program. Specifically, more robust clinical trial evidence and correlating surrogate endpoints with meaningful outcomes and developing an evidence ecosystem to deliver information more readily were seen as key. To note the various potential policy solutions reviewed, policies incentivizing the completion of confirmatory trials garnered the most interest and was thought to have the potential for the most impact.

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