Current and former policymakers have recently spoken about the potential for Medicare to pay a lower price for medicines approved via the Accelerated Approval pathway until they successfully complete post-approval confirmatory trial requirements.
The FDA approval process is poorly understood generally, and any responses to a pilot program or proposed rule would require significant education around the process for the public. Companies with medicines recently approved via Accelerated Approval are potentially at risk of critical media coverage while those with pipeline products along the same pathway could be subject to pilot programs that reduce reimbursement.
About Accelerated Approval
Accelerated Approval allows the FDA to approve medicines for serious conditions with unmet need based on a surrogate endpoint, such as shrinking tumor size or improving a biomarker associated with the disease, rather than the full clinical benefit.
- Drugs approved via Accelerated Approval must have their benefit to patients confirmed by a post-approval confirmatory clinical trial. However, for several years, policymakers have criticized the speed of those trials and the perceived inability of the FDA to remove drugs from the market if they do not show a benefit.
Recent Policymaker Commentary
STAT recently published an article featuring comments made by Centers for Medicare & Medicaid Services (CMS) Chief Medical Officer Lee Fleisher, who said that the agency is considering a range of reimbursement options for drugs approved via Accelerated Approval, including lower reimbursement rates. The article notes that the deadline set by an executive order for HHS to propose demonstration projects that could lower drug prices was last month.
- This is not the first time the notion has been raised, and the idea seems to be gaining some ground. For example, the Institute for Clinical and Economic Review (ICER) put forth this idea in its April 2021 Policy Reform paper and a June 2021 Medicaid and CHIP Payment and Access Commission (MACPAC) recommendation suggested higher rebates for drugs that had not completed confirmatory trials.
Separately, Pink Sheet covered a panel discussion featuring FDA Commissioner Robert Califf and former commissioners all giving support to the concept of differentiated pricing for accelerated approval drugs. Califf, who noted he was not speaking on behalf of the agency, stated, “There is a lot of wisdom in the idea that if you haven't proven that you're effective, that the price shouldn't be as high as if you have proven that.”
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