May 22, 2023
By Kellie Rademacher, Director, Strategy, Evoke Navience
Through the accelerated approval pathway, the FDA can approve drugs earlier by using surrogate endpoints predictive of clinical benefit to validate drugs, and then require confirmatory trials after approval. At the end of 2022, reforms for the accelerated approval pathway were included in the draft of the annual spending bill. The FDA has issued draft guidance requesting feedback on the reform.
In the guidance, the FDA is recommending shifting from single-arm trials to randomized controlled trials (RCT) for oncology products to address the limitations of single-arm trials and suggesting a potential advantage (in appropriate classes) to the RCTs. Long-term follow-up trials could serve as the post marketing requirement to verify clinical benefit.
Potential Impact on Patient Access
There are some potential repercussions of this seemingly beneficial move that may affect patient access to oncolytic options moving forward. First, if RCT’s are required, recruiting participants for randomization into both a treatment and control arm could delay trial starts, resulting in product delays to desperate patients. Second, in oncology there are often multiple treatment regimens recommended based on several factors, the selection of the control arm regimen (if only one regimen is chosen) for the RCT could result in payer restrictions of use with other recommended regimens, decreasing patient access.
It will be interesting to follow comments and the FDA’s final guidance. While well intended the FDA’s efforts to reform the accelerated approval pathway in oncology may result in delayed lifesaving therapies reaching patients, additional costs to plan sponsors, and highly restricted access for patients.
Stay Informed with Evoke Navience
Evoke Navience helps our clients navigate the potential implications of policy shifts on their brand strategies. Connect with our strategy experts for potential implications for your brand. Reach out to Christine Lenthe at Christine.email@example.com to get connected!
 FDA (n.d.). Review of the FDA's Accelerated Approval Pathway. U.S. Department of Health and Human Services Office of Inspector General. Retrieved April 4, 2023. https://oig.hhs.gov/reports-and-publications/workplan/summary/wp-summary-0000608.asp.  Bayer, M. (2022, December 20). FDA's accelerated approval reforms sneak into Congress' last-minute spending bill. Retrieved April 4, 2023. https://www.fiercebiotech.com/biotech/accelerated-approval-reforms-are-precipice-becoming-law-cementing-califf-proposals.  FDA (2023, March 23). Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics; Guidance for Industry; Draft Guidance. Regulations.gov. Retrieved April 4, 2023. https://www.regulations.gov/document/FDA-2023-D-0110-0001.
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