Cancer drugs speed to accelerated approvals, then hit the brakes in confirmatory trials
August 15, 2024This article, authored by Evolent Vice President of Pharmacy Terra Wonsettler, was published in the August 2024 issue of the American Journal of Managed Care’s Evidence Based Oncology.
How does the FDA balance the demand for life-saving drugs with its mandate to ensure a clinical benefit in therapies it approves? For more than 30 years, one method has been the accelerated approval (AA) pathway, which can make a drug available if it meets benchmarks that are associated with improved survival. The use of AA is not the end of the journey, however; drug sponsors have responsibilities to multiple stakeholders after a therapy reaches the market.
The Accelerated Approval Pathway
The number of AA drug applications granted by the FDA has steadily risen. Since the agency instituted AA regulations in 1992, there have been 278 approvals spanning various disease types through 2021.
Notably, AA is gaining momentum in oncology, with 9 FDA-approved oncology therapies in 2024 through June, compared with 5 in the first half of 2023. Approximately one-third of all oncology drug approvals utilize this pathway, more than any other specialty. The AA regulations allow drugs for serious conditions that fill an unmet medical need to be approved based upon a surrogate end point. Therefore, the value of AA lies in its ability to expedite access to potentially life-saving drugs for patients facing serious conditions such as cancer. These drugs have been shown in clinical trial findings to meet their surrogate end points (such as response rate or progression-free survival) which suggest there may be an overall survival (OS) benefit. Although AA allows drugs that address unmet needs to reach patients faster, timely completion and rigorous validation of clinical benefit in confirmatory trials is essential to ensure patient safety and efficacy, avoiding low-value, high-cost care.