FDA Issues Draft Guidance on Accelerated Approval for Oncology Drugs

On March 24, 2023, the FDA issued a draft guidance on the clinical trial considerations for the accelerated approval of oncology drugs. The proposed recommendations include guidelines to fortify and strengthen the clinical trial process for oncology therapeutics.

According to the document released by the FDA, many oncology drugs are designated for one or more accelerated approval pathways due to the severe and potentially fatal nature of many cancer cases. Because of the urgency associated with the condition, clinicians, researchers, and healthcare organizations strive to provide timely access to resources, leading to high percentages of accelerated approval.

Although accelerated approval trials may take many forms, the FDA notes that single-arm trial designs and response endpoints support most accelerated oncology trials. Despite the seemingly complicated status of these clinical trials, there are multiple limitations, such as limited safety data.

Following accelerated approval, researchers are expected to conduct confirmatory trials to reaffirm their trial results and identify potential risks associated with the drug or treatment regimen. However, a report by the HHS in September 2022 noted many delays in these confirmatory trials.

Using data from the FDA Center for Drug Evaluation and Research from the start of accelerated approval in 1992 until December 2021, researchers determined that 104 out of 278 drugs granted accelerated approval still need to complete confirmatory trials. Roughly 34% of those are past the planned completion rate.

Understanding the limitations associated with accelerated approval, the FDA has proposed some recommendations for trials moving forward. The first of these recommendations is randomized controlled clinical trials to support approval. Clinical trial leaders can conduct two trials, one with an accelerated timeline and another concurrent trial with long-term clinical endpoints.

Beyond two concurrent clinical trials, the draft also recommends discussing single-arm trials with the FDA to determine if the use is appropriate. Finally, the organization emphasizes the importance of confirmatory trials following accelerated approval.

While this recommendation is simply a draft and does not indicate any policy changes, the FDA hopes that this will improve clinical trial outcomes and minimize post-market adverse reactions.