Illustration: Rebecca Zisser/Axios
The Food and Drug Administration is eyeing policy changes that could make drugmakers conduct more stringent trials to win fast-track approvals of cancer drugs.
Why it matters: The agency's accelerated approval process has drawn fire for the way it allows manufacturers to launch products based on preliminary evidence and charge high prices before they complete trials.
- Some products don't work out: Merck withdrew its blockbuster cancer therapy Keytruda for some uses after an FDA crackdown on drugs approved through the fast-track process without the required post-marketing data, per Clinical Trials Arena.
Driving the news: The FDA is proposing treatments be put through randomized controlled trials, in which drugmakers measure the effectiveness of an experimental treatment against a conventional one, instead of widely used "single-arm studies" that lack a comparison.
- Single-arm studies would be limited to specific instances, and companies would be encouraged to discuss with FDA in advance whether the expected effect is reasonably likely to predict clinical benefit.
- "Given the limitations of single-arm trials, a randomized controlled trial is the preferred approach to support an application for accelerated approval," the FDA wrote in draft guidance.
The FDA outlined two options for companies: Separate randomized trials to support the accelerated approval of the drug and to then verify its clinical benefit, or a single randomized trial with short and longer-term endpoints.
- The comment period on the guidance runs through May 26, PhRMA, the big drug industry trade group, declined comment on Monday when asked about the draft.
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