Bishal Gyawali: In this perspective piece out in NEJM today, we discuss finding the right balance for accelerated approval of cancer drugs in the context of the recently proposed FDA guidance.
Quoting Bishal Gyawali, an Associate Professor on LinkedIn:
”Super excited about my first NEJM publication!
In this perspective piece out in NEJM today, we discuss finding the right balance for accelerated approval of cancer drugs in the context of the recently proposed FDA guidance. The proposed guidance contains several strengths but we suggest 5 areas of improvement.
1. More clarity regarding when single arm trials are acceptable for AA. AA based on single-arm trials should be exception reserved for rare cancers with no treatment options.
2. PFS should not be considered a clinical endpoint for confirmatory trials. Improving PFS without OS or QOL does NOT confirm clinical benefit.
3. What constitutes clinical meaningfulness as opposed to statistical significance alone should be better defined.
4. More explicit recommendations on other aspects of trial design and interpretation is needed. For example, issues related to appropriate control arm treatment, informative censoring, crossovers etc.
5. Swift action for automatic withdrawal of AA when the confirmatory trial fails to confirm clinical benefit.
We hope that these suggestions are considered when drafting the final guidance. I am very thankful to Aaron Kesselheim and Joseph Ross for their invaluable mentorship and collaboration.”
For the article click here.
Source: Bishal Gyawali/LinkedIn