Last Friday I had the opportunity to attend the National Comprehensive Cancer Network (NCCN) Policy Summit: Designing Clinical Trials in the Era of Multiple Biomarkers and Targeted Therapies. Dr Alan Venook opened the conference by discussing a new era in which we have drugs tailored to each patient's tumor. Venook discussed many issues that may arise in this exciting future.
What struck me about the discussion is that the future is now. According to a recent article in the Washington Post, the FDA has been lobbied to approve a new drug for Duchenne muscular dystrophy based on a twelve person study. According to the article, the drug may help about 2,000 boys alive in the US today and approximately 1 in 30,000 boys born.
How large the trial needs to be to satisfy the FDA depends on how effective the drug is, but if say 500 boys are needed, the trial would have to accrue at least 25% of the disease population.
This may the future. Drug's designed to target very very specific disease populations will necessary aim at very small disease populations. Disease populations that are so small that it may be impossible to design large enough studies to test the drug's effectiveness. How does the FDA approve such drugs?