The FDA recently held an Advisory Committee meeting to consider BioMarin’s investigatory treatment for Duchenne’s muscular dystrophy. The outcome caught many by surprise - and many were watching this AdComm as a "canary in the coal mine" for broader patient group involvement.
The Duchenne’s community has been at the forefront of working with the FDA on drafting guidance on how to bring new therapies to market for Duchenne’s, a deadly disease that has no approved treatments.
The outcome was not entirely expected. The FDA Division provided the Advisory Committee with evidence that showed an imbalance between efficacy and safety and, despite well-prepared and delivered testimony on the part of patient groups, the science under discussion pointed to ambiguous review options inside the FDA.
How does this outcome impact and inform future rare disease development and patient group involvement? YourEncore Executive Partner, Peter J. Pitts, sat down with Brian Daniels, former SVP of Global Development and Medical Affairs at Bristol-Myers Squibb, to share their thoughts.