With an eye toward rare-disease research, the Biotechnology Industry Organization and a Duchenne patient advocacy group are joining forces to help drugmakers design clinical studies with patient input.
The BIO-Parent Project Muscular Dystrophy alliance aims to collect real-world “community-centered” snapshots that hone in on desired patient outcomes, delivery methods and manageable side effects to better inform drug development.
The alliance will issue recommendations on study designs and how the findings can be used in regulatory filings. PPMD piloted a study on the topic in 2013 and has been pushing for a strategy that will allow for special considerations on both benefit and risk.
Patient-centric efforts have seen a surge over the last year and are important components of the 21st Century Cures Act, which aims to include more personalized medicine in the drug development ecosystem.
In particular, Duchenne — which has no specific treatment — has fielded growing attention in the pharma industry, with the FDA calling for more development, allowing for more flexibility and fast-tracking several potential therapies. Still, pleas from patients and families urging for leniency in approval of BioMarin’s drisapersen fell short last month, with an advisory committee voting against recommending the candidate.