A new survey by the Biotech Consortium to Accelerate Innovation reveals that U.S. biotech companies developing treatments for rare diseases prefer to conduct clinical trials domestically but face regulatory hurdles pushing them abroad. The consortium aims to ensure American patients have early access to innovative therapies for conditions like amyotrophic lateral sclerosis and Duchenne muscular dystrophy.
The Biotech Consortium to Accelerate ... therapies for serious neurological and rare diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Angelman syndrome, and Duchenne muscular dystrophy. BCAI's mission is to keep American clinical trials in the U.S., ... The Biotech Consortium to Accelerate Innovation (BCAI) unites more than a dozen innovative biotechnology companies developing transformative therapies for serious neurological and rare diseases, including amyotrophic lateral sclerosis, frontotemporal dementia, Angelman syndrome, and Duchenne muscular dystrophy. BCAI's mission is to keep American clinical trials in the U.S., so American patients have the earliest possible access to U.S.-developed medical innovation. CAMBRIDGE, Mass.--(BUSINESS WIRE)--A new survey reveals that U.S. biotechnology companies focused on developing treatments for rare and serious diseases overwhelmingly prefer to conduct first-in-human (FIH) clinical trials in the United States. However, historical obstacles posed by the U.S. Through strategic cost-saving initiatives, robust business partnerships, educational and networking opportunities, and proactive advocacy, MassBio empowers its members to launch the next generation of medical advancements to deliver the cures and therapies that enhance patient lives. ... 1U.S. Food and Drug Administration. Estimating the Maximum Safe Starting Dose in Initial Clinical Trials for Therapeutics in Adult Healthy Volunteers: Guidance for Industry. Instead, they are asking the FDA to implement the 10x safety margin as outlined in the Agency’s own 2005 guidance, Estimating the Maximum Safe Starting Dose in Initial Clinical Trials1. Restore predictable review timelines that reduce costly holds and rework. Strengthen sponsor-reviewer communication so trial design is driven by science, not procedural ambiguity. Ensure better alignment of review criteria with disease severity and life expectancy.
Original title: “New Survey Finds U.S. Biotechs Advancing Treatments for Rare and Serious Diseases Want to Run Clinical Trials in U.S. but Regulatory Hurdles are Sending Them Abroad”