Posted by Michael Wonder on 01 Jul 2024
FDA opens doors for more treatments for rare diseases through the new START Pilot Program
27 June 2024 - We are at a turning point in understanding the science that is opening doors to rare disease treatments that were unthinkable only a few years ago, such as gene therapies and drugs that work on gene expression.
Despite this progress, there are still many rare diseases that lack treatment options, and we need to do more to alleviate the suffering of patients and their families. For this reason, the FDA is taking steps to help accelerate the development of novel drugs and biologics through the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.
