USA – FDA seeks input on rare disease clinical trials network

To further the establishment of a rare disease clinical trials network, the US Food and Drug Administration (FDA) is asking for input from the public and a broad array of stakeholders.
In a press release, the agency noted that it is “looking to provide a more cooperative approach” in supporting the drug development pipeline for rare diseases.
The call for information comes as the FDA is launching a Rare Disease Cures Accelerator. This accelerator will establish common platforms and approaches that can support rare disease characterization and the development of standard core clinical outcome assessments and endpoints pertaining to rare diseases, as well as provide support for conducting clinical trials in rare disease populations.
These efforts began in 2019 with a $10 million Congressional appropriation. Early initiatives focused on disease characterization and standardizing data measures; the FDA is now turning to the support of clinical trials…