The Food and Drug Administration launched a docket to require inputs and comments from patients, patient advocates, the scientific community, health professionals, regulatory and health authorities and general public regarding practical steps to establish a rare diseases clinical trial network.
Enpr-EMA response to the FDA’s request for information and comments on Rare Disease Clinical Trial Networks has been published on the regulations.gov page, where you can also see other contributions. The document has been prepared by some appointed members from the Enpr-EMA Coordinating Group, including the TEDDY Network, that is cited as a relevant initiative to liaise with and that can contribute to create the framework of collaboration.
The overall aim of the FDA initiative will be to provide a more cooperative approach and common strategy to support the conduct of clinical trials in rare diseases populations. In particular, with this request for information and comments, FDA is interested in investigating what work is currently being done and what work needs to be carried out to improve the design, conduct and completion of rare diseases clinical trials. As underlined also in FDA docket, the goal is to join efforts and create a global network connected with the most relevant research initiatives in the world related to rare diseases in order to support innovation and quality in the drug development pipeline for rare diseases.