Written by Lisa Van Allen, Chair of the RSDSA Advocacy Committee, for the RSDSA blog.
Representatives Doris Matsui (D-CA) and Gus Bilirakis (R-FL) and Senators Amy Klobuchar (D-MN) and Roger Wicker (R-MS), co-chairs of the Rare Disease Congressional Caucus, reintroduced the Scientific External Process for Educated Review of Therapeutics (EXPERT) Act, which seeks to formalize the Externally-Led Scientific-Focused Drug Development (EL-SFDD) meeting at the Food and Drug Administration (FDA).
This legislation seeks to bridge the gap between rare disease expertise and regulatory expertise through the EL-SFDD. These quarterly meetings will provide an opportunity for enhanced collaboration between medical experts, drug sponsors, scientific organizations, and patient advocates to discuss the challenges impacting the development of rare disease treatments, identify scientific opportunities to facilitate development, discuss novel clinical trial designs, and align on endpoints to address unmet medical needs for rare disease patients. Each meeting will focus on a different rare disease topic, and the FDA will report annually on how these sessions are helping to shape and improve its internal review process for rare diseases.
In layman’s terms, this would put researchers in the same room with patients and funders to work together toward shortening the timeline on the creation of drugs that treat rare diseases like CRPS.
For more on this bill, click here.
Contact your legislators today and ask them to support the EXPERT Act.
When you contact your congressional representatives, be sure to also ask them to support funding for Medicare/Medicaid and FDA research. Budget cuts are coming and we need to remind our representatives just how important healthcare services and research are to us. If you have a specific story about how Medicare or Medicaid covers treatment you need, be sure to include it. And then share your letter with us – we want to hear your stories and have examples to share with others.