When I was 29 and seemingly healthy, I had a stroke caused by a rare disease I hadn't known I had. I was fortunate to fully recover after a few weeks of physical therapy and months of speech therapy. I've taken several different blood thinners off-label to prevent another stroke.
I support the 21st Century Cures initiative because it would be truly transformative to our biomedical enterprise. U.S. regulations have not kept up with scientific advances, and change is desperately needed to accelerate the discovery, development and delivery of new treatments to patients.
The House Energy and Commerce Committee spent more than a year soliciting feedback from researchers, patients, physicians, leaders of the Food and Drug Administration (FDA) and National Institutes of Health (NIH), and other stakeholders to identify what improvements to the research and development ecosystem could have the greatest impact for patients. The legislation was considered by the Committee and then received broad bipartisan support when it passed the U.S. House of Representatives by a vote of 344-77 last July.
Despite this widespread support in the House, and by the more than 700 advocacy organizations that signed on to support 21st Century Cures, the U.S. Senate has not yet voted on the bill.
Time for Congress to act is running out. And time is running out for patients who need new treatments. Please call your Senators to urge them to support #CuresNow and the OPEN ACT (HR 971/ S 1421), which would provide an incentive to encourage biopharmaceutical companies to repurpose medicines for rare diseases (resulting in more FDA-approved treatments for patients).
