Help Support the 21st Century Cures Act and the OPEN ACT


The final decisions could be made today on the 21st Century Cures Act and the OPEN ACT – a provision that could be transformational for rare disease patients. CureDuchenne signed the letter of support. Despite outreach from constituents, we are still hearing pushback from Sen. Warren on the OPEN ACT. If you could please take a moment to weigh-in with her office today via email about the critical importance of the OPEN ACT that would be tremendously helpful.

Feel free to send her office an e-mail to Here is a draft e-mail.  Please feel free to customize with specific information on what this means for Duchenne.


Dear Julia –

I’m writing today as a concerned constituent regarding the 21st Century Cures Act and one provision in particular that we believe to be critical for rare disease patients called the OPEN ACT (S 1421). The OPEN ACT is needed life-saving legislation that has the potential to double the number of treatments available to rare disease patients. [insert Duchenne-specific info if possible].

Please see the attached letter which highlights the level of support from the patient community right here in Massachusetts. We are grateful for your support of biomedical research and hope that Sen. Warren will continue her efforts to see 21st Century Cures and the OPEN ACT become law.

Here is the initial letter of support.

The Honorable Elizabeth Warren
United States Senate
Washington, D.C.  20510

Dear Senator Warren:

On behalf of the rare disease patients and their families living in Massachusetts, the undersigned organizations are writing to request your support for bipartisan legislation that would significantly increase the number of treatments for rare diseases. With 95 percent of rare diseases having no FDA-approved treatment, it is critical that Congress enact policies that will bring aid to the nearly 30 million Americans suffering from a rare disease or condition.

We urge you to support the inclusion of S. 1421, the Orphan Product Extensions Now Accelerating Cures and Treatments Act of 2015 (OPEN ACT), in the Senate’s medical research and innovation legislation being considered this fall.  This bipartisan bill sponsored by Senators Amy Klobuchar (D-MN) and Orrin Hatch (R-UT) would lead to the development of hundreds of safe, effective, and affordable rare disease treatments within the next several years by incentivizing drug makers and innovators to “repurpose” already approved drugs for life-threatening rare diseases and pediatric cancers. In exchange for investing the resources to achieve an FDA-approved rare disease indication on the medicine’s label, the OPEN ACT would grant six months of market exclusivity to repurposed drug.

Requiring drug makers and innovators to obtain FDA-approval for the rare disease treatment means that patients can have confidence that the medicines they are taking are thoroughly tested for safety and effectiveness, and that researchers have studied and determine dosage levels. This would decrease unmonitored off-label use and would clear the way for insurance coverage.

As the Senate Health, Education, Labor and Pensions Committee, as well as the full Senate, considers medical research legislation, we would ask that you join the more than 150 rare disease organizations across the country in supporting the inclusion of the OPEN ACT, S. 1421 in this legislative package.


National Organization for Rare Disorders (NORD), Quincy
The Coalition Against Pediatric Pain (TCAPP), Medfield
Rare Disease United Foundation, Attleboro
Sophia’s Fund, Marlborough
Choroideremia Research Foundation, Inc., Springfield
CureDuchenne, Cambridge
TargetCancer Foundation, Cambridge
National Tay-Sachs & Allied Diseases Association, Boston
Amyloidosis Research Consortium, Lincoln
MitoAction, Boston

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