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Join Us

We are asking Patient Organizations to partner with us to help share our goals with their members and when the time is right - to reach out to the FDA and Congress to help implement new regulatory policy that will put Orphan Treatments on the FAST TRACK.

Recent Updates


OPEN ACT (HR 971) Included in 21st Century Cures & Reintroduced in the 114th Congress 21st Century Cures

Support for the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures & Treatments) on Capitol Hill is growing. The bill provides incentives for companies to repurpose or “rarepurpose” existing treatments for rare disease indications. The legislation was included in the draft 21st Century Cures discussion bill released by the Energy & Commerce Committee. Read More


Journal Pens Article on Benefits of Repurposing General

From the Annals of Clinical and Translational Neurology:

Overcoming Obstacles to Repurposing for Neurodegenerative Disease

Repurposing Food and Drug Administration (FDA)-approved drugs for a new indication may offer an accelerated pathway for new treatments to patients but is also fraught with significant commercial, regulatory, and reimbursement challenges. Read More


Rare Disease Patient Voices Bring Change To The Clinical Trials Process 21st Century Cures

From an article in Forbes by Steve Smith:

The voices of rare disease patient advocates play a unique and critical role in the modernization of the clinical trials process, which advocates see as antiquated and unable to sufficiently address their unmet medical needs. Read More