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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


154 Patient Organizations Urge U.S. Senate to Support OPEN ACT, S. 1421 21st Century Cures

A new letter signed by 154 patient organizations urges Senators Lamar Alexander (R-TN), Chairman of the Senate Health, Education, Labor and Pensions (HELP) Committee, and Patty Murray (D-WA), Ranking Member of the Committee, to support the Orphan Product Extensions Now, Accelerating Cures & Treatments (OPEN ACT), S. Read More


We Applaud FDA for Two New Draft Guidances on Rare Disease Drug Development CTP-1

 fda-logoLast month, the FDA outlined their new toxicology requirements for ultra-rare diseases in their draft guidance for industry, titled “Investigational Enzyme Replacement Therapy Products:  Nonclinical Assessment.”  The guidance potentially allows for only three months of chronic toxicology animal studies if there are no adverse findings, which is a significant improvement in policy. Read More


EveryLife Foundation Submits Comments on 21st Century Cures Initiative 21st Century Cures

Starting in 2014, House Energy & Commerce (E&C) Chairman Fred Upton (R-MI) and Representative Diana DeGette (D-CO) embarked on an ambitious and bipartisan effort to improve our nation’s biomedical research and innovation system – the 21st Century Cures Initiative.

The powerful E&C Committee convened dozens of roundtables, hearings, and opportunities for public input and comment as they set out uncover biomedical policy challenges. Read More


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