Included in 21st Century Cures & Reintroduced in the 114th CongressOpenActLogoColorCMYKOL

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. This is a major positive development as Chairman Upton (R-MI) has expressed interest in having the legislation voted on by Memorial Day.


In addition, Reps. Bilirakis (R-FL), Butterfield (D-NC), and McCaul (R-TX) have co-sponsored the stand-alone version of the OPEN ACT, which was officially introduced on Friday, February 13th as HR 971. The bipartisan bill introduction was in concert with strong support from the patient community, with 135 organizations signing-on.


The EveryLife Foundation is still looking for supporting patient organizations to support. Please contact Max Bronstein – to sign-on today.

Overview of Legislation Benefiting the Rare Disease Community

Passed in the 113th Congress:

  • Gabriella Miller Kids First Research Act: Redirects $12.6 Million in funding from political conventions to the National Institutes of Health (NIH) Common Fund to support transformative research for childhood cancer, autism, Fragile X syndrome and other rare pediatric diseases. With the odds stacked against us, more than 70 organizations called on Congress and ensured this essential funding was in the 2015 omnibus appropriations bill.

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Senators Hatch (R-UT) & Bennet (D-CO) Introduce Dormant Therapies Act

From the Office of Senator Hatch:

Washington, D.C.—Senators Orrin Hatch, R-Utah and Michael Bennet, D-Colorado, introduced the Dormant Therapies Act, a bill that will establish a new class of pharmaceuticals known as “dormant therapies” eligible for 15 years of data protection.  This provision will remove the “ticking patent clock” conundrum that forces companies to  prioritize research based on which compounds can be brought quickly to market.

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Sign-on to Support the OPEN ACT: Encourage Companies to Repurpose Drugs for Rare Diseases!

Take action to support the OPEN ACT, HR 971 (Orphan Product Extensions Now, Accelerating Cures & Treatments), which helps remove drug development roadblocks and incentivizes the development of treatments for the benefit of all rare disease patients. Biopharmaceutical companies possess a wealth of scientific knowledge from drugs that have already been approved for common diseases, some of which may have the ability to treat rare diseases.

However, there are inadequate incentives in place for companies to study potential impact for rare disease patients. Some patients may elect to take an approved drug for off-label use, in the hopes that it could treat a rare disease. Off-label usage, however, is typically not studied or tracked for clinical effectiveness. In addition, off-label use is not reimbursed by insurance plans, creating undue and unnecessary financial burdens on patients.

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The Immune Deficiency Foundation Requests Support for Legislation: H.R. 460 Patients Access to Treatment

Under many private insurance plans, patients with rare diseases continue to pay extremely high out-of-pocket costs because their prescribed medication is often placed on a “specialty tier.” Specialty tiers allow plans to impose high coinsurance, in lieu of a co-payment for expensive drugs resulting in higher out-of-pocket spending for patients.  Not only does this place a financial burden on patients, but it has also been shown to discourage adherence to treatment, which can increase overall costs to the healthcare system.

H.R. 460, the Patients’ Access to Treatments Act, would require plans to limit cost-sharing requirements for specialty drug tiers to the level of cost sharing required for non-preferred brand drug tiers. Insurers would be able to charge no more than 10% more for specialty drugs over what is charged for the next lowest tier (non-preferred brand).  For example, if an insurer is currently charging $30 for non-specialty tier brand drugs, HR 460 would require the insurer to charge no more than $33 for specialty drugs. 

Every day, Members of Congress hear from thousands of constituents with serious issues and valuable causes. Raising awareness of the impact this legislation will have on Americans throughout the country has inspired 135 Members of Congress to stand in support of HR 460.  Click here to learn more and to sign on to their letter in support of H.R. 460 PATA.

BioCentury Pens article on popularity of ‘Right To Try” Laws

States’ Rights
By Steve Usdin, Washington Editor
Published on Monday, June 30, 2014

State right-to-try laws, which have been signed by a Democratic governor in Colorado and a Republican in Louisiana, are responses to deep dissatisfaction with the pace of drug development and frustration with the ways biopharma companies have responded to pleas for early access to experimental drugs. Read more

Floor Vote for Newborn Screening Saves Lives Reauthorization Act (H.R. 1281) may be coming this week

The House is scheduled to vote this week – as early as Tuesday – on the Newborn Screening Saves Lives Reauthorization Act (H.R. 1281). Attached is the coalition letter, signed by the EveryLife Foundation as well as many other Rare Disease groups, urging House leadership to schedule this important vote. This bipartisan legislation will help ensure that infants continue to receive comprehensive, effective and lifesaving screenings for genetic and endocrine conditions soon after birth.

NBSSLRA(HR1281) House Vote Letter

Sign on to Support the Undiagnosed Diseases Research & Collaboration Network (HR 1591)

The “Network” created by H.R. 1591 will help reduce the length of time and number of physicians it currently takes to diagnose patients with a rare disease by creating a professional physician collaboration network and providing a means to collect and analyze information on undiagnosed cases.  Add your organization to the list of supporters by contacting Heather Long at

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New Op-Ed With Details on 21st Century Cures Initiative

by Rep. Fred Upton,U.S. Congressman representing Michigan’s 6th District &  Rep. Diana DeGette, Chief Deputy Whip

Originally published by The Huffington Post on May 6th:  Talk to any American adolescent and it quickly becomes clear that keeping pace with 21st century technology is a constant challenge. But staying on top of today’s technology goes far beyond trending hashtags and Instagram likes; it presents a unique opportunity to bring researchers, innovators, caregivers, and patients together in a new way that advances our collective understanding of disease and how we combat it.

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BREAKING NEWS: SGR Deal Includes Two of MODDERN Cures Provisions

Patients advocated for MODDERN Cures at RDLA’s Lobby Day last month.  Our advocacy efforts helped move this important legislation:
Last night, Senate Majority Leader Harry Reid and House Speaker John Boehner agreed on a one-year patch to prevent a drastic cut in physician Medicare payments. The bill, which temporarily suspends the sustainable growth rate (SGR), includes two provisions from MODDERN related to the development of diagnostic tests.

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