The House of Representatives APPROVES the 21st Century Cures Initiative with a vote of 344-77

RDLA applauds the House for approving the 21st Century Cures Act by a strong, bipartisan vote of 344 to 77. This landmark legislation will spur scientific discovery and bio-medical innovation, providing hope for patients and their families, while also protecting jobs and America’s leadership role in healthcare innovation.

Attention now turns to the Senate, and we look forward to working with Senators on their ideas and priorities for creating treatments and cures for rare diseases. We encourage the Senate to move quickly in advancing this legislation through the chamber and to the president’s desk.

If you’d like to know more about the contents of the 21st Century Cures Act, click here for our information page on the legislation.


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.

Read more

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.

Read more

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.

Read more

Update: Funding for Secured for Gabriella Miller Kids First Research Act for Pediatric Cancer

In April, President Obama signed the Gabriella Miller Kids First Research Act authorizing Congress to allocate additional funding for pediatric cancer research through the National Institutes of Health (NIH). Congress, however, has not taken the steps needed to unlock the funding for NIH.

Today, December 10, 2014, Funding for the bill was included in the funding package to be passed by Congress.

Congratulations! This is a great example of what we can accomplish working together as a broad coalition.

If you took action by signing-on to our letter reminding congressional leadership to prioritize funding for rare diseases like childhood cancers THANK YOU!!!


New Rhode Island Law Prescribes Affordable Medical Foods for Rare Diseases

From an Aug. 19 Valley Breeze article , Lincoln, Rhode Island:  Legislation requiring insurance coverage for special baby formula and prescribed food is now law in Rhode Island.

The new measure abolishes the mandated cap on coverage for those suffering from rare diseases and inherited disorders requiring prescribed nutrition mandates. The bills sponsored by state Sen. Ryan W. Pearson of District 19 in Cumberland and Lincoln, and state Rep. Raymond A. Hull of District 6 in Providence and North Providence, induced testimony from patients and family members of patients who require prescribed diets and formulas during the hearing process. Read more

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