The EveryLife Foundation for Rare Diseases: February 2015

The EveryLife Foundation for Rare Diseases: February 2015

Novato, Calif.
Accelerate biotech innovation for rare disease treatments through science-driven public policy.

Currently, there are fewer than 400 approved treatments for 7,000 rare diseases affecting more than 30 million Americans. The science exists for many of these diseases to be treated; however, treatments may never be developed because of roadblocks in the development process, such as a lack of investment and a challenging regulatory environment. The EveryLife Foundation for Rare Diseases works with patient organizations, industry, academic scientists, the Food and Drug Administration (FDA) and National Institutes of Health (NIH) to improve the clinical development process through efforts that include the CureTheProcess 2 campaign, the Rare Disease Scientific Workshop and legislation. And while the Foundation's primary focus is on regulatory issues, it also works to engage the rare disease community by spearheading programs that fulfill unmet need for patients and by supporting other organizations with similar goals.

Of the thousands of rare diseases impacting  Americans today, 95 percent have no treatment whatsoever, and many of the existing treatments are only able to address the symptoms of these ailments. While the National Institutes of Health's (NIH) support of basic research is an incredibly important component to finding cures for these types of diseases, the EveryLife Foundation for Rare Diseases also strives to create more incentives for industry to develop treatments.

The foundation's current campaign, CureTheProcess2, is a grassroots, patient-driven advocacy campaign to remove roadblocks in the drug development process, with three unique goals. The first of these is to encourage the FDA to accommodate a more scientifically rational and flexible application of safety data to allow U.S. patients to have access to early stage clinical trials. The second is to encourage the FDA to create more specialized drug review divisions and allow reviewers access to the latest science to enhance their understanding of the diseases they are reviewing. And finally, the new Orphan Product Extensions Now Accelerating Cures & Treatments (OPEN ACT) legislation would increase incentives for companies to explore the repurposing of already approved drugs for the treatment of rare diseases.

EveryLife also recognizes the compelling effect that sharing personal stories can have on policymakers, and works hard to make the patient voices of the rare disease community heard in Congress. From February 23-27, 2015, the Foundation, through its Rare Disease Legislative Advocates (RDLA) program will host Rare Disease Week on Capitol Hill, including Rare Disease Lobby Day, during which more than 200 advocates will have the opportunity to meet with members of Congress. The foundation will also relay the personal stories of patients unable to attend.

A new initiative for the foundation, Community Congress, is a membership-based program dedicated to bringing patient organizations, industry leaders and other rare disease stakeholder organizations together. The program provides the opportunity for members to learn about the foundation's scientific and policy goals, and help provide valuable insight on prioritizing future initiatives. The congress currently consists of three working groups - public policy, science/regulatory policy and newborn screening - that collaborate on these specific issues to provide an opportunity for continued engagement throughout the year to drive policy forward.

"We view our partnership with Research!America as vital in ensuring that the NIH and FDA are fully funded, so as to foster and accelerate our nation's innovation engine to bring life-saving therapies to patients," said Emil D. Kakkis, M.D., Ph.D., president, EveryLife Foundation for Rare Diseases.

For more information, visit

Media Contacts

Tim Haynes
Senior Director of Communications 

Luck shouldn't play a role in why I'm alive.
Laurie MacCaskill, a seven-year pancreatic cancer survivor