In early 2009, Dr. Emil Kakkis launched the EveryLife Foundation to accelerate biotech innovation for rare diseases. Dr. Kakkis is best known for his work over the last 19 years to develop novel treatments for patients with rare disorders, including MPS and PKU.
The EveryLife Foundation leverages public policy to improve, streamline, and accelerate the drug development process. We work to empower the patients and families of the rare disease community, collaborate with research and industry experts, and engage policy makers who can enact change. We offer a broad spectrum of programs and initiatives to promote the understanding of regulatory challenges and to enable advocates to have an impact on public policy. One of the Foundation programs that has been very successful is Rare Disease Legislative Advocates (RDLA).
In 2009, the Foundation launched a new program to empower patients, caregivers and others in the rare disease community to become legislative advocates at the state and federal levels. Rare Disease Legislative Advocates (RDLA) educates patients about urgent public policy issues and provides them the opportunity to engage with their elected representatives. RDLA hosts a number of advocacy events throughout the year, including Rare Disease Week which hosts more than 250 advocates in Washington D.C., as well as quarterly Rare Disease Congressional Caucus briefings, regional Legislative Conferences, In-District Lobby Days during the summer Congressional recess and the RareVoice Awards which honor patient advocates as well as Congressional staff and government agency staff making a difference for rare disease patients. The Foundation also supports the rare disease community through a grant program, Rare Giving, and holds an annual Rare Artist contest to showcase the artistic talent of the rare disease community.
We are trying to reach patients, caregivers, physicians and others in the rare disease community and empower them to meet with their state and federal representatives to urge the reform necessary to spur the development of more treatments for rare disease. We provide travel stipends through our Rare Giving program to ensure that regulators and policymakers hear directly from patients and their caregivers.
The Foundation’s efforts to increase the predictability of the regulatory process led to the inclusion of legislative language (ULTRA/FAST) in 2012 to improve access to the Food and Drug Administration’s (FDA) accelerated approval pathway for rare diseases, which has been pivotal in enabling rapid access to novel and life-saving treatments for patients. The Foundation is working closely with the rare disease community and Members of Congress to advance the 21st Century Cures Act. A key victory was the inclusion of The Orphan Product Extensions Now, Accelerating Cures and Treatments (OPEN ACT), which would grant an additional six months of exclusivity for drugs that are repurposed for rare disease indications, in the legislation which passed the House of Representatives with broad bipartisan support in July 2015.
Here are some statistics:
- 160+ rare disease organizations support the OPEN ACT.
- 130 organizations were represented at Rare Disease Week on Capitol Hill.
- $100,000 given back to the rare disease community annually through Rare Giving.
Hints and Tips
We believe collaboration with all patient groups and other organizations in the rare disease space is essential to speed treatments to the rare disease patients who need them. Educating patients to be advocates for their cause not only empowers them but helps them feel they have some control in dealing with the difficulties of having or caring for someone with a rare disease.