The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. We can do more with the science we already have and bring life-saving treatments to millions of people suffering from rare diseases.
Currently, there are fewer than 400 approved treatments for 7000 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 Foundation 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 our CureTheProcess Campaign, Rare Disease Workshop Series, and legislation.
The Foundation was the driving force behind the grassroots movement that ensured the ULTRA/FAST language, to improve access to the FDA’s Accelerated Approval pathway for rare diseases, was included into the FDASIA final bill, which was signed by the President in July of 2012. The Foundation is working with rare disease stakeholders, Congress and the FDA to ensure the timely implementation of the legislation.
While the Foundation remains mainly focused on regulatory issues, it also works to grow the rare disease community by spearheading programs that fulfill unmet need for patients and by supporting other organizations working to help empower the rare disease community.
Director of Public and Government Relations
Leslie White, RN
Patient Programs Manager
Foundation and Operations Manager
Patient Advocacy Liaison
Emil D. Kakkis M.D., Ph.D.
President and Founder, EveryLife Foundation for Rare Diseases
Vice President, Commercial Planning, Ultragenyx Pharmaceutical
Managing Director, CVUS Clinical Trials LLC
Jenny Soriano, M.D.
OB/GYN, Kaiser Permanente