EveryLife and Patient Organizations Urge Congress to Open Access to Potentially Life-Saving Gene Therapy

In a letter sent to Congress this week, The EveryLife Foundation, along with Parent Project Muscular Dystrophy, the National MPS Society, the MLD Foundation and dozens of patient advocacy organizations, urged Congress to enact policies that would help ensure prompt and equitable access to transformative, potentially curative treatments, such as gene therapy.

The coalition of patient organizations submitted the letter at a time when Congress is considering various proposals that explore novel payment arrangements for treatments such as gene therapies. The coalition is united in support of a flexible framework that would enable biopharmaceutical companies and payers (insurance plans, provider networks, etc.) to develop customized approaches that ultimately foster patient access.

Gene therapy has the potential to result in not only a better quality of life and patient health outcomes for our patients who are candidates for treatment, but also long-term savings to them individually and the larger health care system. Congress must also consider the value of providing relief to all those affected by genetic diseases of the considerable financial, psychological, and other challenges that these conditions present for all family members. The benefits of these potentially life-saving innovations will not be realized until patients have a pathway to access. Time is of the essence.

View the letter sent to Congress.

About the EveryLife Foundation for Rare Diseases

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy. The Foundation does not speak for patients but instead provides the training, education, resources, and opportunities to make patient voices heard. By activating the patient advocate, the Foundation believes it can change public policy and save lives.