Last week the EveryLife Foundation wrote to President-Elect Biden and his Transition Team laying out our rare disease community’s policy recommendations to be included for consideration as their health advisors set an agenda for 2021 and President Biden’s first term. The recommendations were focused on the issues prioritized through EveryLife’s Community Congress Working Groups, with a particular focus on actions that can be taken by the new Administration specifically.
In the letter, EveryLife stressed the urgent need to accelerate and Inform rare disease therapy development & infrastructure, improve access and coverage for early and accurate diagnoses and care and respond to the COVID-19 pandemic.
The letter noted that while exciting advances in science and medicine have propelled new rare disease therapies into reality, currently 93% of the over 7,000 rare diseases still have no FDA approved treatment. Further many of the transformational therapies that are approved then face access barriers that prohibit or delay life-changing treatments from reaching patients. To overcome these barriers and others facing the rare disease community, the letter included recommendations such as;
- The establishment of a Center of Excellence for Rare Diseases at the FDA
- Within the proposed Center of Excellence, the creation of a Rare Disease Advisory Committee
- Support for the newborn screening program including working with Congress to pass the Newborn Screening Saves Lives Reauthorization Act and the inclusion of robust funding requests for newborn screening programs within the Administration’s budget request
- Support for increased funding of rare disease research including initiatives like the Rare Disease Clinical Research Network and the Undiagnosed Diseases Network
Additionally, the letter noted the important concerns about the COVID-19 vaccine in the rare disease community, informed by the recent US Rare Disease COVID-19 Vaccine Survey and urged the new Administration to consider how the lessons learned during the pandemic can inform long-term policy change in areas such as increased access to telehealth and home health services among others.
EveryLife, in conjunction with the Community Congress Working Groups, will continue to engage around these and other issues as the new Administration finalizes their health teams and begins to assess opportunities to address the significant unmet needs in the rare disease community.
