On January 20, 2022, the Rare Disease Legislative Advocates (RDLA) hosted its first monthly webinar for 2022. In this month’s program, the speakers provided an overview of the progress that was made last year as well as the anticipated policy goals in 2022. This overview touched upon legislation that is specifically relevant to the rare disease patient community.
Speakers for this month’s webinar included:
- Nicholas Manetto, Principal Consultant, Faegre Drinker
- Jamie Sullivan, Director of Public Policy, EveryLife Foundation for Rare Diseases
- Ellie Dehoney, VP of Policy and Advocacy, Research!America
- David Davenport, Public Policy Manager, Personalized Medicine Coalition
- Jennifer Dexter, Policy Director, National Health Council
Nicholas started off the session with an overview of the situation in Congress. In all likelihood, the level of legislative progress is likely to be low until the mid-term elections. Some of the goals for 2022 that are in the spotlight include:
- FDA user fees (Prescription Drug User Fee Agreements, or PDUFA): Congress will have to act by 9/30 to reinstate user fees
- Telehealth extension: Legislation that will extend policies implemented during the pandemic to enhance telehealth access
- Reauthorizing Medicaid funding for the five US territories
- Pandemic responses
Jamie Sullivan says that Congress will have to begin work early in the year in order to enact PDUFA. There are important new provisions in this iteration for the rare disease community, such as pilot projects, rare disease endpoint developments, and changes to the review process.
Another major focus has been CURES 2.0, or the 21st Century Cures Act. The latest version was introduced in November last year and also has several valuable pieces for rare disease, such as the Rare Disease Center of Excellence, improvements to genetic testing access, and more. Unfortunately, the path forwards remains hazy, but sponsors in the House are committed to making advances in 2022.
The Speeding Therapy Access Today (STAT) Act is another critical bill that is on the table in 2022. This includes several provisions that are critical to the rare and ultra rare communities and it was first introduced in March last year.
Newborn Screening has been another area of concern. The Newborn Screening Saves Lives Reauthorization Act expired in 2019, so re-implementing it is a vital goal on the federal level.
RUSP alignment is the component that has been running on the state level, and there was some real progress last year in this area, with multiple states passing RUSP alignment legislation. These laws set an accelerated timeline for states to add new diseases to their screening panels.
Ellie focused more on the research side, and Research!America’s priorities for 2022 include advocating for increasing funding for the NIH and medical research as a whole. Focuses include medical nutrition and wound care, which are traditionally ignored areas.
David discussed some of the progress the Personalized Medicine Coalition has seen, such as the creation of a Personalized Medicine Caucus. PDUFA and CURES 2.0 have also been areas of focus for them. A special piece that was included in CURES was the Precision Medicine Answers for Kids Today Act, which would conduct a demonstration program of genome and genetic testing in children. This would also implement a study from the National Academy of Medicine that would seek to evaluate how testing would improve medical outcomes.
Jennifer touched on areas where the National Health Council plans to play an active role, such as in issues like:
- Healthcare costs
- Medical innovation
- Patient access
- Improving coverage
Rare Disease Week is also coming up from February 22 to March 2. This event will be held virtually again this year, but it will still be a critical moment for rare disease policy advocacy.
To learn more and get registered, click here.
