UPDATED 7/16/21: View the EveryLife Foundation’s comment letter to Representatives Diana DeGette (D-CO) and Fred Upton (R-MI).
On June 22nd 2021, Representatives Diana DeGette (D-CO) and Fred Upton (R-MI) released a Discussion Draft for a 21st Century Cures Act 2.0. On behalf of the EveryLife Foundation, we thank Representatives DeGette and Upton for their unwavering leadership and commitment to building on the successes of the 21st Century Cures Act to advance biomedical research, regulatory science, public health and payment policy innovation so critical for rare disease patients and families.
In December of 2019, EveryLife provided Representatives DeGette and Upton a detailed discussion of priority provisions for a future “Cures 2.0” legislation. We were pleased to see several of these suggestions incorporated into the most current discussion draft. Several provisions of the discussion draft are particularly relevant to the rare disease community, including:
- Establishment of Additional Intercenter Institutes at the Food and Drug Administration (FDA). The creation of a Rare Disease Center of Excellence at the FDA is a priority of the EveryLife Foundation and rare disease community to address the unique challenges across rare diseases, providing solutions that can benefit therapeutic development for many communities. We are pleased to see Cures 2.0 call for the creation of two new Centers, including one focused on diseases affecting less than 200,000 people in the U.S., aligning with the key provision of the Speeding Therapy Access Today Act of 2021 (H.R. 1730/S. 670, STAT Act).
- Improving FDA-CMS communication regarding transformative new therapies. This provision would improve communication between the FDA and the Centers for Medicaid and Medicare Services (CMS) throughout the Breakthrough Therapy approval process, which has the potential to reduce delays in coverage and access after approval. The need for early and substantive communication between FDA and payors, including CMS, is another key issue that the EveryLife-supported STAT Act similarly looks to address.
- Increasing use of real-world evidence. The Discussion Draft calls for better inclusion of real-world evidence for use in regulatory decision making, including utilization of this data as part of post-approval commitments for drugs approved through Breakthrough Therapy Designation or the Accelerated Approvals pathway.
- Realizing the potential for digital health tools, including increased access to telehealth under Medicaid and Children’s Health Insurance Program, extending Medicare Telehealth flexibilities, and accelerating the use of digital biomarkers for clinical trials and real world evidence collection.
- Authorizing the creation of the Advanced Research Projects Agency for Health (ARPA-H). A health related ARPA that could take on high-risk but high-reward biomedical innovation has been a popular concept for more than a decade, but before this year no administration had proposed its establishment. A request for information has been put forward as part of Cures 2.0 to gain insights from stakeholders as to the shared vision for how this agency is structured.
- Expanding access to genetic testing. As a priority identified by the EveryLife Foundation’s Community Congress members, we were particularly pleased to see the inclusion of language establishing a demonstration project within CMS to study providing coverage for “DNA sequencing clinical services” through Medicaid for 5 states (as outlined in the Advancing Access to Precision Medicine Act, H.R. 4393). Similar to past iterations of the legislative language, Cures 2.0 would require a National Academy of Medicine study on the impact of this coverage (improved preventative care, preventative medicine, reducing health disparities), including the impact for patients with rare diseases, and a CMS report on the current state of coverage for genetic and genomic testing.
The EveryLife Foundation exists to empower the rare disease patient community to drive impactful, science-driven legislation and policy that advances the development of treatments, diagnostic opportunities, and access. “Five years ago, our community was instrumental to the success of the landmark 21st Century Cures legislation and we look forward to again working with Reps. DeGette and Upton to ensure transformative rare disease provisions are included in a Cures 2.0 package,” said Julia Jenkins, EveryLife’s Executive Director.
How can you get involved?
- Join the EveryLife Foundation and the Rare Disease Legislative Advocates for Rare Disease Week on Capitol Hill, 2021! Meet fellow advocates, learn about timely rare disease policy issues and help raise support for the STAT Act and creation of a Rare Disease Center of Excellence at the FDA.
- Join the Congressional Caucus Briefing on Wednesday, July 14. Part of Rare Disease week, the briefing will convene policy experts and rare disease stakeholders to educate Congressional staff, the public, and advocates on issues of importance to the rare disease community. You can view previous briefings here.
