Long-Awaited 21st Century Cures Act 2.0 Draft Released

Following months of anticipation by stakeholders including patient organizations, research institutions and medical product developers, Representatives Fred Upton (R-MI) and Diana DeGette (D-DO) released a 21^st Century Cures Act 2.0 discussion draft on June 22.

The bipartisan duo is striving to enact the sequel to their much-embraced 21^st Century Cures Act that became law in late 2016. The original Cures law was assembled over more than two years and included scores of provisions focusing primarily on medical research and Food and Drug Administration (FDA) regulatory issues. Of note, then-Vice President Biden played a significant role in the final passage of Cures 1.0 during the waning days of the Obama administration. The 2016 law included a section named after Biden’s late son to authorize the Vice President’s Cancer Moonshot at the National Cancer Institute.

The effort to develop Cures 2.0 began in late 2019 and included release of a concept paper in spring 2020 — but with the COVID-19 pandemic and other challenges, the effort did not gain much traction beyond that point.

The latest discussion draft expands upon the 2020 concept document and lays out myriad policy proposals under several domains including public health, patients and caregivers, FDA regulatory policies, Centers for Medicare and Medicaid Services (CMS) coverage policies and medical research. Like Cures 1.0, it includes a mix of ideas including several bills championed by other members of Congress.

Highlights of Cures 2.0 include the following:

Medical Research

• The bill makes good on previous statements from its leads that it would authorize an Advanced Research Projects Administration – Health (ARPA-H) that has been proposed by President Biden to pursue transformational breakthroughs in medicine. The discussion draft includes a placeholder for ARPA-H authorizing language while the bipartisan duo continues to solicit feedback from stakeholder on how to structure this new entity.
• The bill also authorizes funding for research grants from multiple agencies that fund scientific researchers to institutions, covering the costs of research disruptions related to the COVID-19 pandemic. Upton and DeGette are sponsoring a standalone bill known as the Research Investment to Spark the Economy “RISE” Act on this issue.

Public Health

• Authorizes a long-term study of those impacted by long COVID as well as a learning collaborative to understand the effects of long COVID.
• Multiple provisions focus on learning lessons from the pandemic to be better prepared for future challenges. This includes a proposal to incent development of novel antimicrobial drugs through which the government would use a subscription-based model to compensate companies for developing such therapies.

Clinical Trials and Therapy Development

• The bill includes provisions to increase diversity of participants in clinical trials as well as provisions to increase FDA’s use of patient experience data in reviewing therapy applications and to expand the use of real-world evidence (RWE).
• Authorizes the creation of two new FDA Intercenter Institutes also known as Centers of Excellence, including one that would focus on rare disease issues. Such centers were originally authorized in Cures 1.0, and FDA established the first such center in oncology in early 2017. Many other disease advocates would like a center to better coordinate agency activities, policies and product reviews.
• The bill includes provisions to improve FDA-CMS communication in the area of breakthrough therapies and would allow an array of studies, including patient registries and those using RWE, to satisfy post-marketing approval requirements under the Accelerated Approval Pathway. The Accelerated Approval pathway has been subject to much criticism lately with stakeholders including the Medicaid and CHIP Payment and Access Commission (MACPAC) recommending larger Medicaid rebates until confirmatory studies are finalized. Further, the Institute for Clinical and Economic Review (ICER) has proposed multiple reforms.
• The legislation also contains proposals to fund grants to support innovative trial designs and use of patient experience data, as well as requesting a report on activities to align regulation of digital health technologies.

Coverage Policies

• Recognizing the gains made in access to telehealth during the Public Health Emergency (PHE), the bill proposes to make such gains permanent by eliminating Medicare’s geographic and originating site requirements and by expanding both the category of providers and types of services eligible for delivery and payment via telehealth. Should Congress fail to act before the end of the PHE, Medicare beneficiaries will no longer have access to these telehealth services. It also requires guidance to facilitate expanded access via telehealth under the Medicaid program.
• The bill would codify in law the Medicare Coverage of Innovative Technologies (MCIT) regulatory pathway that was finalized at the end of the previous administration but has been delayed twice by the Biden administration.
• Establishes a five-state Medicaid pilot program to study coverage of genetic and genomic testing to improve clinical outcomes.
• Authorizes Medicare to cover “genomic precision medicine consultations” by eligible healthcare providers.

Path Forward and Outlook

While the release of the discussion draft is a significant step, the path forward remains unclear. A number of challenges seem likely, including concerns the Democratic majority may have with certain provisions that could be viewed as being too pro-industry as well as an interest in having a concurrent debate on policies to address drug pricing and access.

The calendar remains another possible hurdle, as Congress needs to reauthorize the FDA’s prescription drug user fees (PDUFA) by September 2022. While Cures 1.0 was enacted in 2016 and PDUFA VI was enacted the following year, the truncated timeline could pose a challenge this go-around, and the two measures could run together. And while Reps. Upton and DeGette drove Cures into law in 2016, Upton does not currently hold the committee chairmanship, meaning there is less direct control of the agenda.

Given this uncertainty, those interested in advancing provisions in the draft or adding additional measures should engage aggressively while exploring multiple pathways.