Lawmakers Introduce Cures 2.0 Legislation

Nearly five months after releasing their initial 21^st Century Cures 2.0 draft, Congressman Fred Upton (R-MI) and Congresswoman Diana DeGette (D-CO) introduced the long-anticipated bill on November 15. The bill includes provisions that were hailed by patient groups, life sciences companies and other key stakeholders as setting the stage for key innovations in research and therapy development.

The duo, who led a years-long push to enact the very popular and bipartisan 21^st Century Cures Act in late 2016, are hoping for an encore performance. The original Cures Act included provisions aimed at spurring drug and medical device development, advancing the use of patient-focused drug development approaches, expanding access to treatments, and addressing the opioid crisis. It enjoyed bipartisan support at the time of its passage and has triggered variety of new programs and activities at the U.S. Food and Drug Administration (FDA), National Institutes of Health (NIH), and Centers for Medicare and Medicaid Services (CMS).

Cures 2.0 has been long in the works, with the bill introduction coming nearly two years after the lawmakers released a November 2019 call for ideas. Upton and DeGette initially had hopes that Cures 2.0 could be enacted in 2020 and precede the 2022 scheduled update of the Food and Drug Administration’s prescription drug user fee authorization (PDUFA) law. The COVID-19 pandemic, along with a Congress largely focused on the pandemic response and the reconciliation package, has hindered Upton and DeGette’s ambitions for near-term action, thus the path forward for the legislation remains unclear. But the unveiling of the latest draft, which is largely consistent with the June discussion draft, marks another tangible step forward.

The newly introduced bill is framed by five major headings: medical research, public health, patients and caregivers, payment and reimbursement and FDA regulatory policies. Like the prior draft, it includes several standalone measures as well as items that are popular with leading stakeholders such as authorization of the Advanced Research Projects Agency – Health (ARPA-H) and provisions to advance patient-focused drug development (PFDD), streamline FDA regulatory reviews, enhance Medicare and Medicare coverage of new technologies and make permanent provisions enacted during the pandemic to expand access to telehealth within the Medicare program.

Following are examples of specific provisions within the various headings of the bill:

• Public Health: The bill authorizes research projects to better understand the impact of long COVID along with creation of plans or strategies to better prepare for future possible pandemics and to enhance vaccine awareness. It also directs HHS to establish a grant program aimed at addressing challenges faced by rare disease patients during public health emergencies. Additionally, it authorizes a subscription program to pay for antimicrobial drugs to encourage innovation in that space.
• Patients and Caregivers: This section of the bill includes provisions to increase diversity of participation in clinical trials including through commissioning a Government Accountability Office (GAO) study to identify barriers to enrollment and tasking HHS with an education and awareness campaign, and it requires companies to collect — and FDA to use in its regulatory reviews — patient experience data. Reflecting a growing focus in the advocacy community and Congress on the role of caregivers in addition to patients, Cures 2.0 would also fund educational programs to help caregivers complement clinical care activities. In addition, CMS would be required to collect input on how to promote better health literacy by collaborating with federally subsidized health care programs.
• FDA Reforms: It also authorizes two additional FDA Centers of Excellence, including one on rare diseases, and lays a foundation for greater use of real-world evidence (RWE). Greater use of patient experience and RWE data remain popular with many patient and industry stakeholders who hope their use can accelerate approval of therapies and treatments. This section would also direct FDA to provide grants to grow the science of innovative trial designs and patient experience data, both topics that have been priorities for the agency in recent years and align with proposed activities for PDUFA VII.
• CMS and Coverage Reforms: The bill includes legislation to make permanent the public health emergency changes to Medicare telehealth policy that have helped significantly expand the use of telehealth in Medicare — as well as legislation to increase use of telehealth in Medicaid. It would also seek to drive greater collaboration between FDA and CMS regarding breakthrough therapies, authorize a demonstration program in up to 15 states to expand use of genomic and genetic testing and — in a nod to the informational technology challenge at CMS — task HHS with reporting to Congress on the state of the agency’s computer system. The package also contains a provision to codify a Medicare program to cover breakthrough medical devices just days after a rule to repeal the program issued during the latter months of the Trump administration was finalized.
• Research: In addition to authorizing ARPA-H, Cures 2.0 allocates $25 billion to several federal agencies that fund research to address costs incurred as a result of the COVID-19 pandemic, notably the shuttering of labs and interruption of various research projects.

Regarding ARPA-H, the bill follows introduction of a standalone bill last month by Energy & Commerce Committee Health Subcommittee Chairwoman Anna Eshoo (D-CA) to authorize the agency within the Department of Health and Human Services (HHS). Unlike the Eshoo bill, Cures 2.0 would specifically place the agency within the National Institutes of Health (NIH). Inside or outside of NIH along with authorization levels are major policy questions pertaining to the agency, which is was proposed by the Biden administration in their Fiscal Year 2022 budget and is intended to generate transformational discoveries in medical research.

Political Outlook

Both DeGette and Upton are passionate about this bill, and it has been greeted with enthusiasm by many stakeholders. Although the sponsors recently expressed a desire to get the bill passed before the end of the year, the packed congressional agenda and unclear signals of interest and support from chamber and committee leadership as well as the Senate are all potential obstacles. Given the calendar, an increasingly likely scenario may be that elements of the bill feed into the PDUFA VII package, which will be the subject of congressional hearings and other activities in spring 2022 and must be authorized by the end of September 2022. It is also possible that some aspects of the bill that are high priorities for both chambers and/or the White House could be incorporated into a year-end omnibus package for Fiscal Year 2022 appropriations. Stakeholders will want to pay close attention given all that is going on in Congress as the next few weeks will likely generate some additional clarity on the path forward for Cures 2.0.