New Cures 2.0 Legislation Advances Biomedical Research and Care

Leading legislators have launched the campaign to enact a second version of the 21st Century Cures Act, building on the landmark measure approved by Congress in 2016. Reps. Diana DeGette (D-CO) and Fred Upton (R-MI) again have teamed up to advance a comprehensive (170 pages) bill that would provide billions to establish the new Advanced Research Projects Agency for Health (ARPA-H), enhance pandemic preparedness, and provide added authorities and resources for FDA [see]. Many of its provisions have been applauded by the medical community and biopharma manufacturers, but enactment is not likely any time soon, as a host of legislative priorities already are backing up on Capitol Hill.

A likely scenario is that next year, Cures 2.0 will be incorporated into a broader legislative package to reauthorize several FDA user fee programs that provide critical funding for the agency. Revised fee programs for drugs, generic drugs, biosimilars, and medical devices need to be approved by next summer to meet the Sept. 30, 2022 deadline, a scenario that encourages policymakers to add to the fee measure a wide range of pet projects and policy updates related to public health, biomedical research, and access to medicines and care.

The new Cures legislation similarly aims to build support by including provisions that relate to the COVID-19 pandemic, support patients, enhance FDA programs, and expand Medicare coverage of services and products. The bill seeks further study of factors underlying long COVID, along with development of a national strategy for responding to future pandemics and new models to support development of novel antimicrobial drugs. There’s added support for caregivers and patients and provisions to improve diversity in clinical trials and make broader use of patient experience data in research.

FDA would advance digital health technologies, expand the use of real-world evidence, and support novel trial designs, such as decentralized trials. The policymakers also call for FDA to accelerate designations for breakthrough and regenerative medicines, with specific attention to developing more information on CMC (chemistry, manufacturing, and controls) for expedited programs. FDA and the Centers for Medicare and Medicaid Services would communicate earlier on breakthrough therapies to facilitate coverage, and sponsors could utilize a broader range of evidence to fulfill postapproval study requirements for drugs receiving accelerated approval.

The measure also looks to continue certain more flexible coverage policies authorized for the health emergency, notably wider use of telehealth under Medicaid and Medicare and continued coverage for breakthrough devices and other innovative technologies. It facilitates diagnostic genetic testing of children with rare diseases and authorizes pharmacogenetic consultations for the elderly.

The House Energy & Commerce Committee will consider the measure with an eye to gaining agreement on a package likely to win Congressional approval next year. In budget deliberations earlier this year, the legislators dropped the Biden administration’s pricey ARPA-H proposal, and that may occur again as federal funds get tight. Industry supports the overall bill but will not want any extensive policy debate to delay user fee reauthorization.