Nov 30, 2021

 

Recently, Reps. Diana DeGette (D-CO) and Fred Upton (R-MI) introduced a bill that is universally known as “Cures 2.0.” The bill is the next iteration of legislation passed in 2016 called the 21st Century Cures Act. AASLD has been following the development of this legislation closely since the sponsors began discussions of it in 2019. The bill is 173 pages long and we cannot do justice to all its provisions here. However, we can hit some of the highpoints of interest to members.

 

Title I: Public Health

The bill would create a research focus on long Covid that includes a large national survey of self-identified patients; creation of a learning collaborative of stakeholders; NIH research grants to study long-term pediatric Covid; an NAM study of disparities in long Covid; and HHS-run education and awareness programs regarding long Covid.

The bill also requires a national testing and response strategy for future pandemics; requires the HHS Secretary to develop a plan for rare disease patients to deal with public health emergencies and a federal grant program for implementation; and creates an education program addressing the importance of vaccines.

Finally, this title addresses the creation of a subscription model to pay for novel antimicrobial drugs. HHS would provide companies with a payment to ensure a predictable return on investment and appropriate use of the drug.

 

Title II: Patients and Caregivers

The bill would fund educational programs and training for caregivers to augment a care team and complement a clinical visit; require CMS to solicit input on how the agency can work with federally subsidized health care program stakeholders to promote health literacy; require an update from FDA on diversity in clinical trials; require a GAO study on barriers to clinical trial participation; require HHS to conduct a campaign to increase awareness and understanding, particularly in minority communities, of clinical trials; and establish a task force on making clinicaltrials.gov more user- and patient-friendly.

The bill would also require drug manufacturers/sponsors to collect and report on patient experience data as part of the clinical trial; require FDA to fully consider patient experience data collected during the clinical trial and report patient experience data transparently. The bill also allows Medicare to cover the costs of their beneficiaries in PCORI-funded clinical trials.

 

Title III: Food and Drug Administration

The bill would require the HHS Secretary to submit a report to Congress on efforts to ensure collaboration and alignment across the FDA with respect to regulation of digital health technologies; provide grants in the area of innovative clinical trial design and patient experience data to build the science; and require the HHS Secretary to submit a report to Congress on the current state of cell and gene therapy regulation and foreseeable future regulatory challenges.

The bill would also require HHS to outline approaches to maximize and expand the use of RWE; establish a task force to develop recommendations on ways to encourage patients to engage in real-world data generation; and establish an automatic communication requirement between FDA and CMS for Breakthrough Therapy drugs.

In addition, the bill would direct the Secretary of HHS to establish two additional FDA Centers of Excellence, one related to neuroscience and one focused on rare diseases; fix unforeseen impediments to sponsors to promote designations for investigational drugs if they meet proper criteria: require FDA to publish guidance on the standards and factors it will employ regarding CMC data development and review for expedited programs

It would also allow the use of clinical evidence, patient registries, or other real-world evidence, to fulfill post-approval study requirements to confirm the predicted clinical benefit of a therapy and would direct the Secretary of HHS to convene a multi-stakeholder meeting to explore innovative ways and incentives to foster the adoption of decentralized trials.

 

Title IV: Centers for Medicare and Medicaid Services

The bill would require GAO to report on recommendations to enhance Medicare coverage and reimbursement for innovative health technologies; provide guidance and strategies to states on effectively integrating telehealth into their Medicaid program and CHIP, review the impact of telehealth on patient health and encourage better collaboration; permanently remove Medicare's geographic and originating site restrictions that require a patient to live in a rural area and be physically in a doctor's office or clinic to use telehealth services; and it would allow the Secretary of HHS to permanently expand the health care providers that can offer telehealth services and the services that can be reimbursed under Medicare.

In addition, the bill would codify the Medicare Coverage of Innovative Technology pathway at CMS; require the Secretary of HHS to report on the viability of alternative coverage pathways for innovative technologies; and require the Secretary of HHS to report on the current capabilities and deficiencies of CMS’s computer systems.

The bill would increase access to diagnostic testing by providing federal support for the use of genetic and genomic testing for pediatric patients with rare diseases; it would provide Medicare coverage for pharmacogenetic consultations by qualified clinical pharmacists and genetic counselors; it would prohibit the use of geographic tracking features and biometrics within EVV systems; and would ensure that advances to e-prescribing technology swiftly benefit patients.

Finally, the bill would provide clinician-led clinical data registries with access to Medicare claims data for purposes of research to improve quality and cost efficiency by linking the data with clinical data in registries.

 

Title V: Research

The bill would authorize $6.5B to create ARPA-H within NIH to improve important areas of medicine and health by funding projects that could tackle bold challenges requiring large scale, sustained coordination; create new capabilities (e.g., technologies, data resources, disease models); support high-risk exploration to establish new paradigms; and overcome market failures through critical solutions, including financial incentives.

The legislation would also provide $25 billion to independent research institutions, public laboratories, and universities to continue their work on federally backed projects that were disrupted by the PHE. It would also reauthorize the Research Policy Board to make recommendations on modifying and harmonizing regulation of federally funded research to reduce administrative burden.