#FasterCures 21stCenturyCures

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The 21st Century Cures Act: 2-Year Update

Dec. 13, 2018, marked two years since the signing of the 21st Century Cures Act (P.L. 114-255). In its first two years, the Cures Act has contributed to significant advances in research, changed how stakeholders share health data, and helped modernize the biomedical ecosystem through far-reaching studies such as the All of Us Research Program and the Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative. These advances have reduced obstacles to data sharing and encouraged more patient engagement and the use of real-world data in product development and approval. In addition, the Cures Act provided research funding, prioritized increased support for early-career researchers and high-risk, high-reward research, and provided new abilities for federal agencies to more competitively hire the skilled workforce needed in a 21st Century health-care system.

Through FasterCures’ 21st Century Cures tracker, we continue to monitor the implementation of Cures Act provisions and applaud the U.S. Department of Health and Human Services (HHS) for completing requirements that are improving the biomedical innovation system to the benefit of patients. New for 2019, FasterCures will issue a series of blog posts digging deeper into the impact the Cures Act has had on specific issues of interest. Stay tuned throughout 2019!

In Progress: Some requirements of the section have yet to be completed

Complete: All requirements of the section have all been met

Monitoring: FasterCures is monitoring the use of selected (completed) sections that establish new procedures or processes  

Two Years of Impact: Implementation Highlights

Since its enactment in 2016, the Cures Act has driven improvements in how research is conducted and incentivized, how products are developed and approved, and how health care can more effectively benefit patients. Below are a few key achievements marking these first two years of the Cures Act, as well as updates to watch for in 2019.

National Institutes of Health (NIH)

Sec. 2002: EUREKA prize competitions

The Cures Act required the NIH to support prize competitions to improve health outcomes for serious diseases that represent a significant burden in the U.S. as well as to advance biomedical science. On Sept. 11, 2018, the National Institute on Aging announced its Alzheimer’s prize challenge to improve care navigation and dementia care coordination for older adults. It is accepting submissions until June 30, 2019, and will announce winners in September 2019.

Sec. 2011: Precision Medicine Initiative

On May 6, 2018, the NIH launched the All of Us Research Program, a study aimed to advance the science of precision medicine by involving 1 million participants living in the U.S. So far, the program has enrolled over 150,000 participants, about 75 percent of which are from historically underrepresented groups in biomedical research.

Sec. 2041: Task force on research specific to pregnant women and lactating women

On March 21, 2017, the NIH established the Task Force on Research Specific to Pregnant Women and Lactating Women, which held many meetings throughout 2017-2018.

On Oct. 1, 2018, the task force submitted final recommendations for research on therapies used by pregnant and lactating women.

Food and Drug Administration (FDA)

Sec. 3002: Patient-focused drug development guidance

In May 2017, the FDA released its Plan for Issuance of Patient-Focused Drug Development Guidance under 21st Century Cures Act, Title III, Section 3002.

On Dec. 18, 2017, FDA held a public workshop on “Patient-Focused Drug Development: Guidance 1- Collecting Comprehensive and Representative Input,” including issuing discussion documents. On June 12, 2018, the FDA released the draft guidance.

On Oct. 15-16, 2018, the FDA held a public hearing, “Patient-Focused Drug Development Guidance: Methods to Identify What is Important to Patients and Select, Develop or Modify Fit-for-Purpose Clinical Outcome Assessments.”

On March 19, 2018, the FDA held a public workshop entitled “Patient-Focused Drug Development: Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data.” On Dec. 21, 2018, the FDA issued the draft guidance.

Sec. 3011: Qualification of drug development tools

On July 25, 2018, the FDA published its Surrogate Endpoint Table, listing surrogate endpoints that have supported approval of drugs and biologics.

On Dec. 11, 2018, the FDA held a public meeting, “Drug Development Tool Process under the 21st Century Cures Act and [Prescription Drug User Fee Act] PDUFA VI,” to discuss the drug development tools qualification pathway for animal models, biomarkers, and clinical outcome assessments.

On Dec. 11, 2018, the FDA issued draft guidance, “Biomarker Qualification: Evidentiary Framework.”

Sec. 3021: Novel clinical trial designs

On March 20, 2018, the FDA held a public workshop, “Promoting the Use of Complex Innovative Designs in Clinical Trials,” to inform the development of draft guidance on using complex trial designs to support product approval.

On Aug. 29, 2018, the FDA launched the “Complex Innovative Designs Pilot Meeting Program,” which allows selected drug and biologic companies to discuss the use of these trial designs in their product development programs.

Sec. 3022: Real-world evidence

On Sept. 13, 2017, the Duke-Margolis Center for Health Policy, supported by a cooperative agreement with the FDA, held a public workshop, “A Framework for Regulatory Use of Real-World Evidence,” to discuss the use of real-world data and real-world evidence in drug development and regulatory decision making.

On Dec. 6, 2018, the FDA released its strategic framework to evaluate the use of real-world evidence to support the approval of a new indication for a previously approved drug or to support or satisfy post-approval study requirements.

Sec. 3042: Limited population pathway

The Cures Act established a limited population approval pathway for antibacterial and antifungal drugs. On June 13, 2018, the FDA issued its draft guidance, Limited Population Approval Pathway for Antibacterial and Antifungal Drugs (LPAD).

On Sept. 28, 2018, Arikayce® became the first FDA-approved product under LPAD.

Sec. 3060: Clarifying medical software regulation

On Dec. 13, 2018, the FDA released a report, “Report on Non-Device Software Functions: Impact to Health and Best Practices.”

Sec. 3072: Hiring authority for scientific, technical, and professional personnel

In June 2018, the FDA provided its “FDA 21st Century Cures Workforce Planning Report to Congress,” outlining the agency’s needs and progress in recruiting and retaining scientific, technical, and professional staff.

Government Accountability Office

Sec. 4008: GAO study on patient access to health information

On May 5, 2018, the GAO issued its study on patient access to health information (GAO-18-386).

Centers for Medicare and Medicaid Services (CMS)

Sec. 4011: Medicare site-of-service price transparency

On July 31, 2018, the CMS announced that it would make available a website that provides comparison information between the outpatient prospective payment system and ambulatory surgical center payment and copayment rates.

Sec. 4012: Telehealth services in Medicare

In February 2018, the CMS published an updated Medicare Learning network booklet on telehealth services.

Looking Ahead to 2019

Below we list a few of the actions we are anticipating for 2019.

Sec. 2031: NIH Strategic Plan

We expect the NIH to release its strategic plan for advancing biomedical research and funding in 2019. According to a presentation by the NIH detailing its strategic planning process, the institute is currently seeking input to inform its plan.

Sec. 2041: Task force on research specific to pregnant women and lactating women

The NIH’s Task Force on Research Specific to Pregnant Women and Lactating Women automatically terminates on March 21, 2019 (i.e., two years after establishment), and the HHS Secretary may extend its term for one additional two-year period.

Sec. 3002: Patient-focused drug development guidance

We expect at least two more draft guidances on PFDD from FDA in 2019—one “describing processes and methodological approaches to development of holistic sets of impacts that are most important to patients” and the other “describing approaches to identifying and developing measures for an identified set of impacts (e.g., burden of disease and treatment), which may facilitate collection of meaningful patient input in clinical trials,” both informed by the public meeting held on Oct. 15-16, 2018.

We anticipate that the FDA will hold a number of public meetings in 2019, likely including one to inform draft guidance on “methodologies, standards, and technologies to collect and analyze clinical outcome assessments for purposes of regulatory decision making” and one to inform guidance on “how the Secretary, if appropriate, anticipates using relevant patient experience data and related information, including with respect to the structured risk-benefit assessment framework…to inform regulatory decision making.”  

Sec. 3021: Novel clinical trial designs

The Cures Act requires the FDA to issue draft guidance addressing the use of complex adaptive and other novel trial design in the development of new drugs or biologicals by Dec. 13, 2019. (Relatedly, in September 2018, the FDA issued draft guidance on “Adaptive Designs for Clinical Trials of Drugs and Biologics Guidance for Industry,” meeting a PDUFA requirement.)

Sec. 3051: Breakthrough devices

The Cures Act requires the FDA to issue a report to Congress on the breakthrough device review pathway and opportunities for improvement to better meet patient needs by Jan. 1, 2019. We have yet to see the report, but the FDA will likely share it publicly at some point this year.

Sec. 4004: Information blocking

We expect the Office of the National Coordinator for Health IT to issue its proposed rule on information blocking (as well as other topics, such as interoperability) in 2019 (the proposed rule is currently at the Office of Management and Budget).

21st Century Cures Tracker

For more information on upcoming statutory deadlines and completed actions, we invite you to visit our 21st Century Cures Tracker, which monitors the implementation of the 114 sections in Division A, including the key provisions relevant to biomedical research and innovation. The tracker seeks to highlight progress, measure impact, and identify areas where more resources or action may be needed.

21st Century Cures Impact Blog Series

In 2019 we are excited to enhance the 21st Century Cures Tracker by bringing you a series of resources to more specifically investigate the impact of selected Cures Act provisions on the biomedical and research ecosystems. Stay tuned for additional updates in this series.