January 6, 2017: 21st Century Cures Overview and Implications

January 6, 2017: 21st Century Cures Overview and Implications

Slides: Slides


21st Century Cures Overview and Implications


Gregory Daniel, PhD, MPH, Duke-Margolis Center for Health Policy and the Fuqua School of Business


Clinical research; Drug development; Real-world evidence; 21st Century Cures Act

Key Points

  • The 21st Century Cures Act was signed into law on December 13, 2016. It is aimed at improving and accelerating the discovery, development, and delivery of new biomedical products.
  • The legislation includes four key sections with important implications for clinical research and drug development that include:
    - Data sharing: quality and comprehensiveness; electronic health records interoperability
    - Evidence development: expanded sources of reliable evidence that better reflects real-world clinical care and outcomes
    - Human research subject protection: harmonized regulations; privacy safeguards
    - Patient-centered medicine: rigorous approaches to collecting patient experience data; formalized patient engagement processes to inform benefit/risk decisions
  • 21st Century Cures will advance the Precision Medicine Initiative, support young emerging scientists, and facilitate collaborative research. It will promote patient-focused drug development, patient access to therapies, and antimicrobial innovation and stewardship. The legislation also intends to improve scientific expertise and outreach at FDA.
  • 21st Century Cures reinforces the value of a learning healthcare system, whereby real-world patient experiences inform healthcare decision-making.

Discussion Themes

What are the similarities and differences between real-world surveillance for safety (as in Sentinel) and real-world surveillance for effectiveness and value?

With 21st Century Cures, it will be important to think in a more modern way about how data are measured in the clinical setting. We will need to figure out the best methods for making data credible for optimal use. Guidance on how real-world evidence can support comparative effectiveness research will be helpful.

Uncertainties remain about the impact of leadership changes, funding allocations, and upcoming legislative activities.

For More Information

Read more about the 21st Century Cures Act at https://energycommerce.house.gov/cures.

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