March 31, 2017: FDA and Pragmatic Trials

March 31, 2017: FDA and Pragmatic Trials

Slides: Slides


FDA and Pragmatic Trials


Robert M. Califf, MD, Professor of Medicine, Duke University


FDA; Pragmatic clinical trials; Real-world evidence; People-centered research; NIH Collaboratory; Digital health data; Electronic health records; Learning healthcare system; Safety surveillance

Key Points

  • The national ecosystem of medical product surveillance is shifting from passive to active and is becoming embedded in healthcare systems to leverage real-world evidence. The ecosystem comprises academic health and science systems, healthcare providers, medical products companies, regulators, and patients and consumers. The most positive outcomes will come from an ecosystem that is focused on defining benefits and risks.
  • Calls to action:
    - Organize operational systems that bring together research networks embedded in practice.
    - Establish a robust framework for privacy, confidentiality, and security.
    - Adopt a common approach to configure, store, and reuse digital health data for care, research, surveillance, and public health.
    - Develop and test new methods to reliably answer research questions.
    - Eliminate unnecessary complexity and ensure truly needed safeguards.

Discussion Themes

Real-world evidence has the potential to add findings that reflect actual use in clinical practice. Substantial evidence is associated with tangible outcomes for patients—how they feel and function.
Alliances of activated patients and clinicians can help write guidance for the FDA that focuses on important methods and outcomes.
The clinical ecosystem is moving toward more patient centricity, adaptive designs, use of real-world evidence, and use of central IRBs.
Can we change the academic incentives for investigators so they are rewarded for the "janitorial work" of contributing to the datasets for use in pragmatic trials?

For More Information

Read about the potential of real-world evidence in Real-World Evidence—What Is It and What Can It Tell Us? (Sherman et al., 2016 New England Journal of Medicine).
Read more about efforts to sustain and expand a national network for clinical research at the nonprofit People-Centered Research Foundation (PCRF) website.

#RealWorldEvidence, #pctGR
@Califf001, @PCTGrandRounds, @Collaboratory1, @PCORnetwork