June 30, 2016 – The discussion focused on a new report on real-world evidence issued by the BPC.
The DCRI’s Director of Health Services and Outcomes Research Adrian Hernandez, MD, MHS, was recently invited to participate in a Bipartisan Policy Center (BPC) roundtable focused on advancing the generation and use of real-world evidence.
The BPC, a nonprofit organization devoted to bipartisan solutions to questions of public policy, hosted the meeting at its headquarters in Washington, D.C. on June 23.
At the crux of the discussion was a new report issued by the BPC entitled Using Real World Evidence to Accelerate Safe and Effective Cures. The report expands on the recommendations in BPC’s July 2015 report, Advancing Medical Innovation for a Healthier America, and proposes four key recommendations for modernizing the drug development process through the use of real-world evidence.
Along with an expert panel that included Marc Boutin, CEO of the National Health Council, Andrew von Eschenbach, president of Samaritan Health Initiatives and former U.S. Food and Drug Administration (FDA) commissioner, David Madigan, executive vice president and dean, Faculty of Arts and Sciences at Columbia University, and Marcus D. Wilson, president of HealthCore, Hernandez shared his perspective on how the right policies can shape the future of real-world medicine.
“It is important to have the right policy pathways in place for improving drug development and bringing the best treatments to patients,” Hernandez said. “Real-world evidence complements traditional randomized clinical trials of select populations to help us understand how a new drug or treatment could be beneficial in other populations or for other indications. These insights make it easier for patients to make the right healthcare decisions and for clinicians to better understand the benefit/risk profile of treatments they prescribe.”
The BPC report calls for the following actions to modernize the drug development process through the use of real-world evidence:
- Improve regulatory clarity regarding use of real-world evidence via development of a formal guidance from the FDA regarding the use of real-world evidence to inform regulatory decision-making. The report suggests that the guidance include circumstances under which real-world data could be used as well as the types of real-world data, methods, and the levels of evidence that would be acceptable for use in regulatory review and decision-making.
- Improve methods and data quality for the generation and use of real-world evidence via the FDA’s establishment of a program to promote sharing and evaluation of these methods as they are used in regulatory decision-making. The report also asks that the U.S. Department of Health and Human Services support research to improve methods for the use of real-world evidence and that the FDA require researchers who receive federal funding or utilize real-world evidence make their methods transparent to assess their validity.
- Improve policies for information sharing to support clinical research and counter the current challenge of multiple institutional review board (IRB) approvals when a real-world study is conducted across multiple health systems. Specifically, the report recommends that Congress require the HHS secretary to issue regulations and guidance that facilitate the use of centralized IRBs within 36 months. It also suggests that Congress promote National Institute of Health policies that encourage investigators to voluntarily use single IRBs for grant submissions.
- Explore new adaptive pathways to modernize drug development and support a new era of personalized medicine, including the use of “close monitoring cohorts” to continually generate evidence. The report also calls on the FDA to develop a new program to design and test a new, flexible approach to drug approval that expands the capacity for drug development.
As the Coordinating Center principal investigator for PCORnet, the National Patient-Centered Clinical Research Network, Hernandez noted that PCORnet is one the most robust endeavors to promote the use of real-world data to date.
“With electronic health record and other clinical- and patient-reported data from more than 100,000,000 persons, PCORnet provides a model for the use of real-world evidence to accelerate the nation’s capacity to conduct patient-centered clinical research—including pragmatic clinical trials to generate answers more efficiently,” he said. “This roundtable was an opportunity to emphasize the pressing need for regulatory guidance and policies that will facilitate the use of PCORnet and other tools to bring real-world data to life.”