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What’s the Fuss About Evidence From Clinical Experience? Here’s Why It Matters

At a health care forum in Washington, DC, earlier this month, panelists raised concerns about the ability of the Food and Drug Administration (FDA) to effectively utilize “evidence from clinical experience” to support the approval of a new drug indication or post-approval study requirements. They cited FDA’s ongoing budgetary and staffing issues as part of the problem, but questioned whether this type of evidence would be sufficient for FDA to use in making drug-related decisions. Although FDA already has been using this type of evidence in different ways, the focus has been heightened because of specific provisions included in the House of Representatives’ version of the 21st Century Cures Act.*

The Cures legislation would require FDA to work with stakeholders to establish a program to evaluate the potential use of this evidence, as well as a framework to take into account current sources of data, gaps in data collection activities, and current standards and methodologies for the collection and analysis of data generated through clinical experience. The framework would then be used as the basis for a guidance document.

Taking a step back, it’s important to understand why this type of evidence matters, including what it is and how it can be used effectively in health care. Understanding how treatments work in the real world is an important and complex task to guide care in real-world, clinical practice environments. This could mean, for example, learning how medicines work in tandem with other medicines when patients have more than one condition, whether patients are able to remain adherent as they are juggling other daily challenges, or even determining if there are safety concerns that were not discovered during the clinical trial process. All of this information can provide a fuller picture of how treatments work and offer patients and their providers with a broader understanding about their health care options.

Much of this insight is being gleaned from databases. Among these databases is FDA’s Sentinel Initiative, which is one of the largest uses of real-world evidence in health care and proving vital for monitoring safety and emerging safety concerns. While FDA utilizes the Sentinel program to monitor medical products in use in the marketplace, the agency has acknowledged that “the science of using evidence from clinical experience to establish product effectiveness is still in its infancy. Real progress demands that we develop the methodologies needed to harness its promise.”

We’d agree that methods matter—and that’s why we’ve been working with other organizations to identify best practices and standards for collecting and analyzing real-world clinical experience evidence. Among those efforts, we developed the Comparative Effectiveness Research Collaborative with the Academy of Managed Care Pharmacy and the International Society for Pharmacoeconomics and Outcomes Research to assist decision-makers in their evaluation and use of evidence. The Good Research for Comparative Effectiveness Principles checklist also has helped to guide good practices for the design, conduct, analysis and reporting of observational studies for researchers and others.

In addition, we’ve also been taking a closer look at the best methods and standards for conducting analyses using real-world clinical experience evidence. Our forthcoming research found that there is general agreement on the basic elements required; however, there is variation in how various guidelines and standards recommend those elements should be conducted. Our other research has found that due in part to this variation, research isn’t always “fit for purpose,” or answer the health questions we need to address.

Regardless of whether Cures moves ahead in Congress, evidence from clinical experience is here to stay. And if we are going to make the most of this important information, then it will require all stakeholders to reach agreement on the basic elements needed to ensure the evidence is high-quality, credible and meets their needs.

* NPC does not lobby, nor do we take positions on federal or state legislation.