Health care experts are schedule to gather in Washington, D.C., on Sept. 13 to discuss how we can harness the data we gather in routine health care delivery in the real world and apply that information to support drug approvals, inform safety monitoring, and accelerate new health care innovations. Although many decision-makers are already using real-world data, the Food and Drug Administration is poised to take a significant step forward in how it considers and uses this information.
Understanding how treatments work in the “real world” can help improve the new health care innovation and inform health care decision-making. For example, real-world data can be used to identify unmet needs with existing medications, understand the potential safety and effectiveness of treatments in other forms of cancer, or accelerate the conduct of clinical trials. Real-world evidence has been in use for a long time, but it has grown in importance through investment in health information technology and more sophisticated approaches to collect and analyze data.
Under the 21st Century Cures Act, the FDA must consider how real-world evidence could support the approval of a new indication of a drug or support or satisfy post-approval study requirements. RWE is meant to complement, not replace, knowledge from randomized clinical trials — studies most commonly used for FDA approval of a treatment — and accelerate and add to what we already know about how a treatment works.
In fact, the FDA is already using RWE, but not for new biopharmaceutical indications. Through the FDA’s Sentinel Initiative, the FDA monitors the safety and emerging safety concerns of medical products in use in the marketplace. The Sentinel Initiative database, with more than 170 million lives, is one of the largest uses of this type of information. In July, the FDA cited RWE as a critical factor in its decision to expand the initial approval for transcatheter aortic valve replacement to now include valve-to-valve procedures. And at the end of August, the FDA issued a final guidance on how it intends to utilize real-world data to support regulatory decision-making for medical devices, offering a glimpse at how the agency could use the data for biopharmaceutical-related decisions.
Yet as the FDA has acknowledged, there is a need for rigorous and reliable information. When done correctly, by using high-quality data and strong methodologies, RWE can provide meaningful information; when done poorly, misinterpretations and poor health outcomes for patients are possible. Recent systematic reviews that compared treatment effects results from RCT and RWE studies for the same condition found few differences among high-quality studies based on the study type (RCT vs. RWE) alone. The quality of some data and methods have improved such that RWE can reliably answer many research questions.
High-quality data and credible analytic methods are critical. Over the past decade, several professional societies and research consortium have developed best practices and standards for conducting analyses and using RWE. However, as the National Pharmaceutical Council has outlined in research, there is little agreement among researchers and other stakeholders on how to optimally do this. A consensus-based set of methods and standards for collecting RWE developed in consultation with multiple stakeholders could provide greater clarity. An iterative process would allow these standards to rapidly incorporate new methods innovation, yet still encourage other stakeholders to accept high-quality studies and disregard poor-quality studies.
The regulatory context, decisions, and needs of the FDA are unique — however, recommendations and evidence included in the FDA-approved label serve as a guiding light for many stakeholders. For RWE to be fully accepted and utilized, it will require all stakeholders, including the biopharmaceutical industry, to encourage a learning health system using real-world data. While payers are using data in a variety of applications, from value-based contracting to medication adherence, broader and more consistent use of RWE to update coverage policies based on effectiveness in the real-world is needed. Clinical practice guideline organizations and clinical pathways that serve as a foundation for translating knowledge into clinical practice could more routinely use high-quality RWE to expand the knowledge about treatment effects in broader patient populations and improve the certainty of their recommendations. Patients also are becoming more aware of how their data is being collected and used. Going forward, it will be important to ensure that patients understand what RWE is and determine when it can be trusted to guide patient and provider treatment decisions.
The September meeting, bringing together these diverse stakeholders, is an important next step toward expanding the quality and reliability of real-world evidence for health care decision-making. NPC is looking forward to the conversation.
Jennifer Graff is a PharmD and vice president for comparative effectiveness research at the National Pharmaceutical Council, which sponsors and participates in research on the appropriate use of pharmaceuticals and the clinical and economic value of pharmaceutical innovation.
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