Real-World Evidence in the Real World: Beyond the FDA

Abstract

The 21 Century Cures Act ("Cures Act") relies on the concept of real-world evidence ("RWE") to improve the Food and Drug Administration ("FDA") approval process. This has amplified interest and furthered momentum in applying RWE more broadly, beyond FDA regulation. In this article, we discuss the understandable appeal of RWE's pragmatic application and its many potential benefits. But we also caution that claims about RWE's wide-ranging, ameliorative impact on the health care system are likely overstated. The real world of RWE is messy and uncertain. Successfully incorporating RWE into regular health care system decision-making, beyond the FDA, faces considerable obstacles and limitations. We review the reasons to be wary about RWE as a game-changer. These concerns including data reliability, insufficient incentives for stakeholders to generate and engage with high-quality RWE, and lack of comprehensive regulatory oversight. In addition, the push for RWE may impact the enforcement of the health care fraud and abuse laws, perhaps not in necessarily positive ways. Increased reliance on RWE may have significant implications for off-label fraud enforcement, further conflating the distinction between claims that are false for reimbursement rather than for scientific purposes.

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“…In December 2018, the US Food and Drug Administration (FDA) created a framework for evaluating the potential use of RWE to help support the approval of new indications for approved drugs or to help support or satisfy post-approval study requirements. [26,27] This method is especially suitable for clinical research in severe early childhood diseases.…”

Developing a Canadian Real-World Evidence Action Plan across the Drug Life Cycle

“…Finally, drug development and pharmaceutical policy is a global issue, and thus, the action plan should be developed with an eye toward the ongoing international initiatives in other major jurisdictions, such as those at the European Medicines Agency and the US FDA (Krause and Saver 2018;Plueschke et al 2018). The Canadian action plan should consider the current and ongoing initiatives.…”

“…Healthcare decision-makers such as drug regulators and payers face challenges in curbing rising drug costs and a rapidly growing number of rare indications (CADTH 2016). These challenges, associated with the advent of technological advances in identifying rare conditions and available treatment options, come at a time when there is a demand for quicker access to novel treatments (CADTH 2016(CADTH , 2018Krause and Saver 2018). To address these challenges, decision-makers see the use of real-world evidence (RWE) as an opportunity to make better and more informed decisions related to market access and funding (CADTH 2018).…”

“…The use of and demand for RWE by regulatory, reimbursement and healthcare decisionmakers have quickly expanded (CADTH 2018; Krause and Saver 2018). Currently, the use of RWE has largely been limited to the post-marketing safety evaluation of drugs and supplemental evidence for submissions.…”

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“…10 Real-world evidence is increasingly recognized as an important complement to randomized clinical trial (RCT)derived evidence. [11][12][13] Using data from insurance claims or electronic healthcare records, real-world evidence provides information about the treatment options that are adopted by patients and physicians outside of a clinical trial setting. Real-world evidence is particularly important in those patient populations that are often under-represented in RCTs, such as patients who are typically older, carry higher comorbidity burdens, or are more racially/ethnically diverse.…”