Modernizing Regulatory Evidence with Trials and Real-World Studies
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EDUCATIONAL REVIEW
Modernizing Regulatory Evidence with Trials and Real‑World Studies Nancy A. Dreyer, PhD, MPH, FISPE1 · Marni Hall, PhD, MPH2 · Jennifer B. Christian, PharmD, MPH, PhD, FISPE3 Received: 4 February 2020 / Accepted: 9 February 2020 © The Author(s) 2020
We are taught that randomized controlled trials (RCTs) are the gold standard for evaluating whether a treatment can achieve its intended benefit because they are designed to isolate treatment effects while essentially balancing all other factors, known and unknown. While an elegant tool, the results from RCTs are not always generalizable to less idealized settings and more diverse patients. Real-world evidence (RWE), conducted alongside or as an extension of clinical trials, can play an important role in completing the picture of how well a therapy works, for which patients, and under what conditions. Such evidence has long been used for treatment and reimbursement decisions; the question at hand is whether, when and how RWE should contribute to regulatory decision-making and be considered credible enough to be counted as ‘substantial evidence of effectiveness.’ Among numerous efforts to evaluate RWE suitability for regulatory use, a recent Friends of Cancer Research (Friends) study provides valuable lessons for RCT and RWE zealots alike. Here, we review the complementary nature of RCTs and RWE, discuss the key learnings from the Friends project, and reinforce the call for continued examination of methods and data sources to guide reliable regulatory use of RWE for evaluating therapies.
RCTs and RWE—Fundamental Differences and Complementary Potential RCTs provide scientifically tidy comparisons, but they fall short in their utility for generalizability to more diverse patients, and complex conditions and treatments. By design, * Nancy A. Dreyer [email protected] 1
IQVIA Real World Solutions, 201 Broadway, 5th Floor, Cambridge, MA 02139, USA
2
IQVIA Real-World Solutions, Rockville, MD, USA
3
IQVIA Real World Solutions, Research Triangle Park, NC, USA
patients in RCTs are highly selected and cared for, with active encouragement to adhere to assigned treatments, close monitoring for disease progression, and optimal testing. The endpoints used in RCTs may not be directly relevant to patients and clinicians, especially surrogate endpoints used as substitutes for clinical outcomes that take a long time to develop [1]. For example, 36 of 54 new oncology treatments approved from 2008–2012 used surrogate endpoints for approval, but roughly four years after approval, only 14% showed improvement in overall survival (OS). Half (50%) of those newly approved drugs revealed no survival benefit, with no data available for the remainder [2]. In contrast, real-world data (RWD), an umbrella term for health data that are not collected in the context of highly controlled RCTs [3], captures information about benefits and risks for diverse patients and care settings under more typical conditions. RWE studies designed to supplement RCT results offer
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