Real-world evidence and health technology assessment decision-making: What agencies—and their biopharma stakeholders—need to know

Health technology assessment (HTA) agencies have long relied on evidence from randomized controlled trials (RCTs) in their decision-making on drug efficacy. As interest in real-world evidence (RWE) grows, there is increased opportunity for HTAs to learn from insights on how drugs perform in real clinical practice. In this white paper, created in advance of ISPOR Europe 2019, we explain how RWE can help HTAs bridge the “efficacy-effectiveness gap,” and what that means for biopharma.

"Real-world evidence is not a substitute for a well-conducted controlled trial, but there are many situations where you cannot do a well-controlled trial," said Jens Grueger, Ph.D., Affiliate Professor at the University of Washington, former Head of Global Access at Roche, and President-elect of the Professional Society for Health Economics and Outcomes Research (ISPOR)*. "In those situations, you really want RWE to complement your clinical data, for example, through external control arms."

Read more from Dr. Grueger and Ashley Jaska, M.P.H., Vice President of Analytic Solutions at Aetion, on the case for RWE in HTA decision-making and how biopharma can prepare for a coming shift in paradigm, including use cases, hesitations, and steps global HTA agencies like NICE (UK), ICER (US), and CADTH (Canada) are taking to get ahead of the curve on RWE adoption.

*Dr. Grueger's comments are his personal opinions and do not reflect the position of ISPOR.

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