RESEARCH 1096 JMCP.org | August 2021 | Vol. 27, No. 8 Payer perceptions of the use of real-world evidence in oncology-based decision making Diana Brixner, PhD, RPh; Joseph Biskupiak, PhD, MBA; Gary Oderda, PharmD, MPH; Douglas Burgoyne, PharmD, FAMCP; Daniel C Malone, RPh, PhD, FAMCP; Bhakti Arondekar, PhD, MBA; and Alexander Niyazov, PharmD, RPh, MPH ABSTRACT BACKGROUND: Randomized controlled trials (RCTs), the gold standard of safety and effica- cy evidence, are conducted in select patients that may not mirror real-world populations. As a result, healthcare decision makers may have limited information when making formulary decisions, especially in oncology, given accelerated regulatory approvals and niche patient populations. Real-world evi- dence (RWE) studies may help address these knowledge gaps and help inform oncology formulary decision making. OBJECTIVE: To assess US payer perceptions regarding the use and relevance of RWE in informing oncology formulary decision- making. METHODS: A national survey containing single-answer, multiple-answer, and free- response questions evaluated 4 key areas: (1) the value of RWE, (2) barriers to RWE, (3) sources of RWE, and (4) use of RWE in outcomes-based contracting. The survey was distributed to 221 US payers through the Academy of Managed Care Pharmacy (AMCP) Market Insights program in February 2020. Ten additional respondents were invited to discuss the survey results. The survey results were presented primarily as frequencies of responses and were evaluated by the respondent’s plan size, type, and geography (regional vs national). Differences in respons- es for categorical data were compared using a Pearson Chi-Square or a Fisher’s Exact test. What is already known about this subject • Payers use real-world data in decision making. • How real-world evidence (RWE) is used depends on the available data and therapeutic area. What this study adds • Payers acknowledge RWE on innovative oncology products pre launch is limited, highlighting a need for more early communication with manufacturers under preapproval information exchange (PIE) rules. • The types of RWE valued by payers both before and after launch included total cost of care, burden of illness, treatment patterns, subpopulations, adverse event profiles, off-label usage, and economic data for possible use in contracting. • Payers appeared to be more engaged in conducting their own evaluations of RWE for oncology products than previously. • The primary source of RWE data continues to be administrative claims, but many organizations are turning to electronic health records. • Payers’ interest in outcome-based contracting was high, despite the challenges to conducting these arrangements. Author affiliations Diana Brixner, PhD, RPh; Joseph Biskupiak, PhD, MBA; Gary Oderda, PharmD, MPH; Douglas Burgoyne, PharmD, FAMCP; and Daniel C Malone, RPh, PhD, FAMCP, University of Utah, College of Pharmacy, Salt Lake City. Bhakti Arondekar, PhD, MBA, and Alexander Niyazov, PharmD, RPh, MPH, Pfizer, Inc. AUTHOR CORRESPONDENCE: Diana Brixner, 801.581.3182; Diana.Brixner@utah.edu J Manag Care Spec Pharm. 2021;27(8):1096-105 Copyright © 2021, Academy of Managed Care Pharmacy. All rights reserved.