Payer perceptions of the use of real-world evidence in oncology-based decision making

Diana Brixner, Joseph Biskupiak, Gary Oderda, Douglas Burgoyne, Daniel C. Malone, Bhakti Arondekar, Alexander Niyazov

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


BACKGROUND: Randomized controlled trials (RCTs), the gold standard of safety and efficacy 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 evidence (RWE) studies may help address these knowledge gaps and help inform oncology outcomes-based contracting. The survey was formulary decision making. distributed to 221 US payers through the Academy of Managed Care Pharmacy (AMCP) OBJECTIVE: To assess US payer perceptions Market Insights program in February 2020. regarding the use and relevance of RWE Ten additional respondents were invited to in informing oncology formulary decision-discuss the survey results. The survey results making. were presented primarily as frequencies METHODS: A national survey containing of responses and were evaluated by the single-answer, multiple-answer, and free-respondent’s plan size, type, and geography response questions evaluated 4 key areas: (regional vs national). Differences in respons-(1) the value of RWE, (2) barriers to RWE, es for categorical data were compared using (3) sources of RWE, and (4) use of RWE in a Pearson Chi-Square or a Fisher’s Exact test. Two-tailed values are reported and a level of ≤0.05 was used to indicate statistical significance. RESULTS: The national survey had a 45.9% response rate, with 106 payers responding. Most were from managed care organizations (MCOs; 47.5%) and pharmacy benefit managers (PBMs; 37.4%), with 54.5% from large plans (≥1 million lives) and 45.5% from small plans (<1 million lives). Respondents were largely pharmacists (89.9%), with 55.6% overall indicating their job was a pharmacy administrator. Most (84.9%) used RWE to inform formulary decisions in oncology to support comparative effectiveness in the absence of head-to-head clinical trials (4.1 on a scale of 1=Not At All Useful to 5=Extremely Useful) and validation of National Comprehensive Cancer Network (NCCN) recommendations (4.0). Almost half (41.5%) used RWE results to inform off-label usage decisions. Payers valued RWE pre-launch to inform formulary and contracting decisions and desired real-world comparative effectiveness data post-launch to validate coverage decisions. However, the majority of payers (54.7%) did not conduct their own real-world studies. Commonly considered RWE sources included claims data (79.2%), medical records (68.9%), prospective cohort studies (60.4%), patient registries (36.8%), and patient outcome surveys (33.0%). Barriers to conducting internal RWE studies included the lack of resources and personnel, analytic capabilities, appropriate in-house data, and perceived value in conducting analyses. Payers expressed interest in using outcomes-based contracting in oncology; few have direct experience, and operationalizing through value measurement is challenging. CONCLUSIONS: RWE providing comparative treatment data, validation of NCCN treatment recommendations, and information on off-label usage are appreciated pre launch with post launch validation.

Original languageEnglish (US)
Pages (from-to)1096-1105
Number of pages10
JournalJournal of Managed Care and Specialty Pharmacy
Issue number8
StatePublished - Aug 2021

ASJC Scopus subject areas

  • Pharmacy
  • Pharmaceutical Science
  • Health Policy


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