Payer Perspectives on Future Acceptability of Comparative Effectiveness and Relative Effectiveness Research

Abstract

Objectives: Our objective was to gather perspectives from payers on how comparative effectiveness research (CER) in the United States and relative effectiveness (RE) research in Europe will impact evidentiary standards for access decisions of new drugs by 2020.Methods: We conducted semi-structured interviews with fourteen senior officials representing public and private payers, health technology assessment groups, and pricing and reimbursement bodies in the United States and Europe. An online survey assessed current use of CER/RE evidence and potential trends that might influence its use for decision making by 2020. A semi-structured interview elicited payers' definitions of CER/RE and was structured around four hypothetical cases resembling drugs expected to be more common or poised to create policy challenges by 2020. Topics included acceptance of study designs and analytic methods associated with CER/RE. A systematic content review was done to extract relevant information.Results: According to key informants, randomization will remain an essential component for assessing comparative or relative effectiveness. They anticipate greater use of policy levers such as conditional reimbursement or prior authorization to manage diffusion of new drugs. Case studies provided important insights into situations when certain types of CER evidence may be acceptable (e.g., observational data when differences between drugs are largely convenience).Conclusions: Industry perceptions that CER/RE will change payers' evidentiary requirements in the future are consistent with our findings. Growing investment in payers' own data and increased reliance on policy tools to control diffusion of new drugs may also influence the type of evidence industry will be required to produce by 2020.

Real-World Evidence: Useful in the Real World of US Payer Decision Making? How? When? And What Studies?

“…Nearly all participants indicated that RWE was useful for monitoring safety, conducting utilization management, and examining costs, but was less likely to be considered in P&T decision making, principally because of timeliness. This finding is supported by other studies that report payers relying heavily on RCTs for evaluation of comparative effectiveness [3,4]. In this study, however, similar to other studies, use of specific RWE studies was not based on common areas of agreement such as specific designs (e.g., prospective vs. retrospective studies), data sources (e.g., claims and EHRs), or tier of the journal in which the results are published, but rather whether the study answered relevant questions, transparently described the methods and results, and controlled for potential biases (e.g., design, funding, and authorship).…”

“…A lack of understanding may lead decision makers to mistrust and place a lower importance on information from such studies, limiting their use in the decision-making process. Therefore, decision makers may instead over-rely on familiar sources of evidence, such as randomized controlled trials (RCTs), or use expert opinion [3,4].…”

Real-World Evidence: Bridging Gaps in Evidence to Guide Payer Decisions

“…Using a broad literature review (see electronic supplementary material [ESM]), we identified six articles summarizing payer surveys ( Supplementary Table, see ESM). RCTs were the preferred source of data for coverage decisions made by payers, especially those related to initial market access and pharmacy and therapeutic committee decisions [6][7][8][9]. Payers also used systematic reviews and meta-analyses to assess available evidence and replicability of findings across sources [6].…”

“…Payers also used systematic reviews and meta-analyses to assess available evidence and replicability of findings across sources [6]. Although payers continued to regard RCTs as the gold standard, they valued RWE to fill evidence gaps not addressed by RCTs, such as longterm effectiveness and safety [6,7,9]; head-to-head drug comparisons [6][7][8][9][10]; cost analyses for tiering formulary placement [6,7,9,11]; medication use patterns, including medication adherence [7,9]; identification of relevant responder and non-responder patient subpopulations [8,9]; and patient-reported outcome (PRO) data [10]. Notably, PRO data were considered especially useful in oncology compared with other therapeutic areas, given the high symptom burden and increased need for palliative care [10].…”

The Iterative Development of Medicines Through the European Medicine Agency's Adaptive Pathway Approach

“…The lack of effectiveness in late-stage development is the most frequent reason of clinical trial failures, and safety issues are more frequently observed after the marketing authorization, only when a larger number of patients have been treated. From this point of view, the challenge to the AP approach is to measure efficacy or effectiveness in a real world evidence setting with observational-like study designs, with the opportunity to assess the real acceptable level of uncertainty, also comparing it with the real clinical needs perceived by the patients (29). All these changes allow an “active monitoring” instead of a “passive prediction” and can facilitate a process toward a targeted prescription.…”