Assessing the Impact of Health Economics and Outcomes Research Evidence on Reimbursement Decisions in the United States

Abstract

Objectives: Health economics and outcomes research (HEOR) is a growing field that has gained substantial momentum in recent decades. While studies establishing a product's safety and efficacy have a well-understood role in drug development and commercialization processes, the impact of HEOR in the U.S. is less clear. We sought to understand the impact of HEOR evidence on reimbursement success in the U.S. Methods: We conducted a literature review to understand the evolution of HEOR and existing knowledge about its impact on reimbursement decisions in the U.S. We conduced focus group sessions and planning surveys with pharmaceutical industry HEOR executives to understand the scope of HEOR evidence that is produced and to assess how investments are selected. We developed and administered a survey with this audience to more formally assess how HEOR investment decisions vary based on product and market scenarios. Based on our findings, we crafted detailed hypotheses as to when specific types of HEOR evidence are important to U.S. payers and tested these hypotheses in a survey of HEOR decision-makers in pharmaceutical companies and formulary decision-makers for payer organizations. Results: Previous studies suggest a growing importance of HEOR evidence in U.S. reimbursement decisions; however, much of the existing literature fails to account for the growing scope of HEOR. We defined HEOR to include eight types of evidence that are produced to support new products. Our survey of HEOR executives from pharmaceutical companies suggests significant agreement as to when HEOR is likely to have a positive impact on U.S. reimbursement decisions. Our survey of payers confirmed the growing interest in HEOR evidence. In some instances, the scenarios in which HEOR evidence is perceived to be important varies between pharmaceutical and payer decision-makers. Conclusions: Pharmaceutical companies are investing in a range of HEOR to support payers' reimbursement decisions. This evidence has a substantial role in formulary decision-making processes and its use is expected to grow. There is some evidence of misalignment as to when specific types of evidence are important; understanding when payers value HEOR evidence will allow pharmaceutical companies to produce more relevant evidence in the future.