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PP12-90: Payer Survey Reveals Confusion Around the Definition and Utility of Real-World Evidence in Rare Disease Coverage Decisions

Poster Presenter

David Thompson

Founder and Principal Consultant
Rubidoux Research LLC
United States

Objectives

Amidst growing use of real-world evidence (RWE) in clinical research and regulatory review of treatments for rare diseases, we surveyed payers in the U.S. and Europe to learn how they define RWE and how they value it, relative to other evidence, when making coverage and reimbursement decisions.

Method

We conducted in-depth interviews with payer organizations and a detailed online survey, receiving 64 responses evenly divided between the U.S. and five key European markets. 100% of respondents had experience in rare diseases and had served on pharmacy and therapeutics committees or the equivalent.

Results

The results of our survey, supported by in-depth interviews in a qualitative research phase, suggested that U.S. and European payers are only embracing RWE within limited parameters when it comes to rare-disease coverage decisions. The survey responses reflected significant confusion around terms such as “real-world evidence” and “real-world data.” Furthermore, many payers indicated they are not receptive to RWE-based representations or health economic forecasts in sponsors’ dossiers. In the U.S. only 55% of payers said they were familiar with real-world evidence. In Europe, 37% said the terminology is not widely used in their institutions. To the extent that U.S. payers were familiar with RWE, they were concerned about the lack of standardization when it comes to data sources such as electronic health records. (In the survey, 100% of U.S. payers identified this as a concern, as did more than one-third of European payers). In both geographic regions, payers recognized that regulators value RWE when it comes to post-marketing surveillance. However, few were aware of recent regulatory guidance encouraging the use of RWE in new drug applications—especially for rare-disease treatments. Despite lack of clarity around definitions and apprehensions about how RWE is generated, 60% of U.S. payers in the survey acknowledged RWE can provide robust data on how an agent has behaved in real-world conditions affected by population diversity, comorbidities and adherence. And 42% of European respondents said that RWE mirrored real-world conditions and/or would eventually inform clinical practice. The survey results confirmed our hypothesis that industry-wide educational initiatives focused on RWE could help narrow the perception gap between regulators and manufacturers, on one side, and payers on the other. This will be important as more new treatments arrive on the market with RWE as a component in the evidentiary dossier.

Conclusion

The payer survey and associated research described in this poster support the hypothesis that regulatory bodies and pharmaceutical innovators are moving more quickly than payer organizations in the exploration and utilization of real-world evidence. Potentially, this gap may have adverse consequences when it comes to market access for new treatments—especially in rare diseases where there is high unmet need. In the U.S., for example, the Food and Drug Administration has approved treatments for a host of rare conditions such as Batten’s disease, spinal muscular atrophy, and Merkel cell carcinoma based on single-arm studies in which real-world evidence stands in for the traditional control arm of a seminal study. Yet, despite rapid progress in research and regulatory uses of RWE, many payers responding to our survey lacked a clear or consistent understanding of the new evidentiary models and use cases. In some ways, this isn’t surprising. Payers tend to focus on conditions such as diabetes, cardiovascular disease and common forms of cancer that are a much larger piece of pharmacy and medical budgets, compared with rare diseases. To the extent that they think of RWE at all, it’s in the context of post-marketing surveillance. While the lag in payers’ understanding of RWE has not emerged as a significant market access barrier at the present time, the rapid pace of technological innovation threatens to widen the gap. This could lead to coverage and reimbursement hurdles in the future, as innovations such as “virtual” clinical trials and N-of-1 studies become more common. Keeping up with a fast changing healthcare environment is a challenge for all healthcare stakeholders, not just payers. Through well-structured education exercises, standards setting, and fact-finding missions on the frontiers of evidence sciences, stakeholders can collaborate to close the knowledge gaps on behalf of patients with rare and non-rare conditions.