Tackling the Barriers to Payer Use of Real-World Evidence in Rare Disease

Drug developers and global regulators are rapidly expanding the use of real-world evidence (RWE) to design, test, and review rare disease treatments, but knowledge gaps on the part of payers regarding the validity and value of RWE are limiting patients’ access to such breakthroughs in ways that should be tackled, according to a new report.

Syneos Health looked at payer barriers to the use of RWE and opportunities to advance understanding and engagement by gaining firsthand feedback from more than 300 payers in the U.S. and Europe about the acceptability of RWE as these payers make decisions about rare disease therapies.

The “Real World Value: Advancing Payer Understanding of RWE in Rare Disease” report “reveals that payers are positively disposed to use RWE,” but multistakeholder feedback “points to the need for collaborative, well-structured exercises and standard-setting for payers to advance at the same velocity as other industry stakeholders,” notes Alistair Macdonald, CEO of Syneos Health. “Dialogue is key, and the time to facilitate these conversations is now.”

Among other findings, the report includes these observations from a survey of 64 respondents on Pharmacy and Therapeutic committees, evenly divided between the U.S. and Europe and with experience in rare disease:

  • 55% of U.S. payers and 37% of European payers said terminology for real-world data and RWE is not used widely at their institutions.
  • 60% of U.S. payers said RWE can provide robust data on how an agent has behaved in real-world conditions where population, diversity, comorbidities, and adherence variability come into play, while only 42% of European payers say that RWE mirrored real-world conditions and would eventually inform clinical practices.
  • 100% of payers in the U.S. and Europe expressed a version of the view that not all data are equal in clinical decision making. Furthermore, there is no single, accepted hierarchy of persuasive data on either side of the Atlantic.
  • In the U.S., 100% voiced concern surrounding the lack of standardized—or even interoperable—electronic health records, with more than half describing this barrier as the biggest limitation on data submission compared to European countries that have national health systems.
  • 77% of U.S. payers said primary, peer-reviewed literature is “always meaningful” in their coverage decision making vs. 48% in Europe, where greater trust is put in internal data.

“While payer engagement surrounding RWE in the rare disease space differs from industry and regulators, our research uncovers no trend or obstacles to advancing their understanding as long as regulatory bodies and industry groups are attentive to their concerns,” Macdonald says.

Edited by Gary W. Cramer