Payers Slow to Adopt Real-World Evidence in Rare Diseases

While there is an increased interest in using real-world evidence (RWE) to design, test, and review rare disease treatments, payers may not be as receptive to using RWE when making reimbursement and formulary decisions, according to a new report from Syneos Health.

The report utilized conversations with payers, drug developers, regulatory experts, and patient organizations in the United States and Europe to examine issues around generating and using RWE.

“Our research reveals that payers are positively disposed to use RWE, but knowledge gaps around the validity and value of RWE must be closed to improve access for rare disease patients,” Alistair Macdonald, chief executive officer of Syneos Health, said in a statement. “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. Dialogue is key, and the time to facilitate these conversations is now.”

US respondents seemed to be more knowledgeable or open to RWE. While 55% of US payers were familiar with RWE terms, they said these terms were not widely used at their institutions. Only 37% of European payers said RWE terminology was not widely used. In addition, while 60% of US respondents said RWE can provide robust data in real-world conditions, only 42% of European respondents said RWE mirrored real-world conditions and could inform clinical practice.

All of the US payers noted that there is a lack of standardization and interoperability among electronic health records. More than half said this was the biggest limitation to using RWE. In comparison, only 34% of European respondents felt this lack of standardization was the biggest issue. Acceptance of RWE varies widely throughout Europe: in Italy and Spain, RWE can influence payer decisions at the national and regional levels, but payers in Germany question the validity of RWE from manufacturers.

The report also highlighted a disconnect between payers and regulators or manufacturers. Part of the issue is that rare diseases rank low for payers’ budgetary concerns. In addition, US payers mostly view RWE as something to be used in label extensions or postmarketing surveillance—RWE and real-world data were generally not viewed as tools that can be used to determine safety and efficacy for new products. However, the reality is regulators and increasingly encouraging the use of RWE.

For instance, the FDA has provided formal guidance to spell out guidelines and guardrails, as well as answer questions surrounding data sources, data generation, validity, utility, and more. Former FDA Commissioner Scott Gottlieb, MD, remains a proponent of RWE, and at The American Journal of Managed Care^®’s Patient-Centered Oncology Care^® meeting, he spoke about how the use of RWE will accelerate rapidly and allows regulators to “fill in the blanks.”

“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,” said Macdonald.