At the end of 2018, the U.S. Food and Drug Administration released a framework for real-world evidence, followed it up with expanded guidance on the use of real-world evidence to support the approval of new therapies, and restructured the Office of New Drugs to beef up staffing to accelerate the reviews involving real-world evidence.

In the rare disease space, several products have already won approval based in part on studies that incorporated real-world evidence. This includes BioMarin’s 2017 approval of Brineura to treat CLN2 disease, a form of the neurodegenerative condition Batten disease. The approval was based on a non-randomized, single-arm dose escalation study with patients in a natural history study used as a control. A number of other rare disease approvals have since incorporated real-world evidence.

But while regulators and drug developers have been moving with speed to incorporate the use of real-world evidence into rare disease drug development, a new report finds payers have been slow to embrace such data in their evaluations of the value of rare disease therapies.

The report, Real World Value: Advancing Payer Understanding of RWE in Rare Disease suggests that access to new rare disease treatments, especially in the United States, is hindered by a lack of familiarity among payers with real-world evidence and its growing use in clinical development.

“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,” said Alistair Macdonald, CEO of Syneos Health, a contract research and contract commercial organization that produced the report. “Multi-stakeholder 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.”

The report found a disconnect between regulators and drugmakers at one end, which are moving to incorporate real-world evidence, and payers, who are doing little to incorporate real-world evidence for rare disease reimbursement discussions. The report found such use of real-world evidence “intermittent at best.”

The report includes insights from a survey that included 64 respondents, evenly divided between the United States and Europe who are on payer Pharmacy and Therapeutic committees (Drug and Therapy committees in Europe), have experience in rare disease, are involved in decision making for this category, and also formulate medical policies.

It found that payers do not consider real-world evidence a tool for determining safety and efficacy of therapeutics. Instead, they regard it as something to consider in terms of label extensions and post-marketing surveillance.

While the report found that payers have confusion and concern over real-world evidence data sources, standards, and quality, it found payers were “positively disposed” to real-world evidence in decision support “so long as regulatory bodies and industry groups are attentive to their concerns.” “Dialogue,” the report said, “is key.”

It noted that ad hoc industry groups are working to address the challenges surrounding real-world evidence in healthcare decision making. Among the things regulators and industry will need to address are both transparency and reproducibility of real-world evidence, the report said.

It leaves one to wonder, though, if we are moving to a world in which barriers to approving new therapies for rare diseases are falling with the use of real-world evidence while reimbursement for these therapies will be stymied over a lack of what payers are willing to accept as evidence of value.

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