Closing NICE’s Orphan gap

How patients, industry think NICE should revamp its process for assessing Orphan drugs

As the list of Orphan drugs rejected by NICE grows, industry and patient groups are pressuring the agency to make broader use of tools that currently apply only to selected ultra-Orphan therapies. These tools allow coverage of new drugs during an initial period in which the sponsor must collect additional evidence of cost and clinical effectiveness.

This approach to coverage with evidence development, known as “managed access” in the U.K., is available only for cancer drugs paid for through the Cancer Drugs Fund (CDF), and for selected ultra-Orphan drugs that qualify for NICE’s Highly Specialized Technologies (HST) pathway.

NICE’s introduction of the HST pathway in 2014 was a tacit admission that ultra-Orphan drugs cannot pass the tests for cost-effectiveness used in NICE’s standard technology appraisal (STA) process. HST allows a higher cost per QALY than STA does, and also explicitly permits managed access schemes in cases where additional evidence is needed to confirm clinical and cost effectiveness.

Orphan drugs for larger populations, however, are still being directed to the STA process. They almost never succeed because the costs are too high, and the evidence of their clinical benefits too uncertain.

“The gap between that HST patient threshold and where STA begins to make sense on a per-patient basis is quite large.”

Nick Meade, Genetic Alliance UK

“The gap between that HST patient threshold and where STA begins to make sense on a per-patient basis is quite large,” Nick Meade, director of policy

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