3:58 PM
Sep 14, 2018
 |  BioCentury  |  Regulation

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 for Genetic Alliance UK, told BioCentury. “There’s a problem there.”

Examples of Orphan drugs rejected under STA include cystic fibrosis drug Orkambi lumacaftor/ivacaftor from Vertex Pharmaceuticals Inc., Biogen Inc.’s Spinraza nusinersen to treat spinal muscular atrophy (SMA), and CAR T therapy Yescarta axicabtagene ciloleucel from the Kite Pharma Inc. unit of Gilead Sciences Inc.

Industry executives and patient groups told BioCentury a managed access mechanism could have provided the data needed to assess their long-term benefits, while a risk-sharing payment model could have made them more cost effective.

Their pleas have reached the ear of Parliament, where a group of MPs has asked Genetic Alliance UK to develop alternatives that could enable reimbursement of Orphan drugs while data are collected in the real world.

Missing the cut

Patient groups that spoke with BioCentury had multiple complaints about the way that NICE is evaluating Orphan drugs, including how it determines which pathway to use.

“If we started from scratch, we wouldn’t have the system that we have now,” Meade said.

NICE declined BioCentury’s interview request. The agency has not disclosed a population threshold for the HST pathway and does not publish its reason for reviewing a particular drug under one pathway instead of the other.

Meade said the threshold for HST is believed to be around 500-1,000 patients. But it’s hard to tell based on available examples. Drugs for diseases with as many as 2,500 patients in England and Wales...

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