BioCentury
ARTICLE | Regulation

Closing NICE’s Orphan gap

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

September 14, 2018 10:58 PM UTC

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...