Question to the Department of Health and Social Care:

To ask His Majesty's Government which medicinal products evaluated by the National Institute for Health and Care Excellence (NICE) in each of the past five years have (1) been considered (a) to treat a life threatening or severe limiting disease, (b) to restore patients to full or near full health, and (c) likely to sustain their benefits over a very long period of time, and (2) not been considered to meet the above criteria, in cases where a request was made to be considered as such by the applicant company.

The National Institute for Health and Care Excellence (NICE) concluded a comprehensive review of the methods and processes it uses for health technology assessment with the publication of its updated health technology evaluation manual on 31 January 2022. The review was carried out through extensive engagement with interested parties. As part of the review, NICE considered the circumstances in which it was appropriate to apply a non-reference case discount rate.

The updated manual states that NICE’s committee can consider using a non-reference case discount rate of 1.5% per year for both costs and health effects if the technology is for people who would otherwise die or have a very severely impaired life, if it is likely to restore them to full or near-full health, if the benefits are likely to be sustained over a very long period, and if the committee is confident that there is a highly plausible case for the maintenance of benefits over time and is satisfied that any irrecoverable costs associated with the technology have been appropriately captured or mitigated. The decision on whether a non-reference case discount rate is applied is taken by NICE’s health technology evaluation committees. There are no plans to seek any further clarification from NICE.

The non-reference case discount rate has been considered in several technology appraisals and highly specialised technologies evaluations, when considered appropriate by the committee. This includes both advanced therapeutic medicinal products (ATMPs), for example the evaluations of onasemnogene abeparvovec for spinal muscular atrophy and Strimvelis for adenosine deaminasedeficient severe combined immunodeficiency, and non-ATMPs, for example the evaluation of dinutuximab beta for neuroblastoma.

The full list of products that have or have not been considered to treat a life threatening or severely limiting disease, to restore patients to full or near full health, and that are likely to sustain their benefits over a very long period of time, could only be provided at disproportionate cost.