Question to the Department of Health and Social Care:

To ask Her Majesty’s Government what estimate they have made of the number of patients who will be affected by the proposed £100,000 quality-assured life year threshold for evaluating highly specialised technologies.

We are committed to ensuring that patients with rare and ultra-rare diseases in England are able to access effective treatments.

The National Institute for Health and Care Excellence (NICE) has advised that the proposed arrangements are intended to provide clarity about the level of cost effectiveness below which funding will be made available, automatically and without delay, for highly specialised technologies that receive positive recommendations from NICE.

Subject to the outcome of the current consultation exercise, those products that have a value proposition of more than £100,000 per quality-adjusted life year will be provided with a further opportunity to be considered for use in the National Health Service, through NHS England’s annual process for deciding which new treatments and services will be routinely commissioned.

Furthermore, the proposed changes are intended to clarify NICE’s existing responsibility to determine the period within which funding for recommended products needs to be made available by commissioners of services. This clarity should allow companies and commissioners to work on commercial arrangements, in advance of, and in parallel with, the development of NICE technology appraisal and highly specialised technology guidance. In circumstances where this may not be possible, specific requests from commissioners to vary the timescale for the funding requirement will be considered by NICE.

As these proposals are still subject to possible change, no assessment has been made of the number of patients who may be affected or of any effect on uptake and equity of access to medicines for very rare diseases.