Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the potential merits of establishing a fund for ultra-orphan medicines.

The National Institute for Health and Care Excellence (NICE) has been able to recommend medicines for very rare diseases for NHS funding through its established processes. These are now available to NHS patients in England, including gene therapies for metachromatic leukodystrophy (Libmeldy), spinal muscular atrophy (Zolgensma) and inherited retinal dystrophies (Luxturna).

In June 2022, the Government launched the Innovative Medicines Fund that will support NHS patients in England, including those with very rare diseases, to get early access to the most innovative and effective new treatments, where further data is needed to support a NICE recommendation on routine funding. The IMF is a managed access fund that provides a route for earlier patient access to the most promising new medicines while further evidence is collected to inform a long-term commissioning recommendation.

NICE’s independent committee may consider managed access if it cannot make a recommendation for routine commissioning but believes that further evidence collection during a period of managed access will sufficiently support the case for such a recommendation in the future.