Question to the Department of Health and Social Care:

To ask Her Majesty’s Government how they propose to make available for prescription under the National Health Service orphan and ultra-orphan drugs for the treatment of rare diseases now emerging as a result of recent research discoveries.

It is for clinicians to make prescribing decisions based on their patients’ individual clinical needs.

The National Institute for Health and Care Excellence (NICE) has recommended a number of drugs, designated by the European Union as orphan drugs, in technology appraisal guidance. NICE is also responsible for the evaluation of selected high cost, low volume drugs under its Highly Specialised Technologies Programme.

National Health Service commissioners are legally obliged to provide funding for drugs that have been recommended in NICE technology appraisals or highly specialised technologies evaluations.

We are commissioning an external review of the pathways for the development, assessment, and adoption of innovative medicines and medical technology. This review will consider how to speed up access for NHS patients to cost-effective new diagnostics, medicines and devices.