Question to the Department of Health and Social Care:

To ask the Secretary of State for Health, what steps (a) his Department and (b) the National Institute for Health and Care Excellence is taking to ensure that patients have access to drugs and treatments for ultra-rare diseases.

The UK Strategy for Rare Diseases, published in November 2013, aims to ensure that patients with rare diseases have access to high quality care, including diagnostics, treatments, support and effective drug treatments.

NHS England is responsible for directly commissioning specialised services accessed by comparatively small numbers of patients and has developed service specifications on a national basis, to ensure that patients can access the same high quality services and treatments regardless of where they live in England.

From 1 April 2013 the National Institute for Health and Care Excellence (NICE) took responsibility for the evaluation of selected high cost, low volume drugs under its Highly Specialised Technologies Programme. NHS commissioners are legally obliged to provide funding for drugs that have been recommended in NICE technology appraisals or highly specialised technologies evaluations.