Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, with reference to the findings of the 2017 Office for Health Economics report entitled Comparing Access to Orphan Medicinal Products in the United Kingdom and other European countries, what steps his Department is taking to help ensure that people with rare diseases can access the latest treatments.

The National Institute for Health and Care Excellence (NICE) issues guidance for the National Health Service on whether drugs and other treatments represent an effective use of NHS resources through its technology appraisal (TA) and highly specialised technologies (HST) programmes, including drugs for patients with rare diseases. 86% of NICE’s TA recommendations, and 100% of its HST recommendations, for orphan drugs have been positive since 2013.

Through the Early Access to Medicines Scheme, we are already making important drugs available to patients. So far around 1,500 patients have benefited from the scheme, which enables drugs to be used in clinical practice in parallel with later stages of the regulatory process.