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Muscular Dystrophy

Question for Department of Health and Social Care

UIN 137885, tabled on 25 April 2018

To ask the Secretary of State for Health and Social Care, what assessment he has made of the suitability of the Single Technology Appraisal process for new treatments for patients with Duchenne Muscular Dystrophy.

Answered on

3 May 2018

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based guidance for the National Health Service on whether drugs and other treatments represent an effective use of NHS resources.

NICE assesses most significant new drugs through its technology appraisal programme and has been able to recommend a number of drugs licenced for the treatment of rare diseases for routine use on the NHS. NICE also operates a separate highly specialised technology (HST) evaluation programme for the assessment of very high cost drugs for the treatment of very small numbers of patients. Decisions on the most appropriate programme for individual drugs and other treatments are made through an established topic selection process that includes a public consultation and thorough consideration against a set of published criteria.

NICE published HST guidance recommending ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in July 2016 and is currently developing guidance, through its technology appraisal and HST programmes, on other drugs for the treatment of Duchenne muscular dystrophy.