To ask the Secretary of State for Health and Social Care, with reference to paragraph 4.12 the UK Rare Diseases Strategy, what assessment his Department has made of whether the National Institute for Health and Care Excellence’s Single Technology Appraisal Programme meets the ambition of suitability for assessing rare disease treatments.
Where a company is willing to set a fair price for a drug, the National Institute for Health and Care Excellence (NICE) technology appraisal process has been proven to be suitable for the assessment of drugs for rare diseases. NICE has been able to recommend a number of drugs for patients with rare diseases through its technology appraisal programme; recent examples include dinutuximab beta for neuroblastoma and daratumumab for relapsed and refractory multiple myeloma.
With the aim of improving the lives of all those affected by a rare disease, the United Kingdom Government published the UK Strategy for Rare Diseases in 2013, a high-level framework containing 51 commitments which sets out a seven-year strategic vision (2013-2020). The Government is committed to implementing the Strategy’s commitments and has, in January 2018, published Implementation Plans setting out its actions for England.