Rare Diseases: Medical Treatments
(asked on 6th July 2018) - View Source
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what assessment the Government has made of the merits of the single technology appraisal process for evaluating gene therapies for rare diseases.
This question was answered on 16th July 2018
We have made no such assessment. The National Institute for Health and Care Excellence (NICE) has not evaluated any gene therapies through its technology appraisal programme. NICE published highly specialised technologies guidance recommending use of Strimvelis, a gene therapy, for the treatment of adenosine deaminase deficiency–severe combined immunodeficiency in February 2018.