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Orphan Drugs

Question for Department of Health and Social Care

UIN 32035, tabled on 19 March 2020

To ask the Secretary of State for Health and Social Care, what assessment he has made of the potential merits of establishing a new orphan medicines pathway at NICE to enable orphan medicines which do not qualify for the highly specialised technology evaluation programme to be appraised.

Answered on

27 April 2020

There are no current plans to establish a further National Institute for Health and Care Excellence (NICE) assessment programme for orphan medicines. Where companies are willing to price their drugs fairly in a way that reflects the benefits they bring to patients, NICE has been able to recommend a number of orphan medicines for routine NHS funding through its technology appraisal programme. NICE is reviewing its methods for the development of technology appraisal and highly specialised technology recommendations, including the methods for the assessment of medicines licensed for rare diseases. The creation of an Innovative Medicines Fund will also build on the success of the Cancer Drugs Fund and support access to the most advanced medicines for patients with rare diseases.