Rare Diseases: Drugs

(asked on 21st September 2020) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, how many times a medicine for a rare disease, not eligible for the HST process, has received a conditional approval for reimbursement based on a managed access agreement or similar.


Answered by
Jo Churchill Portrait
Jo Churchill
Minister of State (Department for Work and Pensions)
This question was answered on 22nd October 2020

The National Institute for Health and Care Excellence has made recommendations on two medicines for rare diseases through a managed access agreement that were not eligible for the evaluation through the highly specialised technologies process.

These are:

- Belimumab (Benlysta) for treating active autoantibody-positive systemic lupus erythematosus [TA397] published 22 June 2016; and

- Nusinersen (Spinraza) for treating spinal muscular atrophy [TA588] published on 24 July 2019.

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