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.
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.