Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, how many and what proportion of people with spinal muscular atrophy will be eligible for access to NHS drugs for treatment following the 15 May 2019 announcement by NICE; what his policy is on people who have been excluded from that access; and if he will make a statement.

NHS England, NHS Improvement, the National Institute for Health and Care Excellence (NICE) and Biogen have worked together extensively to reach an arrangement that allows access to nusinersen for most people with spinal muscular atrophy types 1, 2 and 3 along with pre-symptomatic patients.

NICE has advised that because of the difficulties in being confident about the clinical evidence and the cost-effectiveness of nusinersen, it was necessary to agree access criteria for a period of further data collection to try and better understand how nusinersen affects people with spinal muscular atrophy. Therefore a managed access agreement has been reached, which should not only help answer these questions but also address the financial risk and challenges for implementation in the National Health Service.