Question to the Department of Health and Social Care:

To ask Her Majesty's Government what guidance is provided to NHS Trusts about the use of medication not approved by NICE to treat spinal muscular atrophy (SMA).

The National Institute for Health and Care Excellence (NICE) is currently developing technology appraisal guidance for the National Health Service on the use of nusinersen (Spinraza) for the treatment of spinal muscular atrophy (SMA).

NHS England has published an Interim Policy Statement Urgent Clinical Commissioning Policy Statement: Nusinersen for genetically confirmed Spinal Muscular Atrophy (SMA) type 1 for eligible patients under the Expanded Access Programme (EAP) determining the circumstances in which patients will be supported to access nusinersen through the EAP scheme sponsored by Biogen, the manufacturer of nusinersen. A copy is attached. The policy statement enables patients with type 1 SMA to access nusinersen in advance of NICE’s guidance.

In the absence of final guidance from NICE, clinicians can apply to NHS England for funding in exceptional cases through the individual funding request process.