Spinal Muscular Atrophy: Drugs:Written question - HL10601

Asked on: 10 October 2018
Department of Health and Social Care
Spinal Muscular Atrophy: Drugs
To ask Her Majesty's Government what assessment they have made of making financial resources available to patients with spinal muscular atrophy (SMA) to obtain medication not approved by NICE.
Answered by: Lord O'Shaughnessy
Answered on: 17 October 2018

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.

Grouped Questions: HL10600 | HL10602

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