Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what progress has been made on concluding a managed access treatment agreement for the use of Spinraza for patients with spinal muscular atrophy types 2 and 3.

The National Institute for Health and Care Excellence (NICE) is currently developing guidance on the use of Spinraza (nusinersen) for the treatment of spinal muscular atrophy, including types 2 and 3.

NHS England and NICE have been in discussions with the manufacturer of Spinraza, Biogen, to explore whether there are commercial flexibilities that might enable NICE to recommend the drug as a clinically and cost effective use of NHS resources. Biogen has now submitted a revised submission for NICE’s independent Appraisal Committee to consider and a meeting has been arranged for 6 March 2019 to consider the recommendations. 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.