Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, pursuant to the Answer of 8 July 2019 to Question 369611 on Spinal Muscular Atrophy (SMA), what the evidential basis is that Spinraza does not work effectively on people with SMA that are excluded from access to that drug following the announcement of 15 May 2019 by NICE on that drug; and if he will make a statement.

The National Institute for Health and Care Excellence (NICE) is currently developing recommendations for the National Health Service on the use of Spinraza for the treatment of spinal muscular atrophy (SMA) through its technology appraisal programme. NICE recommends technologies for use in the NHS when the evidence demonstrates that they are clinically effective and represent a cost-effective use of NHS resources.

There are several subtypes of SMA and, in the case of Spinraza, NICE has advised that it was not presented with evidence in SMA type 0 and SMA type 4. Evidence presented for the SMA type 3 population, who had lost the ability to walk, was limited and immature and resulted in its independent Appraisal Committee concluding that the size and nature of the benefits of Spinraza for this population were uncertain. Therefore, they were not included in the managed access arrangement (MAA) agreed by NHS England and Biogen.

Following publication of the initial MAA, the company presented further clinical evidence and the MAA was extended to include paediatric patients, who have recently (in the previous 12 months) lost the ability to walk independently.

Uniquely for this type of arrangement, during the five-year course of the MAA, should evidence become available on the potential benefits of Spinraza for type 3 SMA patients that are currently not included in the MAA, NICE will review that evidence to see whether it would support a change in the MAA inclusion criteria.