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    Has all of the relevant evidence been taken into account?
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    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?
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    Are the recommendations sound and a suitable basis for guidance to the NHS?
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The content on this page is not current guidance and is only for the purposes of the consultation process.

1 Recommendations

1.1 Risdiplam is not recommended, within its marketing authorisation, for treating 5q spinal muscular atrophy (SMA) in people 2 months and over, with a clinical diagnosis of SMA types 1, 2 or 3 or with one to four SMN2 copies.

1.2 This recommendation is not intended to affect treatment with risdiplam that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children and young people, this decision should be made jointly by the clinician and the child/young person and/or their parents or carers.

Why the committee made these recommendations

SMA is a rare genetic condition and there is an unmet need for effective treatments that could slow disease progression.

There is no evidence on risdiplam for babies with pre-symptomatic SMA. Clinical evidence shows that risdiplam improves motor function in SMA types 1 to 3. Also, there is some evidence suggesting that people with type 1 SMA who have risdiplam live for longer. But there is no direct evidence comparing risdiplam with best supportive care for type 1 SMA. And there is no long-term evidence, so the estimated long-term benefits are highly uncertain.

The committee considered a wide range of issues in its decision-making. In particular, it discussed the rarity and severity of SMA, risdiplam's innovative oral administration, uncertainties in the evidence, and whether risdiplam should be considered as an end-of-life treatment.

The cost-effectiveness estimates presented are much higher than what NICE usually considers an acceptable use of NHS resources. So, even taking these other factors into account, risdiplam cannot currently be recommended.