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    a. Has all of the relevant evidence been taken into account?

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    b. Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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    c. Are the recommendations sound and a suitable basis for guidance to the NHS?

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1 Recommendations

1.1

Setmelanotide can be used as an option to treat obesity and hyperphagia in genetically confirmed Bardet-Biedl syndrome (BBS) in people aged 6 to 17 years. These people can carry on having setmelanotide as adults until they need to stop. Setmelanotide can only be used if the company provides it according to the commercial arrangement (see section 2).

1.2

This recommendation is not intended to affect treatment with setmelanotide 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 healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers.

What this means in practice

Setmelanotide must continue to be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option.

There is enough evidence to show that setmelanotide provides benefits and value for money, so it can be used routinely across the NHS in this population.

Why the committee made these recommendations

This evaluation reviews the evidence for setmelanotide for treating obesity and hyperphagia in BBS (see NICE highly specialised technology guidance 31, from now HST31). The discussion for this review is mainly about setmelanotide for adults who have not started the drug before age 18 years, for whom setmelanotide was not recommended in HST31. The recommendations for people starting treatment aged 6 to 17 years, and carrying on having it as adults, are no different from those in HST31.

Usual treatment for obesity and hyperphagia for adults with BBS (who have not started setmelanotide before age 18 years) is best supportive care. This includes dietary restrictions and lifestyle changes, including exercise, to manage symptoms. Liraglutide, semaglutide and tirzepatide may also be used for adults who meet the relevant eligibility criteria for treating overweight or obesity.

Results from clinical trials suggest that:

  • setmelanotide may reduce weight and body mass index (BMI) in people 6 years and over with BBS

  • there is more benefit for people who start setmelanotide aged between 6 and 17 years than for people who start treatment as adults

  • hunger scores and quality of life are improved with setmelanotide in the short term, although hunger scores may not reliably reflect changes in hyperphagia.

The updated trial evidence suggests that setmelanotide's treatment effect on weight-related outcomes in adults may be maintained in the longer term. But this is also uncertain. Setmelanotide has not been compared with liraglutide, semaglutide or tirzepatide for treating obesity in adults with BSS.

For adults with BBS who have not started setmelanotide before age 18 years, there are uncertainties in the company's revised economic model, including:

  • the average age of diagnosis in adults

  • the clinical effectiveness of liraglutide, semaglutide or tirzepatide for treating overweight and obesity compared with setmelanotide

  • the expected distribution of hyperphagia severity in adults before setmelanotide treatment

  • the approach used for modelling changes in hyperphagia

  • whether setmelanotide has added liver or kidney benefits in a mixed population of children, young people and adult above the benefits seen from reducing BMI

  • how common the visual and cognitive symptoms of BBS are in people who would have treatment in the NHS

  • potential uncaptured benefits.

Because of the uncertainties in the clinical evidence and economic model, it was not possible to determine the most likely cost-effectiveness estimates for setmelanotide in people starting treatment as adults. So, it should not be used for this population.

In people who start having setmelanotide aged between 6 and 17 years, and keep having it in adulthood, the cost-effectiveness estimates are within the range that NICE considers an acceptable use of NHS resources. So, it can be used for this population.