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Draft guidance consultation

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1 Recommendations

1.1 Setmelanotide is not recommended, within its marketing authorisation, for treating obesity and hyperphagia in genetically confirmed Bardet-Biedl syndrome (BBS) in people aged 6 years and over.

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 clinician consider it appropriate to stop. For children or young people, this decision should be made jointly by them, their clinician, and their parents or carers.

Why the committee made these recommendations

BBS is a debilitating rare genetic condition that severely affects the quality of life of people with the condition, their families and carers. It causes early onset severe obesity and hyperphagia (characterised by a feeling similar to starvation), and affects many different parts of the body. These symptoms are managed with best supportive care, which includes dietary restrictions and lifestyle changes, including exercise.

Results from clinical trials suggest that setmelanotide may reduce weight and body mass index (BMI) in people aged 6 years and over. They also suggest that hunger scores and quality of life are improved with setmelanotide in the short term, although hunger scores may not reliably reflect changes in hyperphagia. Follow up in the trials is short, so the long-term effects of setmelanotide are uncertain.

In addition to the uncertainties in the clinical evidence, there are issues with the economic model. These include assumptions:

  • that everyone has severe hyperphagia when they enter the model

  • about setmelanotide's effect on BMI‑Z in people aged under 18 years

  • that all people whose condition responds to setmelanotide have mild hyperphagia

  • on the quality-of-life values related to BMI

  • about the effect on quality of life of BBS comorbidities not related to obesity

  • on what the quality-of-life benefits are for carers of people whose condition responds to treatment.

This means that it is not possible to determine the most likely cost-effectiveness estimates. So, setmelanotide cannot be recommended and more data is needed.