Setmelanotide is recommended as an option for treating obesity and hyperphagia in genetically confirmed Bardet-Biedl syndrome (BBS) in people aged 6 years and over, only if they are aged between 6 and 17 years when treatment starts. These people can carry on having setmelanotide as adults until they need to stop. Setmelanotide is only recommended if the company provides it according to the commercial arrangement.
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 them, their healthcare professional, 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, and their families and carers. It causes hyperphagia (characterised by a feeling similar to starvation), which can lead to early onset severe obesity 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. There seems to be more benefit for people who start setmelanotide aged between 6 and 17 years than for people who start as adults. The results 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.
There are also uncertainties in the economic model. Some benefits of setmelanotide may not have been captured adequately and some may have been overestimated in the cost-effectiveness analysis. So, the size of any uncaptured benefits is unknown. In the population of children, young people and adults (which reflects the current population in the NHS), the uncertainties in the evidence mean that the cost-effectiveness estimates for setmelanotide are higher than what NICE normally considers an acceptable use of NHS resources within the context of a highly specialised technology. But setmelanotide is estimated to be cost effective in people who start taking it aged between 6 and 17 years. So, setmelanotide is recommended for these people.