NICE recommends obesity medication for Bardet-Biedl syndrome

The National Institute for Health and Care Excellence (NICE) recently published recommendations for the use of setmelanotide when treating obesity and hyperphagia in genetically confirmed Bardet-Biedl syndrome (BBS).

BBS, a rare genetic condition, causes hyperphagia which can lead to early onset severe obesity. Currently those living with the condition manage these symptoms with supportive care such as dietary restrictions, exercise and lifestyle changes.

Recent clinical trials suggest that setmelanotide may reduce weight and body mass index in people aged 6 years and over, with increased benefits for people who start setmelanotide aged between 6 and 17 years, than for people who start as adults.

The researchers indicated 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.

 As the follow up in the trials is short, the long-term effects of setmelanotide are uncertain and researchers indicated that some benefits of setmelanotide may not have been captured adequately, while some may have been overestimated in the cost-effectiveness analysis.

Therefore, although 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, they have estimated setmelanotide is cost effective in people who start taking it aged between 6 and 17 years.