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  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of the criteria considered by the committee, and the clinical and economic considerations reasonable interpretations of the evidence?

  • Question on Consultation

    Are the provisional recommendations sound and a suitable basis for guidance on the use of setmelanotide in the context of national commissioning by NHS England?

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    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of race, gender, disability, religion or belief, sexual orientation, age, gender reassignment, pregnancy and maternity?
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Introduction

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1 Recommendations

1.1 The committee was minded not to recommend setmelanotide as an option for treating obesity and controlling hunger caused by pro-opiomelanocortin (POMC) deficiency, including proprotein convertase subtilisin/kexin type 1, or leptin receptor (LEPR) deficiency in people 6 years and over.

1.2 The committee recommends that NICE requests further information from the company, which should be made available for the second evaluation committee meeting. This should include exploratory analyses using the committee's preferred assumptions with the following:

  • using the UK patient population distribution for the pooled population, as well as scenario analyses for sub-populations by deficiency type and age

  • varying setmelanotide's long-term treatment effect on body mass index

  • using alternative utility values for severe hyperphagia.

Why the committee made these recommendations

POMC and LEPR deficiencies are rare genetic disorders of obesity that severely affect the quality of life of people with them, and their families and carers. They cause early onset, extreme obesity and hyperphagia (characterised by a feeling similar to starvation) and are linked with many chronic conditions. They are also likely to shorten life expectancy. Current management (best supportive care) focuses on dietary restrictions and lifestyle changes, including exercise.

Results from clinical trials suggest that setmelanotide may reduce weight and body mass index (BMI) in people with obesity caused by POMC and LEPR deficiencies. Evidence also suggests that hunger and quality of life are improved with setmelanotide. However, follow up in the trials is short, so the long-term effects of setmelanotide are uncertain. Also, it has not been compared with best supportive care.

There are uncertainties in the economic modelling, including:

  • the proportion of people with the 2 different conditions in the model

  • setmelanotide's long-term effect on BMI

  • how setmelanotide affects hyperphagia

  • the dosing of setmelanotide

  • how long people having best supportive care live

  • the stopping rate for setmelanotide

  • quality‑of‑life values for severe hyperphagia

  • what the discount rate for health benefits should be.

Because of the uncertainties in the clinical trials and the economic model, it is unclear whether the criteria for a quality-adjusted life-year weighting has been met (that is, extra health and quality-of-life benefits of setmelanotide are considered to be substantial). Also, the cost-effectiveness estimates are higher than what NICE usually considers acceptable for highly specialised technologies.

So, setmelanotide is not considered an appropriate use of NHS resources within the context of a highly specialised service and cannot be recommended.