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    Selumetinib for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over

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    1 Recommendations

    1.1 The committee was minded not to recommend selumetinib as an option for treating symptomatic and inoperable plexiform neurofibromas (PN) associated with type 1 neurofibromatosis (NF1) in children aged 3 and over.

    1.2 The committee recommends that NICE requests further clarification and analyses from the company, which should be made available for the second evaluation committee meeting, and should include:

    • a patient-level model, including:

      • a progression-free state for the best supportive care arm

      • allows progression to happen after the age of 18

      • accounts for the progressive nature of the condition, age and location of PN

      • clinical outcomes that are important to people with PN, carers and clinicians

    • the possibility for selumetinib treatment to continue beyond the age of 18

    • utility values obtained from patients in the trial in the analysis by using a mapping algorithm or validation of the time trade off utilities by the mapped utilities

    • carer disutility values that are:

      • dependent on PN location and morbidity experienced

      • applied to 1 carer

      • in both selumetinib and best support care arms

    • the length of utility benefit waning after stopping treatment to be 1 year

    • a breakdown of full resource use costs for selumetinib and best supportive care arms, with comparison between the 2 arms

    • inclusion of 2 additional MRIs in the selumetinib arm.

    Why the committee made these recommendations

    NF1 is a genetic disease that affects multiple organ systems. Around 25% of people with NF1 develop non-malignant peripheral nerve sheath tumours called PN. PN can affect multiple body regions and reach extremely large sizes. Most PNs associated with NF1 are symptomatic, and can cause pain, disfigurement and difficulties with physical functioning. If a PN is inoperable, people have best supportive care, including pain management, physiotherapy, psychological support and sometimes procedures such as a tracheostomy to alleviate severe airway morbidities.

    Clinical trial evidence suggests that selumetinib is effective at reducing the volume and size of PN compared with best supportive care.

    However, the company uses a simplistic model structure based on the volume of PN. It does not account for the heterogeneity of the disease or include clinical outcomes, such as pain, which are important to people with PN and their parents or carers. The committee acknowledged that selumetinib may be an effective treatment option for people with inoperable PN, however the economic model was not appropriate to estimate the costs and benefits of selumetinib. Further information is needed before a recommendation can be made.