Selumetinib is recommended, within its marketing authorisation, for treating symptomatic and inoperable plexiform neurofibromas (PN) associated with type 1 neurofibromatosis (NF1) in children aged 3 and over, only if the company provides selumetinib according to the commercial arrangement.
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 PN 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.
Because of limited available evidence, 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. But, the committee acknowledged that selumetinib may be an effective treatment option for people with inoperable PN, even when considering the uncertainty in the economic modelling and the benefits not captured by the model. Based on the same considerations, it also provides value for money within the context of a highly specialised service. Selumetinib is therefore recommended for use in the NHS.