NICE has approved entrectinib (Rozlytrek, Roche) for use within England's Cancer Drugs Fund as an option for treating neurotrophic tyrosine receptor kinase (NTRK) fusion-positive solid tumours in adults and children 12 years and older if:
- The disease is locally advanced or metastatic or surgery could cause severe health problems, and
- They have not had an NTRK-inhibitor before, and
- They have no satisfactory treatment options.
The key clinical evidence comes from a pooled analysis of 66 people across 13 tumour types, which showed a clinically relevant overall response rate across tumour types. However, there was considerable uncertainty about the extent to which the response translated into clinically meaningful survival benefits.
At the most recent data-cut, median follow-up was short and the survival data were immature. The number of patients with specific cancers was very small, creating uncertainty in the robustness of all survival data. Furthermore, there is no direct evidence of entrectinib’s effectiveness compared with established management.
The cost-effectiveness estimates for entrectinib are uncertain because of limitations in the data. Some of these estimates are higher than the normal NICE threshold, meaning entrectinib cannot be recommended for routine use in the NHS. Providing entrectinib through the Cancer Drugs Fund will help to address some of the uncertainty in the evidence.