NICE has approved larotrectinib for use within the Cancer Drugs Fund as an option for treating neurotrophic tyrosine receptor kinase (NTRK) fusion-positive solid tumours in adults and children if:
- The disease is locally advanced or metastatic or surgery could cause severe health problems, and
- There are no satisfactory treatment options.
The key clinical evidence for larotrectinib comes from a pooled analysis of 102 patients in three single-arm clinical trials.
NAVIGATE (n=62) is an ongoing trial for patients ≥12 years of age with locally advanced or metastatic tumours with NTRK gene fusions who have had prior therapy or who would be unlikely to clinically benefit from standard care. NAVIGATE contributed 62 patients to the pooled analysis.
SCOUT (n=32) is an ongoing trial for children with locally advanced or metastatic solid tumours or primary central nervous system (CNS) tumours.
LOXO‑TRK‑14001 (n=8) was a dose-finding study in patients with solid tumours with NTRK gene fusions.
The group analysed for efficacy was further split by patients with primary CNS tumours (n=9) and all other patients (n=93). In its decision, NICE noted the small patient numbers in each trial and that the trials were single arm and did not include a control group. However, given the rarity of the gene fusion, the NICE appraisal committee concluded that the evidence was appropriate for decision making.
The cost-effectiveness estimates for larotrectinib remain uncertain and collecting data through the Cancer Drugs Fund will help to address these uncertainties.
NICE concluded that larotrectinib has the potential to be a cost-effective use of NHS resources at its current price and, hence, it is recommended for use through the Cancer Drugs Fund while these data are collected.