Larotrectinib is recommended 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
they have no satisfactory treatment options.
It is recommended only if the conditions in the managed access agreement for larotrectinib are followed.
This recommendation is not intended to affect treatment with larotrectinib that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children and young people, this decision should be made jointly by the clinician and the child or young person or their parents or carers.
There is no standard treatment for NTRK fusion-positive solid tumours, so current treatment is based on where in the body the cancer starts. Larotrectinib is a histology-independent treatment. This means that it targets a genetic alteration, NTRK gene fusion, that is found in many different tumour types irrespective of where the cancer starts.
Evidence from trials suggests that tumours with NTRK gene fusions shrink in response to larotrectinib. But it is difficult to know how well larotrectinib works because it is not compared with other treatments in the trials. Also, there is evidence that larotrectinib works well for some types of NTRK fusion-positive tumour, but little or no evidence for other types.
The cost-effectiveness estimates for larotrectinib are very uncertain because:
they are based on data from a population that is different to that seen in NHS clinical practice and
there is substantial uncertainty about how long people would live after their disease gets worse.
Collecting more data would help to address some of the uncertainties in the clinical evidence. Larotrectinib has the potential to be a cost-effective use of NHS resources at its current price so it is recommended through the Cancer Drugs Fund while these data are collected.