NICE publishes draft recommendation for larotrectinib

The National Institute for Health and Care Excellence (NICE) has issued draft guidance rejecting the use of larotrectinib on the NHS for patients with NTRK fusion-positive solid tumours, which includes a small number of people with cholangiocarcinoma (bile duct cancer).

What does this mean?

NICE is responsible for assessing new medicines and making recommendations about whether they should be adopted for routine use by the NHS in England, based upon their cost and how effective they are.

NICE’s draft recommendation for larotrectinib is that it should not be used by the NHS in England.

It is important to note that this is a draft recommendation, and it is still under consultation. It is not uncommon for new treatments to receive a negative recommendation at this stage of NICE’s process. We know that four-in-five (80%) cancer drugs go on to be approved after further review.

What about Scotland, Wales and Northern Ireland?

• In Scotland, the Scottish Medicines Consortium (SMC) assesses drugs to decide whether they will be funded on the NHS. This is a separate process from NICE so they may reach a different decision. The SMC has not yet assessed larotrectinib, but they have approved NHS funding for another treatment that targets NTRK fusion-positive tumours – entrectinib.
• In Wales, the All Wales Medicines Strategy Group (AWMSG) assesses whether the NHS will fund the drug. This usually follows NICE guidance.
• In Northern Ireland, the Department of Health, Social Services and Public Safety (DHSSPS) look at NICE guidance to decide whether to follow their decision. Usually, the NICE guidance is followed.

What is larotrectinib?

Larotrectinib is a treatment that targets a specific gene fault, known as NTRK fusions, that are found in some tumours and encourage cancer cells to grow quickly. NTRK fusions are present in fewer than 1-in-100 cholangiocarcinoma patients.

Larotrectinib may be suitable for patients who meet all the following criteria:

• their disease is locally advanced, can’t be removed through surgery, or has spread (metastasised).
• they have no other satisfactory treatment options.
• their tumour displays a NTRK gene fusion.

Clinical trial evidence suggests that tumours with NTRK gene fusions shrink in response to larotrectinib, but it is difficult to know how well it works because:

• it was not compared with other treatments in the trials.
• the people in the trials did not fully reflect the people who would have larotrectinib in the NHS.
• there is evidence that larotrectinib works well for some types of NTRK fusion-positive tumours and less well for some other types, and for some tumour types there is little or no evidence.

Why have NICE made this draft recommendation?

Within their draft recommendation, NICE acknowledges that the evidence suggests tumours with NTRK gene fusions shrink when treated with larotrectinib. However, due to uncertainties in the data, it has not been possible for them to conclude that larotrectinib offers “value for money” for the NHS.

This draft recommendation from NICE is based on the evidence they have received and heard so far, it is not a final decision.

The pharmaceutical company and other stakeholders (including patients) now have an opportunity to respond to the decision and submit further evidence.

AMMF’s reaction to the announcement

Paul Howard, Head of Policy and Research at AMMF, said:

“Whilst NTRK gene fusions are relatively uncommon in cholangiocarcinoma, the current treatment options for these patients are very limited and offer little benefit. Therefore, this recommendation by NICE, whilst not final, is very disappointing.

“We understand that there is very limited evidence for how effective larotrectinib is for patients with NTRK fusion-positive cholangiocarcinoma due to the small number involved in clinical trials. However, the treatment has been shown to shrink some tumours with NTRK gene fusions, whilst being better tolerated than other treatment options.

“We are hopeful that NICE and the pharmaceutical company can find a solution that will enable the small number of NHS patients with NTRK fusion-positive cholangiocarcinoma to access this treatment which can potentially extend survival and improve quality of life.”

What does this mean for patients already receiving larotrectinib?

This recommendation will not affect patients who started treatment with larotrectinib in the NHS before this guidance was published. Those people may continue without change to the funding arrangements in place for them, until they and their NHS healthcare professional consider it appropriate to stop.

What happens next?

NICE is now welcoming additional evidence and comments from the pharmaceutical company, the public, and charities. Their committee will then meet again to consider new evidence and comments before publishing a final decision.

AMMF will respond to the consultation, advocating for access to this treatment for people diagnosed with NTRK fusion-positive cholangiocarcinoma.

How you can help

The voices of those personally affected by cholangiocarcinoma could help to shape the final decision.

• If you or a loved one has experience of receiving treatment with larotrectinib, AMMF would like to hear from you. Please email [email protected] before 22 April 2026.
• NICE is welcoming comments on their draft recommendation from members of the public via their website. If you would like to share your experiences of cholangiocarcinoma, and what access to a treatment like larotrectinib could mean for you or your loved ones, please complete their online form by 5pm on Friday 24 April 2026.

AMMF is extremely grateful to everyone who has already shared their experiences and supported our evidence submission for this NICE appraisal. Click here to read a report of the findings from our online survey.