Nice update: treatment recommended for bile duct cancer

What’s the guidance?

TA722 Pemigatinib for treating relapsed or refractory advanced cholangiocarcinoma with FGFR2 fusion or rearrangement

Does it replace earlier guidance?
No. Pemigatinib was not recommended at the appraisal consultation draft stage, but the company has provided further information to address the uncertainties highlighted by the committee. This has enabled the committee to conclude pemigatinib is most likely within the range that Nice considers a cost-effective use of NHS resources for a life-extending treatment at end of life.

Who is it for and what does it cover?

Pemigatinib (also known as Pemazyre and made by the Incyte Corporation) is recommended within its conditional marketing authorisation for treating a rare form of bile duct cancer – locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement.

Pemigatinib is an option for adults whose cancer has progressed after chemotherapy and around 50 people will be eligible for treatment with it. Evidence suggests that pemigatinib works better than current treatments at this stage of cancer. Current treatment is active symptom control with or without chemotherapy.

What are the benefits?

This is a life-extending treatment for people with this extremely rare form of bile duct cancer. Treatment options for this form of cancer have not improved in a long time and Nice recognised there was an urgent unmet clinical need for people who have this disease. The decision comes after the company was able to work with Nice in addressing the concerns highlighted by the committee in the previous draft guidance. This recommendation is a great example of how Nice and companies, by working closely and collaboratively, can really help make a difference to patients.

What are the financial implications?

Nice do not expect this guidance to have a significant impact on resources. The resource impact of implementing the recommendations in England will be less than £5m per year in England (or approximately £9,000 per 100,000 population, based on a population for England of 56.3 million people).

This is because the population size is small. It is estimated around 50 people per year are eligible for treatment with pemigatinib. There may be additional costs incurred for FGFR2 testing, which is not currently done for this population group.

Gary Shield is resource impact assessment manager at Nice