A CT scan showing a case of cholangiocarcinoma
The drug pemigatinib (Pemazyre) will now be an option for people with a rare type of bile duct cancer that has continued to advance or spread after initial chemotherapy.
The National Institute of Health and Care Excellence (NICE) has approved the use of this drug in adults with bile duct cancer (cholangiocarcinoma) that has an unusual form of a molecule called FGFR2 on its surface and doesn’t respond to standard treatment.
Kruti Shrotri, head of policy development at Cancer Research UK, said that this decision is “good news” for people affected by this rare type of advanced bile duct cancer.
“There are currently very few effective treatments for this type of cancer. This decision should help widen the options available for those patients whose cancer has relapsed or doesn’t respond to other treatment.”
NICE estimates that around 50 people will be eligible for the treatment.
An urgent need for new treatments
There are currently no effective treatments available in the NHS to treat this kind of bile duct cancer, and treatment for the condition hasn’t improved for over a decade.
People with advanced bile duct cancer with an unusual form of the FGFR molecule (called an FGFR ‘fusion or rearrangement’) who don’t respond to treatment are currently only given therapies to control their symptoms, sometimes with modified FOLFOX chemotherapy, a combination of 3 chemotherapy drugs.
Patient experts told the NICE committee about the difficulty of being diagnosed with a cancer for which there are very few treatment options, especially as they are often told that they have a poor outlook while still feeling well.
Pemigatinib represents new hope for these patients whose options are extremely limited.
How does the treatment work?
Pemigatinib belongs to a group of medicines called ‘protein kinase inhibitors’. These drugs work by blocking molecules in the body called protein kinases, which help send messages within and between cells.
Pemigatinib targets a specific type of protein kinase called ‘fibroblast growth factor receptors’, which are found on the surface of cancer cells and are involved in their growth and spread.
As a protein kinase inhibitor, pemigatinib can block the FGFRs from sending messages and prevent the cancer from growing.
What’s the evidence?
The clinical evidence for the effectiveness of this treatment came from a phase 2, open label study. The trial included 146 patients with advanced bile duct cancer that hadn’t responded to treatment who each had various alterations to the FGFR molecule on their cancer cells.
107 patients in the trial had FGFR fusions or rearrangements, 38 (35.5%) of whom responded to the treatment. 3 of these patients had no detectable cancer by the end of the study.
As a single-arm trial, the study did not directly compare the use of pemigatinib with current treatment options, so the data had to be compared indirectly with data about current treatments. This indirect comparison indicated that pemigatinib may be more effective than current treatments, but this wasn’t certain.
As a rare cancer, it’s difficult to get enough people to take part in these kinds of trials to get a robust comparison.
The NICE committee concluded that this uncertainty was acceptable due to the lack of effective treatment options and progress in developing new treatments, meaning that there is an urgent unmet need for these patients.
Overall, the treatment was considered cost-effective for use in the NHS by NICE and will now be an option on the NHS in England. NICE decisions are usually adopted in Wales and Northern Ireland as well, so the decision is likely to affect patients in all 3 nations. Scotland has a separate process for reviewing drugs.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE said: “I am pleased we are able to recommend this 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 we recognise there is an urgent unmet clinical need for people who have this disease.
“Today’s decision comes after the company was able to work with us 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.”