NICE recommends Pfizer’s Xalkori in rare lung cancer

NICE has issued a Final Appraisal Determination that recommends the use of Pfizer’s Xalkori (crizotinib) in England under the Cancer Drugs Fund (CDF) for patients with ROS1-positive advanced non-small cell lung cancer (NSCLC).

The decision means that ROS1 diagnostic testing will also be available in England.

ROS1-positive advanced NSCLC is a rare type of lung cancer, which occurs in less than 1% of NSCLC patients.

Until now, a lack of available treatment options has meant that this form of cancer has been treated with cytotoxic chemotherapy with unpleasant side effects. Xalkori is taken orally and offers improved quality of life.

However, the route to this recommendation again highlights the challenges faced in gaining market access for treatments for rare conditions, with only small patient populations available to prove effectiveness in trials.

Craig Eagle, Pfizer UK’s head of Oncology, said: “Our priority is to secure access to crizotinib for patients with ROS1-positive advanced NSCLC. The decision to make the treatment available via the CDF is therefore good news for patients in England."

However, Eagle went on, “ROS1-positive advanced NSCLC is a particularly rare type of lung cancer, occurring in less than 1% of NSCLC patients. As such, there are limited data available for this small patient cohort, which presents a challenge for the current model of NICE assessment. As cancer science becomes more sophisticated and treatments for rarer cancers more targeted, this problem will become increasingly evident.

“We are committed to continuing to work in collaboration with all parties towards a long-term solution on how to appropriately assess the limited data available when working in small patient populations, to ensure timely routine access to modern cancer medicines, now and in the future.”

NICE had rejected the drug in first draft guidance because of uncertainties over its cost effectiveness based on a small, single-arm trial of 53 patients.

But it noted advice from clinical experts that the median progression-free survival (PFS) figure of 19.8 months in the trial cohort was clinically meaningful as lung cancer patients treated with chemotherapy show PFS of five months in untreated disease, and three months in previously-treated disease.

Overall survival data were absent, as so many of the patients on the trial were still alive at follow-up after 25.4 months.

NICE’s independent appraisal committee therefore accepted Xalkori showed promise in this form of the disease, and the proposal went forward under the CDF.

Dr Sanjay Popat, Consultant Medical Oncologist at The Royal Marsden NHS Foundation Trust said: “I am really pleased that both Pfizer and NICE have come to an agreement. Until now, there have been no targeted treatment options available through the NHS for patients with this rare type of lung cancer and neither has testing for ROS1 been routinely approved. This approval is an important step forward for our patients.”

The Scottish Medicines Consortium’s (SMC) appraisal process for crizotinib in ROS1-positive advanced NSCLC is underway, with a decision expected in June. Pfizer and Merck have a strategic alliance exploring routes to access for patients in Wales and Northern Ireland.

NICE already recommends Xalkori for ALK-positive lung cancer.