NICE has published draft guidance recommending that Pfizer's Xalkori (crizotinib) to treat ROS1-positive advanced non-small-cell lung cancer is made available via the Cancer Drugs Fund (CDF).

ROS1-positive lung cancer is a recently discovered subtype of the disease, and NICE said that the evidence for Xalkori's effectiveness in treating it is limited.

In January, the appraisal committee concluded that Xalkori appears to be a promising treatment but limited data meant the cost-effectiveness estimates are highly uncertain.

Pfizer was invited to submit a proposal for including crizotinib in the CDF. A deal has been agreed with NHS England where Pfizer has agreed to provide it at a confidential discounted price while further data is gathered for NICE to review.

The data collection will run for five years, with annual reviews after year two to assess whether sufficient information has been gathered. It will continue to be available on CDF after the data collection has ended and while NICE reviews it again.

This decision means ROS1 diagnostic testing will also be available in England. Published studies indicate ROS1-positivity to occur in less than 1% of NSCLC patients.

Craig Eagle, Head of Oncology, Pfizer UK said: “Our priority is to secure access to crizotinib for patients with ROS1-positive advanced non-small cell lung cancer (NSCLC). The decision to make the treatment available via the Cancer Drugs Fund is therefore good news for patients in England.

"This process has, however, demonstrated the underlying access challenges that exist for people with rarer cancers. ROS1-positive advanced NSCLC is a particularly rare type of lung cancer. 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.”

The news comes just days after Xalkori was granted Breakthrough Therapy designation by the US FDA for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with MET exon 14 alterations with disease progression on or after platinum-based chemotherapy and for the treatment of patients with relapsed or refractory systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive.