Background: Crizotinib (Xalkori) has demonstrated clinical activity in ALK (Anaplastic Lymphoma Kinase) positive non-small cell lung cancer (NSCLC), and is registered in Australia and New Zealand for this indication. Access to a particular therapy, in a life threatening situation, where treatment options are also limited, prior to market approval is often a challenge. Crizotinib access filled this gap and addressed the unmet need.
Methods: Pfizer enabled a Global Access Program to provide compassionate access to crizotinib prior to market authorisation. The program was made available from Jan 2012 and closed for patients in Australia in Sep 2013, whereas continued for New Zealand patients well into 2014. Minimal patient data as required to administer the program was captured on these patients. Duration of treatment was determined from the electronic data base run and maintained by Pfizer.
Results: A total of 82 patients were enrolled and 67 of these had an ALK-positive advanced NSCLC. 11 patients never received Crizotinib and were excluded from the analysis. For the 56 patients who were initiated on therapy with crizotinib, 47 belonged to Australia and 9 to New Zealand. Median age was 54.9 years. At the data cut-off date of 31 July 2015, 16 patients remained alive and on treatment with crizotinib, while 40 had been deceased or inactive. Of the 56 patients, 90% had received at least one prior chemotherapy, 5 being first line NSCLC patients. For both first and second line patients, the median time on treatment was 355 days, while for the second line patients alone, the median time on treatment was 337 days. No new safety signals were reported and all deaths were attributed to disease progression.
Conclusion: Treatment with crizoitnib was well tolerated with minimal dose reduction or discontinuation of therapy and treatment duration compares well with published data from the controlled clinical trial experience in both first and second line patients.