Treatment with unfunded drugs in oncology: the impact of access programmes and clinical trials

Where proven cancer therapies remain unfunded, patients are faced with difficult decisions regarding personally covering some or all of the treatment cost. Clinical trials provide an alternative form of access to unfunded drugs.

To examine the patient population and utilisation of the colorectal cancer cetuximab Interim Access Programme (IAP) in Australia.

We retrospectively analysed de-identified data collected as part of the costsharing colorectal cancer cetuximab IAP in Australia, which extended from June 2005 until November 2009. The impact of the CO20 clinical trial that opened in early 2008 and provided free access to cetuximab was also examined.

Eight hundred and fifty-eight patients received ≥1 treatment on the IAP. The median age of participants was lower than the general colorectal cancer population (61 vs 71 years). A greater uptake by males was limited to patients over 65 years old. Socioeconomic status correlated with programme enrolment, and there appeared to be lower uptake among regional patients. The majority (93%) of patients received combination treatment with irinotecan, with a trend towards increasing use of single-agent cetuximab over time. The median time-on-treatment was longer in patients treated with combination therapy than with monotherapy (12 vs 8 weeks). The CO20 trial had minimal impact on IAP enrolment and approximately doubled the number of Australian patients receiving cetuximab.

Patients' age and gender in older patients impacted on participation in the IAP. Both the IAP and the CO20 trial contributed to a substantial proportion of Australian patients accessing an unfunded treatment, with an estimated 50% of the eligible patient population receiving cetuximab.