Expanded access programmes: patient interests versus clinical trial integrity

The first-line phase 3 study with tremelimumab did not show an improvement in its primary endpoint, overall survival, and the drug was never licensed.2,7 At least a third of patients randomly assigned to the control group who were alive at the end of the study or censored at any point had received ipilimumab at some stage, which might well have contributed to the negative outcome of the study.3 This conclusion was supported by the finding that outcomes for patients in US and non-US sites were different, with treatment with tremelimumab resulting in significantly better survival in patients treated in non-US centres, where the expanded access programme for ipilimumab was less readily available, and no difference in US centres, where ipilimumab was readily available via the expanded access programme. [...]patient information and consent forms should explicitly state that involvement in a clinical trial might limit access to future treatments that are made available as part of an expanded access programme.