Back in October, a couple of articles were published in the Journal of Thoracic Oncology that described results in a compassionate use program with Tarceva (erlotinib), one coming out of Asia, and the other out of Europe. These trials together enrolled thousands of patients who then received single agent Tarceva, and while the results were in keeping with our expectations, it provides an occasion to consider the merits of compassionate use programs for new agents in oncology. (For the basics of phases of clinical trials and drug development, check out this review post on the subject.)
Compassionate use programs are generally initiated with agents that have already undergone fairly extensive testing but are not yet commercially available for routine use. They remain a sensitive topic, since for some patients these new drugs may be very helpful, and a much wider range of patients hold out tremendous hope that a new drug not yet commercially available will emerge as remarkably helpful for them. Providing a treatment at no charge that would likely to be quite expensive once it’s approved, the advantages to patients are readily obvious.
