Overall Survival Is an Outdated Requisite for Drug Approval in Cancer Therapy

This transcript has been edited for clarity.

Hello. I'm Dr Maurie Markman from Cancer Treatment Centers of America in Philadelphia.

I want to briefly discuss a very important but controversial topic: the mandated role of overall survival in phase 3 randomized trials in the approval of antineoplastic agents for use in the United States.

There has been a major shift by the US Food and Drug Administration (FDA) to include progression-free survival as an acceptable endpoint in randomized phase 3 trials for the approval of drugs. This is appropriate, in my opinion. But a number of individuals, including prominent academics, have claimed that this is a meaningless surrogate, and unless you can prove overall survival or perhaps some other evidence of benefit, then drugs should not be approved.^[1,2,3] The gold standard, in the opinion of these individuals, is overall survival.

There are a number of examples as to why this insistence on overall survival in randomized phase 3 trials is problematic.

Therapy given after a patient progresses while on treatment in a trial can have many potential benefits. Cancer is becoming more of a chronic illness, particularly in clinical settings where we have new and improved anti-cancer agents. After a patient progresses, they still may receive up to three or more agents that can impact favorably on survival.