The primary goal of cancer drug therapy is to prolong life or improve quality of life. Overall survival (OS) is the most reliable clinical trial endpoint to inform regulatory approvals of new cancer drugs. However, most cancer drugs approved in the US and Europe lack evidence of OS benefit. For instance, more than two-thirds of cancer drug approvals granted by the US Food and Drug Administration (FDA) between 2008 and 2012 were based on surrogate endpoints, and only 14% of drugs initially approved on the basis of surrogate endpoints had demonstrated OS benefits after a median follow-up of 4.4 years. Further, drugs met the threshold for clinically meaningful benefit in less than half of trials supporting solid tumor indication approvals by the FDA between 2006 and 2016. Similarly, among cancer drugs approved by the European Medicines Agency (EMA) between 2009 and 2013, only 51% showed significant improvement in survival or quality of life at a minimum of 3.3 years follow-up, and survival gains over existing therapy were marginal.
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