[Skip to Content]
[Skip to Content Landing]
Views 1,790
Citations 0
Research Letter
July 3, 2019

Surrogate End Points and Patient-Reported Outcomes for Novel Oncology Drugs Approved Between 2011 and 2017

Author Affiliations
  • 1Cardinal Health Specialty Solutions, Cardinal Health, Dublin, Ohio
  • 2Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill
  • 3Aptitude Health, Atlanta, Georgia
JAMA Oncol. Published online July 3, 2019. doi:10.1001/jamaoncol.2019.1760

The US Food and Drug Administration (FDA) may approve drugs based on surrogate end points that reasonably predict that a drug provides clinical benefit.1,2 If approved via the accelerated approval pathway, the FDA may require postmarketing studies to confirm the perceived clinical benefit. Assessment of patient-reported outcomes (PROs), defined as any report on a patient’s health that comes directly from the patient, can also play a key role in understanding benefits and tolerability of oncology drugs.3 We sought to examine the use of surrogate end points for overall survival (OS) in new oncology drug approvals, to evaluate the use of PROs in trials supporting approvals, and to determine whether oncology drugs initially approved without evidence of OS or PRO benefits demonstrated improvements in either measure postapproval.