Cancer Drugs Approved on the Basis of a Surrogate End Point and Subsequent Overall Survival: An Analysis of 5 Years of US Food and Drug Administration Approvals | Targeted and Immune Cancer Therapy | JAMA Internal Medicine | JAMA Network
[Skip to Navigation]
Access to paid content on this site is currently suspended due to excessive activity being detected from your IP address 18.204.227.34. Please contact the publisher to request reinstatement.
1.
Fauber  J.  FDA approves cancer drugs without proof they’re extending lives.Milwaukee Sentinel. October 26, 2014. http://www.jsonline.com/watchdog/watchdogreports/fda-approves-cancer-drugs-without-proof-theyre-extending-lives-b99348000z1-280437692.html. Accessed August 24, 2015.
2.
Prasad  V, Kim  C, Burotto  M, Vandross  A.  The strength of association between surrogate end points and survival in oncology: a systematic review of trial-level meta-analyses.  JAMA Intern Med. 2015;175(8):1389-1398.PubMedGoogle ScholarCrossref
3.
US Government Accountability Office.  FDA needs to enhance its oversight of drugs approved on the basis of surrogate endpoints. GAO-09-866. http://www.gao.gov/products/GAO-09-866. Published September 23, 2009. Accessed August 24, 2015.
4.
Herper  M.  The FDA is basically approving everything: here’s the data to prove it. Forbes. http://www.forbes.com/sites/matthewherper/2015/08/20/the-fda-is-basically-approving-everything-heres-the-data-to-prove-it/. Posted August 20, 2015. Accessed August 24, 2015.
Research Letter
Health Care Reform
December 2015

Cancer Drugs Approved on the Basis of a Surrogate End Point and Subsequent Overall Survival: An Analysis of 5 Years of US Food and Drug Administration Approvals

Author Affiliations
  • 1Medical Oncology Service, National Cancer Institute, National Institutes of Health, Bethesda, Maryland
  • 2Division of Hematology and Medical Oncology, Knight Cancer Center, Oregon Health and Sciences University, Portland
JAMA Intern Med. 2015;175(12):1992-1994. doi:10.1001/jamainternmed.2015.5868

Most contemporary approvals of new cancer drugs are made on the basis of a surrogate end point, such as response rate or progression-free survival (PFS).1 When the approval is based on a surrogate end point, subsequent studies are advised and often obligated to clarify the drug’s effect on overall survival. One such drug is bevacizumab, which received accelerated approval on the basis of PFS for patients with metastatic breast cancer. Later findings revealed no improvement in overall survival and significant toxicity, which required a removal of marketing authorization.2

A 2009 Government Accountability Office report criticized the US Food and Drug Administration (FDA) for failing to enforce postmarketing study commitments for surrogate approvals. Among the more than 400 postmarketing studies requested, approximately 30% were pending, ongoing, delayed, or terminated years later, yet the FDA never exercised its authority to remove a product from the market.3 For these reasons, we sought to investigate how often cancer drugs are approved based on a surrogate end point, whether subsequent studies for these drugs are reported, and whether the drugs improve overall survival.

×