Cost-effectiveness analyses of targeted oral anti-cancer drugs: a systematic review

Pharmacoeconomics. 2014 Jul;32(7):651-80. doi: 10.1007/s40273-014-0160-z.


Background: Over the last 15 years, a paradigm shift in oncology has led to the approval of dozens of targeted oral anti-cancer medications (OAMs), which have become the standard of care for certain cancers. While more convenient for patients than infused drugs, the possibility of non-adherence and the frequently high costs of targeted OAMs have proven controversial.

Objective: Our objective was to perform the first comprehensive review of cost-effectiveness analyses (CEAs) of targeted OAMs.

Methods: A literature search in PubMed, The Cochrane Library, and the Health Technology Assessment (HTA) reports published by the National Institute for Health Research HTA Programme in the UK was performed, covering articles published in the 5 years prior to 30 September 2013. Our inclusion criteria were peer-reviewed English-language full-text original research articles with a primary focus on CEA related to targeted OAMs. We categorized these articles by treatment setting (i.e. cancer site/type, line of therapy, and treatment and comparator) and synthesized information from the articles into summary tables.

Results: We identified 41 CEAs covering nine of the 18 targeted OAMs approved by the US FDA as of December 2012. These medications were studied in seven cancers, most often as second-line therapy for advanced-stage patients. In over half of treatment settings where a targeted OAM was compared with treatment that was not a targeted OAM, targeted OAMs were considered cost effective. Limitations in interpreting these findings include the risk of bias due to author conflicts of interest, cross-country variation, and difficulties in generalizing clinical trial evidence to community practice.

Conclusions: Several types of cost-effectiveness studies remain under-represented in the literature on targeted OAMs, including those for follow-on indications approved after the initial indication for a drug and for off-label indications, head-to-head comparisons of targeted OAMs with other targeted OAMs and targeted intravenous therapies, and studies that adopt a perspective other than the payer's. Keeping up with the increasing number of approved targeted OAMs will also prove an important challenge for economic evaluation.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review
  • Systematic Review

MeSH terms

  • Administration, Oral
  • Antineoplastic Agents / administration & dosage*
  • Antineoplastic Agents / economics*
  • Antineoplastic Agents / therapeutic use
  • Cost-Benefit Analysis
  • Drug Costs*
  • Humans
  • Molecular Targeted Therapy / economics*
  • Molecular Targeted Therapy / methods
  • Neoplasms / drug therapy*
  • Neoplasms / economics
  • Neoplasms / metabolism


  • Antineoplastic Agents