Impact of Availability of Companion Diagnostics on the Clinical Development of Anticancer Drugs

Mol Diagn Ther. 2017 Jun;21(3):337-343. doi: 10.1007/s40291-017-0267-y.


Background: Companion diagnostics permit the selection of patients likely to respond to targeted anticancer drugs; however, it is unclear if the drug development process differs between drugs developed with or without companion diagnostics. Identification of differences in study design could help future clinical development.

Patients and methods: Anticancer drugs approved for use in solid tumors between 28 September 2000 and 4 January 2014 were identified using a search of the US FDA website. Phase III trials supporting registration were extracted from the drug label. Each published study was reviewed to obtain information about the phase I and II trials used for the development of the respective drug.

Results: We identified 35 drugs and 59 phase III randomized trials supporting regulatory approval. Fifty-three phase I trials and 47 phase II trials were cited in the studies and were used to support the design of these phase III trials. The approval of drugs using a companion diagnostic has increased over time (p for trend 0.01). Expansion cohorts were more frequently observed with drugs developed with a companion diagnostic (62 vs. 20%; p = 0.005). No differences between drugs developed with or without a companion diagnostic were observed for the design of phase I and II studies.

Conclusions: The approval of drugs developed with a companion diagnostic has increased over time. The availability of a companion diagnostic was associated with more frequent use of phase I expansion cohorts comprising patients selected by the companion diagnostic.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Agents*
  • Clinical Trials, Phase III as Topic
  • Drug Approval
  • Humans
  • Molecular Diagnostic Techniques*
  • Neoplasms / drug therapy
  • Patient Selection*
  • Randomized Controlled Trials as Topic


  • Antineoplastic Agents