Presurgical window of opportunity trial design as a platform for testing anticancer drugs: Pros, cons and a focus on breast cancer

Marcello Maugeri-Saccà, Maddalena Barba, Patrizia Vici, Laura Pizzuti, Domenico Sergi, Teresa Catenaro, Luigi Di Lauro, Marcella Mottolese, Daniele Santini, Michele Milella, Ruggero De Maria

Research output: Contribution to journalReview article


The high attrition rate is a major issue in anticancer drug development. Among the alternative trial designs, presurgical window of opportunity trials envision a short course treatment in the time window between diagnostic biopsy and surgery in a moderately-sized patient population. This approach allows testing therapeutics when pre- and post-treatment tumor tissues are available for comprehensive molecular analyses. The emerging evidence may help define the ability of a given agent to modulate its target(s) and help obtain a broader picture of the molecular changes operated by the treatment. The resulting gain may outweigh the potential harms for patients in the early disease setting. Window of opportunity trials have been extensively applied to breast cancer. Overall, a wider use of these trial designs might lead to the identification of potential responders, ineffective drugs or combinations, and ultimately contribute to enhance the efficiency of the clinical developmental process.

Original languageEnglish
Pages (from-to)132-142
Number of pages11
JournalCritical Reviews in Oncology/Hematology
Publication statusPublished - Oct 1 2016



  • Attrition rate
  • Breast cancer
  • Cancer therapeutics
  • Window of opportunity trials

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Geriatrics and Gerontology

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