Pragmatic solutions for optimizing oncology drug development trials

Jaap Verweij, Carole Longson, Marie von Lilienfeld-Toal, David Cahane, Thomas R. Jeffry Evans, Victoria L. Chiou, Denis Lacombe, Gwenaelle Gravis, Daniel A. Goldstein

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review


Practical strategies are needed for strengthening the development of cancer drugs, focusing on optimization of treatment dosage, and improvements in the diversity of patients enrolled in clinical trials. Treatment optimization (both pre- and post-marketing) offers potential to deliver a range of benefits including a reduction in the burden of side-effects to patients and cost of drugs to health care systems. For immune checkpoint inhibitors (ICIs), dosage optimization involves reducing duration of treatment, extending interval administration, and a ‘stop and go’ approach where treatment is resumed if patients progress. Such initiatives will be necessary to optimize effectiveness and achieve the economic savings needed for global access to these drugs. Strategies are needed to increase patient diversity so that clinical trials start to represent the patient groups the interventions being studied intend to treat. The potential of decentralized trials is explored in greater depth as one of the ways to increase clinical trial diversity.

Original languageEnglish
Title of host publicationComprehensive Precision Medicine, First Edition, Volume 1-2
ISBN (Electronic)9780128240106
StatePublished - 1 Jan 2023


  • Decentralized trial
  • Dose optimization
  • Dose refinement
  • Trial population diversity


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