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Updated: Jun 25, 2026

Evaluating the Effectiveness of Cancer Drug Sensitization In Vitro and In Vivo
Published on: February 6, 2015
Improving the evaluation of new cancer treatments: challenges and opportunities
Mace L Rothenberg1, David P Carbone, David H Johnson
1Vanderbilt-Ingram Cancer Center, 777 Preston Research Building, Nashville, Tennessee 37232-6307, USA. mace.rothenberg@vanderbilt.edu
Abstract:
There are, at present, ten times more anticancer drugs being tested in clinical trials than there were 15 years ago. Many of the new classes of agents, however, are predicted to work in only small subpopulations of patients, target unconventional aspects of tumour development and interact with other agents in an unpredictable manner. How can clinical trials be re-designed to accommodate the new features of targeted anticancer drugs?
Insights
The number of anticancer drugs in clinical trials has surged. New targeted therapies require redesigned clinical trials to effectively evaluate their efficacy in specific patient groups.
Area of Science:
- Oncology
- Clinical Pharmacology
- Drug Development
Background:
- Significant increase in the number of anticancer drugs undergoing clinical trials.
- Emergence of novel targeted anticancer agents with specific mechanisms of action.
- Challenges posed by the heterogeneity of patient responses to new therapies.
Purpose of the Study:
- To address the need for redesigned clinical trials for targeted anticancer drugs.
- To explore innovative trial designs accommodating novel therapeutic strategies.
- To optimize the evaluation of anticancer agents in specific patient subpopulations.
Main Methods:
- Review of current clinical trial methodologies.
- Analysis of the characteristics of novel targeted anticancer drugs.
- Exploration of adaptive and biomarker-driven trial designs.
Main Results:
- Current trial designs may not be optimal for targeted therapies.
- Small patient subpopulations necessitate focused trial recruitment.
- Unpredictable drug interactions require careful trial planning.
Conclusions:
- Clinical trials must evolve to incorporate targeted drug development.
- Biomarker-based stratification and adaptive designs are crucial.
- Redesigned trials are essential for efficient evaluation of novel anticancer agents.
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