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Innovative trial design in precision oncology

Apostolia M Tsimberidou1, Peter Müller2, Yuan Ji3

  • 1Department of Investigational Cancer Therapeutics, Phancise I Clinical Trials Program, The University of Texas MD Anderson Cancer Center, 1400 Holcombe Boulevard, Unit 455, Houston, TX 77030, United States.

Seminars in Cancer Biology
|October 6, 2020
PubMed

Insights

Innovative clinical trials, including master protocols, accelerate precision oncology by matching molecular profiles to targeted therapies. Despite challenges with small patient subgroups, these trials rapidly translate cancer drug discoveries into improved patient outcomes.

Area of Science:

  • Oncology
  • Genomics
  • Clinical Trial Design

Background:

  • Genomic profiling enables targeted therapies for cancer by identifying specific biomarkers and molecular pathways.
  • Precision oncology trials are evolving to incorporate novel agents based on patient molecular alterations and diverse tumor types.

Purpose of the Study:

  • To explore the role of innovative clinical trial designs, particularly master protocols, in advancing precision oncology.
  • To highlight how master protocols facilitate drug development by matching patient molecular profiles with experimental treatments.

Main Methods:

  • Utilizing master protocols for simultaneous sub-studies matching tumor molecular profiles with experimental arms.
  • Incorporating real-world data, patient-reported outcomes, and N-of-1 trials to enhance evidence for personalized treatment.

Main Results:

  • Master protocols accelerate the development of novel cancer therapies by enabling efficient drug testing in targeted patient populations.
  • Innovative trial designs, despite potential limitations of small subpopulations, are crucial for translating genomic discoveries into clinical benefits.

Conclusions:

  • Precision oncology trials with master protocols are key to rapidly advancing cancer treatment by personalizing therapies based on genomic data.
  • Continued innovation in clinical trial design is essential for overcoming challenges and maximizing the impact of precision oncology for cancer patients.
Keywords:
Basket trialBiomarkersPersonalized medicineReal world dataUmbrella trial

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