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New clinical trial designs in the era of precision medicine

Elena Garralda1,2, Rodrigo Dienstmann1,3, Alejandro Piris-Giménez1

  • 1Medical Oncology Department, Vall d'Hebron University Hospital and Institute of Oncology (VHIO), Universitat Autònoma de Barcelona, Spain.

Molecular Oncology
|January 31, 2019
PubMed

Insights

Precision cancer medicine (PCM) advances target common genetic deregulations across tumor types. Novel adaptive clinical trial designs accelerate biomarker-drug development for more efficient cancer therapy.

Area of Science:

  • Oncology
  • Translational Medicine
  • Clinical Trial Design

Background:

  • Precision Cancer Medicine (PCM) leverages genetic insights to match targeted therapies with specific molecular deregulations.
  • The landscape of cancer therapeutics is rapidly expanding with novel agents, including immune-oncology drugs.
  • Regulatory agencies are increasingly approving new drugs based on early-phase trial data.

Purpose of the Study:

  • To review the evolution of early-stage clinical trial designs in the context of PCM.
  • To discuss the challenges and opportunities presented by novel trial designs for drug development.
  • To explore how these advancements can facilitate the realization of PCM for patients.

Main Methods:

  • Review of current trends in early-stage clinical trial design, including adaptive, basket, and umbrella studies.
  • Analysis of the role of biomarker-drug co-development in optimizing trial efficiency.
  • Examination of collaborative frameworks among academic institutions, clinicians, pharmaceutical companies, and regulatory agencies.

Main Results:

  • Significant evolution in early-stage clinical trial designs, characterized by rapid dose-escalation followed by large expansion cohorts.
  • Emergence of adaptive trial designs (basket and umbrella) to optimize biomarker-drug development.
  • Establishment of enhanced collaboration frameworks among all stakeholders in drug development.

Conclusions:

  • New clinical trial designs offer opportunities for a more efficient drug development process.
  • Optimized collaboration and adaptive trial designs are crucial for advancing Precision Cancer Medicine.
  • These advancements hold the potential to make PCM a clinical reality for more cancer patients.
Keywords:
Basket of BasketsCancer Core Europeadaptive designsbasket protocolsumbrella protocols

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