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The Next Horizon of Drug Development: External Control Arms and Innovative Tools to Enrich Clinical Trial Data

Kelly H Zou1, Chelsea Vigna2, Aniketh Talwai2

  • 1Viatris, Canonsburg, PA, USA.

Therapeutic Innovation & Regulatory Science
|March 26, 2024
PubMed

Insights

External control arms (ECAs) and digital health tools like virtualization and decentralized clinical trials (DCTs) streamline complex clinical trials. Tokenization links trial data with real-world data (RWD) for richer insights and reduced costs.

Area of Science:

  • Clinical trial design and operations
  • Digital health technologies
  • Real-world data utilization

Background:

  • Clinical trials (CTs) face escalating costs and complexity, especially for novel therapies in oncology and rare diseases targeting smaller patient populations.
  • Traditional site-centric trial designs present significant patient burden and can limit diversity.

Purpose of the Study:

  • To describe external control arms (ECAs) and other innovative tools like virtualization, decentralized clinical trials (DCTs), and tokenization for enhancing clinical trial design and data analysis.
  • To highlight how these technologies can create more patient-centric, diverse, and cost-effective clinical trials.

Main Methods:

  • Construction of ECAs involves identifying, cleaning, and standardizing external data sources, followed by subject matching with the clinical trial cohort.
  • Leveraging digital health technologies, virtualization, and DCTs to shift from site-centric to virtual, decentralized, and patient-centric trial models.
  • Utilizing tokenization to link clinical trial data with real-world data (RWD) for longitudinal outcome measurement.

Main Results:

  • ECAs can be augmented with subject-level meta-analysis and simulated data for robust analysis.
  • Virtualization and DCTs reduce patient burden, encourage diversity, and enable real-world data integration.
  • Tokenization facilitates comprehensive, longitudinal outcome measures by connecting CT data with RWD.

Conclusions:

  • External control arms, virtualization, DCTs, and tokenization offer powerful strategies to enrich clinical trial data.
  • These tools support informed decision-making, reduce operational costs, and enhance the insights derived from clinical trial data.
  • Adoption of these technologies can lead to more efficient, patient-friendly, and insightful clinical research, particularly in challenging therapeutic areas.
Keywords:
Clinical trialDecentralized clinical trialsExternal control armPatient diversityReal-world dataReal-world evidenceRecord linkage

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