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Updated: Sep 8, 2025

Evidence-based Knowledge Synthesis and Hypothesis Validation: Navigating Biomedical Knowledge Bases via Explainable AI and Agentic Systems
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Evidence-based Knowledge Synthesis and Hypothesis Validation: Navigating Biomedical Knowledge Bases via Explainable AI and Agentic Systems

Published on: June 13, 2025

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Hope is Not a Strategy: Using Robust Real-World Evidence to Make Better Clinical Development Decisions

Nicolle M Gatto1,2, Ulka B Campbell3,4

  • 1Aetion, Inc., 5 Pennsylvania Plaza, New York, NY, 10001, USA. nicolle.gatto@aetion.com.

Therapeutic Innovation & Regulatory Science
|July 18, 2025
PubMed

Insights

Systematic generation of real-world evidence (RWE) is crucial for improving clinical trial design and decision-making. This approach enhances understanding of patient populations, leading to more effective drug development.

Area of Science:

  • Clinical Research
  • Pharmacoeconomics
  • Biostatistics

Background:

  • Clinical development faces delays and failures due to reliance on suboptimal trial designs based on expert opinion or historical data.
  • Representative, real-world point-of-care data is underutilized for understanding patient populations and indication characteristics.
  • Systematic generation of real-world evidence (RWE) is essential for informed clinical development decision-making.

Purpose of the Study:

  • To make integrated real-world evidence (RWE) generation more compelling and practicable.
  • To provide a blueprint for designing real-world studies in a phased approach aligned with clinical development investment.
  • To facilitate wider adoption of systematic RWE generation early in the development process.

Main Methods:

  • Addressing concerns raised by biopharmaceutical leaders regarding RWE generation.
  • Developing a phased approach for designing real-world studies.
  • Integrating RWE generation into the clinical development lifecycle.

Main Results:

  • Current RWE generation practices are not systematic, despite recognized value.
  • Proposed strategies aim to overcome barriers to integrated RWE generation.
  • A phased approach can align RWE studies with clinical development investment.

Conclusions:

  • Systematic RWE generation, starting early in development, is essential for evidence-based decision-making.
  • Robust RWE ensures timely data availability for sponsors, regulators, and payers.
  • Improved RWE practices can enhance clinical development efficiency and public health outcomes.
Keywords:
Clinical developmentReal-world dataReal-world evidence

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