New designs in early clinical drug development.
A Mansinho1, V Boni2, M Miguel2
1Medical Oncology Division, Centro Hospitalar Universitário Lisboa Norte - Hospital de Santa Maria, Lisbon, Portugal; START Madrid, Centro Integral Oncológico Clara Campal, Madrid, Spain.
Summary
New drug development faces challenges due to cancer
Area of Science:
- Oncology
- Clinical Pharmacology
- Drug Development
Background:
- Massive data availability fuels drug development, particularly for cancer.
- Cancer's heterogeneity (mutations, varied responses) challenges traditional clinical trials.
- Existing early-phase trial designs are insufficient for novel drug mechanisms and toxicity profiles.
Purpose of the Study:
- To review innovative early-phase clinical trial designs.
- To address the limitations of classical trial designs in oncology.
- To adapt trial methodologies for diverse drug classes and targets.
Main Methods:
- Review of current and emerging early-phase clinical trial designs.
- Analysis of how different drug classes necessitate tailored trial strategies.
- Discussion of optimizing decision-making for advancing drug candidates.
Main Results:
- Classical 'one-size-fits-all' trials are inadequate for heterogeneous diseases like cancer.
- Novel trial designs are crucial for efficiency, cost reduction, and informed go/no-go decisions.
- Tailored designs are needed post-maximum tolerated dose/optimum biological dose identification.
Conclusions:
- Adapting early-phase trial designs is essential for modern drug development in oncology.
- More informative and transformative designs are required to meet the complexity of new therapeutics.
- Optimized early-phase studies enhance the transition from bench to bedside.
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