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Biomarkers in early cancer drug development: limited utility
1Division of Hematology and Oncology, Weill Medical College of Cornell University, New York, New York, USA. robgmd@hotmail.com
Abstract:
Sponsors bringing a novel antineoplastic agent into human testing are faced with a slew of decisions regarding dosing, schedule, and regulatory path (e.g., indications, patient population, trial design, and end points), often with a meager scientific foundation on which to base these critical decisions. Thus, the cost (in both time and money) to bring innovative small-molecule drugs or biologics to cancer patients has been enormous, with only a handful of approvals per year despite the more than 700 drugs in clinical development and the more than 1,300 in preclinical testing.(1) A biomarker, defined as "a characteristic that is objectively measured and evaluated as an indicator of normal biologic processes, pathogenic processes, or pharmacologic responses to a therapeutic intervention,"(2) has intuitive appeal as a means of shortcutting drug development processes largely through optimizing the drug's dose and schedule (pharmacodynamic biomarkers), selecting patients most likely to benefit from the therapeutic intervention (predictive biomarkers), and acting as a substitute for a true clinical outcome (outcome biomarkers or surrogate end points).(3) However, the enthusiasm for widespread adoption of biomarker studies in early drug development is unjustified because of statistical and cost considerations, as well as a lack of historic evidence for their usefulness.
Insights
Biomarkers offer potential to streamline cancer drug development but their early use is cautioned against. Statistical, cost, and evidence-based limitations hinder widespread adoption despite intuitive appeal.
Area of Science:
- Oncology
- Pharmacology
- Biostatistics
Background:
- Developing novel antineoplastic agents involves complex decisions on dosing, schedule, and regulatory strategy.
- High costs and limited success rates plague innovative cancer drug development, with few approvals annually.
- Biomarkers are proposed to optimize drug development by guiding dose, patient selection, and outcomes.
Purpose of the Study:
- To evaluate the justification for widespread adoption of biomarker studies in early-stage drug development.
- To assess the utility of biomarkers in streamlining the development of novel antineoplastic agents.
- To examine the statistical, economic, and historical evidence supporting biomarker use.
Main Methods:
- Review of existing literature and data on biomarker utility in early drug development.
- Analysis of statistical and cost considerations associated with biomarker-guided trials.
- Evaluation of historical evidence for biomarker effectiveness in accelerating drug approvals.
Main Results:
- The enthusiasm for early biomarker adoption is not fully supported by current evidence.
- Statistical and financial challenges limit the practical application of biomarker studies.
- Lack of robust historical data questions the proven usefulness of biomarkers in accelerating development.
Conclusions:
- Widespread adoption of biomarker studies in early oncology drug development is currently unjustified.
- Further research is needed to establish the statistical validity and cost-effectiveness of biomarkers.
- Careful consideration of limitations is essential before integrating biomarkers into novel therapeutic development pathways.
