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Published on: September 20, 2019
Phase 0 trials: an industry perspective
Helen Eliopoulos1, Vincent Giranda, Robert Carr
1Abbott Laboratories, Abbott Park, Illinois, USA.
Abstract:
Worldwide, cancer is a leading cause of morbidity and mortality. An increased understanding of the disease and its process has resulted in a multitude of new targeted therapies. The costs as well as time from drug discovery to market, however, remain staggeringly high and protracted, with the majority of compounds never reaching phase III. The concept of an exploratory or phase 0 trial was introduced as a mechanism to enhance and accelerate the overall process of new oncologic drug development. Performance of a phase 0 study allows researchers to better understand the pharmacokinetic and pharmacodynamic properties of compounds in human subjects before initiation of phase I trials. Data gleaned from a phase 0 trial are beneficial not only in prioritizing promising compounds but also in allowing the modification of phase I study design before initiation. To date, few researchers have taken advantage of the potential benefits of phase 0 trials. This review focuses on the purpose as well as the potential merits of phase 0 trials from the perspective of a pharmaceutical company. The review summarizes the experience of a team of researchers with ABT-888, a novel poly (ADP-ribose) polymerase agent that inhibits an enzyme critical for repairing damage to DNA, which is one of the first compounds to be investigated using the phase 0 clinical trial design.
Insights
Phase 0 trials accelerate oncology drug development by providing early human data on pharmacokinetics and pharmacodynamics. This approach helps prioritize promising compounds and refine subsequent trial designs.
Area of Science:
- Oncology
- Clinical Pharmacology
- Drug Development
Background:
- Cancer remains a leading cause of death globally, driving the need for efficient drug development.
- Despite advances, new targeted cancer therapies face high costs and lengthy development timelines.
- Most drug candidates fail before Phase III trials, highlighting inefficiencies in the drug development pipeline.
Purpose of the Study:
- To review the utility and benefits of exploratory or Phase 0 clinical trials in oncology drug development.
- To examine how Phase 0 trials can enhance the efficiency of bringing new cancer therapies to market.
- To present a case study of ABT-888, a poly (ADP-ribose) polymerase inhibitor, developed using a Phase 0 trial design.
Main Methods:
- Review of the concept and rationale behind Phase 0 trials.
- Analysis of pharmacokinetic and pharmacodynamic data obtained from Phase 0 studies.
- Case study illustrating the application of Phase 0 trials in early drug development.
Main Results:
- Phase 0 trials enable early assessment of drug behavior in humans, informing go/no-go decisions.
- Data from Phase 0 studies can optimize the design of subsequent Phase I trials.
- The use of Phase 0 trials can potentially reduce attrition rates and development costs for oncology drugs.
Conclusions:
- Phase 0 trials offer significant advantages for pharmaceutical companies by providing crucial early data.
- These trials can de-risk drug development and accelerate the identification of effective cancer therapies.
- Wider adoption of Phase 0 trials is recommended to improve the efficiency of oncology drug development.
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