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Precision Oncology Clinical Trials: A Systematic Review of Phase II Clinical Trials with Biomarker-Driven, Adaptive
Hyerim Ha1, Hee Yeon Lee2, Jee Hyun Kim3
1Department of Internal Medicine, Inha University Hospital, Inha University College of Medicine, Incheon, Korea.
Abstract:
Novel clinical trial designs are conducted in the precision medicine era. This study aimed to evaluate biomarker-driven, adaptive phase II trials in precision oncology, focusing on infrastructure, efficacy, and safety. We systematically reviewed and analyzed the target studies. EMBASE and PubMed searches from 2015 to 2023 generated 29 eligible trials. Data extraction included infrastructure, biomarker screening methodologies, efficacy, and safety profiles. Government agencies, cancer hospitals, and academic societies with accumulated experiences led investigator-initiated precision oncology clinical trials (IIPOCTs), which later guided sponsor-initiated precision oncology clinical trials (SIPOCTs). Most SIPOCTs were international studies with basket design. IIPOCTs primarily used the central laboratory for biomarker screening, but SIPOCTs used both central and local laboratories. Most of the studies adapted next-generation sequencing and/or immunohistochemistry for biomarker screening. Fifteen studies included an independent central review committee for outcome investigation. Efficacy assessments predominantly featured objective response rate as the primary endpoint, with varying results. Nine eligible studies contributed to the United States Food and Drug Administration's marketing authorization. Safety monitoring was rigorous, but reporting formats lacked uniformity. Health-related quality of life and patient-reported outcomes were described in some protocols but rarely reported. Our results reveal that precision oncology trials with adaptive design rapidly and efficiently evaluate anticancer drugs' efficacy and safety, particularly in specified biomarker-driven cohorts. The evolution from IIPOCT to SIPOCT has facilitated fast regulatory approval, providing valuable insights into the precision oncology landscape.
Insights
Adaptive phase II trials in precision oncology accelerate drug evaluation. Investigator-initiated trials evolved into sponsor-initiated trials, facilitating faster regulatory approvals and improving cancer treatment insights.
Area of Science:
- Oncology
- Clinical Trial Design
- Precision Medicine
Background:
- The precision medicine era necessitates novel clinical trial designs.
- Adaptive phase II trials are crucial for evaluating targeted therapies in oncology.
Purpose of the Study:
- To evaluate biomarker-driven, adaptive phase II trials in precision oncology.
- Focus on infrastructure, efficacy, and safety of these trial designs.
Main Methods:
- Systematic review of EMBASE and PubMed databases (2015-2023).
- Analysis of 29 eligible trials, extracting data on infrastructure, biomarker screening, efficacy, and safety.
- Investigated the evolution from investigator-initiated precision oncology clinical trials (IIPOCTs) to sponsor-initiated precision oncology clinical trials (SIPOCTs).
Main Results:
- Biomarker screening utilized next-generation sequencing and/or immunohistochemistry, with IIPOCTs favoring central labs and SIPOCTs using both central and local labs.
- Objective response rate was the primary efficacy endpoint in most studies.
- Nine trials contributed to US Food and Drug Administration marketing authorization; safety monitoring was rigorous but reporting lacked uniformity.
Conclusions:
- Adaptive precision oncology trials efficiently assess anticancer drug efficacy and safety in biomarker-defined cohorts.
- The progression from IIPOCTs to SIPOCTs streamlines regulatory approval and advances precision oncology.
- Further standardization in safety reporting and inclusion of patient-reported outcomes are warranted.
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