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Patient-Reported Outcomes in Early-Phase Oncology Clinical Trials: A Stepping Stone to a Patient-Centered Drug
Frederic Fiteni1,2, Adeline Meilhoc3, Olivier Blin4
1Medical Oncology Department, University Hospital, 30900, Nîmes, France. frederic.fiteni@chu-nimes.fr.
The Patient
|November 11, 2025
Summary
Integrating patient-reported outcomes (PROs) into phase I clinical trials improves accuracy in defining dose-limiting toxicities (DLTs) and setting the recommended phase II dose (RP2D). This enhances patient-centered drug development and trial design.
Area of Science:
- Clinical Pharmacology
- Oncology Drug Development
- Patient-Reported Outcomes Research
Background:
- Phase I clinical trials determine the recommended phase II dose (RP2D) based on maximum tolerated dose (MTD) and dose-limiting toxicities (DLTs).
- Clinician assessments of toxicities using NCI-CTCAE may underestimate patient symptoms.
- Patient-reported outcomes (PROs) offer direct patient input on adverse events, complementing clinician evaluations.
Purpose of the Study:
- To explore the integration of PROs into phase I clinical trials for more accurate DLT definition and RP2D determination.
- To highlight the potential of PROs in optimizing later-stage trial design and evaluating drug benefit, particularly in rare oncologic subsets.
- To address the need for methodological guidelines for early PRO integration in drug development.
Main Methods:
- Review of current practices in phase I trial dose selection.
- Analysis of the role of clinician-assessed toxicities versus patient-reported outcomes (PROs).
- Discussion of the potential impact of PRO integration on trial design and decision-making.
Main Results:
- PROs, such as the NCI PRO-CTCAE, can provide crucial patient insights into adverse events, potentially leading to more accurate DLT identification.
- Integrating PROs can inform better RP2D decisions and optimize sample size calculations for subsequent trials.
- PRO data can facilitate health-related quality of life (HRQoL) comparisons, aiding in the assessment of drug efficacy, especially in specific patient populations.
Conclusions:
- Integrating PROs into phase I trials enhances the accuracy of DLT assessment and RP2D selection.
- PROs offer significant potential to improve patient-centeredness, optimize trial design, and strengthen the overall drug development process.
- Standardized methodologies are needed to facilitate the broader adoption of PROs in early-phase clinical research.
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