Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes

Wages, Nolan A.; Lin, Ruitao

doi:10.1080/19466315.2023.2288013

Statistics in Biopharmaceutical Research

2024

DOI: 10.1080/19466315.2023.2288013

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Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes

Nolan A. Wages,

Ruitao Lin

Abstract: Supplemental Figure S1: A sample of 69 dose-toxicity curves generated from the Clertant and O'Quigley family of curves for a target C-DLT rate of 0.25 and a target P-DLT rate of 0.35. Supplemental Figure S2: Comparison of the percentage of correct MTD selection (PCS) for the PRO-CRM, PRO-ISO, and the PRO-Optimal methods over the 69 dose-toxicity curves in Figure S1. The mean PCS for the PRO-CRM is 52.7%, the mean PCS for the PRO-ISO is 56.8%, and the mean PCS for the PRO-Optimal is 62.6%.

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Cited by 4 publications

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SOHO State of the Art Updates and Next Questions | Measuring Patient-Reported Outcomes (PROs) and Treatment Tolerability in Patients With Hematologic Malignancies

Major,

Dueck,

Thanarajasingam

2024

Clinical Lymphoma Myeloma and Leukemia

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SOHO State of the Art Updates and Next Questions | Measuring Patient-Reported Outcomes (PROs) and Treatment Tolerability in Patients With Hematologic Malignancies

Major,

Dueck,

Thanarajasingam

2024

Clinical Lymphoma Myeloma and Leukemia

View full text Add to dashboard Cite

Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials – insights from an expert virtual roundtable

Yap,

Lee Aiyegbusi,

Alger

et al. 2024

eClinicalMedicine

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Patient and public involvement and engagement in the development of innovative patient-centric early phase dose-finding trial designs

Alger,

Van Zyl,

Aiyegbusi

et al. 2024

Res Involv Engagem

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Background In light of the FDA’s Project Optimus initiative, there is fresh interest in leveraging Patient-reported Outcome (PRO) data to enhance the assessment of tolerability for investigational therapies within early phase dose-finding oncology trials. Typically, dose escalation in most trial designs is solely reliant on clinician assessed adverse events. Research has shown a disparity between patients and clinicians when assessing whether an investigational therapy is tolerable, leading to the recommendation of potentially intolerable doses for further investigation in subsequent trials. It is also increasingly recognized that patient and public involvement and engagement (PPIE) plays a pivotal role in enriching trial design and conduct. However, to our knowledge, no PPIE has explored the optimal integration of PROs in the development of advanced statistical trial designs within early phase dose-finding oncology trials. Methods A virtual PPIE session was held with nine participants on 18th October 2023 to discuss the incorporation of PROs within a dose-finding trial design. This cross disciplinary session was developed and led by a team of statisticians, clinical specialists, qualitative experts, and trial methodologists. Following the session, in-depth perspectives were provided by two patient advocates who actively engaged in the PPIE session. We discuss the importance of PPIE in shaping advanced dose-finding trial designs, share insights from patients on integrating PROs to inform treatment tolerability, and present a template for meaningful patient involvement in trial design development. Results Participants generally supported the introduction of PROs within dose-finding trials but showed some apprehensiveness as to how PROs may reduce the size of the recommended dose (and potentially efficacious effect). Some participants shared that they may be reluctant to record the real severity of their symptoms via PROs if it would mean that they would have to discontinue treatment. They discussed that PROs could be used to assess tolerability rather than toxicity of a dose. Conclusions Amplifying patient voice in the development of patient-centric dose-finding trial designs is now essential. This paper offers an exemplary illustration of how trialists and methodologists can effectively incorporate patient voice in the future development of advanced dose-finding trial designs.

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Isotonic Phase I Cancer Clinical Trial Design Utilizing Patient-Reported Outcomes

Cited by 4 publications

References 45 publications

SOHO State of the Art Updates and Next Questions | Measuring Patient-Reported Outcomes (PROs) and Treatment Tolerability in Patients With Hematologic Malignancies

SOHO State of the Art Updates and Next Questions | Measuring Patient-Reported Outcomes (PROs) and Treatment Tolerability in Patients With Hematologic Malignancies

Advancing patient-centric care: integrating patient reported outcomes for tolerability assessment in early phase clinical trials – insights from an expert virtual roundtable

Patient and public involvement and engagement in the development of innovative patient-centric early phase dose-finding trial designs

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