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Published on: January 12, 2018
Patient and public involvement and engagement in the development of innovative patient-centric early phase
Emily Alger1, Mary Van Zyl2, Olalekan Lee Aiyegbusi3,4
1Clinical Trial and Statistics Unit, Institute of Cancer Research, London, UK.
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.
Insights
Patient-reported outcomes (PROs) can improve tolerability assessments in early-phase oncology trials. Patient involvement is key to integrating PROs effectively into advanced dose-finding trial designs.
Area of Science:
- Oncology clinical trial design
- Patient-reported outcomes research
- Statistical methodology in clinical trials
Background:
- FDA's Project Optimus highlights need for PROs in early-phase oncology trials.
- Clinician-assessed adverse events alone may not capture true treatment tolerability.
- Patient and Public Involvement and Engagement (PPIE) is crucial for trial enrichment.
Purpose of the Study:
- Explore optimal integration of PROs in advanced statistical dose-finding trial designs.
- Investigate patient perspectives on using PROs for tolerability assessment.
- Develop a template for patient involvement in trial design.
Main Methods:
- Virtual PPIE session with nine participants.
- Cross-disciplinary team including statisticians, clinicians, and trial methodologists.
- In-depth perspectives from two patient advocates.
Main Results:
- General support for PROs in dose-finding trials.
- Concerns that PROs might lead to dose reduction, impacting efficacy.
- Patients suggested PROs for assessing tolerability, not just toxicity.
Conclusions:
- Patient voice is essential for patient-centric dose-finding trial design.
- Effective incorporation of patient input can enhance advanced trial designs.
- This work provides a model for integrating patient perspectives in trial development.
