Presentation (CTI)

OBJECTIVES: Regulatory and Health Technology Assessment (HTA) bodies increasingly recognize patient preference information (PPI) as valuable patient experience data (PED), a central tenet of patient-focused drug development (PFDD). PPI complements other PED types by capturing patients' treatment preferences and willingness to trade different attributes of an intervention. PPI can support identification of unmet need, patient-focused endpoint development, and benefit-risk assessment. However, PPI studies are typically limited to patients reflecting on hypothetical treatments and prior experiences. Additionally, in-trial/exit interviews are increasingly recognized by regulators as a means to generate valuable PED reflecting actual experiences of treatments under evaluation. Such insights can provide evidence to contextualize trial data, substantiate patient-focused measurement strategies, and inform endpoint responder definitions. Eliciting PPI during in-trial interviews provide opportunities to understand and contextualize patient preferences before-and-after treatment exposure.
METHODS: This review identifies and summarizes existing literature/commentary regarding collecting PPI in clinical trials. Methodological considerations for successful study design, analysis and integration is outlined. Scenarios where in-trial PPI can generate high-impact evidence for sponsors and stakeholders are proposed.
RESULTS: While in-trial PPI can enhance data collection efficiencies, reduce patient burden, and increase generalizability to the target population, there is limited guidance/published examples of integrating PPI within in-trial interviews. To maximize value and impact, it is critical that PPI activities are robust and tailored to trial contexts. Selecting suitable PPI elicitation methods is critical, dependent on population characteristics, feasibility, and resource constraints. Appropriate framing of preference tasks/questioning in alignment with the research question will ensure relevance for decision-makers. Determining optimal timing and frequency for PPI activities must balance data quality with trial integrity and minimizing measurement bias.
CONCLUSIONS: Collecting PPI within clinical trials offers significant potential to enhance patient-centricity and support regulatory and HTA decision-making. Addressing methodological and practical challenges through further case studies and guidance is essential to support generating fit-for-purpose evidence.