Conference Agenda

The absence of clear PRO objectives has lead to uncertainty in cancer clinical trials, causing confusion in their analysis and reporting. Current practices often show inconsistent terminology, an inflation of analyses, and potentially conflicting results, hindering decision-making and cross-validation of PRO results across studies. Using estimands that reflect relevant and concise PRO objectives can improve the design, analysis, interpretation, and communication of PRO results. The terminology and framework of the intial SISAQOL recommendations will be presented, including a taxonomy of PRO objectives. We will also demonstrate how the estimand framework was used to bridge the gap from research question to actual analysis.

Unobserved data is a persistent issue in PRO analyses due to voluntary patient participation, characterized by intercurrent events and missing data. Intercurrent events affect endpoint interpretation and must be aligned with the intended PRO objective, especially in oncology where events like death are not uncommon. Strategies for handling missing data should consider potential informative relationships and assess robustness. Reporting data availability at each assessment timepoint using standardized completion rates and available data rates is recommended to address selection bias in an uniform way across trials.

Single Arm Trials (SATs) face additional challenges due to the lack of a randomized concurrent control. Specifying PRO objectives using the ICHE9 (R1) estimand framework is crucial as current practices are hampered by unclear objectives. Strategies for handling terminal events like death vary, with the while-alive strategy often preferred for quality of life. Causal inference methods offer new opportunities for generating evidence, but untestable causal assumptions must be plausible and supported by sensitivity analyses. This session will explore these strategies and their application in SATs.

This session will highlight key SISAQOL-IMI recommendations for communicating PRO results from oncology trials, focusing on visualization. Key principles for transparency include aligning figures with pre-specified objectives. Best practices for including sample size details, missing data, and intercurrent events will be presented, along with a template for integrating intercurrent event data into PRO figures. Strategies for reporting statistical significance and distinguishing confirmatory analyses from exploratory findings will be discussed, aiming to improve the accessibility of PRO results for both expert and non-specialist readers.

The heterogeneous terminology around thresholds for interpreting PRO data poses challenges in selecting such thresholds. SISAQOL-IMI has established a harmonized terminology differentiating types of PRO score interpretation thresholds, focusing on patient- and group-level data. These types of threshold correspond to specific statistical analysis methods to support their correct implementation and interpretation. This session will provide recommendations on reporting the use of PRO score interpretation thresholds in cancer clinical trial data analysis and interpretation.

This interactive panel discussion will bring together the speakers to delve into the practical implementation of SISAQOL-IMI recommendations. The panel will include all session presenters and the discussion will focus on the challenges in harmonizing PRO objectives. Speakers will be asked to identify areas where these recommendations are expected to have practice-changing impact. Attendees will have the opportunity to engage with the panelists through a Q&A format, fostering a dynamic exchange of ideas and experiences aimed at improving the utility of PRO data in cancer clinical trials.