Collection of Post-treatment PRO Data in Oncology Clinical Trials

Abstract

As patient-reported outcome (PRO) measures are being included more frequently in oncology clinical trials, regulatory and health technology assessment agencies have begun to request long-term, post-treatment PRO data to supplement traditional survival/progression endpoints. These data may be collected as part of cohort extension or registry studies to describe long-term outcomes of study participants after concluding their cancer treatment. While post-treatment PRO data may be expected to satisfy regulatory and payer expectations, significant practical barriers exist for the efficient incorporation of these data into oncology clinical trials, such as subject attrition, protocol deviations, and treatment crossover. The incorporation of post-treatment PRO assessments is a resource-intensive task requiring clear objectives for how the data will be analyzed and interpreted by both sponsors and regulators. Incorporating PRO data collection via electronic modalities (e.g., smartphone, web) may be a less expensive and more feasible option for incorporating long-term follow-up, reducing the frequency of manual study staff follow-up and expensive clinic visits. It is essential to include well-defined estimands for the statistical analysis, as well as to document limitations associated with the long-term follow-up data-collection approach. Analytical techniques will likely rely on descriptive and model-based statistics, and conclusions about treatment differences will likely be limited to preliminary findings of effectiveness (instead of efficacy). Finally, communications with health authorities and regulatory agencies regarding the LTFU study design and analysis should occur as early as possible to ensure that the PRO data to be collected offer an opportunity to properly evaluate the research question(s) of interest.

Keywords: Cancer; Long-term follow-up; Oncology clinical trials; Patient-reported outcome; Post-treatment data.