Recommendations For The Collection Of Patient-Reported Outcomes (PRO) Data In The Post-Progression And Post-Treatment Setting Following Oncology Clinical Trials
Author(s)
Oladapo O1, D'Alessio D2, Vallow S2
1Novartis Pharmaceuticals Corporation, Jacksonville, FL, USA, 2Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA
OBJECTIVES: Patient-reported outcomes (PROs) in the post-progression and post-treatment setting are important for continued understanding of the patient experience following oncology clinical trials. This research aims to summarize published literature and recommendations for the collection of PRO data post-progression and the use of this data in HTA decision-making. METHODS: A targeted literature review was performed to identify publications from within the last 5 years. A search was performed on the Food and Drug Administration (FDA), European Medicines Agency (EMA) and various HTA agency websites for guidelines or recommendations. RESULTS: The literature review yielded 14 publications that were included in the analysis, with recommendations for protocol considerations, leveraging technology to facilitate data collection, prespecifying statistical analysis plans, and detailing causes of missing data. EMA advises that study duration should robustly support planned analysis and that PROs should be collected after progression to compare trends over time, where feasible. FDA advises that patients who fail to report due to dropout or treatment discontinuation should continue to provide PRO data, and the protocol should state how this data will be collected if withdrawal is unavoidable. The Australian Health Ministers’ Advisory Council highlights the need for further understanding of the transition between and after active treatment of cancer. The 8 HTA agencies reviewed did not provide specific recommendations for collection of this data. CONCLUSIONS: Continued collection of PRO data after disease progression and study treatment gives valuable insight into patients’ experiences, even as they experience changing health states. It also provides a comprehensive interpretation of disease and treatment-related impacts and is utilized by regulators and HTAs to differentiate treatments and contextualize study results, as well as patients and physicians to inform treatment selection. Current recommendations provide valuable insight, however, more guidance is needed to thoroughly inform PRO collection in this setting.
Conference/Value in Health Info
2021-05, ISPOR 2021, Montreal, Canada
Value in Health, Volume 24, Issue 5, S1 (May 2021)
Code
PCN223
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Clinical Outcomes Assessment, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Oncology