The SISAQOL-IMI Recommendations on Standardizing the Use of Thresholds for Interpretation of Patient-Reported Outcome Results in Cancer Clinical Trials
Author(s)
Johannes Giesinger1, Nathan Clarke, PhD2, Kim Cocks, PhD, CStat2, Jammbe Musoro, PhD3, Morten Aa Petersen, MSc4, Micha Pilz, MPH5, khadija Rerhou Rantell, PhD6, Antoine Regnault, PhD7, Joseph C. Cappelleri, MPH, MS, PhD8.
1Asst.Prof., Innsbruck Medical University, Innsbruck, Austria, 2Adelphi Values Ltd, Bollington, United Kingdom, 3European Organisation for Research and Treatment of Cancer, Brussels, Belgium, 4Bispebjerg and Frederiksberg Hospital, Copenhagen, Denmark, 5Medical University of Innsbruck, Innsbruck, Austria, 6Medicines and Healthcare products Regulatory Agency (MHRA), London, United Kingdom, 7Modus Outcomes, Lyon, France, 8Pfizer Inc, New York, NY, USA.
1Asst.Prof., Innsbruck Medical University, Innsbruck, Austria, 2Adelphi Values Ltd, Bollington, United Kingdom, 3European Organisation for Research and Treatment of Cancer, Brussels, Belgium, 4Bispebjerg and Frederiksberg Hospital, Copenhagen, Denmark, 5Medical University of Innsbruck, Innsbruck, Austria, 6Medicines and Healthcare products Regulatory Agency (MHRA), London, United Kingdom, 7Modus Outcomes, Lyon, France, 8Pfizer Inc, New York, NY, USA.
OBJECTIVES: The multi-stakeholder SISAQOL-IMI consortium has developed international guidelines for standardisation of analysis of patient-reported outcome (PRO) data in cancer trials, including guidance on interpreting PRO trial results from different types of statistical analyses and the appropriate use of PRO score interpretation thresholds.
METHODS: The SISAQOL-IMI recommendations emerged from a comprehensive, multi-round consensus process involving 41 organisations over a four-year span and build on the estimand framework. This diverse group included clinicians, patients, academics, industry representatives, regulatory authorities, and HTA bodies. The work of the consortium is readily accessible through various resources, including an online platform (www.sisaqol-imi.org). The harmonised terminology and recommendations for issues related to PRO score interpretation thresholds were developed through five in-person consensus rounds, a series of virtual meetings, a patient workshop, and stakeholder surveys.
RESULTS: The work on the use of PRO score interpretation thresholds resulted in 27 recommendations specific to the use of such thresholds in cancer clinical trials. A key achievement is the establishment of a harmonised terminology for PRO score interpretation thresholds, which provides clear definitions for various threshold types, including differentiating between patient- and group-level thresholds. Crucially, these recommendations link specific threshold types to appropriate statistical analysis methods, thereby instilling correct implementation and valid interpretation of PRO data. Furthermore, key criteria for threshold selection are detailed and advocate for, e.g., anchor-based methods—especially those leveraging patient-centred anchors—to infuse meaning into the interpretation of PRO data. Detailed terminology, recommendations, and accompanying examples will be presented at the conference.
CONCLUSIONS: The SISAQOL-IMI recommendations and terminology for PRO score interpretation thresholds will significantly enhance the analysis, interpretation, and transparent reporting of PRO data in cancer clinical trials, leading to a more valid and critically appraisable understanding of PRO-based endpoints. It will establish a solid foundation for future research to address existing gaps, such as defining thresholds for interpreting group-level differences.
METHODS: The SISAQOL-IMI recommendations emerged from a comprehensive, multi-round consensus process involving 41 organisations over a four-year span and build on the estimand framework. This diverse group included clinicians, patients, academics, industry representatives, regulatory authorities, and HTA bodies. The work of the consortium is readily accessible through various resources, including an online platform (www.sisaqol-imi.org). The harmonised terminology and recommendations for issues related to PRO score interpretation thresholds were developed through five in-person consensus rounds, a series of virtual meetings, a patient workshop, and stakeholder surveys.
RESULTS: The work on the use of PRO score interpretation thresholds resulted in 27 recommendations specific to the use of such thresholds in cancer clinical trials. A key achievement is the establishment of a harmonised terminology for PRO score interpretation thresholds, which provides clear definitions for various threshold types, including differentiating between patient- and group-level thresholds. Crucially, these recommendations link specific threshold types to appropriate statistical analysis methods, thereby instilling correct implementation and valid interpretation of PRO data. Furthermore, key criteria for threshold selection are detailed and advocate for, e.g., anchor-based methods—especially those leveraging patient-centred anchors—to infuse meaning into the interpretation of PRO data. Detailed terminology, recommendations, and accompanying examples will be presented at the conference.
CONCLUSIONS: The SISAQOL-IMI recommendations and terminology for PRO score interpretation thresholds will significantly enhance the analysis, interpretation, and transparent reporting of PRO data in cancer clinical trials, leading to a more valid and critically appraisable understanding of PRO-based endpoints. It will establish a solid foundation for future research to address existing gaps, such as defining thresholds for interpreting group-level differences.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
PCR243
Topic
Clinical Outcomes, Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Oncology