Implementing SISAQOL Recommendations: Insights From Cancer Clinical Trials
Author(s)
Ahu Alanya, PhD1, Anne Esser, MA1, Lotte Van der Weijst, PhD1, Micha J. Pilz, PhD2, Máire O'Donnell, PhD1, Johannes M. Giesinger, PhD2, Madeline Pe, PhD1.
1Quality of Life Department, European Organisation for Research and Treatment of Cancer, Brussels, Belgium, 2Medical University of Innsbruck, Innsbruck, Austria.
1Quality of Life Department, European Organisation for Research and Treatment of Cancer, Brussels, Belgium, 2Medical University of Innsbruck, Innsbruck, Austria.
OBJECTIVES: Objective:
Despite the increasing collection of patient-reported outcomes (PRO) data in cancer clinical trials, optimal incorporation of these endpoints for decision-making by different stakeholders remains unclear. The SISAQOL (Setting International Standards in Analysing
Patient-Reported Outcomes and Quality of Life Endpoints) consortiums brought together organisations representing regulatory agencies, European HTA bodies, oncology societies, academic clinical trial organisations, pharmaceutical industry, and patient groups to provide recommendations on how to incorporate PROs in cancer clinical trials. In 2020, SISAQOL released recommendations to address poorly defined PRO endpoints and missing data; SISAQOL-IMI later expanded these with estimand framework-based guidance. This paper reviewed cancer clinical trials citing the 2020 recommendations, to evaluate implementation, identify areas for improvement, and highlight how the new SISAQOL-IMI guidance may address ongoing challenges.
METHODS: A forward citation search on PubMed, Scopus, and Google Scholar conducted on July 9, 2024, identified 22 trial publications citing the 2020 SISAQOL recommendations. The publication and the trial protocol (available for 20 of 22 trials) were both reviewed.
RESULTS: Despite citing the SISAQOL 2020 recommendations, the objective for the PRO endpoint was still not indicated or unclear in 9 out of 22 studies. The majority of trials reported descriptive PRO endpoints without prior hypotheses, power calculations, or multiplicity corrections. Approximately half of the trials did not pre-specify PRO scales of interest in the protocol, though some highlighted specific scales later in the publications. Reporting on multiple scales, timepoints, and PRO variables was common, with mean change from baseline being the most popular endpoint.
CONCLUSIONS: There is a need for clearer PRO endpoint prioritization and better alignment between protocols and publications including clearer distinction between the main and supplementary analyses. The new set of SISAQOL recommendations, along with other stakeholder initiatives, offer further practical guidance to improve PRO design, analysis and interpretation of PRO data.
Despite the increasing collection of patient-reported outcomes (PRO) data in cancer clinical trials, optimal incorporation of these endpoints for decision-making by different stakeholders remains unclear. The SISAQOL (Setting International Standards in Analysing
Patient-Reported Outcomes and Quality of Life Endpoints) consortiums brought together organisations representing regulatory agencies, European HTA bodies, oncology societies, academic clinical trial organisations, pharmaceutical industry, and patient groups to provide recommendations on how to incorporate PROs in cancer clinical trials. In 2020, SISAQOL released recommendations to address poorly defined PRO endpoints and missing data; SISAQOL-IMI later expanded these with estimand framework-based guidance. This paper reviewed cancer clinical trials citing the 2020 recommendations, to evaluate implementation, identify areas for improvement, and highlight how the new SISAQOL-IMI guidance may address ongoing challenges.
METHODS: A forward citation search on PubMed, Scopus, and Google Scholar conducted on July 9, 2024, identified 22 trial publications citing the 2020 SISAQOL recommendations. The publication and the trial protocol (available for 20 of 22 trials) were both reviewed.
RESULTS: Despite citing the SISAQOL 2020 recommendations, the objective for the PRO endpoint was still not indicated or unclear in 9 out of 22 studies. The majority of trials reported descriptive PRO endpoints without prior hypotheses, power calculations, or multiplicity corrections. Approximately half of the trials did not pre-specify PRO scales of interest in the protocol, though some highlighted specific scales later in the publications. Reporting on multiple scales, timepoints, and PRO variables was common, with mean change from baseline being the most popular endpoint.
CONCLUSIONS: There is a need for clearer PRO endpoint prioritization and better alignment between protocols and publications including clearer distinction between the main and supplementary analyses. The new set of SISAQOL recommendations, along with other stakeholder initiatives, offer further practical guidance to improve PRO design, analysis and interpretation of PRO data.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
MSR128
Topic
Clinical Outcomes, Methodological & Statistical Research, Patient-Centered Research
Topic Subcategory
PRO & Related Methods
Disease
No Additional Disease & Conditions/Specialized Treatment Areas