Dorme L1, Pe M1, Coens C1, Basch EM2, Calvert M3, Campbell AK4, Cleeland C5, Cocks K6, Collette L1, Devlin N7, Dueck AC8, Flechtner HH9, Gotay C10, Griebsch I11, Grønvold M12, Johnson LL13, King MT14, Kluetz PG13, Koller M15, Malone D16, Martinelli F1, Mitchell SA17, Musoro J1, O'Connor D18, Oliver K19, Piault-Louis E20, Piccart M21, Quinten C22, Reijneveld JC23, Christoph S24, Sloan J25, Ashley WS17, Soltys KM26, Sridhara R27, Taphoorn MJ28, Velikova G12, Bottomley A1
1EORTC, Brussels, Belgium, 2University of North Carolina at Chapel Hill, Chapel Hill, NC, USA, 3University of Birmingham, Birmingham, UK, 4Patient Relevant Evidence, San Francisco, CA, USA, 5University of Texas MD Anderson Cancer Center, Houston, TX, USA, 6Adelphi Values Ltd, Bollington, UK, 7University of Melbourne, Melbourne, VIC, Australia, 8Mayo Clinic, Scottsdale, AZ, USA, 9University of Magdeburg, Magdeburg, Germany, 10University of British Columbia, Vancouver, BC, Canada, 11Boehringer Ingelheim International GmbH, Ingelheim, Germany, 12University of Southampton, Southampton, UK, 13FDA, Silver Spring, MD, USA, 14University of Sydney, Sydney, Australia, 15University Hospital Regensburg, Regensburg, Germany, 16University of Arizona, Tucson, AZ, USA, 17National Cancer Institute (NCI), Bethesda, MD, USA, 18Medicines and Healthcare products Regulatory Agency, London, UK, 19International Brain Tumour Alliance (IBTA), Surrey, UK, 20Genentech, Inc., South San Francisco, CA, USA, 21Institut Jules bordet, Université Libre de Bruxelles, Brussels, Belgium, 22European Centre for Disease Prevention and Control (ECDC), Stockholm, Sweden, 23VU University Medical Center, Amsterdam, Netherlands, 24Institute for Quality and Efficiency in Health Care (IQWIG), Cologne, Germany, 25Mayo Clinic, Rochester, MN, USA, 26Health Canada, Ottawa, ON, Canada, 27US Food and Drug Administration (FDA), Silver Spring, MD, USA, 28Leiden University Medical Center, Leiden, Netherlands

Presentation Documents

OBJECTIVES : Patient-reported outcome (PRO) data, such as symptoms and health-related quality of life, are increasingly being captured in cancer randomized clinical trials (RCTs) to provide valuable information on treatment risks, benefits and tolerability. Our literature review showed heterogeneity in analysis, interpretation and reporting of these data in various cancer fields, hindering comparability in results across trials. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) Consortium was convened to set recommendations for PRO analysis in cancer RCTs.

METHODS : The Consortium comprises 40 international experts including PRO researchers and statisticians, representatives from regulatory bodies, academic societies, pharmaceutical industry, cancer institutes and patient organizations to work on four priorities: (a) specification of well-defined PRO research objectives, (b) recommendations for appropriate statistical PRO analysis methods, (c) standardization of statistical terminology and (d) development of guidelines for analyzing missing data. Recommendations were developed through literature review, surveys, meeting discussions and ratified through voting in a final meeting.

RESULTS : A taxonomy of research objectives was developed. Appropriate statistical methods, with the exception of summary measures, were proposed. Consensus was reached on the taxonomy of research objectives and statistical methods, along with a missing data definition and two rates to report missing data occurrence. While some statements concerning handling missing data or statistical analyses are still to be discussed, many statements were ratified for each of the priorities.

CONCLUSIONS : A robust first set of PRO analyses recommendations were established in a joint process with diverse international stakeholders. Addressing the needs and requirements of these stakeholders provides a strong foundation for widespread endorsement of these recommendations. Ultimately, we expect enhanced interpretability and better impact of PRO data in cancer RCTs from harmonization of current research practices.

Disclaimer: This publication reflects solely the views of the individual authors

Conference/Value in Health Info

2019-11, ISPOR Europe 2019, Copenhagen, Denmark





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