THE SISAQOL INITIATIVE- ESTABLISHING INTERNATIONAL STANDARDS AND RECOMMENDATIONS FOR THE ANALYSIS OF PATIENT-REPORTED OUTCOMES AND QUALITY OF LIFE DATA IN ONCOLOGY RANDOMIZED CLINICAL TRIALS

Author(s)

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

ISPOR Nov 2019 - SISAQOL.pdf

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

Code

PCN457

Disease

Oncology

Presentation