SISAQOL-IMI consensus-based guidelines to design, analyse, interpret, and present patient-reported outcomes in cancer clinical trials

Cecilie D Amdal¹, Ragnhild S Falk¹, Ahu Alanya², Michael Schlichting³, Satrajit Roychoudhury⁴, Vishal Bhatnagar⁵, Lisa M Wintner⁶, Antoine Regnault⁷, Anders Ingelgård⁸, Corneel Coens², Saskia le Cessie⁹, Bernhard Holzner⁶, Jane Chang⁴, Martin Taphoorn¹⁰, Paul Cislo⁴, Johannes M Giesinger⁶, Joseph C Cappelleri⁴, Vivek Pawar¹¹, Silene Ten Seldam¹², Elektra J Papadopoulos¹³, Melanie J Calvert¹⁴, Kenth L Joseph¹, Andrew Bottomley², Ingolf Griebsch⁸, Juan I Arraras¹⁵, Guro L Astrup¹, Ethan Basch¹⁶, Andrej Belančić¹⁷, Michael Brundage¹⁸, Alicyn Campbell¹⁹, Khadija Rerhou Rantell²⁰, Kim Cocks²¹, Nathan Cherny²², Sonya Eremenco²³, Montse Ferrer²⁴, Mallorie H Fiero⁵, Christoph Gerlinger²⁵, Els Goetghebeur²⁶, Ulrich Grouven²⁷, Alexandra Lauer⁸, Olalekan Lee Aiyegbusi¹⁴, Abigirl Machingura², Junki Mizusawa²⁸, Geert Molenberghs²⁹, Morten Aa Petersen³⁰, Jaap C Reijneveld³¹, Jolie Ringash³², Gerhard Rumpold³³, Claudia Rutherford³⁴, Chantal Quinten³⁵, Kavita Sail¹³, Maxime Sasseville³⁶, Willi Sauerbrei³⁷, Anja Schiel³⁸, Ashley W Smith³⁹, Claire Snyder⁴⁰, Galina Velikova⁴¹, Xin S Wang⁴², Kristin Bjordal¹, Madeline Pe⁴³; SISAQOL-IMI Consortium

Collaborators, Affiliations

Collaborators

SISAQOL-IMI Consortium:
Solomon Alexi, Imad Al-Naesan, Jennifer Black, Florien Boele, Kris Bogaerts, Sarah Böhme, Ting-Yu Chen, Francesca Chiavaro, Vincent Claus, Charlie Cleeland, Cheryl Coon, Samantha Cruz Rivera, Gracia Dekanic Arbanas, Gregoire Desplanques, Nnadozie Emechebe, Kristina Fitzgerald, Olatz Garin, Miaomiao Ge, Alexandra Gilbert, Mogens Groenvold, Franziska Gross, Erica Horodniceanu, Laura Lee Johnson, Katie Joyner, Rajesh Kamalakar, Madeleine King, Paul Kluetz, Karin Kuljanic, Nicola Latino, Limin Liu, Morine Maguri, Francesca Martinelli, Flora Mazerolle, Sandra Mitchell, Kate Morgan, Jammbe Musoro, David Ness, Irene Nsiah, Sjoukje Oosting, Rosemary Peacock

Affiliations

¹ Oslo University Hospital, Oslo, Norway.
² Quality of Life Department, European Organisation for Research and Treatment of Cancer Headquarters, Brussels, Belgium.
³ Merck, Healthcare KGaA, Darmstadt, Germany.
⁴ Pfizer, New York, NY, USA.
⁵ US Food and Drug Administration, Silver Spring, MD, USA.
⁶ University Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.
⁷ Modus Outcomes, Lyon, France.
⁸ Boehringer Ingelheim, Ingelheim am Rhein, Germany.
⁹ Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, Netherlands.
¹⁰ Department of Neurology, Leiden University Medical Center, Leiden, Netherlands.
¹¹ EMD Serono, Billerica, MA, USA.
¹² Myeloma Patients Europe, Brussels, Belgium.
¹³ AbbVie, Mettawa, IL, USA.
¹⁴ Centre for Patient Reported Outcomes Research and NIHR Biomedical Research Centre, University of Birmingham, Birmingham, UK.
¹⁵ Hospital Universitario de Navarra, Pamplona, Spain.
¹⁶ American Society for Clinical Oncology, Alexandria, VA, USA.
¹⁷ Clinical Hospital Center Rijeka, Rijeka, Croatia.
¹⁸ Queen's University at Kingston, Kingston, ON, Canada.
¹⁹ Patient Relevant Evidence, San Francisco, CA, USA.
²⁰ Medicines and Healthcare products Regulatory Agency, London, UK.
²¹ Adelphi Values, Bollington, UK.
²² European Society for Medical Oncology, Lugano, Switzerland.
²³ Critical Path Institute, Tucson, AZ, USA.
²⁴ Hospital del Mar Research Institute, Consorcio de Investigación Biomédica en Red de Epidemiología y Salud Pública, Instituto de Salud Carlos III, Barcelona, Spain.
²⁵ Bayer AG, Berlin, Germany; Gynaecology, Obstetrics and Reproductive Medicine, University Medical School of Saarland, Homburg, Germany.
²⁶ Department of Applied Mathematics, Computer Science and Statistics, Ghent University, Ghent, Belgium.
²⁷ Institute for Quality and Efficiency in Health Care, Cologne, Germany.
²⁸ Japan Clinical Oncology Group, National Cancer Center Hospital, Tokyo, Japan.
²⁹ KU Leuven, Leuven, Belgium; Hasselt University, Hasselt, Belgium.
³⁰ Region Hovedstaden, Copenhagen, Denmark.
³¹ Amsterdam University Medical Center, Amsterdam, Netherlands.
³² University Health Network, Toronto, ON, Canada.
³³ Evaluation Software Development, Innsbruck, Austria.
³⁴ Faculty of Medicine and Health and Susan Wakil School of Nursing and Midwifery, Sydney Quality of Life Office, University of Sydney, Sydney, NSW, Australia.
³⁵ European Medicines Agency, Amsterdam, Netherlands.
³⁶ Health Canada, Ottawa, ON, Canada.
³⁷ Faculty of Medicine and Medical Center, University of Freiburg, Freiburg, Germany.
³⁸ Norwegian Medical Products Agency, Oslo, Norway.
³⁹ National Cancer Institute, Bethesda, MD, USA.
⁴⁰ Johns Hopkins University School of Medicine, Baltimore, MD, USA.
⁴¹ Leeds Institute of Medical Research, University of Leeds, Leeds, UK; Leeds Cancer Centre, St James's Hospital, Leeds, UK.
⁴² University of Texas MD Anderson Cancer Center, Houston, TX, USA.
⁴³ Quality of Life Department, European Organisation for Research and Treatment of Cancer Headquarters, Brussels, Belgium. Electronic address: madeline.pe@eortc.org.

PMID: 41308689
DOI: 10.1016/S1470-2045(25)00520-0

Review

SISAQOL-IMI consensus-based guidelines to design, analyse, interpret, and present patient-reported outcomes in cancer clinical trials

Cecilie D Amdal et al. Lancet Oncol. 2025 Dec.

. 2025 Dec;26(12):e683-e693.

doi: 10.1016/S1470-2045(25)00520-0.

Authors

Collaborators

SISAQOL-IMI Consortium:
Solomon Alexi, Imad Al-Naesan, Jennifer Black, Florien Boele, Kris Bogaerts, Sarah Böhme, Ting-Yu Chen, Francesca Chiavaro, Vincent Claus, Charlie Cleeland, Cheryl Coon, Samantha Cruz Rivera, Gracia Dekanic Arbanas, Gregoire Desplanques, Nnadozie Emechebe, Kristina Fitzgerald, Olatz Garin, Miaomiao Ge, Alexandra Gilbert, Mogens Groenvold, Franziska Gross, Erica Horodniceanu, Laura Lee Johnson, Katie Joyner, Rajesh Kamalakar, Madeleine King, Paul Kluetz, Karin Kuljanic, Nicola Latino, Limin Liu, Morine Maguri, Francesca Martinelli, Flora Mazerolle, Sandra Mitchell, Kate Morgan, Jammbe Musoro, David Ness, Irene Nsiah, Sjoukje Oosting, Rosemary Peacock

Affiliations

¹ Oslo University Hospital, Oslo, Norway.
² Quality of Life Department, European Organisation for Research and Treatment of Cancer Headquarters, Brussels, Belgium.
³ Merck, Healthcare KGaA, Darmstadt, Germany.
⁴ Pfizer, New York, NY, USA.
⁵ US Food and Drug Administration, Silver Spring, MD, USA.
⁶ University Hospital of Psychiatry II, Medical University of Innsbruck, Innsbruck, Austria.
⁷ Modus Outcomes, Lyon, France.
⁸ Boehringer Ingelheim, Ingelheim am Rhein, Germany.
⁹ Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, Netherlands.
¹⁰ Department of Neurology, Leiden University Medical Center, Leiden, Netherlands.
¹¹ EMD Serono, Billerica, MA, USA.
¹² Myeloma Patients Europe, Brussels, Belgium.
¹³ AbbVie, Mettawa, IL, USA.
¹⁴ Centre for Patient Reported Outcomes Research and NIHR Biomedical Research Centre, University of Birmingham, Birmingham, UK.
¹⁵ Hospital Universitario de Navarra, Pamplona, Spain.
¹⁶ American Society for Clinical Oncology, Alexandria, VA, USA.
¹⁷ Clinical Hospital Center Rijeka, Rijeka, Croatia.
¹⁸ Queen's University at Kingston, Kingston, ON, Canada.
¹⁹ Patient Relevant Evidence, San Francisco, CA, USA.
²⁰ Medicines and Healthcare products Regulatory Agency, London, UK.
²¹ Adelphi Values, Bollington, UK.
²² European Society for Medical Oncology, Lugano, Switzerland.
²³ Critical Path Institute, Tucson, AZ, USA.
²⁴ Hospital del Mar Research Institute, Consorcio de Investigación Biomédica en Red de Epidemiología y Salud Pública, Instituto de Salud Carlos III, Barcelona, Spain.
²⁵ Bayer AG, Berlin, Germany; Gynaecology, Obstetrics and Reproductive Medicine, University Medical School of Saarland, Homburg, Germany.
²⁶ Department of Applied Mathematics, Computer Science and Statistics, Ghent University, Ghent, Belgium.
²⁷ Institute for Quality and Efficiency in Health Care, Cologne, Germany.
²⁸ Japan Clinical Oncology Group, National Cancer Center Hospital, Tokyo, Japan.
²⁹ KU Leuven, Leuven, Belgium; Hasselt University, Hasselt, Belgium.
³⁰ Region Hovedstaden, Copenhagen, Denmark.
³¹ Amsterdam University Medical Center, Amsterdam, Netherlands.
³² University Health Network, Toronto, ON, Canada.
³³ Evaluation Software Development, Innsbruck, Austria.
³⁴ Faculty of Medicine and Health and Susan Wakil School of Nursing and Midwifery, Sydney Quality of Life Office, University of Sydney, Sydney, NSW, Australia.
³⁵ European Medicines Agency, Amsterdam, Netherlands.
³⁶ Health Canada, Ottawa, ON, Canada.
³⁷ Faculty of Medicine and Medical Center, University of Freiburg, Freiburg, Germany.
³⁸ Norwegian Medical Products Agency, Oslo, Norway.
³⁹ National Cancer Institute, Bethesda, MD, USA.
⁴⁰ Johns Hopkins University School of Medicine, Baltimore, MD, USA.
⁴¹ Leeds Institute of Medical Research, University of Leeds, Leeds, UK; Leeds Cancer Centre, St James's Hospital, Leeds, UK.
⁴² University of Texas MD Anderson Cancer Center, Houston, TX, USA.
⁴³ Quality of Life Department, European Organisation for Research and Treatment of Cancer Headquarters, Brussels, Belgium. Electronic address: madeline.pe@eortc.org.

PMID: 41308689
DOI: 10.1016/S1470-2045(25)00520-0

Abstract

Standardising the implementation of patient-reported outcomes (PROs) in clinical trials is crucial for evaluating the benefits and risks of cancer treatments. The Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI) has developed 146 consensus-based recommendations for designing, analysing, interpreting, and presenting PROs in cancer clinical trials. This initiative, undertaken from 2021 to 2025, involved experts, including statisticians, PRO measurement experts, clinicians, and patient representatives from 41 organisations representing regulatory agencies, academia, the pharmaceutical industry, health-technology assessment bodies, and patient advocates. SISAQOL-IMI provides guidance on the implementation of PROs in randomised controlled trials and single-arm trials, terminology, definitions and the selection of PRO score interpretation thresholds, and for visualising PRO results for different audiences. To facilitate the implementation of these standards, in addition to this Policy Review, four key outputs are available: an interactive table, a guidebook, plain language materials, and a glossary.

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Conflict of interest statement

Declaration of interests KB, JMG, BH, and GR received funding paid to their institution from the Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI) grant to conduct this work. ABe, CQ, NC, and MF received travel grants from SISAQOL-IMI to participate in the annual meeting. EB received consulting fees as a scientific advisor for AZ, Navigating Cancer, Verily, N-Power Medicine, Savor, Thyme Care, and Resilience Health. AC was an employee of Genentech from 2011 to 2019 and then AZ from 2020 to 2024; however, the current work was done as part of PRE LLC and on external time. AC has a patent unrelated to this work (AZ ownership); and reports stocks, leadership roles, receipt of equipment and other services, and other financial and non-financial interest from AZ. PC, JC, and SR are employees of Pfizer and receive stocks and have stock options. MJC received grants paid to her institution from the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, Health Data Research UK, Innovate UK, Macmillan Cancer Support, GSK, Research England, European Commission and the European Federation of Pharmaceutical Industries and Associations, Brain Tumour Charity, Gilead, Janssen, NIHR, UK Research and Innovation, and Merck; revenue from commercial licence of the symptom burden questionnaire–long COVID; consulting fees from Aparito, Boehringer Ingelheim, CIS Oncology, Merck, Genentech, GSK, Pfizer, Vertex Pharmaceuticals, ICON, Halfloop, the University of Michigan, Northwestern University, EuroQoL, Shionogi, and Cell and Gene Therapy Catapult; payment or honoraria from the University of Maastricht, Sount-Eastern Norway Regional Health Authority, Cochrane Portugal, and Singapore National Medical Research Council; has a leadership role in PROTEUS Consortium for which she receives a consultancy fee; and her family member has stocks in GSK. OLA received grants and contracts paid to University of Birmingham from NIHR Biomedical Research Centres, NIHR Applied Research Collaborations, NIHR Blood and Transplant Research Unit in Precision Transplant and Cellular Therapeutics, Anthony Nolan Trust, Sarcoma UK, and Merck; and personal consulting fees paid through the University of Birmingham from Merck, GSK, Gilead, Boehringer Ingelheim, and Innovate UK. MB received an unrestricted research grant paid to his institution from Pfizer. KC received travel grants from SISAQOL-IMI to attend the annual meeting; and is an employee for Adelphi Values who consult with pharmaceutical companies. SE had membership fees paid by PRO Consortium member firms to the Critical Path Institute (CPATH) to support staff time to participate in the Consortium; received grants and broad agency announcement contract payments from the US Food and Drug Administration to CPATH; travel funds to participate in the American College of Neuropsychopharmacology 2022 Annual meeting; and travel grants to attend the SISAQOL-IMI meeting. CG is an employee of Bayer and holds stock options. MSc is an employee of Merck and received stocks and stock options. EJP is an employee of AbbVie. JCR is the Chair of the European Organisation for Research and Treatment of Cancer (EORTC) quality of life group (unpaid); is a member of a scientific advisory board for EpilepsieNL (unpaid); and received travel grants to attend the annual SISAQOL-IMI meeting. StS reports that her employer Myeloma Patients Europe receives grant and sponsorship from various pharmaceutical companies, which are listed on their webpage.(28) CS received funding from Pfizer and Genentech to her institution; personal consulting fees from Shionogi and Movember; and travel costs from Shionogi to present on a panel at the CPATH COA Consortium meeting. GV received grants from NIHR, Pfizer, and Yorkshire Cancer Research all paid to institution; consulting fees paid from Pfizer, Roche, and Seagen; payments and honoraria from Pfizer, Roche, Novartis, Eisai, and Sanofi; travel support from Pfizer and Roche; payment as part of her participation on a data safety monitoring board or advisory board from Roche, Seagen, and AZ; and holds leadership roles as part of the EORTC board of directors and National Cancer Research Institute Chair of Living with and Beyond Cancer group. All other authors declare no competing interests.

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SISAQOL-IMI consensus-based guidelines to design, analyse, interpret, and present patient-reported outcomes in cancer clinical trials

Collaborators

Affiliations

SISAQOL-IMI consensus-based guidelines to design, analyse, interpret, and present patient-reported outcomes in cancer clinical trials

Authors

Collaborators

Affiliations

Abstract

Conflict of interest statement

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LinkOut - more resources

Full Text Sources

Medical

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