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International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL Consortium

Publikation: Bidrag til tidsskriftReviewpeer review

  • Corneel Coens
  • Madeline Pe
  • Amylou C Dueck
  • Jeff Sloan
  • Ethan Basch
  • Melanie Calvert
  • Alicyn Campbell
  • Charles Cleeland
  • Kim Cocks
  • Laurence Collette
  • Nancy Devlin
  • Lien Dorme
  • Hans-Henning Flechtner
  • Carolyn Gotay
  • Ingolf Griebsch
  • Mogens Groenvold
  • Madeleine King
  • Paul G Kluetz
  • Michael Koller
  • Daniel C Malone
  • Francesca Martinelli
  • Sandra A Mitchell
  • Jammbe Z Musoro
  • Daniel O'Connor
  • Kathy Oliver
  • Elisabeth Piault-Louis
  • Martine Piccart
  • Chantal Quinten
  • Jaap C Reijneveld
  • Christoph Schürmann
  • Ashley Wilder Smith
  • Katherine M Soltys
  • Martin J B Taphoorn
  • Galina Velikova
  • Andrew Bottomley
Vis graf over relationer

Patient-reported outcomes (PROs), such as symptoms, function, and other health-related quality-of-life aspects, are increasingly evaluated in cancer randomised controlled trials (RCTs) to provide information about treatment risks, benefits, and tolerability. However, expert opinion and critical review of the literature showed no consensus on optimal methods of PRO analysis in cancer RCTs, hindering interpretation of results. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium was formed to establish PRO analysis recommendations. Four issues were prioritised: developing a taxonomy of research objectives that can be matched with appropriate statistical methods, identifying appropriate statistical methods for PRO analysis, standardising statistical terminology related to missing data, and determining appropriate ways to manage missing data. This Policy Review presents recommendations for PRO analysis developed through critical literature reviews and a structured collaborative process with diverse international stakeholders, which provides a foundation for endorsement; ongoing developments of these recommendations are also discussed.

TidsskriftThe Lancet Oncology
Udgave nummer2
Sider (fra-til)e83-e96
Antal sider14
StatusUdgivet - feb. 2020

Bibliografisk note

Funding Information:
MC reports personal fees from Astellas, Takeda, Glaukos, Merck, Daiichi Sankyo, and the Patient-Centered Outcomes Research Institute; and grants from Health Data Research UK, Innovate UK, National Institute for Health Research (NIHR), Macmillan, and UCB Pharma, outside the submitted work. AC reports having been an employee of Genentech until Jan 31, 2019. KC reports grants from European Organization for Research and Treatment of Cancer (EORTC); and personal fees from Bristol-Myers Squibb, Endomag, Celgene, and Amgen, outside the submitted work. IG is an employee of Boehringer Ingelheim International, which provided an unrestricted education grant to EORTC. MKo reports grants from EORTC, Biofrontera, and Komitee Forschung Naturmedizin; and personal fees from Janssen-Cilag, Lily, and Verband Forschender Arzneimittelhersteller, outside the submitted work. KO reports grants from Bristol-Myers Squibb, Roche, Novocure, Lilly, Pfizer, MagForce, Novartis, Medac, Photonamic, Northwest Biotherapeutics, VBL Therapeutics, AbbVie, Elekta, Apogenix, and Bayer, outside the submitted work. EP-L is an employee of Genentech. MPi reports being a member of the Radius advisory board; grants to Institut Jules Bordet from Radius, Synthon, and Servier; grants and personal fees to Institut Jules Bordet from AstraZeneca, Lilly, Merck Sharp & Dohme, Novartis, Pfizer, and Roche-Genentech; and personal fees from Odonate, Camel-IDS, Crescendo Biologics, Periphagen, Huya, Debiopharm, PharmaMar, G1 Therapeutics, Menarini, Seattle Genetics, Immunomedics, and Oncolytics, outside the submitted work. GV reports personal fees from Roche, Eisai, Novartis, and Pfizer; and grants from the NIHR UK Government, Breast Cancer NOW, and EORTC, outside the submitted work. AB reports grants to EORTC from Boehringer Ingelheim International, Genentech, and the EORTC research fund during the conduct of the study; grants from Merck outside the submitted work; and reports being a member of the EORTC Quality of Life Group executive committee. All other authors declare no competing interests.

Funding Information:
The European Organisation for Research and Treatment of Cancer received an unrestricted education grant from Boehringer Ingelheim to initiate this work and from Genentech, a member of the Roche Group, for continuity funding. We thank the International Society for Quality of Life Research and the International Society for Pharmacoeconomics and Outcomes Research for their support and review of the final manuscript and we thank Gina Mazza for the help with the survey research in the missing data part. This publication reflects the views of the individual authors and should not be construed to represent official views or policies of the US Food and Drug Administration, US National Cancer Institute, Medicines and Healthcare products Regulatory Agency, Institute for Quality and Efficiency in Health Care, Health Canada, the UK National Health Service, the National Institute for Health Research, or the UK Department of Health. This study received no funding from the US National Institutes of Health (NIH). AWS and SAM are employed by NIH. No other authors were fully or partly NIH funded, employed by NIH, or are in receipt of an NIH grant.

ID: 59912774