TY - JOUR

T1 - International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials

T2 - recommendations of the SISAQOL Consortium

AU - Coens, Corneel

AU - Pe, Madeline

AU - Dueck, Amylou C

AU - Sloan, Jeff

AU - Basch, Ethan

AU - Calvert, Melanie

AU - Campbell, Alicyn

AU - Cleeland, Charles

AU - Cocks, Kim

AU - Collette, Laurence

AU - Devlin, Nancy

AU - Dorme, Lien

AU - Flechtner, Hans-Henning

AU - Gotay, Carolyn

AU - Griebsch, Ingolf

AU - Groenvold, Mogens

AU - King, Madeleine

AU - Kluetz, Paul G

AU - Koller, Michael

AU - Malone, Daniel C

AU - Martinelli, Francesca

AU - Mitchell, Sandra A

AU - Musoro, Jammbe Z

AU - O'Connor, Daniel

AU - Oliver, Kathy

AU - Piault-Louis, Elisabeth

AU - Piccart, Martine

AU - Quinten, Chantal

AU - Reijneveld, Jaap C

AU - Schürmann, Christoph

AU - Smith, Ashley Wilder

AU - Soltys, Katherine M

AU - Taphoorn, Martin J B

AU - Velikova, Galina

AU - Bottomley, Andrew

AU - Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data Consortium

N1 - Copyright © 2020 Elsevier Ltd. All rights reserved.

PY - 2020/2

Y1 - 2020/2

N2 - 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.

AB - 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.

U2 - 10.1016/S1470-2045(19)30790-9

DO - 10.1016/S1470-2045(19)30790-9

M3 - Review

C2 - 32007209

VL - 21

SP - e83-e96

JO - The Lancet Oncology

JF - The Lancet Oncology

SN - 1470-2045

IS - 2

ER -