Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI): stakeholder views, objectives, and procedures.

Madeline Pe ,Vishal Bhatnagar ,Martine Piccart ,Maarten Voorhaar ,Rajesh Kamalakar ,Cristián Frigolett Catalan ,Jane Chang ,Lindsey Murray ,Bethany J Wolf ,Morten Aa Petersen ,Michael Tschuggnall ,Anders Ingelgaard ,Kristin Bjordal ,Olalekan Lee Aiyegbusi ,Ivana Rede ,Saskia Le Cessie ,Miaomiao Ge ,Barbara Kiesewetter ,Elisabeth G.e De Vries ,Christoph Schürmann ,Doranne Thomassen ,Olatz Garín ,Sjoukje F Oosting ,Mitsumi Terada ,Sonya Eremenco ,Ragnhild Sørum Falk ,Florien Boele ,Rickard Sandin ,Kathy Oliver ,Lisa Campbell ,Micha J Pilz ,Kristina Fitzgerald ,Yolanda Barbáchano ,Mogens Grøenvold ,Nathan I Cherny ,Kenth Louis Hansen Joseph ,Galina Velikova ,Bishal Gyawali ,Gemma Vilagut ,Jinma Ren ,Franziska Gross ,Sandra A Mitchell ,Linda Dirven ,Willi Sauerbrei ,Corinne De Vries ,Ahu Alanya ,Bernhard Holzner ,Yun Su ,Xin Shelley Wang ,Paul G Kluetz ,Katherine Soltys ,Bryce B Reeve ,Ingolf Griebsch ,Michael Schlichting ,Claudia Rutherford ,Geert Verbeke ,Kavita Sail ,Michael Brundage ,Vivek Pawar ,Khadija Rantell ,Felix Schoepf ,Lisa M Wintner ,Kate Morgan ,Francesco Pignatti ,Juan Ignacio Arrarás ,Elektra J Papadopoulos ,Alexander Russell-Smith ,Urania Dafni ,Daniel O’connor ,Carla Mamolo ,Sean Turner ,Gerhard Rumpold ,Ananda Plate ,Felipe Roitberg ,Kelly Van Lancker ,Jaap C Reijneveld ,Stephen Joel Coons ,Melanie Calvert ,Rohini Sen ,Laura Lee Johnson ,Montse Ferrer ,Thomas Schlüter ,Paul Cislo ,Erica Horodniceanu ,Ralf Herold ,Cat N Bui ,Lori Williams ,Alicyn Campbell ,Febe Brackx ,Kris Bogaerts ,Geert Molenberghs ,Brendon Wong ,Abigirl Machingura ,Rebecca M Speck ,Rosemary Peacock ,Gregoire Desplanques ,Samantha Cruz Rivera ,Martin J.b Taphoorn ,Ariel Alonso Abad ,Nnadozie Emechebe ,Dongsheng Tu ,Chantal Quinten ,Ashley Wilder Smith ,Maxime Sasseville ,Johannes M Giesinger ,Jorge Barriuso ,Joseph C Cappelleri ,Kim Cocks ,Patrizia De Besi ,Alexandra Gilbert ,Barbara Peil ,Antoine Regnault ,Jan Geißler ,Limin Liu ,Francesca Martinelli ,Andrew Bottomley ,Jammbe Z Musoro ,Duška Petranović ,C Cleeland ,Junki Mizusawa ,Gracia Dekanic Arbanas ,Mallorie H Fiero ,Corneel Coens ,Silene Ten Seldam ,Anja Schiel ,Karen Keating ,Jayne Galinsky ,Els Goetghebeur ,Ethan Basch ,Cecilie Delphin Amdal ,Tove Ragna Reksten ,Karin Kuljanić ,Jun‐Yong Choi ,Beate Wieseler ,Jennifer A Black ,Satrajit Roychoudhury ,Madeleine King ,Jolie Ringash ,Claire Snyder ,Christoph Gerlinger ,Lisa Rodríguez

doi:10.1016/s1470-2045(23)00157-2

Abstract

Patient-reported outcomes (PROs), such as symptoms, functioning, and other health-related quality-of-life concepts are gaining a more prominent role in the benefit-risk assessment of cancer therapies. However, varying ways of analysing, presenting, and interpreting PRO data could lead to erroneous and inconsistent decisions on the part of stakeholders, adversely affecting patient care and outcomes. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials-Innovative Medicines Initiative (SISAQOL-IMI) Consortium builds on the existing SISAQOL work to establish recommendations on design, analysis, presentation, and interpretation for PRO data in cancer clinical trials, with an expanded set of topics, including more in-depth recommendations for randomised controlled trials and single-arm studies, and for defining clinically meaningful change. This Policy Review presents international stakeholder views on the need for SISAQOL-IMI, the agreed on and prioritised set of PRO objectives, and a roadmap to ensure that international consensus recommendations are achieved.

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