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.
Competing Interests: Declaration of interests SR is a current employee of Pfizer and a former employee of Novartis Pharma. JC, PC, and JR are current employees of Pfizer. VP and IG are current employees of EMD Serono. MS is a current employee of Merk. GV has received consulting fees or payment from or related to the following organisations: Pfizer, Eisai, Roche, Novartis, AstraZeneca, Sanofi, Seattle Genetics, the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Group, and the EORTC Board. KO's organisation has received sponsorship funding or grants for various annual programmes and activities from Bristol Myers Squibb, Novocure, Pfizer, Bayer, Novartis, Northwest Biotherapeutics, Karyopharm, MagForce, Medac, Photonamic, Apogenix, Elekta, and GW Pharmaceuticals/Jazz Pharmaceuticals; consulting fees from Bristol Myers Squibb and Novartis; and honoraria from Sanofi, Sharing Progress in Cancer Care, and Seagen. KO participated in an advisory board for Novartis, Novocure, Seagen, Eisai, Bristol Myers Squibb, and Sanofi, and has leadership roles in a number of organisations. All other authors declare no competing interests.
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