Moving forward toward standardizing analysis of quality of life data in randomized cancer clinical trials

Andrew Bottomley¹, Madeline Pe¹, Jeff Sloan², Ethan Basch³, Franck Bonnetain⁴, Melanie Calvert⁵, Alicyn Campbell⁶, Charles Cleeland⁷, Kim Cocks⁸, Laurence Collette¹, Amylou C Dueck⁹, Nancy Devlin¹⁰, Hans-Henning Flechtner¹¹, Carolyn Gotay¹², Eva Greimel¹³, Ingolf Griebsch¹⁴, Mogens Groenvold¹⁵, Jean-Francois Hamel¹⁶, Madeleine King¹⁷, Paul G Kluetz¹⁸, Michael Koller¹⁹, Daniel C Malone²⁰, Francesca Martinelli¹, Sandra A Mitchell²¹, Carol M Moinpour²², Jammbe Z Musoro¹, Daniel O'Connor²³, Kathy Oliver²⁴, Elisabeth Piault-Louis⁶, Martine Piccart²⁵, Francisco L Pimentel^{26

27}, Chantal Quinten²⁸, Jaap C Reijneveld²⁹, Christoph Schürmann³⁰, Ashley Wilder Smith²¹, Katherine M Soltys³¹, Rajeshwari Sridhara¹⁸, Martin J B Taphoorn³², Galina Velikova³³, Corneel Coens¹

Affiliations

¹ 1 Quality of Life Department, European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium.
² 2 Alliance Statistics and Data Center, Mayo Clinic, Rochester, MN, USA.
³ 3 Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC, USA.
⁴ 4 Methodology and Quality of Life Unit in Cancer, INSERM U1098, University Hospital of Besançon, Besançon, France.
⁵ 5 Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, College of Medical and Dental Sciences, University of Birmingham, Birmingham, UK.
⁶ 6 Genentech, San Francisco, CA, USA.
⁷ 7 Department of Symptom Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
⁸ 8 Adelphi Values, Bollington, UK.
⁹ 9 Alliance Statistics and Data Center, Mayo Clinic, Scottsdale, AZ, USA.
¹⁰ 10 Office of Health Economics, London, UK.
¹¹ 11 Clinic for Child and Adolescent Psychiatry and Psychotherapy, University of Magdeburg, Magdeburg, Germany.
¹² 12 School of Population and Public Health, University of British Columbia, Vancouver, BC, Canada.
¹³ 13 Department of Obstetrics and Gynecology, Medical University Graz, Graz, Austria.
¹⁴ 14 Boehringer Ingelheim, Frankfurt, Germany.
¹⁵ 15 Department of Public Health, University of Copenhagen and Bispebjerg Hospital, Copenhagen, Denmark.
¹⁶ 16 Methodology and Biostatistics Department, University Hospital of Angers UNAM, Angers, France.
¹⁷ 17 School of Psychology and Sydney Medical School, University of Sydney, Sydney, NSW, Australia.
¹⁸ 18 US Food and Drug Administration, Silver Spring, MD, USA.
¹⁹ 19 Center for Clinical Studies, University Hospital Regensburg, Regensburg, Germany.
²⁰ 20 College of Pharmacy, University of Arizona, Tucson, AZ, USA.
²¹ 21 Outcomes Research Branch, Healthcare Delivery Research Program, Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, MD, USA.
²² 22 Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
²³ 23 Medicines and Healthcare Products Regulatory Agency, London, UK.
²⁴ 24 International Brain Tumour Alliance, Surrey, UK.
²⁵ 25 Internal Medicine/Oncology, Institut Jules Bordet, Brussels, Belgium.
²⁶ 26 Blueclinical Phase I, Porto, Portugal.
²⁷ 27 Centro de Estudos e Investigação em Saúde da Universidade de Coimbra, Coimbra, Portugal.
²⁸ 28 European Centre for Disease Prevention and Control, Surveillance and Response Support Unit, Epidemiological Methods Section, Stockholm, Sweden.
²⁹ 29 VU University Medical Center, Department of Neurology & Brain Tumor Center, Amsterdam, The Netherlands.
³⁰ 30 Institute for Quality and Efficiency in Health Care, Cologne, Germany.
³¹ 31 Health Canada, Ottawa, ON, Canada.
³² 32 Leiden University Medical Center/Haaglanden Medical Center, Leiden/The Hague, The Netherlands.
³³ 33 Leeds Institute of Cancer and Pathology, University of Leeds, St James's Hospital, Leeds, UK.

PMID: 30141714
DOI: 10.1177/1740774518795637

Free article

Moving forward toward standardizing analysis of quality of life data in randomized cancer clinical trials

Andrew Bottomley et al. Clin Trials. 2018 Dec.

Free article

. 2018 Dec;15(6):624-630.

doi: 10.1177/1740774518795637. Epub 2018 Aug 24.

Authors

Affiliations

¹ 1 Quality of Life Department, European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium.
² 2 Alliance Statistics and Data Center, Mayo Clinic, Rochester, MN, USA.
³ 3 Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC, USA.
⁴ 4 Methodology and Quality of Life Unit in Cancer, INSERM U1098, University Hospital of Besançon, Besançon, France.
⁵ 5 Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, College of Medical and Dental Sciences, University of Birmingham, Birmingham, UK.
⁶ 6 Genentech, San Francisco, CA, USA.
⁷ 7 Department of Symptom Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
⁸ 8 Adelphi Values, Bollington, UK.
⁹ 9 Alliance Statistics and Data Center, Mayo Clinic, Scottsdale, AZ, USA.
¹⁰ 10 Office of Health Economics, London, UK.
¹¹ 11 Clinic for Child and Adolescent Psychiatry and Psychotherapy, University of Magdeburg, Magdeburg, Germany.
¹² 12 School of Population and Public Health, University of British Columbia, Vancouver, BC, Canada.
¹³ 13 Department of Obstetrics and Gynecology, Medical University Graz, Graz, Austria.
¹⁴ 14 Boehringer Ingelheim, Frankfurt, Germany.
¹⁵ 15 Department of Public Health, University of Copenhagen and Bispebjerg Hospital, Copenhagen, Denmark.
¹⁶ 16 Methodology and Biostatistics Department, University Hospital of Angers UNAM, Angers, France.
¹⁷ 17 School of Psychology and Sydney Medical School, University of Sydney, Sydney, NSW, Australia.
¹⁸ 18 US Food and Drug Administration, Silver Spring, MD, USA.
¹⁹ 19 Center for Clinical Studies, University Hospital Regensburg, Regensburg, Germany.
²⁰ 20 College of Pharmacy, University of Arizona, Tucson, AZ, USA.
²¹ 21 Outcomes Research Branch, Healthcare Delivery Research Program, Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, MD, USA.
²² 22 Fred Hutchinson Cancer Research Center, Seattle, WA, USA.
²³ 23 Medicines and Healthcare Products Regulatory Agency, London, UK.
²⁴ 24 International Brain Tumour Alliance, Surrey, UK.
²⁵ 25 Internal Medicine/Oncology, Institut Jules Bordet, Brussels, Belgium.
²⁶ 26 Blueclinical Phase I, Porto, Portugal.
²⁷ 27 Centro de Estudos e Investigação em Saúde da Universidade de Coimbra, Coimbra, Portugal.
²⁸ 28 European Centre for Disease Prevention and Control, Surveillance and Response Support Unit, Epidemiological Methods Section, Stockholm, Sweden.
²⁹ 29 VU University Medical Center, Department of Neurology & Brain Tumor Center, Amsterdam, The Netherlands.
³⁰ 30 Institute for Quality and Efficiency in Health Care, Cologne, Germany.
³¹ 31 Health Canada, Ottawa, ON, Canada.
³² 32 Leiden University Medical Center/Haaglanden Medical Center, Leiden/The Hague, The Netherlands.
³³ 33 Leeds Institute of Cancer and Pathology, University of Leeds, St James's Hospital, Leeds, UK.

PMID: 30141714
DOI: 10.1177/1740774518795637

Abstract

Background: There is currently a lack of consensus on how health-related quality of life and other patient-reported outcome measures in cancer randomized clinical trials are analyzed and interpreted. This makes it difficult to compare results across randomized controlled trials (RCTs) synthesize scientific research, and use that evidence to inform product labeling, clinical guidelines, and health policy. The Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data for Cancer Clinical Trials (SISAQOL) Consortium aims to develop guidelines and recommendations to standardize analyses of patient-reported outcome data in cancer RCTs.

Methods and results: Members from the SISAQOL Consortium met in January 2017 to discuss relevant issues. Data from systematic reviews of the current state of published research in patient-reported outcomes in cancer RCTs indicated a lack of clear reporting of research hypothesis and analytic strategies, and inconsistency in definitions of terms, including "missing data,""health-related quality of life," and "patient-reported outcome." Based on the meeting proceedings, the Consortium will focus on three key priorities in the coming year: developing a taxonomy of research objectives, identifying appropriate statistical methods to analyze patient-reported outcome data, and determining best practices to evaluate and deal with missing data.

Conclusion: The quality of the Consortium guidelines and recommendations are informed and enhanced by the broad Consortium membership which includes regulators, patients, clinicians, and academics.

Keywords: Guidelines; cancer clinical trials; health-related quality of life; patient-reported outcomes; standards.

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Moving forward toward standardizing analysis of quality of life data in randomized cancer clinical trials

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Moving forward toward standardizing analysis of quality of life data in randomized cancer clinical trials

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