Rebecca Mercieca-Bebber1,2,3, Douglas Williams4, Margaret-Ann Tait4, Claudia Rutherford4,5, Lucy Busija6, Natasha Roberts7,8, Michelle Wilson9,10, Chindhu Shunmuga Sundaram4, Jessica Roydhouse11. 1. NHMRC Clinical Trials Centre, University of Sydney, Locked bag 77, Camperdown, NSW, 1450, Australia. Rebecca.Mercieca@sydney.edu.au. 2. Faculty of Science, School of Psychology, University of Sydney, Camperdown, NSW, Australia. Rebecca.Mercieca@sydney.edu.au. 3. Faculty of Medicine, Sydney Medical School, Central Clinical School, University of Sydney, Camperdown, NSW, Australia. Rebecca.Mercieca@sydney.edu.au. 4. Faculty of Science, School of Psychology, University of Sydney, Camperdown, NSW, Australia. 5. Cancer Nursing Research Unit (CNRU), Sydney Nursing School, University of Sydney, Camperdown, NSW, Australia. 6. Biostatistics Group, Department of Epidemiology and Preventive Medicine, Monash University, Melbourne, Australia. 7. Royal Brisbane and Women's Hospital (RBWH), Herston, Australia. 8. Queensland University of Technology (QUT), Brisbane, Australia. 9. Auckland City Hospital, Auckland, New Zealand. 10. University of Auckland, Auckland, New Zealand. 11. Department of Health Services, Policy & Practice, Brown University School of Public Health, Providence, USA.
Abstract
AIMS: A proxy is someone other than a patient who reports a patient's outcomes as if they are the patient. Due to known discordance with patient reports, proxies are often not recommended in clinical trials; however, proxies may be needed in certain research contexts. We aimed to identify and describe trials registered on the Australian New Zealand Clinical Trials Registry (ANZCTR) with proxy-reported endpoints. METHODS: ANZCTR was systematically searched from inception (2005) to 31 March 2017 for trials with proxy-reported endpoints. Primary and secondary endpoints for each trial retrieved by the search were individually coded (proxy-reported: yes/no), and trials with confirmed proxy-reported endpoints were included in the analysis. RESULTS: Of 13,666 registered trials, 469 (3.4%) included a proxy-reported endpoint (867 individual proxy-reported endpoints in total: 62% family member proxy, 22% health professional). Proxy endpoint inclusion did not significantly increase over time (r = 0.18, p = 0.59). Mental health (11.5%), stroke (10.3%) and neurological (8.3%) trials had the highest proportion of trials using proxies. Of the 469 trials, 123 (26.2%) studies involved paediatric patients. DISCUSSION: Proxy-reported endpoints are included in a small but notable number of studies, which may indicate other types of outcomes are used for patients unable to self-report, or that these patients are under-researched.
AIMS: A proxy is someone other than a patient who reports a patient's outcomes as if they are the patient. Due to known discordance with patient reports, proxies are often not recommended in clinical trials; however, proxies may be needed in certain research contexts. We aimed to identify and describe trials registered on the Australian New Zealand Clinical Trials Registry (ANZCTR) with proxy-reported endpoints. METHODS: ANZCTR was systematically searched from inception (2005) to 31 March 2017 for trials with proxy-reported endpoints. Primary and secondary endpoints for each trial retrieved by the search were individually coded (proxy-reported: yes/no), and trials with confirmed proxy-reported endpoints were included in the analysis. RESULTS: Of 13,666 registered trials, 469 (3.4%) included a proxy-reported endpoint (867 individual proxy-reported endpoints in total: 62% family member proxy, 22% health professional). Proxy endpoint inclusion did not significantly increase over time (r = 0.18, p = 0.59). Mental health (11.5%), stroke (10.3%) and neurological (8.3%) trials had the highest proportion of trials using proxies. Of the 469 trials, 123 (26.2%) studies involved paediatric patients. DISCUSSION: Proxy-reported endpoints are included in a small but notable number of studies, which may indicate other types of outcomes are used for patients unable to self-report, or that these patients are under-researched.
Entities:
Keywords:
Clinical trial endpoint; Clinical trial registration; Outcome measures; Proxy-reported outcomes; Quality of life
Authors: Jessica K Roydhouse; Matthew L Cohen; Henrik R Eshoj; Nadia Corsini; Emre Yucel; Claudia Rutherford; Katarzyna Wac; Allan Berrocal; Alyssa Lanzi; Cindy Nowinski; Natasha Roberts; Angelos P Kassianos; Veronique Sebille; Madeleine T King; Rebecca Mercieca-Bebber Journal: Qual Life Res Date: 2021-07-12 Impact factor: 4.147