Adaptive Trial Designs in Rheumatology: Report from the OMERACT Special Interest Group

Tim Pickles; Rieke Alten; Maarten Boers; Vivian Bykerk; Jared Christensen; Robin Christensen; Hubert van Hoogstraten; Lee S Simon; Lai-Shan Tam; Ernest H Choy

doi:10.3899/jrheum.181054

Adaptive Trial Designs in Rheumatology: Report from the OMERACT Special Interest Group

J Rheumatol. 2019 Oct;46(10):1406-1408. doi: 10.3899/jrheum.181054. Epub 2019 Feb 15.

Authors

Tim Pickles^{1

2}, Rieke Alten^{1

2}, Maarten Boers^{1

2}, Vivian Bykerk^{1

2}, Jared Christensen^{1

2}, Robin Christensen^{1

2}, Hubert van Hoogstraten^{1

2}, Lee S Simon^{1

2}, Lai-Shan Tam^{1

2}, Ernest H Choy^{3

4}

Affiliations

¹ From the CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University, Cardiff, UK; Schlosspark Klinik, Charité University Medicine, Berlin, Germany; Department of Epidemiology and Biostatistics; Amsterdam Rheumatology and Immunology Center; Amsterdam University Medical Centers, location VUmc, Amsterdam, the Netherlands; Department of Rheumatology, Hospital for Special Surgery, Weill Cornell Medical College; Pfizer, New York, New York; Sanofi, Bridgewater, New Jersey; SDG LLC, Cambridge, Massachusetts, USA; Rebecca McDonald Centre for Arthritis and Autoimmune Disease, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada; Musculoskeletal Statistics Unit, The Parker Institute, Bispebjerg and Frederiksberg Hospital; Department of Rheumatology, Odense University Hospital, Odense, Denmark; Division of Rheumatology, Department of Medicine and Therapeutics, and Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong.
² T. Pickles, MSc, CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University; R. Alten, MD, PhD, Schlosspark Klinik, Charité University Medicine; M. Boers, MD, PhD, MSc, Department of Epidemiology and Biostatistics, and Amsterdam Rheumatology and Immunology Center, and Amsterdam University Medical Centers VUmc; V. Bykerk, MD, FRCPC, Department of Rheumatology, Hospital for Special Surgery, Weill Cornell Medical College, and Rebecca McDonald Centre for Arthritis and Autoimmune Disease, Mount Sinai Hospital, University of Toronto; J. Christensen, MD, Pfizer; R. Christensen, PhD, MSc, Musculoskeletal Statistics Unit, The Parker Institute, Bispebjerg and Frederiksberg Hospital, and Department of Rheumatology, Odense University Hospital; H. van Hoogstraten, MD, PhD, Sanofi; L.S. Simon, MD, SDG LLC; L.S. Tam, MD, Division of Rheumatology, Department of Medicine and Therapeutics, and Faculty of Medicine, The Chinese University of Hong Kong; E.H. Choy, MD, CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University.
³ From the CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University, Cardiff, UK; Schlosspark Klinik, Charité University Medicine, Berlin, Germany; Department of Epidemiology and Biostatistics; Amsterdam Rheumatology and Immunology Center; Amsterdam University Medical Centers, location VUmc, Amsterdam, the Netherlands; Department of Rheumatology, Hospital for Special Surgery, Weill Cornell Medical College; Pfizer, New York, New York; Sanofi, Bridgewater, New Jersey; SDG LLC, Cambridge, Massachusetts, USA; Rebecca McDonald Centre for Arthritis and Autoimmune Disease, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada; Musculoskeletal Statistics Unit, The Parker Institute, Bispebjerg and Frederiksberg Hospital; Department of Rheumatology, Odense University Hospital, Odense, Denmark; Division of Rheumatology, Department of Medicine and Therapeutics, and Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong. choyeh@cardiff.ac.uk.
⁴ T. Pickles, MSc, CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University; R. Alten, MD, PhD, Schlosspark Klinik, Charité University Medicine; M. Boers, MD, PhD, MSc, Department of Epidemiology and Biostatistics, and Amsterdam Rheumatology and Immunology Center, and Amsterdam University Medical Centers VUmc; V. Bykerk, MD, FRCPC, Department of Rheumatology, Hospital for Special Surgery, Weill Cornell Medical College, and Rebecca McDonald Centre for Arthritis and Autoimmune Disease, Mount Sinai Hospital, University of Toronto; J. Christensen, MD, Pfizer; R. Christensen, PhD, MSc, Musculoskeletal Statistics Unit, The Parker Institute, Bispebjerg and Frederiksberg Hospital, and Department of Rheumatology, Odense University Hospital; H. van Hoogstraten, MD, PhD, Sanofi; L.S. Simon, MD, SDG LLC; L.S. Tam, MD, Division of Rheumatology, Department of Medicine and Therapeutics, and Faculty of Medicine, The Chinese University of Hong Kong; E.H. Choy, MD, CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University. choyeh@cardiff.ac.uk.

PMID: 30770514
DOI: 10.3899/jrheum.181054

Abstract

Objective: Adaptive trial design was developed initially for oncology to improve trial efficiency. If optimized for rheumatology, it may improve trial efficiency by reducing sample size and time.

Methods: A systematic review assessed design of phase II clinical trials in rheumatoid arthritis.

Results: Fifty-six trials were reviewed. Most trials had 4 groups (1 control and 3 intervention), with an average group size of 34 patients. American College of Rheumatology 20 measured at 16 weeks was the most commonly used primary endpoint.

Conclusion: The next step is to undertake a systematic review of adaptive designs used in early-phase trials in nonrheumatic conditions.

Keywords: ADAPTIVE TRIAL DESIGN; EARLY-PHASE CLINICAL TRIAL; OMERACT CORE OUTCOME SET; RHEUMATOID ARTHRITIS; SYSTEMATIC REVIEW.

Publication types

Systematic Review

MeSH terms

Antirheumatic Agents / therapeutic use*
Arthritis, Rheumatoid / drug therapy*
Clinical Trials as Topic*
Humans
Outcome Assessment, Health Care
Public Opinion
Research Design*
Rheumatology / methods*

Substances

Antirheumatic Agents