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Adaptive Trial Designs in Rheumatology: Report from the OMERACT Special Interest Group
Tim Pickles1,2, Rieke Alten1,2, Maarten Boers1,2
1From 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.
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.
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