Document Detail

Creative trial design in RA: optimizing patient outcomes.
MedLine Citation:
PMID:  23381561     Owner:  NLM     Status:  Publisher    
The rheumatology community has witnessed remarkable advances in the management of rheumatoid arthritis (RA) made possible by the development of highly effective biologic DMARDs. Robust randomized controlled trials of clinical efficacy, equipped with validated outcome measures, ensured these therapies could enter the clinical arena and thus substantially improve patient outcomes. Current management principles, which follow a 'treat-to-target' paradigm, advocate tight control of disease activity with the aim of achieving clinical remission. However, efficacy trials are not yet aligned with this approach, hampering patient recruitment. This impediment and the usual approach of inclusion of previously failed treatment arms (to protect methodological concerns) is prompting reappraisal of RA trial design and the consideration of more pragmatic studies that reflect real-life practice. In addition, the aspirations of the rheumatology community to strive for personalized medicine means innovative approaches to trial design are needed to complement the efficacy trial. This Review appraises the current trial landscape and provides insights and key concepts from other fields such as oncology as to the potential utility (as well as the limitations) of pragmatic trial designs such as adaptive trials and biomarker-driven trials.
Maya H Buch; Sue Pavitt; Mahesh Parmar; Paul Emery
Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2013-2-05
Journal Detail:
Title:  Nature reviews. Rheumatology     Volume:  -     ISSN:  1759-4804     ISO Abbreviation:  Nat Rev Rheumatol     Publication Date:  2013 Feb 
Date Detail:
Created Date:  2013-2-5     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101500080     Medline TA:  Nat Rev Rheumatol     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
NIHR Leeds Musculoskeletal Biomedical Research Unit, Division of Rheumatic & Musculoskeletal Disease, University of Leeds, Chapel Allerton Hospital, Chapeltown Road, Leeds LS7 4SA, UK.
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