Arthritis Clinical Trials at a Crossroad

Pope, Janet; Thorne, John; Karsh, Jacob; Keystone, Edward; Bennett, Linda

doi:10.3899/jrheum.140717

Cited by 4 publications

(2 citation statements)

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“…In a 2015 editorial in The Journal of Rheumatology , Pope et al . [1] wrote:Over the last 2 decades, we have seen advances in the clinical management of rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, systemic sclerosis, vasculitis, and systemic lupus erythematosus. Yet trial designs and entry criteria required for drug development have not kept pace with medical care and thus no longer reflect patients seen in typical rheumatology practices in Canada, the United States and Western Europe [1].…”

Section: Discussionmentioning

confidence: 99%

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Early phase and adaptive design clinical trials in rheumatoid arthritis: a systematic review of early phase trials

Pickles

Christensen

Tam

et al. 2018

Rheumatology Advances in Practice

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Objective Adaptive designs can enable highly sophisticated and efficient early phase trials, but the clinical inference from these trials is surrounded by complexity, and currently there is a paucity but steadily increasing amount of use of these designs in all fields of medicine. We aim to review early phase trials in RA to discover those that have used adaptive designs and benchmark trial characteristics. Methods From an OVID search for journal articles reporting the results of early phase trials in rheumatology, 35 studies were found, with 9 subsequently excluded; 11 were added from manual searches and 19 from searching the references. Study characteristics were extracted from the 56 papers (describing 62 trials), including the number of arms, number of patients, the primary outcome and when it was measured. Result One early phase trial using an adaptive design was found. The benchmark early phase trial in RA is a phase II double-blinded randomized trial, with four arms (one control and three intervention), each with 34 patients, and ACR20 measured at 16 weeks as the primary outcome. Conclusion The one adaptive design reviewed here, and a simulation study found in the search, both indicate that adaptive designs can be applied to early phase trials in RA. We have described the benchmark, which the efficiency of early phase trials using an adaptive design needs to exceed. These efficient designs could drive down numbers required, time for data collection and thus cost. Changes have been suggested, but more needs to be done.

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Section: Discussionmentioning

confidence: 99%

“…Randomized controlled trials are the gold standard in evidence-based medicine. However, many clinical trials struggle to meet the recruitment target and time line [1]. This issue is not unique to rheumatology; randomized controlled trials are resource intensive in terms of time, personnel, finance and the available patient pool.…”

Section: Introductionmentioning

confidence: 99%