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Transforming clinical trials in rheumatology: towards patient-centric precision medicine

Nature Reviews Rheumatology volume 16, pages 590–599 (2020)Cite this article

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Abstract

Despite the success of targeted therapies in the treatment of inflammatory arthritides, the lack of predictive biomarkers drives a ‘trial and error’ approach to treatment allocation, leading to variable and/or unsatisfactory responses. In-depth characterization of the synovial tissue in rheumatoid arthritis, as well as psoriatic arthritis and spondyloarthritis, is bringing new insights into the diverse cellular and molecular features of these diseases and their potential links with different clinical and treatment-response phenotypes. Such progress raises the tantalizing prospect of improving response rates by matching the use of specific agents to the cognate target pathways that might drive particular disease subtypes in specific patient groups. Innovative patient-centric, molecular pathology-driven clinical trial approaches are needed to achieve this goal. Whilst progress is clearly being made, it is important to emphasize that this field is still in its infancy and there are a number of potential barriers to realizing the premise of patient-centric clinical trials.

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Fig. 1: Evolution of trial design in rheumatoid arthritis.
Fig. 2: Emergent biomarker-driven trial designs.
Fig. 3: Biomarker-driven trial designs.
Fig. 4: Biomarker-driven adaptive trial design.

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Authors and Affiliations

  1. Centre for Experimental Medicine & Rheumatology, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, London, UK

    Costantino Pitzalis

  2. CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University School of Medicine, Cardiff University, Cardiff, UK

    Ernest H. S. Choy

  3. Division of Musculoskeletal and Dermatological Sciences, University of Manchester, Manchester, UK

    Maya H. Buch

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C.P., E.H.S.C. and M.H.B. made substantial contributions to discussion of the content and researched data for the article; C.P. wrote the article and C.P., E.H.S.C. and M.H.B. reviewed and edited the manuscript before submission.

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Correspondence to Costantino Pitzalis.

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Nature Reviews Rheumatology thanks A. Jorgensen and the other, anonymous, reviewer(s) for their contribution to the peer review of this work.

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Pitzalis, C., Choy, E.H.S. & Buch, M.H. Transforming clinical trials in rheumatology: towards patient-centric precision medicine. Nat Rev Rheumatol 16, 590–599 (2020). https://doi.org/10.1038/s41584-020-0491-4

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