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. 2024 Jun 12;83(7):826-829.
doi: 10.1136/ard-2023-224652.

Current myositis clinical trials and tribulations

Didem Saygin  1 , Victoria Werth  2 , Julie J Paik  3 , Jin Kyun Park  4 , Merrilee Needham  5 , Ingrid E Lundberg  6 , Lisa Christopher-Stine  7
Affiliations

Current myositis clinical trials and tribulations

Didem Saygin et al. Ann Rheum Dis. .

Abstract

With improved understanding of disease pathogenesis and availability of outcome measures, there has been a remarkable increase in the number of therapeutic clinical trials in idiopathic inflammatory myopathies (myositis) over the last three years reaching as many as five trials per site. These trials share similar design and inclusion/exclusion criteria resulting in a competitive clinical trial landscape in myositis. While these are exciting times for the myositis field, we have a number of concerns about the design and conduct of the myositis trials. These include competitive landscape, lengthy placebo arms, underrepresentation of minority groups among participants, use of patient reported outcome measures with limited/no data on validity in myositis, antiquated disease classification criteria, and unclear performance of the ACR/EULAR Myositis Response Criteria in skin-predominant patients despite inclusion of these patients in trials. In this viewpoint, we further discuss these concerns and offer potential solutions such as including patient perspectives in the trial design and adoption of innovative frameworks.

Keywords: Dermatomyositis; Polymyositis; Therapeutics.

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Conflict of interest statement

Competing interests: DS, JKP, MN, IEL, and LCS are members of the OMERACT Myositis Working Group. JJP received funding from NIAMS (K23AR073927); grants from Pfizer Inc, CORBUS, Kezar, Priovant and EMD Serono; royalty from Uptodate; and consulting fees from Pfizer, Kezar, Alexion, EMD Serono, Priovant and Guidepoint Inc. IEL received support from Swedish Research Council; consulting fees from Corbus Pharmaceutical, EMD Serono, ArgenX, Pfizer, Galapagos, Bristol Myers Squibb and Chugai; honoraria from Boehringer-Ingelheim; and currently holds stock in Roche and Novartis. MN received honorarium from Abcuro, Novartis, Sanofi-Aventis, Genzyme and Teva; participated on Data Safety Monitoring Board or Advisory Board for Abcuro, Teva and Sanofi-Genzyme; and received materials from Sanofi-Aventis. VW received grants from Pfizer, Corbus, CSL Behring, Priovant, Rome, Ventus and Horizon; received consulting fees from Pfizer, Janssen, Neovacs, Octapharma and CSL Behring; and University of Pennsylvania owns the copyright for the Cutaneous Dermatomyositis Disease Area and Severity Index. LCS received grants from Pfizer, Corbus, Kezar; royalties for IP related to anti-HMGCR assay from Inova Diagnostics; consulting fees from Janssen, Boehringer-Ingelheim, Mallincrodt, EMD Serono, ArgenX, Allogene, Pfizer, Horizon Therapeutics, Octopharma and NuVig; received payment for expert testimony from Bendin Sumrall and Ladner LLC, Feldman, Kleidman Coffey & Sappe LLP, Downs Ward Bender Hauptmann & Herzog, PA, Sulloway and Hollis, received support for attending meetings and travel from Octapharma and has patent with Inova Diagnostics/RDL.