Current myositis clinical trials and tribulations.
Didem Can TrabulusVictoria WerthJulie J PaikJin-Kyun ParkMerrilee NeedhamIngrid E LundbergLisa Christopher-StinePublished in: Annals of the rheumatic diseases (2024)
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.
Keyphrases
- clinical trial
- interstitial lung disease
- end stage renal disease
- patient reported
- myasthenia gravis
- ejection fraction
- newly diagnosed
- phase iii
- chronic kidney disease
- systemic sclerosis
- machine learning
- peritoneal dialysis
- prognostic factors
- study protocol
- single cell
- double blind
- oxidative stress
- deep learning
- randomized controlled trial
- electronic health record
- open label
- idiopathic pulmonary fibrosis
- soft tissue