Correspondence on 'Current myositis clinical trials and tribulations' by Saygin et al

Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11110%2F24%3A10481947" target="_blank" >RIV/00216208:11110/24:10481947 - isvavai.cz</a>
Result on the web
<a href="https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=iV_CAAR60m" target="_blank" >https://verso.is.cuni.cz/pub/verso.fpl?fname=obd_publikace_handle&handle=iV_CAAR60m</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1136/ard-2024-225751" target="_blank" >10.1136/ard-2024-225751</a>

Result language
angličtina
Original language name
Correspondence on 'Current myositis clinical trials and tribulations' by Saygin et al
Original language description
We read the viewpoint titled 'current myositis clinical trials and tribulations' with great interest. We congratulate the authors for addressing critical patient-centred and science-forward concerns in idiopathic inflammatory myopathies (IIMs) clinical trials. These valuable insights highlight current pitfalls and the need for more effective engagement of patients, physicians and trial methodology experts in the development of clinical trial designs for complex rare diseases. Lengthy placebo arms, trial recruitment and equity/diversity challenges, outdated classification criteria, and outcome measures that insufficiently demonstrate therapeutic responsiveness in large subsets of patients, such as skin-predominant involvement, cripple the current landscape of IIM trials, levying a toll on a rare diseases' premium resource: patient life-years.
Czech name
—
Czech description
—

Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
—
OECD FORD branch
30226 - Rheumatology

Publication year
2024
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

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