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

Kód výsledku v 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>

Výsledek na webu

<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>

Jazyk výsledku

angličtina

Název v původním jazyce

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

Popis výsledku v původním jazyce

We read the viewpoint titled &apos;current myositis clinical trials and tribulations&apos; 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 Lengthy placebo arms, trial recruitment and equity/diversity challenges, outdated classification criteria, and outcome measures that insufficiently demonstrate therapeutic responsiveness in large subsets of patients, such as skin-predominant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&apos; premium resource: patient life-years.

Název v anglickém jazyce

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

Popis výsledku anglicky

We read the viewpoint titled &apos;current myositis clinical trials and tribulations&apos; 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 Lengthy placebo arms, trial recruitment and equity/diversity challenges, outdated classification criteria, and outcome measures that insufficiently demonstrate therapeutic responsiveness in large subsets of patients, such as skin-predominant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&apos; premium resource: patient life-years.

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30226 - Rheumatology

Projekt

—

Návaznosti

V - Vyzkumna aktivita podporovana z jinych verejnych zdroju

Rok uplatnění

2024

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Annals of the Rheumatic Diseases

ISSN

0003-4967

e-ISSN

1468-2060

Svazek periodika

83

Číslo periodika v rámci svazku

11

Stát vydavatele periodika

GB - Spojené království Velké Británie a Severního Irska

Počet stran výsledku

3

Strana od-do

e22

Kód UT WoS článku

001194569600001

EID výsledku v databázi Scopus

2-s2.0-85190147666