TY - JOUR
T1 - Towards a novel clinical outcome assessment for systemic lupus erythematosus
T2 - first outcomes of an international taskforce
AU - Connelly, Kathryn
AU - Eades, Laura E.
AU - Koelmeyer, Rachel L.
AU - Ayton, Darshini R
AU - Golder, Vera
AU - Kandane-Rathnayake, Rangi K.
AU - Gregory, Kate
AU - Brunner, Hermine
AU - Burke, Laurie
AU - Arnaud, Laurent
AU - Askanase, Anca Dinu
AU - Aranow, Cynthia B
AU - Vital, Ed
AU - Pons-Estel, Guillermo
AU - Dantata, Khadija
AU - Andersen, Jeanette
AU - Cornet, Alain
AU - Buie, Joy
AU - Sun, Ying
AU - Tanaka, Yoshiya
AU - Simon, Lee
AU - Lahoud, Youmna
AU - Friedman, Alan
AU - Kalunian, Kenneth
AU - Zuraw, Qing
AU - Werth, Victoria
AU - Garces, Sandra
AU - Morand, Eric F.
AU - the TRM-SLE Consortium
N1 - Publisher Copyright:
© 2023. Springer Nature Limited.
PY - 2023/9
Y1 - 2023/9
N2 - Systemic lupus erythematosus (SLE) is a disease of high unmet therapeutic need. The challenge of accurately measuring clinically meaningful responses to treatment has hindered progress towards positive outcomes in SLE trials, impeding the approval of potential new therapies. Current primary end points used in SLE trials are based on legacy disease activity measures that were neither specifically designed for the clinical trial context, nor developed according to contemporary recommendations for clinical outcome assessments (COAs), such as that substantial patient input should be incorporated into their design. The Treatment Response Measure for SLE (TRM-SLE) Taskforce is a global collaboration of SLE clinician-academics, patients and patient representatives, industry partners and regulatory experts, established to realize the goal of developing a new COA for SLE clinical trials. The aim of this project is a novel COA designed specifically to measure treatment effects that are clinically meaningful to patients and clinicians, and intended for implementation in a trial end point that supports regulatory approval of novel therapeutic agents in SLE. This Consensus Statement reports the first outcomes of the TRM-SLE project, including a structured process for TRM-SLE development.
AB - Systemic lupus erythematosus (SLE) is a disease of high unmet therapeutic need. The challenge of accurately measuring clinically meaningful responses to treatment has hindered progress towards positive outcomes in SLE trials, impeding the approval of potential new therapies. Current primary end points used in SLE trials are based on legacy disease activity measures that were neither specifically designed for the clinical trial context, nor developed according to contemporary recommendations for clinical outcome assessments (COAs), such as that substantial patient input should be incorporated into their design. The Treatment Response Measure for SLE (TRM-SLE) Taskforce is a global collaboration of SLE clinician-academics, patients and patient representatives, industry partners and regulatory experts, established to realize the goal of developing a new COA for SLE clinical trials. The aim of this project is a novel COA designed specifically to measure treatment effects that are clinically meaningful to patients and clinicians, and intended for implementation in a trial end point that supports regulatory approval of novel therapeutic agents in SLE. This Consensus Statement reports the first outcomes of the TRM-SLE project, including a structured process for TRM-SLE development.
UR - http://www.scopus.com/inward/record.url?scp=85168789787&partnerID=8YFLogxK
U2 - 10.1038/s41584-023-00993-7
DO - 10.1038/s41584-023-00993-7
M3 - Review Article
C2 - 37433880
AN - SCOPUS:85168789787
SN - 1759-4790
VL - 19
SP - 592
EP - 602
JO - Nature Reviews Rheumatology
JF - Nature Reviews Rheumatology
IS - 9
ER -