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Minimal clinically important difference, low disease activity state, and patient acceptable symptom state:
Florence Tubach1, George A Wells, Philippe Ravaud
1Département d'Epidémiologie, Biostatistique et Recherche Clinique, Groupe Hospitalier Bichat-Claude Bernard (AP-HP), INSERM U738, Faculté Xavier Bichat (Université Paris 7), Paris, France.
The Journal of Rheumatology
|October 6, 2005
Summary
Establishing minimal clinically important difference (MCID) and low disease activity state (LDAS) as clinical trial targets is crucial. Methodological challenges in defining these thresholds require standardization for reliable patient outcome assessment.
Area of Science:
- Rheumatology
- Clinical Trial Methodology
- Patient-Reported Outcomes
Background:
- Minimal clinically important difference (MCID) and low disease activity state (LDAS) are established treatment targets in clinical trials.
- Numerous methodological issues persist regarding the definition and application of these targets.
Purpose of the Study:
- To address key methodological challenges in defining MCID and LDAS thresholds.
- To discuss the impact of these challenges on clinical trial outcomes and patient assessments.
Main Methods:
- Review and discussion of existing methodological issues concerning MCID and LDAS threshold determination.
- Consideration of criteria-based versus composite criteria, LDAS versus patient acceptable symptom state (PASS), and statistical approaches.
Main Results:
- Significant methodological questions remain regarding threshold definition, including the use of individual criteria versus composite measures.
- The distinction between LDAS and PASS, optimal question wording, statistical methods, and confounding factors require further investigation.
Conclusions:
- Standardization of methods for determining MCID and LDAS thresholds is essential.
- Recommendations for threshold determination should be data-driven, incorporate expert opinion, and potentially be endorsed by consensus groups like OMERACT.