Session Information
Session Title: Rheumatoid Arthritis-Clinical Aspects III: Outcome Measures, Socioeconomy, Screening, Biomarkers in Rheumatoid Arthritis
Session Type: Abstract Submissions (ACR)
Background/Purpose:
Rheumatoid Arthritis (RA) is a chronic autoimmune disease, affecting around 1.3 million people in the US. A patient-centred focus is increasingly advocated by healthcare authorities, making assessments of the impact of novel treatments on patient-reported outcomes (PRO) important. PRO data should be interpreted in context of whether it represents a clinically meaningful change. Minimal important difference/minimal clinically important difference (MID/MCID) provide the benchmarks to define responders in clinical trials. The objective of the study was to review MIDs/MCIDs for PROs commonly applied in clinical trials in patients with RA.
Methods:
Articles were identified in MEDLINE, EMBASE, and PsycINFO databases using pre-defined search terms related to RA, differential diagnosis, key PROs and MIDs/MCIDs. Reference lists of identified papers were manually searched. FDA/EMA regulatory guidance and reports, PRO user manuals, PRO Labels and PROQOLID were searched to identify information on MIDs/MCIDs. The following PROs in RA were included in the review: Health Assessment Questionnaire Disability Index (HAQ-DI), 0-100mm pain visual analogue scale (VAS), 0-100mm patient global assessment of disease activity (PtGA), Medical Outcomes Study Short-Form Health Survey (SF-36v2), EuroQol (EQ-5D-3L), Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue), and Work Productivity and Activity Impairment Questionnaire: Rheumatoid Arthritis (WPAI:RA). MIDs/MCIDs were prioritised as follows: Available in: 1) RA, 2) related inflammatory conditions, 3) general population. If MIDs/MCIDs were sparse, the next approach was taken.
Results:
The search identified 1019 abstracts; 32 abstracts met inclusion and exclusion criteria. From the reference list search, an additional 17 abstracts met the criteria, totalling 49 articles for in-depth review. MIDs/MCIDs were identified for an RA population for the HAQ-DI (≥0.22 units), Pain (VAS) (-11.9mm for minimal improvement), PtGA (-9.1mm for minimal improvement), SF-36v2 (change of 2.0 to 3.8 points for Physical Component Summary (PCS) score and 2.7 to 4.6 points for Mental Component Summary (MCS) score), EQ-5D-3L (0.06 to 0.20 [improvement]), FACIT-Fatigue (3.5 to 4.1 points). No MID/MCID information for the WPAI:RA was identified. Most articles focused on the MIDs/MCID’s for change over time within a treatment group. MID’s/MCID’s used in clinical trials in RA were not always consistent with the literature: often applied responder definitions for HAQ-DI ranged from 0.22 to 0.3 units, was 10mm for Pain (VAS) and PtGA, was 3 to 4 points for FACIT-Fatigue, and was a change of 2.5 points for the PCS and MCS of the SF-36v2.
Conclusion:
MID/MCID information is available for the core PRO measures in RA. However, some gaps in information remains for the newer PRO measures in RA. Further research to explore patients’ perspectives of what constitutes important differences in symptoms, Health-Related Quality of Life, and functioning, and information on important differences between treatment groups, may be beneficial to extrapolate meaning from clinical trial data to the real-world experience of patients.
Disclosure:
H. Kitchen,
Adelphi Values Ltd – paid consultant to conduct literature review,
5;
B. B. Hansen,
Novo Nordisk A/S,
3;
L. Abetz,
Adelphi Values Ltd – paid consultant to conduct literature review,
5;
L. Højbjerre,
Novo Nordisk A/S,
3;
M. Strandberg-Larsen,
Novo Nordisk A/S,
3.
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