Table 5Outcome measures and study eligibility criteria

Key Questions, Outcomes of Interest, and Specific MeasuresStudy Eligibility Criteria
KQ 1/KQ 2:a
Efficacy/effectiveness
KQ 1:
  • Disease activityb
  • Radiographic joint damage
  • Remission
KQ 2:
  • Functional capacity
  • Quality of life
  • Patient-reported symptoms
Study Design
  • Head-to-head double-blind RCTs
  • Systematic reviews
  • Prospective, controlled cohort studies
Minimum Study Duration
  • RCT—3 months
  • Observational—3 months
Study Population
  • Ages 19 or older
  • Patients with RA
Sample Size
  • RCT N ≥100
  • Observational N ≥100
KQ 3:
Harms, tolerability, adherence, adverse effects
Study Design
  • Head-to-head double-blind RCTs
  • Systematic reviews
  • Observational studies, prospective and retrospective
Minimum Study Duration
  • RCT—3 months
  • Observational—3 months
Study Population
  • Ages 19 or older
  • Patients with RA
Sample Size
  • RCT N ≥100
  • Observational N ≥100
KQ 4:
Benefits and harms in subgroups based on stage, history of prior therapy, demographics, concomitant therapies, comorbidities
Study Design
  • Head-to-head double-blind RCTs
  • Systematic reviews
  • Observational studies
Minimum Study Duration
  • RCT—3 months
  • Observational—3 months
Study Population
  • Ages 19 or older
  • Patients with RA
Sample Size
  • RCT N ≥100
  • Observational N ≥100

KQ = Key Question; N = number of subjects enrolled (i.e. sample size); RA = rheumatoid arthritis; RCT = randomized controlled trial

a

We divided the assessment of efficacy/effectiveness into two KQs based on two groups of outcomes: those addressing disease activity, radiographic measures, and remission (KQ 1) and those addressing functional capacity, quality of life, and other patient reported symptoms (KQ 2). We did this to group measures that are based on more objective measures under KQ 1 and those that are based more on subjective patient reported outcomes under KQ 2.

b

Disease activity reflects the overall RA activity. Measures of disease activity, such as the American College of Rheumatology 20 percent response (ACR 20), 50 percent response (ACR 50), 70 percent response (ACR 70), and the Disease Activity Score (DAS), include assessment of some or all of the following: the number of swollen and tender joints, the patient’s global assessment of his/her disease activity, the physician’s global assessment of the patient’s disease activity, patient’s pain score, patient’s physical function score, and acute phase reactants (C-reactive protein). Appendix G provides additional details about these measures.

From: Methods

Cover of Drug Therapy for Rheumatoid Arthritis in Adults: An Update
Drug Therapy for Rheumatoid Arthritis in Adults: An Update [Internet].
Comparative Effectiveness Reviews, No. 55.
Donahue KE, Jonas DE, Hansen RA, et al.

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