Table 5.
Selected Patient-reported Outcomes
| Outcome Measure | Components | Recall Period | Year Published | MCID | Approved (Agency) | AECOPD Validation |
|---|---|---|---|---|---|---|
| Symptom/activity scales | ||||||
| EXACT-PRO (130) | Questions: 14 | 1 d | 2010 | Undefined | Yes (FDA, EMA) | Yes |
| Domains: breathlessness, cough and sputum, chest symptoms | ||||||
| Scale: 0 (asymptomatic) to 100 (severely symptomatic) | ||||||
| E-RS (131) | Questions: 11 | 1 d | 2014 | 1–3 units, varies by domain | Yes (FDA, EMA) | Yes |
| Domains: breathlessness, cough and sputum, chest symptoms | ||||||
| Scale: 0 (asymptomatic) to 40 (severely symptomatic) | ||||||
| MMRC* (131) | Questions: 1 | Undefined | 1952 | Undefined | Yes (FDA, EMA) | No |
| Domains: undefined | ||||||
| Scale: grade 1 (breathless with strenuous exercise) to 5 (breathless with minimal activity) | ||||||
| Health-related QoL | ||||||
| SGRQ-C (95) | Questions: 50 | 4 wk, 3 mo, 1 yr | 1992 | 4 units | Yes (FDA) | No |
| Domains: symptoms, activity, impact of disease on daily life | ||||||
| Scale: 0 (no impairment) to 100 (completely impaired) | ||||||
| CRQ (132) | Questions: 20 | 2 wk | 1987 | 0.5 per domain | No | No |
| Domains: dyspnea, emotional function, fatigue, and mastery | ||||||
| Scale: 1 (severe impairment) to 7 (no impairment) per domain | ||||||
| SF-36 (133) | Questions: 36 | 1 wk, 4 wk | 1992 | Undefined | Yes (FDA) | Yes |
| Domains: physical and mental subscales: physical function, mental health, energy, health perception, physical role limitation, mental role limitation, social function, and pain | ||||||
| Scale: 0 (negative health) to 100 (positive health) | ||||||
| CAT (131) | Questions: 8 | Undefined | 2009 | 1–3 | Yes (FDA) | Yes |
| Domains: undefined | ||||||
| Scale: 0 (no impact) to 40 (severe impact) | ||||||
| Multidimensional | ||||||
| Modified BODE (134, 135) | Questions: 4 areas | N/A | 2004 | Undefined | Yes (FDA, EMA) | No |
| Domains: FEV1 (% predicted), 6-MWT, dyspnea scale score, BMI | ||||||
| Scale: 0 (best) to 10 (worst) | ||||||
| PROactive (98) | Questions: D-PPAC: 30, C-PPAC: 35 | 7 d | 2014 | Undefined | No | Yes |
| Domains: amount of activity, difficulty of activity (performed daily or in clinic) | ||||||
| Scale: Yet to be established |
Definition of abbreviations: 6-MWT = 6-minute-walk test; AECOPD = acute exacerbation of chronic obstructive pulmonary disease; BMI = body mass index; BODE = body mass index, airflow obstruction, dyspnea, and exercise; CAT = COPD Assessment Test; COPD = chronic obstructive pulmonary disease; C-PPAC = Clinical Visit PROactive Physical Activity in COPD; CRQ = Chronic Respiratory Questionnaire; D-PPAC = Daily PROactive Physical Activity in COPD; EMA = European Medicines Agency; E-RS = Evaluating Respiratory Symptoms; EXACT-PRO = The Exacerbations of Chronic Pulmonary Disease Tool–Patient-reported Outcome; FDA = U.S. Food and Drug Administration; MCID = minimal clinically important difference; MMRC = Modified Medical Research Council; N/A = not applicable; QoL = quality of life; SF-36 = 36-item Short-Form Health Survey; SGRQ = St. George’s Respiratory Questionnaire; SGRQ-C = COPD-specific SGRQ.
There are several characteristics that the FDA takes into account when reviewing a new or modified patient-reported outcome: concepts being measured, number of items, conceptual framework of the instrument, medical condition for intended use, population for intended use, data collection method, administration mode, response options, recall period, scoring, weighting of items or domains, format, respondent burden, and translation or cultural adaptive availability (136).
FDA classified as activity scale but also considered in symptom scale.