～臨床開発担当者のための PRO ... - jpma.or.jp

1 Patient Reported Outcomes PRO 7 Ve r 1 . 0 2016 6 .. 1 1 PRO .. 2 PRO .. 2 PRO .. 3 .. 5 2 FDA PRO .. 7 PRO CONCEPTUAL FRAMEWORK .. 7 CONTENT VALIDITY .. 8 PRO .. 9 3 PRO .. 11 .. 11 PRO .. 12 PRO .. 12 PRO .. 13 .. 20 4 PRO .. 23 .. 23 .. 24 .. 25 .. 26 .. 28 .. 29 1) PRO .. 29 2) .. 32 3) 34 4) PRO .. 36 5) PRO .. 36 6) PRO .. 38 7) .. 38 8) .. 40 .. 43 .. 46 1 Patient Reported Outcome PRO HR-QOL Health related quality of life 2015 7 2015 8 7 PRO PRO PRO measure PRO ePRO electronic PRO PRO PRO

2 PRO PRO 1 PRO 2 FDA PRO 3 PRO PRO PRO PRO 4 PRO PRO [1, 2] PRO 2 1 PRO PRO PRO QOL Quality of Life QOL QOL [3] QOL QOL Health Related QOL, HR-QOL QOL 1948 WHO [4] PRO PRO QOL FDA [5] PRO PRO 2001 2 PRO [6] PRO PRO 3 PRO 1- 1 3 1- 1 SF-36 EQ-5D VA S MPQ PainDETECT WOMAC ODI JOABPEQ BPI EORTC-QLQ FA C T-G PSQI ESS BASDAI ACR core set DAS28 COPD SGRQ AQLQ HADS BDI SDISS SF-36 : MOS(Medical Outcome Study) 36-item Short-Form health survey, EQ-5D : EuroQol 5 Dimension, VAS : Visual Analog Scale, MPQ.

3 McGill Pain Questionnaire, WOMAC : Western Ontario & McMaster Universities Osteoarthritis Index, ODI : Oswestry Disability Index, JOABPEQ : the Japanese Orthopaedic Association Back Pain Evaluation Questionnaire, BPI : Brief Pain Inventory, EORTC-QLQ : the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire, FAC T-G : Functional Assessment of Cancer-general, PSQI : Pittsburgh Sleep Quality Index, ESS : Epworth Sleepiness Scale, BASDAI : Bath Ankylosing Spondylitis Disease Activity Index, ACR core set : American College of Rheumatology core set DAS28 : Disease Activity Score 28, SGRQ : St George s Respiratory Questionnaire, AQLQ : Asthma Quality of Life Questionnaire, HADS : Hospital Anxiety and Depression scale, BDI : Beck Depression Inventory, SDISS : Sheehan Disability Scale Electronic PRO [ePRO] ePRO PRO site PRO eDiary ePRO [7] PRO 4 [6] 1.

4 PRO 2. caregiver 3. 4. FEV1 HbA1c 3,4 4 4 1 PRO ICH-E9 QOL [8] PRO 22 1 26 12 5 208 24 PRO 1- 2 24 7 6 8 PRO 1- 2 PRO 1- 2 PRO * ** PRO 112,116, 119 7 (24) VA S 821 2 (2) 239 2 (8) SF-NDI.

5 Short Form of the Nepean Dyspepsia Index 259 1 (2) 634 1 (26) A 399 6 (46) HAQ : Health Assessment Questionnaire VA S 121 1 (3) VRS : Verbal Rating Scale 114 1 (1) 625 1 (14) 449 1 (2) Total four-symptom score 131 1 (5) * 87 3 ** 208 5 2007 2013 [9] 30,012 1887 PRO FDA 2009 PRO Guidance for Industry Patient-Reported Outcome Measures[5] PRO PRO 2 PRO PRO FDA Comparative Effectiveness PRO FDA-DIA stat workshop [10] PRO Critical Path institute C-PAT H PRO [11]

6 EMA PRO 2005 Reflection Paper on the Regulatory Guidance for the use of health related Quality of Life [3] 2009 Qualification of novel methodologies for drug development [12] Guideline on the evaluation of anticancer medicinal products in man [13] 2012 PRO Appendix 2016 11 The use of patient-reported outcome (PRO) measures in oncology studies [14] PRO 3 17 [15] QOL PRO 6 Patient-focused Drug Development [16] PRO 7 2 FDA PRO 2009 12 FDA FDA Guidance for Industry PRO Measures Use in Medical Product Development to Support Labeling Claims [5] FDA PRO PRO PRO 4 PRO ISPOR(International Society for Pharmacoeconomics and Outcomes Research) Patient-Reported Outcome.

7 PRO / [17] PRO conceptual framework PRO PRO FDA PRO 1 PRO framework conceptual framework 2- 1 2- 1 PRO ISPOR Patient-Reported Outcome: PRO / [17] 8 PRO PRO 2- 2 PRO PRO 2- 2 X ISPOR Patient-Reported Outcome.

8 PRO / [17] content validity content validity PRO validation PRO 9 PRO FDA PRO FDA PRO PRO PRO PRO PRO PRO

9 FDA FDA PRO PRO 2- 1 10 2- 1 PRO (reliability) (test-retest reliability) PRO [6] (Inter-interviewer reproducibility) PRO [6] (construct validity) ( (criterion-related validity) PRO (PRO vs ) PRO (change detection))

10 11 3 PRO QOL PRO PRO 3- 1 PRO PRO PRO PRO 3- 1 PRO 2015 8 PRO Figure 4 PRO CRO 12 PRO PRO Standard Operating Procedure SOP Table 3 PRO PRO relevant PRO PRO PRO PRO PRO PRO PRO [ ] PRO ePROVIDE [ ]