厚生労働省科学研究班開発
患者報告アウトカム(Patient-Reported Outcome:PRO)
使用についてのガイダンス集
［ 目次 ］
臨床試験のためのPatient-Reported Outcome（PRO）使用ガイダンス
3. 参考文献・参考書籍
諸外国のPRO関連ガイダンスの系統的レビューより得られた主要な参考文献・書籍を以下に示す。
| 項目 | 著者名 | タイトル | 書誌事項 |
| 1.1- 1.2 |
Basch E. | The Missing Voice of Patients in Drug-Safety Reporting | N Engl J Med. 2010; 362(10): 865-9.［PubMed］ |
| 下妻 晃二郎 | QOL評価研究の歴史と展望 | 行動医学研究. 2015; 21(1): 4-7. | |
| 2.1 | Acquadro C. | Incorporating the patient's perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001. | Value Health. 2003; 6(5): 522-31.［PubMed］ |
| 2.2 | European Medicines Agency. | Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man. The use of patient-reported outcome (PRO) measures in oncology studies. 2016. | https://www.ema.europa.eu/en/appendix-2-guideline-evaluation-anticancer-medicinal-products-man-use-patient-reported-outcome-pro-measures-oncology-studies-scientific-guideline |
| ICH Assembly. | ICH Reflection paper Proposed ICH Guideline Work to Advance Patient Focused Drug Development. | https://admin.ich.org/sites/default/files/2021-06/ICH_ReflectionPaper_PFDD_FinalRevisedPostConsultation_2021_0602.pdf | |
| Mayo NE. | Montreal Accord on Patient-Reported Outcomes (PROs) use series e Paper 2: terminology proposed to measure what matters in health. | J Clin Epidemiol. 2017; 89: 119-24.［PubMed］ | |
| US. Food and Drug Administration. | FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient's Voice in Medical Product Development and Regulatory Decision Making. | https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical | |
| US. Food and Drug Administration. | Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 2009. | https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-reported-outcome-measures-use-medical-product-development-support-labeling-claims | |
| 国際医薬経済・アウトカム研究学会日本部会 ワーキンググループ 監訳. | 患者報告アウトカム（Patient-Reported Outcome: PRO）の測定法：医薬品/医療機器における適応申請のための方法. | https://ispor-jp.org/wp-content/uploads/2022/10/patient-reported-outcome-pro.pdf | |
| 2.3 | Walton MK. | Clinical Outcome Assessments: Conceptual Foundation-Report of the ISPOR Clinical Outcomes Assessment - Emerging Good Practices for Outcomes Research Task Force. | Value Health. 2015; 18(6): 741-52.［PubMed］ |
| Mokkink LB. | COSMIN Risk of Bias checklist for systematic reviews of Patient-Reported Outcome Measures. | Qual Life Res. 2018; 27(5): 1171-9.［PubMed］ | |
| Prinsen CA. | COSMIN guideline for systematic reviews of patient-reported outcome measures. | Qual Life Res. 2018; 27(5): 1147-57.［PubMed］ | |
| Reeve BB. | ISOQOL recommends minimum standards for patient-reported outcome measures used in patient-centered outcomes and comparative effectiveness research. | Qual Life Res. 2013; 22(8): 1889-905.［PubMed］ | |
| Terwee CB. | COSMIN methodology for evaluating the content validity of patient-reported outcome measures: A Delphi study. | Qual Life Res. 2018; 27(5): 1159-70.［PubMed］ | |
| 2.4 | Fayers P & Machin D. | Quality of Life: The Assessment, Analysis and Interpretation of Patient-reported Outcomes | 3rd edition. Wiley, 2016 |
| ピーター・M・フェイヤーズ 著（福原俊一 監訳）. | QOL評価学 測定、解析、解釈のすべて. | 中山書店．2005 | |
| 2.5 | O'Brien BC. | Standards for reporting qualitative research: a synthesis of recommendations. | Acad Med. 2014; 89(9): 1245-51.［PubMed］ |
| Pope C & Mays N (eds.) | Qualitative research in health care 3rd edition. | BMJ Books, Blackwell Publishing, 2006 | |
| Rothrock NE. | Developing a Valid Patient-Reported Outcome Measure. | Clin Pharmacol Ther. 2011; 90(5): 737-42.［PubMed］ | |
| Tong A. | Consolidated criteria for reporting qualitative research (COREQ): a 32-item checklist for interviews and focus groups. | Int J Qual Health Care. 2007; 19(6): 349-57.［PubMed］ | |
| 2.6 | Anfray C. | Reflection paper on copyright, patient reported outcome instruments and their translations. | Health Qual Life Outcomes. 2018; 16(1): 224.［PubMed］ |
| Wild D. | ISPOR Task Force for Translation and Cultural Adaptation. Principles of Good Practice for the Translation and Cultural Adaptation Process for Patient-Reported Outcomes (PRO) Measures: report of the ISPOR Task Force for Translation and Cultural Adaptation. | Value Health. 2005; 8(2): 94-104.［PubMed］ | |
| 2.7 | Byrom B, Muehlhausen W. | Electronic Patient-Reported Outcome Measures: An Implementation Handbook For Clinical Research. | Amazon, 2018 |
| Coons SJ. | Recommendations on evidence needed to support measurement equivalence between electronic and paper-based patient-reported outcome (PRO) measures: ISPOR ePRO Good Research Practices Task Force report. | Value Health. 2009; 12(4): 419-29.［PubMed］ | |
| Ly JJ. | Training on the Use of Technology to Collect Patient-Reported Outcome Data Electronically in Clinical Trials: Best Practice Recommendations from the ePRO Consortium. | Ther Innov Regul Sci. 2019; 53(4): 431-40.［PubMed］ | |
| Matza LS. | Pediatric Patient-Reported Outcome Instruments for Research to Support Medical Product Labeling: Report of the ISPOR PRO Good Research Practices for the Assessment of Children and Adolescents Task Force. | Value Health. 2013; 16(4): 461-79.［PubMed］ | |
| Morel T. | Measuring what matters to rare disease patients - reflections on the work by the IRDiRC taskforce on patient-centered outcome measures. | Orphanet J Rare Dis. 2017; 12(1): 171.［PubMed］ | |
| Wailoo AJ. | Mapping to Estimate Health-State Utility from Non-Preference-Based Outcome Measures: An ISPOR Good Practices for Outcomes Research Task Force Report. | Value Health. 2017; 20(1): 18-27.［PubMed］ | |
| 白岩健，船越大，村澤秀樹，下妻晃二郎，斎藤信，福田敬. | 医療経済評価におけるマッピングとMAPS 声明について. | 保健医療科学 2018; 67(4): 422-6. | |
| 下妻晃二郎. | 医療の効率性と公平性（総論） | 日内会誌. 2014; 103(5): 1203-9. | |
| 2.8 | Brundage M. | Patient-reported outcomes in randomized clinical trials: development of ISOQOL reporting standards. | Qual Life Res, 2013; 22(6): 1161-75.［PubMed］ |
| Calvert M. | Reporting of Patient-Reported Outcomes in Randomized Trials: The CONSORT-PRO extension. | JAMA. 2013;27;309(8):814-22.［PubMed］ | |
| Calvert M. | Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols: The SPIRIT-PRO Extension. | JAMA. 2018; 319(5): 483-94.［PubMed］ | |
| 2.9 | Bottomley A. | Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards. | Lancet Oncol. 2016; 17(11): e510-e514.［PubMed］ |
| Coens C. | International standards for the analysis of quality-of-life and patient-reported outcome endpoints in cancer randomised controlled trials: recommendations of the SISAQOL Consortium. | Lancet Oncol. 2020; 21(2): e83-e96.［PubMed］ | |
| Fiero MH. | Demystifying the estimand framework: a case study using patient-reported outcomes in oncology. | Lancet Oncol. 2020; 21(10): e488-e494.［PubMed］ | |
| 2.10 | King MT. | A point of minimal important difference (MID): A critique of terminology and methods. | Expert Rev Pharmacoecon Outcomes Res. 2011; 11(2): 171-84.［PubMed］ |
| Revicki D. | Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. | J Clin Epidemiol. 2008; 61(2): 102-9. ［PubMed］ | |
| Cocks K. | Evidence-based guidelines for interpreting change scores for the European Organisation for the Research and Treatment of Cancer Quality of Life Questionnaire Core 30. | Eur J Cancer 2012; 48(11): 1713-21.［PubMed］ | |
| Wyrwich KW. | Methods for interpreting change over time in patient-reported outcome measures. | Qual Life Res. 2013; 22(3): 475-83.［PubMed］ | |
| 2.11 | Wilson H. | Beyond study participants: a framework for engaging patients in the selection or development of clinical outcome assessments for evaluating the benefits of treatment in medical product development. | Qual Life Res. 2018; 27(1): 5-16.［PubMed］ |
| Facey K, Hansen HP, and Single A (eds.) | Patient involvement in health technology assessment (Developing Patient-Reported and Relevant Outcome Measures). | Adis, Spting Nature.2017 | |
| 2.12 | Rivera SC. | Ethical Considerations for the Inclusion of Patient-Reported Outcomes in Clinical Research: The PRO Ethics Guidelines. | JAMA. 2022; 327(19): 1910-9.［PubMed］ |
| その他 | Banerjee AK. | Patient-reported outcome measures in safety event reporting: PROSPER consortium guidance. | Drug Saf. 2013; 36(12): 1129-49.［PubMed］ |
| Sajobi TT. | Scoping review of response shift methods: current reporting practices and recommendations. | Qual Life Res. 2018; 27(5): 1133-46.［PubMed］ |