References
- Makady A, de Boer A, Hillege H, et al. What is real-world data? A review of definitions based on literature and stakeholder interviews. Value Health. 2017;20(7):858–865.
- Berger ML, Sox H, Willke RJ, et al. Good practices for real-world data studies of treatment and/or comparative effectiveness: recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making. Pharmacoepidemiol Drug Saf. 2017;26(9):1033–1039.
- Flaherty KT, Le DT, Lemery S. Tissue-agnostic drug development. Am Soc Clin Oncol Educ Book. 2017;37:222–230.
- Franklin JM, Glynn RJ, Martin D, et al. Evaluating the use of nonrandomized real-world data analyses for regulatory decision making. Clin Pharmacol Ther. 2019;105(4):867–877.
- Makady A, Ham RT, de Boer A, et al. Policies for use of real-world data in health technology assessment (HTA): a comparative study of six HTA agencies. Value Health. 2017;20(4):520–532.
- Beaulieu-Jones BK, Finlayson SG, Yuan W, et al. Examining the use of real-world evidence in the regulatory process. Clin Pharmacol Ther. 2020;107(4):843–852.
- Di Maio M, Perrone F, Conte P. Real-world evidence in oncology: opportunities and limitations. Oncologist. 2020;25(5):e746–e52.
- Wu J, Wang C, Toh S, et al. Use of real-world evidence in regulatory decisions for rare diseases in the United States—current status and future directions. Pharmacoepidemiol Drug Saf. 2020;29(10):1213–1218.
- Ramagopalan SV, Wasiak R. Life after COVID-19: R WE going to help? J Comp Eff Res. 2020;9(8):525–526.
- ICMRA meeting: COVID-19 real-world evidence and observational studies. 2021; [cited 2022 Feb 23]; Available from: https://www.icmra.info/drupal/en/covid-19/10may2021.
- COVID-19 research database; [cited 2022 Feb 23]. Available from: https://covid19researchdatabase.org/.
- Observational Health Data Sciences and Informatics (OHDSI); [cited 2022 Feb 23]; Available from: https://www.ohdsi.org/.
- EMA Conditional Marketing Authorisation; [cited 2022 Feb 23]; Available from: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/conditional-marketing-authorisation.
- FDA Emergency Use Authorization; [cited 2022 Feb 23]; Available from: https://www.fda.gov/emergency-preparedness-and-response/mcm-legal-regulatory-and-policy-framework/emergency-use-authorization.
- Renoux C, Azoulay L, Suissa S. Biases in evaluating the safety and effectiveness of drugs for the treatment of COVID-19: designing real-world evidence studies. Am J Epidemiol. 2021;190(8):1452–1456.
- Annual Real World Evidence Conference: applying lessons learned from RWE in the time of COVID-19 to the future. 2020; [cited 2022 Feb 23]; Available from: https://healthpolicy.duke.edu/events/annual-real-world-evidence-conference-applying-lessons-learned-rwe-time-covid-19-future.
- Zou KH, Li JZ, Imperato J, et al. Harnessing real-world data for regulatory use and applying innovative applications. J Multidiscip Healthc. 2020;13:671–679.
- Hersh WR, Weiner MG, Embi PJ, et al. Caveats for the use of operational electronic health record data in comparative effectiveness research. Med Care. 2013;51(8 Suppl 3):S30–S7.
- Cave A, Kurz X, Arlett P. Real-world data for regulatory decision making: challenges and possible solutions for Europe. Clin Pharmacol Ther. 2019;106(1):36–39.
- Welsing PM, Oude Rengerink K, Collier S, et al. Series: pragmatic trials and real world evidence: Paper 6. Outcome measures in the real world. J Clin Epidemiol. 2017;90:99–107.
- Orsini LS, Berger M, Crown W, et al. Improving transparency to build trust in real-world secondary data studies for hypothesis testing-why, what, and how: recommendations and a road map from the real-world evidence transparency initiative. Value Health. 2020;23(9):1128–1136.
- Lim JCW, Gau CS, Kang JH, et al. Use of real world endpoints to drive next generation healthcare – viewpoints of regulators, payors, clinicians and patients in Asia Pacific region. Presented at Virtual ISPOR Asia Pacific 2020, 14–16 September; 2020.
- Dreyer NA, Velentgas P, Westrich K, et al. The GRACE checklist for rating the quality of observational studies of comparative effectiveness: a tale of hope and caution. J Manag Care Spec Pharm. 2014;20(3):301–308.
- Duke Margolis Center for Health Policy. A framework for regulatory use of real-world evidence. 2017; [cited 2022 Feb 23]; Available from: https://healthpolicy.duke.edu/sites/default/files/2020-08/rwe_white_paper_2017.09.06.pdf.
- Miksad RA, Abernethy AP. Harnessing the power of real-world evidence (RWE): a checklist to ensure regulatory-grade data quality. Clin Pharmacol Ther. 2018;103(2):202–205.
- US Food and Drug Administration. Framework for the FDA's real-world evidence program. 2018; [cited 2022 Feb 23]; Available from: https://www.fda.gov/media/120060/download.
- Duke Margolis Center for Health Policy. Determining real-world data’s fitness for use and the role of reliability. 2019; [cited 2022 Feb 23]; Available from: https://healthpolicy.duke.edu/sites/default/files/2019-11/rwd_reliability.pdf.
- European Medicines Agency. EMA regulatory science to 2025: strategic reflection. 2020. [cited 2022 Feb 23]; Available from: https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/ema-regulatory-science-2025-strategic-reflection_en.pdf.
- US Food and Drug Administration. Real-world data: assessing registries to support regulatory decision-making for drug and biological products guidance for industry (draft guidance). 2021; [cited 2022 Feb 23]; Available from: https://www.fda.gov/media/154449/download.
- US Food and Drug Administration. Considerations for the use of real-world data and realworld evidence to support regulatory decision-making for drug and biological products (draft guidance). 2021; [cited 2022 Feb 23]; Available from: https://www.fda.gov/media/154714/download.
- Center for Drug Evaluation. Guideline on using real-world data to generate real-world evidence (trial). 2021; [cited 2021 Jul 1]; Available from: https://redica.com/wp-content/uploads/NMPA_-Attachment_-_Guiding-Principles-of-Real-World-Data-Used-to-Generate-Real-World-Evidence-Trial_.pdf.
- Center for Drug Evaluation. Guideline on using real-world evidence to support drug research & development and evaluation (trial). 2020; [cited 2021 Jul 1]; Available from: https://www.nmpa.gov.cn/yaopin/ypggtg/ypqtgg/20200107151901190.html.
- Taiwan Food and Drug Administration. Real-world evidence supports the basic considerations of drug research and development. FDA Drug No. 1091405905. 2020; [cited 2021 May 17]; Available from: https://www.fda.gov.tw/tc/newsContent.aspx?cid=3&id=26255.
- Pharmaceuticals and Medical Devices Agency. Basic principles on utilization of registry for applications; [cited 2021 Jul 1]; Available from: https://www.pmda.go.jp/files/000240810.pdf.
- REAL World Data in Asia for Health Technology Assessment in Reimbursement (REALISE) working group. 2020; [cited 2022 Feb 23]; Available from: https://hiper.nus.edu.sg/wp-content/uploads/2020/12/REALISE-Full-guidance_updated-20201101.pdf.
- Lou J, Kc S, Toh KY, et al. Real-world data for health technology assessment for reimbursement decisions in Asia: current landscape and a way forward. Int J Technol Assess Health Care. 2020;36(5):474–480.
- Petracci F, Ghai C, Pangilinan A, et al. Use of real-world evidence for oncology clinical decision making in emerging economies. Future Oncol. 2021;17(22):2951–2960.
- Nishioka K, Makimura T, Ishiguro A, et al. Evolving acceptance and use of RWE for regulatory decision making on the benefit/risk assessment of a drug in Japan. Clin Pharma and Therapeutics. 2022;111(1):35–43.
- Pharmaceuticals and Medical Devices Agency. Points to consider for ensuring the reliability in utilization of registry data for applications. 2018; [cited 2022 Feb 23]; Available from: https://www.pmda.go.jp/files/000240811.pdf.
- Pharmaceuticals and Medical Devices Agency. Points to consider for ensuring the reliability in conducting post-marketing database surveillance. 2018; [cited 2022 Feb 23]; Available from: https://www.pmda.go.jp/files/000223003.pdf.
- Yamaguchi M, Inomata S, Harada S, et al. Establishment of the MID-NET(®) medical information database network as a reliable and valuable database for drug safety assessments in Japan. Pharmacoepidemiol Drug Saf. 2019;28(10):1395–1404.
- The Japanese Ministry of Health, Labor, and Welfare. Ministerial ordinance on standards for investigation and testing after manufacture and sales of pharmaceuticals. 2018; [cited 2022 Feb 23]; Available from: https://www.pmda.go.jp/files/000220721.pdf.
- Yasunaga H. Protection of personal information in real-world data in Japan. Ann Clin Epidemiol. 2020;2(1):1–2.
- Center for Drug Evaluation. Technical guidelines (trial) for real-world research and support for drug research and development and review of children. 2020; [cited 2021 1 July]; Available from: https://www.nmpa.gov.cn/directory/web/nmpa/xxgk/ggtg/qtggtg/20200901104448101.html.
- Center for Medical Device Evaluation (NMPA). Guideline on using real-world evidence to support medical device evaluation (Trial). 2020; [cited 2021 2 July]; Available from: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20201126090030150.html.
- Taiwan Food and Drug Administration. Guidelines for clinical research using electronic medical record data. 2020; [cited 2021 17 July]; Available from: https://www.fda.gov.tw/TC/newsContent.aspx?cid=3&id=26558.
- Huang LY, Gau CS. Lessons learned from the reimbursement policy for immune checkpoint inhibitors and real-world data collection in Taiwan. Int J Technol Assess Health Care. 2020;37(1):e26.
- Xie J, Wu EQ, Wang S, et al. Real-world data for healthcare research in China: call for actions. Value Health Reg Issues. 2022;27:72–81.
- Jin F, Yao C, Yan X, et al. Gap between real-world data and clinical research within hospitals in China: a qualitative study. BMJ Open. 2020;10(12):e038375.
- Mhatre SK, Machado RJM, Ton TGN, et al. Real-world progression-free survival as an endpoint in advanced non-small-cell lung cancer: replicating atezolizumab and docetaxel arms of the oak trial using data derived from electronic health records. 2022. p. 41. Available from: https://doi.org/10.1101/2022.05.02.22274571.
- Hsieh CY, Su CC, Shao SC, et al. Taiwan’s national health insurance research database: past and future. Clin Epidemiol. 2019;11:349–358.
- Wang X, Birch S, Zhu W, et al. Coordination of care in the Chinese health care systems: a gap analysis of service delivery from a provider perspective. BMC Health Serv Res. 2016;16(1):571.
- Sheller MJ, Edwards B, Reina GA, et al. Federated learning in medicine: facilitating multi-institutional collaborations without sharing patient data. Sci Rep. 2020;10(1):12598.
- Heads of Medicines Agencies and European Medicines Agency, 2019. HMA-EMA Joint Big Data Taskforce phase II report: ‘Evolving Data-Driven Regulation’; [cited 2022 Feb 23]; Available from: https://www.ema.europa.eu/en/documents/other/hma-ema-joint-big-data-taskforce-phase-ii-report-evolving-data-driven-regulation_en.pdf.
- Healthcare IT News. RWE Alliance aims to boost policies and practices around real-world evidence. 2021; [cited 2022 Feb 23]; Available from: https://www.healthcareitnews.com/news/rwe-alliance-aims-boost-policies-and-practices-around-real-world-evidence.