Regulatory requirements and optimization of multiple criteria decision analysis to quantify the benefit-risk assessment of medical devices

References

• Kaule S, Bock A, Dierke A, et al. Medical Device Regulation and current challenges for the implementation of new technologies. Curr Dir Biomed Eng. 2020;6(3):334–337.
   Google Scholar
• Fu B, Li X, Scott J, et al. A new framework to address challenges in quantitative benefit-risk assessment for medical products. Contemp Clin Trials. 2020;95:106073.
   PubMed Web of Science ®Google Scholar
• White paper: benefit-risk determination: a quantitative approach (RQM+). 2022. [cited 12 Mar 2023]. Available from: https://resources.rqmplus.com/benefit-risk-analysis-white-paper-download
   Google Scholar
• Arlegui H, Bollaerts K, Salvo F, et al. Benefit–risk assessment of vaccines part I: a systematic review to identify and describe studies about quantitative benefit–risk models applied to vaccines. Drug Saf. 2020;43(11):1089–1104.
   PubMed Web of Science ®Google Scholar
• Coplan PM, Noel RA, Levitan BS, et al. Development of a framework for enhancing the transparency, reproducibility and communication of the benefit–risk balance of medicines. Clin Pharmacol Ther. 2011;89(2):312–315.
   PubMed Web of Science ®Google Scholar
• Sun S, Heske S, Mercadel M, et al. Predicting regulatory product approvals using a proposed quantitative version of FDA’s benefit–risk framework to calculate net-benefit score and benefit–risk ratio. Ther Innov Regul Sci. 2021;55(1):129–137.
   PubMed Web of Science ®Google Scholar
• Colopy MW, Damaraju CV, He W, et al. Benefit-risk evaluation and decision making: some practical insights[J]. Ther Innov Regul Sci. 2015;49(3):425–433.
   PubMed Web of Science ®Google Scholar
• Mt-Isa S, Wang N, Hallgreen CE, et al. Review of methodologies for benefit and risk assessment of medication. London (UK): Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium; 2013.
   Google Scholar
• Smith MY, van Til J, Disantostefano RL, et al. Quantitative benefit–risk assessment: state of the practice within industry. Ther Innov Regul Sci. 2021;55(2):415–425.
   PubMed Web of Science ®Google Scholar
• Mt-Isa S, Hallgreen CE, Wang N, et al. Balancing benefit and risk of medicines: a systematic review and classification of available methodologies. Pharmacoepidemiol Drug Saf. 2014;23(7):667–678.
   PubMed Web of Science ®Google Scholar
• The Global Harmonization Task Force. GHTF SG5/N2R8: clinical evaluation (Obsolete). 2007. [cited 12 Mar 2023]. Available from: https://www.imdrf.org/sites/default/files/docs/ghtf/archived/sg5/technical-docs/ghtf-sg5-n2r8-2007-clinical-evaluation-070501.pdf
   Google Scholar
• International Medical Device Regulators Forum. IMDRF GRRP WG/N47FINAL: essential principles of safety and performance of medical devices and IVD medical devices. 2018. [cited 12 Mar 2023]. Available from: https://www.imdrf.org/documents/essential-principles-safety-and-performance-medical-devices-and-ivd-medical-devices
   Google Scholar
• International Medical Device Regulators Forum. IMDRF MDCE WG/N56FINAL: clinical evaluation. 2019. [cited 12 Mar 2023]. Available from: https://www.imdrf.org/documents/clinical-evaluation .
   Google Scholar
• Directive 2007/47/EC of the European Parliament and of the Council of 5 September 2007 amending Council Directive 90/385/EEC on the approximation of the laws of the Member States relating to active implantable medical devices. (Obsolete). 2007. [cited 12 Mar 2023]. Available from: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32007L0047
   Google Scholar
• Regulation (EU) 2017/745 of the European Parliament and of the Council of 5 April 2017 on medical devices. MDR. 2017. [cited 12 Mar 2023]. Available from: https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=OJ:L:2017:117:TOC
   Google Scholar
• European Commission, MEDDEV. Clinical evaluation: a guide for manufacturers and notified bodies under directives 93/42/EEC and 90/385/EEC. 2016. [cited 12 Mar 2023]. Available from: https://ec.europa.eu/docsroom/documents/17522
   Google Scholar
• Center for Devices and Radiological Health. Guidance for industry and Food and Drug Administration Staff: Factors to consider when making benefit-risk determinations in medical device premarket approval and de novo classifications. 2019. [cited 12 Mar 2023]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/factors-consider-when-making-benefit-risk-determinations-medical-device-premarket-approval-and-de
   Google Scholar
• Center for devices and radiological health. guidance for investigational device exemption sponsors, sponsor-investigators and food and drug administration staff: factors to consider when making benefit-risk determinations for medical device investigational device exemptions. 2017. [cited 12 Mar 2023]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/factors-consider-when-making-benefit-risk-determinations-medical-device-investigational-device; .
   Google Scholar
• Center for devices and radiological health. guidance for industry and food and drug administration staff: benefit-risk factors to consider when determining substantial equivalence in premarket notifications (510(k)) with different technological characteristics. 2018. [cited 12 Mar 2023]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/benefit-risk-factors-consider-when-determining-substantial-equivalence-premarket-notifications-510k
   Google Scholar
• Center for devices and radiological health. guidance for industry and food and drug administration staff: consideration of uncertainty in making benefit-risk determinations in medical device premarket approvals, de novo classifications, and humanitarian device exemptions. 2019. [cited 12 Mar 2023]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/consideration-uncertainty-making-benefit-risk-determinations-medical-device-premarket-approvals-de
   Google Scholar
• National Medical Products Administration. Decree No. 14: “technical guidelines for medical device clinical evaluation”. (Obsolete) 2015. [cited 12 Mar 2023]. Available from: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20150519120001314.html
   Google Scholar
• National medical products administration. Decree No. 18: “Basic principles of medical device safety and performance. 2020. [cited 12 Mar 2023]. Available from: https://www.nmpa.gov.cn/ylqx/ylqxggtg/ylqxzhdyz/20200310172701477.html
   Google Scholar
• National medical products administration. Decree No. 73: “technical guidelines for medical device clinical evaluation.” Available from: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20210928170338138.html; 2021. [cited 12 Mar. 2023].
   Google Scholar
• National Medical Products Administration. Decree No. 79: “Notice by the national medical products administration on guidelines for medical device benefit-risk assessment, registration, and technical review.“ [cited 12 Mar 2023]. 2019 Available from: https://www.nmpa.gov.cn/xxgk/ggtg/qtggtg/20191115170501683.html; .
   Google Scholar
• European Medicines Agency, Benefit-risk methodology project work Package 2 report: applicability of current tools and processes for regulatory benefit-risk assessment. 2010 [cited 12 Mar 2023]. Available from: https://www.ema.europa.eu/en/documents/report/benefit-risk-methodology-project-work-package-2-report-applicability-current-tools-processes_en.pdf
   Google Scholar
• Keeney RL, Raiffa H. Decisions with multiple objectives: preferences and value trade-Offs. Cambridge UK: Cambridge University Press; 1993.
   Google Scholar
• Leong J, Salek S, Walker S. Benefit-risk assessment of medicines. London: ADIS Press, Springer International; 2015. p. 77–81.
   Google Scholar
• Mussen F, Salek S, Walker S. A quantitative approach to benefit-risk assessment of medicines - part 1: the development of a new model using multi-criteria decision analysis. Pharmacoepidemiol Drug Saf. 2007;16(Suppl 1):S2–S15.
   PubMedGoogle Scholar
• Thokala P, Devlin N, Marsh K, et al. Multiple criteria decision analysis for health care decision making–An introduction. Value Health. 2016;19(1):1–13.
   PubMed Web of Science ®Google Scholar
• Marsh K, IJzerman M, Thokala P, et al. Multiple criteria decision analysis for health care decision making–emerging good practices. Value Health. 2016;19(2):125–137.
   PubMed Web of Science ®Google Scholar
• Marsh K, Lanitis T, Neasham D, et al. Assessing the value of health care interventions using multi-criteria decision analysis: a review of the literature. Pharmacoeconomics. 2014;32:345–365.
   PubMed Web of Science ®Google Scholar
• Broekhuizen H, Groothuis-Oudshoorn CG, van Til JA, et al. A review and classification of approaches for dealing with uncertainty in multi-criteria decision analysis for healthcare decisions. Pharmacoeconomics. 2015;33:445–455.
   PubMed Web of Science ®Google Scholar
• Jiang Q, He W, editors. Benefit-risk assessment methods in medical product development: bridging qualitative and quantitative assessments. New York: CRC Press; 2017.
   Google Scholar
• European medicines agency, benefit-risk methodology project work package 1 report: description of the current practice of benefit-risk assessment for centralised procedure products in the EU regulatory network. 2011 [cited 12 Mar 2023]. Available from: https://www.ema.europa.eu/en/documents/report/benefit-risk-methodology-project-work-package-1-report-description-current-practice-benefit-risk_en.pdf
   Google Scholar
• Raftery J, Young A, Stanton L, et al. Clinical trial metadata: defining and extracting metadata on the design, conduct, results and costs of 125 randomised clinical trials funded by the national institute for health research health technology assessment programme. Health Technol Assess. 2015;19(11):1–138.
   Web of Science ®Google Scholar
• Neugebauer EAM, Rath A, Antoine SL, et al. Specific barriers to the conduct of randomised clinical trials on medical devices. Trials. 2017;18(1):427.
   PubMedGoogle Scholar
• Páez A, Rovers M, Hutchison K, et al. Beyond the RCT: when are randomized trials unnecessary for new therapeutic devices, and what should we do instead? Ann Surg. 2022;275(2):324–331.
   PubMed Web of Science ®Google Scholar
• MDCG 2020-6. Regulation (EU) 2017/745: clinical evidence needed for medical devices previously CE marked under Directives 93/42/EEC or 90/385/EEC A guide for manufacturers and notified bodies2020 [cited 12 Mar 2023]. Available from: https://health.ec.europa.eu/system/files/2020-09/md_mdcg_2020_6_guidance_sufficient_clinical_evidence_en_0.pdf
   Google Scholar
• Bernard A, Vaneau M, Fournel I, et al. Methodological choices for the clinical development of medical devices. Med Devices (Auckl). 2014;7:325–334.
   PubMedGoogle Scholar
• Agapova M, Devine EB, Bresnahan BW, et al. Applying quantitative benefit-risk analysis to aid regulatory decision making in diagnostic imaging: methods, challenges, and opportunities. Acad Radiol. 2014;21(9):1138–1143.
   PubMed Web of Science ®Google Scholar