A Survey to Assess the Current Status of Structured Benefit-Risk Assessment in the Global Drug and Medical Device Industry

European Medicines Agency (2011), Work package 2 report: applicability of current tools and processes for regulatory benefit‐risk assessment, In: Benefit‐risk methodology project. Report No.: EMA/549682/2010. http://www.ema.europa.eu/docs/en_GB/document_library/Report/2010/10/WC500097750.pdf.

US Food and Drug Administration (2013). Structured approach to benefit-risk assessment in drug regulatory decision-making. Draft PDUFA V implementation plan. Fiscal Years 2013–2017. http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM329758.pdf.

Angelis A, Phillips L. Structured decision-making in drug regulation at the FDA and EMA. Br J Clin Pharmacol. 2021;87(7):395–405.

Article  PubMed  Google Scholar 

Levitan BS, Andrews EB, Gilsenan A, Ferguson J, Noel RA, Coplan PM, Mussen F. Application of the BRAT framework to case studies: observations and insights. Clin Pharmacol Ther. 2011;89(2):217–24.

Article  CAS  PubMed  Google Scholar 

US Food and Drug Administration (2019). Factors to consider when making benefit-risk determinations in medical device premarket approval and De Novo classifications. Guidance for industry and food and drug administration staff. https://www.fda.gov/media/99769/download.

PROTECT Benefit‐Risk Group (2013). Benefit‐risk integration and representation. http://www.imi-protect.eu/benefitsRep.shtml.

Keeney RL, Raiffa H, Meyer RF. Decisions with multiple objectives: preferences and value trade-offs. Cambridge: Cambridge University Press; 1993.

Book  Google Scholar 

Hammond JS, Keeney RL, Raiffa H. Smart Choices: a practical guide to making better decisions. Boston, MA: Harvard University Press; 1999.

Google Scholar 

Mt-Isa S, Ouwens M, Robert V, Gebel M, Schacht A, Hirsch I. Structured Benefit-risk assessment: a review of key publications and initiatives on frameworks and methodologies. Pharm Stat. 2015;15(4):324–32.

Article  PubMed  Google Scholar 

CIOMS Working Group XII—Benefit-Risk Balance for Medicinal Products (2019). Benefit-Risk Balance for Medicinal Products—Update of CIOMS IV Proposal to form a CIOMS Expert Working Group. https://cioms.ch/wp-content/uploads/2019/10/CIOMS-WGXII-Concept-Note-_12Feb2019.docx.

European Medicines Agency (2020). EMA Regulatory Science to 2025—Strategy reflection. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/ema-regulatory-science-2025-strategic-reflection_en.pdf.

US Food and Drug Administration (2018). Benefit‐risk assessment in drug regulatory decision-making—Draft PDUFA VI Implementation Plan. Fiscal Years 2018–2022. Available at https://www.fda.gov/media/112570/download.

IMI PREFER (2021). CHMP & EUnetHTA parallel Scientific Advice. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/chmp-eunethta-parallel-scientific-advice-qualification-framework-points-consider-method-selection_en.pdf.

Rodriguez L, Renz C, Edwards B, Wang WB. trends and recent progress in benefit-risk assessment planning for medical products and devices. In: Wang W, Munsaka M, Buchanan J, Li J, editors. Drug safety and benefit-risk evaluation. Boca Raton: Chapman and Hall/CRC; 2021. https://doi.org/10.1201/9780429488801.

Chapter  Google Scholar 

Smith MY, van Til J, DiSantostefano R, Hauber BA, Marsh K. Quantitative benefit-risk assessment: state of the practice within industry. Ther Innov Regul Sci. 2021;55:415–25.

Article  PubMed  Google Scholar 

Kürzinger ML, Douarin L, Uzun I, El-Haddad C, Hurst W, Juhaeri J, Tcherny-Lessenot S. Structured benefit–risk evaluation for medicinal products: review of quantitative benefit–risk assessment findings in the literature. Ther Adv Drug Saf. 2020;11:1–12.

Article  Google Scholar 

Wolka A, Warner M, Bullok K, Wang J, Radawski C, Noel R. Incorporation of a benefit–risk assessment framework into the clinical overview of marketing authorization applications. Ther Innov Regul Sci. 2016;50(1):130–4.

Article  PubMed  Google Scholar 

Warner MR, Wolka AM, Noel RA. Implementing benefit–risk assessment for the periodic benefit–risk evaluation report. Ther Innov Regul Sci. 2016;50(3):342–6.

Article  PubMed  Google Scholar 

Smith MY, Benattia I, Strauss C, Bloss L, Jiang Q. Structured benefit–risk assessment across the product lifecycle: practical considerations. Ther Innov Regul Sci. 2017;51(4):501–8.

Article  PubMed  Google Scholar 

FDA guidance—Benefit-risk assessment for new drug and biological products, guidance for industry. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/benefit-risk-assessment-new-drug-and-biological-products

CIOMS Working group XII: Benefit-risk balance for medicinal products. CIOMS Working Group report. https://cioms.ch/working-groups/working-group-xii/

Tervonen T, Veldwijk J, Payne K, Ng X, Levitan B, Lackey LG, Marsh K, Thokala P, Pignatti F, Donnelly A, Ho M. Quantitative benefit-risk assessment in medical product decision making: a good practices report of an ISPOR Task Force. Value Health. 2023;26(4):449–60.

Article  PubMed  Google Scholar 

Lackey LG, Ng X, Veldwijk J, Thokala P, Levitan B, Payne K, Ho M, Tervonen T. Illustrating emerging good practices for quantitative benefit-risk assessment: a hypothetical case study of systemic biologic treatments for plaque psoriasis. Value Health. 2023;26(4):519–27.

Article  PubMed  Google Scholar 

PREFER Recommendations—Why, when and how to assess and use patient preferences in medical product decision-making. https://www.imi-prefer.eu/recommendations/

Sullivan T, Zorenyi G, Feron J, Smith M, Nord M. A structured benefit-risk assessment operating model for investigational medicinal products in the pharmaceutical industry. Ther Innov Regul Sci. 2023;57(4):849–64.

Article  PubMed  PubMed Central  Google Scholar 

Colopy MW, Gakava L, Chen C. Planning benefit-risk assessments using visualizations. Ther Innov Regul Sci. 2023;57(6):1123–35.

Article  PubMed  Google Scholar 

Norton JD. A longitudinal model and graphic for benefit-risk analysis, with case study. Ther Innov Regul Sci. 2011;45(6):741–7.

Google Scholar 

Crowe BJ, Xia HA, Berlin JA, Watson DJ, Shi H, Lin SL, Kuebler J, Schriver RC, Santanello NC, Rochester G, Porter JB, Oster M, Mehrotra DV, Li Z, King EC, Harpur ES, Hall DB. Recommendations for safety planning, data collection, evaluation and reporting during drug, biologic and vaccine development: a report of the safety planning, evaluation, and reporting team. Clin Trials. 2009;6(5):430–40.

Article  PubMed  Google Scholar 

Hendrickson BA, Wang W, Ball G, Bennett D, Bhattacharyya A, Fries M, Kuebler J, Kurek R, McShea C, Tremmel L. Aggregate safety assessment planning for the drug development life-cycle. Ther Innov Regul Sci. 2021;55:717–32.

Article  PubMed  Google Scholar 

FDA—Center for Devices and Radiological Health (2016). Patient Preference Information—Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling, Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-preference-information-voluntary-submission-review-premarket-approval-applications

FDA Guidance—Patient-focused drug development: collecting comprehensive and representative input. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/patient-focused-drug-development-collecting-comprehensive-and-representative-input

Kaul S, Stockbridge N, Butler J. Benefit-risk tradeoffs in assessment of new drugs and devices. Circulation. 2020;142:1974–88.

Article  PubMed  Google Scholar 

Ho MP, Gonzalez JM, Lerner HP, Neuland CY, Whang JM, McMurry-Heath M, Hauber AB, Irony T. Incorporating patient-preference evidence into regulatory decision making. Surg Endosc. 2015;29(10):2984–93.

Article  PubMed  Google Scholar 

Gebel M, Fechtner S, Tamm M, Steffen Jeske S, Fiala-Buskies S, Krüsmann F, Pap AF (2022). BReasy: An R shiny application for structured benefit-risk assessment. https://github.com/Bayer-Group/BIC-BReasy

Comments (0)

No login
gif