Synonimous with Benefit-Risk Assessment. Formal methods to assess benefits and harms of interventions, used for medical decision making in clinical settings, making recommendations in CPG development, in approval process of medicines and devices.
Gail MH, Costantino JP, Bryant J, Croyle R, Freedman L, Helzlsouer K, Vogel V: Weighing the risks and benefits of tamoxifen treatment for preventing breast cancer. J Natl Cancer Inst 1999;91:1829-46. doi: 10.1093/jnci/91.21.1829 PMID: 10547390 URL: https://pubmed.ncbi.nlm.nih.gov/10547390/
MCDAについて広く解説されている。Keeney & Raiffaの方法の実例の解説がある。
Thokala P, Devlin N, Marsh K, Baltussen R, Boysen M, Kalo Z, Longrenn T, Mussen F, Peacock S, Watkins J, Ijzerman M: Multiple Criteria Decision Analysis for Health Care Decision Making-An Introduction: Report 1 of the ISPOR MCDA Emerging Good Practices Task Force. Value Health 2016;19:1-13. PMID: 26797229 URL: https://pubmed.ncbi.nlm.nih.gov/26797229/
上記のThokala Pの報告の後半に相当し、ISPORの公式の報告として出版されている。
Marsh K, IJzerman M, Thokala P, Baltussen R, Boysen M, Kalo Z, Lonngren T, Mussen F, Peacock S, Watkins J, Devlin N, ISPOR Task Force: Multiple Criteria Decision Analysis for Health Care Decision Making-Emerging Good Practices: Report 2 of the ISPOR MCDA Emerging Good Practices Task Force. Value Health 2016;19:125-37. PMID: 27021745 URL: https://pubmed.ncbi.nlm.nih.gov/27021745/
効果推定値の不確実性から正味の益の不確実性を推定する方法。
Wen S, Zhang L, Yang B: Two approaches to incorporate clinical data uncertainty into multiple criteria decision analysis for benefit-risk assessment of medicinal products. Value Health 2014;17:619-28. PMID: 25128056 URL: https://pubmed.ncbi.nlm.nih.gov/25128056
GRADEワーキンググループの益と害のバランス=正味の益に関するコンセプトペーパー。Certainty of net benefitという考えは、USPSTFと同じで、推奨の強さを決める主要要素。Appendixに具体的な計算法が記載されている。
Alper BS, Oettgen P, Kunnamo I, Iorio A, Ansari MT, Murad MH, Meerpohl JJ, Qaseem A, Hultcrantz M, Schunemann HJ, Guyatt G, GRADE Working Group: Defining certainty of net benefit: a GRADE concept paper. BMJ Open 2019;9:e027445. PMID: 31167868 URL: https://pubmed.ncbi.nlm.nih.gov/31167868/
定量的ベネフィット・リスク分析に関するISPORの公式の報告。実際的な手順を解説。
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;26:449-460. doi: 10.1016/j.jval.2022.12.006 PMID: 37005055 URL: https://pubmed.ncbi.nlm.nih.gov/37005055/
Ho M, Saha A, McCleary KK, Levitan B, Christopher S, Zandlo K, Braithwaite RS, Hauber AB, Medical Device Innovation Consortium’s Patient Centered Benefit-Risk Steering Committee: A Framework for Incorporating Patient Preferences Regarding Benefits and Risks into Regulatory Assessment of Medical Technologies. Value Health 2016;19:746-750. PMID: 27712701 URL: https://pubmed.ncbi.nlm.nih.gov/27712701/
Phillippo DM, Dias S, Welton NJ, Caldwell DM, Taske N, Ades AE: Threshold Analysis as an Alternative to GRADE for Assessing Confidence in Guideline Recommendations Based on Network Meta-analyses. Ann Intern Med 2019;170:538-546. doi: 10.7326/M18-3542 PMID: 30909295 URL: https://pubmed.ncbi.nlm.nih.gov/30909295/
Eiring Ø, Brurberg KG, Nytrøen K, Nylenna M: Rapid methods including network meta-analysis to produce evidence in clinical decision support: a decision analysis. Syst Rev 2018;7:168. doi: 10.1186/s13643-018-0829-z PMID: 30342549 URL: https://pubmed.ncbi.nlm.nih.gov/30342549/
複数のアウトカムに対する介入の効果を対照と比較して、集約し、正味の益 net benefitを計算するには、効果推定値の尺度(スケール)をすべてのアウトカムに対して同じにする必要があります。治癒・非治癒のような二値変数 binary/dichotomous variableの場合は、前回の投稿で解説したように、リスク差を用いれば共通の尺度になり、絶対効果を表し、直線関係が維持されます。しかし、連続変数アウトカムも取り扱う場合は、何らかの方法で変換する必要があります。
文献: Rutten-van Molken M, Leijten F, Hoedemakers M, Tsiachristas A, Verbeek N, Karimi M, Bal R, de Bont A, Islam K, Askildsen JE, Czypionka T, Kraus M, Huic M, Pitter JG, Vogt V, Stokes J, Baltaxe E, SELFIE consortium: Strengthening the evidence-base of integrated care for people with multi-morbidity in Europe using Multi-Criteria Decision Analysis (MCDA). BMC Health Serv Res 2018;18:576. doi: 10.1509/jmkr.41.4.392.47020 PMID: 30041653
SELFIEプロジェクトのウェブサイトSUSTAINABLE INTEGRATED CARE MODELS FOR MULTI-MORBIDITY DELIVERY, FINACING AND PERFOMANCE (SELFIE)
Thokala P, Devlin N, Marsh K, Baltussen R, Boysen M, Kalo Z, Longrenn T, Mussen F, Peacock S, Watkins J, Ijzerman M: Multiple Criteria Decision Analysis for Health Care Decision Making-An Introduction: Report 1 of the ISPOR MCDA Emerging Good Practices Task Force. Value Health 2016;19:1-13. PMID: 26797229
Marsh K, IJzerman M, Thokala P, Baltussen R, Boysen M, Kalo Z, Lonngren T, Mussen F, Peacock S, Watkins J, Devlin N, ISPOR Task Force: Multiple Criteria Decision Analysis for Health Care Decision Making-Emerging Good Practices: Report 2 of the ISPOR MCDA Emerging Good Practices Task Force. Value Health 2016;19:125-37. PMID: 27021745
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;26:449-460. doi: 10.1016/j.jval.2022.12.006 PMID: 37005055
Wen S, Zhang L, Yang B: Two approaches to incorporate clinical data uncertainty into multiple criteria decision analysis for benefit-risk assessment of medicinal products. Value Health 2014;17:619-28. PMID: 25128056
Ho M, Saha A, McCleary KK, Levitan B, Christopher S, Zandlo K, Braithwaite RS, Hauber AB, Medical Device Innovation Consortium’s Patient Centered Benefit-Risk Steering Committee: A Framework for Incorporating Patient Preferences Regarding Benefits and Risks into Regulatory Assessment of Medical Technologies. Value Health 2016;19:746-750. PMID: 27712701
Schünemann HJ, Neumann I, Hultcrantz M, Brignardello-Petersen R, Zeng L, Murad MH, Izcovich A, Morgano GP, Baldeh T, Santesso N, Cuello CG, Mbuagbaw L, Guyatt G, Wiercioch W, Piggott T, De Beer H, Vinceti M, Mathioudakis AG, Mayer MG, Mustafa R, Filippini T, Iorio A, Nieuwlaat R, Marcucci M, Coello PA, Bonovas S, Piovani D, Tomlinson G, Akl EA, GRADE Working Group: GRADE guidance 35: update on rating imprecision for assessing contextualized certainty of evidence and making decisions. J Clin Epidemiol 2022;150:225-242. doi: 10.1016/j.jclinepi.2022.07.015 PMID: 35934266
Hultcrantz M, Rind D, Akl EA, Treweek S, Mustafa RA, Iorio A, Alper BS, Meerpohl JJ, Murad MH, Ansari MT, Katikireddi SV, Östlund P, Tranæus S, Christensen R, Gartlehner G, Brozek J, Izcovich A, Schünemann H, Guyatt G: The GRADE Working Group clarifies the construct of certainty of evidence. J Clin Epidemiol 2017;87:4-13. doi: 10.1016/j.jclinepi.2017.05.006 PMID: 28529184