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Published in: BMC Medical Informatics and Decision Making 1/2015

Open Access 01-12-2015 | Research article

Estimating the value of medical treatments to patients using probabilistic multi criteria decision analysis

Authors: Henk Broekhuizen, Catharina G. M. Groothuis-Oudshoorn, A. Brett Hauber, Jeroen P. Jansen, Maarten J. IJzerman

Published in: BMC Medical Informatics and Decision Making | Issue 1/2015

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Abstract

Background

Estimating the value of medical treatments to patients is an essential part of healthcare decision making, but is mostly done implicitly and without consulting patients. Multi criteria decision analysis (MCDA) has been proposed for the valuation task, while stated preference studies are increasingly used to measure patient preferences. In this study we propose a methodology for using stated preferences to weigh clinical evidence in an MCDA model that includes uncertainty in both patient preferences and clinical evidence explicitly.

Methods

A probabilistic MCDA model with an additive value function was developed and illustrated using a case on hypothetical treatments for depression. The patient-weighted values were approximated with Monte Carlo simulations and compared to expert-weighted results. Decision uncertainty was calculated as the probability of rank reversal for the first rank. Furthermore, scenario analyses were done to assess the relative impact of uncertainty in preferences and clinical evidence, and of assuming uniform preference distributions.

Results

The patient-weighted values for drug A, drug B, drug C, and placebo were 0.51 (95 % CI: 0.48 to 0.54), 0.51 (95 % CI: 0.48 to 0.54), 0.54 (0.49 to 0.58), and 0.15 (95 % CI: 0.13 to 0.17), respectively. Drug C was the most preferred treatment and the rank reversal probability for first rank was 27 %. This probability decreased to 18 % when uncertainty in performances was not included and increased to 41 % when uncertainty in criterion weights was not included. With uniform preference distributions, the first rank reversal probability increased to 61 %. The expert-weighted values for drug A, drug B, drug C, and placebo were 0.67 (95 % CI: 0.65 to 0.68), 0.57 (95 % CI: 0.56 to 0.59), 0.67 (95 % CI: 0.61 to 0.71), and 0.19 (95 % CI: 0.17 to 0.21). The rank reversal probability for the first rank according to experts was 49 %.

Conclusions

Preferences elicited from patients can be used to weigh clinical evidence in a probabilistic MCDA model. The resulting treatment values can be contrasted to results from experts, and the impact of uncertainty can be quantified using rank probabilities. Future research should focus on integrating the model with regulatory decision frameworks and on including other types of uncertainty.
Literature
2.
go back to reference Marsh K, Lanitis T, Neasham D, Orfanos P, Caro J. Assessing the value of healthcare interventions using multi-criteria decision analysis: a review of the literature. Pharmacoeconomics. 2014;32:1–21.CrossRef Marsh K, Lanitis T, Neasham D, Orfanos P, Caro J. Assessing the value of healthcare interventions using multi-criteria decision analysis: a review of the literature. Pharmacoeconomics. 2014;32:1–21.CrossRef
3.
go back to reference Belton V, Stewart TJ. Multiple criteria decision analysis: an integrated approach. 2nd ed. Dordrecht: Kluwer Academic Publishers; 2002.CrossRef Belton V, Stewart TJ. Multiple criteria decision analysis: an integrated approach. 2nd ed. Dordrecht: Kluwer Academic Publishers; 2002.CrossRef
7.
go back to reference 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:S2–S15. doi:10.1002/pds.CrossRefPubMed 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:S2–S15. doi:10.​1002/​pds.CrossRefPubMed
11.
go back to reference Goetghebeur MM, Wagner M, Khoury H, Levitt RJ, Erickson LJ, Rindress D. Bridging health technology assessment (HTA) and efficient health care decision making with multicriteria decision analysis (MCDA): applying the EVIDEM framework to medicines appraisal. Med Decis Mak. 2012;32:376–88. doi:10.1177/0272989X11416870.CrossRef Goetghebeur MM, Wagner M, Khoury H, Levitt RJ, Erickson LJ, Rindress D. Bridging health technology assessment (HTA) and efficient health care decision making with multicriteria decision analysis (MCDA): applying the EVIDEM framework to medicines appraisal. Med Decis Mak. 2012;32:376–88. doi:10.​1177/​0272989X11416870​.CrossRef
12.
go back to reference Tony M, Wagner M, Khoury H, Rindress D, Papastavros T, Oh P, et al. Bridging health technology assessment (HTA) with multicriteria decision analyses (MCDA): field testing of the EVIDEM framework for coverage decisions by a public payer in Canada. BMC Health Serv Res. 2011;11:329. doi:10.1186/1472-6963-11-329.PubMedCentralCrossRefPubMed Tony M, Wagner M, Khoury H, Rindress D, Papastavros T, Oh P, et al. Bridging health technology assessment (HTA) with multicriteria decision analyses (MCDA): field testing of the EVIDEM framework for coverage decisions by a public payer in Canada. BMC Health Serv Res. 2011;11:329. doi:10.​1186/​1472-6963-11-329.PubMedCentralCrossRefPubMed
13.
14.
go back to reference Facey K, Boivin A, Gracia J, Hansen HP, Lo Scalzo A, Mossman J, et al. Patients’ perspectives in health technology assessment: a route to robust evidence and fair deliberation. Int J Technol Assess Health Care. 2010;26:334–40. doi:10.1017/S0266462310000395.CrossRefPubMed Facey K, Boivin A, Gracia J, Hansen HP, Lo Scalzo A, Mossman J, et al. Patients’ perspectives in health technology assessment: a route to robust evidence and fair deliberation. Int J Technol Assess Health Care. 2010;26:334–40. doi:10.​1017/​S026646231000039​5.CrossRefPubMed
16.
go back to reference MDIC PCBR project group members. A framework for incorporating information of patient preferences regarding benefit and risk into regulatory assessments of new medical technology. Medical Device Innovation Consortium; 2015. http://mdic.org/PCBR/. Accessed 30 Nov 2015. MDIC PCBR project group members. A framework for incorporating information of patient preferences regarding benefit and risk into regulatory assessments of new medical technology. Medical Device Innovation Consortium; 2015. http://​mdic.​org/​PCBR/​. Accessed 30 Nov 2015.
17.
18.
go back to reference Weernink MGM, Janus SIM, van Til J a, Raisch DW, van Manen JG, IJzerman MJ. A Systematic Review to Identify the Use of Preference Elicitation Methods in Healthcare Decision Making. Pharmaceut Med 2014. doi:10.1007/s40290-014-0059-1. Weernink MGM, Janus SIM, van Til J a, Raisch DW, van Manen JG, IJzerman MJ. A Systematic Review to Identify the Use of Preference Elicitation Methods in Healthcare Decision Making. Pharmaceut Med 2014. doi:10.​1007/​s40290-014-0059-1.
20.
go back to reference Briggs AH, Weinstein MC, Fenwick EAL, Karnon J, Sculpher MJ, Paltiel AD. Model parameter estimation and uncertainty: a report of the ISPOR-SMDM modeling good research practices task force-6. Med Decis Making. 2012;32(5);722-33. doi:10.1177/0272989X12458348.CrossRefPubMed Briggs AH, Weinstein MC, Fenwick EAL, Karnon J, Sculpher MJ, Paltiel AD. Model parameter estimation and uncertainty: a report of the ISPOR-SMDM modeling good research practices task force-6. Med Decis Making. 2012;32(5);722-33. doi:10.​1177/​0272989X12458348​.CrossRefPubMed
21.
go back to reference Claxton K. Exploring Uncertainty in Cost-Effectiveness Analysis. Pharmacoeconomics. 2008;26(9):781–98.CrossRefPubMed Claxton K. Exploring Uncertainty in Cost-Effectiveness Analysis. Pharmacoeconomics. 2008;26(9):781–98.CrossRefPubMed
22.
go back to reference Broekhuizen H, Groothuis-Oudshoorn C, van Til J, Hummel M, IJzerman M. A review and classification of approaches for dealing with uncertainty in multi-criteria decision analysis for healthcare decisions. Pharmacoeconomics. 2015;33:445–55.PubMedCentralCrossRefPubMed Broekhuizen H, Groothuis-Oudshoorn C, van Til J, Hummel M, IJzerman M. A review and classification of approaches for dealing with uncertainty in multi-criteria decision analysis for healthcare decisions. Pharmacoeconomics. 2015;33:445–55.PubMedCentralCrossRefPubMed
23.
go back to reference Hamilton M. A rating scale for depression. Neurol Neurosurg Psychiatry. 1960;23:56–62.CrossRef Hamilton M. A rating scale for depression. Neurol Neurosurg Psychiatry. 1960;23:56–62.CrossRef
24.
go back to reference Montgomery S, Asberg M. A new depression scale designed to be sensitive to change. Br J Psychiatry. 1979;134:382–9.CrossRefPubMed Montgomery S, Asberg M. A new depression scale designed to be sensitive to change. Br J Psychiatry. 1979;134:382–9.CrossRefPubMed
25.
go back to reference Hummel JM, Volz F, van Manen JG, Danner M, Dintsios CM, IJzerman MJ, et al. Using the analytic hierarchy process to elicit patient preferences. Patient. 2012;5:1–13.CrossRef Hummel JM, Volz F, van Manen JG, Danner M, Dintsios CM, IJzerman MJ, et al. Using the analytic hierarchy process to elicit patient preferences. Patient. 2012;5:1–13.CrossRef
27.
go back to reference Keeney R, Raiffa H. Decisions with multiple objectives. Cambridge: Cambridge University Press; 1976. Keeney R, Raiffa H. Decisions with multiple objectives. Cambridge: Cambridge University Press; 1976.
28.
go back to reference Holden W. Benefit-risk analysis: A brief review and proposed quantitative approaches. Drug Saf. 2003;26:853–62.CrossRefPubMed Holden W. Benefit-risk analysis: A brief review and proposed quantitative approaches. Drug Saf. 2003;26:853–62.CrossRefPubMed
29.
30.
go back to reference Lynd LD, Najafzadeh M, Colley L, Byrne MF, Willan AR, Sculpher MJ, et al. Using the incremental net benefit framework for quantitative benefit-risk analysis in regulatory decision-making - a case study of alosetron in irritable bowel syndrome. Value Heal. 2009;13:1–7. doi:10.1111/j.1524-4733.2009.00595.x. Lynd LD, Najafzadeh M, Colley L, Byrne MF, Willan AR, Sculpher MJ, et al. Using the incremental net benefit framework for quantitative benefit-risk analysis in regulatory decision-making - a case study of alosetron in irritable bowel syndrome. Value Heal. 2009;13:1–7. doi:10.​1111/​j.​1524-4733.​2009.​00595.​x.
31.
go back to reference Lynd LD, Marra CA, Najafzadeh M, Sadatsafavi M. A quantitative evaluation of the regulatory assessment of the benefits and risks of rofecoxib relative to naproxen: an application of the incremental net-benefit framework. Pharmacoepidemiol Drug Saf. 2010;19:1172–80. doi:10.1002/pds.CrossRefPubMed Lynd LD, Marra CA, Najafzadeh M, Sadatsafavi M. A quantitative evaluation of the regulatory assessment of the benefits and risks of rofecoxib relative to naproxen: an application of the incremental net-benefit framework. Pharmacoepidemiol Drug Saf. 2010;19:1172–80. doi:10.​1002/​pds.CrossRefPubMed
32.
33.
go back to reference Tervonen T, van Valkenhoef G, Buskens E, Hillege HL, Postmus D. A stochastic multicriteria model for evidence-based decision making in drug benefit-risk analysis. Stat Med. 2011;30:1419–28. doi:10.1002/sim.4194.CrossRefPubMed Tervonen T, van Valkenhoef G, Buskens E, Hillege HL, Postmus D. A stochastic multicriteria model for evidence-based decision making in drug benefit-risk analysis. Stat Med. 2011;30:1419–28. doi:10.​1002/​sim.​4194.CrossRefPubMed
35.
go back to reference Marshall DA, Burgos Liz L, Eng II, Ijzerman MJ, Osgood ND, Padula WV, et al. Applying dynamic simulation modeling methods in health care delivery research — the simulate checklist : report of the ispor simulation modeling emerging good practices task force. Value Heal. 2015;18:5–16. doi:10.1016/j.jval.2014.12.001.CrossRef Marshall DA, Burgos Liz L, Eng II, Ijzerman MJ, Osgood ND, Padula WV, et al. Applying dynamic simulation modeling methods in health care delivery research — the simulate checklist : report of the ispor simulation modeling emerging good practices task force. Value Heal. 2015;18:5–16. doi:10.​1016/​j.​jval.​2014.​12.​001.CrossRef
36.
go back to reference Giabbanelli PJ, Crutzen R. Creating groups with similar expected behavioural response in Randomized Controlled Trials: a fuzzy cognitive map approach. BMC Med Res Methodol. 2014;14:130.PubMedCentralCrossRefPubMed Giabbanelli PJ, Crutzen R. Creating groups with similar expected behavioural response in Randomized Controlled Trials: a fuzzy cognitive map approach. BMC Med Res Methodol. 2014;14:130.PubMedCentralCrossRefPubMed
38.
go back to reference Zafiropoulos N, Phillips L, Pignatti F, Luria X. Evaluating benefit-risk: An agency perspective. Regul Rapp. 2012;9:5–8. Zafiropoulos N, Phillips L, Pignatti F, Luria X. Evaluating benefit-risk: An agency perspective. Regul Rapp. 2012;9:5–8.
41.
go back to reference Rogowski W, Payne K, Schnell-Inderst P, Manca A, Rochau U, Jahn B, et al. Concepts of “personalization” in personalized medicine: implications for economic evaluation. Pharmacoeconomics. 2015;33:49–59. doi:10.1007/s40273-014-0211-5.CrossRefPubMed Rogowski W, Payne K, Schnell-Inderst P, Manca A, Rochau U, Jahn B, et al. Concepts of “personalization” in personalized medicine: implications for economic evaluation. Pharmacoeconomics. 2015;33:49–59. doi:10.​1007/​s40273-014-0211-5.CrossRefPubMed
Metadata
Title
Estimating the value of medical treatments to patients using probabilistic multi criteria decision analysis
Authors
Henk Broekhuizen
Catharina G. M. Groothuis-Oudshoorn
A. Brett Hauber
Jeroen P. Jansen
Maarten J. IJzerman
Publication date
01-12-2015
Publisher
BioMed Central
Published in
BMC Medical Informatics and Decision Making / Issue 1/2015
Electronic ISSN: 1472-6947
DOI
https://doi.org/10.1186/s12911-015-0225-8

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