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Decision Making in Health and Medicine: Integrating Evidence and Values
Abstract
Decision making in health care involves consideration of a complex set of diagnostic, therapeutic and prognostic uncertainties. Medical therapies have side effects, surgical interventions may lead to complications, and diagnostic tests can produce misleading results. Furthermore, patient values and service costs must be considered. Decisions in clinical and health policy require careful weighing of risks and benefits and are commonly a trade-off of competing objectives: maximizing quality of life vs maximizing life expectancy vs minimizing the resources required. This text takes a proactive, systematic and rational approach to medical decision making. It covers decision trees, Bayesian revision, receiver operating characteristic curves, and cost-effectiveness analysis, as well as advanced topics such as Markov models, microsimulation, probabilistic sensitivity analysis and value of information analysis. It provides an essential resource for trainees and researchers involved in medical decision modelling, evidence-based medicine, clinical epidemiology, comparative effectiveness, public health, health economics, and health technology assessment.
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“Another approach is to use within-cycle corrections (WCC).2,22”
From An Introductory Tutorial on Cohort State-Transition Models in R Using a Cost-Effectiveness Analysis Example (2023) · cites this paper as [22]
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“Decision thresholds depend on treatment consequences—the relative potential for benefit and harm (28).”
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Self-citationWhen should decision-analytic modeling be used in the economic evaluation of health care?
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“A decision tree is a visual representation of all the possible options and the consequences that may follow each option [9].”
BackgroundEpigenome-based cancer risk prediction: rationale, opportunities and challenges
Widschwendter, M., Jones, A., Evans, I., Reisel, D., Dillner, J., Sundström, K., Steyerberg, E. W., Vergouwe, Y., Wegwarth, O., Rebitschek, F. G., et al. (2018) · Nature Reviews Clinical Oncology · doi:10.1038/nrclinonc.2018.30 · Cited by 163
Toward an evidence-based toxicology
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Impact of hospital volume on the economic value of computer navigation for total knee replacement.
Slover, J., Tosteson, A., Bozic, K., Rubash, H., & Malchau, H. (2008) · Journal of Bone and Joint Surgery. American volume · doi:10.2106/jbjs.g.00888 · Cited by 114
New Insights on Vaginal Birth After Cesarean: Can It Be Predicted?
Eden, K., McDonagh, M., Denman, M., Marshall, N., Emeis, C. L., Fu, R., Janik, R., Walker, M., & Guise, J. (2010) · Obstetrics and Gynecology · doi:10.1097/aog.0b013e3181f2de49 · Cited by 112
Slobogean, G., Noonan, V., & O'Brien, P. (2010) · Journal of shoulder and elbow surgery · doi:10.1016/j.jse.2009.10.021 · Cited by 107
“These utilities represent a desirability for a specific health state, and generic instruments that report utilities obtain these values by asking large population-based samples to assign a numeric value to each health state described by the instrument.(9) Utilities are essential for performing economic analyses becaus…”
BackgroundYang, Q., Zimmerman, J., Steinfeld, A., Carey, L., & Antaki, J. F. (2016) · Proceedings of the 2016 CHI Conference on Human Factors in Computing Systems · doi:10.1145/2858036.2858373 · Cited by 105
Uncertainty and patient heterogeneity in medical decision models
Groot Koerkamp, B., Weinstein, M. C., Stijnen, T., Heijenbrok-Kal, M. H., & Hunink, M. G. M. (2010) · Medical Decision Making · doi:10.1177/0272989x09342277 · Cited by 105
“We applied a halfcycle correction for all analyses.(11) Tables 1 to 3 in the Web appendix present all model parameters with their 95% uncertainty interval and sources.”
BackgroundMethodInfluentialSelf-citationBollen, K., & Horowitz, J. (2008) · Applied Animal Behaviour Science · doi:10.1016/j.applanim.2007.07.007 · Cited by 104
Spillover Effects on Caregivers’ and Family Members’ Utility: A Systematic Review of the Literature
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Self-citationSurgeon perception of risk and benefit in the decision to operate.
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Learning curve for coronary CT angiography: what constitutes sufficient training?
Pugliese, F., Hunink, M. G. M., Gruszczyńska, K., Alberghina, F., Malagó, R., van Pelt, N. V., Mollet, N., Cademartiri, F., Weustink, A., Meijboom, W., et al. (2009) · Radiology · doi:10.1148/radiol.2512080384 · Cited by 98
Self-citationDisutility of Illness for Caregivers and Families: A Systematic Review of the Literature
Wittenberg, E., & Prosser, L. A. (2013) · PharmacoEconomics · doi:10.1007/s40273-013-0040-y · Cited by 99
“, 1 - (1 - EQ-VAS score) [46], prior to calculation.”
MethodSelf-citationHealth State Utilities: A Framework for Studying the Gap Between the Imagined and the Real
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Emergency planning and mitigation at Vesuvius: A new evidence-based approach
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“The methodology is used in diagnosis and decision making in patient management and treatment, and for deriving guidelines in clinical practice (Hunink and Glasziou, 2001; Sackett et al., 2000).”
MethodGriebsch, I., Knowles, R., Brown, J., Bull, C., Wren, C., & Dezateux, C. (2007) · International Journal of Technology Assessment in Health Care · doi:10.1017/s0266462307070304 · Cited by 92
“Where data were not available from these sources, input parameters were obtained as subjective probabilities (26) from two or three pediatric cardiologists who provided individual estimates that were negotiated”
MethodDecision-analytic modeling to evaluate benefits and harms of medical tests: uses and limitations
Trikalinos, T. A., Siebert, U., & Lau, J. (2009) · Medical Decision Making · doi:10.1177/0272989x09345022 · Cited by 88
“Transparent and reproducible approaches, such as decisionanalytic modeling, are often necessary in evaluating the comparative clinical utility of medical tests.(8;9) Herein, we discuss the rationale for and impact of using modeling to assess medical tests for the broad audience of researchers who perform comparative e…”
BackgroundCost-effectiveness analysis of custom total knee cutting blocks.
Slover, J., Rubash, H., Malchau, H., & Bosco, J. (2012) · Journal of Arthroplasty · doi:10.1016/j.arth.2011.04.023 · Cited by 85
“Cost-effectiveness was measured using the incremental cost-effectiveness ratio (ICER), which is the ratio of the difference in cost divided by the difference in effectiveness between 2 different treatment strategies [17,9].”
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“This model incorporates the components of the clinical matrix, assesses economic consequences, and considers various time frames.(40) The data elements considered by the current model represent a combination of observable effects and speculative concerns.”
BackgroundUncertainty in Clinical Medicine
Djulbegović, B., Hozo, I., & Greenland, S. (2011) · Elsevier eBooks · doi:10.1016/b978-0-444-51787-6.50011-8 · Cited by 115
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“However, unlike most generic quality of life measures, health state values are unique because they quantify society's preferences or value for defined health states.(7) The health state reported by a subject is converted into a single numeric value where 0 represents death and 1 describes full health.”
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Integration of Meta-analysis and Economic Decision Modeling for Evaluating Diagnostic Tests
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Tapper, E. B., Hunink, M. G. M., Afdhal, N. H., Lai, M., & Sengupta, N. (2016) · PLoS ONE · doi:10.1371/journal.pone.0147237 · Cited by 68
Karger, R., Donner-Banzhoff, N., Müller, H., Kretschmer, V., & Hunink, M. G. M. (2007) · Platelets · doi:10.1080/09537100601100366 · Cited by 60
“Whether these predictive values are clinically useful depends on the possible consequences of the medical decision to label a patient as diseased, which can be formalized by finding the so-called treatment threshold [63].”
BackgroundSelf-citationIs Hip Arthroscopy Cost-effective for Femoroacetabular Impingement?
Shearer, D., Kramer, J., Bozic, K., & Feeley, B. (2012) · Clinical Orthopaedics and Related Research · doi:10.1007/s11999-011-2023-7 · Cited by 66
“Disutilities are transient decreases in utility resulting from undesirable events such as the period of pain and decreased function after major surgery [19].”
BackgroundSlover, J., Haas, J., Quirno, M., Phillips, M., & Bosco, J. (2011) · Journal of Arthroplasty · doi:10.1016/j.arth.2010.03.009 · Cited by 66
“A decision model [14] was used to evaluate the cost of the SA screening program.”
MethodJones, H. E., Hickman, M., Kasprzyk‐Hordern, B., Welton, N. J., Baker, D. R., & Ades, A. E. (2014) · The Science of The Total Environment · doi:10.1016/j.scitotenv.2014.02.101 · Cited by 65
“The choice of distribution should re fl ect the target properties of theparameterand thedistributionof any data informingit (see e.g. Hunink, 2001, p351).”
A Cost-Effectiveness Analysis Comparing 3 Anterior Cruciate Ligament Graft Types
Genuario, J. W., Faucett, S. C., Boublik, M., & Schlegel, T. (2012) · American Journal of Sports Medicine · doi:10.1177/0363546511426088 · Cited by 65
“Cost-effectiveness analysis (CEA; see Table 1) is a well-established tool to guide these decisions.(10,14) Cost-effectiveness analysis enables one to compare the benefits and costs associated with different treatment paradigms to determine which treatment choice is ultimately most effective and least costly.”
Decisions from Experience: From Monetary to Medical Gambles
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“In research on medical decision making, it is widely thought that patients’ choices can and should be modeled in terms of the expected utility framework (Hunink et al., 2001; Russell & Schwartz, 2012).”
BackgroundThe Combined Analysis of Uncertainty and Patient Heterogeneity in Medical Decision Models
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“Conversions were conducted using indices commonly employed to adjust for inflation [25], the Bureau of Labor Statistics Consumer Price Index Medical Care inflation rates [26,27], and the Statistics Canada Health and Personal Care Consumer Price Index [28].”
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The Half-Cycle Correction: Banish Rather Than Explain It
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Randomized study designs for lifestyle interventions: a tutorial
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Self-citationKarády, J., Mayrhofer, T., Ivanov, A., Foldyna, B., Lu, M. T., Ferencik, M., Pursnani, A., Salerno, M., Udelson, J., Mark, D., et al. (2020) · JAMA Network Open · doi:10.1001/jamanetworkopen.2020.28312 · Cited by 57
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Is quantitative benefit-risk modelling of drugs desirable or possible?
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“The cohort simulation takes into account an annual rate of discounting at 3% [24], and the cycle-length is 1 year.”
Cost-Effectiveness of Antivenoms for Snakebite Envenoming in 16 Countries in West Africa
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“Country and GDP/Capita ($) [49] Increm Cost Effect Ratio [ICER]/DALY ($) (95% Conf.”
BackgroundMethodInfluentialLinear Regression Metamodeling as a Tool to Summarize and Present Simulation Model Results
Jalal, H. J., Dowd, B. E., Sainfort, F., & Kuntz, K. M. (2013) · Medical Decision Making · doi:10.1177/0272989x13492014 · Cited by 52
“, 95% confidence interval) while the rest of the parameters are held equal to their mean values.(1) An analyst also may be interested in identifying threshold values at which the preferred intervention changes.”
BackgroundGastric Bypass is a Cost-Saving Procedure: Results from a Comprehensive Markov Model
Faria, G., Preto, J., & Costa-Maia, J. (2013) · Obesity Surgery · doi:10.1007/s11695-012-0816-8 · Cited by 50
“The Markov model allows for a more accurate representation of most time-dependent and repeating occurrences and is useful for the cost-effectiveness analysis of several clinical states [15, 16].”
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“Sensitivity analysis is a test of the stability of conclusions conducted by varying a variable, or variables, over a stated range and evaluating the effect of this variation on the outcome, in this study, the ICER, of the model [14].”
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Lukkien, D., Stolwijk, N. E., Askari, S. I., Hofstede, B. M., Nap, H. H., Boon, W., Peine, A., Moors, E. H. M., & Minkman, M. M. N. (2024) · JMIR Nursing · doi:10.2196/55962 · Cited by 55
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Value-of-information analysis to guide future research in colorectal cancer screening.
Hassan, C., Hunink, M. G. M., Laghi, A., Pickhardt, P., Zullo, A., Kim, D. H., Iafrate, F., & di Giulio, E. (2009) · Radiology · doi:10.1148/radiol.2533090234 · Cited by 43
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“Measuring values and incorporating them into the decision-making process ideally should be done from different points of view.(31,32) The perspective of the policy maker could be quite different from the perspective of the treating clinician and of the patient.”
BackgroundComparative effectiveness of treatment options for pediatric craniopharyngiomas.
Ali, Z. S., Bailey, R., Daniels, L. B., Vakhshori, V., Lewis, D., Hossain, A., Sitterley, K., Lee, J., Storm, P., Heuer, G., et al. (2014) · Journal of Neurosurgery: Pediatrics · doi:10.3171/2013.11.peds1320 · Cited by 42
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Self-citationCost-effectiveness of primary HPV screening for cervical cancer in Germany—a decision analysis
Sroczynski, G., Schnell-Inderst, P., Mühlberger, N., Lang, K., Aidelsburger, P., Wasem, J., Mittendorf, T., Engel, J., Hillemanns, P., Petry, K. U., et al. (2011) · European Journal of Cancer · doi:10.1016/j.ejca.2011.03.006 · Cited by 40
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Alarid-Escudero, F., Krijkamp, E., Enns, E. A., Yang, A., Hunink, M. G. M., Pechlivanoglou, P., & Jalal, H. (2023) · Medical Decision Making · doi:10.1177/0272989x221103163 · Cited by 39
“2,22 In this tutorial, we use Simpson’s 1/3rd rule by multiplying the rewards (e.g., costs and effectiveness) by 1 / 3 in the first and last cycles, by 4 / 3 for the odd cycles, and by 2 / 3 for the even cycles.”
Self-citationAdvancing the Power of Economic Evidence to Inform Investments in Children, Youth, and Families
Steuerle, E., Jackson, L., Families, & Youth Board On Children (2016) · National Academies Press eBooks · doi:10.17226/23481 · Cited by 38
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Cost-effectiveness of Antivenoms for Snakebite Envenoming in Nigeria
Habib, A., Lamorde, M., Dalhat, M., Habib, Z., & Kuznik, A. (2015) · PLoS Neglected Tropical Diseases · doi:10.1371/journal.pntd.0003381 · Cited by 37
“41 [34,35].”
Evaluation and review of pharmacoeconomic models
Hay, J. W. (2004) · Expert Opinion on Pharmacotherapy · doi:10.1517/14656566.5.9.1867 · Cited by 33
“A final modelling approach, known as Monte Carlo simulation, can be applied to any of the modelling methods already described, or to arbitrary model structures or modelling hybrids [23,24].”
MethodA case against default effect sizes in sport and exercise science
Caldwell, A., & Vigotsky, A. (2020) · PeerJ · doi:10.7717/peerj.10314 · Cited by 36
A cost analysis of Behcet's syndrome in Turkey.
Sut, N., Seyahi, E., Yurdakul, S., Şenocak, M., & Yazici, H. (2006) · Rheumatology · doi:10.1093/rheumatology/kel382 · Cited by 36
“The cost was analysed in two parts: direct and indirect costs [9–14].”
BackgroundMethodInfluentialLu, G., Brazier, J., & Ades, A. (2013) · Value in Health · doi:10.1016/j.jval.2012.07.003 · Cited by 36
“them into treatment effect estimates on the generic QOL scale [3–5].”
BackgroundWattson, D. A., Hunink, M. G. M., DiPiro, P., Das, P., Hodgson, D., Mauch, P., & Ng, A. (2014) · International Journal of Radiation Oncology, Biology, Physics · doi:10.1016/j.ijrobp.2014.06.013 · Cited by 35
“We constructed a Markov state transition model to evaluate the strategies of annual LDCT screening versus no screening among HL survivors, using Data TreeAge Pro 2013 (TreeAge Software, Inc, Williamstown, MA) (18).”
MethodSelf-citationManagement of patients with acoustic neuromas: A Markov decision analysis
Morrison, D. (2010) · The Laryngoscope · doi:10.1002/lary.20819 · Cited by 35
Bärnighausen, T., & Bloom, D. E. (2009) · Social Science & Medicine (1967) · doi:10.1016/j.socscimed.2008.11.009 · Cited by 35
Ofstad, E., Frich, J., Schei, E., Frankel, R., & Gulbrandsen, P. (2014) · Patient Education and Counseling · doi:10.1016/j.pec.2014.08.005 · Cited by 34
Eckermann, S., & Coelli, T. (2013) · Social Science & Medicine (1967) · doi:10.1016/j.socscimed.2012.10.020 · Cited by 34
Distraction from randomization in diagnostic research.
Biesheuvel, C., Grobbee, D., & Moons, K. (2006) · Annals of Epidemiology · doi:10.1016/j.annepidem.2005.10.004 · Cited by 33
Miksch, F., Jahn, B., Espinosa, K., Chhatwal, J., Siebert, U., & Popper, N. (2019) · PLoS ONE · doi:10.1371/journal.pone.0221564 · Cited by 32
van Kempen, B. J. H., Ferket, B. S., Steyerberg, E. W., Max, W. B., Hunink, M. G. M., & Fleischmann, K. E. (2015) · International Journal of Cardiology · doi:10.1016/j.ijcard.2015.10.171 · Cited by 32
Self-citationPandya, A., Weinstein, M., Salomon, J., Cutler, D. M., & Gaziano, T. (2014) · Circulation. Cardiovascular Quality and Outcomes · doi:10.1161/circoutcomes.113.000397 · Cited by 31
“Strategies were ranked by cost, and then incremental cost-effectiveness ratios were calculated; inefficient strategies were ruled out by strong dominance (higher incremental costs and lower incremental QALYs) or weak dominance (if they had higher incremental costs per QALY than a more effective strategy) per conventio…”
MethodSelf-citationKapoor, A., Chuang, W., Radhakrishnan, N. S., Smith, K. J., Berlowitz, D., Segal, J., Katz, J. N., & Losina, E. (2012) · PharmacoEconomics (Auckland) · doi:10.2165/11535210-000000000-00000 · Cited by 30
Health related quality of life in genital herpes: a pilot comparison of measures
Fisman, D. N. (2005) · Sexually Transmitted Infections · doi:10.1136/sti.2004.011619 · Cited by 26
Effectiveness, benefit harm and cost effectiveness of colorectal cancer screening in Austria
Jahn, B., Sroczynski, G., Bundo, M., Mühlberger, N., Puntscher, S., Todorović, J., Rochau, U., Oberaigner, W., Koffijberg, H., Fischer, T., et al. (2019) · BMC Gastroenterology · doi:10.1186/s12876-019-1121-y · Cited by 29
Should warfarin be discontinued before a dental extraction? A decision-tree analysis.
Balevi, B. (2010) · Oral surgery, oral medicine, oral pathology, oral radiology, and endodontics · doi:10.1016/j.tripleo.2010.03.018 · Cited by 29
The Influence of Influence Diagrams in Medicine
Pauker, S., & Wong, J. (2005) · Decision Analytics · doi:10.1287/deca.1060.0060 · Cited by 29
“Of two recent texts that deal with decision modeling in medicine (Chapman and Sonnenberg 2000, Hunink et al. 2001), only one deals explicitly with the influence diagram representation, and only in passing; evaluation is not discussed, so it is really not presented as a practical tool (Chapman and…”
BackgroundSelf-citationPharmaceutical Benefit–Risk Communication Tools: A Review of the Literature
Balog‐Way, D. H. P., Blazsin, H., Löfstedt, R. E., & Bouder, F. E. (2016) · Drug Safety · doi:10.1007/s40264-016-0466-1 · Cited by 30
Choice of a mechanical valve or a bioprosthesis for AVR: does CABG matter?
Puvimanasinghe, J., Takkenberg, J., Eijkemans, M., Steyerberg, E., van Herwerden, L. A., Grunkemeier, G., Habbema, J. F., & Bogers, A. (2003) · European Journal of Cardio-Thoracic Surgery · doi:10.1016/s1010-7940(03)00085-x · Cited by 28
“Further, it permits adjusting of hazards for the individual patient depending on prior events, thereby accommodating the variability with groups of subjects [15].”
Background
Metadata
- Authors
- Hunink, M. G. M., Weinstein, M. C., Wittenberg, E., Drummond, M. F., Pliskin, J. S., Wong, J. B., & Glasziou, P. P.
- Affiliations
- Erasmus University Rotterdam (NL)
- Harvard University (US)
- University of York (GB)
- Ben-Gurion University of the Negev (IL)
- Tufts University (US)
- Bond University (AU)
- ORCID
- Venue
- Cambridge University Press eBooks
- Publisher
- Cambridge University Press
- Published
- 2014-10-05
- OpenAlex
- W3023658191
- Cited by
- 425
- References
- 0
- Citations by year
- 2026: 17 · 2025: 25 · 2024: 17 · 2023: 23 · 2022: 37 · 2021: 37 · 2020: 35 · 2019: 22 · 2018: 24 · 2017: 18 · 2016: 17 · 2015: 17 · 2014: 20
- FWCI
- 31.10
- Type
- book
- Language
- en
- Open access
- closed
- Locations
- Cambridge University Press eBooks (publishedVersion)
- Topics
- Social Sciences › Economics, Econometrics and Finance › Economics and Econometrics › Health Systems, Economic Evaluations, Quality of Life
- Health Sciences › Medicine › Family Practice › Clinical Reasoning and Diagnostic Skills
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Epidemiology
- Keywords
- clinical decision-making, clinical decision analysis, decision tree, receiver operating characteristic, cost-effectiveness analysis, Markov model, microsimulation, probabilistic sensitivity analysis, value of information analysis, health policy, risk-benefit trade-off, diagnostic uncertainty, treatment side effects, health economics, health technology assessment
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