Skip to main content
Key Specialties
Cardiology Diabetology Endocrinology Gastroenterology Geriatrics and Gerontology Gynecology and Obstetrics Hematology Infectious Disease Internal Medicine Nephrology Neurology Oncology Pediatrics Respiratory Medicine Rheumatology Surgery
Congress Coverage
2024 ACC Congress 2023 ESMO Congress 2023 EASD Congress 2023 ERS Congress 2023 ESC Congress 2023 EHA Congress 2023 ASCO Annual Meeting
Clinical Collections
Advances in Alzheimer’s

At a glance: The ONWARDS insulin icodec trials

A round-up of the ONWARDS phase 3 clinical trials evaluating the novel weekly insulin icodec in people with diabetes.
ADVANCED SEARCH
Log in
Top
BMC Medical Research Methodology
Published in: BMC Medical Research Methodology 1/2011

Open Access 01-12-2011 | Research article

Structuring and validating a cost-effectiveness model of primary asthma prevention amongst children

Authors: G Feljandro P Ramos, Sandra Kuiper, Edward Dompeling, Antoinette DI van Asselt, Wim JC de Grauw, J André Knottnerus, Onno CP van Schayck, Tjard RJ Schermer, Johan L Severens

Published in: BMC Medical Research Methodology | Issue 1/2011

Login to get access

Abstract

Background

Given the rising number of asthma cases and the increasing costs of health care, prevention may be the best cure. Decisions regarding the implementation of prevention programmes in general and choosing between unifaceted and multifaceted strategies in particular are urgently needed. Existing trials on the primary prevention of asthma are, however, insufficient on their own to inform the decision of stakeholders regarding the cost-effectiveness of such prevention strategies. Decision analytic modelling synthesises available data for the cost-effectiveness evaluation of strategies in an explicit manner. Published reports on model development should provide the detail and transparency required to increase the acceptability of cost-effectiveness modelling. But, detail on the explicit steps and the involvement of experts in structuring a model is often unevenly reported. In this paper, we describe a procedure to structure and validate a model for the primary prevention of asthma in children.

Methods

An expert panel was convened for round-table discussions to frame the cost-effectiveness research question and to select and structure a model. The model's structural validity, which indicates how well a model reflects the reality, was determined through descriptive and parallel validation. Descriptive validation was performed with the experts. Parallel validation qualitatively compared similarity between other published models with different decision problems.

Results

The multidisciplinary input of experts helped to develop a decision-tree structure which compares the current situation with screening and prevention. The prevention was further divided between multifaceted and unifaceted approaches to analyse the differences. The clinical outcome was diagnosis of asthma. No similar model was found in the literature discussing the same decision problem. Structural validity in terms of descriptive validity was achieved with the experts and was supported by parallel validation.

Conclusions

A decision-tree model developed with experts in round-table discussions benefits from a systematic and transparent approach and the multidisciplinary contributions of the experts. Parallel validation provides a feasible alternative to validating novel models. The process of structuring and validating a model presented in this paper could be a useful guide to increase transparency, credibility, and acceptability of (future, novel) models when experts are involved.
Appendix
Available only for authorised users
Literature
1.
Johansson SGO, Haahtela T: Introduction. Prevention of Allergy and Allergic Asthma: World Allergy Organization Project Report and Guidelines. Edited by: Johansson SGO, Haahtela T. 2004, Basel, CH: Karger, XIII-XIX. [Ring J, Adorini L, Berek C, Blaser K, Capron M, Denburg JA, Holgate ST, Marone G, Saito H (Series Editors): Chemical Immunology and Allergy, vol. 84.]
2.
Peters SP, Ferguson G, Deniz Y, Reisner C: Uncontrolled asthma: a review of the prevalence, disease burden and options for treatment. Respir Med. 2006, 100: 1139-1151. 10.1016/j.rmed.2006.03.031.CrossRefPubMed
3.
Lane S, Molina J, Plusa T: An international observational prospective study to determine the Cost of Asthma eXacerbations (COAX). Respir Med. 2006, 100: 434-450. 10.1016/j.rmed.2005.06.012.CrossRefPubMed
4.
Bahadori K, Doyle-Waters MM, Marra C, Lynd L, Alasaly K, Swiston J, FitzGerald JM: Economic burden of asthma: a systematic review. BMC Pulm Med. 2009, 9: 24-10.1186/1471-2466-9-24.CrossRefPubMedPubMedCentral
5.
Williams AE, Lloyd AC, Watson L, Rabe KF: Cost of scheduled and unscheduled asthma management in seven European Union countries. Eur Respir Rev. 2006, 15: 4-9. 10.1183/09059180.06.00009801.CrossRef
6.
Sculpher MJ, Price M: Measuring costs and consequences in economic evaluation in asthma. Respir Med. 2003, 97: 508-520. 10.1053/rmed.2002.1474.CrossRefPubMed
7.
Arshad SH: Primary prevention of asthma and allergy. J Allergy Clin Immunol. 2005, 116: 3-14. 10.1016/j.jaci.2005.03.043.CrossRefPubMed
8.
Arshad SH, Bateman B, Matthews SM: Primary prevention of asthma and atopy during childhood by allergen avoidance in infancy: a randomised controlled study. Thorax. 2003, 58: 489-493. 10.1136/thorax.58.6.489.CrossRefPubMedPubMedCentral
9.
Chan-Yeung M, Watson W, Ferguson A, Dimich-Ward H, Rousseau R, Lilley M, DyBuncio A, Becker A: The Canadian Childhood Asthma Primary Prevention Study: outcomes at 7 years of age. J Allergy Clin Immunol. 2005, 116: 49-55. 10.1016/j.jaci.2005.03.029.CrossRefPubMed
10.
Brunekreef B, Smit J, de Jongste J, Neijens H, Gerritsen J, Postma D, Aalberse R, Koopman L, Kerkhof M, Wijga A, van Strien R: The prevention and incidence of asthma and mite allergy (PIAMA) birth cohort study: design and first results. Pediatr Allergy Immunol. 2002, 13 (Suppl 15): 55-60.CrossRefPubMed
11.
Marks GB, Mihrshahi S, Kemp AS, Tovey ER, Webb K, Almqvist C, Ampon RD, Crisafulli C, Belousova EG, Mellis CM, Peat JK, Leeder SR: Prevention of asthma during first 5 years of life: a randomized controlled trial. J Allergy Clin Immunol. 2006, 118: 53-61. 10.1016/j.jaci.2006.04.004.CrossRefPubMed
12.
van Schayck OCP, Maas T, Kaper J, Knottnerus AJA, Sheikh A: Is there any role for allergen avoidance in the primary prevention of childhood asthma?. J Allergy Clin Immunol. 2007, 119: 1323-1328. 10.1016/j.jaci.2007.02.024.CrossRefPubMed
13.
Maas T, Kaper J, Sheikh A, Knottnerus JA, Wesseling G, Dompeling E, Muris JW, van Schayck CP: Mono and multifaceted allergen reduction interventions for preventing asthma in children at high risk of developing asthma. Cochrane Database Syst Rev. 2009, 3: CD006480-PubMed
14.
Schönberger HJAM, Dompeling E, Knottnerus JA, Maas T, Muris JWM, van Weel C, van Schayck CP: The PREVASC study: the clinical effect of a multifaceted educational intervention to prevent childhood asthma. Eur Respir J. 2005, 25: 660-670. 10.1183/09031936.05.00067704.CrossRefPubMed
15.
Kuiper S, Maas T, van Schayck CP, Muris JW, Schönberger HJ, Dompeling E, Gijsbers B, van Weel C, Knottnerus JA, PREVASC Group: The primary prevention of asthma in children study: design of a multifaceted prevention program. Pediatr Allergy Immunol. 2005, 16: 321-331. 10.1111/j.1399-3038.2005.00278.x.CrossRefPubMed
16.
Maas T, Dompeling E, Muris J, Wesseling , Knottnerus A, van Schayck O: Randomized controlled trial on environmental exposure reduction aimed at prevention of asthma in susceptible children: outcome of an intervention suitable for implementing in primary care [abstract]. Eur RespirJ. 2010, 36: 367s-
17.
Kuiper S, Muris JW, Dompeling E, Kester AD, Wesseling G, Knottnerus JA, van Schayck CP: Interactive effect of family history and environmental factors on respiratory tract-related morbidity in infancy. J Allergy Clin Immunol. 2007, 120: 388-395. 10.1016/j.jaci.2007.03.038.CrossRefPubMed
18.
Kuiper S, Muris JW, Dompeling E, van Schayck CP, Schönberger HJ, Wesseling G, Knottnerus JA: Association between first-degree familial predisposition of asthma and atopy (total IgE) in newborns. Clin Exp Allergy. 2006, 36: 594-601. 10.1111/j.1365-2222.2006.02467.x.CrossRefPubMed
19.
Brennan A, Akehurst R: Modelling in health economic evaluation: what is its place? what is its value?. Pharmacoeconomics. 2000, 17: 445-459. 10.2165/00019053-200017050-00004.CrossRefPubMed
20.
Sculpher MJ, Claxton K, Drummond M, McCabe C: Whither trial-based economic evaluation for health care decision making?. Health Econ. 2006, 15: 677-687. 10.1002/hec.1093.CrossRefPubMed
21.
Barton P, Bryan S, Robinson S: Modelling in the economic evaluation of health care: Selecting the appropriate approach. J Health Serv Res Policy. 2004, 9: 110-118. 10.1258/135581904322987535.CrossRefPubMed
22.
Karnon J, Brown J: Selecting a decision model for economic evaluation: A case study and review. Health Care Manag Sci. 1998, 1: 133-140. 10.1023/A:1019090401655.CrossRefPubMed
23.
Brennan A, Chick SE, Davis R: A taxonomy of model structures for economic evaluation of health technologies. Health Econ. 2006, 15: 1295-1310. 10.1002/hec.1148.CrossRefPubMed
24.
Stahl JE: Modelling methods for pharmacoeconomics and health technology assessment: An overview and guide. Pharmacoeconomics. 2008, 26: 131-148. 10.2165/00019053-200826020-00004.CrossRefPubMed
25.
Evans C, Crawford B: Expert use in pharmacoeconomic studies: Guidance and future use. Pharmacoeconomics. 2000, 17: 545-553. 10.2165/00019053-200017060-00002.CrossRefPubMed
26.
van Keulen HM, Mesters I, Brug J, Ausems M, Campbell M, Resnicow K, Zwietering PJ, van Breukelen G, van Mechelen W, Severens JL, de Vries H: Vitalum study design: RCT evaluating the efficacy of tailored print communication and telephone motivational interviewing on multiple health behaviors. BMC Public Health. 2008, 8: 216-10.1186/1471-2458-8-216.CrossRefPubMedPubMedCentral
27.
Albers-Heitner P, Berghmans B, Joore M, Lagro-Janssen T, Severens J, Nieman F, Winkens R: The effects of involving a nurse practitioner in primary care for adult patients with urinary incontinence: The PromoCon study (Promoting Continence). BMC Health Serv Res. 2008, 8: 84-10.1186/1472-6963-8-84.CrossRefPubMedPubMedCentral
28.
29.
Evans C: The use of consensus methods and expert panels in pharmacoeconomic studies. Pharmacoeconomics. 1997, 12: 121-129. 10.2165/00019053-199712020-00003.CrossRefPubMed
30.
van der Fels-Klerx IHJ, Goosens LHJ, Saatkamp HW, Horst SHS: Elicitation of quantitative data from a heterogeneous expert panel: Formal process and application in animal health. Risk Anal. 2002, 22: 67-81. 10.1111/0272-4332.t01-1-00007.CrossRefPubMed
31.
Steuten L, Palmer S, Vrijhoef B, van Merode F, Spreeuwenberg C, Severens H: Cost-utility of a disease management program for patients with asthma. Int J Technol Assess Health Care. 2007, 23: 184-191.CrossRefPubMed
32.
Detsky AS, Naglie G, Krahn MD, Naimark D, Redelmeier DA: Primer on medical decision analysis: Part 2--Building a tree. Med Decis Making. 1997, 17: 126-135. 10.1177/0272989X9701700202.CrossRefPubMed
33.
Weinstein MC, O'Brien B, Hornberger J, Jackson J, Johannesson M, McCabe C, Luce BR: Principles of good practice for decision analytic modelling in health-care evaluation: Report of the ISPOR Task Force on Good Research Practices--Modeling Studies. Value Health. 2003, 6: 9-17. 10.1046/j.1524-4733.2003.00234.x.CrossRefPubMed
34.
McCabe C, Dixon S: Testing the validity of cost-effectiveness models. Pharmacoeconomics. 2000, 17: 501-513. 10.2165/00019053-200017050-00007.CrossRefPubMed
35.
Hammerschmidt T, Goertz A, Wagenpfeil S, Neiss A, Wutzler P, Banz K: Validation of health economic models: The example of EVITA. Value Health. 2003, 6: 551-559. 10.1046/j.1524-4733.2003.65241.x.CrossRefPubMed
36.
Akehurst R, Anderson P, Brazier J, Brennan A, Briggs A, Buxton M, Cairns J, Calvert N, Claxton K, Dixon S, Fryback D, Gallivan S, Green C, Lloyd A, McCabe C, Mitchell A, Nicholl J, O'Brien B, Roberts J, Sculpher M, Severens H, Sullivan S, Veenstra D: Decision analytic modelling in the economic evaluation of health technologies: A consensus statement. Pharmacoeconomics. 2000, 17: 443-444. 10.2165/00019053-200017050-00003.CrossRef
37.
Russell LB: Comparing model structures in cost-effectiveness analysis. Decis Making. 2005, 25: 485-486. 10.1177/0272989X05281155.CrossRef
38.
Warner JO: Origins of asthma. Childhood Asthma. Edited by: Szefler SJ, Pedersen S. 2006, New York: Taylor & Francis, [Lenfant C (Series Editor): Lung Biology in Health and Disease, vol. 209].
39.
Alton V, Eckerlund I, Norlund A: Health economic evaluations: How to find them. Int J Tech Assess Health Care. 2006, 22: 512-517.CrossRef
40.
Sassi F, Archard L, McDaid D: Searching literature databases for health care economic evaluations: How systematic can we afford to be?. Med Care. 2002, 40: 387-394. 10.1097/00005650-200205000-00004.CrossRefPubMed
41.
Drummond MF, Sculpher MJ, Torrance GW, O'Brien BJ, Stoddart GL: Methods for the Economic Evaluation of Health Care Programmes. 2005, Oxford: Oxford University Press, 3
42.
Schönberger HJAM, van Schayck CPM: Prevention of asthma in genetically predisposed children in primary care--from clinical efficacy to a feasible intervention programme. Clin Exp Allergy. 1998, 28: 1325-1331. 10.1046/j.1365-2222.1998.00416.x.CrossRefPubMed
43.
Hakkaart-van Roijen L, Tan SS, Bouwmans CAM: Manual for cost investigation. 2010, Amstelveen, Netherlands: Dutch College of Health Insurers
44.
Dirksen WJ, Geijer RMM, de Haan M, de Koning G, Flikweert S, Kolnaar BGM: NHG-Standaard astma bij kinderen. Huisarts Wet. 1998, 41: 130-143.
45.
Nuijten MJ, Wittenberg W, Lebmeier M: Cost-effectiveness of palivizumab for respiratory syncytial virus prophylaxis in high-risk children: A UK analysis. Pharmacoeconomics. 2007, 25: 55-71. 10.2165/00019053-200725010-00006.CrossRefPubMed
46.
Omnes LF, Bousquet J, Scheinmann P, Neukirch F, Jasso-Mosqueda G, Chicoye A, Champion L, Fadel R: Pharmacoeconomic assessment of specific immunotherapy versus current symptomatic treatment of allergic rhinitis and asthma in France. Eur Ann Allergy Clin Immunol. 2007, 39: 148-156.PubMed
47.
Resch B, Gusenleitner W, Nuijten MJ, Lebmeier M, Wittenberg W: Cost-effectiveness of palivizumab against respiratory syncytial viral infection in high-risk children in Austria. Clin Ther. 2008, 30: 749-760. 10.1016/j.clinthera.2008.03.014.CrossRefPubMed
48.
El Hassan NO, Sorbero ME, Hall CB, Stevens TP, Dick AW: Cost-effectiveness analysis of palivizumab in premature infants without chronic disease. Arch Pediatr Adolesc Med. 2006, 160: 1070-1076. 10.1001/archpedi.160.10.1070.CrossRef
49.
Brüggenjürgen B, Reinhold T, Brehler R, Laake E, Wiese G, Machate U, Willich SN: Cost-effectiveness of specific subcutaneous immunotherapy in patients with allergic rhinitis and allergic asthma. Ann Allergy Asthma Immunol. 2008, 101: 316-324. 10.1016/S1081-1206(10)60498-X.CrossRefPubMed
50.
Buxton MJ, Drummond MF, van Hout BA, Prince RL, Sheldon TA, Szucs T, Vray M: Modelling in economic evaluation: An unavoidable fact of life. Health Econ. 1997, 6: 217-227. 10.1002/(SICI)1099-1050(199705)6:3<217::AID-HEC267>3.0.CO;2-W.CrossRefPubMed
51.
Akl EA, Grant BJB, Guyatt GH, Montori VM, Schünemann : A decision aid for COPD patients considering inhaled steroid therapy: development and before and after pilot testing. BMC Med Inform Decis Mak. 2007, 7: 12-10.1186/1472-6947-7-12.CrossRefPubMedPubMedCentral
52.
Weiss K, Haus M, Iikura Y: The costs of allergy and asthma and the potential benefit of prevention strategies. Prevention of Allergy and Allergic Asthma: World Allergy Organization Project Report and Guidelines. Edited by: Johansson SGO, Haahtela T. 2004, Basel CH: Karger, XIII-XIX. [Ring J, Adorini L, Berek C, Blaser K, Capron M, Denburg JA, Holgate ST, Marone G, Saito H (Series Editors): Chemical Immunology and Allergy, vol. 84.]
53.
Schwappach DLB: The economic evaluation of prevention--Let's talk about values and the case of discounting. Int J Public Health. 2007, 52: 335-336. 10.1007/s00038-007-0231-4.CrossRefPubMed
54.
Shiba K, Kashara K, Nakajima H, Adachi M: Structural changes of the airway wall impair respiratory function, even in mild asthma. Chest. 2002, 122: 1622-1666. 10.1378/chest.122.5.1622.CrossRefPubMed
55.
Lukrafka JL, Fuchs SC, Moreira LB, Picon RV, Fischer GB, Fuchs FD: Performance of the ISAAC Questionnaire to establish the prevalence of asthma in adolescents: A population-based study. J Asthma. 2010, 47: 166-169. 10.3109/02770900903483766.CrossRefPubMed
56.
Annemans L: Methodological issues in evaluating cost effectiveness of adjuvant aromatase inhibitors in early breast cancer: A need for improved modelling to aid decision making. Pharmacoeconomics. 2008, 26: 409-423. 10.2165/00019053-200826050-00005.CrossRefPubMed
57.
Gass SI: Decision-aiding models: validation, assessment, and related issues for policy analysis. Oper Res. 1983, 31: 603-631. 10.1287/opre.31.4.603.CrossRef
58.
Drummond MF, Barbieri M, Wong JB: Analytic choices in economic models of treatments for rheumatoid arthritis: what makes a difference?. Med Decis Making. 2005, 25: 520-533. 10.1177/0272989X05280561.CrossRefPubMed
59.
Goldhaber-Fiebert JD, Stout NK, Goldie SJ: Empirically evaluating decision-analytic models. Value Health. 2010, 13: 667-674. 10.1111/j.1524-4733.2010.00698.x.CrossRefPubMed
Metadata
Title
Structuring and validating a cost-effectiveness model of primary asthma prevention amongst children
Authors
G Feljandro P Ramos
Sandra Kuiper
Edward Dompeling
Antoinette DI van Asselt
Wim JC de Grauw
J André Knottnerus
Onno CP van Schayck
Tjard RJ Schermer
Johan L Severens
Publication date
01-12-2011
Publisher
BioMed Central
Published in
BMC Medical Research Methodology / Issue 1/2011
Electronic ISSN: 1471-2288
DOI
https://doi.org/10.1186/1471-2288-11-150

Other articles of this Issue 1/2011

BMC Medical Research Methodology 1/2011 Go to the issue

Research article

Re-evaluating a vision-related quality of life questionnaire with item response theory (IRT) and differential item functioning (DIF) analyses

Research article

Quantifying, displaying and accounting for heterogeneity in the meta-analysis of RCTs using standard and generalised Qstatistics

Research article

Reliability of the Marlowe-Crowne social desirability scale in Ethiopia, Kenya, Mozambique, and Uganda

Research article

Parametric methods outperformed non-parametric methods in comparisons of discrete numerical variables

Research article

Potential risk factors associated with human encephalitis: application of canonical correlation analysis

Research article

Opt-out as an acceptable method of obtaining consent in medical research: a short report