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
Clinical Collections
Advances in Alzheimer’s

Keynote webinar | Spotlight on medication adherence

LIVE: Thursday 27th June 2024, 18:00-19:30 (CEST)
Join our expert panel to discover why you need to understand the drivers of non-adherence in your patients, and how you can optimize medication adherence in your clinics to drastically improve patient outcomes.
ADVANCED SEARCH
Log in
Top
Cost Effectiveness and Resource Allocation
Published in: Cost Effectiveness and Resource Allocation 1/2010

Open Access 01-12-2010 | Research

Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decisionmaking framework to growth hormone for Turner syndrome patients

Authors: Mireille M Goetghebeur, Monika Wagner, Hanane Khoury, Donna Rindress, Jean-Pierre Grégoire, Cheri Deal

Published in: Cost Effectiveness and Resource Allocation | Issue 1/2010

Login to get access

Abstract

Objectives

To test and further develop a healthcare policy and clinical decision support framework using growth hormone (GH) for Turner syndrome (TS) as a complex case study.

Methods

The EVIDEM framework was further developed to complement the multicriteria decision analysis (MCDA) Value Matrix, that includes 15 quantifiable components of decision clustered in four domains (quality of evidence, disease, intervention and economics), with a qualitative tool including six ethical and health system-related components of decision. An extensive review of the literature was performed to develop a health technology assessment report (HTA) tailored to each component of decision, and content was validated by experts. A panel of representative stakeholders then estimated the MCDA value of GH for TS in Canada by assigning weights and scores to each MCDA component of decision and then considered the impact of non-quantifiable components of decision.

Results

Applying the framework revealed significant data gaps and the importance of aligning research questions with data needs to truly inform decision. Panelists estimated the value of GH for TS at 41% of maximum value on the MCDA scale, with good agreement at the individual level (retest value 40%; ICC: 0.687) and large variation across panelists. Main contributors to this panel specific value were "Improvement of efficacy", "Disease severity" and "Quality of evidence". Ethical considerations on utility, efficiency and fairness as well as potential misuse of GH had mixed effects on the perceived value of the treatment.

Conclusions

This framework is proposed as a pragmatic step beyond the current cost-effectiveness model, combining HTA, MCDA, values and ethics. It supports systematic consideration of all components of decision and available evidence for greater transparency. Further testing and validation is needed to build up MCDA approaches combined with pragmatic HTA in healthcare decisionmaking.
Appendix
Available only for authorised users
Literature
1.
Schlander M: The use of cost-effectiveness by the National Institute for Health and Clinical Excellence (NICE): no(t yet an) exemplar of a deliberative process. J Med Ethics 2008, 34: 534–539. 10.1136/jme.2007.021683PubMedCrossRef
2.
Williams I, McIver S, Moore D, Bryan S: The use of economic evaluations in NHS decision-making: a review and empirical investigation. Health Technol Assess 2008, 12: iii. ix-iii,175CrossRef
3.
Nord E, Daniels N, Kamlet M: QALYs: some challenges. Value Health 2009,12(Suppl 1):S10-S15. 10.1111/j.1524-4733.2009.00516.xPubMedCrossRef
4.
Drummond M, Evans B, LeLorier J, Karakiewicz P, Martin D, Tugwell P, et al.: Evidence and values: requirements for public reimbursement of drugs for rare diseases--a case study in oncology. Can J Clin Pharmacol 2009, 16: e273-e281.PubMed
5.
Drummond MF, Schwartz JS, Jonsson B, Luce BR, Neumann PJ, Siebert U, et al.: Key principles for the improved conduct of health technology assessments for resource allocation decisions. Int J Technol Assess Health Care 2008, 24: 244–258.PubMed
6.
Jehu-Appiah C, Baltussen R, Acquah C, Aikins M, d'Almeida SA, Bosu WK, et al.: Balancing equity and efficiency in health priorities in Ghana: the use of multicriteria decision analysis. Value Health 2008, 11: 1081–1087. 10.1111/j.1524-4733.2008.00392.xPubMedCrossRef
7.
Browman GP, Manns B, Hagen N, Chambers CR, Simon A, Sinclair S: 6-STEPPPs: A modular tool to facilitate clinician participation in fair decisions for funding new cancer drugs. Journal of Oncology Practice 2008, 4: 2–7. 10.1200/JOP.0812001PubMedCentralPubMedCrossRef
8.
Johnson AP, Sikich NJ, Evans G, Evans W, Giacomini M, Glendining M, et al.: Health technology assessment: a comprehensive framework for evidence-based recommendations in Ontario. Int J Technol Assess Health Care 2009, 25: 141–150. 10.1017/S0266462309090199PubMedCrossRef
9.
10.
Martin DK, Pater JL, Singer PA: Priority-setting decisions for new cancer drugs: a qualitative case study. Lancet 2001, 358: 1676–1681. 10.1016/S0140-6736(01)06714-9PubMedCrossRef
11.
Mullen PM: Quantifying priorities in healthcare: transparency or illusion? Health Serv Manage Res 2004, 17: 47–58. 10.1258/095148404322772723PubMedCrossRef
12.
Wilson E, Sussex J, Macleod C, Fordham R: Prioritizing health technologies in a Primary Care Trust. J Health Serv Res Policy 2007, 12: 80–85. 10.1258/135581907780279495PubMedCrossRef
13.
Camidge DR, Oliver JJ, Skinner C, Attwood B, Nussey F, Jodrell D, et al.: The impact of prognosis without treatment on doctors' and patients' resource allocation decisions and its relevance to new drug recommendation processes. Br J Clin Pharmacol 2008, 65: 224–229. 10.1111/j.1365-2125.2007.02996.xPubMedCentralPubMedCrossRef
14.
Wilson EC, Peacock SJ, Ruta D: Priority setting in practice: what is the best way to compare costs and benefits? Health Econ 2008.
15.
Tappenden P, Brazier J, Ratcliffe J, Chilcott J: A stated preference binary choice experiment to explore NICE decision making. Pharmacoeconomics 2007, 25: 685–693. 10.2165/00019053-200725080-00006PubMedCrossRef
16.
Baltussen R, Niessen L: Priority setting of health interventions: the need for multi-criteria decision analysis. Cost Eff Resour Alloc 2006, 4: 14. 10.1186/1478-7547-4-14PubMedCentralPubMedCrossRef
17.
Baltussen R, Stolk E, Chisholm D, Aikins M: Towards a multi-criteria approach for priority setting: an application to Ghana. Health Econ 2006, 15: 689–696. 10.1002/hec.1092PubMedCrossRef
18.
Baltussen R, ten Asbroek AH, Koolman X, Shrestha N, Bhattarai P, Niessen LW: Priority setting using multiple criteria: should a lung health programme be implemented in Nepal? Health Policy Plan 2007, 22: 178–185. 10.1093/heapol/czm010PubMedCrossRef
19.
Peacock S, Mitton C, Bate A, McCoy B, Donaldson C: Overcoming barriers to priority setting using interdisciplinary methods. Health Policy 2009, 92: 124–132. 10.1016/j.healthpol.2009.02.006PubMedCrossRef
20.
Culyer AJ: Equity of what in healthcare? Why the traditional answers don't help policy--and what to do in the future. Healthc Pap 2007,8(Spec No):12–26.PubMedCrossRef
21.
22.
Daniels N, Sabin J: Limits to health care: fair procedures, democratic deliberation, and the legitimacy problem for insurers. Philos Public Aff 1997, 26: 303–350. 10.1111/j.1088-4963.1997.tb00082.xPubMedCrossRef
23.
Hutton J, Trueman P, Facey K: Harmonization of evidence requirements for health technology assessment in reimbursement decision making. Int J Technol Assess Health Care 2008, 24: 511–517. 10.1017/S0266462308080677PubMedCrossRef
24.
Lehoux P, Williams-Jones B: Mapping the integration of social and ethical issues in health technology assessment. Int J Technol Assess Health Care 2007, 23: 9–16. 10.1017/S0266462307051513PubMedCrossRef
25.
Autti-Ramo I, Makela M: Ethical evaluation in health technology assessment reports: an eclectic approach. Int J Technol Assess Health Care 2007, 23: 1–8.PubMed
26.
Hofmann B: Toward a procedure for integrating moral issues in health technology assessment. Int J Technol Assess Health Care 2005, 21: 312–318. 10.1017/S0266462305050415PubMedCrossRef
27.
Goetghebeur MM, Wagner M, Khoury H, Levitt RJ, Erickson LJ, Rindress D: Evidence and Value: Impact on DEcisionMaking - the EVIDEM framework and potential applications. BMC Health Serv Res 2008, 8: 270. 10.1186/1472-6963-8-270PubMedCentralPubMedCrossRef
28.
Bondy CA: Care of girls and women with Turner syndrome: A guideline of the Turner Syndrome Study Group. J Clin Endocrinol Metab 2007, 92: 10–25. 10.1210/jc.2006-1374PubMedCrossRef
29.
Stochholm K, Juul S, Juel K, Naeraa RW, Gravholt CH: Prevalence, incidence, diagnostic delay, and mortality in Turner syndrome. J Clin Endocrinol Metab 2006, 91: 3897–3902. 10.1210/jc.2006-0558PubMedCrossRef
30.
Gruskin S, Daniels N: Process is the point: justice and human rights: priority setting and fair deliberative process. Am J Public Health 2008, 98: 1573–1577. 10.2105/AJPH.2007.123182PubMedCentralPubMedCrossRef
31.
Persad G, Werthiemer A, Emanuel EJ: Principles for allocation of scarce medical interventions. Lancet 2009, 373: 423–431. 10.1016/S0140-6736(09)60137-9PubMedCrossRef
32.
33.
34.
35.
Vuorenkoski L, Toiviainen H, Hemminki E: Drug reimbursement in Finland-a case of explicit prioritizing in special categories. Health Policy 2003, 66: 169–177. 10.1016/S0168-8510(03)00042-3PubMedCrossRef
36.
Giacomini M: One of these things is not like the others: the idea of precedence in health technology assessment and coverage decisions. Milbank Q 2005, 83: 193–223. 10.1111/j.1468-0009.2005.00344.xPubMedCentralPubMedCrossRef
37.
Baxter L, Bryant J, Cave CB, Milne R: Recombinant growth hormone for children and adolescents with Turner syndrome. Cochrane Database Syst Rev 2007, CD003887.
38.
Freemantle N, Hessel F: The applicability and generalizability of findings from clinical trials for health-policy decisions. Pharmacoeconomics 2009, 27: 5–10. 10.2165/00019053-200927010-00002PubMedCrossRef
39.
Gerkens S, Crott R, Cleemput I, Thissen JP, Closon MC, Horsmans Y, et al.: Comparison of three instruments assessing the quality of economic evaluations: a practical exercise on economic evaluations of the surgical treatment of obesity. Int J Technol Assess Health Care 2008, 24: 318–325. 10.1017/S0266462308080422PubMedCrossRef
40.
Shrout PE, Fleiss JL: Intraclass correlations: uses in assessing rater reliability. Psychol Bull 1979, 86: 420–428. 10.1037/0033-2909.86.2.420PubMedCrossRef
41.
42.
Sibbald SL, Singer PA, Upshur R, Martin DK: Priority setting: what constitutes success? A conceptual framework for successful priority setting. BMC Health Serv Res 2009, 9: 43. 10.1186/1472-6963-9-43PubMedCentralPubMedCrossRef
43.
Lomas J: Decision support: a new approach to making the best healthcare management and policy choices. Healthc Q 2007, 10: 16–18.PubMedCrossRef
44.
Daniels N: Normal functioning and the treatment-enhancement distinction. Camb Q Healthc Ethics 2000, 9: 309–322. 10.1017/S0963180100903037PubMed
45.
Sniderman AD, Furberg CD: Why guideline-making requires reform. JAMA 2009, 301: 429–431. 10.1001/jama.2009.15PubMedCrossRef
46.
47.
Taylor DW, Fowler E: The importance of patient participation in health policy decision making consumer engagement in health technology assessment: Recommendations for the mandate and makeup of a citizens' council as created under Ontario's Transparent Drug System for Patients Act. 2007.
48.
49.
Haverkamp F, Ranke MB: The ethical dilemma of growth hormone treatment of short stature: a scientific theoretical approach. Horm Res 1999, 51: 301–304. 10.1159/000023417PubMedCrossRef
50.
Verweij M, Kortmann F: Moral assessment of growth hormone therapy for children with idiopathic short stature. J Med Ethics 1997, 23: 305–309. 10.1136/jme.23.5.305PubMedCentralPubMedCrossRef
51.
Stiggelbout AM, Vogel-Voogt E, Noordijk EM, Vliet Vlieland TP: Individual quality of life: adaptive conjoint analysis as an alternative for direct weighting? Qual Life Res 2008, 17: 641–649. 10.1007/s11136-008-9325-6PubMedCentralPubMedCrossRef
Metadata
Title
Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decisionmaking framework to growth hormone for Turner syndrome patients
Authors
Mireille M Goetghebeur
Monika Wagner
Hanane Khoury
Donna Rindress
Jean-Pierre Grégoire
Cheri Deal
Publication date
01-12-2010
Publisher
BioMed Central
Published in
Cost Effectiveness and Resource Allocation / Issue 1/2010
Electronic ISSN: 1478-7547
DOI
https://doi.org/10.1186/1478-7547-8-4

Other articles of this Issue 1/2010

Cost Effectiveness and Resource Allocation 1/2010 Go to the issue

Review

The scope of costs in alcohol studies: Cost-of-illness studies differ from economic evaluations

Research

To what extent does recurrent government health expenditure in Uganda reflect its policy priorities?

Research

Cost-effectiveness of a pressure ulcer quality collaborative

Research

A health economic model for evaluating a vaccine for the prevention of herpes zoster and post-herpetic neuralgia in the UK

Research

Estimation of the cost of treatment by chemotherapy for early breast cancer in Morocco

Research

Cost-utility of Intravenous Immunoglobulin (IVIG) compared with corticosteroids for the treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in Canada