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Cost-effectiveness analysis of health technologies when evidence is scarce
Linköping University, Department of Medicine and Health Sciences, Health Technology Assessment. Linköping University, The Institute of Technology.
2010 (English)Doctoral thesis, comprehensive summary (Other academic)
Abstract [en]

Given the increasing pressures on health care budgets, economic evaluation is used in many countries to assist decision-making regarding the optimal use of competing health care technologies. Although the standard methods of estimating cost-effectiveness underpinning these decisions have gained widespread acceptance, concerns have been raised that many technologies would not be considered for funding, due to scarcity of evidence. However, as long as the amount and quality of evidence used for the analysis are properly characterized, scarce evidence per se should not be seen as a hindrance to perform cost-effectiveness analyses. Characterizing uncertainty appropriately, though, may pose a challenge even when there is a large body of evidence available, and even more so when evidence is scarce. The aims of this thesis are to apply a methodological framework of cost-effectiveness analysis and explore methods for characterising uncertainty when evidence is scarce. Three case studies associated with limited evidence provide economic evaluations on current decision problems, investigate the feasibility of using the framework, and explore methods for characterizing uncertainty when evidence is scarce.

The results of the case studies showed that, given current information, providing transfemoral amputees with C-Leg and Airsonett Airshower to patients with perennial allergic asthma could be considered cost-effective whereas screening for hyperthrophic cardiomyopathy among young athletes is unlikely to be cost-effective. In the cases of C-Leg and Airsonett Airshower conducting further research is likely to be cost-effective. The case studies indicate that it is feasible to apply methods of cost-effectiveness in health care for technologies not commonly evaluated due to lack of evidence. The analysis showed that failing to account for individual experts’ might have a substantial effect on the interpretation of the results of cost-effectiveness analysis. Formal expert elicitation is a promising method of characterizing uncertainty when evidence is missing, and thus enable cost-effectiveness and value of further research to be appropriately estimated in such situations.

In conclusion, this thesis shows that scarcity of evidence should not preclude the use of cost-effectiveness analysis. On the contrary, in such cases it is probably more important than ever to use a framework that enable us to define key parameters for a decision problem and identify available evidence in order to determine cost-effectiveness given current information and provide guidance on further data collection.

Place, publisher, year, edition, pages
Linköping: Linköping University Electronic Press , 2010. , 41 p.
Series
Linköping Studies in Science and Technology. Dissertations, ISSN 0345-7524 ; 1324
National Category
Health Care Service and Management, Health Policy and Services and Health Economy
Identifiers
URN: urn:nbn:se:liu:diva-56595ISBN: 978-91-7393-363-6 (print)OAI: oai:DiVA.org:liu-56595DiVA: diva2:320610
Public defence
2010-06-18, Aulan i Hälsans hus, Campus US, Linköpings universitet, Linköping, 09:00 (English)
Opponent
Supervisors
Note
In the list of included articles in the Ph.D. thesis the title of manuscript IV is "Cost-effectiveness of screening for hypertrophic cardiomyopathy in young athletes" but the title of the included article in the Ph.D. thesis is "Screening for hypertrophic cardiomyopathy in young athletes: A cost-effectiveness analysis".Available from: 2010-05-26 Created: 2010-05-26 Last updated: 2010-05-26Bibliographically approved
List of papers
1. Cost-effectiveness of clean air administered to the breathing zone in allergic asthma
Open this publication in new window or tab >>Cost-effectiveness of clean air administered to the breathing zone in allergic asthma
2010 (English)In: CLINICAL RESPIRATORY JOURNAL, ISSN 1752-6981, Vol. 4, no 2, 104-110 p.Article in journal (Refereed) Published
Abstract [en]

Introduction: Airsonett Airshower (AA) is a novel non-pharmaceutical treatment for patients with perennial allergic asthma that uses a laminar airflow directed to the breathing zone of patients during sleep. It has been shown that AA treatment in addition to optimized standard therapy significantly increases asthma-related quality of life among adolescent asthmatics. However, the cost-effectiveness of AA treatment has not yet been assessed. As reimbursement decisions are increasingly guided by results from the cost-effectiveness analysis, such information is valuable for health-care policy-makers. Objective: The objective of this study was to estimate the cost-effectiveness of adding AA treatment with allergen-free air during night sleep to optimized standard therapy for adolescents with perennial allergic asthma compared with placebo. Materials and Methods: A probabilistic Markov model was developed to estimate costs and health outcomes over a 5-year period. Costs and effects are presented from a Swedish health-care perspective (QALYs). The main outcome of interest was cost per QALY gained. Results: The Airshower strategy resulted in a mean gain of 0.25 QALYs per patient, thus yielding a cost per QALY gained of under 35 000 as long as the cost of Airshower is below 8200. Conclusions: Adding AA treatment to optimized standard therapy for adolescents with perennial allergic asthma compared with placebo is generating additional QALYs at a reasonable cost. However, further studies taking more detailed resource use and events such as exacerbations into account would be needed to fully evaluate the cost-effectiveness of AA treatment. Please cite this paper as: Brodtkorb T-H, Zetterstrom O and Tinghog G. Cost-effectiveness of clean air administered to the breathing zone in allergic asthma.

Keyword
asthma, cost-effectiveness, decision modeling, quality of life
National Category
Medical and Health Sciences
Identifiers
urn:nbn:se:liu:diva-54712 (URN)10.1111/j.1752-699X.2009.00156.x (DOI)000275766600007 ()
Available from: 2010-04-06 Created: 2010-04-06 Last updated: 2015-09-22
2. Cost-effectiveness of C-Leg compared to non microprocessor controlled knees: a modeling approach
Open this publication in new window or tab >>Cost-effectiveness of C-Leg compared to non microprocessor controlled knees: a modeling approach
2008 (English)In: Archives of Physical Medicine and Rehabilitation, ISSN 0003-9993, E-ISSN 1532-821X, Vol. 89, no 1, 24-30 p.Article in journal (Refereed) Published
Abstract [en]

Objective: To estimate the costs and health outcomes of C-Leg and non–microprocessor-controlled (NMC) knees using a decision-analytic model.

Design: Data on costs, rates and duration of problems, knee survival, and health-related quality of life were obtained from interviews with patients and prosthetists with experience of both C-Leg and NMC knees. Interview data were assessed in a decision-analytic Markov model to estimate cost-effectiveness from a health care perspective.

Setting: Outpatient.

Participants: A population sample of 20 patients currently using the C-Leg and prior experience of nonmicroprocessor knees, and 5 prosthetists.

Interventions: Not applicable.

Main Outcome Measure: Incremental cost per quality-adjusted life year (QALY).

Results: The mean incremental cost (in 2006 Euros) and QALYs for the C-Leg was €7657 and 2.38, respectively, yielding a cost per QALY gained of €3218.

Conclusions: It is important to provide decision-makers with relevant information on costs and health outcomes of different treatment strategies on actual decision problems despite limited evidence. The results of the study, taking into account both costs and a broadly defined health outcome in terms of QALY, show that given existing albeit limited evidence the C-Leg appears to yield positive health outcomes at an acceptable cost.

Place, publisher, year, edition, pages
Institutionen för medicin och hälsa, 2008
Keyword
Artificial limbs, Costs and cost analysis, Decision support techniques, Quality of life, Rehabilitation
National Category
Medical and Health Sciences
Identifiers
urn:nbn:se:liu:diva-11142 (URN)10.1016/j.apmr.2007.07.049 (DOI)
Note
Original publication: Thor-Henrik Brodtkorb, Martin Henriksson, Kasper Johannesen-Munk, Fredrik Thidell, Cost-effectiveness of C-Leg compared to non microprocessor controlled knees: a modelling approach, 2008, Archives of Physical Medicine and Rehabilitation, (89), 1, 24-30. http://dx.doi.org/10.1016/j.apmr.2007.07.049. Copyright: Elsevier B.V., http://www.elsevier.com/Available from: 2008-02-27 Created: 2008-02-27 Last updated: 2017-12-13
3. Eliciting priors to characterize uncertainties in decision analytic models
Open this publication in new window or tab >>Eliciting priors to characterize uncertainties in decision analytic models
(English)Manuscript (preprint) (Other academic)
Abstract [en]

Background: Expert opinions are often used in decision models when evidence is scarce. This study describes the details of a formal elicitation exercise to estimate parameter values and their associated uncertainty and compares the results in term of cost-effectiveness and value of information with results from only eliciting mean values from experts.

Methods: Elicited distributions for 11 unknown parameters where incorporated into a previously developed cost-effectiveness model for prosthetic knees for amputees. The original model included elicited mean values for the missing values, thus ignoring any uncertainty across experts’ beliefs.

Results: The incremental cost-effective ratio (ICER) for the analysis based on the current elicited distributions was substantially higher (€13 625) than the ICER in the original analysis (€3 258). Even decision uncertainty, at a €35 000 threshold, increased significantly, increasing the value of further research from €355 100 in the original analysis, to €5 987 444 for the current elicited values.

Conclusions: Failing to account for the individual expert’s uncertainty might have a considerable impact on the result of cost-effectiveness analyses. Formal expert elicitation offers a plausible method to generate prior distributions representing the experts’ uncertainty and thereby more appropriately account for the true uncertainty of the decision.

National Category
Economics and Business
Identifiers
urn:nbn:se:liu:diva-56593 (URN)
Available from: 2010-05-26 Created: 2010-05-26 Last updated: 2010-05-26
4. Screening for hypertrophic cardiomyopathy in young athletes: A cost-effectiveness analysis
Open this publication in new window or tab >>Screening for hypertrophic cardiomyopathy in young athletes: A cost-effectiveness analysis
(English)Manuscript (preprint) (Other academic)
Abstract [en]

Background: Screening to prevent sudden cardiac death among young athletes has been debated for some time and several countries have already introduced pre-participation cardiovascular screening to identify sports active individuals at risk. Although, hypertrophic cardiomyopathy (HCM) is the most common underlying disease that is documented to be detectable by screening the cost-effectiveness of such a screening strategy is still unclear.

Methods: A screening program to detect HCM in young athletes was compared to a non screening strategy. Prevalence of HCM, mortality risks and test characteristics were estimated from published sources and formal expert elicitation. These estimates were incorporated in a decision analytic model to estimate costs and health outcomes, expressed in life years and quality adjusted life years (QALYs), over a lifetime perspective.

Results: The screening strategy was associated with a mean incremental cost of €93 and a mean incremental gain of 0.0005 life years, yielding a cost per life year gained of €196 205. Taking quality of life into account, the screening strategy was associated with a loss of 0.034 QALY.

Conclusions: The study shows that screening young athletes for hypertrophic cardiomyopathy is not likely to yield survival benefits at a cost normally considered to be cost-effective and if quality of life is considered in the analysis screening is associated with higher costs and a loss of QALYs. Thus, based on the present findings a strategy of screening young athletes for hypertrophic cardiomyopathy is unlikely to be cost effective.

National Category
Medical and Health Sciences
Identifiers
urn:nbn:se:liu:diva-56594 (URN)
Available from: 2010-05-26 Created: 2010-05-26 Last updated: 2010-05-26Bibliographically approved

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