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1.  Clinical and economic consequences of vancomycin and fidaxomicin for the treatment of Clostridium difficile infection in Canada 
Current treatment options for Clostridium difficile are limited. Recent trials have demonstrated the noninferiority of the new macrocyclic antibiotic fidaxomicin to vancomycin, the current standard treatment for more severe disease. Fidaxomicin has also been associated with fewer recurrences; however, the increased cost compared with vancomycin has precluded it from becoming the new standard therapy. The authors of this article conducted a cost-effectiveness analysis for vancomycin versus fidaxomicin, and investigated implications for the use of these antibiotics to treat C difficile infections in the Canadian health care system.
Clostridium difficile infection (CDI) represents a public health problem with increasing incidence and severity.
To evaluate the clinical and economic consequences of vancomycin compared with fidaxomicin in the treatment of CDI from the Canadian health care system perspective.
A decision-tree model was developed to compare vancomycin and fidaxomicin for the treatment of severe CDI. The model assumed identical initial cure rates and included first recurrent episodes of CDI (base case). Treatment of patients presenting with recurrent CDI was examined as an alternative analysis. Costs included were for study medication, physician services and hospitalization. Cost effectiveness was measured as incremental cost per recurrence avoided. Sensitivity analyses of key input parameters were performed.
In a cohort of 1000 patients with an initial episode of severe CDI, treatment with fidaxomicin led to 137 fewer recurrences at an incremental cost of $1.81 million, resulting in an incremental cost of $13,202 per recurrence avoided. Among 1000 patients with recurrent CDI, 113 second recurrences were avoided at an incremental cost of $18,190 per second recurrence avoided. Incremental costs per recurrence avoided increased with increasing proportion of cases caused by the NAP1/B1/027 strain. Results were sensitive to variations in recurrence rates and treatment duration but were robust to variations in other parameters.
The use of fidaxomicin is associated with a cost increase for the Canadian health care system. Clinical benefits of fidaxomicin compared with vancomycin depend on the proportion of cases caused by the NAP1/B1/027 strain in patients with severe CDI.
PMCID: PMC4028674  PMID: 24855476
Clostridium difficile; Cost effectiveness; Fidaxomicin; Recurrence; Vancomycin
2.  Growth Hormone Research Society Workshop Summary: Consensus Guidelines for Recombinant Human Growth Hormone Therapy in Prader-Willi Syndrome 
Recombinant human GH (rhGH) therapy in Prader-Willi syndrome (PWS) has been used by the medical community and advocated by parental support groups since its approval in the United States in 2000 and in Europe in 2001. Its use in PWS represents a unique therapeutic challenge that includes treating individuals with cognitive disability, varied therapeutic goals that are not focused exclusively on increased height, and concerns about potential life-threatening adverse events.
The aim of the study was to formulate recommendations for the use of rhGH in children and adult patients with PWS.
We performed a systematic review of the clinical evidence in the pediatric population, including randomized controlled trials, comparative observational studies, and long-term studies (>3.5 y). Adult studies included randomized controlled trials of rhGH treatment for ≥ 6 months and uncontrolled trials. Safety data were obtained from case reports, clinical trials, and pharmaceutical registries.
Forty-three international experts and stakeholders followed clinical practice guideline development recommendations outlined by the AGREE Collaboration ( Evidence was synthesized and graded using a comprehensive multicriteria methodology (EVIDEM) (
Following a multidisciplinary evaluation, preferably by experts, rhGH treatment should be considered for patients with genetically confirmed PWS in conjunction with dietary, environmental, and lifestyle interventions. Cognitive impairment should not be a barrier to treatment, and informed consent/assent should include benefit/risk information. Exclusion criteria should include severe obesity, uncontrolled diabetes mellitus, untreated severe obstructive sleep apnea, active cancer, or psychosis. Clinical outcome priorities should vary depending upon age and the presence of physical, mental, and social disability, and treatment should be continued for as long as demonstrated benefits outweigh the risks.
PMCID: PMC3789886  PMID: 23543664
4.  From efficacy to equity: Literature review of decision criteria for resource allocation and healthcare decisionmaking 
Resource allocation is a challenging issue faced by health policy decisionmakers requiring careful consideration of many factors. Objectives of this study were to identify decision criteria and their frequency reported in the literature on healthcare decisionmaking.
An extensive literature search was performed in Medline and EMBASE to identify articles reporting healthcare decision criteria. Studies conducted with decisionmakers (e.g., focus groups, surveys, interviews), conceptual and review articles and articles describing multicriteria tools were included. Criteria were extracted, organized using a classification system derived from the EVIDEM framework and applying multicriteria decision analysis (MCDA) principles, and the frequency of their occurrence was measured.
Out of 3146 records identified, 2790 were excluded. Out of 356 articles assessed for eligibility, 40 studies included. Criteria were identified from studies performed in several regions of the world involving decisionmakers at micro, meso and macro levels of decision and from studies reporting on multicriteria tools. Large variations in terminology used to define criteria were observed and 360 different terms were identified. These were assigned to 58 criteria which were classified in 9 different categories including: health outcomes; types of benefit; disease impact; therapeutic context; economic impact; quality of evidence; implementation complexity; priority, fairness and ethics; and overall context. The most frequently mentioned criteria were: equity/fairness (32 times), efficacy/effectiveness (29), stakeholder interests and pressures (28), cost-effectiveness (23), strength of evidence (20), safety (19), mission and mandate of health system (19), organizational requirements and capacity (17), patient-reported outcomes (17) and need (16).
This study highlights the importance of considering both normative and feasibility criteria for fair allocation of resources and optimized decisionmaking for coverage and use of healthcare interventions. This analysis provides a foundation to develop a questionnaire for an international survey of decisionmakers on criteria and their relative importance. The ultimate objective is to develop sound multicriteria approaches to enlighten healthcare decisionmaking and priority-setting.
PMCID: PMC3495194  PMID: 22808944
Decisionmaking; Resource allocation; Priority-setting; Criteria; Healthcare
5.  Burden of community-acquired and nosocomial rotavirus gastroenteritis in the pediatric population of Western Europe: a scoping review 
Rotavirus affects 95% of children worldwide by age 5 years and is the leading cause of severe dehydrating diarrhea. The objective of this review was to estimate the burden of rotavirus gastroenteritis (RVGE) in the Western European pediatric population.
A comprehensive literature search (1999-2010) was conducted in PubMed and other sources (CDC; WHO, others). Data on the epidemiology and burden of RVGE among children < 5 years-old in Western Europe --including hospital-acquired disease--were extracted.
76 studies from 16 countries were identified. The mean percentage of acute gastroenteritis (AGE) cases caused by rotavirus ranged from 25.3%-63.5% in children < 5 years of age, peaking during winter. Incidence rates of RVGE ranged from 1.33-4.96 cases/100 person- years. Hospitalization rates for RVGE ranged from 7% to 81% among infected children, depending on the country. Nosocomial RVGE accounted for 47%-69% of all hospital-acquired AGE and prolonged hospital stays by 4-12 days. Each year, RVGE incurred $0.54- $53.6 million in direct medical costs and $1.7-$22.4 million in indirect costs in the 16 countries studied. Full serotyping data was available for 8 countries. G1P[8], G2P[4], G9P[8], and G3P[8] were the most prevalent serotypes (cumulative frequency: 57.2%- 98.7%). Serotype distribution in nosocomial RVGE was similar.
This review confirms that RVGE is a common disease associated with significant morbidity and costs across Western Europe. A vaccine protecting against multiple serotypes may decrease the epidemiological and cost burden of RVGE in Western Europe.
PMCID: PMC3342230  PMID: 22429601
Rotavirus; Burden of illness; Gastroenteritis; Pediatric population
6.  Field testing of a multicriteria decision analysis (MCDA) framework for coverage of a screening test for cervical cancer in South Africa 
Systematic and transparent approaches to priority setting are needed, particularly in low-resource settings, to produce decisions that are sound and acceptable to stakeholders. The EVIDEM framework brings together Health Technology Assessment (HTA) and multi-criteria decision analysis (MCDA) by proposing a comprehensive set of decision criteria together with standardized processes to support decisionmaking. The objective of the study was to field test the framework for decisionmaking on a screening test by a private health plan in South Africa.
Liquid-based cytology (LBC) for cervical cancer screening was selected by the health plan for this field test. An HTA report structured by decision criterion (14 criteria organized in the MCDA matrix and 4 contextual criteria) was produced based on a literature review and input from the health plan. During workshop sessions, committee members 1) weighted each MCDA decision criterion to express their individual perspectives, and 2) to appraise LBC, assigned scores to each MCDA criterion on the basis of the by-criterion HTA report.
Committee members then considered the potential impacts of four contextual criteria on the use of LBC in the context of their health plan. Feedback on the framework and process was collected through discussion and from a questionnaire.
For 9 of the MCDA matrix decision criteria, 89% or more of committee members thought they should always be considered in decisionmaking. Greatest weights were given to the criteria "Budget impact", "Cost-effectiveness" and "Completeness and consistency of reporting evidence". When appraising LBC for cervical cancer screening, the committee assigned the highest scores to "Relevance and validity of evidence" and "Disease severity". Combination of weights and scores yielded a mean MCDA value estimate of 46% (SD 7%) of the potential maximum value. Overall, the committee felt the framework brought greater clarity to the decisionmaking process and was easily adaptable to different types of health interventions.
The EVIDEM framework was easily adapted to evaluating a screening technology in South Africa, thereby broadening its applicability in healthcare decision making.
PMCID: PMC3330006  PMID: 22376143
7.  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 
Consistent healthcare decisionmaking requires systematic consideration of decision criteria and evidence available to inform them. This can be tackled by combining multicriteria decision analysis (MCDA) and Health Technology Assessment (HTA). The objective of this study was to field-test a decision support framework (EVIDEM), explore its utility to a drug advisory committee and test its reliability over time.
Tramadol for chronic non-cancer pain was selected by the health plan as a case study relevant to their context. Based on extensive literature review, a by-criterion HTA report was developed to provide synthesized evidence for each criterion of the framework (14 criteria for the MCDA Core Model and 6 qualitative criteria for the Contextual Tool). During workshop sessions, committee members tested the framework in three steps by assigning: 1) weights to each criterion of the MCDA Core Model representing individual perspective; 2) scores for tramadol for each criterion of the MCDA Core Model using synthesized data; and 3) qualitative impacts of criteria of the Contextual Tool on the appraisal. Utility and reliability of the approach were explored through discussion, survey and test-retest. Agreement between test and retest data was analyzed by calculating intra-rater correlation coefficients (ICCs) for weights, scores and MCDA value estimates.
The framework was found useful by the drug advisory committee in supporting systematic consideration of a broad range of criteria to promote a consistent approach to appraising healthcare interventions. Directly integrated in the framework as a "by-criterion" HTA report, synthesized evidence for each criterion facilitated its consideration, although this was sometimes limited by lack of relevant data. Test-retest analysis showed fair to good consistency of weights, scores and MCDA value estimates at the individual level (ICC ranging from 0.676 to 0.698), thus lending some support for the reliability of the approach. Overall, committee members endorsed the inclusion of most framework criteria and revealed important areas of discussion, clarification and adaptation of the framework to the needs of the committee.
By promoting systematic consideration of all decision criteria and the underlying evidence, the framework allows a consistent approach to appraising healthcare interventions. Further testing and validation are needed to advance MCDA approaches in healthcare decisionmaking.
PMCID: PMC3248909  PMID: 22129247
8.  Burden of rotavirus gastroenteritis in the pediatric population in Central and Eastern Europe 
Human Vaccines  2011;7(5):523-533.
Rotaviral gastroenteritis (RVGE) is the leading cause of severe diarrhea in children under five years of age worldwide. This comprehensive review aims to estimate the burden of RVGE among children in Central and Eastern Europe.
This literature search captured 38 studies pertaining to RVGE infection in the region. Among children under 15 years of age, RVGE accounted for between 22.0% and 55.3% of all cases of acute gastroenteritis per year. For most countries RVGE was most common in the winter months, although it was reported year round in Bulgaria. Geographical comparison of genotyping data revealed that three genotype combinations, G1P[8], G4P[8] and G2P[4] were present in all countries for which full genotyping data was available. Genotype predominance varied on a season to season basis within each country. Only limited data was available for healthcare resource utilization and economic burden for this region.
An extensive search of the biomedical literature (1999–2009) was conducted in major databases. Studies pertaining to the epidemiology and burden of rotavirus in Central and eastern Europe were captured and data from each country was systematically extracted and compared.
RVGE is a common disease associated with significant morbidity and mortality. While three genotype combinations currently predominate in the region, the dominance of a certain serotype can change dramatically from year to year and from country to country. A vaccination program with broad serotype coverage may help to decrease the burden of RVGE in Central and Eastern Europe.
PMCID: PMC3166495  PMID: 21422818
rotaviral gastroenteritis; burden of illness; Central and Eastern Europe
9.  Burden of rotavirus gastroenteritis in the Middle Eastern and North African pediatric population 
Rotavirus gastroenteritis (RVGE) is the most common cause of severe childhood diarrhea worldwide. Objectives were to estimate the burden of RVGE among children less than five years old in the Middle East (Bahrain, Iran, Iraq, Israel, Jordan, Kuwait, Oman, Qatar, Saudi Arabia, Syria, UAE, Yemen), North Africa (Algeria, Egypt, Libya, Morocco, Tunisia) and Turkey.
A comprehensive literature search was conducted in major databases on the epidemiology and burden of rotavirus among children less than five years old between 1999 and 2009. Data from each country was extracted and compared.
The search identified 43 studies. RVGE was identified in 16-61% of all cases of acute gastroenteritis, with a peak in the winter. RVGE-related hospitalization rates ranged from 14% to 45%, compared to 14%-28% for non-RVGE. Annually, RVGE caused up to 112 fatalities per 100,000 in certain countries in the region. Hospitalization costs ranged from $1.8 to $4.6 million annually, depending on the country. The most recent literature available showed that G1P[8] was the most prevalent genotype combination in 8 countries (range 23%-56%). G2P[4] was most prevalent in 4 countries (26%-48%). G9P[8] and G4P[8] were also frequently detected.
RVGE is a common disease associated with significant morbidity, mortality, and economic burden. Given the variety and diverse rotavirus types in the region, use of a vaccine with broad and consistent serotype coverage would be important to help decrease the burden of RVGE in the Middle East and North Africa.
PMCID: PMC3022719  PMID: 21214934
10.  Economic evaluation of high-dose (80 mg/day) atorvastatin treatment compared with standard-dose (20 mg/day to 40 mg/day) simvastatin treatment in Canada based on the Incremental Decrease in End-Points Through Aggressive Lipid-Lowering (IDEAL) trial 
The Canadian Journal of Cardiology  2009;25(11):e362-e369.
The Incremental Decrease in End-Points Through Aggressive Lipid-Lowering (IDEAL) trial demonstrated incremental cardiovascular benefit of treatment with high-dose atorvastatin (80 mg/day) versus standard-dose simvastatin (20 mg/day to 40 mg/day) in 8888 patients with a previous myocardial infarction (MI) over a median follow-up period of 4.8 years.
To assess the cost-effectiveness of high-dose atorvastatin versus standard-dose simvastatin treatment in patients with a history of MI from a Canadian societal perspective.
In a within-trial analysis, end point-related events, resources used and productivity losses occurring during the IDEAL trial were aggregated by treatment arm on an intention-to-treat basis to calculate the incremental cost per event avoided. Additionally, quality-adjusted survival was projected using a lifetime Markov model. Transition probabilities, workdays lost, use of study medication and cardiovascular hospitalization rates were based on IDEAL trial data. Hospitalization, study medication and productivity costs were included. Probabilistic and deterministic sensitivity analyses were performed.
Compared with standard-dose simvastatin, atorvastatin 80 mg led to 0.099 fewer events per patient and cost savings over 4.8 years of treatment. Over a lifetime horizon, atorvastatin 80 mg led to 0.023 quality-adjusted life years (QALYs) gained per patient at an incremental cost of $26,795/QALY gained. The incremental cost-effectiveness ratio remained below $50,000/QALY in 78% of 1000 simulations. Exclusion of indirect costs resulted in an incremental cost-effectiveness ratio of $38,834/QALY. Results were relatively sensitive to baseline age, but robust with respect to sex, baseline low-density lipoprotein cholesterol levels, diabetes status and hospitalization costs.
From a Canadian societal perspective, high-dose atorvastatin is cost-effective compared with standard-dose simvastatin in patients with a previous MI.
PMCID: PMC2776564  PMID: 19898698
Atorvastatin; Clinical outcomes; Cost-effectiveness; Cost utility; Markov model; Simvastatin
11.  Combining multicriteria decision analysis, ethics and health technology assessment: applying the EVIDEM decisionmaking framework to growth hormone for Turner syndrome patients 
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.
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.
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.
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.
PMCID: PMC2856527  PMID: 20377888
12.  Evidence and Value: Impact on DEcisionMaking – the EVIDEM framework and potential applications 
Healthcare decisionmaking is a complex process relying on disparate types of evidence and value judgments. Our objectives for this study were to develop a practical framework to facilitate decisionmaking in terms of supporting the deliberative process, providing access to evidence, and enhancing the communication of decisions.
Extensive analyses of the literature and of documented decisionmaking processes around the globe were performed to explore what steps are currently used to make decisions with respect to context (from evidence generation to communication of decision) and thought process (conceptual components of decisions). Needs and methodologies available to support decisionmaking were identified to lay the groundwork for the EVIDEM framework.
A framework was developed consisting of seven modules that can evolve over the life cycle of a healthcare intervention. Components of decision that could be quantified, i.e., intrinsic value of a healthcare intervention and quality of evidence available, were organized into matrices. A multicriteria decision analysis (MCDA) Value Matrix (VM) was developed to include the 15 quantifiable components that are currently considered in decisionmaking. A methodology to synthesize the evidence needed for each component of the VM was developed including electronic access to full text source documents. A Quality Matrix was designed to quantify three criteria of quality for the 12 types of evidence usually required by decisionmakers. An integrated system was developed to optimize data analysis, synthesis and validation by experts, compatible with a collaborative structure.
The EVIDEM framework promotes transparent and efficient healthcare decisionmaking through systematic assessment and dissemination of the evidence and values on which decisions are based. It provides a collaborative framework that could connect all stakeholders and serve the healthcare community at local, national and international levels by allowing sharing of data, resources and values. Validation and further development is needed to explore the full potential of this approach.
PMCID: PMC2673218  PMID: 19102752
13.  Methicillin-resistant Staphylococcus aureus: A public health issue with economic consequences 
Methicillin-resistant Staphylococcus aureus (MRSA) has become endemic worldwide in hospitals, and community-associated MRSA is spreading into the community at large.
To estimate the current cost of MRSA in Canada and to assess the magnitude of this public health issue.
An extensive review of the literature was conducted to gather epidemiology, health care resource utilization and cost data for MRSA in Canadian settings. The current MRSA burden was estimated using available cost data and the most recent epidemiology data.
The rate of MRSA in Canadian hospitals increased from 0.46 to 5.90 per 1000 admissions between 1995 and 2004, while community-associated MRSA continued to spread into the community. Patients harbouring MRSA required prolonged hospitalization (average 26 days of isolation per patient), special control measures, expensive treatments and extensive surveillance. Total cost per infected MRSA patient averaged $12,216, with hospitalization being the major cost driver (81%), followed by barrier precautions (13%), antimicrobial therapy (4%) and laboratory investigations (2%). The most recent epidemiological data, combined with available cost data, suggest that direct health care cost attributable to MRSA in Canada, including cost for management of MRSA-infected and-colonized patients and MRSA infrastructure, averaged $82 million in 2004 and could reach $129 million in 2010.
MRSA is a costly public health issue that needs to be tackled if the growing burden of this disease in Canadian hospitals and in the community is to be limited.
PMCID: PMC2542887  PMID: 18923684
Costs; Methicillin-resistant; Staphylococcus aureus; Review

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