- Email: [email protected]
SF6D VALUE SETS: A SYSTEMATIC REVIEW
A282
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 1 - A 3 1 8
Objectives: Real world evidence (RWE) offers easily accessible information on treatments outside the realm of clinical trials. RWE is gaining significance as it explores various crucial outcomes pertaining to delivery of care in clinical practice. The objective of this study was to assess the trends and utilization of RWE based information in heart failure and asthma to support market access in the US. Methods: A literature review was performed in PubMed to identify studies conducted on heart failure and asthma in a RWE setting in the US. The review included all studies in English, regardless of design. Studies were further stratified by type of RWE data sources, number of individuals, and type of research questions. Results: In total, 805 studies (678 for heart failure indication and 127 for asthma indication) were included in the analyses. In heart failure, the majority of the studies were registries (54%) followed by medical records (16%), claims (15%), and other data sources (14%). However, in asthma, the majority of the studies used claims data (51%), followed by medical records, registries and other data sources which were represented in 20%, 10%, and 19% of studies, respectively. Common research questions addressed in those RWE data sources were epidemiology, burden of illness (humanistic and economic burden), treatment patterns and compliance, resource utilization, unmet needs and data on patient management. Conclusions: RWE covers a large patient pool unlike controlled trials. Multiple research questions can be addressed using RWE, though no single data source is likely to cover all questions. This study concluded that RWE may act as an important instrument to support market access strategies by filling data gaps and by providing more comprehensive real world results. These can be utilized further for the development of budget impact analysis models in the US.
PHP143 ARE NON-RANDOMIZED CONTROLLED STUDIES BEING PUBLISHED IN TOP MEDICAL JOURNALS? Ip Q , Malone D C University of Arizona, Tucson, AZ, USA .
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Objectives: The role of real world evidence (RWE) and comparative effectiveness research (CER) has received increasing attention in recent years. Healthcare decision makers are often informed of these studies and findings through peer-reviewed publications. The purpose of this study was to examine the publication of studies that are not clinical randomized trials in major medical journals. Methods: Table of contents from 5 journals (New England Journal of Medicine (NEJM), Journal of American Medical Association (JAMA), Lancet, Circulation, and the British Medical Journal (BMJ)) were examined from January 2011 to December, 2015 to identify non-randomized controlled trials (non-RCTs). To be classified as a study of interest, the research must have examined clinical and/ or cost outcomes in which interventions are not randomly assigned and involved 2 or more interventions. Non-RCTs were sub-classified into one of the following categories: retrospective cohort or case control; prospective cohort; meta-analysis (MA); network meta-analysis (NMA); modeled cost-effectiveness analysis (CEA); cost-effectiveness with randomized controlled trial (CEA-RCT); cost-consequence analysis (CCA); discrete event simulation (DES); and prediction model derivation/ validation (PM). Descriptive, epidemiological evaluation, association, and trend studies were excluded. Results: A total of 575 publications met inclusion criteria. Journals with the highest to lowest number of studies of interest were BMJ (N= 269), JAMA (N= 102), Circulation (N= 100), Lancet (N= 75), and NEJM (N= 29). The most common study type was meta-analysis (N= 322). Only eight CEA-RCT studies were published during the study time frame. A total of 72 retrospective and 36 prospective cohort studies were published, with 12 of the prospective studies being published in NEJM. For all 5 journals, publication of non-RCTs peaked in 2012. Conclusions: Publication of non-RCT studies is not common, especially in NEJM. Among the five studied journals, the BMJ published the highest number of non-RCT studies and the vast majority were meta-analyses.
PHP144 RANKING COUNTRIES BY QUALITY-ADJUSTED LIFE EXPECTANCY, MEDICAL EXPENDITURES, AND COST EFFECTIVENESS Ghushchyan V 1, Chobanyan A 1, Sullivan P W 2 University of Armenia, Yerevan, Armenia, 2Regis University, Denver, CO, USA .
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1American
Objectives: Quality-Adjusted Life Years (QALYs) are widely used in cost-effectiveness analysis and have been calculated for different population groups and disease states; however, the literature falls short in providing country-specific QALYs or as it is commonly referred quality-adjusted life expectancy (QALE). The aim of this paper is: 1) to develop county-level QALE; 2) to calculate country-specific average costeffectiveness ratios (ACERs); 3) to rank countries based on QALE and ACERs, and 4) to analyze how economic, social and environmental factors affect country-specific QALE. Methods: The Medical Expenditure Panel Survey data was used to estimate the relationship between EQ-5D-3L health utility index, age and some prevalent disease states after controlling for socio-demographic characteristics. Next, these estimates were combined with data on country-specific average life expectancy, disease prevalence (Global burden of disease study) and medical expenditures (World Health Organization) to derive country-specific health utility scores, QALE and ACERs. Finally, using country level regression analysis, we analyze how country-specific social, economic and environmental factors affect the QALE. Results: There were 83 countries in the sample. Countries were ranked based on life expectancy, healthcare expenditures, QALE and ACERs. Adjusted analysis of all countries revealed that those with higher per-capita healthcare expenditures generally had higher QALE. Better sanitation level and larger forest area were associated with greater QALE. In addition, higher CO2 emissions and greater inequality, measured by GINI index, were associated with lower QALE. These factors explained 81% of variations in QLAE. The US had the highest healthcare expenditures per capita; however it ranked 33rd in QALE. As a result, the US had the worst (highest) ACER. Conclusions: Healthcare expenditures have a positive impact on QALE, but are not the only significant
factor. Sanitation, forest area, CO2 emissions and income distribution also have a significant effect on it. PHP145 SF6D VALUE SETS: A SYSTEMATIC REVIEW Poder T G 1, Gandji E W 2 de l’Estrie - CHUS, Sherbrooke, QC, Canada, 2CERDI, Clermont-Fd, France .
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1CIUSSS
Objectives: The SF6D is a preference-based measure designed to calculate QALY. Preference-based measures are standardized multidimensional health state classifications with preference or utility weights elicited from a sample of the population. There is actually a concern that valuations may differ between countries because of differences in culture, thus invalidating the use of values obtained from one country in another. The aim of this article is to assess whether these valuations differ from one country to another for the SF6D. Methods: We performed a systematic review of articles that developed value sets for the SF6D. The data search was conducted in PubMed, ScienceDirect and Scopus. This search was completed by a scanning of the researchgate page of John Brazier, inventor of the SF6D, and of the SF6D webpage hosted by the University of Sheffield. The search was performed up to November 2014 and all languages were considered. The quality of the articles was assessed with the CREATE checklist developed by Xie et al. (2014). Methodological differences were assessed. Results: From a total of 466 articles, 21 were selected. This corresponded to 8 different countries (UK, Spain, Portugal, Brazil, USA, HongKong, Japan and Australia) and to 9 different surveys (2 surveys in Hong-Kong). Most studies used the standard gamble method to elicit health state preferences. Other studies used the discrete choice experiment method (N= 2) or the lottery equivalent method (N= 1). Various econometric methods were used to derive algorithms to calculate QALY. However, when studies used the same elicitation method and the same econometric methods, obvious differences were found in the distribution of values and in the size of the coefficients, revealing different values for health states in different countries. Conclusions: Value sets for the SF6D are different from one country to another and there is a need to develop value sets for more countries to consider cultural differences. PHP146 AVAILABILITY OF COST EFFECTIVENESS INFORMATION ON THE HIGHEST-COST DRUGS AND PROCEDURES IN THE UNITED STATES Bungay K 1, Cohen J T 2, Chambers J 1, Salem M 1, Ciarametaro M 3, Neumann P J 1 1Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies,Tufts Medical Center, Boston, MA, USA, 2Center for the Evaluation of Value and Risk in Health,Tufts Medical Center, Boston, MA, USA, 3National Pharmaceutical Council, Washington, DC, USA .
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Objectives: Cost-effectiveness analyses can help inform formulary and medical benefit decisions, but there are questions about the availability and usefulness of (published, high quality) data. We examined the availability of “usable” costeffectiveness information for prescription drugs and procedures with the highest population-wide annual U.S. expenditures. Methods: We systematically searched the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry, which contains over 4,000 cost-utility analyses and 12,000 cost-per-quality adjusted life year (QALY) ratios published through 2013. We limited the sample to US-focused articles published after 2007 that received a quality rating of 4 or higher on a 1-7 scale. We then reviewed cost-effectiveness ratios for the top 25 pharmaceuticals, ranked by 2014 sales, and the top 25 medical procedures ranked by population-wide expenditures. Results: Most of the nearly 900 ratios did not meet our inclusion criteria (> 75% were non-US studies). We identified 37 ratios for 19 pharmaceuticals. No ratios met the criteria for 4 (16%) top-selling drugs (esomeprazole, glatiramer, tiotropium and celecoxib). In numerous cases, studies of the same drug appeared to report discordant findings. Further investigation revealed that these ratios differed by population or disease stage (e.g., hepatitis with cirrhosis, or without; Crohn’s maintenance treatment, or treatment of severe disease). We identified 52 ratios for 17 top-expenditure procedures. No ratios met the criteria for 8 (32%) of these procedures (e.g., including lumpectomy, appendectomy, tonsillectomy, and bronchoscopy). In many cases, ratios for procedures collectively represented similar interventions. For example, there are multiple ratios reported for “spinal fusion”; these ratios pertain to multiple indications and techniques (cervical or lumbar, different materials or fusion methods). Conclusions: Despite the growing number of published cost effectiveness analyses, in many notable cases, there is a paucity of usable ratios available for US decision makers. PHP147 SELF-REPORTED POPULATION HEALTH BASED ON EUROQOL EQ-5D: POPULATION NORMS FROM ARGENTINA, BRAZIL, CHILE AND URUGUAY Rey-Ares L 1, Kind P 2, Fernandez G 3, Andrade M V 4, Zarate V 5, Perna A 3, Noronha K 4, Augustovski F 1 1Institute for Clinical Effectiveness and Health Policy (IECS), Buenos Aires, Argentina, 2HSE University, St Petersburg, Russia, 3Fondo Nacional de Recursos, Montevideo, Uruguay, 4Federal University of Minas Gerais, Belo Horizonte, Brazil, 5Universidad de los Andes, Santiago, Chile .
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Objectives: To describe self-reported health related quality of life (HRQL) from general population of Argentina, Brazil, Chile and Uruguay and to report population norms based on EQ-5D. Methods: We included data from the 2009 national health risk survey in Argentina (n= 41,392) and from the participants of the EQ-5D valuation studies Brazil (n= 3,362, Viegas Andrade 2013), Chile (n= 2000, Zárate 2011) and Uruguay (n= 792). We estimated mean VAS values and EQ-5D index by age, gender and the presence of limitations in any of the EQ-5D domains. We also explored the most frequent health states (those which include at least 90% of the population). Results: Self-rated mean VAS was 76.5 (IC95% 76.2-76.8), 83.8 (83.3- 84.3), 75.7 (74.8-76.6) and 79.8 (78.6-80.9) in Argentina, Brazil, Chile and Uruguay respectively. The percentage of individuals without limitations was 60.9%, 44.3%, 49.8%, and 44.6% in Argentina, Brazil, Chile and Uruguay respectively. The EQ-5D TTO-based index values showed slightly higher values in Argentina and Uruguay (0.910; CI95%
VA L U E I N H E A LT H 1 9 ( 2 0 1 6 ) A 1 - A 3 1 8
Objectives: Real world evidence (RWE) offers easily accessible information on treatments outside the realm of clinical trials. RWE is gaining significance as it explores various crucial outcomes pertaining to delivery of care in clinical practice. The objective of this study was to assess the trends and utilization of RWE based information in heart failure and asthma to support market access in the US. Methods: A literature review was performed in PubMed to identify studies conducted on heart failure and asthma in a RWE setting in the US. The review included all studies in English, regardless of design. Studies were further stratified by type of RWE data sources, number of individuals, and type of research questions. Results: In total, 805 studies (678 for heart failure indication and 127 for asthma indication) were included in the analyses. In heart failure, the majority of the studies were registries (54%) followed by medical records (16%), claims (15%), and other data sources (14%). However, in asthma, the majority of the studies used claims data (51%), followed by medical records, registries and other data sources which were represented in 20%, 10%, and 19% of studies, respectively. Common research questions addressed in those RWE data sources were epidemiology, burden of illness (humanistic and economic burden), treatment patterns and compliance, resource utilization, unmet needs and data on patient management. Conclusions: RWE covers a large patient pool unlike controlled trials. Multiple research questions can be addressed using RWE, though no single data source is likely to cover all questions. This study concluded that RWE may act as an important instrument to support market access strategies by filling data gaps and by providing more comprehensive real world results. These can be utilized further for the development of budget impact analysis models in the US.
PHP143 ARE NON-RANDOMIZED CONTROLLED STUDIES BEING PUBLISHED IN TOP MEDICAL JOURNALS? Ip Q , Malone D C University of Arizona, Tucson, AZ, USA .
.
.
Objectives: The role of real world evidence (RWE) and comparative effectiveness research (CER) has received increasing attention in recent years. Healthcare decision makers are often informed of these studies and findings through peer-reviewed publications. The purpose of this study was to examine the publication of studies that are not clinical randomized trials in major medical journals. Methods: Table of contents from 5 journals (New England Journal of Medicine (NEJM), Journal of American Medical Association (JAMA), Lancet, Circulation, and the British Medical Journal (BMJ)) were examined from January 2011 to December, 2015 to identify non-randomized controlled trials (non-RCTs). To be classified as a study of interest, the research must have examined clinical and/ or cost outcomes in which interventions are not randomly assigned and involved 2 or more interventions. Non-RCTs were sub-classified into one of the following categories: retrospective cohort or case control; prospective cohort; meta-analysis (MA); network meta-analysis (NMA); modeled cost-effectiveness analysis (CEA); cost-effectiveness with randomized controlled trial (CEA-RCT); cost-consequence analysis (CCA); discrete event simulation (DES); and prediction model derivation/ validation (PM). Descriptive, epidemiological evaluation, association, and trend studies were excluded. Results: A total of 575 publications met inclusion criteria. Journals with the highest to lowest number of studies of interest were BMJ (N= 269), JAMA (N= 102), Circulation (N= 100), Lancet (N= 75), and NEJM (N= 29). The most common study type was meta-analysis (N= 322). Only eight CEA-RCT studies were published during the study time frame. A total of 72 retrospective and 36 prospective cohort studies were published, with 12 of the prospective studies being published in NEJM. For all 5 journals, publication of non-RCTs peaked in 2012. Conclusions: Publication of non-RCT studies is not common, especially in NEJM. Among the five studied journals, the BMJ published the highest number of non-RCT studies and the vast majority were meta-analyses.
PHP144 RANKING COUNTRIES BY QUALITY-ADJUSTED LIFE EXPECTANCY, MEDICAL EXPENDITURES, AND COST EFFECTIVENESS Ghushchyan V 1, Chobanyan A 1, Sullivan P W 2 University of Armenia, Yerevan, Armenia, 2Regis University, Denver, CO, USA .
.
.
.
1American
Objectives: Quality-Adjusted Life Years (QALYs) are widely used in cost-effectiveness analysis and have been calculated for different population groups and disease states; however, the literature falls short in providing country-specific QALYs or as it is commonly referred quality-adjusted life expectancy (QALE). The aim of this paper is: 1) to develop county-level QALE; 2) to calculate country-specific average costeffectiveness ratios (ACERs); 3) to rank countries based on QALE and ACERs, and 4) to analyze how economic, social and environmental factors affect country-specific QALE. Methods: The Medical Expenditure Panel Survey data was used to estimate the relationship between EQ-5D-3L health utility index, age and some prevalent disease states after controlling for socio-demographic characteristics. Next, these estimates were combined with data on country-specific average life expectancy, disease prevalence (Global burden of disease study) and medical expenditures (World Health Organization) to derive country-specific health utility scores, QALE and ACERs. Finally, using country level regression analysis, we analyze how country-specific social, economic and environmental factors affect the QALE. Results: There were 83 countries in the sample. Countries were ranked based on life expectancy, healthcare expenditures, QALE and ACERs. Adjusted analysis of all countries revealed that those with higher per-capita healthcare expenditures generally had higher QALE. Better sanitation level and larger forest area were associated with greater QALE. In addition, higher CO2 emissions and greater inequality, measured by GINI index, were associated with lower QALE. These factors explained 81% of variations in QLAE. The US had the highest healthcare expenditures per capita; however it ranked 33rd in QALE. As a result, the US had the worst (highest) ACER. Conclusions: Healthcare expenditures have a positive impact on QALE, but are not the only significant
factor. Sanitation, forest area, CO2 emissions and income distribution also have a significant effect on it. PHP145 SF6D VALUE SETS: A SYSTEMATIC REVIEW Poder T G 1, Gandji E W 2 de l’Estrie - CHUS, Sherbrooke, QC, Canada, 2CERDI, Clermont-Fd, France .
.
.
.
1CIUSSS
Objectives: The SF6D is a preference-based measure designed to calculate QALY. Preference-based measures are standardized multidimensional health state classifications with preference or utility weights elicited from a sample of the population. There is actually a concern that valuations may differ between countries because of differences in culture, thus invalidating the use of values obtained from one country in another. The aim of this article is to assess whether these valuations differ from one country to another for the SF6D. Methods: We performed a systematic review of articles that developed value sets for the SF6D. The data search was conducted in PubMed, ScienceDirect and Scopus. This search was completed by a scanning of the researchgate page of John Brazier, inventor of the SF6D, and of the SF6D webpage hosted by the University of Sheffield. The search was performed up to November 2014 and all languages were considered. The quality of the articles was assessed with the CREATE checklist developed by Xie et al. (2014). Methodological differences were assessed. Results: From a total of 466 articles, 21 were selected. This corresponded to 8 different countries (UK, Spain, Portugal, Brazil, USA, HongKong, Japan and Australia) and to 9 different surveys (2 surveys in Hong-Kong). Most studies used the standard gamble method to elicit health state preferences. Other studies used the discrete choice experiment method (N= 2) or the lottery equivalent method (N= 1). Various econometric methods were used to derive algorithms to calculate QALY. However, when studies used the same elicitation method and the same econometric methods, obvious differences were found in the distribution of values and in the size of the coefficients, revealing different values for health states in different countries. Conclusions: Value sets for the SF6D are different from one country to another and there is a need to develop value sets for more countries to consider cultural differences. PHP146 AVAILABILITY OF COST EFFECTIVENESS INFORMATION ON THE HIGHEST-COST DRUGS AND PROCEDURES IN THE UNITED STATES Bungay K 1, Cohen J T 2, Chambers J 1, Salem M 1, Ciarametaro M 3, Neumann P J 1 1Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies,Tufts Medical Center, Boston, MA, USA, 2Center for the Evaluation of Value and Risk in Health,Tufts Medical Center, Boston, MA, USA, 3National Pharmaceutical Council, Washington, DC, USA .
.
.
.
.
.
.
.
Objectives: Cost-effectiveness analyses can help inform formulary and medical benefit decisions, but there are questions about the availability and usefulness of (published, high quality) data. We examined the availability of “usable” costeffectiveness information for prescription drugs and procedures with the highest population-wide annual U.S. expenditures. Methods: We systematically searched the Tufts Medical Center Cost-Effectiveness Analysis (CEA) Registry, which contains over 4,000 cost-utility analyses and 12,000 cost-per-quality adjusted life year (QALY) ratios published through 2013. We limited the sample to US-focused articles published after 2007 that received a quality rating of 4 or higher on a 1-7 scale. We then reviewed cost-effectiveness ratios for the top 25 pharmaceuticals, ranked by 2014 sales, and the top 25 medical procedures ranked by population-wide expenditures. Results: Most of the nearly 900 ratios did not meet our inclusion criteria (> 75% were non-US studies). We identified 37 ratios for 19 pharmaceuticals. No ratios met the criteria for 4 (16%) top-selling drugs (esomeprazole, glatiramer, tiotropium and celecoxib). In numerous cases, studies of the same drug appeared to report discordant findings. Further investigation revealed that these ratios differed by population or disease stage (e.g., hepatitis with cirrhosis, or without; Crohn’s maintenance treatment, or treatment of severe disease). We identified 52 ratios for 17 top-expenditure procedures. No ratios met the criteria for 8 (32%) of these procedures (e.g., including lumpectomy, appendectomy, tonsillectomy, and bronchoscopy). In many cases, ratios for procedures collectively represented similar interventions. For example, there are multiple ratios reported for “spinal fusion”; these ratios pertain to multiple indications and techniques (cervical or lumbar, different materials or fusion methods). Conclusions: Despite the growing number of published cost effectiveness analyses, in many notable cases, there is a paucity of usable ratios available for US decision makers. PHP147 SELF-REPORTED POPULATION HEALTH BASED ON EUROQOL EQ-5D: POPULATION NORMS FROM ARGENTINA, BRAZIL, CHILE AND URUGUAY Rey-Ares L 1, Kind P 2, Fernandez G 3, Andrade M V 4, Zarate V 5, Perna A 3, Noronha K 4, Augustovski F 1 1Institute for Clinical Effectiveness and Health Policy (IECS), Buenos Aires, Argentina, 2HSE University, St Petersburg, Russia, 3Fondo Nacional de Recursos, Montevideo, Uruguay, 4Federal University of Minas Gerais, Belo Horizonte, Brazil, 5Universidad de los Andes, Santiago, Chile .
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.
.
.
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.
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Objectives: To describe self-reported health related quality of life (HRQL) from general population of Argentina, Brazil, Chile and Uruguay and to report population norms based on EQ-5D. Methods: We included data from the 2009 national health risk survey in Argentina (n= 41,392) and from the participants of the EQ-5D valuation studies Brazil (n= 3,362, Viegas Andrade 2013), Chile (n= 2000, Zárate 2011) and Uruguay (n= 792). We estimated mean VAS values and EQ-5D index by age, gender and the presence of limitations in any of the EQ-5D domains. We also explored the most frequent health states (those which include at least 90% of the population). Results: Self-rated mean VAS was 76.5 (IC95% 76.2-76.8), 83.8 (83.3- 84.3), 75.7 (74.8-76.6) and 79.8 (78.6-80.9) in Argentina, Brazil, Chile and Uruguay respectively. The percentage of individuals without limitations was 60.9%, 44.3%, 49.8%, and 44.6% in Argentina, Brazil, Chile and Uruguay respectively. The EQ-5D TTO-based index values showed slightly higher values in Argentina and Uruguay (0.910; CI95%