Application of economic evidence in health technology assessment and decision-making for the allocation of health resources in Latin America: Seven key topics and a preliminary proposal for implementation

This technical note1 discusses the application of economic evidence in health technology assessments for decision-making on the allocation of health resources. There is already recognition in Latin America that the economic dimensions of health interventions, such as cost-effectiveness and budgetary impact, are critical dimensions that should always be considered when making decisions about the coverage or inclusion of technologies in benefits packages. However, there are still barriers and constraints that prevent the evaluation of economic evidence in the region from being an integral part of all decision- making processes, with serious implications for the equity and efficiency with which health resources are allocated. The purpose of this technical note is to provide elements and tools that contribute, in a practical way, to overcoming these barriers, answering the questions asked by health systems that are beginning to apply economic evidence in their evaluation and decision-making processes. How do we know if a technology or intervention is cost-effective in our context? What cost-effectiveness threshold should be applied? How might non-economic criteria and dimensions influence our cost-effectiveness threshold? What limit should be considered when a technology implies a high budgetary impact in a particular health system? Given the existing difficulties in generating local economic evidence, what can the economic evidence generated in other jurisdictions tell us? How can economic evidence be taken into account in a fragmented health system? Consideration of these aspects is key to ensuring fairer, more transparent allocation of health resources and thus achieving more efficient and equitable health systems in Latin America

Determining the efficiency path to universal health coverage : cost-effectiveness thresholds for 174 countries based on growth in life expectancy and health expenditures

BACKGROUND: Assessment of the efficiency of interventions is paramount to achieving equitable health-care systems. One key barrier to the widespread use of economic evaluations in resource allocation decisions is the absence of a widely accepted method to define cost-effectiveness thresholds to judge whether an intervention is cost-effective in a particular jurisdiction. We aimed to develop a method to estimate cost-effectiveness thresholds on the basis of health expenditures per capita and life expectancy at birth and empirically derive these thresholds for 174 countries. METHODS: We developed a conceptual framework to assess how the adoption and coverage of new interventions with a given incremental cost-effectiveness ratio will affect the rate of increase of health expenditures per capita and life expectancy at the population level. The cost-effectiveness threshold can be derived so that the effect of new interventions on the evolution of life expectancy and health expenditure per capita is set within predefined goals. To provide guidance on cost-effectiveness thresholds and secular trends for 174 countries, we projected country-level health expenditure per capita and life expectancy increases by income level based on World Bank data for the period 2010-19. FINDINGS: Cost-effectiveness thresholds per quality-adjusted life-year (QALY) ranged between US$87 (Democratic Republic of the Congo) and$95 958 (USA) and were less than 0·5 gross domestic product (GDP) per capita in 96% of low-income countries, 76% of lower-middle-income countries, 31% of upper-middle-income countries, and 26% of high-income countries. Cost-effectiveness thresholds per QALY were less than 1 GDP per capita in 168 (97%) of the 174 countries. Cost-effectiveness thresholds per life-year ranged between $78 and$80 529 and between 0·12 and 1·24 GDP per capita, and were less than 1 GDP per capita in 171 (98%) countries. INTERPRETATION: This approach, based on widely available data, can provide a useful reference for countries using economic evaluations to inform resource-allocation decisions and can enrich international efforts to estimate cost-effectiveness thresholds. Our results show lower thresholds than those currently in use in many countries. FUNDING: Institute for Clinical Effectiveness and Health Policy (IECS)

Quality of life in type 2 diabetes mellitus patients requiring insulin treatment in Buenos Aires, Argentina: a cross-sectional study

Background: Decision-makers have begun to recognize Health-Related Quality of Life (HRQoL) as an important and measurable outcome of healthcare interventions; and HRQoL data is increasingly being used by policy-makers to prioritize health resources. Our objective was to measure HRQoL in a group of Type 2 Diabetes Mellitus (T2DM) patients receiving insulin treatment in Buenos Aires, Argentina. Methods: We conducted a cross-sectional study of patients with T2DM over 21 years of age, treated with either Neutral Protamine Hagedorn (NPH) insulin or Insulin Glargine (IG), who had not changed their baseline schedule in the last 6 months. The recruitment was during 2006–7 in nine private diabetes specialists’ offices in Buenos Aires, Argentina. A standardized diabetes-specific HRQoL questionnaire, the Audit of Diabetes Dependent Quality of Life (ADDQoL), was used. Results: A total of 183 patients were included (93 receiving NPH and 90 receiving IG). The mean QoL score was: 0.98 (SD: 0.89) and the diabetes specific QoL was: -1.49 (SD: 0.90). T2DM had a negative impact on HRQoL with a mean Average Weighted Impact (AWI) score on QoL of -1.77 (SD: 1.58). The greatest negative impact was observed for domains: ‘ worries about the future’ , ‘ freedom to eat’ , ‘ living conditions ’, ‘ sex life’ , and ‘ family life’ . The mean AWI score was -1.71 (SD: 1.48) in patients treated with IG and -1.85 (SD: 1.68) in patients receiving NPH, this difference was not statistically significant. Conclusion: The ADDQoL questionnaire is a tool that can be used in Argentina to measure the QoL of patients with diabetes when evaluating diabetes care programs. The scores of QoL in our selected population did not differ from those reported in high-income countries. We expect that the results of this study will increase healthcare providers’ awareness of patients’ perceived QoL and help to overcome the barriers that delay insulin treatment; mainly clinical inertia and patient resistanc

Challenges faced in transferring economic evaluations to middle income countries

BACKGROUND: Decision makers in middle income countries are using economic evaluations (EEs) in pricing and reimbursement decisions for pharmaceuticals. However, whilst many of these jurisdictions have local submission guidelines and local expertise, the studies themselves often use economic models developed elsewhere and elements of data from countries other than the jurisdiction concerned. The objectives of this study were to describe the current situation and to assess the challenges faced by decision makers in transferring data and analyses from other jurisdictions. METHODS: Experienced health service researchers in each region conducted an interview survey of representatives of decision making bodies from jurisdictions in Asia, Central and Eastern Europe, and Latin America that had at least one year’s experience of using EEs. RESULTS: Representatives of the relevant organizations in 12 countries were interviewed. All 12 jurisdictions had developed official guidelines for the conduct of EEs. All but one of the organizations evaluated studies submitted to them, but 9 also conducted studies and 7 commissioned them. Nine of the organizations stated that, in evaluating EEs submitted to them, they had consulted a study performed in a different jurisdiction. Data on relevant treatment effect was generally considered more transferable than those on prices/unit costs. Views on the transferability of epidemiological data, data on resource use and health state preference values were more mixed. Eight of the respondents stated that analyses submitted to them had used models developed in other jurisdictions. Four of the organizations had a policy requiring models to be adapted to reflect local circumstances. The main obstacles to transferring EEs were the different patterns of care or wealth of the developed countries from which most economic evaluations originate. CONCLUSIONS: In middle income countries it is commonplace to deal with the issue of transferring analyses or data from other jurisdictions. Decision makers in these countries face several challenges, mainly due to differences in current standard of care, practice patterns or GDP between the developed countries where the majority of the studies are conducted and their own jurisdiction

Budget Impact analysis of venetoclax for management of acute myeloid leukemia from the perspective of the social security and the private sector in Argentina

OBJECTIVE: This study aimed to estimate the budget impact of the incorporation of venetoclax for the treatment of patients with Acute Myeloid Leukemia (AML) over 75 years of age or those with comorbidities and contraindications for the use of intensive chemotherapy, from the perspective of the social security and the private third-party payers in Argentina. METHODS: A budget impact model was adapted to estimate the cost difference between the current scenario (azacitidine, decitabine and low doses of cytarabine) and the new scenario (incorporation of venetoclax) for a third-party payer over a time horizon of three years. Input parameters were obtained from a literature review, validated or complemented by expert opinion using a modified Panel Delphi approach. All direct medical costs were estimated by the micro-costing approach and were expressed in US dollars (USD) as of September 2020 (1 USD = 76.18 Argentine pesos). RESULTS: For a third-party payer with a cohort of 1,000,000 individuals covered, incorporating venetoclax was associated with an average budget impact per-member per-month (PMPM) of $0.11 USD for the social security sector and$0.07 USD for the private sector. The duration of treatment with venetoclax was the most influential parameter in the budget impact results. CONCLUSION: The introduction of venetoclax was associated with a positive and slight budget impact. These findings are informative to support policy decisions aimed to expand the current treatment landscape of AML

Measuring the Benefits of Healthcare: DALYs and QALYs – Does the Choice of Measure Matter? A Case Study of Two Preventive Interventions

Abstract Background: The measurement of health benefits is a key issue in health economic evaluations. There is very scarce empirical literature exploring the differences of using quality-adjusted life years (QALYs) or disability-adjusted life years (DALYs) as benefit metrics and their potential impact in decision-making. Methods: Two previously published models delivering outputs in QALYs, were adapted to estimate DALYs: a Markov model for human papilloma virus (HPV) vaccination, and a pneumococcal vaccination deterministic model (PNEUMO). Argentina, Chile, and the United Kingdom studies were used, where local EQ-5D social value weights were available to provide local QALY weights. A primary study with descriptive vignettes was done (n=73) to obtain EQ-5D data for all health states included in both models. Several scenario analyses were carried-out to evaluate the relative importance of using different metrics (DALYS or QALYs) to estimate health benefits on these economic evaluations. Results: QALY gains were larger than DALYs avoided in all countries for HPV, leading to more favorable decisions using the former. With discounting and age-weighting –scenario with greatest differences in all countries– incremental DALYs avoided represented the 75%, 68%, and 43% of the QALYs gained in Argentina, Chile, and United Kingdom respectively. Differences using QALYs or DALYs were less consistent and sometimes in the opposite direction for PNEUMO. These differences, similar to other widely used assumptions, could directly influence decision-making using usual gross domestic products (GDPs) per capita per DALY or QALY thresholds. Conclusion: We did not find evidence that contradicts current practice of many researchers and decision-makers of using QALYs or DALYs interchangeably. Differences attributed to the choice of metric could influence final decisions, but similarly to other frequently used assumptions

Implications of global pricing policies on access to innovative drugs: : the case of trastuzumab in seven Latin American countries

Background: Differential pricing, based on countries’ purchasing power, is recommended by the WHO to secure affordable medicines. However, in developing countries innovative drugs often have similar or even higher prices than in high-income countries. We evaluated the potential implications of trastuzumab global pricing policies in terms of cost-effectiveness (CE), coverage and accessibility for patients with breast cancer in Latin America (LA). Methods: A Markov model was designed to estimate life years (LYs), quality-adjusted life years (QALYs) and costs from a health care perspective. To better fit local cancer prognosis, a base case scenario using transition probabilities from clinical trials was complemented with two alternative scenarios with transition probabilities adjusted to reflect breast cancer epidemiology in each country. Findings: Incremental discounted benefits ranged from 0.87 to 1.00 LY and 0.51 to 0.60 QALY and incremental CE ratios from USD 42,104 to USD 110,283 per QALY (2012 US dollars), equivalent to 3.6 gross domestic product per capita (GDPPC) per QALY in Uruguay and to 35.5 GDPPC in Bolivia. Probabilistic sensitivity analysis showed 0% probability that trastuzumab is CE if the willingness-to-pay (WTP) threshold is one GDPPC per QALY, and remained so at three GDPPC threshold except for Chile and Uruguay (4.3% and 26.6% respectively). Trastuzumab price would need to decrease between 69.6% to 94.9% to became CE in LA. Interpretation: Although CE in other settings, trastuzumab was not CE in LA. The use of health technology assessment to prioritize resource allocation and support price negotiations is critical to making innovative drugs available and affordable in developing countries

Health and economic burden of disease of sugar-sweetened beverage consumption in four Latin American and Caribbean countries : a modelling study

OBJECTIVE: Overweight and obesity are important contributors to the non-communicable disease burden. The consumption of sugar-sweetened beverages (SSBs) has been associated with an increased risk of type 2 diabetes mellitus (T2DM), cardiovascular disease, cancer and other conditions. The objective of this study was to estimate the burden of disease attributable to the consumption of SSBs and the costs to the healthcare systems in Argentina, Brazil, El Salvador, and Trinidad and Tobago. DESIGN: Following a systematic review of models, a comparative risk assessment framework was developed to estimate the health and economic impact associated with the consumption of SSBs. SETTING: Argentina, Brazil, El Salvador, and Trinidad and Tobago. PARTICIPANTS: Overall population. PRIMARY AND SECONDARY OUTCOME MEASURES: The model estimated the effects of SSB consumption on health through two causal pathways: one mediated by body mass index (BMI) and health conditions associated with BMI and another that reflected the independent effects of SSB consumption on T2DM and cardiovascular diseases. RESULTS: The model results indicated that for all four countries, in 1 year, SSB consumption was associated with 18 000 deaths (3.2% of the total disease-related deaths), seven million disease events (3.3% of the total disease-related events), a half-million DALYs and US$2 billion in direct medical costs. This included 1.5 million cases of overweight and obesity in children/adolescents (12% of the excess weight cases) and 2.8 million cases in adults (2.8%); 2.2 million cases of type 2 diabetes (19%); 200 000 cases of heart disease (3.8%); 124 000 strokes (3.9%); 116 000 cases of musculoskeletal disease (0.2%); 102 000 cases of kidney disease (0.9%); and 45 000 episodes of asthma (0.4%). The Trinidad and Tobago population were the most affected by disease events. CONCLUSIONS: The study results indicate that the consumption of SSBs is associated with a significant burden of disease and death in Latin America and the Caribbean