Challenges of Health Technology Assessment in Pluralistic Healthcare Systems: An ISPOR Council Report.

Health technology assessment (HTA) has been growing in use over the past 40 years, especially in its impact on decisions regarding the reimbursement, adoption, and use of new drugs, devices, and procedures. In countries or jurisdictions with "pluralistic" healthcare systems, there are multiple payers or sectors, each of which could potentially benefit from HTA. Nevertheless, a single HTA, conducted centrally, may not meet the needs of these different actors, who may have different budgets, current standards of care, populations to serve, or decision-making processes. This article reports on the research conducted by an ISPOR Health Technology Assessment Council Working Group established to examine the specific challenges of conducting and using HTA in countries with pluralistic healthcare systems. The Group used its own knowledge and expertise, supplemented by a narrative literature review and survey of US payers, to identify existing challenges and any initiatives taken to address them. We recommend that countries with pluralistic healthcare systems establish a national focus for HTA, develop a uniform set of HTA methods guidelines, ensure that HTAs are produced in a timely fashion, facilitate the use of HTA in the local setting, and develop a framework to encourage transparency in HTA. These efforts can be enhanced by the development of good practice guidance from ISPOR or similar groups and increased training to facilitate local use of HTA.

EQ-5D-5L Health-State Values for the Mexican Population.

OBJECTIVE: To generate a value set for the Mexican adult general population to support and facilitate the inclusion of quality-adjusted life years (QALYs) into the health technology assessment process of the Mexican healthcare authorities. METHODS: A representative sample of the Mexican adult population stratified by age, sex and socio-economic status was used. Following version 2.0 of the EuroQol EQ-5D-5L valuation protocol, trained interviewers guided participants in completing composite time trade-off (cTTO) and discrete-choice experiment (DCE) tasks included in the EQ-VT software. Generalized least squares, Tobit and Bayesian models were used for cTTO data. The choice of value set model was based on criteria that included: theoretical considerations, parsimony, logical ordering of coefficients, and statistical significance. RESULTS: Based on quality control criteria and interviewer judgment, 1000 out of 1032 participants provided useable responses. Participants' demographic characteristics were similar to the 2010 Mexican Population Census and followed the socioeconomic structure defined by the Mexican Association of Marketing Research and Public Opinion Agencies (AMAI). The predicted index values in the final cTTO model (a heteroscedastic censored model with Bayesian estimation) ranged from - 0.5960 to 1, with 19.7% of all predicted health state scores less than 0 (i.e., worse than dead). CONCLUSION: This study has generated the first value set representing the stated preferences of the Mexican adult population for use in estimating QALYs. The resulting EQ-5D-5L value set is technically robust and will facilitate health economic analyses as well as quality-of-life studies.

[Cost-effectiveness analysis of etanercept compared with other biologic therapies in the treatment of rheumatoid arthritis]. / Etanercept y otras terapias biológicas en artritis reumatoide. Un análisis de costo-efectividad.

OBJECTIVE: to conduct cost-effectiveness analysis of etanercept compared with other biologic therapies in the treatment of moderate or severe rheumatoid arthritis in patients with previous unresponse to immune selective anti-inflammatory derivatives failure. METHODS: a pharmacoeconomic model based on decision analysis to assess the clinical outcome after giving etanercept, infliximab, adalimumab or tocilizumab to treat moderate or severe rheumatoid arthritis was employed. Effectiveness of medications was assessed with improvement rates of 20 % or 70 % of the parameters established by the American College of Rheumatology (ACR 20 and ACR 70). RESULTS: the model showed that etanercept had the most effective therapeutic response rate: 79.7 % for ACR 20 and 31.4 % for ACR 70, compared with the response to other treatments. Also, etanercept had the lowest cost ($149,629.10 per patient) and had the most cost-effective average ($187,740.40 for clinical success for ACR 20 and $476,525.80 for clinical success for ACR 70) than the other biologic therapies. CONCLUSIONS: we demonstrated that treatment with etanercept is more effective and less expensive compared to the other drugs, thus making it more efficient therapeutic option both in terms of means and incremental cost-effectiveness ratios for the treatment of rheumatoid arthritis.

Objetivo: analizar la relación costo-efectividad del etanercept en comparación con otras terapias biológicas para tratar la artritis reumatoide moderada o severa en pacientes con falla previa a fármacos antirreumáticos modificadores de la enfermedad. Métodos: se empleó un modelo farmacoeconómico basado en el análisis de decisiones para valorar la evolución clínica con etanercept, infliximab, adalimumab o tocilizumab para tratar artritis reumatoide moderada o severa. Los parámetros de efectividad fueron las tasas de mejoría igual o superior a 20 % (ACR 20) y de mejoría igual o superior a 70 % (ACR 70). Resultados: etanercept tuvo la mayor efectividad terapéutica: en 79.7 % de los pacientes se observó una ACR 20 y en 31.4 %, una ACR 70. También tuvo el menor costo asociado ($149 629.1 por paciente) y fue más costo-efectiva ($187 740.4 por éxito clínico para obtener ACR 20 y $476 525.8 por éxito clínico para obtener ACR 70) que las demás terapias biológicas. Conclusiones: el etanercept fue el fármaco más efectivo y menos costoso, por lo que resulta la opción terapéutica más eficiente, desde el punto de vista de la relación costo-efectividad media y en términos incrementales para el tratamiento de la artritis reumatoide.

Análisis del costo y la efectividad de los esquemas de administración de factores de coagulación para el manejo de niños con hemofilia A en México / Cost-effectiveness analysis of prophylaxis vs. ''on demand'' approach in the management in children with hemophilia A in Mexico

Introducción. La hemofilia A es causada por la deficiencia del factor VIII. El tratamiento consiste principalmente en aumentar la concentración del FVIII en la sangre utilizando productos de remplazo. El objetivo de este trabajo fue estimar los beneficios clínicos y económicos del manejo profiláctico con factor VIII en niños con hemofilia A en México. Métodos. Se realizó la evaluación económica del manejo profiláctico (PROF) y del tratamiento sobre demanda (SD). Las estrategias comparadas fueron el manejo profiláctico -consistente en FVIII recombinante 25 UI/kg cada tercer día- vs. tratamiento sobre demanda -consistente en FVIII derivado de plasma 40 UI/kg. Se reportó el número de sangrados evitados (SE). Se empleó una tasa de descuento del 5%. Los resultados se expresaron en pesos mexicanos. Resultados. El costo de incremental de PROF respecto a SD fue de $7,727,554 pesos. El manejo con PROF ofrece una reducción de 112 SE frente al manejo de SD (162.9 vs. 50.7). El costo por SE fue de $68,876 pesos. Conclusiones. Un abordaje con PROF mejora la calidad de vida respecto al manejo SD y reduce el número de hemorragias que enfrentan los niños con hemofilia A. PROF es una alternativa costo-efectiva (RCEI $68,876 pesos) para reducir sangrados de acuerdo con la disponibilidad de pago establecida por las autoridades de salud en México.

Background. Hemophilia A is due to a deficiency of factor VIII. Treatment consists primarily of increasing the concentration of FVIII in the blood using replacement products. The aim of this study was to estimate the clinical and economic benefits of prophylactic management with factor VIII in children with hemophilia A in Mexico. We undertook this study to estimate the clinical and economic benefits of prophylactic management (PROF) with factor VIII (FVIII) in children with severe hemophilia in Mexico. Methods. We carried out an economic evaluation of PROF vs. treatment on demand (OD). The strategies compared were management with PROF consisting of recombinant FVIII (rFVIII) 25 IU kg every other day vs. OD management consisting of plasma-derived FVIII (pdFVIII) 40 IU kg. A Markov model was performed with a time horizon of 16 years in patients with severe hemophilia for 2 years, reporting the number of events of bleeding averted (BA). We used a discount rate of 5%. The results are expressed in Mexican pesos (2012). Results. The incremental cost of PROF regarding SD was $7,727,554 pesos. PROF management provides a reduction of 112 BA vs. OD management (162.9 vs. 50.7). Cost per BA was $68,876 pesos. Conclusions. Management with PROF reduces the number of bleeding events facing children with hemophilia A compared to OD management. PROF is a cost-effective alternative to reduce bleeding ($68,876 pesos per BA) according to the willingness to pay established by health authorities in Mexico.

Health care costs of osteopenia, osteoporosis, and fragility fractures in Mexico.

UNLABELLED: Different sources were used to estimate the 2010 health care costs of managing low bone density (osteopenia/osteoporosis) plus caring fragility fractures in Mexico at 411 million USD. Figures are projected to rise 42 % by 2020. Preventive and timely interventions are required to decrease the financial burden of these entities. INTRODUCTION: Osteopenia, osteoporosis, and fragility fractures (FF) are a public health concern. The study purpose was to estimate the health care costs of these conditions in Mexico during 2010 and project them to 2015 and 2020. METHODS: Prevalence of osteopenia and osteoporosis was derived from international data. The Mexican version of FRAX® algorithm was used to assess risk for a major FF (hip, clinical spine, forearm, and proximal humerus) in osteopenic and osteoporotic population aged over 40 years. The estimates were applied to national demographic projections. Only direct medical costs composed by routine non-pharmacological management of osteopenia/osteoporosis besides the costs owing to medical care of major FF were considered into the analysis. Resource use for managing osteopenia/osteoporosis was defined from local sources (clinical practice guidelines, published literature, and expert opinion); unit costs were gathered from official lists. Costs for medical care of FF were based on diagnosis-related groups. RESULTS: In population aged ≥40 years, prevalence of osteopenia and osteoporosis in 2010 was 32.8 and 8 %, respectively. A total of 75,763 FF occurred that year. Costs of managing osteopenia and osteoporosis were 154.9 million USD, whereas medical costs due to FF reached 256.2 million USD. Therefore, the annual health care costs of these entities in 2010 were 411 million USD. Total costs will be 19.2 % higher in 2015, and by 2020, the figures will have increased by 41.7 %. CONCLUSIONS: Low bone density entails substantial epidemiological and financial burden in Mexico, and their impact will grow considerably during the next years.

Economic evaluation of duloxetine as a first-line treatment for painful diabetic peripheral neuropathy in Mexico.

OBJECTIVE: To perform an economic evaluation of duloxetine, pregabalin, and both branded and generic gabapentin for managing pain in patients with painful diabetic peripheral neuropathy (PDPN) in Mexico. RESEARCH DESIGN AND METHODS: The analysis was conducted using a 3-month decision model, which compares duloxetine 60 mg once daily (DUL), pregabalin 150 mg twice daily (PGB), and gabapentin 600 mg three-times daily (GBP) for PDPN patients with moderate-to-severe pain. A systematic review was performed and placebo-adjusted risk ratios for achieving good pain relief (GPR), adverse events (AE), and withdrawal owing to intolerable AE were calculated. Direct medical costs included drug acquisition and additional visits due to lack of efficacy (poor pain relief) or intolerable AE. Unit costs were taken from local sources. Adherence rates were used to estimate the expected drug costs. All costs are expressed in 2010 Mexican Pesos (MXN). Utility values drawn from published literature were applied to health states. The proportion of patients with GPR and quality-adjusted life years (QALY) were assessed. RESULTS: Branded-GBP was dominated by all the other options. PGB was more costly and less effective than DUL. Compared with branded-GBP and PGB, DUL led to savings of 1.01 and 1.74 million MXN (per 1000 patients). The incremental cost per QALY gained with DUL used instead of generic-GBP was $102 433 MXN. This amount is slightly lower than the estimated gross domestic product per capita in Mexico for 2010. During a second-order Monte Carlo simulation, DUL had the highest probability of being cost-effective (61%), followed by generic-GBP (25%) and PGB (14%). LIMITATIONS: Study limitations include a short timeframe and using data from different dosage schemes for GBP and PGB. CONCLUSIONS: This study suggests that DUL provides overall savings and better health outcomes compared with branded-GBP and PGB. Administering DUL rather than generic-GBP is a cost-effective intervention to manage PDPN in Mexico.

Costo Efectividad del Tratamiento de Tumores Neuroendócrinos Pancreáticos Avanzados no Operables con Sunitinib en México.

OBJECTIVES: Sunitinib had showed a substantial clinical benefit in patients with non-resectable pancreatic Neuroendocrine Tumors (NET). The objective of this study was to estimate the cost-effectiveness of sunitinib in the treatment of non-resectable pancreatic NET, from the perspective of the Social Security Mexican Institute (IMSS). METHODS: A Markov model (2-week cycles) was used to estimate the health and economic consequences of sunitinib 37.5mg/day+best supportive care (BSC) regarding placebo+BSC (ten-years horizon, discount rate: 5%). Effectiveness measures were: overall survival (OS), progression-free survival (PFS) and quality adjusted life years (QALY). Resource utilization (BSC, adverse events management, medical follow-up) was estimated through a survey with Mexican oncologists (n=10). Unit costs of medication and medical resources were obtained from institutional sources. Sensitivity analyses were performed and acceptability curves were constructed. RESULTS: Sunitinib+BSC gained 0.49 years (PFS), 1.18 years (OS) and 0.70 QALY against placebo+BSC. Sunitinib+BSC increased medical direct costs (2011 US$) per patient in $20,854, which was driven by acquisition costs of sunitinib and medical follow up before progression. ICER's were $42,157, $17,662 and $29,808 per progression-free year, life-year and QALY gained, respectively, which remained robust through±25% changes in main parameters. At willingness to pay higher than $40,000, $22,400 and $37,600 sunitinib+BSC becomes the most cost-effective alternative in regards to PFS, OS and QALYs, respectively. CONCLUSION: At IMSS, sunitinib+BSC would provide substantial clinical benefits to patients suffering unresectable pancreatic NET, although the latter would increase medical costs of treatment and clinical follow up.

Cost Utility of Sirolimus versus Tacrolimus for the Primary Prevention of Graft Rejection in Renal Transplant Recipients in Mexico.

OBJECTIVE: Therapies for end-stage renal disease improve quality of life, and survival. In Mexico, clinicians often must choose between different therapies without the availability of comparative outcomes evaluation. The present study evaluates the comparative cost-utility of sirolimus (SIR) versus tacrolimus (TAC) for the primary prevention of graft rejection in renal transplant recipients in Mexico. METHODS: We used modeling techniques to estimate the cost-effectiveness of SIR versus TAC to prevent graft rejection in patients with end-stage renal disease in the Mexican setting. The model estimates the cost of quality-adjusted life-year (QALY) per patient. We applied a 20-year horizon (1-year Markov cycles). Cost-effectiveness was expressed in terms of cost per QALY. All costs are presented in 2011 US dollars. Probabilistic sensitivity analyses were conducted. RESULTS: The total cost for the SIR treatment arm over the 20-year duration of the model is estimated to be $136,778. This compares with $142,624 for the TAC treatment arm, resulting in an incremental cost of SIR compared with that of TAC of-$5,846. Over 20 years, SIR was estimated to have 8.18 QALYs compared with 7.33 QALYs for TAC. The resulting incremental utility of SIR compared with that of TAC is 0.84 QALY gained. SIR is estimated to be both less costly and more effective than TAC, indicating that it is the dominant strategy. Notably, results suggest that SIR has a 78% probability of being dominant over the TAC strategy and a 100% probability of having an incremental cost-effectiveness ratio at or below $10,064 (1 GDP) per QALY. CONCLUSIONS: These analyses suggest that in the Mexican setting, the use of SIR in place of TAC for the prevention of graft rejection in this population is likely to be cost saving.

[Economic analysis of parecoxib in the management of postsurgical pain in gynecology]. / Análisis económico de parecoxib en el tratamiento del dolor postoperatorio en ginecología.

BACKGROUND: Poorly managed postoperative pain has a negative impact in healing patients and costs of care. METHODS: A model to estimate economic and health consequences of parecoxib 40 mg and morphine 12 mg regarding ketorolac 30 mg, on the management of postoperative pain in gynecologic laparotomy surgery from the perspective of the Mexican Social Security Institute (IMSS) was developed. A systematic review to identify the proportion of patients that rated their analgesic treatment as "excellent" or "good" in the Patient Global Evaluation of Study Medication, 12 hours after administration of the analgesic (responders), was performed. The patients who rated "fair" or "poor" their treatment were administered additional 4 mg of morphine. Costs in the model correspond to the acquisition costs of analgesics in which the institution would incur. RESULTS: The proportion and cost per responder were: morphine: 14.44% and $192.79, ketorolac: 32.44% and $34.82, parecoxib: 35.51% and $121.25.Treatment with morphine was more expensive and less effective than both, ketorolac and parecoxib, while the cost per additional percent point of responders with parecoxib (compared to ketorolac) was $28.15. For the management of postoperative pain, ketorolac and parecoxib are more effective and less expensive than morphine, additionally parecoxib would be an alternative for patients with contraindication to ketorolac use. CONCLUSION: The management of postoperative pain with parecoxib is more effective and, in the context of IMSS, less expensive than morphine, also constitutes an alternative with a reasonable incremental cost compared to ketorolac.