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Las enfermedades raras son aquellas que tienen baja prevalencia y que, por lo tanto, el desarrollo de medicamentos para tratarlas no es rentable para las empresas farmacéuticas debido a la baja demanda. A pesar de que ya se cuenta con diferentes políticas públicas alrededor del mundo para incentivar a las industrias farmacéuticas a investigar estos medicamentos, conocidos como medicamentos huérfanos, su desarrollo conlleva muchas dificultades en las evaluaciones clínicas y el precio final para el público es muy elevado. Si bien en años recientes se ha planteado el uso de tecnología de impresión en 3D para producir estos medicamentos o incluso recurrir a otros medicamentos previamente aprobados para tratar enfermedades raras, existe un historial de mal uso de las legislaciones por parte de las empresas con el fin de generar beneficios comerciales, por lo que estas políticas deben reforzarse para que cumplan su propósito; ayudar a una población muy vulnerable. El objetivo del presente texto es exponer los resultados de una revisión documental sobre el panorama científico y sociopolítico en el que se encuentra el problema de las enfermedades raras y los medicamentos huérfanos, así como las posibles soluciones que se están desplegando para abordarlo. Deriva de un estudio que se desarrolla en el momento actual en la Universidad Autónoma Metropolitana, de Ciudad de México.

The strange illnesses are those that have low prevalence and that, therefore, the development of medications to treat them is not profitable for the pharmaceutical companies due to the drop demands. Although it is already counted with different political public around the world to motivate to the pharmaceutical industries to investigate these medications, well-known as orphan medications, their development bears many difficulties in the clinical evaluations and the final price for the public it is very high. Although in recent years he/she has thought about the use of impression technology in 3D to produce these medications or even to appeal to other medications previously approved to treat strange illnesses, a record of wrong use of the legislations exists on the part of the companies with the purpose of generating commercial benefits, for what these politicians should be reinforced so that they complete its purpose; to help a very vulnerable population. The objective of the present text is to expose the results of a documental revision on the scientific and sociopolitical panorama in which is the problem of the strange illnesses and the orphan medications, as well as the possible solutions that they are spreading to approach it. It derives of a study that is developed in the current moment in the Metropolitan Autonomous University, of Mexico City.

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Rotavirus (RV) infection causes acute rotavirus gastroenteritis (RVGE) in infants. Safe and effective RV vaccines are available, of which Mexico has included one in its national immunization program (NIP) since 2007. Health outcome gains, expressed in quality-adjusted life years (QALYs), and cost improvements are important additional factors for the selection of a NIP vaccine. These two factors were analyzed here for Mexico over one year implementing three RV vaccines: 2-dose Rotarix (HRV), versus 3-dose RotaTeq (HBRV), and 3-dose Rotasiil (BRV-PV), presented in a 1-dose or 2-dose vial). HRV would annually result in discounted QALY gains of 263 extra years compared with the other vaccines by averting an extra 24,022 homecare cases, 10,779 medical visits, 392 hospitalizations, and 12 deaths. From a payer's perspective and compared with HRV, BRV-PV 2-dose vial and BRV-PV 1-dose vial would annually result in $13,548,179 and $4,633,957 net savings, respectively, while HBRV would result in $3,403,309 extra costs. The societal perspective may also show savings compared with HRV for BRV-PV 2-dose vial of $4,875,860, while BRV-PV 1-dose vial and HBRV may show extra costs of $4,038,363 and $12,075,629 respectively. HRV and HBRV were both approved in Mexico, with HRV requiring less investment than HBRV with higher QALY gains and cost savings. The HRV vaccine produced those higher health gains due to its earlier protection and greater coverage achieved after its schedule completion with two doses only, providing full protection at four months of age instead of longer periods for the other vaccines.

Rotavirus (RV) infection causes acute diarrhea in infants and can be life-threatening. Several safe and effective vaccines against RV and its complications exist. For many governments choosing vaccines for national immunization programs, total costs or savings and health gains are important factors in the selection process. We compared the costs and health benefits of three RV vaccines for Mexico: HRV, HBRV, and BRV-PV, that have different dosing schedules: two doses for HRV and three doses for HBRV and BRV-PV. HRV is currently part of the national immunization program in Mexico. HRV would result in more health benefits as it incurs fewer RV-related cases, medical visits, hospitalizations, and infant deaths than the other vaccines due to its early protection achieved after only two doses to complete its schedule. However, from a payer's perspective, the least expensive vaccine was BRV-PV, while HRV was less expensive than HBRV. From a societal perspective, also accounting for families' costs and loss in income due to an infant's RV disease, and the families' costs and loss in income when accompanying the infant to the vaccination center, the HRV vaccine was less expensive than HBRV and BRV-PV presented in a 1-dose vial, while more expensive than BRV-PV presented in a 2-dose vial. HRV and HBRV are both approved in Mexico, although HBRV requires a greater investment at lower health benefits than HRV, from both a payer's and a societal perspective. A 2-dose vaccination scheme is an important asset for the economic value of this vaccination program.

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OBJECTIVES: This study aimed to determine the cost-utility of ocrelizumab versus rituximab in patients with RRMS, from the perspective of the Colombian healthcare system. METHODOLOGY: Cost-utility study based on a Markov model, with a 50-year horizon and payer perspective. The currency was the US dollar for the year 2019, with a cost-effectiveness threshold of $5180 defined for Colombian health system. The model used annual cycles according to the health status determined by the disability scale. Direct costs were considered, and the incremental cost-effectiveness ratio per 1 quality-adjusted life-year (QALY) gained was used as the outcome measure. A discount rate of 5% was applied to costs and outcomes. Multiple one-way deterministic sensitivity analyses and 10 000 Monte Carlo simulation were conducted. RESULTS: For the treatment of patients with RRMS, ocrelizumab versus rituximab had an incremental cost-effectiveness ratio of $73 652 for each QALY gained. After 50 years, 1 subject treated with ocrelizumab earns 4.8 QALYs >1 subject treated with rituximab, but at a higher cost of $521 759 versus $168 752, respectively. Ocrelizumab becomes a cost-effective therapy if its price is discounted > 86% or if there is a high willingness to pay. CONCLUSIONS: Ocrelizumab was not a cost-effective drug as compared with rituximab in treating patients with RRMS in Colombia.

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BACKGROUND: Preference heterogeneity in health valuation has become a topic of greater discussion among health technology assessment agencies. To better understand heterogeneity within a national population, valuation studies may identify latent groups that place different absolute and relative importance (i.e., scale and taste parameters) on the attributes of health profiles. OBJECTIVE: Using discrete choice responses from a Peruvian valuation study, we estimated EQ-5D-5L values on a quality-adjusted life-year (QALY) scale accounting for latent heterogeneity in scale and taste, as well as controlling heteroskedasticity at task level variation. METHOD: We conducted a series of latent class analyses, each including the 20 main effects of the EQ-5D-5L and a power function that relaxes the constant proportionality assumption (i.e., discounting) between value and lifespan. Taste class membership was conditional on respondent-specific characteristics and their experience with the composite time trade-off (cTTO) tasks. Scale class membership was conditional on behavioral characteristics such as survey duration and self-stated difficulty level in understanding tasks. Each analysis allowed the scale factor to vary by task type and completion time (i.e., heteroskedasticity). RESULTS: The results indicated three taste classes: a quality-of-life oriented class (33.35%) that placed the highest value on levels of severity, a length-of-life oriented class (26.72%) that placed the highest value on lifespan, and a middle class (39.71%) with health attribute effects lower than the quality class and lifespan effect lower than the length-of-life oriented class. The EQ-5D-5L values ranged from - 2.11 to 0.86 (quality-of-life oriented class), from - 0.38 to 1.02 (middle class), and from 0.36 to 1.01 (length-of-life oriented class). The likelihood of being a member of the quality-of-life class was highly dependent on whether the respondent completed the cTTO tasks (p-value < 0.001), which indicated that the cTTO tasks might cause the Peru respondents to inflate the burden of health problems on a QALY scale compared to those who did not complete the cTTO tasks. The results also showed two scale classes as well as heteroskedasticity within each scale class. CONCLUSION: Accounting for taste and scale classes simultaneously improveds understanding of preference heterogeneity in health valuation. Future studies may confirm the differences in taste between classes in terms of the effect of quality of life and lifespan attributes. Furthermore, confirmatory evidence is needed on how behavioral variables captured within a study protocol may enhance analyses of preference heterogeneity.

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BACKGROUND: Early pregnancy loss, also referred to as miscarriage, is common, affecting approximately 1 million people in the United States annually. Early pregnancy loss can be treated with expectant management, medications, or surgical procedures-strategies that differ in patient experience, effectiveness, and cost. One of the medications used for early pregnancy loss treatment, mifepristone, is uniquely regulated by the Food and Drug Administration. OBJECTIVE: This study aimed to compare the cost-effectiveness from the healthcare sector perspective of medical management of early pregnancy loss, using the standard of care medication regimen of mifepristone and misoprostol, with that of office uterine aspiration. STUDY DESIGN: We developed a decision analytical model to compare the cost-effectiveness of early pregnancy loss treatment with medical management with that of office uterine aspiration. Data on medical management came from the Pregnancy Failure Regimens randomized clinical trial, and data on uterine aspiration came from the published literature. The analysis was from the healthcare sector perspective with a 30-day time horizon. Costs were in 2018 US dollars. Effectiveness was measured in quality-adjust life-years gained and the rate of complete gestational sac expulsion with no additional interventions. Our primary outcome was the incremental cost per quality-adjust life-year gained. Sensitivity analysis was performed to identify the key uncertainties. RESULTS: Mean per-person costs were higher for uterine aspiration than for medical management ($828 [95% confidence interval, $789-$868] vs $661 [95% confidence interval, $556-$766]; P=.004). Uterine aspiration more frequently led to complete gestational sac expulsion than medical management (97.3% vs 83.8%; P=.0001); however, estimated quality-adjust life-years were higher for medical management than for uterine aspiration (0.082 [95% confidence interval, 0.8148-0.08248] vs 0.079 [95% confidence interval, 0.0789-0.0791]; P<.0001). Medical management dominated uterine aspiration, with lower costs and higher confidence interval. The probability that medical management is cost-effective relative to uterine aspiration is 97.5% for all willingness-to-pay values of ≥$5600/quality-adjust life-year. Sensitivity analysis did not identify any thresholds that would substantially change outcomes. CONCLUSION: Although office-based uterine aspiration more often results in treatment completion without further intervention, medical management with mifepristone pretreatment costs less and yields similar quality-adjust life-years, making it an attractive alternative. Our findings provided evidence that increasing access to mifepristone and eliminating unnecessary restrictions will improve early pregnancy care.

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RESUMEN Introducción: Villa Clara es la provincia más envejecida del país, pues el adulto medio ocupa un alto porciento dentro de su población. Durante esta etapa de la vida, si bien algunos cambios fisiológicos se deben a factores genéticos y biológicos personales, también es importante considerar la influencia del modo de vida, experimentado en la actual calidad de vida del adulto medio, que puede ser mejorada con la actividad física. Objetivos: Diseñar una multimedia como herramienta educativa e instructiva para mejorar la calidad de vida del adulto medio, en la Facultad de Tecnología de la Salud-Enfermería, de la Universidad de Ciencias Médicas de Villa Clara. Métodos: Se realizó un estudio de tipo innovación tecnológica en el período de septiembre de 2018 a septiembre de 2019. El universo de estudio estuvo constituido por 397 profesores, que constituyen el total de docentes a tiempo completo; la muestra fue de 40 profesores. Como criterio de exclusión se consideró a los que no estaban ubicados en el rango de edad correspondiente con el adulto medio. Las variables fueron: edad, peso, talla, sexo, ejercicios físicos, enfermedades crónicas no transmisibles y tipo de alimentación. Se utilizaron métodos teóricos, empíricos y de nivel matemático-estadístico. Resultados: El producto final proporcionó beneficios, pues con él se motivó al adulto medio hacia el desarrollo de habilidades y capacidades que le permitan tomar decisiones correctas para elevar la calidad de vida. Conclusiones: La multimedia educativa constituye una vía novedosa y motivadora para influir en la calidad de vida del adulto medio.

ABSTRACT Introduction: Villa Clara is the oldest province in the country, since middle-aged adults occupy a high percentage of its population. During this stage of life, although some physiological changes are due to individuals' genetic and biological factors, it is also important to consider the influence of lifestyle, experienced in the current quality of life in middle-aged adults, which can be improved with physical activity. Objective: to design a multimedia, at the Faculty of Health Technology and Nursing in the University of Medical Sciences of Villa Clara, as an educational and instructive tool to improve the quality of life in middle-aged adults. Methods: a technological innovation study was carried out from September 2018 to September 2019. The universe of study consisted of 397 teachers, who constituted the total number of full-time teachers; the sample was 40 teachers. Those who were not in the age range corresponding to middle-aged adults were considered as an exclusion criterion. Age, weight, height, gender, physical exercises, non-communicable chronic diseases and type of diet were among the variables studied. Theoretical, empirical and mathematical-statistical methods were used. Results: the final product provided benefits, since middle-aged adults were motivated towards the development of skills and abilities, allowing them to make correct decisions in order to improve their quality of life. Conclusions: educational multimedia is a novel and motivating way to influence the quality of life in middle-aged adults.

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BACKGROUND: Preoperative opioid is associated with poor postoperative outcomes for several surgical specialties, including neurosurgical, orthopedic, and general surgery. Patients with symptomatic adult spinal deformity (SASD) are among the highest patient populations reporting opioid use prior to surgery. Surgery for SASD has been demonstrated to improve patient reported quality of life, however, little medical economic data exists evaluating impact of preoperative opioid use upon surgical cost-effectiveness for SASD. The purpose of this study was to evaluate the impact that preoperative opioid use has upon SASD surgery including duration of intensive care unit (ICU) and hospital stay, postoperative complications, patient reported outcome measures (PROMs), and surgical cost-effectiveness using a propensity score matched analysis model. METHODS: Surgically treated SASD patients enrolled into a prospective multi-center SASD study were assessed for preoperative opioid use, and divided into two cohorts; preoperative opioid users (OPIOID) and preoperative opioid non-users (NON). Propensity score matching (PSM) was used to control for patient age, medical comorbidities, spine deformity type and magnitude, and surgical procedures for OPIOID vs NON. Preoperative and minimum 2-year postoperative PROMs, duration of ICU and hospital stay, postoperative complications, and opioid use at one and two years postoperative were compared for OPIOID vs NON. Preoperative, one year, and minimum two-year postoperative SF6D values were calculated, and one- and two-year postoperative QALYs were calculated using SF6D change from baseline. Hospital costs at the time of index surgery were calculated and cost/QALY compared at one and two years postop for OPIOID vs NON. RESULTS: 261/357 patients (mean follow-up 3.3 years) eligible for study were evaluated. Following the PSM control, OPIOID (n=97) had similar preoperative demographics, smoking and depression history, spine deformity magnitude, and surgery performed as NON (n=164; p>0.05). Preoperatively, OPIOID reported greater NRS back pain (7.7 vs 6.7) and leg pain (5.2 vs 3.9), worse ODI (50.8 vs 36.9), worse SF-36 PCS (28.8 vs 35.6), and worse SRS-22r self-image (2.3 vs 2.5) than NON, respectively (p<0.05). OPIOID had longer ICU (41.2 vs 21.4 hours) and hospital stay (10.6 vs 8.0 days) than NON, respectively (p<0.05). At last postoperative follow up, OPIOID reported greater NRS back pain (4.1 vs 2.3) and leg pain (2.9 vs 1.7), worse ODI (32.4 vs 19.4), worse SF-36 PCS (37.4 vs 47.0), worse SRS-22r self-image (3.5 vs 4.0), and lower SRS-22r treatment satisfaction score (2.5 vs 4.5) than NON, respectively (p<0.05). At last follow-up postoperative Cost/QALY was higher for OPIOID ($44,558.31) vs NON ($34,304.36; p<0.05). At last follow up OPIOID reported greater postoperative opioid usage than NON [41.2% vs. 12.9%, respectively; odds ratio =4.7 (95% CI=2.6-8.7; p<0.05)]. CONCLUSIONS: Prospective, multi-center, matched analysis demonstrated SASD patients using opioids prior to SASD surgery reported worse preoperative and postoperative quality of life, had longer ICU and hospital stay, had less cost effectiveness of SASD surgery. Preoperative opioid users also reported lower treatment satisfaction, and reported greater postoperative opioid use than non-users. These data should be used to council patients on the negative impact preoperative opioid use can have on SASD surgery.

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BACKGROUND: Chronic obstructive pulmonary disease (COPD) is an inflammatory lung disease characterized by long-term breathing problems and airflow limitations. International guidelines recommend using bronchodilators like long-acting beta- and muscarinic antagonists, and inhalational corticosteroids. OBJECTIVES: The cost-effectiveness of single-inhaler triple therapy containing fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) was compared to the treatments Fluticasone Furoate/Vilanterol (FF/VI), Umeclidinio/Vilanterol (UMEC/VI) and Fluticasone Propionate 250 mcg/Salmeterol 25mcg + Tiotropio 18 mcg (FP/SAL/TIO) for patients with COPD from the Chilean public health system perspective. METHODS: A cost-effectiveness analysis was performed, including a deterministic and probabilistic sensitivity analysis over a 25-year time horizon. Two scenarios were assessed to study the effect of a 3%-discount for costs and outcomes on FF/UMEC/VI. RESULTS: The incremental cost-effectiveness (ICER) of FF/UMEC/VI versus FF/VI was $10,076/QALY, being a cost-effective alternative to a threshold of one Gross Domestic Product per capita (GDPpc), while versus FP/SAL/TIO the ICER increased to $50,288/QALY, showing to be a non-cost effective alternative to 1 GDPpc, but at a threshold of 3 GDPpc. CONCLUSION: FF/UMEC/VI appears to be a cost-effective intervention for treating COPD compared to FF/VI. However, FF/UMEC/VI compared to FP/SAL/TIO showed an ICER above the threshold of 1 GDPpc, but, in comparison with lower price, the ICER was below 3 GDPpc.

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Resumen Introducción: El mercurio circula por el aire; persiste en suelos, sedimentos y agua, y causa efectos en la salud humana. Las mujeres en edad fértil y los neonatos son la población más vulnerable. Objetivo: Analizar las evidencias sobre la carga de enfermedad ocasionada por la exposición a mercurio, así como el impacto económico sobre el sistema de salud. Metodología: Revisión de alcance de la literatura, de las bases de datos PUBMED y EPISTEMONIKOS, búsqueda manual de documentos técnicos de entidades oficiales de diferentes continentes. Resultados: Se identificaron 311 registros en bases de datos y 4 en búsqueda manual en entidades oficiales; 19 artículos fueron incluidos. Discusión: Predomina la afectación del desarrollo neurológico y cognitivo en niños de madres expuestas y lactantes. Los costos se midieron por la pérdida del coeficiente intelectual. Conclusión: Efectos en salud por la exposición a metilmercurio se traducen en gastos para la sociedad y los sistemas de salud.

Abstract Introduction: Mercury circulates through the air, persists in soils, sediments and water, and can affect human health. Women of childbearing age and newborns are the most vulnerable population. Objective: To analyze the evidence on the burden of disease caused by mercury exposure, as well as the economic impact on the health system. Methodology: Review of the literature, PUBMED and EPISTEMONIKOS databases, manual search of technical documents of official entities from different continents. Results: A total of 311 records were identified in databases and four in manual searches from official entities; 19 articles were included. Discussion: Neurological and cognitive development in children of exposed mothers and infants are more predominant. Costs were measured by IQ loss. Conclusion: Health effects of methylmercury exposure translate into costs for society and health systems.

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OBJECTIVES: Although several randomized clinical trials performed in children 6 years and older with Omalizumab as add-on therapy have reported improvements in diverse clinical outcomes, the evidence regarding its cost effectiveness is not sufficient, especially in less-affluent countries, where the clinical and economic burden of the disease is the greatest. The aim of the present study was to perform a cost-utility analysis of adding omalizumab to standard treatment for treating pediatric patients with uncontrolled severe allergic asthma in Colombia, a middle-income country (MIC). METHODS: A Markov-type model was developed to estimate costs and health outcomes of a simulated cohort of pediatric patients with persistent asthma treated over a 5-year period. The effectiveness data and transition probabilities were obtained from various sources, including systematic reviews with meta-analysis. Cost data were obtained from official databases provided by the Colombian Ministry of Health. The study was carried out from the perspective of the national healthcare system in Colombia. The main outcome was the variable ''quality-adjusted life-years'' (QALYs). RESULTS: For the base-case analysis, the cost-utility analysis showed that compared with the standard treatment strategy, the omalizumab strategy involved higher costs (US$72,142.3 vs. $20,243.4 average cost per patient) and greater gain in QALYs (0.8718 vs. 0.8222 QALYs on average per patient). The incremental cost-utility ratio (ICUR) of omalizumab compared with standard treatment was US$82,748.1 per QALY CONCLUSIONS: This study shows that in Colombia, an MIC, compared with standard treatment, omalizumab is not a cost-effective strategy for treating pediatric patients with uncontrolled severe allergic asthma.

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