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      Cuestiones controvertidas en evaluación económica (III). Intervenciones sanitarias en tres situaciones especiales: enfermedades raras, tratamientos al final de la vida y externalidades en las evaluaciones Translated title: Controversial Issues in Economic Evaluation (III): health Care Interventions in Special Situations

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          Abstract

          El desarrollo de la evaluación económica de intervenciones sanitarias se ha convertido en una herramienta de apoyo en la toma decisiones sobre financiación pública y fijación de precios de nuevas intervenciones sanitarias. La aplicación cada vez más extensiva de estas técnicas ha llevado a la identificación de situaciones particulares en las que, por diversas razones, podría ser razonable tener en cuenta consideraciones especiales a la hora de aplicar los principios generales de evaluación. En este artículo, que cierra una serie de tres, debatiremos, empleando la técnica Metaplan, sobre la evaluación económica en tres situaciones especiales: las enfermedades raras y los tratamientos al final de la vida, así como la consideración de las externalidades en las evaluaciones, apuntando finalmente algunas líneas de investigación futuras para resolver los principales problemas identificados en estos ámbitos.

          Translated abstract

          The development of the economic evaluation of health care interventions has become a support tool in making decisions on pricing and reimbursement of new health interventions. The increasingly extensive application of these techniques has led to the identification of particular situations in which, for various reasons, it may be reasonable to take into account special considerations when applying the general principles of economic evaluation. In this article, which closes a series of three, we will discuss, using the Metaplan technique, about the economic evaluation of health interventions in special situations such as rare diseases and end of life treatments, as well as consideration of externalities in assessments, finally pointing out some research areas to solve the main problems identified in these fields.

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          Most cited references 51

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          Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer.

          The Orphan Drug Act incentivizes medication development for rare diseases, offering substantial financial benefits to the manufacturer. Orphan products constitute most new drug approvals in oncology, but safety and efficacy questions have emerged about some of these agents. To define characteristics of orphan cancer drugs and their pivotal clinical trials and to compare these with nonorphan drugs. We identified all new orphan and nonorphan drugs approved between 2004 and 2010 to treat cancer. We then collected data on key development variables from publicly available information on the US Food and Drug Administration's Web site and in the Code of Federal Regulations. We assessed clinical testing dates, approved indications, and regulatory characteristics (regular vs accelerated review, advisory committee review, postmarketing commitments). We then compared design features (randomization, blinding, primary end point) of pivotal trials supporting approval of orphan and nonorphan drugs and rates of adverse safety outcomes (deaths not attributed to disease progression, serious adverse events, dropouts) in pivotal trials. Fifteen orphan and 12 nonorphan drugs were approved between January 1, 2004, and December 31, 2010. Pivotal trials of orphan drugs had smaller participant numbers (median, 96 [interquartile range {IQR}, 66-152] vs 290 [IQR, 185-394] patients exposed to the drug; P < .001) and were less likely to be randomized (30% vs 80%; P = .007). Orphan and nonorphan pivotal trials varied in their blinding (P = .04), with orphan trials less likely to be double-blind (4% vs 33%). Primary study outcomes also varied (P = .04), with orphan trials more likely to assess disease response (68% vs 27%) rather than overall survival (8% vs 27%). More treated patients had serious adverse events in trials of orphan drugs vs trials of nonorphan drugs (48% vs 36%; odds ratio, 1.72; 95% confidence interval, 1.02-2.92; P = .04). Compared with pivotal trials used to approve nonorphan cancer drugs, pivotal trials for recently approved orphan drugs for cancer were more likely to be smaller and to use nonrandomized, unblinded trial designs and surrogate end points to assess efficacy.
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            Societal views on NICE, cancer drugs fund and value-based pricing criteria for prioritising medicines: a cross-sectional survey of 4118 adults in Great Britain.

            The criteria used by the National Institute for Health and Clinical Excellence (NICE) for accepting higher incremental cost-effectiveness ratios for some medicines over others, and the recent introduction of the Cancer Drugs Fund (CDF) in England, are assumed to reflect societal preferences for National Health Service resource allocation. Robust empirical evidence to this effect is lacking. To explore societal preferences for these and other criteria, including those proposed for rewarding new medicines under the future value-based pricing (VBP) system, we conducted a choice-based experiment in 4118 UK adults via web-based surveys. Preferences were determined by asking respondents to allocate fixed funds between different patient and disease types reflecting nine specific prioritisation criteria. Respondents supported the criteria proposed under the VBP system (for severe diseases, address unmet needs, are innovative--provided they offered substantial health benefits, and have wider societal benefits) but did not support the end-of-life premium or the prioritisation of children or disadvantaged populations as specified by NICE, nor the special funding status for treatments of rare diseases, nor the CDF. Policies introduced on the basis of perceived--and not actual--societal values may lead to inappropriate resource allocation decisions with the potential for significant population health and economic consequences. Copyright © 2012 John Wiley & Sons, Ltd.
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              Propuesta de guía para la evaluación económica aplicada a las tecnologías sanitarias

              La evaluación económica de tecnologías sanitarias se ha convertido en los últimos años en Europa en una herramienta de primer orden para los gestores sanitarios en sus estrategias de asignación de recursos sanitarios y de adopción de nuevas tecnologías. España fue uno de los países pioneros en realizar propuestas de estandarización metodológica aplicable a estudios de evaluación económica. No obstante, la falta de decisión política y de apoyo de los gestores sanitarios a este tipo de herramientas hizo que las propuestas cayeran en desuso. Sin embargo, lo esperable es que a medio plazo sea cada vez más habitual que las nuevas tecnologías sanitarias financiadas por el Sistema Nacional de Salud deban aportar una adecuada evidencia de su valor terapéutico y social en comparación con su coste. Llegado ese momento, los actores del sistema requerirán una serie de reglas claras y consensuadas por parte de los agentes del sistema sobre las cuestiones técnicas o metodológicas que deben respetar los estudios de evaluación de tecnologías sanitarias. Por este motivo, las presentes recomendaciones orientan sobre cómo realizar y analizar los estudios de evaluación económica de calidad. Las recomendaciones aparecen bajo 17 encabezamientos o dominios, y bajo cada recomendación hay además un comentario, en el cual se justifican y discuten las propuestas en relación con otras opciones posibles.
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                Author and article information

                Contributors
                Role: ND
                Role: ND
                Role: ND
                Role: ND
                Journal
                resp
                Revista Española de Salud Pública
                Rev. Esp. Salud Publica
                Ministerio de Sanidad y Consumo (Madrid )
                1135-5727
                June 2015
                : 89
                : 3
                : 237-247
                S1135-57272015000300002

                http://creativecommons.org/licenses/by/4.0/

                Product
                Product Information: SciELO Spain
                Categories
                Health Policy & Services

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