Reflections on the ISPOR Special Task Force on U.S. Value Frameworks: Implications of a Health Economics Approach for Managed Care Pharmacy.

In 2016, The Professional Society for Health Economics and Outcomes Research (ISPOR) formed a special task force (STF) to review approaches and methods to support the definition and use of high-quality U.S. value frameworks. As the leadership group of that initiative, we present our perspective, focusing on implications for the managed care pharmacy community. Our reflections are organized by 9 key observations and conclude with a summary recommendation. We begin by emphasizing the importance of distinguishing among "perspectives" and "decision contexts." Possible perspectives include patient, payer, provider, health care sector, and societal. Decision contexts range from formulary inclusion to guideline development to clinical shared decision making, and multiple perspectives can be taken on each of these decisions. The STF focused on value in the context of including a new medicine in a formulary and, thus, health plan, using a health economics approach that compares marginal benefit (gross value) and marginal (opportunity) cost, yielding the net value. Health care is unique compared with other markets. While economists often use market purchases as indicators of value, they also recognize that this does not work well in health care, since most patent-protected drugs are covered by insurance. To assess the likely health and economic impact, health economists often employ cost-effectiveness analysis, using the quality-adjusted life-year (QALY), a metric that combines mortality and morbidity into a single preference-based index. We strongly endorse the STF's recommendation that payers should use the cost-per-QALY metric as a starting point. However, like the STF, and many of those stakeholders who provided input, we recognize that this metric has some limitations in theory and in practice. Nonetheless, the cost-per-QALY metric is a pragmatic tool that can be augmented to address some of its limitations by integrating other elements of value, particularly those related to uncertainty, such as financial risk protection, health risk protection, the value of hope, real option value, and the value of knowing. The resulting adjusted ratio can be compared with a willingness-to-pay threshold or combined in a measure of net monetary benefit. Alternatively, the array of elements can be valued using multi-criteria decision analysis. We end with the key recommendation that further development and testing of these promising approaches is needed to improve the deliberative process of health technology assessment. DISCLOSURES: No outside funding supported the writing of this article. The authors are leaders of the ISPOR Special Task Force on U.S. Value Frameworks. Willke is employed by ISPOR. Garrison and Neumann have nothing to disclose. The opinions expressed in this article should be considered as belonging only to the authors.

Early benefit assessment for pharmaceuticals in Germany: lessons for policymakers.

Since 2011, Germany's Pharmaceutical Market Restructuring Act has mandated that all newly introduced drugs are subject to an assessment of their benefits in relation to a comparator, typically the current standard treatment. For drugs found to have some additional benefit, the manufacturer and the statutory health insurers negotiate a price. For drugs found to have no additional benefit, their price is set in reference to the price of the comparator. This new system is intended to reduce spending on expensive new drugs that are no more effective than existing treatments, while encouraging pharmaceutical companies to invest in innovative drugs that improve health outcomes. The German experience provides lessons for the United States, where comparative effectiveness research is publicly funded but public insurance programs are limited in their ability to use its findings to make coverage or pricing decisions.

Impacto de las recomendaciones del Sistema Español de Farmacovigilancia en la publicación de casos de reacciones adversas a medicamentos / Impact of the spanish pharmacovigilance system recommendation son the publication of cases of adverse drug reactions

Objetivo. Evaluar el impacto de lasrecomendaciones que el Comité Técnico delSistema Español de Farmacovigilancia envióa los editores de revistas médicas españolasen el año 2004 sobre la información mínimaque deberían contener los casos publicadosde reacciones adversas a medicamentos.Diseño. Estudio observacional.Emplazamiento. Base de datos nacional desospechas de reacciones adversas (FEDRA).Participantes. Notificaciones de publicacionesde sospechas de reacciones adversasregistradas en FEDRA y publicadas enlos años previo (2003) y posterior (2005)al envío de las recomendaciones.Mediciones principales. Información en losapartados: sexo, edad, dosis, motivo de laprescripción, duraciones del tratamiento y dela reacción, secuencia temporal, efecto de laretirada y causas alternativas de laspublicaciones de sospechas de reaccionesadversas registradas en FEDRA. Secompararon los resultados de los 2 años.Resultados. No se observaron diferenciassignificativas en la información contenidaen las publicaciones de casos de reaccionesadversas entre los años 2003 y 2005.Los apartados que con mayor frecuenciapresentaban información incompleta eranlas dosis y duraciones de los tratamientosy de la reacción adversa. Sólo un tercio,aproximadamente, de las publicacionescontenían información completa.Conclusiones. Es necesario mejorarel contenido de la información de laspublicaciones de casos de reacciones adversasa medicamentos para que esta mejordocumentación contribuya a mejorarla evaluación de las señales de alerta

Objective. To assess the impact of therecommendations that, the TechnicalCommittee of the SpanishPharmacovigilance System sent, in 2004,to the editors of Spanish medical journalson the minimum information required forpublication of adverse drug reaction casereports.Design. Observational study.Setting. National suspected adverse drugreactions database (FEDRA).Participants. Published adverse drug reactionreports registered in FEDRA, publishedin the years before (2003) and after (2005)the recommendations were issued.Main measurements. The following dataelements were analysed: sex, age, dose,disease treated with the suspecteddrug, length of treatment and adverse drugreaction, temporal sequence, withdrawaleffect, and alternative causes. The resultsof the 2 years were compared.Results. The information in the case reportspublished between years 2003 and 2005was not significantly different. The dataelements more often incomplete were dose,length of treatment, as well as length ofadverse reaction. Approximately one thirdof the published case reports included fullinformation.Conclusions. There seems to be a needto improve the data elements contentof published adverse drug reactions casereports, so that such documentation can contribute to improve the assessment of alert signals (AU)