Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

Abstract

BackgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.ObjectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.MethodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.ResultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or ‘opportunity cost’-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.ConclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-014-0235-x) contains supplementary material, which is available to authorized users.

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

“…Besides existing healthcare and research infrastructures, physicians collaborating in clinical trials on behalf of their patients and the scientific community producing the basic knowledge on diseases, an increasingly significant role is played by individual patients who share their experiences and risk their own health by participating in research programs [35] . As pointed out by Paulden et al [32] , socioeconomic policies together with access to diagnosis and treatment facilities are key elements in the final success of coverage decision for ODs. Moreover, disease advocacy groups not only contribute to accelerate research and product development, but are generally active in informing patients about new treatments, hence contributing to decrease marketing costs [77] .…”

“…Although there is agreement in considering a broader approach to set the value of pharmaceuticals in general, including their societal value, and regardless of some pilot experiences which are currently under development, there is no internationally agreed-on taxonomy for valuebearing criteria or measuring instruments [26,32,33,[41][42][43] . Despite these limited advances, different factors have been considered for value-bearing pharmaceuticals, including the disease and patient characteristics, the possibility of improvement either in health (effect size for efficacy and safety) or non-health outcomes (procedural attributes) [44] , and the increasingly recognised value of innovation [45,46] .…”

“…Despite these limited advances, different factors have been considered for value-bearing pharmaceuticals, including the disease and patient characteristics, the possibility of improvement either in health (effect size for efficacy and safety) or non-health outcomes (procedural attributes) [44] , and the increasingly recognised value of innovation [45,46] . Though some other issues could be considered in the specific field of RDs and ODs -such as rarity and disease severity, lack of suitable treatment alternatives, evidence of efficacy and safety, degree of product innovation, societal therapeutic impact, and potential drug use for more than 1 indication -, they are not consistently used in value assessments for coverage decisions of ODs [26,32,42,47,48] .…”

“…In the case of ODs, several of the above-reported domains become critical [9,12,[20][21][22][23][24][25] , feeding an open worldwide debate about the appropriateness of existing HTA methods for health technologies devoted to RDs [26][27][28][29] . Increasingly, considerations of using an extended set of criteria beyond the conventional core assessment of safety, efficacy, and cost-effectiveness is gaining acceptance even among the HTA community [30][31][32] .…”

Más allá Del análisis Coste-Efectividad. Precios Basados en el Valor y Financiación Orientada a Resultados Como Vía de Sostenibilidad Para el SNS en España

“…Las primeras propuestas, más ambiguas, proponían la incorporación de ponderaciones sociales en el análisis (50), mientras que posteriormente os nuevos enfoques se orientaron más hacia métodos de evaluación multi-criterio, que ofrecen la oportunidad de incorporar todos los elementos relevantes de un medicamento en la decisión de financiación de una manera estructurada, transparente y consistente. Este tipo de enfoque es especialmente útil en el caso de los medicamentos huérfanos, campo en el que empiezan exister numerosas propuestas prácticas para su implementación (49,(51)(52)(53)(54)(55).…”