Principles for consistent value assessment and sustainable funding of orphan drugs in Europe

Abstract

The European Orphan Medicinal Products (OMP) Regulation has successfully encouraged research to develop treatments for rare diseases resulting in the authorisation of new OMPs in Europe. While decisions on OMP designation and marketing authorisation are made at the European Union level, reimbursement decisions are made at the national level. OMP value and affordability are high priority issues for policymakers and decisions regarding their pricing and funding are highly complex. There is currently no European consensus on how OMP value should be assessed and inequalities of access to OMPs have previously been observed. Against this background, policy makers in many countries are considering reforms to improve access to OMPs. This paper proposes ten principles to be considered when undertaking such reforms, from the perspective of an OMP manufacturer. We recommend the continued prioritisation of rare diseases by policymakers, an increased alignment between payer and regulatory frameworks, pricing centred on OMP value, and mechanisms to ensure long-term financial sustainability allowing a continuous and virtuous development of OMPs. Our recommendations support the development of more consistent frameworks and encourage collaboration between all stakeholders, including research-based industry, payers, clinicians, and patients.

Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs?

“…Current incentives to develop and market orphan drugs do not promote long-term innovation [22] or address some of the pressing concerns regarding treatment affordability for patients and third party payers [23]. In addition, patients may end up paying twice for the same drug through public funds to develop orphan drugs and the cost of treatment [17].…”

“…cost-effectiveness) for the approval and reimbursement of orphan drugs. While some authors argue that the special status argument for public funding and reimbursement of orphan drugs does not stand up to critical assessment [34], others have strengthened the importance of using economic evaluation, political debate [23], and social dialogue [23, 35] to balance distributive justice to orphan drug development and access. Further, the outcome measure of effectiveness, whether premature mortality, quality adjusted life years, achieving a minimum acceptable life expectancy, or any other proposed metrics remains unclear [22, 34].…”

“…The dearth of information on the patients’ health status and overall costs of rare diseases prior to patients gaining access to orphan drugs makes it challenging to assess the cost-effectiveness of orphan drugs. Thus, balancing the cost of economic incentives for the development of orphan drugs against the overall benefits and improvements in health outcomes remains of critical importance [17, 23, 33, 40, 52]. Otherwise, there is a risk of aggravating market failures and perpetuating inefficiencies.…”

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

“…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] .…”

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).…”