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.
Highlights
The growth in the number of approved orphan medicinal products (OMPs) in Europe since the introduction of legislation in 2000 has been a success for rare disease patients, the scientific and medical community, European and national policymakers, and the pharmaceutical industry
While the regulatory framework has been adapted to the specificities of OMPs by creating an expert Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA), many national pricing and reimbursement systems have not been adapted to respond to the specific challenges of these types of drugs
Given the magnitude of the public health challenge in rare diseases it is important that pricing and reimbursement systems for OMPs are reformed to provide more consistent assessment of value and to be more closely aligned with regulatory frameworks for OMPs. This paper addresses this problem from the perspective of a biopharmaceutical manufacturer that has invested – and continues to invest – significantly in OMP research and development
Summary
The growth in the number of approved orphan medicinal products (OMPs) in Europe since the introduction of legislation in 2000 has been a success for rare disease patients, the scientific and medical community, European and national policymakers, and the pharmaceutical industry. Regional and local inequalities of access to OMPs have previously been observed in Europe, where Member States have responsibility for health policy [10,11,12,13] Such variations can be due to restrictions of reimbursement or funding, either because of a negative assessment by policymakers or because the drug has not been considered for reimbursement [10,11,14,15,16,17]. Despite important initiatives to improve conformity of OMP assessment methods for reimbursement purposes at a European level, there is as yet no consensus on how OMP value should be evaluated [20] This uncertainty has important consequences for healthcare policy makers, manufacturers and patients. The ambition is to create a European OMP value framework that rewards innovation and ensures sustainability of the OMP model
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