Abstract
BackgroundSince incentives were introduced to promote orphan drugs in Europe, several dozens of drugs have been registered at the European level. However, patient access on a national level remains very heterogeneous across Europe. This can be explained by healthcare organization and drug reimbursement, which are within the purview of each Member State. We studied access to orphan drugs in France from the patients’ point of view, including marketing but also ease of supply from patients’ perspective, financial and time-based dimensions.ResultsWe identified 91 registered orphan drugs in Europe, corresponding to 115 orphan drug–therapeutic indication pairs. In France, 78.3% (90/115) of these pairs were marketed: 100% were available to inpatients and 75.6% were available to outpatients. The median period between granting of the European marketing authorization and publication of the reimbursement decision was 360 days. The broadest availability—through community pharmacies—was guaranteed in only 31.1% of cases. Prescriptions were mainly restricted either to hospital-based doctors or to specialists. Inpatients were not financially responsible for these prescriptions and 72% of the orphan drug–therapeutic indication pairs available to outpatients were fully covered by national health insurance in France.ConclusionsPatient access to orphan drugs is not universal in France. Access to reimbursement has a strong impact on patients’ effective access to orphan drugs, which may be restricted by difficulties with assessing the clinical value of these drugs and with pricing issues. Prescribing restrictions and drug delivery systems influence the ease of patients’ supply for reimbursed orphan drugs for patients. Patients do not seem to be limited by financial issues, but the growing budgetary impact of orphan drugs is worrisome from a societal point of view.
Highlights
Rare diseases affect around 30 million people in the European Union (EU) and many of them are life-threatening [1]
Our results show that 78.3% of approved orphan drug–therapeutic indication pairs were marketed, among which only 31.1% had the broadest availability and 72% were fully reimbursed for outpatients
As we focused on registered orphan drugs, our study underestimates the real access to drugs for rare diseases in France
Summary
Rare diseases affect around 30 million people in the European Union (EU) and many of them are life-threatening [1]. Inspired by the US Orphan Drug Act, it provides a set of incentives to promote the development and marketing of drugs for serious and rare diseases lacking a satisfactory therapeutic alternative. Drugs are designated as orphan drugs during the development process. They are evaluated to obtain European marketing authorization (MA). Since incentives were introduced to promote orphan drugs in Europe, several dozens of drugs have been registered at the European level. Patient access on a national level remains very heterogeneous across Europe. This can be explained by healthcare organization and drug reimbursement, which are within the purview of each Member State. We studied access to orphan drugs in France from the patients’ point of view, including marketing and ease of supply from patients’ perspective, financial and time-based dimensions
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