Independent Registries Are Cost-Effective Tools to Provide Mandatory Postauthorization Surveillance for Orphan Medicinal Products
نویسندگان
چکیده
ObjectivesOrphan medicinal products (OMPs) often receive market authorization under conditions imposed by regulators for ongoing postauthorization surveillance (PAS) to answer questions that remain at the time of entry. This may be provided through industry-funded registries (IFRs). Nevertheless, data in these not sufficient quality and always accessible regulatory review. We propose a mandatory independent registry is an efficient cost-effective tool PAS OMPs.MethodsUsing from Canadian Fabry Disease Initiative, we reviewed costs per unique patient sites participating both national IFRs disease compared completeness Initiative published documents IFRs.ResultsThe collection were 17% 36% (depending on site) lower than collect IFRs, collected was higher IFRs. Data annually guide indications publicly funded therapy. Even when enrollment ceased requirement therapy, 77% patients continued enroll registry, suggesting structure acceptable patients.ConclusionsIndependent are tools should mandated agencies as preferred OMPs. Countries with health systems consider investment infrastructure
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ژورنال
عنوان ژورنال: Value in Health
سال: 2021
ISSN: ['1098-3015', '1524-4733']
DOI: https://doi.org/10.1016/j.jval.2020.10.006