Independent Registries Are Cost-Effective Tools to Provide Mandatory Postauthorization Surveillance for Orphan Medicinal Products
| Autor: | Kaye LeMoine, Syed Wasim, Marie-Françoise Arthus, Michael West, Robin H. Lachmann, Larry D. Lynd, Daniel G. Bichet, Cheryl Rockman-Greenberg, Sandra Sirrs, Carla E. M. Hollak, Chantal F. Morel |
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| Přispěvatelé: | Endocrinology, AGEM - Amsterdam Gastroenterology Endocrinology Metabolism |
| Rok vydání: | 2020 |
| Předmět: |
Canada
Orphan Drug Production postauthorization surveillance Cost-Benefit Analysis registry 03 medical and health sciences 0302 clinical medicine medicine Product Surveillance Postmarketing Humans Enzyme Replacement Therapy 030212 general & internal medicine Registries health care economics and organizations Data collection Actuarial science 030503 health policy & services Health Policy Data Collection Public Health Environmental and Occupational Health Authorization medicine.disease Fabry disease Fabry Disease Business National registry orphan medicinal products 0305 other medical science Healthcare system |
| Zdroj: | Value in health, 24(2), 268-273. Elsevier Limited |
| ISSN: | 1524-4733 1098-3015 |
| Popis: | Objectives Orphan 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 market entry. This surveillance may be provided through industry-funded registries (IFRs). Nevertheless, data in these registries may not be of sufficient quality to answer these questions and may not always be accessible for regulatory review. We propose that a mandatory independent registry is an efficient and cost-effective tool for PAS for OMPs. Methods Using data from the Canadian Fabry Disease Initiative, we reviewed costs per unique patient from sites participating in both the independent national registry and IFRs for Fabry disease and compared data completeness from the Canadian Fabry Disease Initiative to that in published documents from IFRs. Results The costs of data collection through the independent registry were 17% to 36% (depending on site) lower than costs to collect data in the IFRs, and completeness of data collected through the independent registry was higher than that through the IFRs. Data from the independent registry were reviewed annually to guide indications for publicly funded Fabry disease therapy. Even when enrollment ceased to be a requirement to receive therapy, 77% of patients continued to enroll in the registry, suggesting the structure was acceptable to patients. Conclusions Independent registries are cost-effective and efficient tools and should be mandated by regulatory agencies as the preferred tool for PAS for OMPs. Countries with publicly funded health systems should consider investment in registry infrastructure for OMPs. |
| Databáze: | OpenAIRE |
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