@article{5f06a12e264f4eb88cac22b462172bd4,
title = "Independent Registries Are Cost-Effective Tools to Provide Mandatory Postauthorization Surveillance for Orphan Medicinal Products",
abstract = "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.",
keywords = "Fabry disease, orphan medicinal products, postauthorization surveillance, registry",
author = "Sirrs, {Sandra M.} and Marie-Francoise Arthus and Bichet, {Daniel G.} and Cheryl Rockman-Greenberg and Kaye LeMoine and Morel, {Chantal F.} and Robin Lachmann and Lynd, {Larry D.} and Syed Wasim and West, {Michael L.} and Carla Hollak",
note = "Funding Information: Author Contributions: Concept and design: Sirrs, Rockman-Greenberg, Lynd, West, Hollak, Acquisition of data: Sirrs, Arthus, Bichet, Morel, Lachmann, Wasim, Hollak, Analysis and interpretation of data: Sirrs, Arthus, Bichet, West, Hollak, Drafting of Manuscript: Sirrs, Arthus, Bichet, Lachmann, Wasim, Hollak, Critical revision of paper for content: Sirrs, Rockman-Greenberg, Lemoine, Morel, Lachmann, Lynd, West, Hollak, Statistical analysis: Sirrs, Provision of study materials or patients: Sirrs, Arthus, Bichet, Rockman-Greenberg, Lemoine, Morel, West, Administrative, technical, or logistic support: Sirrs, Lemoine, Wasim, Supervision: Lynd, Conflict of Interest Disclosures: Dr Sirrs reported receiving grants and personal fees from Sanofi Genzyme, Takeda, and Amicus Therapeutics outside the submitted work, and grants from Actelion outside the submitted work. Dr Bichet reported receiving grants and personal fees from Amicus Therapeutics and Sanofi-Genzyme during the conduct of this study; personal fees from Takeda during conduct of the study; and personal fees from ?UpToDate? outside the submitted work. Dr Morel reported receiving grants from Genzyme, Shire, and government during the conduct of this study; and personal fees from Amicus and Shire Takeda outside the submitted work. Dr Lachmann reported receiving personal fees and non-financial support from Sanofi-Genzyme, Takeda, and Biomarin outside the submitted work. Dr Lynd reported receiving honoraria for consulting services from Boehringer Ingelheim outside the submitted work; grants from Pfizer Canada, Astra Zeneca, and the Canadian Institutes of Health Research outside the submitted work. Dr Wasim reported receiving grants from the Canadian Fabry Disease Initiative outside the submitted work; and personal fees from Amicus Therapeutics outside the submitted work. Dr West reported receiving grants and personal fees from Amicus Therapeutics, Takeda Shire, and Sanofi-Genzyme Corp outside the submitted work, and grants from Idorsia, Protalix outside the submitted work. In addition, Dr West has a Lenti virus gene therapy for Fabry disease patent with royalties paid, and patent issued for an Edmonton Fabry biomarker panel. Dr Hollak reported being involved in pre-marketing studies with Sanofi-Genzyme, Protalix, and Idorsia outside the submitted work. No other disclosures were reported. Funding/Support: The authors received no financial support for this research. Acknowledgment: The authors would like to acknowledge Dr Aneal Khan who provided insights on this study, investigators in the Canadian Fabry Disease Initiative who did not participate in this manuscript, the Canadian Fabry Association, and all the patients and families who support rare disease registries. Publisher Copyright: {\textcopyright} 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research Copyright: Copyright 2021 Elsevier B.V., All rights reserved.",
year = "2021",
month = feb,
doi = "https://doi.org/10.1016/j.jval.2020.10.006",
language = "English",
volume = "24",
pages = "268--273",
journal = "Value in Health",
issn = "1098-3015",
publisher = "Elsevier Limited",
number = "2",
}