Registries for orphan drugs: generating evidence or marketing tools?

Orphanet Journal of Rare Diseases

Robin Lachmann¹  Mirjam Langeveld²  Carla E. M. Hollak²  Sandra Sirrs³  Vincent van der Wel⁴  Saco J. de Visser⁴  Sibren van den Berg⁴  Hanka Dekker⁵ 

[1] Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery;Department of Endocrinology and Metabolism, Amsterdam University Medical Centers, location Academic Medical Center, University of Amsterdam;Division of Endocrinology and Metabolism, University of British Columbia;Platform Medicine for Society at Amsterdam University Medical Centers, location Academic Medical Center, University of Amsterdam;VKS, The Dutch patient association for Inherited Metabolic Diseases;

关键词: Registries;    Orphan drugs;    Real world data;    Disease registries;   

DOI  :  10.1186/s13023-020-01519-0

来源: DOAJ

【 摘 要 】

Abstract Independent disease registries for pre-and post-approval of novel treatments for rare diseases are increasingly important for healthcare professionals, patients, regulators and the pharmaceutical industry. Current registries for rare diseases to evaluate orphan drugs are mainly set up and owned by the pharmaceutical industry which leads to unacceptable conflicts of interest. To ensure independence from commercial interests, disease registries should be set up and maintained by healthcare professionals and patients. Public funding should be directed towards an early establishment of international registries for orphan diseases, ideally well before novel treatments are introduced. Regulatory bodies should insist on the use of data from independent disease registries rather than company driven, drug-oriented registries.

【 授权许可】

Unknown