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.
Highlights
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
Several studies suggest that orphan drugs are associated with a higher return on investment than drugs licensed for common diseases resulting in intense pharmaceutical industry interest in rare diseases [2, 3]
Box 22660, 1100, DD, Amsterdam, The Netherlands 2Platform Medicine for Society at Amsterdam University Medical Centers, location Academic Medical Center, University of Amsterdam, Amsterdam, The Netherlands Full list of author information is available at the end of the article at an annual growth rate of 11.2% and orphan drugs are expected to make up more than 18% of worldwide prescription drug costs by 2024 [4]
Summary
Enter registries Following debates on dosing and differences in the natural disease course, a 1995 Health Technology Assessment meeting resulted in a recommendation to seek better evidence to support the appropriate use of this costly therapy [18]. For this purpose, Genzyme set up a global registry in 1991 containing data on both treated and untreated patients: run and financed by Genzyme, analyses performed by Genzyme and publications supported by Genzyme. An international board of Gaucher treating physicians helped to operate the registry: meetings were held, with key opinion leaders, paid by Genzyme, discussing treatment goals and diagnostic
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