Registries for orphan drugs: generating evidence or marketing tools?
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(2020) 15:235
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Open Access
Registries for orphan drugs: generating evidence or marketing tools? Carla E. M. Hollak1,2* , Sandra Sirrs3, Sibren van den Berg2, Vincent van der Wel2, Mirjam Langeveld1, Hanka Dekker4, Robin Lachmann5 and Saco J. de Visser2
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. Keywords: Registries, Orphan drugs, Real world data, Disease registries
Orphans Rare diseases (defined in the EU as affecting fewer than 1 in 2000 people) are individually rare but collectively common, affecting 6–8% of the population [1]. Orphan drugs, drugs for rare diseases, are developed under specific regulations in the EU, the United States and Canada with incentives to stimulate pharmaceutical companies to develop medicines for rare diseases. The idea is that investments in orphans would not be commercially attractive. However, 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]. While the number of treatable rare conditions is still relatively low, the orphan drug market is expanding * Correspondence: [email protected] 1 Department of Endocrinology and Metabolism, Amsterdam University Medical Centers, location Academic Medical Center, University of Amsterdam, F5-170, P.O. Box 22660, 1100, DD, Amsterdam, The Netherlands 2 Platform 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]. The majority are for oncology indications followed by cell- and gene-based therapeutics, the latter typically indicated for ultra-rare inherited diseases [5].
The development process and registries Orphan designation is granted to an applicant at the beginning of the drug development process based on the “medical plausibility” of the proposed active substance’s effect on a rare disease. However, conducting clinical studies to prove safety and effectiveness can be challengi
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