Orphan Medicines for Pediatric Use: A Focus on the European Union
| Autor: | Claire Hill-Venning, Thomas C. Kühler, David King, Hans Hoogland, Solange Corriol-Rohou, Angelika Joos, Genevieve Le Visage, Winona Rei Bolislis, Victoria Kitcatt |
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| Rok vydání: | 2019 |
| Předmět: |
Prioritization
Drug Industry Orphan Drug Production 02 engineering and technology 030204 cardiovascular system & hematology Orphan diseases 03 medical and health sciences Rare Diseases 020210 optoelectronics & photonics 0302 clinical medicine Drug Development 0202 electrical engineering electronic engineering information engineering Humans media_common.cataloged_instance Medicine Pharmacology (medical) European Union European union Child media_common Pharmacology business.industry Corporate governance Public relations Incentive Drug development European policy business |
| Zdroj: | Clinical Therapeutics. 41:2630-2642 |
| ISSN: | 0149-2918 |
| DOI: | 10.1016/j.clinthera.2019.10.006 |
| Popis: | Purpose European policy makers have provided a number of incentives for the development of medicines for orphan diseases as early as 1999 through the Orphan Regulation and created obligations for medicines developers to investigate their products in children through the Paediatric Regulation adopted in 2006. This article describes the challenges that developers of orphan medicines are facing with pediatric indications, discusses the interplay between the Orphan Regulation and the Paediatric Regulation, and provides some recommendations on how to optimize drug development under the current European Union regulatory framework. Methods This article discusses the European Union's Orphan Regulation, Paediatric Regulation, and the implications of the intersection of the regulations on the development of orphan medicines for pediatric use. Findings Although these regulations have been successful in meeting their objectives separately, different regulatory frameworks entail separate governance, multiple assessments, varying approaches and priorities to unmet medical needs, and joined-up regulatory process coordination. Better integration of regulatory pathways would therefore be helpful in stimulating more global drug development of pediatric orphan medicines, including optimizing the interaction between both regulations, using innovative drug development approaches while considering alternatives to randomized clinical trials, better identification and prioritization of unmet medical needs in pediatrics, and ensuring the alignment of regulatory processes. Implications Rare diseases are categorized as “orphan diseases” because their occurrence in a small number of patients means that, regardless of the apparent high unmet medical need, there is limited public and market interest to justify the high development risk and significant investment to develop new treatments. However, unexplored potential within the area, as well as a conducive regulatory environment, can further support the development of medicines to treat rare diseases, including for children. |
| Databáze: | OpenAIRE |
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