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National Authority of Medicines and Health Products, Portugal
Posters & Accepted Abstracts: J Develop Drugs
Learning Objectives: Discuss the strategic aspects, experiences and learnings with regard to regulatory framework for pediatric orphan drug. Share the experience from paediatric development process of medicines and review the opportunities offered by the different legislations for treatment of rare diseases; discuss the regulatory aspects that may impact approval of pediatric orphan drugs Proposal Details: Drug development is no longer possible without considering children and the orphan indication and/or rare disease pediatric setting involves unique considerations in addition to the more common challenges of clinical research and therefore poses extra challenges in conducting a research study. The medicinal products for paediatric use pose a challenge due to the heterogeneity of the considered age groups and the impossibility of extrapolating clinical results between adults and children. These difficulties could impact in childrenâ�?�?s access to medicines innovation. European legislation in medicines has been in line with the path of the United States Food and Drug Administration with new procedures for granting marketing authorization now include accelerated and conditional approvals, leading to quicker access of new drugs to patients. In this evolving scenario, guidelines on the evaluation of medicinal products are subject to continuous revision. We intend to present the current status and forthcoming activities related to principles behind successful development of paediatric orphan drugs, focusing on development challenges and critical needs and provide information on various strategies designed to overcome these challenges. We will present recent examples of the pediatric orphan drug application process from designation to approval (orphan market exclusivity, similarity and significant benefit) and future developments such as the introduction of rare paediatric diseases designations and potential implications for orphan drugs.