What is it about?

Are you as curious as me to find out what the outcomes of the Paediatric and Orphan drug Regulations were when it comes to development of orphan medicines for paediatric use and what one can do to further stimulate development? Well, both regulations basically delivered on their objectives regarding orphan drugs for children. The Paediatric Regulation rendered more research, which was one intended key deliverable, as evidenced by the number of PIPs (paediatric investigation plans) for orphan designated medicines – albeit this was not necessarily mirrored by an increase in the number of approved medicines for children which was another hoped for outcome. The Orphan Regulation resulted in a significant number of Orphan Designated research products intended for children only, adults only, or children and adults together. And, as with any drug development programs, attrition rates are high, but the orphan drugs that made it all the way to the market were primarily indicated for adults only, and maybe more comforting, for children and adults alike.

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Why is it important?

Good, however, is not good enough – indeed, there are a couple of tweaks that easily could be implanted which would propel the EU-regulatory Orphan and Paediatric systems from good to great. Imagine if the COMP and the PDCO would be even better coordinated than they are, if modeling and simulation methods were more broadly recognized, or if alternative clinical trial design were more widely accepted.


Good regulations are in place and practical improvements that do not require the Regulations to be reworked would contribute to more efficient orphan drug development programs for children.

Thomas Kuhler
Sanofi SA

Read the Original

This page is a summary of: Orphan Medicines for Pediatric Use: A Focus on the European Union, Clinical Therapeutics, November 2019, Elsevier,
DOI: 10.1016/j.clinthera.2019.10.006.
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