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The importance and challenge of pediatric trials of hemophilia drugs

Nature Medicine volume 20, pages 465–466 (2014)Cite this article

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To the Editor:

A recent Opinion piece by Peyvandi et al.1 was critical of the European requirement that pediatric data on hemophilia medicines be included in initial marketing authorization applications (MAAs).The passion the authors expressed for expediting the availability of new treatments for adult patients is commendable. However, the Opinion may leave the false impression that the source of the requirement for pediatric data before drug approval is the European Paediatric Regulation and the associated mandate for a Paediatric Investigation Plan (PIP). In actuality, the Paediatric Regulation is not the basis for requiring that pediatric data for hemophilia products be included in the initial MAA.

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References

  1. Peyvandi, F., Rosendaal, F.R., O'Mahony, B. & Mannucci, P.M. Nat. Med. 20, 117 (2014).

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  2. Baxter. Baxter submits application for European approval of Rixubis, recombinant factor IX (nonagoc gamma) for hemophilia B, http://www.baxter.com/press_room/press_releases/2013/10_31_13_rixubis.html (2013).

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  1. Pediatric Center of Excellence, Quintiles, Durham, North Carolina, USA

    Robin Huff

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  1. Robin Huff
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Correspondence to Robin Huff.

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Huff, R. The importance and challenge of pediatric trials of hemophilia drugs. Nat Med 20, 465–466 (2014). https://doi.org/10.1038/nm.3539

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