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Children 2015, 2(1), 108-130; doi:10.3390/children2010108

Cystic Fibrosis Treatment: A Paradigm for New Pediatric Medicines, Globalization of Drug Development and the Role of the European Medicines Agency

1
Consulting, Aeussere Baselstrasse 308, 4125 Riehen, Switzerland
2
Internal Medicine and Pharmacy, Division of Clinical Pharmacology, University of Utah, Salt Lake, UT 84108, USA
3
Divisions of Adolescent Medicine and Clinical Pharmacology, Clinical Trials Office, University of Utah, Salt Lake City, UT 84108, USA
*
Author to whom correspondence should be addressed.
Academic Editor: Sari Acra
Received: 19 January 2015 / Revised: 10 March 2015 / Accepted: 11 March 2015 / Published: 23 March 2015
(This article belongs to the Special Issue Development of Medicines for Paediatric and Rare Diseases)
View Full-Text   |   Download PDF [552 KB, uploaded 23 March 2015]

Abstract

The European Pediatric Pharmaceutical Legislation wants children to benefit more from pharmaceutical progress. In rare diseases, concerns have been raised that this legislation might damage research and stymie drug development. We discuss the role of the European Medicines Agency (EMA) and its Pediatric Committee (PDCO) in the development of ivacaftor, first-in-class for cystic fibrosis (CF) patients with the G551D mutation (and eight other mutations later) and of lumacaftor and ataluren, two more potential break-through CF medications. Ivacaftor was USA-approved early 2012 and six months later in the EU. Registration was based on the same data. We analyzed these drugs’ EU pediatric investigation plans (PIPs) and compared the PIP-studies with the pediatric CF studies listed in www.clinicaltrials.gov. The ivacaftor PIP studies appear to reflect what the developer planned anyway, apart from a study in 1–23-month-olds, which has not yet started. The total negotiation time for the current PIP version was approximately 5.5 years. For companies that develop drugs in pediatric diseases, e.g., CF, PIPs represent considerable additional procedural workload with minimal or no additional benefit for the patients. New drugs for pediatric diseases should not be hampered by additional, unnecessary and costly bureaucracy, but be registered as rapidly as possible without compromising safety. View Full-Text
Keywords: better medicines for children; pediatric drug development; cystic fibrosis; pediatric legislation; pediatric pharmaceutical legislation; EU pediatric regulation; FDA Innovation and Safety Act (FDASIA); Pediatric Committee (PDCO) better medicines for children; pediatric drug development; cystic fibrosis; pediatric legislation; pediatric pharmaceutical legislation; EU pediatric regulation; FDA Innovation and Safety Act (FDASIA); Pediatric Committee (PDCO)
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Rose, K.; Spigarelli, M.G. Cystic Fibrosis Treatment: A Paradigm for New Pediatric Medicines, Globalization of Drug Development and the Role of the European Medicines Agency. Children 2015, 2, 108-130.

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