Pediatric drug regulation: International perspectives.

There was a time when the predominant approach to exposing children to new drugs was to protect children from research. This has evolved over the past several decades into protecting children through research. To encourage pediatric studies and approval of pediatric medicines, governments have provided financial incentives as well as obligations/requirements for pharmaceutical companies to carry out pediatric studies in certain circumstances. The unique considerations for children have been acknowledged by the various governments and drug regulatory agencies through international dialogue and cooperation among patient and patient care representatives, regulatory agencies, and academic, clinical and manufacturing stakeholders. We describe pediatric drug regulation in five of the largest international drug regulatory agencies and additionally discuss efforts at international cooperation and discussion in pediatric drug regulation.

Drug-induced cardiac abnormalities in premature infants and neonates.

The Cardiac Safety Research Consortium (CSRC) is a transparent, public-private partnership that was established in 2005 as a Critical Path Program and formalized in 2006 under a Memorandum of Understanding between the United States Food and Drug Administration and Duke University. Our continuing goal is to advance paradigms for more efficient regulatory science related to the cardiovascular safety of new therapeutics, both in the United States and globally, particularly where such safety questions add burden to innovative research and development. This White Paper provides a summary of discussions by a cardiovascular committee cosponsored by the CSRC and the US Food and Drug Administration (FDA) that initially met in December 2014, and periodically convened at FDA's White Oak headquarters from March 2015 to September 2016. The committee focused on the lack of information concerning the cardiac effects of medications in the premature infant and neonate population compared with that of the older pediatric and adult populations. Key objectives of this paper are as follows: Provide an overview of human developmental cardiac electrophysiology, as well as the electrophysiology of premature infants and neonates; summarize all published juvenile animal models relevant to drug-induced cardiac toxicity; provide a consolidated source for all reported drug-induced cardiac toxicities by therapeutic area as a resource for neonatologists; present drugs that have a known cardiac effect in an adult population, but no reported toxicity in the premature infant and neonate populations; and summarize what is not currently known about drug-induced cardiac toxicity in premature infants and neonates, and what could be done to address this lack of knowledge. This paper presents the views of the authors and should not be construed to represent the views or policies of the FDA or Health Canada.