tainty and more risk with the new compounds now in the pipeline, in terms of both the costs of studies and market size. Because none of these factors bode well for encouraging drug makers to test their products in children, Dr. Murphy stressed that incentives may become more important than ever.

An additional challenge is funding for off-patent studies. Dr. Mathis and others suggested that relying solely on FNIH for funding for these studies is unrealistic. Dr. Nelson also argued that the process used by NIH to get contracts for studies of these drugs, as well as for on-patent drugs that a sponsor chooses not to study, is cumbersome and unnecessarily lacking in transparency.

A number of participants, including Dr. Gorman and Ms. Jarrett, agreed that the infrastructure needed to conduct multisite pediatric studies is lacking. Enhancing this infrastructure would require not only the additional training in therapeutics discussed above, but also increased funding for practice-based research networks. In addition to better training, increasing the numbers of trained pediatric clinical investigators is also important; the debt forgiveness for students incentive mentioned earlier could be helpful in this regard.

Pediatric-specific adverse events must also be defined so that pediatricians and parents will know what to look for and how to manage events that occur. Dr. Murphy and Dr. Snodgrass advocated for a more active surveillance system to identify adverse events, especially after a pediatric indication has been approved for marketing. They also stressed the importance of better communication to prescribers and consumers so they will know not only how to deal with adverse events, but also how to use the medications properly.

Finally, lessons can be learned from the European Union’s recent regulations, which represent a coordinated effort to improve the study of on-patent, off-patent, and orphan drugs. The compensation fund and information sheets that are integral to vaccine drug development can also serve as models for pediatric drug development.



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