formulation issues for parenteral drugs: the safety and feasibility of different volumes and concentrations, the safety of various preservatives, the appropriate packaging of vials or prefilled syringes for pediatric use, and so forth.

There is no simple approach to the evaluation of a biologic product for use in the pediatric population. One needs to think about the intended action or the indication of the particular biologic and the disease or condition that it is proposed to treat. Questions that should be considered during development include the following: Is the disease unique or very common in the pediatric population? Are there alternative therapies? What are the safety and efficacy profiles of those alternative therapies that will make it imperative or important to develop a new agent? What is the relevance and suitability of the adult safety and efficacy data? Will it be possible to extrapolate efficacy data from older to younger children? The answers to these questions are among the considerations for optimizing the program development for biologics for the pediatric population.

The evaluation of the clinical efficacy of biologics is similar to that for drugs and devices. There may be unique safety concerns for biologic products because of their distinctive properties that will require different types of monitoring, such as for adventitious agents or for immune reactions. Little is known about the long-term effects (e.g., years) of treatment with therapeutic biologics, especially the effects on growing children. As more and more biologics are licensed for use for chronic treatment, it will be important to collect and examine data from postmarketing use, including studies from registries and Phase 4 clinical trials (controlled studies following market approval), to further the understanding of this important class of agents.



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