There are several factors to be considered when facing the problem of therapeutic orphans. First, there is the scientific one. We are all convinced that there is much need for more data concerning the use of drugs in children, infants, and neonates. Second, there is a regulatory factor involving political and technical institutions. Finally, we have to consider the economic interest of the pharmaceutics industry in investing money in research that would hardly generate sufficient return.
There is no need to stress here that improved research in pediatric clinical pharmacology is required. Despite the fact that the number of clinical trials in children has increased, in many cases the use of drugs still depends on extrapolation of data from adults, and adequate scientific documentation for their use in children is lacking. We need additional studies to optimize dosages at various ages throughout childhood, and we still need studies on clinical efficacy and tolerability of drugs in children.
However, we must recognize that when the pharmaceutics industry has an economic interest in developing a drug for use in children, we generally have good clinical data and reasonably large studies. Just think of many studies on antibiotics or the many drugs used in the treatment of otitis media or pharyngitis.
But in many cases, there is simply no economic advantage for the pharmaceutics industry to conduct studies in children. These studies would be time-consuming, often necessarily multicentered, and costly. This is true for a number of drugs. We also must consider that negative results in children could reflect unfavorably on the use of the same drug in adults and limit the profit of the industry.
Performing such studies therefore would depend primarily on the economic support of nonprofit or governmental organizations. The example of the network of pediatric clinical pharmacology centers developed …