Healthy Skepticism Library item: 10529

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Publication type: Journal Article

Holdsworth MT.
Pediatric drug research--the road less traveled.
Ann Pharmacother 2003 Apr; 37:(4):586-91
http://www.theannals.com/cgi/content/full/37/4/586

Abstract:

The above evidence points to a glaring deficiency in pediatric drug development research in the US. It is apparent that the cumbersome and costly drug approval process does not serve children well. Solutions that will substantially alter this situation will necessitate a significant change in the existing system. Incentives for the pharmaceutical industry in the form of patent extensions are somewhat effective, yet mainly benefit children only after several years of marketing. It seems that some incentives are also necessary at the time of marketing, if industry should bear part of the responsibility for seeking drug approval data in children. Requirements for pediatric studies of all new drugs would be overly burdensome for the pharmaceutical industry. Such requirements would likely succumb to the multiple loopholes that have negated much of the power of the Final Rule. Instead, incentives such as fast-track approval for drugs with significant pediatric data would be a possible approach. While this was initially bemoaned as a dangerous venture for the HIV population, it has contributed to significant clinical advances in this area of medicine. It is also inappropriate to rely on the pharmaceutical industry for the majority of pediatric drug research. The federal government should shoulder the majority of the responsibility for promoting pediatric drug development research. The ability of pharmaceutical companies to obtain a significant return on investment via application of the agent in a large population of patients is critical. The variety and relative rarity of significant illnesses in the pediatric population will often be a losing venture for the industry. This would likely necessitate a significant shift in funding within NIH and perhaps the creation of a center within NICHD devoted to drug development in children. While it may be argued that many of the existing centers support some pediatric research, it is clear that the current system is falling well short of the mark needed to positively influence pediatric labeling. Such a center within NICHD could promote alternative study designs for drugs currently on the market but without remaining patent life. By offering grants to promote a change in the current drug delivery/patient care interface, such funding might permit the badly needed pharmacoepidemiology research in the field. The paradigm shift within NIH that would be necessary for such a significant change would be substantial. However, there is no patient population that is more deserving of our focus and attention than children with serious illnesses. I hope that there will soon be the groundswell in advocacy that will be necessary to bring about significant change in the lives of such children.

Keywords:
Aging/physiology* Child Child Advocacy*/trends Clinical Trials/methods* Clinical Trials/standards Drug Approval Drugs, Investigational/pharmacology* Humans National Institutes of Health (U.S.) Research/methods* Research/standards United States United States Food and Drug Administration