Stimulation programs for pediatric drug research – do children really benefit?

Abstract

Most drugs that are currently prescribed in pediatrics have not been tested in children. Pediatric drug studies are stimulated in the USA by the pediatric exclusivity provision under the Food and Drug Administration Modernization Act (FDAMA) that grants patent extensions when pediatric labeling is provided. We investigated the effectiveness of these programs in stimulating drug research in children, thereby increasing the evidence for safe and effective drug use in the pediatric population. All drugs granted pediatric exclusivity under the FDAMA were analyzed by studying the relevant summaries of medical and clinical pharmacology reviews of the pediatric studies or, if these were unavailable, the labeling information as provided by the manufacturer. A systematic search of the literature was performed to identify drug utilization patterns in children. From

Finding Better Ways to Fill Gaps in Pediatric Health Research

“…Because the percentage of children using a prescription drug is relatively high in the first year of life, and off-label and unlicensed prescribing rates are particularly high for neonates, this finding supports the conclusion that the age distribution of children participating in trials does not reflect the need for research. 1 In addition, we found fewer registered trials for communicable, maternal, perinatal, and nutritional conditions (22% of trials; 71% of the global burden) than for noncommunicable diseases (74% of trials; 21% of the global burden). This disparity is also present in adult trials, 5 but it is larger for pediatric trials, potentially reflecting that the lack of health R&D for populations in developing countries disproportionately affects children.…”

“…Methodological and ethical challenges associated with pediatric research increase the costs of running pediatric trials, and the market for pediatric products is relatively small. 1,2 The subsequent knowledge gaps and lack of child-specific product development have resulted in high off-label and unlicensed medication prescription rates in children. 2 The past decade has seen increased recognition of this problem, and both the United States and the European Union have implemented legislative measures to stimulate pediatric health research by providing incentives and funding for pediatric studies and by requiring pediatric studies for new drug applications when appropriate.…”

“…2 The past decade has seen increased recognition of this problem, and both the United States and the European Union have implemented legislative measures to stimulate pediatric health research by providing incentives and funding for pediatric studies and by requiring pediatric studies for new drug applications when appropriate. [1][2][3] Although these measures have led to an increase in the number of pediatric trials being conducted and to several knowledge gaps being addressed, 3 the trials remain driven largely by market incentives, and evaluations of the measures in the United States 1 and the European Union 3 show that other areas remain neglected. Moreover, the majority of the global pediatric disease burden lies with populations in low-and middle-income countries, for whom the lack of health R&D remains a significant problem.…”

“…A discussion of how this might be achieved for pediatric research gaps in various geographic contexts is needed, to determine what measures could address the gaps left by existing measures in the United States and European Union. [1][2][3] …”

Analysis of Pediatric Clinical Drug Trials for Neuropsychiatric Conditions

“…41,42 Consequently, the drugs studied under the exclusivity program may more closely mirror adult than pediatric medical needs. Nonetheless, the BPCA and PREA have succeeded in encouraging both efficacy and safety studies in children and in expanding indications for existing adult drugs to pediatric populations.…”