Analysis of Pediatric Clinical Drug Trials for Neuropsychiatric Conditions

Abstract

BACKGROUND AND OBJECTIVE: Neuropsychiatric conditions represent a large and increasing disease burden in children. A number of drugs are available for the treatment of these conditions, but most drugs have not been adequately tested in children, and off-label drug use remains widespread. We sought to define and quantify recent and ongoing clinical research on the use of neuropsychiatric drugs in children. METHODS: Drug trials registered in ClinicalTrials.gov between 2006 and 2011 and studying neuropsychiatric conditions were selected and classified based on the drug’s Food and Drug Administration (FDA) approval status in children. We measured the proportion of trials seeking to expand the use of a drug to pediatric patients and the proportion of available drugs studied in children. RESULTS: Only 10% of neuropsychiatric trials focused on children. Of 303 drugs studied in both pediatric and adult populations, 90% lacked FDA approval in children and 97% were not approved in children for the indication studied. However, only 19% of all neuropsychiatric drugs were under study in pediatric populations, with as few as 8% of either antidepressant or antipsychotic drugs. Overall, 76% of pediatric drug trials examined a drug previously unapproved in children and 26% explored the use of a drug for a new indication. CONCLUSIONS: Despite the rising prevalence of neuropsychiatric disease and the paucity of FDA-approved pediatric drugs, only a small proportion of trials focus on pediatric populations and these trials cover only a fraction of available drugs. This deficiency is most pronounced for depression and schizophrenia.

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“…Ambas agencias poseen un sistema similar por el cual, por un lado, exigen a la industria realizar estudios en la población infantojuvenil, mientras que otorgan incentivos económicos (Murthy, 2013). Para la FDA estas leyes, vigentes desde 1997, son Best pharmaceutical for children act (BPCA) y Pediatric research equity act (PREA), mientras que en lo que respecta a la autoridad europea, en el año 2007 se estableció el Comité Pediátrico de la Agencia Europea de Medicamentos (EMA-PDCO) e hizo obligatorio el Pediatric Investigation Plan (PIP), es decir, estudios en la población pediátrica cuyos resultados deben incluirse en la documentación de la Autorización de Comercialización, a menos que se conceda una excusa fundada para no hacerlo (waiver).…”

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