[How and when to create a pediatric file for authorization to market a new drug. A proposal of the SNIP clinical group (National Syndicate of Pharmaceutical Industry)].
In order to recommend the pediatric use of a new drug, the registration file should include several types of studies. Existing data on toxicological and pharmacological studies in animal and human have to be thoroughly examined. In addition, the splitting potential of single doses to be adapted to the child should be studied, according to the galenic formula. In all cases, the clinical file should have one pharmacokinetic study on acute intake of one or several doses in the specific age bracket. When the efficacy of a drug has been demonstrated in adults for the same disease, and if the results are extrapolated to children, the file should include one or several open studies designs in order to assess the tolerability. On the contrary, for example when the disease is different in or specific for children, one or several double blind randomized studies against reference treatment are necessary. All those studies should show the therapeutical value of the drug, and should allow precise recommendations for the dosages, depending on the body weight and/or the body surface compatible with the galenic formula.
Vasmant
D
D
Département de Pharmacologie Clinique, Hôpital Trousseau, Paris.
Abadie
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E
D002648:Child; D002986:Clinical Trials as Topic / Q000592:standards; D004353:Drug Evaluation, Preclinical / Q000592:standards; D004345:Drug Industry; D015507:Drugs, Investigational / Q000592:standards*; D004992:Ethics, Medical; D006801:Humans; D016134:Organizational Policy; D010297:Paris; D012042:Registries / Q000592:standards*; D012957:Societies, Pharmaceutical / Q000458:organization & administration*
D015507:Drugs, Investigational
Comment et quand bâtir un dossier pédiatrique pour l'Autorisation de Mise sur le Marché d'un nouveau médicament. Une proposition du groupe clinique du SNIP.