美国儿童用药独占权介绍

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1、PediatricExclusivityPediatricExclusivityandDrugDevelopmentRequirementsintheOverallPediatricPopulationPreparedbyBeckloffAssociates,Inc.1INTRODUCTIONANDOVERVIEW简介Althoughchildrensufferfrommanyofthesamediseasesasadultsandareoftentreatedwiththesamedrugs,

2、onlyasmallfractionofthedrugsmarketedandusedastherapiesintheUnitedStateshavebeenstudiedinpediatricpatients0–16yearsofage.Recentdatademonstratesthatonlyabout25%ofapproveddrugsmarketedintheUnitedStateshaveadequatepediatricdatatosupportapprovalofproductl

3、abelingbyFDAfordosing,safety,orefficacyinchildren.1Thislackofappropriatepediatrictestingandlabelingincreasesthechancesofincorrectdosing,therebyexposingpediatricpatientstoeitheranincreasedriskofadversereactionsorlessthanoptimaltherapeuticbenefits.2Ina

4、ddition,thefailureofpharmaceuticalcompaniestomanufacturedrugsindosageformsthatcanbeeasilyandproperlyusedinallpediatricagegroupsdeniespediatricpatientsaccesstonecessarymedications.虽然儿童患有许多与成年人相同的疾病，治疗往往也用同样的药物，只有一小部分在美国销售和治疗使用的药品在儿科病人0–16岁中进行过研究。近期的数据

5、表明，在美国上市批准的药品中只有约25％有足够的儿科数据支持美国FDA批准的剂量，安全性或有效性在儿童中使用的标签[1]。这缺乏适当的儿科试验和标签的情况剂量使用不当的几率增加，从而使患儿处于不良反应风险加而最佳的治疗效果降低的境地[2]。此外，制药公司未能提供适用于各年龄层的准确、简便的剂量规格，使得儿童患者获得必要的药物变得更难。Mostdrugsprescribedforchildrenlacksufficientpharmacokinetic,pharmacodynamic,effica

6、cy,orsafetydatatosupportuseinthepediatricpopulation.Thepharmaceuticalindustrytraditionallyhashadlittleincentivetoundertakepediatricclinicaltrials,whichhasresultedinoff-labeluseinthepediatricpopulation,rangingfrom60to90%inchildrenandnewborns.3Reasonsc

7、itedforthelackofstudiesinthepediatricpopulationincludesmallertargetpopulations,increasedrecruitmentchallenges,consentandethicalissues,andlowerpotentialformarketreturnoncorporateinvestmentcomparedwiththatseeninadultpopulations.3Legislationhasbeenenact

8、edintheUnitedStatesoverthepast10yearstoprovideincentivesforpharmaceuticalindustrysponsorstoconductdrugstudiesinthepediatricpopulation.Toencouragepharmaceuticalsponsorstoundertakeclinicalstudiesinthedevelopmentofpediatricindicationsfordrugsandbiologic