the best pharmaceuticals for children act anne zajicek, m.d., pharm.d. pediatric medical officer...
TRANSCRIPT
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The Best Pharmaceuticals for Children Act
Anne Zajicek, M.D., Pharm.D.Pediatric Medical OfficerNational Institute of Child Health and Human DevelopmentNational Institutes of Health
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Best Pharmaceuticals for Children Act
Enacted January 2002, exclusivity provision will sunset October 2007Continues exclusivity provision of FDAMAPurpose: pediatric labeling
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Pediatric Labeling
Pre-approval: Pediatric Research Equity ActOn-patent: Best Pharmaceuticals for Children Act which
continues the exclusivity provisions of FDAMA Studies supported by the Foundation for the NIH
Off-patent: Best Pharmaceuticals for Children Act
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Best Pharmaceuticals for Children Act
In pediatric oncology prioritize new drugs for study assure timely access to new
treatments develop pre-clinical models of
pediatric cancers
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Master List of all Off-Patent Drugs which lack adequate pediatric labeling
N=200
Consultation with experts in pediatric practice and research
Develop, prioritize, publish an Annual List
N=5-15
Consider for prioritizing: Availability of S/E dataAre additional data needed?Will new studies produce health benefits?Reformulation?
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Priority List
Developed by NIHIn consultation with Institutes and Centers of the National
Institutes of Health Federal Agencies
Food and Drug Administration Centers for Disease Control and Prevention
Pediatric subspecialists and subspecialty groups
Advocacy groups
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Drugs on the Priority List: January 2003
FR 68: 13; Jan 21, 2003
AzithromycinBaclofen*BumetanideDobutamineDopamineFurosemideHeparin
LithiumLorazepamRifampinSodium nitroprussideSpironolactone
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Drugs on the Priority List: August 2003FR 68: 156; Aug 13, 2003
Ampicillin/sulbactamDiazoxideIsofluraneLindaneMeropenem
Metoclopramide*Piperacillin/ tazobactamPromethazine
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Drugs on the Priority List: February 2004
FR 69:30; February 13, 2004
AmpicillinKetamineVincristineActinomycin-DMetolazone
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Drugs on the Priority List: January 2005FR 70:17; January 27, 2005
IvermectinHydrocortisone valerateHydrochlorothiazideEthambutolGriseofulvinMethadoneHydroxychloroquineSevelamer*Morphine*
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Drugs on the Priority List: April 2006
FR 70:79; April 25, 2006
ADHD: methylphenidate Hypertension: diureticsParasitic Diseases: albendazole, mebendazoleInfluenza: amantidine, rimantidineCancer: methotrexate, daunomycinPoisonings: pralidoximeSickle Cell Anemia: hydroxyurea
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Drugs on the Priority List:March 2007
FR 72: 14588; March 28, 2007
Infectious Diseases: Methicillin-resistant Staph aureus infection Clindamycin, tetracyclines, trimethoprim-
sulfamethoxazoleHypertension: clinical trial designsNeonatal Research: clinical trial designsCancer: Neuroblastoma 13-cis retinoic acid
Asthma: clinical trial designs in young children
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FDA Issues a Written Request:On-Patent
From Priority ListFDA issues
Written Request (WR)
WR sent toHolder of
NDA
Accept
Decline
Referredto FNIH
for funding
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FDA Issues a Written Request: Off-Patent
From Priority ListFDA issues
Written Request (WR)
WR sent toHolders of
NDA/aNDA
Accept
Decline
Referredto NIH
for contract
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Contracting Process
WR referred to NIH
RFC* RFP* FedBizOpps
Proposals peer-reviewed
Contract(s) awarded
*RFC=request for contract*RFP=request for proposal
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Results So Far…
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Studies On-Going under BPCA
Lorazepam: Clinical studies for treatment of status epilepticus Study 1: PK Study 2: Efficacy, safety study comparing
lorazepam to diazepamLorazepam: Clinical studies of sedation of children on ventilators in an intensive care unitNitroprusside: Clinical studies to reduce blood pressure during surgery to reduce blood loss Lithium: Clinical studies to define treatment of mania in children with bipolar disorder Baclofen: Clinical studies of oral baclofen to treat spasticity, most commonly from cerebral palsy
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Studies On-Going
Ketamine - Preclinical studies to evaluate the scientific and safety concerns about the use as an anesthetic in childrenHydroxyurea- Clinical studies to improve treatment of children with sickle cell disease (NHLBI)Methylphenidate – Preclinical and clinical evaluation of pharmacokinetics and safety to understand reports of cytogenetic toxicity (NIEHS)Morphine – preclinical basic science evaluations of the developmental expression of opioid receptors to better understand management of pain in children of different developmental stages and safety issues in treating pain in neonates
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Studies On-Going under BPCA
Vincristine: Studies to evaluate neurotoxicity, PK in children (NCI-COG)Actinomycin-D: Studies to evaluate hepatotoxicity/ VOD, PK in children (NCI-COG)Methotrexate: Clinical studies to evaluate neurocognitive outcomes of pediatric patients with high risk acute lymphoblastic leukemia (NCI-COG)Daunomycin: Pharmacokinetics, safety, efficacy of daunomycin to treat childhood cancers and relationship to body weight (NCI-COG)
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Vincristine and Actinomycin-D
Retrospective chart review of Demographics Treatment history Toxicity
Neurotoxicity Veno-occlusive disease
Line-clearing methodPharmacokinetic modelingProspective PK study
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SummaryPartnership with FDANIH responsible for prioritizing list of drugs for study sponsoring clinical studies in children
that will improve pediatric therapeutics