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The Best Pharmaceuticals for Children Act

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Study 2: Efficacy, safety study comparing lorazepam to diazepam. Lorazepam: Clinical studies of sedation of children on ventilators in an intensive care unit ... – PowerPoint PPT presentation

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Title: The Best Pharmaceuticals for Children Act


1
The Best Pharmaceuticals for Children Act
  • Anne Zajicek, M.D., Pharm.D.
  • Pediatric Medical Officer
  • National Institute of Child Health and Human
    Development
  • National Institutes of Health

2
Best Pharmaceuticals for Children Act
  • Enacted January 2002, exclusivity provision will
    sunset October 2007
  • Continues exclusivity provision of FDAMA
  • Purpose pediatric labeling

3
Pediatric Labeling
  • Pre-approval Pediatric Research Equity Act
  • On-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

4
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

5
Master List of all Off-Patent Drugs which lack
adequate pediatric labeling N200
  • Consider for prioritizing
  • Availability of S/E data
  • Are additional data needed?
  • Will new studies produce
  • health benefits?
  • Reformulation?

Consultation with experts in pediatric practice
and research
Develop, prioritize, publish an Annual
List N5-15
6
Priority List
  • Developed by NIH
  • In 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

7
Drugs on the Priority List January 2003 FR 68
13 Jan 21, 2003
  • Azithromycin
  • Baclofen
  • Bumetanide
  • Dobutamine
  • Dopamine
  • Furosemide
  • Heparin
  • Lithium
  • Lorazepam
  • Rifampin
  • Sodium nitroprusside
  • Spironolactone

8
Drugs on the Priority List August 2003 FR 68
156 Aug 13, 2003
  • Ampicillin/sulbactam
  • Diazoxide
  • Isoflurane
  • Lindane
  • Meropenem
  • Metoclopramide
  • Piperacillin/ tazobactam
  • Promethazine

9
Drugs on the Priority List February 2004 FR
6930 February 13, 2004
  • Ampicillin
  • Ketamine
  • Vincristine
  • Actinomycin-D
  • Metolazone

10
Drugs on the Priority List January 2005 FR
7017 January 27, 2005
  • Ivermectin
  • Hydrocortisone valerate
  • Hydrochlorothiazide
  • Ethambutol
  • Griseofulvin
  • Methadone
  • Hydroxychloroquine
  • Sevelamer
  • Morphine

11
Drugs on the Priority List April 2006 FR 7079
April 25, 2006
  • ADHD methylphenidate
  • Hypertension diuretics
  • Parasitic Diseases albendazole, mebendazole
  • Influenza amantidine, rimantidine
  • Cancer methotrexate, daunomycin
  • Poisonings pralidoxime
  • Sickle Cell Anemia hydroxyurea

12
Drugs on the Priority List March 2007 FR 72
14588 March 28, 2007
  • Infectious Diseases Methicillin-resistant Staph
    aureus infection
  • Clindamycin, tetracyclines, trimethoprim-sulfameth
    oxazole
  • Hypertension clinical trial designs
  • Neonatal Research clinical trial designs
  • Cancer Neuroblastoma
  • 13-cis retinoic acid
  • Asthma clinical trial designs in young children

13
FDA Issues a Written Request On-Patent
Accept
WR sent to Holder of NDA
From Priority List FDA issues Written Request
(WR)
Referred to FNIH for funding
Decline
14
FDA Issues a Written Request Off-Patent
Accept
WR sent to Holders of NDA/ aNDA
From Priority List FDA issues Written Request
(WR)
Referred to NIH for contract
Decline
15
Contracting Process
WR referred to NIH
RFC
FedBizOpps
RFP
Proposals peer-reviewed
RFCrequest for contract RFPrequest for
proposal
Contract(s) awarded
16
Results So Far
17
Studies On-Going under BPCA
  • Lorazepam Clinical studies for treatment of
    status epilepticus
  • Study 1 PK
  • Study 2 Efficacy, safety study comparing
    lorazepam to diazepam
  • Lorazepam Clinical studies of sedation of
    children on ventilators in an intensive care unit
  • Nitroprusside 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

18
Studies On-Going
  • Ketamine - Preclinical studies to evaluate the
    scientific and safety concerns about the use as
    an anesthetic in children
  • Hydroxyurea- 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

19
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)

20
Vincristine and Actinomycin-D
  • Retrospective chart review of
  • Demographics
  • Treatment history
  • Toxicity
  • Neurotoxicity
  • Veno-occlusive disease
  • Line-clearing method
  • Pharmacokinetic modeling
  • Prospective PK study

21
Summary
  • Partnership with FDA
  • NIH responsible for
  • prioritizing list of drugs for study
  • sponsoring clinical studies in children that will
    improve pediatric therapeutics
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