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Lessons Learned

Lessons Learned. Need for partnerships Need for an objective prioritization process Feasibility determination: frequency of use/condition defined for children Need for infrastructure, so that infrastructure is not the driver of feasibility. NIH Role. Prioritize therapeutic areas/drugs

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Lessons Learned

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  1. Lessons Learned • Need for partnerships • Need for an objective prioritization process • Feasibility determination: frequency of use/condition defined for children • Need for infrastructure, so that infrastructure is not the driver of feasibility

  2. NIH Role • Prioritize therapeutic areas/drugs • Sponsor clinical trials • Submit data to FDA for labeling change

  3. 2007: Therapeutic Areas • Consider for prioritizing: • Therapeutic gaps • Potential health benefits • of research • Adequacy of necessary • infrastructure Consultation with experts in pediatric practice and research Develop, prioritize, publish an Annual List of Therapeutic Areas and Specific Needs

  4. 2007 Legislative Improvements • Proposed Pediatric Study Request (PPSR) • Therapeutic Areas • Infrastructure

  5. 2012 Re-authorization

  6. Prioritization • Objective, not constrained by infrastructure • Maximize public involvement • Input in therapeutic areas, medications • Input in determining which therapeutic areas, specific diseases, medications

  7. Prioritization: Areas of Focus • 2005: hypertension • 2006: infectious diseases • 2007: formulations • 2008: biodefense • 2009: adolescents, OTC treatments for cough and cold, atypical antipsychotics • 2010: gastroenterology, endocrinology, neurology • 2011: hematology, pulmonary, renal, formulations

  8. Prioritized Therapeutic Areas Cardiovascular DiseaseHypertension, Hypotension, Dyslipidemia Respiratory DiseaseAsthma, Pulmonary Hypertension Infectious DiseasesMRSA, General Infections, Tinea capitis, Tuberculosis, Parasitic Infections, Influenza Psychiatry ADHD, Bipolar Disease, Safety of Atypical Antipsychotics Adolescent MedicineGeneral therapeutic needs, Over the counter meds General PediatricsCorticosteroids, Cold and Cough Medicines FormulationsTechnology advances OncologyNeuroblastoma, Leukemia, Solid Tumors, Formulations Neurology Cerebral Palsy, Migraines, Seizures Intensive Care Anesthesia, Sedation DermatologyAtopic Dermatitis, Severe inflammatory skin disease RheumatologyConnective tissue disorders Bio-defense research Nerve agent exposure, Organophosphate poisoning, Cyanide toxicity Renal Diseases Chronic Kidney Disease, Anemia Hematology Sickle Cell Disease, Thrombosis Rare diseases/Endocrine Fragile X, Type-1 Diabetes, Formulations Gastroenterology GE Reflux, Cholestatic disease, Cyclic vomiting Neonatology BPD, Pain, Neonatal abstinence syndrome, NEC Special considerations Intellectual and developmental disabilities Pediatric Formulations, Devices, Adolescent therapeutics 8

  9. BPCA Process Prioritize PPSR/WR Sponsor Label Change Publish Submit Data to FDA

  10. Leveraging Existing Infrastructure

  11. Clinical Trials • National Cancer Institute/ Children’s Oncology Group • Vincristine • Actinomycin-D • Daunomycin • Methotrexate • Isotretinoin

  12. Clinical Trials • National Heart, Lung and Blood Institute: Hydroxyurea (Baby HUG)

  13. Clinical Trials • Health Research Services Administration: Pediatric Emergency Care and Applied Research Network (PECARN): Lorazepam for Status Epilepticus

  14. Data Collections for Clinical Trials Construction • NHLBI: Pediatric Respiratory Outcomes Program (PROP)-respiratory medications • NICHD: Neonatal Research Network (hypotension trial feasibility), Critical Care Research Network (medications used to treat status asthmaticus in the ICU)

  15. Infrastructure: Pharmacology Expertise • Training • Co-fund T32 training with NIGMS • Sites: Mayo, Penn, Vanderbilt, UCSF, UCLA, Univ of Chicago, Duke, Jefferson

  16. Outcome Measures: Partnership with NCRR/CTSAs • Partnership with NCRR/CTSAs • Purpose: research on outcome measures in the areas of • Neonatology • Cardiovascular medicine • Neurology

  17. Snapshot of BPCA Projects (18 recommended for funding) Topic Areas: 8-neonatology, 5-neurology, 4-hypertension/hypotension, 3-“other”; >1 topic area possible 1 Duke Development of a PK algorithm to improve neonatal outcomes 2 UTHSC Advanced MRI to assess neonatal care and outcome 3 Columbia Targets and Barriers for Hydroxyurea use in Sickle Hemoglobinopathies 4 Utah Improving Management of the Neonatal Abstinence Syndrome 5 U Pitt Cardiac Outcome Measures for Pediatric Muscular Dystrophy • UNC Outcome Measures for chronic lung disease of prematurity 7 U-CO Small volume fentanyl PK/PD & PG in neonates • UC-Davis Outcome Measures for Trials in Children with Autism • Vanderbilt Wireless Home-Based Tools for studying sleep in Autism 10 U-MI Pediatric Cardiac Intensive Care Data Standards Repository 11 Stanford Methadone vs. Morphine PD/PD in infants after cardiac surgery 12 Indiana Predictors of Vincristine-induced peripheral neuropathy 13 UAB Nasal Potential Difference Studies Utilizing CFTR Modulators 14 CWRU Efficacy Outcomes Measures in Antihypertensive Trials in Children 15 CWRU Effect of BMI on Exposure-Response Relationships to Lisinopril in Children 16 U-Wash Advancing Patient Reported Outcomes (PROs) in children with Cystic Fibrosis 17 AECOM Pediatric hypertension outcome measures 18 Tufts Improving BPD predictors and outcomes for clinical trials

  18. NIH-FDA Formulations PlatformIntra-Agency Agreement • Purpose: Develop an approach for producing oral dosage forms of various BCS class drugs, that are: stable in heat and humidity, tasteless/taste masked, preferably solid orally dissolvable dosage forms, in clinically useful dosage increments • http://bpca.nichd.nih.gov/collaborativeefforts/initiatives/index.cfm

  19. NIH-FDA Formulations Platform Inter-Agency Agreement

  20. Creation of New Infrastructure • NICHD T32 RFA • Sites: Indiana, Kansas City Mercy, Cincinnati

  21. U54 Research in Pediatric Developmental Pharmacology Centers Program • Pediatric Pharmacology Research Units Network sunset in 2009 • Need for model for basic/ translational /clinical drug development • Four sites funded: • Children’s National Medical Center • SUNY • Indiana University • University of CA San Diego

  22. Created Infrastructure: Duke Pediatric Trials Network • Flexible infrastructure to perform pediatric clinical trials • Cores: • Management (site performance) • Study design/ clinical pharmacology • Recruitment • Formulations development • Data analysis • Device development/validation

  23. Conclusion • Development of strong NIH and HHS partnerships • Slow but steady progress in completion of clinical trials and data submission for labeling • Continuing need for access to observational data for efficacy, safety in children

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