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Overview of Pediatric Drug Development

Overview of Pediatric Drug Development. Dianne Murphy, MD Director Office of Counterterrorism and Pediatric Drug Development Center for Drug Evaluation and Research February 2, 2004. Goals.

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Overview of Pediatric Drug Development

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  1. Overview of Pediatric Drug Development Dianne Murphy, MD Director Office of Counterterrorism and Pediatric Drug Development Center for Drug Evaluation and Research February 2, 2004

  2. Goals • Provide condensed historical background to better understand how we arrived at the present place in pediatric drug development • Review FDA’s Pediatric Drug Development responsibilities

  3. Acronyms • FDAMA – Food and Drug Administration Modernization Act • BPCA – Best Pharmaceuticals for Children Act • PREA – Pediatric Research Equity Act • WR – Written Request (FDA issues) • PPSR – Proposed Pediatric Study Request (sponsor submits)

  4. Historical Perspective • Tragedies in children lead to new laws • 1902 - Biologics Control Act • diphtheria antitoxin contaminated with live tetanus bacilli • 1938 - Food, Drug and Cosmetic Act • sulfanilamide • 1962 - Amendments • thalidomide

  5. BENCHMARKSPediatric Drug Development • 1977 – AAP statement concerning the need to conduct trials in children • 1979 – FDA requires trials in children parallel to adult process • 1994 – FDA requires sponsors to update label; introduces “extrapolations” • 1997 – Congress passes FDAMA/Exclusivity Provision – “Incentives” (voluntary) • 1998 – FDA publishes Pediatric Rule (mandatory)

  6. BENCHMARKSPediatric Drug Development (cont.) • 2002 –Congress passes Best Pharmaceuticals for Children Act (BPCA) • renewed Exclusivity • provides process for “off-patent” drug development • public posting of results • reporting of all AE’s for 1 year after Exclusivity granted • 2003 – Congress passes Pediatric Research Equity Act (PREA) • Requires the study of drugs and biologics for pediatric population except in defined situations • Creates Pediatric Advisory Committee

  7. Impact of Legislation • Proposals from Industry 336 • FDA-issued Written Requests 283 • Exclusivity Determinations 101 • Exclusivity Granted 91 • New Labels 63

  8. Tamoxifen Quinapril Paroxetine Nefazodone Citalopram Pravastatin Vinorelbine Sertraline Oxybutynin Atorvastatin Simvastatin Busulfan Cetirizine Losartan 1-Year Post-ExclusivityReporting of Adverse Events Products

  9. Ethical Issues • Pediatric Advisory Subcommittee Meetings have addressed: • Patients vs. subjects in pediatric trials (11/15/99) • Placebo controlled trials (9/11/00) • Vulnerable pediatric populations (4/24/01)

  10. Prior Peds Advisory Subcommittee Discussion 1999 • November: Issues regarding a Pediatric Drug Development Program for the Treatment of Insomnia 2000 • September: Pediatric Psychotropic Drug Use Issues 2001 • April: Treatment of Chronic Hepatitis C in Children 2002 • June: Reflux disease; GERD-Template

  11. Prior Peds Advisory Subcommittee Discussion (cont.) 2003 • March: Development of Antiretroviral Drugs in HIV-Infected and HIV-Exposed Neonates Younger than four weeks of age • June: Current Epidemiology and Therapeutic Interventions Relevant to Hyperbilirubinemia in the Term and Near-Term Newborn • October: Clinical Risk Management of HPA Axis Suppression in Children with Atopic Dermatitis being treated with Topical Corticosteroids Tracking Cancer Risk among Children with Atopic Dermatitis who are treated with Topical Calcineurin Inhibitors

  12. ONGOING LESSONS LEARNED 1- Pharmacokinetics are more variable than anticipated 2-Adverse reactions that are pediatric specific are being defined 3-Trial designs are being modified as we learn from submitted studies 4-Ethical issues have to be reassessed from the pediatric perspective

  13. General Principles* • Pediatric patients should be given medicines that have been properly evaluated for their use in the intended population • Product development programs should include pediatric studies when pediatric use is anticipated • Pediatric development should not delay adult studies nor adult availability • Shared responsibility among companies, regulatory authorities, health professionals, and society as a whole * from ICH E-11

  14. To Find out More Internet: http://www.fda.gov/cder/pediatric Peds Line: (301) 594-7337

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