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Division of Pediatric Drug Development

Division of Pediatric Drug Development . Shirley Murphy, MD Director, DPDD March 3, 2003. DPDD. Objectives. Provide an overview of DPDD Update on activities NICHD/FDA Partnership Consultative Activities Across FDA New Labels. Medical Officers D. Birenbaum L. Grylack

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Division of Pediatric Drug Development

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  1. Division of Pediatric Drug Development Shirley Murphy, MD Director, DPDD March 3, 2003 DPDD

  2. Objectives • Provide an overview of DPDD • Update on activities • NICHD/FDA Partnership • Consultative Activities Across FDA • New Labels

  3. Medical Officers D. Birenbaum L. Grylack L. Mathis L. Cooper S. Iyasu B. Lindsay S. Cummins J. Filie S. Buckman H. Sachs S. Olnes Project Managers G. Carmouze R. Addy K. Phucas Support Staff J. Angel P. Curry L. Gilmer Who is in Division of Pediatric Drug Development?

  4. Division of Pediatric Drug Development

  5. What is the Role of Division of Pediatric Drug Development? • Foster pediatric drug development within FDA • Partner with NICHD to identify and obtain pediatric information for off-patent drugs • Provide consultative service to all centers within FDA

  6. What is the Role of Division of Pediatric Drug Development? • Conduct detailed review of proposed pediatric trials for on-patent drugs • Contribute to resolution of scientific/ethical issues • Disseminate new pediatric labeling information

  7. What is the Role of Division of Pediatric Drug Development? • Partnership with NIH/NICHD • “FDA-NICHD: Marriage Made In Congress” FDA/DPDD NIH/NICHD

  8. FDA/NIH Partnership • Develop “List of Drugs for Which Pediatric Studies are Needed” • Long, involved iterative process lead by Bill Rodriguez/George Giacoia • Input obtained from Advisory Committee, FDA Divisions, NIH Divisions, AAP, USP, and Experts.

  9. Development of the List • The BPCA stipulates that in developing and prioritizing the list, the NIH shall consider; • the availability of information concerning the safe and effective use of the drug in the pediatric population • whether additional information is needed • whether new pediatric studies concerning the drug may produce health benefits in the pediatric population

  10. List Development 5/19/00 FDA publishes list of 426 drugs where further pediatric studies are needed 6/11/02 Pediatric Advisory Subcommittee Meeting 7/02 NIH gives list of 426 drugs to USP USP removes some on-patent drugs 7/15/02 NIH sends new list of 284 drugs to FDA FDA reviews list - removes on-patent drugs, biologics, 505b approvals, off-market drugs, duplicates & groups drugs by division/disease

  11. List Development 9/18/02 FDA sends refined list of 180 drugs back to NIH NIH ranks 180 drugs with input from NIH institutes, AAP and experts NIH divides drug list Lower Priority High Priority (List kept at NIH)

  12. List Development 10/21/02 NIH sends 34 Drug High Priority List back to FDA FDA review divisions rank drugs on public health benefit, other approved products in class, other options & adds inpatient/outpatient data 11/27/02 FDA sends 34 Drug List with above information to NIH NIH obtains written review by subspecialty expert on each drug

  13. List Development 12/10/02 NIH convenes expert panel (AAP, COD, pharmacologists, subspecialty pediatricians, FDA, USP) & each drug is reviewed & scored by NIH system (100 high-500 low) Scores totaled by NIH 12/13/02 FDA/NIH meet & select 12 drugs from top 16 1/21/03 NIH publishes “List of Drugs for Which Pediatric Studies are Needed”

  14. Azithromycin Baclofen Bumetanide Dobutamine Dopamine Furosemide Heparin Lithium Lorazepam Nitroprusside Rifampin Spironolactone 2003 List of Drugs for Which Pediatric Studies Are Needed

  15. FDA/NIH Off-Patent Drug Process • BPCA establishes a process to study off-patent drugs in collaboration with NIH • DPDD performs existing literature/label review • DPDD writes detailed plan for studies (Written Request) • No response to WR, NICHD issues Request for Contract (RFC)

  16. Process for the Study of Off-Patent Drugs Priority List of Off-Patent Drugs Industry agrees to conduct studies FDA issues Written Request yes Industry declines to conduct studies no Industry has 30 days to respond Referral to NIH

  17. FDA/NIH Partnership • RFC for Coordinating Center Published in Federal Business Opportunities • RFCs to be published • Nitroprusside - to reduce blood pressure in pediatric patients • Lorazepam - long-term, continuous sedation in ICU setting • Lorazepam - treatment of status epilepticus • 5 other off-patent drugs in process

  18. FDA/NIH Partnership • NICHD/FDA Newborn Drug Development Initiative • BPCA recognizes neonates as a specific pediatric subpopulation • Goal: foster the development of safe and effective drug therapies for the pre-term and neonatal population

  19. FDA/NIH Partnership • NICHD/FDA to conduct workshop in early 2004 to frame the state of the art and define research priorities for pain control, cardiac, neurological, and pulmonary diseases • Planning meeting with 50 multidisciplinary experts held 2/10/03 • Working groups established

  20. Consultative Service To All Centers Within FDA • Devices • Cochlear Implants • Breast Implants • GI Devices • Other • CFSAN • Infant formula additives • Other • phthalates • scientific/safety/ ethical issues • Formal Consulting Process with Counter-Terrorism

  21. Prussian Blue Label • Treatment of internal contamination of radioactive and non-radioactive Cesium or Thallium • Efficacy extrapolated from adult data, safety and efficacy supported by information from pediatric patients exposed in the Goiânia accident • 27 pediatric patients received PB after exposure to Cesium • Reduced half life by 46% in adolescents and 43% in children

  22. Isotretinoin Famotidine Omeprazole Mometasone Montelukast Tamoxifen Lamivudine Atorvastatin Simvastatin Cetirizine Pravastatin Vinorelbine Atomoxetine Fluoxetine Busulfan Labeled Products(6/02 - 2/03)

  23. Labeled Products with Significant New Information Montelukast (Singulair) - prophylaxis and chronic treatment of asthma • dose, PK & AE profile in pts. 12-23 mo. & 2-5yrs. • new formulations: 4mg chewable tablet & 4mg oral granule

  24. Labeled Products with Significant New Information Mometasone (Elocon) -Corticosteroid responsive dermatoses Cream & Ointment: • evidence of HPA Axis suppression in ped patients 6-23mo. • skin atrophy in pediatric patients 6mo-2yr. • Should not be used for treatment of diaper dermatitis Lotion: • Safety and effectiveness have not been established in ped patients below 12 yrs and use is not recommended • Should not be used for treatment of diaper dermatitis

  25. Labeled Products with Significant New Information Tamoxifen (Nolvadex) • safety and effectiveness studied in female patients aged 2-10 yrs. with McCune-Albright Syndrome and precocious puberty treated for up to 12 months. Long term effects have not been established. • relative to prestudy baseline • 50% reduction in frequency of vaginal bleeding • Reduction in mean rate of increase of bone age • linear growth rate reduced in majority of patients during treatment • mean uterine volume increased after 6 months of therapy and doubled at end of 1 yr. study

  26. Labeled Products with Significant New Information Pravastatin (Pravachol) - Heterozygous Familial Hypercholesterolemia • new indication in boys and girls 8-18yrs Atorvastatin (Lipitor) - Heterozygous Familial Hypercholesterolemia • new indication adolescent boys and girls (post-menarche) Simvastatin (Zocor) - Heterozygous Familial Hypercholesterolemia • new indication in adolescent boys and girls (at least one year post menarche) 10-17yrs.

  27. Labeled Products with Significant New Information Vinorelbine (Navelbine) • no meaningful clinical activity in a variety of tumors (e.g., recurrent solid malignant tumors, including rhabdomyosarcoma/undiff sarcoma, neuroblastoma, and CNS tumors) Atomoxetine (Strattera) - ADHD down to 6yrs. • First non-stimulant (SNRI) drug labeled for ADHD • NME/non-scheduled drug • unknown whether final adult height or weight is affected by treatment • patients on long-term treatment should be monitored

  28. Labeled Products with Significant New Information Fluoxetine (Prozac) - Major Depressive Disorder (MDD) Obsessive Compulsive Disorder (OCD) • Effectiveness established MDD 8-17yrs. & OCD 7-17yrs. • Decreased weight gain observed with use of fluoxetine as with other SSRIs • In one 19 week clinical trial ped subjects treated with fluoxetine gained an average of 1.1cm less in height (p=0.04) and 1.1kg less in weight (p=0.008) than those treated with placebo • height and weight should be monitored periodically in pediatric patients treated with fluoxetine

  29. Labeled Products with Significant New Information Fluoxetine (Prozac) - MDD & OCD (con’t.) • Mania/hypomania led to discontinuation of 1.8% of fluoxetine treated patients vs 0% of placebo controlled patients in 3 placebo controlled trials combined • regular monitoring for the occurrence of mania/hypomania is recommended

  30. Summary “The FDA is the most interesting and fun place you could ever work” Jane Henney, MD 5/02 (Former FDA Commissioner) Please send me the CVs of all your friends and colleagues.

  31. To Learn More: Internet: www.fda.gov/cder/pediatrics Peds Line: 301-594-PEDS Email: pdit@cder.fda.gov

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