Pediatrics

Pediatric Drug Development

Contents:

• Best Pharmaceuticals for Children Act (BPCA)
  • BPCA/Pediatric Exclusivity Statistics
• Pediatric Research Equity Act (PREA)
  • Pediatric Rule Statistics
• Guidances for Industry
• Regulations
• Pediatric Advisory Subcommittee of the Anti-Infective Drugs Advisory Committee
• Counter-Terrorism Measures for Use in Pediatrics
• Presentations by CDER Staff on Pediatric Issues
• Organization
• Historical References
• Other Resources
• How to Contact Us

Best Pharmaceuticals for Children Act (BPCA)

• Best Pharmaceuticals for Children Act, January 4, 2002 (Public Law No. 107-109).  Optional Format: PDF   (posted 1/31/2002)

BPCA/Pediatric Exclusivity

• The Pediatric Exclusivity Provision - January 2001 Status Report to Congress  [PDF ]  (posted 1/9/2001)
• FDA Modernization Act: Section 111  (posted 11/21/97)
• Guidance for Industry: Qualifying for Pediatric Exclusivity Under Section 505A of the Federal Food, Drug and Cosmetic Act.  Optional Format: PDF    (issued 9/1999; posted 10/4/1999)

BPCA/Pediatric Exclusivity Statistics

• Pediatric Exclusivity Labeling Changes (updated 8/4/2004)
• Breakdown of Requested Studies Report  (updated 10/6/2004)
• Written Requests Issued (updated 10/6/2004)
• Pediatric Exclusivity Granted (updated 10/29/2004)
• Pediatric Exclusivity Statistics (updated 10/6/2004)
• Spectrum of Diseases/Conditions  (updated 10/6/2004)
• Summaries of Medical and Clinical Pharmacology Reviews  (updated  9/17/2004)
  Federal Register Notice:
  • Summaries of Medical and Clinical Pharmacology Reviews of Pediatric Studies; Availability (69FR33043) [Optional Format: PDF ] (issued 6/14/2004)
  • Summaries of Medical and Clinical Pharmacology Reviews of Pediatric Studies; Availability (68FR40678) [Optional Format: PDF ] (issued 7/08/2003)

Written Request Templates

• General Written Request for Pediatric Studies.  Optional Format: Word  (updated 7/1/2004)
• Written Request for Antidepressants.  Optional Format: Word97  (updated  1/06/2003)
• Written Request for Drugs Indicated to Treat HIV Infection. Optional Format: Word (updated 8/19/2004)
• Written Request for Obsessive-Compulsive Disorder (OCD) Drugs.  Optional Format: Word97 (updated 1/06/2003)
• Sample Written Request for Oral Antihypertensives.
  (This page is being revised and is currently unavailable as of 11/9/2001.)
• Written Request for Oncology Drugs.  Optional Format: Word 97 (updated 01/06/2003)

BPCA/Off-Patent Drugs

• Approved Active Moieties for which FDA has Issued a Written Request for Pediatric Studies under Section 3 of the Best Pharmaceuticals for Children Act  (updated 9/30/2004)
• List of Drugs for Which Pediatric Studies are Needed
  • Federal Register Notice (69 FR 7243)  List of Drugs for Which Pediatric Studies Are Needed   Optional Format: PDF    Issued February 13, 2004.  (posted 2/25/2004)
  • Federal Register Notice (68 FR 48402)  List of Additional Drugs for Which Pediatric Studies Are Needed  Optional Format: PDF   Issued August 13, 2003.  (posted 11/03/2003)
  • Federal Register Notice (68 FR 2789) List of Drugs for Which Pediatric Studies are Needed  [PDF]  Issued January 21, 2003.  (posted 11/03/2003)

BPCA/Access to New Therapeutic Agents for Pediatric Cancer

• Patient Access to New Therapeutic Agents for Pediatric Cancer,  December 2003: Report to Congress. [Word] (posted 2/11/2004)

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Pediatric Research Equity Act (PREA)

• Pediatric Research Equity Act of 2003 (Public Law No: 108-155)  Optional Format: PDF   (posted 12/16/2003)

Documents/Information Related to the Pediatric Rule

• 1998 Pediatric Final Rule: Regulations Requiring Manufacturers to Assess the Safety and Effectiveness of New Drugs and Biological Products in Pediatric Patients.  Optional Format:  PDF   (11/27/98)
• Guidance for Industry: Recommendations for Complying with the Pediatric Rule.  Optional Format: Word97
• Advance Notice of Proposed Rulemaking (ANPRM): Obtaining Timely Pediatric Studies of and Adequate Pediatric Labeling for Human Drugs and Biologics.  Optional Format:  PDF
• Press Release: Bush Administration Will Seek New Legislation for Mandatory Pediatric Drug Testing (12/16/02)

Pediatric Rule Statistics

• Pediatric Rule Update (updated 05/02/2003)
• Pediatric Rule Labeling Changes (posted 01/24/2003)

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Guidances for Industry

• ICH-E11 Clinical Investigation of Medicinal Products in the Pediatric Population  [PDF ]  (issued 12/2000, posted 12/14/2000)
• Guidance for Industry:  Qualifying for Pediatric Exclusivity Under Section 505A of the Federal Food, Drug, and Cosmetic Act.  Optional Format: PDF (issued 9/1999, posted 10/4/1999)
• Guidance for Industry: Pediatric Oncology Studies In Response to a Written Request.  Optional Format: PDF (issued 6/2000, posted 6/19/2000)
• Guidance for Industry: General Considerations for Pediatric Pharmacokinetic Studies for Drugs and Biological Products [PDF ] (issued 11/1998, posted 11/12/1998)
• Guidance for Industry: Content and Format of Pediatric Use Supplements [PDF ] (issued 5/1996)

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Regulations

• 1994 Pediatric Final Rule: Specific Requirements on Content and Format of Labeling for Human Prescription Drugs; Revision of "Pediatric Use" Subsection in the Labeling.  (21 CFR Part 201)
• Interim Rule: Additional Safeguards for Children in Clinical Investigations of FDA-Regulated Products.   Optional Format: PDF   (posted 4/26/2001)
• 21 CFR 201.57 Specific Requirements on Content and Format of Labeling for Human Prescription Drugs   Optional Format: PDF

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Pediatric Advisory Subcommittee of the Anti-Infective Drugs Advisory Committee

Consensus Statements from Pediatric Advisory Subcommittee Meetings

• FDA Pediatric Ethics Working Group Consensus Statement on the Pediatric Advisory Subcommittee’s April 24, 2001 Meeting (posted 11/28/2003)   
• FDA Ethics Working Group Consensus Statement on the Pediatric Advisory Subcommittee’s November 15, 1999 Meeting (5/11/2000)
• FDA Pediatric Ethics Working Group Consensus Statement on the Pediatric Advisory Subcommittee’s September 11, 2000 Meeting (posted 9/7/2001)

Meetings of the Subcommittee

• March 3, 2003
•  Agenda and Transcripts
• June 11, 2002
  • Agenda and Transcripts
• April 23-24, 2001
• Agenda  [PDF ]
• Transcripts  [PDF ]  [Text (RTF)]
• September 11-12, 2000
•  Agenda  [PDF ]  [Word97]
• Transcripts  [PDF ]  [Text (RTF)]
• November 15-16, 1999
• Agenda and Questions (posted 11/9/1999)
• Transcripts (posted 12/10/1999)
• April 23, 1999
• Agenda (posted 4/20/1999)
• Transcripts (posted 5/14/1999)

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Counter-Terrorism Measures for Use in Pediatrics

• Pediatric Measures for Counter-Terrorism
  (Topics including: anthrax, radiation emergencies, chemical agents, and vaccines for all patient populations)

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Presentations by CDER Staff on Pediatric Issues

(Presentation slides do not provide complete information regarding labeling changes. For more complete labeling change information, including hyperlinks to the labeling of most products referenced in the presentation, please see: http://www.fda.gov/cder/pediatric/labelchange.htm.)

2004

• The Role of the FDA in Pediatric Research – July 16, 2004, Dianne Murphy, M.D.  Presented at the Glaser Pediatric Research Network Symposium (posted 9/2/2004)
• Regulatory Framework for Pediatric Drug Development in the U.S. – June 16, 2004, Shirley Murphy, M.D. Presented at the Drug Information Association Annual Meeting. (posted 6/24/2004)
• Impact of Pediatric Initiatives – June 15, 2004, Dianne Murphy, M.D. Presented at the Drug Information Association Annual Meeting. (posted 6/16/2004)
• FDA Perspective on FDAMA: Successes, Failures; Future Directions – May 3, 2004, Rosemary Roberts, M.D. (posted 6/1/2004)

2003

• Pediatric Advisory Subcommittee of the Anti-Infective Drugs Advisory Committee, June 12, 2003:
  • Section 17, Best Pharmaceuticals for Children Act, Adverse Event Reporting. Solomon Iyasu, MD.  Optional Format: PowerPoint (Posted 6/16/2003)
• Drug Information Association Meeting: "Pediatric Studies Initiative" March 27-28, 2003:
• Best Pharmaceuticals for Children Act: an FDA Implementation Perspective.  Debra Birenbaum, M.D.  (posted 4/10/2003)
• Fruits of the Pediatric Initiatives: a CDER Update.  Debra Birenbaum, M.D.  (posted 4/10/2003)
• Pediatric Advisory Subcommittee of the Anti-Infective Drugs Advisory Committee, March 3, 2003:
• Adverse Event Tracking as Mandated by the Best Pharmaceuticals for Children Act.  Solomon Iyasu, M.D., M.P.H.  (posted 3/14/2003)
• Division of Pediatric Drug Development .  Shirley Murphy, M.D.  (posted 3/14/2003)
• Overview of Postmarketing Safety Surveillance in FDA (For Drugs and Biologics).  Min Chen, M.S., R.Ph.
• Pediatric Drug Development: Statistical Update.  Terrie Crescenzi, R.Ph.  (posted 3/14/2003)
• Pediatrics Update. Dianne Murphy, M.D. (posted 3/14/2003)

2002

• The Federal Government: Recent Changes at FDA and the Implications for Pediatric Clinical Trials -- Rosemary Roberts, M.D.   Presented at Washington University in St. Louis, October 15, 2002
• Drug Information Association Annual Meeting, 2002
• Pediatric Initiatives: a Regulatory Perspective of the U.S. Experience -- June 17, 2002,  Debra Birenbaum, M.D.  (posted 7/9/2002)
• Adequacy of Current Laws Governing Research in Children -- May, 2002, Rosemary Roberts, M.D. (posted 5/22/2002)
• 2002 FDA Science Forum: Children's Health Issues:
  • Mercury, Lead and Pesticides: Developmental and Reproductive Effects -- February 20, 2002, Ana Maria Osorio, M.D., M.P.H. (posted 7/22/2002)
  • CDER's Experience: What We Have Learned from The Pediatric Initiative -- February 20, 2002, William Rodriguez, M.D. (posted 7/19/2002)

2001

• What’s So Special About Children?  Rosemary Roberts, M.D.  Presented at the American College of Toxicology Annual Meeting, November 6, 2001

2000

• Drug Information Association, Annual Meeting, 2000:
  • Integrating Pediatric Studies into Drug Development Regulatory Perspectives, Victor Raczkowski, M.D. (7/25/2000)
  • Update on FDA’s Pediatric Labeling Regulations and Initiatives on Patient Drug Information, Victor Raczkowski, M.D.   (7/25/2000)
• Problems and Issues with Implementation of FDAMA and the Final Rule - DIA, April 3, 2000- Dianne Murphy, M.D, (4/7/2000)
• Where Have We Been and Where are we Heading? - DIA April 3, 2000 - Rosemary Roberts, M.D. (4/7/2000)

1999 

• Pediatric Initiatives Update,  Murray M. Lumpkin, M.D.,  October 20, 1999  
• Pediatric Initiatives, 1999 Regulatory Affairs Professional Society Annual Conference: Leanne Cusumano, Regulatory Counsel, CDER (10/13/1999)
• Drug Information Association, Annual Meeting, 1999:
• Safety Assessment in Pediatrics, Rosemary Roberts, M.D., Medical Officer, Pediatrics Team, Center for Drug Evaluation and Research 
• Exclusivity Statistics, Dianne Murphy, M.D. Associate Director for Pediatrics (posted 7/02/1999)
• 1999 Pediatric Initiatives, Dianne Murphy, M.D., Associate Director for Pediatrics (posted 7/02/1999)
• Pediatric Subcommittee; Anti-Infective Drugs Advisory Committee, 4/23/1999
• 1999 Food & Drug Law Institute Annual Education Conference - Exclusivity: Pediatrics & 505(b)(2)s, Leanne Cusumano (1/14/2000)  

1998

• Drug Information Association Workshop: Pediatric Drug Development: Successes and New Solutions, Rosemary Roberts, M.D., Medical Officer, Pediatrics Team, Center for Drug Evaluation and Research, April 20-21, 1998

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Organization

• CDER Organization Chart
• Office of Counter-Terrorism and Pediatric Drug Development/Division of Pediatric Drug Development Organization Chart

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Historical References

• List of Approved Drugs for Which Additional Pediatric Information May Produce Health Benefits in the Pediatric Population (updated 5/22/2001)
• Pediatric Cancer Therapeutics: Letter of Interest (2/15/2000)
• Frequently Asked Questions on Pediatric Exclusivity (505A), The Pediatric "Rule," and their Interaction (posted 7/27/1999)
• FDA Acts to make Drugs Safer for Children (11/27/1998)
• Draft List Published of Drugs Needing Pediatric Data - March 25, 1998
• Press Release: FDA Proposes to Require Pediatric Data Prior to Drug and Biologic Product Approvals - August 13, 1997
• Proposed Rule: Regulations Requiring Manufacturers to Assess the Safety and Effectiveness of New Drugs and Biological Products in Pediatric Patients.  Optional Format: PDF   (posted 8/15/97)
• Public Meeting Agenda and Transcripts: Pediatric Proposed Rule, October 27, 1997
• From News Along the Pike,  January, 1997:
  "Pediatric Corner:  Center IDs Top 10 Drugs Used Off-Label in Out-Patient Setting"   [PDF  ]

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Other Resources

• American Academy of Pediatrics
• ClinicalTrials.gov, a service of the National Institutes of Health, provides consumers with easy and free access to information on clinical studies for a wide range of diseases and conditions.
• "Drug Research and Children." FDA Consumer article
• National Institute of Child Health and Human Development
• "Ten Guiding Principles for Teaching Children and Adolescents about Medicine" (From the U.S. Pharmacopoeia)

How to Contact Us

• Phone:  (301) 594-PEDS  ((301) 594-7337)
• E-mail:  pdit@cder.fda.gov
• Pediatric Comment and Feedback Form

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FDA/Center for Drug Evaluation and Research
Last Updated: October 29, 2004
Originator: OCTAP/DPDD
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