# Presentation: 313388

## Best Pharmaceuticals for Children Act: an FDA Implementation Perspective

**Debra Birenbaum, M.D.**
**Lead Medical Officer**
** ****Division Pediatric Drug Development**
**OCTAP, CDER, FDA**
**March 27, 2003**

## Objectives

**Describe highlights of the Best Pharmaceuticals for Children Act (BPCA)**
**Discuss processes in the implementation of the Act**
**Describe the FDA/NIH Partnership**

## Best Pharmaceuticals for Children Act

**Signed into law  January 4, 2002**
**Provides processes for studying “off-patent” as well as “on-patent” drugs**
**Re-authorized pediatric exclusivity incentives as they apply to drugs* approved under Section 505 of the Federal Food, Drug and Cosmetic Act**
**Sunsets October 1, 2007**
**  *****most biologic pharmaceuticals are approved under the Public Health Service  (PHS) Act and therefore do not qualify for pediatric exclusivity**
**  **

## Best Pharmaceuticals for Children Act

**Provides opportunity for FDA and NIH to collaborate on the drug development process, for drugs that may be administered to children**
**provides processes for studying “off-patent” drugs, as well as “on-patent” drugs**

## Other BPCAHighlights

**Adds neonates**
**Requires adequate representation of racial and ethnic groups**
**Establishes an Office of Pediatric Therapeutics at FDA (ethical issues and post-marketing safety)**
**Mandates public dissemination of pediatric information**

## BPCAOn Patent Drugs

## Process for the Study of On-Patent Drugs

**FDA issues **
**Written Request**

**Industry agrees **
**to conduct studies**

**Industry declines**
**to conduct studies**

**Referral to Foundation**
** ****for NIH**

**Industry has 180 days to respond**

**Industry submits**
**a Proposed Pediatric Study Request**

**FDA determines**
**public health benefit **
**to support pediatric studies**

** ****yes**

** ****yes**

** ****no**

## FDA On-Patent Drug Exclusivity Process

**Background Research/Extensive Literature Review **
**          ****Sponsor**
**Proposed Pediatric****	****Development of Written Request by **
**Study Request (PPSR) ****	****Reviewing Division**
**	****	****	****Reviewed by PdIT         ****	****     **
**	****      ****FDA            ****	****           WR Issued**
**            ****	****        ****	****	**
**	****    ****	****Sponsor Completes Studies****	****	**
**	****or FDA****	****Reviewing Division Reviews                                       ****	**
**	****	**** ****Exclusivity Determined by**
**	****	**** ****Exclusivity Board****	****	****	**

## BPCAOff-Patent Drugs

## An FDA/ NIH Partnership

- Partnership with NIH/NICHD
- “FDA-NICHD:  Marriage Made In Congress”

- FDA/DPDD

- NIH/NICHD

## Process for the Study of Off-Patent Drugs

**Priority List of**
**Off-Patent Drugs**

**FDA issues **
**Written Request**

**Industry agrees **
**to conduct studies**

**Industry declines**
**to conduct studies**

**Referral to NIH**

**Industry has 30 days to respond**

**yes**

**no**

## FDA/NIH Off-Patent Drug Process

**Drug selected from List**
**DPDD performs existing literature/label review**
**DPDD writes a detailed plan studies (Written Request)**
**FDA Review Division Input****	****	****	****    NIH/NICHD Input**
**	****	****	****	****	****Review by PdIT**
**Written Request Issued to Holders of Approved Applications**
**                                       ****yes                                no**
**	****	****	****	****	****	****	**
**           ****Industry does studies****	****	****	****NICHD**

## 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.**
**List of 12 drugs published in Federal Register 1/21/03**

## 2003 List of Drugs for Which Pediatric Studies Are Needed

**Azithromycin**
**Bumetanide**
**Dopamine**
**Heparin**
**Lorazepam**
**Rifampin**

**Baclofen**
**Dobutamine**
**Furosemide**
**Lithium**
**Nitroprusside**
**Spironolactone**

## FDA/NIH Partnership

**FDA recently issued off-patent drugs WRs for Nitroprusside and lorazepam**
**Both drugs referred to NIH **
**NIH Published RFC for Coordinating Center in Federal Business Opportunities**
**RFCs to be published in Federal Register**** **
**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**

## Goal

**On-Patent Exclusivity Process**
** ****and**
**Off-Patent BPCA Process**
**New Pediatric Information in Label**

## What is the Role of Division Pediatric Drug Development?

**FDA Resource to foster pediatric drug development throughout FDA**
**Partner with NICHD to identify and obtain Pediatric information on             off-patent drugs**
**Consultative service to all Centers within FDA**

## What is the Role of Division Pediatric Drug Development?

**Provide detailed review of proposed pediatric trials for on-patent drugs**
**Contribute to resolution of scientific/ethical issues**
**Disseminate new pediatric labeling information**

## Division of Pediatric Drug Development - who are we?

**Medical Officers**
**Project Managers**
**Support Staff**

## Pediatrics Structure

**OCTAP**

**Office of the FDA**
**Commissioner**

**International Activities & Strategic Initiatives**

**CDER**

**Division of **
**Pediatric Drug Development**

**Office of Pediatric Therapeutics**

## Office of Pediatric Therapeuticsa BPCA Provision

**Duties**
**“****Coordination and facilitation of all activities of FDA that may have any effect on a pediatric population **_***OR***_
**the practice of pediatrics **_***OR***_
**may in any way involve pediatric issues”**** **

## Office of Pediatric Therapeutics: a BPCA Provision

**Current**** ****Functions**
**Ethics**
**Adverse Event Reporting****	****	**

## Office of Pediatric Therapeutics

**How to avoid overlap and duplication with Division of Pediatric Drug Development? **
**Solution  = Efficiencies**

## Efficiencies

## To learn more:

**www.fda.gov/cder/pediatric**
*** ***_**Peds Line**_**:**
**    ****(301) 594-PEDS***** ***
_**Email address:**_***   *****pdit@cder.fda.gov**

**To learn more:**