# Presentation: 771143

## Update onPediatric Initiatives	

**Murray M. Lumpkin, M.D.**
**Deputy Center Director**
**Center for Drug Evaluation and Research**
**20 October 1999**
**PPRU Steering Committee**
**Bethesda, Maryland**

## Exclusivity Process

**Proposed Pediatric**
**Study Request**

**Written Request**
** ****from FDA**

**Submission of **
**Study Reports**

**FDA Determines**
**Whether Reports**
**Meet the Terms **
**of the Request**

**Written Agreement**
**on Studies**

**(4 mos)**

**60 days**

**90 days**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Exclusivity Process

**Proposed Pediatric**
**Study Request**

**Written Request**
** ****from FDA**

**Submission of **
**Study Reports**

**FDA Determines**
**Whether Reports**
**Meet the Terms **
**of the Request**

**Written Agreement**
**on Studies**

**(4 mos)**

**60 days**

**90 days**

**159**

**101 Written Requests**
**35 Incomplete Responses**
**1 Withdrawal of the PPSR**
**20 WR Amendments**
**33 Pending (10 >4 mo)**

**10 Exclusivity granted**
**(9 moieties)**
**2 Pending determinations**
**1 Exclusivity denied**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Moietieswith Exclusivity Granted

**Midazolam**
**Ranitidine**
**Abacavir**
**Ibuprofen**
**Propofol**
**Ammonium Lactate**
**3 not yet approved moieties**

- Murray M. Lumpkin, M.D.

- 10/20/99

## PPSRs by Division

**Cardiorenal****	****	****	****25**
**Neuropharm****	****	****20**
**Anti-inflammatory****	****20**
**Anti-viral****	****	****	****20**
**Metabolic/Endo****	****	****16**
**Anesthetic/Abuse****	****12**
**Pulmonary****	****	****	****12**

- Murray M. Lumpkin, M.D.

- 10/20/99

## PPSRs by Division

**Dermatology****	****	****9**
**GI****	****	****	****	****	****7**
**Special Pathogens****	****7**
**Oncology****	****	****	****5**
**OTC****	****	****	****	****3**
**Anti-infective****	****	****1**
**Reproductive****	****	****0**
**Medical Imaging****	****0**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Studies Requested	

**TOTAL****	****	****	****	****228**
**Total # of patients****	****14,772+**
**# of studies specifying # of pts: 102**
**Efficacy and Safety****	****88****	****	****39%**
**PK & Safety****	****	****68****	****	****30%**
**PK/PD****	****	****	****	****25****	****	****10%**
**Safety****	****	****	****	****40****	****	****18%**
**Other****	****	****	****	****  7****	****	****  3%**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Efficacy Studies

**Hypertension****	****	****18****	****(18)**
**Depression****	****	****	****14****	****(  7)**
**Asthma****	****	****  ****	****  7****	****(  4)**
**Cholesterol Lowering 5****	****(  4)**
**Anesthesia/Sedation  4****	****(  4)**
**Atopic Dermatitis****	****  4****	****(  2)**
**Obs Compulsive Dis****	****  3  ****	****(  3)**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Efficacy Studies (contd)

**Intraocular pressure****	****	****3****	****(3)**
**Migraines****	****	****	****	****3****	****(3)**
**Seizures****	****	****	****	****3****	****(2)**
**Diabetes****	****	****	****	****2****	****(2)**
**HIV****	****	****	****	****	****2****	****(2)**
**Influenza****	****	****	****	****2****	****(1)**
**Anxiety Disorders****	****	****2****	****(1)**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Efficacy Trials (contd)

**Malaria****	****	****	****	****2****	****(1)**
**Allergic Conjunctivitis****	****2****	****(2)**
**Allergic Rhinitis****	****	****	****1****	****(1)**
**Eye Allergies****	****	****	****1****	****(1)**
**Hepatitis B****	****	****	****	****1****	****(1)**
**Hepatitis C****	****	****	****	****1****	****(1)**
**Complicated UTI****	****	****1****	****(1)**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Efficacy Trials (contd)

**Smoking Cessation****	****	****1****	****(1)**
**Oropharyng candidiasis****	****1****	****(1)**
**JRA****	****	****	****	****	****1****	****(1)**
**Obesity****	****	****	****	****1****	****(1)**
**Transplant rejection****	****	****1****	****(1)**
**End stage renal disease****	****1****	****(1)**
**Acne****	****	****	****	****	****1****	****(1)**
**TOTAL****	****	****	****	****	****88****	****(61)**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Template Written Requests

**Antidepressants**
**HIV infection**
**Oral Antihypertensives**
**On web site**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Issues

**No real precedents:  sometimes sponsors want minimalistic approach**
**Questions on end points**
**Questions on extrapolatability**
**Many want extreme precision in our making a request and ultimate flexibility in their fulfilling of it**
**No resources given us for this initiative - yet quite resource intensive - lost user fees for pediatric supplements**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Other initiatives

**Pediatric Subcommittee**
**April****	****	**
**Procedural / Gen Discussion**
**November**
**Ethics - Normal volunteers**
**Insomnia - ? Pediatric disease**
**Ethics - placebo-controlled trials, payments for participation**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Other initiatives

**International Conference on Harmonization**
**Pediatric Clinical Development Guidance**
**Step Two in Washington earlier this month  --  out for comment**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Other initiatives

**Guidance Documents**
**Final Guidance on Exclusivity**
**Draft Guidance on PK  (expect final before end of year)**
**Draft Guidance on Pediatric Rule (hopefully before end of year)**
**Rough Draft of Guidance on Clinical Trials  (in more depth than ICH)**

- Murray M. Lumpkin, M.D.

- 10/20/99

## Web Site Information

**www.fda.gov/cder/pediatric/**
**index.htm**
**www.fda.gov**

- Murray M. Lumpkin, M.D.

- 10/20/99