# Presentation: 412711

## 	Exclusivity Statistics

**   ****Dianne Murphy, M.D.****	****	****	****	****	****Associate Director for ****	****	****	****	****	****	****     Pediatrics**

- FDA

## An Act105th Congress

**The “Food and Drug Administration Modernization Act of 1997”**
**Sec. 111**
**Pediatric Studies of Drugs**
**Signed into law 11/21/97**

- FDA

## Slide 3

## Slide 4

## TOTAL PPSR Actions

- 76 Written Requests
- 20 Incomplete Responses
- 1 PPSR Withdrawal
- 13 Amended Written Requests
**TOTAL ACTIONS = 110**
**PPSRs RECEIVED = 130**
**PPSRs PENDING = 39**
**PPSRs > 4 months = 14**

## Exclusivity Summary

**As of May 1999**
**Granted Exclusivity:****	****5**
**Pending Review:****	****3**

## Average Time to Issuance of Written Requests

## Rate of Submissions Over Time

**Rate of Submissions Over Time**

## Written Request Types of of StudiesAs of May 31, 1999

- Data is from 76 Written Requests
- Efficacy/Safety: 63
- PK/Safety: 58
- PK/PD: 10
- Safety: 26
- Other: 14
- TOTAL: 171 studies

## Efficacy Trials (as of 5/31/99)Probable:  Different Disease Expression or Response to Therapy

- Hypertension:	     	16
- Depression:	     	14
- Anesthesia/Sedation:	       	  4
- Cholesterol Lowering:       	  3
- Asthma:		      	  3
- OCD:	          	  3
- HIV:		       	  2
- Influenza:  	     	  2
- Seizures:		       	  2
- Malaria:		       	  2

- GAD:		       	2
- Allergic Conjunctivitis:  	2
- Hepatitis B:	  	1
- Hepatitis C:		1
- Antibiotic:		       	1
- Smoking Cessation:     	1
- Migraines:		      	1
- JRA:		       	1
- Diabetes:		       	1
- Anti-fungal:		1
- 		   TOTAL:              63

## Safety Studies

- 26 Safety “only” studies
- 20 Products for which Written Requests have been issued
- Patient numbers: 30 (270) to 3,000
- as of 5/31/99

## Slide 12

## Slide 13

## Percentage of AERs cases by age1997-1998

- 0-30 days			0.5%
- 0-16 years			 5%
- 17 years and up	        70%
- age unknown		 25%

## Slide 15

## “Other” Studies

- Prophylaxis
- OTC Actual Use Studies
- Combination of Efficacy, Safety and PK

## Ages Included in Written Requests

- Preterms:				  1
- 0 - < 2 months:			13
- 2 months - < 2 yrs:		24
- 2 yrs. - 12 yrs:			29
- 12 yrs. - 16 yrs:			26
- Other:					22

## AGES:  OTHER

- Neonate - Tanner Stage 3
- 0 - 3 months
- 10 months - 19 yrs.
- 1 - 3 yrs.
- 2 yrs. - 6 yrs.
- 3 yrs. - adolescence
- 10 yrs.- 17 yrs.

## Number of Pediatric PatientsAs of May 31, 1999

- 31 Written Requests specified number of patients in studies
-    9332 - TOTAL # of patients
- (-) 3800   (# of patients involved in one  			long-term study)
- =	 5532 Patients / 30 Written Requests
-     An average of 184 patients per WR

## Approved Drug Products to which FDA has issued a Written Request for Pediatric Studies 

## Approved Drug Products to which FDA has issued a Written Request for Pediatric Studies 

## Approved Drug Products to which FDA has issued a Written Request for Pediatric Studies

## Approved Drug Products to which FDA has issued a Written Request for Pediatric Studies 

## ImpactEnormous

- Cultural/mind-set change
- Drug development planning process change
-  “Need” change for pediatric information and science
- Increase in numbers of children involved in clinical trials

## Issues

- Ethical Concerns
- Trial Design Problems
- Regulatory “Bumps”
- Implementation Infrastructure
- Tracking Systems
- Immediate vs. Long Term Research Issues

## Committees and Working Groups

- External Liaisons
- AAP/ICH/NIH-PPRU
- Communications and Education
- Ethics
- Program Implementation
- PdIT/Preclinical/Tracking/Clinical Database
- Clinical Policy Development
- Biopharm/PK Guidance/Pedicomm/ Formulations/Pediatric Guidance (Clinical Trials, Age Concerns, Scenarios)
- Pediatric Advisory Subcommittee

## To Find out More

- Internet:
- www.fda.gov/cder/pediatric
- PEDS Line:
- 301-594-PEDS (7337)