# Presentation: 747122

## Exclusivity	- Pediatrics	- 505(b)(2)s

**Leanne Cusumano**
- Regulatory Counsel
- Center for Drug Evaluation and Research 301-594-2041pdit@cder.fda.gov

- FDA

## What is exclusivity?

- Exclusivity is marketing protection.
- Exclusivity prevents the marketing of an identical generic or 505(b)(2) product for a certain period of time (all 505 blocked by orphan)
- Exclusivity is enforced by FDA
- Exclusivity prevents either the submission or approval of an application

## How many types of exclusivity are there?

- Hatch-Waxman
- New molecular entity - 5 year
- New use - 3 year
- applications
- supplements
- 1st filed ANDA - 180 day marketing
- Orphan Drug Exclusivity - 7 year
- Pediatric Exclusivity

## What is pediatric exclusivity?

- Pediatric exclusivity is an incentive developed by Congress to encourage sponsors to conduct pediatric studies.
- Pediatric exclusivity does not stand alone - it attaches only to existing exclusivity or patents.

## Effect of Pediatric Exclusivity

- attaches to exclusivity or patent life of the active moiety as EXCLUSIVITY extension
- adds 6 months of exclusivity
- possible delay of generic approvals
- once submitted, maximum of 90 days
- six month period runs during delay

## Applications to Which Pediatric Exclusivity Applies

- Applications
- containing the active moiety
- held by the same sponsor
- Unapproved applications upon approval
- Later-filed NDAs & Supplements
- data earned prior ped. ex and
- essential to approval of later-filed NDA or supp.

## Moieties in Multiple Products

- Monotherapy
- montherapy exclusivities
- monotherapy patents
- Combination Therapy
- combination protections
- each montherapy’s protections

## How does a sponsor qualify for pediatric exclusivity?

- FDA issues an official Written Request for pediatric studies
- Sponsor conducts studies in accordance with a Written Agreement or commonly accepted scientific principles
- Sponsor files reports of studies that meet the terms of the Written Request

## Written Requests

- FDA assesses what studies on the moiety are needed to produce a health benefit in the pediatric population.
- Recommend sponsors provide a proposed pediatric study request as a first step.
- Written Request may or may not reflect some or all elements in the sponsor’s proposal.
- FDA can issue a Written Request without  a sponsor’s request.

## Can you earn more than one 6-month period of pediatric exclusivity?

- Response to the Written Request is a supplement for a new use.
- A new use is a use that is not included in the approved labeling of an approved drug, including a new pediatric population.
- The supplement qualifies for 3-year Waxman-Hatch exclusivity
- The 6-month period of pediatric exclusivity attaches *only *to the 3-year exclusivity.

## Sunset

*FDA can issue a Written Request for a drug (active moiety) after January 1, 2002, only if:*
- an application for the drug is submitted on or before January 1, 2002; and
- the drug was in commercial distribution on November 21, 1997; and
- the drug is on the List on January 1, 2002; and
- FDA finds that there is
- continuing need for information and
- the drug may provide health benefits

## Sunset

*Studies submitted on or after January 1, 2002, can earn pediatric exclusivity if:*
- the studies are submitted in response to a Written Request issued on or before January 1, 2002, and
- the Written Request is for a drug (active moiety) for which an application was submitted on or before January 1, 2002.
*FDA cannot issue a Written Request for a drug (active moiety) after January 1, 2002, if the first application for the drug is submitted after January 1, 2002.*

## Report to Congress

- Due January 1, 2001
- Must address:
- effectiveness in obtaining pediatric information
- adequacy of incentive
- economic impact
- suggestions for modification

## Actions as of 12/1/99

- 119 Written Requests
- 39 Incomplete Responses
- 1 PPSR Withdrawal
- 44 Amended Written Requests
**PPSRs RECEIVED = 184**
**TOTAL ACTIONS = 203**
**PPSRs PENDING = 36**
**PPSRs > 4 months = 14**

## Pediatric Exclusivity Determinations as of 12/1/99

**Granted on Approved Moieties:****	****9**
**Granted on Unapproved Moieties:  3**
**Pending Review:****	****2**
**Denials:****	****2**

## Pediatric Exclusivity Summary

- Extends 3/5/7 year exclusivity by 6 months
- Grants 6 months exclusivity after expiration of a submitted patent
- Approval not necessary - ped. ex. determined within 60/90 days after filing of responsive reports of studies
- Sunset January 1, 2002

## Resources on Pediatric Exclusivity

- The Statute:  21 U.S.C. 505a
- Guidance for Industry*:  Qualifying for Pediatric Exclusivity, *including Frequently Asked Questions
- www.fda.gov/cder/pediatric
- PEDS Line:  301-594-PEDS (7337)
- EMail:  pdit@cder.fda.gov
- Pediatric Advisory Subcommittee

## How do 505(b)(2) applications fit into the world of exclusivity?

- Like ANDAs
- Blocked by exclusivity
- Required to file patent certifications
- Unlike ANDAs
- Able to earn exclusivity
- May differ from RLD in more ways than those permitted by suitability petition process

## What is a 505(b)(2) application?

- A new drug application containing
- One or more investigations necessary to approval that
- were not conducted by applicant and for which
- applicant has no right of reference

## What information can an applicant rely on?

- Published literature specific to the approval sought in the NDA
- FDA’s finding of safety and effectiveness for an RLD

## Examples of 505(b)(2) applications

- Dosage form
- Strength
- Route of administration
- Substituted active ingredient in combo.
- Formulation
- Dosing regimen
- Active ingredient
- Intentional bioinequivalence

- Combo. of individually approved products
- Indication
- Rx/OTC Switch
- OTC monograph
- Naturally derived or recombinant active ingredient
- NME

## Resources on 505(b)(2) applications

- 21 USC 355(b)(2)
- 21 CFR 314.50 - NDAs
- 21 CFR 314.54 - 505(b)(2) applications
- 21 CFR 314.108 - Exclusivity
- DRAFT Guidance for Industry:  *Applications Covered by Section 505(b)(2)*

## Exclusivity & Patent Listings

- Published in the Orange Book
- Available in hard copy and at www.fda.gov/cder/ob/default.htm
- Lists all current exclusivities and submitted patents

## Where can I get more information?

- Leanne Cusumano, 301-594-2041
- GC (Kim Dettelbach), 301-827-1148
- OGD
- Pediatric Implementation Team
- 301-594-PEDS
- Terrie Crescenzi, PM