1. Interactive Session: Presentation of Scenarios and Q&A
Linda Bowen
Meredith Brown-Tuttle
Amy Grant
And YOU the audience

2. Disclaimer
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These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. Drug Information Association, DIA and DIA logo are registered trademarks or trademarks of Drug Information Association Inc. All other trademarks are the property of their respective owners.

3. We welcome your participation!
Interactive Session
Scenarios representing real-life development and life-cycle management challenges
Demonstration of thought process, information gathering, and analysis of the situation
Participation from the audience
Demonstration of use of Regulatory Strategy Plan throughout life-cycle of the product
We welcome your participation!

4. Special Attention to Specific Regulatory ProceduresEU US
Fast Track; Rolling Submissions;
Frequent Agency Meetings
Conditional Approval
Accelerated Approval (Subpart H) Accelerated Development (Subpart E)
Approval under Exceptional Circumstances
Accelerated Assessment
Priority Review
Scientific Advice
EOP1/EOP2 Meetings/
Special Protocol Assessment (SPA)
Named Patient Use
Single Patient IND/Physician IND
Orphan designation

5. Data Protection Type US EU Orphan 7 years 10 years NCE 5 years 10 (CP)
8+2+1 (National/ MRP/DCP/CP)
New indication
3 years
+1 year marketing protection for new indication with significant therapeutic benefit
Pediatric/Paediatric
6 months added to current protection
6 months SPC extension
For orphan products: 2-year extension of the 10-year market exclusivity
PUMA years (pediatric use marketing authorization)
Generic
180 days to first ANDA filer
No protection

6. Details from the Original Applications
Indication: post-surgical pain
Dosage form: two separate traditional NDAs/ 505(b)(1) applications for IV and tablet dosage forms; both dosage forms approved 1986 (US) and tablet approved in 1990 via the national procedure and marketed in the UK, France and Italy (EU)
No remaining patent protection/data exclusivity
Underwent standard review
Indicated for adults only 6

7. Our Goal
To develop a strategy for a new dosage form, to accommodate either a new indication and/or target population, or both. The following scenarios are indicative of the types of issues you would encounter when developing a strategy. 7

8. The Scenarios Pediatric Indication & exclusivity
Least burdensome pathway to approval
Orphan Designation
Priority/Accelerated Assessment/ Fast Track
Risk Minimization 8

9. Scenario 1: Pediatric Indication
Is there a possibility of a pediatric indication for post-surgical pain with the new dosage form? Other indications? Could we get 6 months of pediatric exclusivity for this product in the US and EU? How would we account for this in the regulatory plan? 9

10. Scenario 2: Pathway to Approval
What would be the least burdensome U.S. regulatory pathway for approval of:
New dosage form, but no new indication?
New dosage form and a new indication?
How would you support your application? 10

11. Scenario 3: Orphan designation
Would the new indication, adult and/or pediatric neuropathic pain, support an application for orphan designation in the US and EU? What would be the benefits of orphan designation? What would be the regulatory pathway? 11

12. Scenario 4: Priority/Fast Track
Would the new indication, adult and/or pediatric neuropathic pain, be eligible for:
Priority review (US)
Accelerated Assessment (EU)
Fast Track Designation (US) 12

13. Scenario 5: Risk Management
Although the earlier dosage forms were approved without a risk management plan, would the company have to provide a
REMS to FDA for a new NDA or supplement? What about in Europe? 13