1. Partnering with Regulatory Intelligence Speakers: Amy Grant, MS Viropharma Incorporated Penny Levin, MS Cephalon Danielle Ziernicki, Pharm.D. Johnson & Johnson Pharmaceuticals Group 1

2. The views expressed in this presentation are the personal views of the authors and do not necessarily represent the views of their respective companies.

4.  What is regulatory information?  What is regulatory intelligence?  How we interact and engage with other intelligence groups  Partnering Benefits  Case studies

5. Regulatory information Regulatory information is obtained through reliable regulatory resources extracted from fact-based primary information. –Health Authority Information –Regulatory News Sources & Databases It has official citation/ reference source. Data gathering methodology can be reproduced. It is the foundation of regulatory policy and intelligence. 5

6. 6 Laws, Regulations, GuidanceAdvisory MeetingsApprovals Health Authority alerts and websites Ad Hoc Health Authority Queries (Freedom of Information) Regulatory NewsRegulatory Databases

7. Regulatory Information  Primary Source: ◦ Evidence-based ◦ Scientifically focused ◦ Rigorous ◦ Health Authority public information of record reflecting their assessments, guidance, and perspective ◦ Published medical & scientific information available in bibliographic databases, clinical trial registries, and other clinical resources Competitive Information  Primary Sources ◦ Evidence-based ◦ Commercially focused ◦ Rigorous ◦ Market Research ◦ Published medical & scientific information available in bibliographic databases, commercial trade journals, and other commercially focused resources ◦ Soft Intelligence:  Direct conversations 7

8. Regulatory Intelligence (RI) Gathering & analyzing publicly available regulatory information; communicating implications of that information & monitoring the current regulatory environment for opportunities to shape future regulation, guidance, policy and legislation*. Competitive Intelligence (CI)  The continuous process involving the legal and ethical collection of information, monitoring the competitive environment, analyzing the findings in the context of internal issues, and controlled dissemination of actionable intelligence to decision makers. ** * DIA Regulatory Intelligence Network (RING) definition **Abbreviated definition from Strategic and Competitive Intelligence Professionals (SCIP) definition

9. 9 Analysis of Regulatory Pathway & Hurdles Interpretation of laws, regulation and guidance Analysis of regulatory precedent for policy and therapeutic area key intelligence topics (KITS) Regulatory Strategy Strategy

10. Compound Strategy Due diligence strategy Indication Sequencing Guides Establishes regulatory precedent /pathway Challenges & critical success factors Asseses likelihood of success Identifies Health Authority requests TA project challenges Situational analysis Regulatory precedent Addresses To Provide Regulatory Expertise that……

11. Approaches to enhance or streamline drug development Recommends Policy Health Authority Guidance (via commenting) Influences Supports Decisions To Provide Regulatory Expertise that…… Mergers/Acquisitions Go/ No-go Decisions

12. Key Intelligence Questions (KIQ) Regulatory Intelligence KIQ’s Key Intelligence Topic (KIT) Competitive Intelligence KIQ’s

13.  Business Intelligence  Clinical Development  Commercial  Corporate Communications  Federal Government Affairs  Global Medical Affairs  Legal  Medical Communications  Project Management  State Government Affairs * Others… (depends on the size of company and scope of the department )

14. Set regular meetings Informal and formal Partner for meeting conference coverage (medical meetings)Collaborate & Share Expertise Alerts Documents Provides best answer/ rounded answer to the customer Cite one another’s work!Share partnership success with management

15. Showcases strengths of each group and how collaboration yields an even stronger font of intelligence for the organization. Garners all expertisePrevents duplication of effortsYields time & cost saving

16. RI KIQ’s  Laws  Regulation  Guidance  Policy  Approval Precedent  Basis of Approval (FDA Review/ EPARs)  Regulatory hurdles  Health Authority + or – opinion  FDA Advisory Committee Meetings  Safety Information  Withdraws  Recalls  DHCPLs CI KIQ’s  Market forces  Marketing plan  Market research  Competitor speed to market  Probability of success  Sales force alignment  Pricing and reimbursement  More… Case Study #1 KIT = Regulatory Precedence of a Tx and Indication

17. REGULATORY RESEARCH Question: Can you identify a regulatory precedence for approval of a stem cell therapy for a congestive heart failure (CHF) indication? SUMMARY A broad search was first conducted of the FDA website and IDRAC databases to see if any stem cell therapy was approved for the treatment of CHF. No stem cell therapies have been approved to-date for the treatment of CHF. The next strategy was to review all treatments approved for CHF and to understand the requirements for approval, the study endpoints employed in the pivotal trials, and the analyses used. Though these were not stem cell therapies, the strategy may prove valuable to discuss with a regulatory agency when precedence has not been established. Following review of all approved treatments for CHF, a review of clinicaltrials.gov was conducted in efforts to obtain information about potential competitive stem cell therapies in development and their regulatory strategies for obtaining approved for a CHF indication. The search was stratified by type of stem cell such that one search was conducted for embryonic stem cells, another one for neural stem cells, another one for mesenchymal, another for autogenic, and a last for allogeneic. A total of 83 study results were obtained. The breakdown of these is as follows:….. Search Timeframe: 2006-2001 for relevancy Sources: 1. FDA website 2. IDRAC website 3. Google 4. Clinical trials.gov References/Links: - see table below Report prepared by: J. Doe, 7/20/2011 KIQ The RI Process Search Gather Analysis Summarize for action Case Study #1: KIT: Regulatory Precedence in CHF

18. RI KIQ’s  Laws  Regulation  Guidance  Policy  Approval Precedent  Basis of Approval (FDA Review/ EPARs)  Regulatory hurdles  Health Authority + or – opinion  FDA ADCOMS  Safety Information  Withdraws  Recalls  DHCPLs CI KIQ’s  Market forces  Marketing plan  Market research  Competitor speed to market  Probability of success  Sales force alignment  Pricing and reimbursement  More… Case Study #2 KIT = Policy Related – PDUFA V Reauthorization

19. REGULATORY RESEARCH Question: Can you identify the impact PDUFA V will have on our company? Answer: PDUFA V is intended to increase first-time approval cycles. Proposals within PDUFA V reauthorization will increase the review cycle by 2 months for all NMEs and may add an additional 3 months for major amendments to applications  Participate in trade organization working groups to exchange information and negotiate proposal  Assemble steering team and conduct interviews with colleagues for their perspective on policy changes  Present and  Benchmark internal and industry standards Summary of Other Pertinent PDUFA V Proposals… References/Links: - see table below Report prepared by: J. Doe, 7/20/2011 KIQ The RI Process Research Collaborate Analysis Summarize for action Case Study #2: KIT: PDUFA V Reauthorization

21. Team: Clinical: What are the clinical studies and results? Commercial: What are the marketing plans, price points, reimbursement schemes, and other commercial aspects? Medical Affairs: What are Healthcare Providers saying? Will a positive approval decision change Physicians’ practice? Regulatory Affairs: What are the regulatory hurdles and potential regulatory outcomes? Legal: Any impact on IP, exclusivity, or other legal aspects related to our business?

22. Regulatory Hurdles and Opportunities, e.g., priority review, pediatrics, etc. Relevant Regulations and Guidances Advisory Committee Meeting(s) Approval Letter – Postmarketing Requirements and Commitments Label Disclosable Info from Health Authorities, e.g., FDA Approval Packages and European Public Assessment Reports (EPARs) Regulatory

23. 23 Contact Information Amy Grant amy.grant@viropharma.com Penny Levin plevin@cephalon.com Danielle Ziernicki dziernicki@its.jnj.com