- ExLPharma holds annual conferences on investigator-initiated trials (IITs) to discuss governance models, planning, operations, and the role of medical science liaisons (MSLs). - Key topics included developing a review committee and charter, strategic planning documents, budgeting, standard operating procedures, and metrics to measure program success. - The role of MSLs in IITs is to provide scientific feedback, study information, and liaise across functions like medical information and clinical development to support investigators. - Prioritizing IITs involves aligning them with product strategies and evaluating proposals based on strategic fit, scientific design, and investigator/site capabilities.

1. Highlights from ExLPharma’s 12th Investigator Initiated Trials September 27-28, 2010 Burlingame, CA

2. A Governance Modelfor Review & Decisions of Investigator- initiated Research

3. Investigator-initiated Studies Unsolicited Non-Clinical and Clinical Investigator - Driven Typically single center Duration up to two years Scientifically driven Support drug and /or funding

4. Building a Committee Charter Scope and Purpose Establish strategic review committee Acquire appropriate membership Define roles and responsibilities Establish meeting logistics Establish operating principles Agree to decisions rights Develop key metrics Measure success

5. Planning: Develop Strategy Global Strategy Document Provides a disease area with consolidated documentation of scientific areas of focus Includes key regional considerations Identifies areas of research interest for investigator initiated studies to enable: An avenue for investigators worldwide to design and conduct their own research Feasibility of new scientific ideas Discovery of emerging trends and signals Transparency and avoid duplication of studies

6. Planning: Budget Planning Generate budget and average cost per study Tie to areas of research strategy Forecasting Management of budget

7. Planning: Operations Identify key stakeholders Internal and external Acquire qualified staff to support programs Establish standard SOPs / guidelines Include timelines Identify training needs Develop standardized tools for investigators Submission /Concept templates

8. Planning: Operations Technology Web portal, IT support Document management Establish Metrics to measure progress Performance Compliance Cycle time

11. Review of research proposal

12. Complete and quality proposal

13. Scientific merit (scientific importance, methodological rigor)

14. Alignment with therapeutic area strategy

15. Uniqueness (should not compete or conflict with company research programs

17. Experienced

18. Sufficient resources to conduct the study

19. Past performance

20. Feasibility of study timelines

22. Setting Expectations What you should expect from potential investigators A well written proposal Ability to conduct and complete study Ability to meet study timelines Submission of complete and timely quarterly progress updates To seek expert statistical help in analyzing the data Ability to submit well written abstract and manuscript

23. Setting Expectations What investigators should expect from you Prompt response to a proposal Thorough scientific review of the proposal Timely decision on acceptance or rejection of proposal Clear rationale regarding rejection Upon study completion, timely and discretionary comments of abstract and manuscript

24. Elements of Success A well structured operating model that focuses on rigorous scientific review of investigator-initiated proposals will ensure: Program Integrity Transparency of Intent Decisions are based on good science

25. Elements of Success Build trust and respect Communication should be clear, complete and timely Establish and agree to expectations Effective documentation and tracking will ensure a compliant and efficient program

26. Bringing MSL Value to Investigator Initiated Trials

27. Roles and Responsibilities of the MSL Feedback Presentations Studies Scientific information

28. Characteristics of the MSL Educational Preparation Experience Competencies

29. Core Competencies of the MSL Technical expertise Communication Relationship building Team work

30. Medical Science Liaisons Work Across Multiple Functions Medical Information Commercial Partners Publications MSL Clinical Development Legal

31. Investigator Initiated Trials (IIT) RESOURCING question: “In relation to other trials in the program, how important is the IIT to the development of drugs?”

34. Pursue combinations as disease appropriate

35. Extend IP protection on new therapeutic options

36. Broad regulatory approval

38. Broadly explore

40. Prioritization of IITs: Trial Priority High Medium Low Resourcing MSL Operations/Contracts Timelines

41. Methodology for the IIT Program Goals/Objectives IIT Resource Allocation Team review and prioritization

42. Evaluation of IIT Proposals Three Criteria for Evaluation Strategy Design Site

43. Strategic Development

44. Measuring MSL Value Balanced Score Card Strategy Map KPI/KPM Key Performance Indicators Key Performance Measures Total Quality Management

45. Balanced Score Card R.Kaplan and D.Norton (1992)

46. Strategy Map R.Kaplan and D.Norton (1996)

47. Key Performance Indicators/Measures Strategy Objectives SMART format KPIs

48. KPI/KPM Some examples are: New investigators acquired Analysis of individuals applying to become investigators Status of existing investigators Investigator attrition Project activation, monitoring, and completion

49. Total Quality Management (TQM) Technology Organization People

50. Conclusion MSLs are a highly educated group of clinicians/researchers and an important part of the development of new drugs. Evaluation of the value MSLs bring to the IIT program should focus on controllable areas and objectives.

51. Still have any questions? For additional information on ExLPharma’s Investigator Initiated Trials Conferences, please visit www.exlpharma.com