Concept Paper #3: Risk Assessment of Observational Data Session 1: Good Pharmacovigilance Practices Linda S. Hostelley on behalf of PhRMA

PhRMA Supports FDA Efforts on Risk Management Risk Management is a Continuum Positive Benefit Risk is Key Benefits and Risks Drive RMP Evidence-Based Decisions Systems-Based Approach Impact on Drug Development Collaboration is Essential 2

Session 1: Good Pharmacovigilance Practices Question 1: How can the quality of spontaneously reported case reports be improved? PhRMA Response: PhRMA supports FDA’s efforts to improve the quality of individual spontaneously reported cases Balance must exist between the significance of the clinical event reported and the intrusiveness of follow-up with the treating healthcare professional Simplify reporting Identify best practices Educate all partners to understand the importance of adverse experience reporting and the importance of quality reports Collaboration between industry, FDA, academia, healthcare and patient organizations is essential 3

Session 1: Good Pharmacovigilance Practices Question 2: What are the possible advantages or disadvantages of applying data mining techniques to spontaneous report databases for the purpose of identifying safety signals? PhRMA Response: Data mining techniques may be a useful adjunct to traditional pharmacovigilance methods Data mining techniques provide a systematic, efficient approach to screening large adverse event databases for potential safety signals Limitations of the underlying data and the data mining techniques must be fully appreciated to avoid false positive causality conclusions Research efforts should be targeted to reduce false positives Collaboration between industry and FDA to define best practices and to optimize use of data mining technologies in pharmacovigilance and clinical risk management 4

Session 1: Good Pharmacovigilance Practices Question 3: What are the possible advantages or disadvantages of performing causality assessments at the individual case level? PhRMA Response: Inadequacy of spontaneous report data is problematic With exception of cases with positive re-challenge, little or no advantage in preparing causality assessment on an individual case Lack methodology for reliable and consistent individual causality assessment High likelihood of misinterpretation 5

Session 1: Good Pharmacovigilance Practices Summary PhRMA supports development of best practices for improving the quality of spontaneous reports and for developing the methodologies for application of data mining techniques PhRMA supports education of all partners involved in post- marketing surveillance 6

Session 1: Good Pharmacovigilance Practices Additional Question for the FDA What criteria should be used to determine whether a Pharmacovigilance Plan describing pharmacovigilance efforts above and beyond post-marketing spontaneous reporting is warranted? 7