1. Patient Engagement in Drug Development: Experiences, Good Practices and Lessons Learned Lana Skirboll VP Science Policy Sanofi October 28, 2016, National Health Council 1

2. Some of the (real and potential) uses of PFDD Discovery - Research targets - Unmet need - Disease burden - Treatment burden - Natural history of disease Development - Barriers to participation - Trial design - Clinical endpoints - Delivery system & dosage - Return of results - RWD Submission & Approval - Benefit-risk assessment - Prioritization of COAs - Improved acceptance of novel endpoints - Labeling Post-Approval - RWD for post- market surveillance - Use for payer/provider decision making - REMS/Risk management 2

3. Questions being raised about PFDD in regulatory decision-making ● FDA use of PFDD in regulatory decision making is still evolving. ● It premature to talk about ‘good practices’ when ‘practice’ itself is still emerging. ● The many sources of PFDD innovation 3 FDA Patient Industry GOAL: An agreed upon framework for: the collection of valid and reproducible patient voice data and how it will be used in the review and approval of drugs and devices.

4. The Perpetual Conundrum: Validated PV Data The collection of valid and reproducible patient voice data using tools and methods that are both valid and reproducible ● Is it a reasonable goal that the right tools/methodologies could reach all (or most) the patients, especially in this time of social media and internet connectivity? ● How do we do this without further slowing the process? ● How to get there? We need behavioral scientists who know how to develop valid tools and methodologies that are fit for purpose and that represent human opinion and the components of human decision making. We need to test tools them, refine them and validate them for use by ALL. FDA needs a validation process so we do not have the reinvent the wheel.

5. 5 Burden of disease Burden of treatment Tolerance for risk Expectation of benefit A validated survey tool that reaches 1000’s of patients!!! Querying patients one at a time VS One Example: A Validated Survey Tool?

6. PFDD -- Why bother? ( Motherhood and Apple Pie!) ● Patients benefit from being heard ● Industry can develop better products ● FDA hears from the all the stakeholders ● Payers ensure they are paying for what matters ● Physicians deliver the best treatment option 6

7. 7 Back-up slides

8. External stimuli – Cures and Innovation Bills ● 21 st Century Cures – would: ● define patient experience data ● require FDA to establish process to guide entity wishing to develop patient experience data ● allow FDA to request patient experience data ● require FDA to publish draft guidance on the use of patient experience data, registries, patient beliefs on B-R ● require FDA to hold regular workshops to gain input on methodologies and collection of patient experience data. ● Senate Innovation Bill – would: ● define patient data (as well as patient experience data) ● require FDA to make public a statement on patient experience data and related information submitted as part of a drug/biologic application including whether it was reviewed as part of the application ● Require FDA to publish guidance as above ● PDUFA VI 8

9. External stimuli – PDUFA VI ● PDUFA VI ● Provides resources to increase FDA staff capacity/capabilities within review divisions ● Requires FDA to develop a series of guidance documents: 1.Comprehensive and representative patient/caregiver data collection and taxonomy 2.Methods to identify what impacts are most important to patients 3.Out of list of impacts, what to measure in a clinical trial 4.Methods to incorporate/develop COAs into endpoints robust enough for regulatory decision-making ● Requires FDA to maintain a repository of tools for stakeholders revise relevant MAPPs and SOPPs hold a public meeting to gather input on enhancing patient engagement in clinical trials 9