Gwendolyn Ryals, Look at Me Artwork from The Creative Center Julia Brown, Vice President Government Affairs and Market Access CADTH Symposium April 2015 RWE is the New Black: A Perspective from Industry

1 What is Real World Evidence (RWE)? Generated using data from usual care setting in a range of non-interventional (observational) studies, including:  Primary data collections  Analyses of secondary data RWE can be generated using so called “pragmatic” randomized trials that are conducted in more usual care settings that employ broad patient inclusion criteria and that investigate effectiveness (rather than efficacy) of different treatments. A working definition of RWE…

2 Growing demand and increasing data availability moving RWE close to tipping point RWE is growing in importance and relevance at accelerated pace Growing demand – Focus on value and transparency Increasing supply – Easier availability of data/analytics  Societal trends (e.g., reform, aging population, emerging middle class) increasing utilization  Markets becoming increasingly demanding as health care systems seek meaningful innovation, value and transparency  Substantial economic / pricing pressure; increased legal, regulatory, and business scrutiny  More data from new data sources, e.g., sensors, self- reported data  Better insights from structured and unstructured data  Governments (e.g., US, UK, Denmark) playing an active role making health data liquid  Engaged patients – more involved in care, sharing and generating data

3 RWE is becoming increasingly important IMS Health

4 Stakeholders responding globally Regulators ▪ Mainly focused on pharmacovigilance ▪ Adaptive licensing concepts and corresponding use of RWE data Payors ▪ Selected payers use RWE for formulary decisions (US) or cost/quality analyses (GER) ▪ Emerging outcomes based/risk-sharing contracts Providers/clinicians ▪ Usage of internal RWE datasets for cost/quality monitoring and physician incentives ▪ Creating registries to generate RWE HTA bodies ▪ Broad variation of use, some systematic (e.g, UK, Italy) others more research focused (e.g., US) Patients ▪ Interest in real-world evidence mixed with questions on data privacy ▪ Starting to “create” real-world data via forums Academia ▪ Broad use of RWE as source of insight to medical research ▪ Partnerships with other stakeholders

5 But there are significant geographic variations RWE used for national market access and general reimburse- ment RWE mainly used for utilization/ safety or formularies (e.g., payer/reg- ional/hos- pital) Sporadic useFrequent use ▪ Limited RWE use for benefit assessments and reimbursement negotiations – RWE has low weight vs. clinical data ▪ Some payors using cost- benefit assessment (e.g., WellPoint) ▪ Major investments in RWE analytics Use of RWE by stakeholders in health system Main application of RWE data France Germany Italy UK US Spain  RWE data used for conditio- ned reimbursement and ma- naged entry agreements (especially for high cost innovative drugs) ▪ RWE mainly used to identify potential post-marketing safety concerns Japan

6 Going forward, RWE use will increase – albeit at different speeds RWE used for national market access and general reimburse- ment RWE mainly used for utilization/ safety or formularies (e.g., payer/reg- ional/hos- pital) Sporadic useFrequent use ▪ Slower - RWE likely to play a somewhat larger role during drug re-evaluations post launch ▪ Faster - Higher number of payers and providers conducting HTA-type assessments (driven by data availability) Use of RWE by stakeholders in health system Main application of RWE data ▪ Faster - RWE expected to play significantly larger role during drug evaluations (3-5 yrs post launch) Faster - RWE likely to be used more frequently at national level for access and during re- evaluations at regional level ▪ Faster - RWE use likely to increase due to increasing data availability ▪ Slower – role of HTA and RWE for market access and reimbur- sement likely to increase slowly Outlook: At what speed will RWE usage increase? France Germany Italy UK US Spain Japan ▪ Slower - opportunity to increase use of RWE via Public Know- ledge Assessment process

7 What do we need to do in Canada to catch up? From… …To Mindset ▪ Systematic and proactive; earlier and patient-centric ▪ Ad hoc and reactive; post- launch and product-centric Capabilities ▪ Broad awareness, collaboration and information sharing ▪ Low awareness, variable capabilities and no framework for incorporation into decision-making Process ▪ Iterative and strategic use of RCT and RWE ▪ Fragmented and serial lacking cohesive approach Data/infra- structure ▪ Greater access, coordination ▪ Limited access and coordination Partnerships ▪ Coordinated and integrated partnerships ▪ One-off collaborations Funding ▪ Limited funding ▪ Pre-planned and coordinated

8 In Summary  Engagement and collaboration from all stakeholders: – Patients, HTA bodies, payers, healthcare professionals, academia, industry…  Framework for RWE generation and incorporation into HTA reviews, public policy and formulary decision-making  Generation of data that balances commercial and public interests  Appropriate allocation of resources and willingness to risk-share  Prioritization of patient outcomes over short-term cost- containment