Gwendolyn Ryals, Look at Me Artwork from The Creative Center Janey Shin, Director, Real World Evidence Government Affairs and Market Access CADTH Symposium April 2016 Adaptive Pathways: A New Option for Canada? A Perspective from Industry

2 International Landscape: Keeping pace with scientific innovation while addressing health system and societal needs NEWDIGs FDA (Sentinel, MDEpiNet, UDI) EMA (MAPP) IMI (ADAPT SMART, GetReal) Timely access to new medicines and technology for patients Key opportunity for industry to engage with regulators, HTAs, payers, patients, HCPs Increasing use of RWE to better understand benefit and risk MARS EXCITE

Leveraging RWE to Support Adaptive Pathways 3 Sources of Real World Data:  EMRs/EHRs  Registries  Observational Studies  Chart Audits  PROs  Randomized Pragmatic Studies  Administrative Claims Data Sources of Real World Data:  EMRs/EHRs  Registries  Observational Studies  Chart Audits  PROs  Randomized Pragmatic Studies  Administrative Claims Data

Managed Entry Agreements: Managing uncertainty with evidence not just with dollars Adaptive Pathways provides a mechanism for Access with Evidence schemes (such as, Coverage with Evidence Development and Performance-based Risk Sharing) To build successful schemes we must acknowledge:  Innovative products and related services delivering important and cost-effective outcomes  Different approaches to different kinds of drugs (e.g. oncology, first in class, drugs for rare diseases, biologics)  Generating meaningful data that can inform the healthcare system  Driving appropriate prescribing, utilization and compliance 4

Challenges  Assessment of Value  Acceptability of evidence outside of RCTs  Managing uncertainty  Generating and Accessing RWD/RWE  Allocation of resources, cost, and timely development of registries  Ability to link databases; inter-provincial data sharing  Timely access to robust secondary data (EMRs/EHRs)  Implementation and Resources  Innovative Product Listing Agreements  Infrastructure and Monitoring of outcomes 5 How will Adaptive Pathways be successful within the current Canadian context of various legislative frameworks: PMPRB, Health Canada, HTA, pCPA, formulary decisions by payers?

Opportunities and Benefits  Early access for patients to novel medicines  Ability for patients to engage in adaptive process  Engagement and robust discussions with all stakeholders  Building trust: Collaboration with industry, payers, HTA, regulators, system leaders, health care providers, patients, data custodians  Public/private partnerships  Improve understanding of outcomes in real-world settings  Advanced analytics and methodology  Increase efficiency of knowledge generation

Taking and sharing risk  Current regulatory, HTA assessment, reimbursement, and pricing environment needs to align with evolving, innovative science: engagement and flexibility from all stakeholders is critical  Generating evidence that focuses on value for the whole health system  Onus on industry to bring solutions to the table – onus on all to resource and execute those solutions