1. Continuous Improvement & Real World Evidence: A Public Payer’s Perspective Suzanne McGurn, Assistant Deputy Minister and Executive Officer Ontario Public Drug Programs Tuesday April 12, 2016 CADTH Symposium 2016

2. Approaching a Crossroad 2 Program growth expected to continue at 3-5% annually – can’t expect to be able to afford everything. Over 4,300 drug products listed on the Ontario Drug Benefit (ODB) Formulary with many more reimbursed under the ODB’s Exceptional Access Program. o Approximately 1/3 of Formulary and other listings are supported by Product Listing Agreements that predate the pan-Canadian Pharmaceutical Alliance, with many coming up for renegotiation. o Formulary listing or funding status of several products have never been reassessed, despite rapidly evolving treatment landscapes in some therapeutic areas, such as Cancer and Hepatitis C. Where do we go from here?

3. Important Questions to be Addressed 3 Is the first to market, first to door, first to cue approach serving everyone’s needs well? How do we continue to manage the volume of new products coming to market in Canada? How do we approach reassessing older products to ensure continued value in evolving treatment landscapes? 1 2 3

4. How Have Our Needs from HTA Producers and Evaluators Changed? 4 HTA Producers Cost-neutral pricing with a “positive” HTA recommendation not enough to secure market access; public plans shifting focus to long-term sustainability, feasibility of implementation, evaluation vs alternatives. “Strong” evidence is not enough; thoughtful trial development imperative to inform decision making (trial duration, surrogate vs. clinically relevant outcome measurement etc). Consistent pan-Canadian market access approach and streamlined interactions through pCPA office to improve consistency and transparency. HTA Evaluators Current volume of 20+ active negotiations unsustainable; need for approach that will help to “sort” products. Guidance from HTA Evaluators on “sorting”? Need for public plans to re-negotiate or negotiate on products (old drugs or otherwise) that are not “new”. Guidance from HTA Evaluators on how to best approach?

5. How Have HTA Recommendations Changed? Addition of Do Not List at Submitted Price is allowing for careful consideration of pricing and affordability issues for public drug plans at the pCPA level, while still acknowledging a drug’s clinical benefit. List with Clinical Criteria and/or Conditions is providing guidance to public drug plans in determining who will benefit most from new therapies and under which conditions they should be reimbursed to maximize affordability and positive outcomes for patients. 5 FINAL RECOMMENDATION (pERC, CDEC) LIST LIST WITH CLINICAL CRITERIA AND/OR CONDITIONS DO NOT LIST AT SUBMITTED PRICE DO NOT LIST CLEAR RECOMMENDATIONSCLINICAL/COST EFFECTIVENESS UNCERTAIN

6. Quality Improvement & Evidence Evaluation Determine & Validate Problem Outcomes & Objectives Develop, Analyze & Evaluate Solutions Decision Implementation Planning Implementation Monitoring and Evaluation Review and Assess 6 POLICY DEVELOPMENT CYCLE Increased need to focus our efforts on continuous quality improvement and assessment of product performance against comparators to support continued listing of both new and old products. This focus presents an opportunity to: 1)Re-evaluate evidence through HTA for both products and classes. 2) Extend research efforts beyond traditional Phase IV post-market clinical studies with a focus on Real World Evidence.

7. HTA for New and Old Drug Products 7 FINAL RECOMMENDATION (pERC, CDEC) LIST LIST WITH CRITERIA/ CONDITIONS DO NOT LIST AT SUBMITTED PRICE DO NOT LIST CLEAR RECOMMENDATIONS CLINICAL/COST EFFECTIVENESS UNCERTAIN PAN-CANADIAN PHARMACEUTICAL ALLIANCE NEGOTIATIONS PLA Terms INDIVIDUAL JURISDICTIONS CLINICAL/COST EFFECTIVENESS AS DETERMINED THROUGH HTA RECOMMENDATION NEW DRUGS: SUBMISSION TO CADTH OLD DRUGS OLD DRUGS (RFA?)

8. Challenges with Real World Evidence for Payers Real world evidence does not replace need for appropriate, well-designed clinical trials with meaningful outcomes. Data quality and access o Access to high-quality data varies across Canadian jurisdictions and by indication. What is the appropriate level of evidence to support decision making or maintain funding arrangements? o Going beyond anecdotal evidence o What are meaningful outcomes in post-market space? Difficulties with delisting or ceasing funding of drug where evidence is inconclusive or poor. o Source of potential contentious issues. 8

9. Where do we go from here? Collaboration, Collaboration, Collaboration o Widespread coordination of efforts, including on an international scale, is ideal and would support evidence generation in specific areas like expensive drugs for rare diseases Incorporating real world evidence more formally into the R&D continuum. o Opportunity for manufacturers, payers, regulators and patient groups to collaborate on appropriate protocol and important questions that need answers. Need for streamlined approach to when and how real world evidence is considered in decision-making process. o Importance of accountability for data collection, reporting and analysis and appropriate timelines. o Incorporation in outcome-based funding models (patient or disease level), risk-sharing agreements etc. 9