REAL WORLD DATA GENERATION FOR MARKET ACCESS PURPOSES. Marcin Balcerzak Research Team Lead Farenta Oy

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2 REAL WORLD DATA GENERATION FOR MARKET ACCESS PURPOSES Marcin Balcerzak Research Team Lead Farenta Oy

3 AGENDA Real world evidence in market access landscape: demands & requirements Case studies: Product entry / effectiveness research Mapping market opportunities Challenge and defense of value Farenta Research system setting

4 CLINICAL TRIALS VS. REAL WORLD Clinical trials By nature and regulation conducted in controlled environment Patients recruited according to certain procedures Reality Millions of patients and HCPs reality is not so structured Not every diagnosis is correct Patients has often more than one disease Drugs administrated to strict protocols Not all drugs prescribed to patients are taken according prescription Every investigator knows it. Considering how much money is spend for healthcare acknowledging role of real-world evidence can not be refused

5 1. ISPOR Real World Task Force Report (2006) Using Real World Data For Coverage And Payment Decisions. 2. The vision of real world data Harnessing the opportunities in the UK (2011) DEFINITIONS AND SOURCES data used for decision making that are not collected in conventional randomized controlled trials 1...collected outside the controlled constraints of conventional randomized trials to evaluate what is happening in normal clinical practice 2 RWD is scattered across a system: Observational clinical trails, Pragmatic clinical trials, Health records, Claims databases Patient registers, Patient-reported outcomes, Social media

6 McKinsey PMP practice perspectives on RWD WHERE AND WHEN APPLY RWE? REAL-WORLD DATA HAS BECOME A MAJOR CONTRIBUTOR THROUGHOUT LIFECYCLE Real World Evidence (RWE) creates opportunities along the whole value chain, A comprehensive RWE strategy needs to enhance a cross-functional approach, it has implications for Development and Commercial functions

7 TRANSFERING RWD BETWEEN COUNTRIES? Clinical outcomes Patient reported outcomes (in some cases) Safety data Natural history of disease Prescribing patterns X Utility data for economic modeling? Epidemiology? (depends on country & disease) Treatment pathways X Resource use X Service delivery X Patient experience of service X

8 IS EVIDENCE ENOUGH FOR DECISION MAKING? Relevance: population comparators endpoints timeframe policy Credibility: design data analysis reporting interpretation conflict of interest

9 RWD BENEFITS Estimates of effectiveness Comparison of multiple alternative interventions Estimates of the risk benefit profile Examination of clinical outcomes in a diverse study population Results on a broader range of outcomes Data on resource use Information on how a product is dosed and applied in clinical practice Data in circumstances where there is an urgency to provide reimbursement Interim evidence

10 CASE STUDIES

11 PRODUCT ENTRY Meeting material of the ehealth Stakeholder Group (E02769). Source: Sebastian Gaiser, Johnson&Johnson Not yet cases that RWE granted medicine market authorization However, RWE can help development on: Understanding standard of care Reveal unmet needs Recruit patients Budget impact Manage entry But, not all products are medicines

12 EFFECTIVENESS RESEARCH Case study: pharmaceutical pictograms commercialization Practical effectiveness of pharmaceutical pictograms use: effect on treatment adherence (6 domains: messages conveyed and recalled by pictograms) Setting: pharmacy practice-based research network, Farenta Research Prospective randomized study comparing changes in adherence between standard dispensing practice and standard practice supported with pictograms with permission of

13 EFFECTIVENES RESEARCH Percentage of patients adherent (proper behavior) in each domain Control: standard practice Intervention: pictograms baseline follow-up baseline follow-up omitting doses omitting doses dosing time crusing tabs. dosing time crusing tabs. 0% 100% 0% 100% number of tabs. taking with water number of tabs. taking with water dosing freq. n=117 dosing freq. n=104 with permission of % pts. with 1 adhernece problem % pts. with 1 adhernece problem

14 GAIN / PROTECT MARKET POSITION Gain identify underserved patient populations, identify high-cost areas (risk-based product pricing) identify subpopulations with superior product response show product adventage vs. competitor challenge current standard

15 Brand s market share Reason of discontionuation Reason of discontionuation Reasons of discontinuation Reason of discontionuation AEs adverse events, LoE lack of efficacy SHIFT OR PROTECT MARKET OPPORTUNITIES Real therapeutic pathways Brands used in subsequent lines 100% 80% 60% 40% 20% 0% 1st trial (n=79) 2nd trial (n=80) 1 color = 1 brand 3rd trial (n=41) 4th trial (n=18) 5th trial (n=7) Strengths and disadvantages of leading brands 50% 40% 30% 20% 10% 50% 40% 0% 15,9% Medikinet CR 30% 20% 10% 50% 40% 30% 20% 10% 0% 19,0% 36,4% 15,9% 0,0% 0,0% 0,0% Equasym Retard 13,1% 11,9% 9,5% 2,4% 18,2% 28,6% 4,5% 4,5% 2,3% 2,3% 7,1% 4,8% 2,4% 0,0% 7,1% 7,1% 9,5% 2,4% Concerta

16 CHALLENGE AND DEFENSE OF VALUE Analyses affecting your products may already be under way Challenges to come from any direction Real world evidence may help to defend product value Germany : IQWiG excluded insulin analogues from reimbursement launched real-world studies demonstrated better total cost of care performance of analogues individual payers restored access

17 CHALLENGE AND DEFENSE OF VALUE 1st JAN 2017: change in reimbursement of diabetes medicines other than insulins (they were moved from upper (100%) to lower special reimbursement class (65%) Finnish diabetes type 2 longitudinal study: 6 months follow up results (NOV-DEC 2016 MAY-JUNE 2017): discontinuations and initiations of main classes of non-insulin anti-diabetic medicines discontinuations initiations SGLT-2 incretine mimetics glinides DPP-4 inh. glitazone combinations sulfonylureas metformin

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19 UNIQUE CONCEPT WITH MANY POSSABILITIES What kind of information can be obtained with Farenta Research? R&D HEOR Market Access Medical & Safety Marketing Epidemiology research Using RWD not only RCTs HTA/ BI models Adverse event/ signal detection Patient flow & market understanding Trial simulation & recruitment Improved outcomes research Value demonstration Drug utilization / additional monitoring Launch planning/ trigger programs Unmeet need & burden research HEOR productivity Outcomesbased pricing research Risk management plans Campaign planning and effectiveness Standards of treatment Managed entry PSP and patient services evaluation Services evaluation Portfolio evaluation therapy schemes & pathways Comparative effectives research

20 WHAT IS FARENTA RESEARCH? pharmacy practice-based research network connects pharmacies and their personnel around different research projects engages patients to participate in real-life studies in different therapeutic areas provides research electronic solutions and ensures common standards of data collection

21 BENEFITS FOR PHARMACEUTICAL COMPANIES Evidence reflecting patients perspective outside-in approach Data collected is limited in other sources Studies meet needs of different pharmaceutical industry functions System is universal (e.g. different TAs) Data independence Data generalizability Data control Data focus Adaptability (start, stop, continue, change)

22 SYSTEM FEATURES Used to perform cross-sectional and prospective studies Offering possibility of many interview scenarios Studies designed to meet different stakeholders needs Focus on patient-reported outcomes and economic outcomes Tailored methodologies Standardized, electronic data collection End-to-End service

23 SATISFIED CUSTOMERS - Research Services

24 THANK YOU! Contact details: Marcin Balcerzak Research Team Lead