The Vision For Real World Data – Harnessing the Opportunities for the UK Dr Rick Lones BM FFPM MRCGP

1. The Vision For Real World Data – Harnessing the Opportunities for the UK Dr Rick Lones BM FFPM MRCGP Executive Medical Director, UK and Ireland, Bristol-Myers Squibb and Co-Chair, ABPI Real World Data Campaign Team 13th February 2012

2. Objectives • What do we mean by Real World Data?                 • Where does RWD fit in the new NHS?  • Importance of RWD to Industry

3. Page 2 The UK context “RCTs, long regarded at the ‘gold standard' of evidence, have been put on an undeserved pedestal. Their appearance at the top of ‘hierarchies’ of evidence is inappropriate; and hierarchies, themselves, are illusory tools for assessing evidence. They should be replaced by a diversity of approaches that involve analysing the totality of the evidence-base.” Prof. Sir Michael Rawlins Harverian Oration to Royal College of Practitioners, 2008

4. Page 3 ABPI strategic vision • ABPI committed to supporting and driving the UK to be a centre of excellence in RWD • RWD Campaign team • White Paper Sept 2011 • Practical Guide May 2011 • Training and Master Classes

5. A useful definition “Data collected outside the controlled constraints of conventional randomized clinical trials to evaluate what is happening in normal clinical practice”

6. Real world data complements and augments RCT data Complexity and co-morbidities . . . . . . Post approval ‘market’ population . . . . . . . . . . . . . . . . . . . .. . . . . . . . . . . . . . . .. . . . . Pre-market population . . . . . . Disease specificity

7. RCT data vs. RW data

8. Page 7 What do you measure with RWD? • Outcomes • Clinical • Patient reported • Resource use • NHS • Patient • Societal • Treatment pathways • Service models • Patient preference, experience, compliance

9. Page 8 Existing electronic health records New datasets Prospectively Retrospectively Collected using a variety of methodologies Non interventional research Audits Service evaluation Surveys How do you get Real World Data?

10. Page 9 The UK regulatory framework

11. Page 10 The UK NHS environment • Decision makers at national and local level increasingly require broader and more sophisticated evidence on which to base informed choices • White Paper: Equity and Excellence: liberating the NHS (July 2010) • Focus on quality and outcomes and NOT targets • Patient perspective is increasingly important • Patient experience and satisfaction • PROMs • Introduction of Value–Based Pricing (2014) – need to be underpinned by robust evidence of value

12. Page 11 Why does the NHS need RWD?

13. Importance of RWD to the pharmaceutical industry Gg gg The UK share of clinical trial activity is declining… …could a growth in RWD research fill the gap?

14. Supporting Industry Objectives • Demonstrating Value • Resource Use: Individual Episodes of Care and Total NHS Resources • Outcomes: Clinical outcomes, PROs • Service Delivery: Patient or Clinical Satisfaction, Capacity, Patient Journeys Ggg Ggg Especially important if RCT data collected outside the UK

15. Supporting Industry Objectives • Getting HTA Ready • Ensure a robust economic and budget impact argument • Avoids reliance on RCT data or ‘expert’ opinion • Option to collect in specific UK countries (AWMSG, SMC) Ggg ggg Ggg ggg

16. Supporting Industry Objectives • Capturing Early Clinical Experience • Suitable alternative to Phase IV study (esp. high costs diseases/orphan drugs) • Local requirements for new medicines • Evaluating Joint Working • ABPI Framework advises that outcomes should be measured • Baselines need to be established and tracked Ggg ggg Ggg ggg

17. Supporting Industry Objectives • Informing Internal Decision Making • Clarity on market segments and size of market • Competitor products in practice • Documenting Product Safety • May be regulatory requirement for approval (RMP, PSUR, PAES) • Support for labelling changes Ggg ggg Ggg ggg

18. Alignment with Clinical Development Plan Discovery Pre-Clinical Phase 1 Phase 2 Phase 3 LCM • RCTs focus on EFFICACY, SAFETY and QUALITY; they are SLOW and cost £MILLIONS • RW Studies focus on EFFECTIVENESS, a more realistic picture of what can be achieved in normal clinical practice; they are FASTER and typically cost £THOUSANDS

19. Product Licensing: From “One-Off” ……… Level of understanding of benefit-risk Withdrawal, Backlash Warning, Dear Dr MA Pharmacovigilance Drug Development Phase Time

20. Product Licensing: ……….to “Live Licence”? Level of understanding of benefit-risk Amended MA/SmpC Amended MA/SmpC Outcomes Research Earlier conditional MA MA Pharmacovigilance, Post Marketing Studies Drug Development Phase Time

21. The UK as a Global Player in RWD • The UK has a significant and disproportionate influence on other markets • Impact of NICE globally • Unique “cradle to grave” healthcare system • Wealth of electronic databases developed over last 20 years • Streamlining of regulatory & governance frameworks ongoing • Strong links with academia

22. The Future of RWD in the UK • The UK could become a World Class Leader in RWD, but we must: • Strengthen the case for the UK to develop its capabilities • Encourage initiatives to overcome existing challenges The Introduction of Value Based Pricing offers a significant opportunity for industry