1. Patients’ Perspective on HTA and Off-label use David Head MBA Chief Executive RP Fighting Blindness 25th Annual EuroMeeting 4-6 March 2013 RAI, Amsterdam Netherlands

2. Disclaimer The views and opinions expressed in the following PowerPoint slides are those of the individual presenter and should not be attributed to Drug Information Association, Inc. (“DIA”), its directors, officers, employees, volunteers, members, chapters, councils, Special Interest Area Communities or affiliates, or any organization with which the presenter is employed or affiliated. These PowerPoint slides are the intellectual property of the individual presenter and are protected under the copyright laws of the United States of America and other countries. Used by permission. All rights reserved. Drug Information Association, DIA and DIA logo are registered trademarks or trademarks of Drug Information Association Inc. All other trademarks are the property of their respective owners. 2

3. Context: What is RP Fighting Blindness? UK Charity Patient Organisation Research Funder Provider of Patient Information Provider of Patient Support 3 RP Fighting Blindness

4. Context: What is RP? Retinitis pigmentosa – a genetic condition Degeneration of retinal cells Progressive sight loss Currently not preventable or curable Orphan Disease affects 2 – 3m worldwide Off label prospects Devices and drugs in clinical trials 4 Retinitis Pigmentosa

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6. Not very scientific but …. Have you ever heard of Healthcare Technology Assessments? 93% said no Have you heard the term "Off Label Use“ 84% said no 6 Straw Poll

7. Industry Stakeholders 7 Pharma Patients Patient Organisations Regulators

8. Trust in the Doctor Prognosis Family Pain Trust in the Healthcare System Friends Fears Media Patient Decision Making 8

9. Patient Perspective 9 Video embedded here

10. Patient Perspective 10

11. The Obvious Suspects Expediting trials and approvals Access to therapies Safety Risk benefit decisions Real life and quality of life issues 11 Patient Organisation Priorities

12. No patient population perception on HTAs and off-label use Patient Organisation and Experts are a different kettle of fish These are self-selecting groups Work is needed to build trust in pharma and regulators, but patients trust their healthcare professionals Note: My perspective is inevitably tainted 12 Summary

13. PharmaPatientsRegulators Old Stakeholder Map

14. Pharma Patients Regulators We are all people first

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16. Patients’ Perspective on HTA and Off-label use David Head MBA Chief Executive RP Fighting Blindness 25th Annual EuroMeeting 4-6 March 2013 RAI, Amsterdam Netherlands