1. Dr Alan G Wade alan@patientsdirect.org

2. Patient Centricity The Key to Real World Data Dr Alan G Wade alan@patientsdirect.org 2Eye for Pharma Washington DC 14-15 Oct 2014

3. Real World Data Real World Data comes directly from patients/carers/family members Appropriate because –No filtering by HCPs –More open answers –Can be anonymous -no embarrassment factor –No fear of conflict or confrontation with HCP –Time to reflect and provide a full account of disease or condition 16 3Eye for Pharma Washington DC 14-15 Oct 2014

4. Patient Knowledge Balanced Pharmacovigila nce Compliance Understanding Pathways – Disease, Investigation, Treatment HTA Assessment Adaptive Licensing Market Access Using data from patients www.patientsdirect.org 4Eye for Pharma Washington DC 14-15 Oct 2014

5. Big Data Research (RCTs) Physicians Database entries REGISTRIES Laboratory Results Other Miscellaneous datasets What data does pharma use? 5Eye for Pharma Washington DC 14-15 Oct 2014

6. Existing registries Strengths Large numbers Immediate access Longitudinal data(retrospective) Prescribing data Weaknesses Inherent biases Representative population Diagnostic drift Patient level data Surrogate outcomes Completeness of data Family social and work history Patient attitudes To effectively use any registry it is important to understand how and why it has been developed and its strengths and weaknesses Claims Databases e - Medical Records 6Eye for Pharma Washington DC 14-15 Oct 2014

7. What the Patient Knows What the Patient Shares What the physician understands What the Physician records Big Data What data is in a registry? Ask the patient – but how? 7Eye for Pharma Washington DC 14-15 Oct 2014

8. Collecting patients and data Identify data required Develop On-line and telephone collection Recruit patient population Develop questionnaire Collect one-off or longitudinal data Collate and analyse data Produce report/publication Develop protocol www.patientsdirect.org 8Eye for Pharma Washington DC 14-15 Oct 2014

9. Brief – “Define Impact on Family of a Child Contracting a Flu Like Illness” Methodology Baseline Survey – 946 Families – 3695 Individuals Follow Up every two weeks – 540 “flu like illnesses” Influenza Survey 2012/13 9Eye for Pharma Washington DC 14-15 Oct 2014

10. “ILI” Influenza Like Illness Outcomes In 22% of child ILI an adult had to take time off work (50% for ≥2 days) 30% of adult ILIs were linked to child ILIs (of those adults who had to take time off work 70% were off for > 2 days) Quantified NHS Use Poster prepared for at ISPOR 2014 prior to full publication 38 10Eye for Pharma Washington DC 14-15 Oct 2014

11. Bespoke – ask required questions Prospective Hosted on independent site Interactive Multi-national coverage Structured patient registries 11Eye for Pharma Washington DC 14-15 Oct 2014

12. RetrospectiveStructured prospective Presence of YOUR required data++++ Representative population++ Large numbers+++++ Linkage of data fields of interest?+++ Confirmation of diagnosis++ Standardised measurement++++ Validated outcomes+++ Patient level data++++ Social,economic and attitudinal data-+++ Response to unexpected findings++++ Immediacy of data+++++ Longitudinal data+++ Cost++ Registries 12Eye for Pharma Washington DC 14-15 Oct 2014

13. Daily Mail, Tuesday, March 4, 2014 13Eye for Pharma Washington DC 14-15 Oct 2014

14. Outlet for their feelings and views – might be a threat to their relationship if they report problems to their healthcare professional – we’re neutral Altruistic - Assist in developing new and better treatments Obtain better information and knowledge through participation Feel valued through regular contact/ interaction Desire to make sure the patients voice is heard Why do people participate? 14...the benefits and attraction to each individual will differ but we believe the main reasons are : Eye for Pharma Washington DC 14-15 Oct 2014

15. Is Patient Data Reliable? No ulterior motive Answer what they feel and know Why would they be untruthful? –No or very minimal incentive –usually to charity Accurate? –Depends partially on the skill of the questions Diagnosis –85% of prospective diagnosis is from clinical history 16 15Eye for Pharma Washington DC 14-15 Oct 2014

16. Individualised to project Media/advertising –Electronic advertising - Google, Facebook, Twitter –Traditional advertising –newspapers –Editorials/ Feature articles –Patient Groups, websites –TV, radio Physicians/databases – point of delivery e.g. vaccines Consider –Target population –Demographics –Disease –Cost efficiency Recruitment – The Key www.patientsdirect.org 16Eye for Pharma Washington DC 14-15 Oct 2014

17. Considerations –Age? –Education? –Social class? –Carer reporting? Alz Dis children On-line or alternative reporting? If inappropriate – provide alternative reporting methodology – live telephone interview 17Eye for Pharma Washington DC 14-15 Oct 2014

18. Population Recruitment Targeting – Chronic back pain in younger sufferers Targeting younger sufferers Number of Participants Age 20 Response On-Line Telephone

19. Chronic Back Pain (IBP) 19Eye for Pharma Washington DC 14-15 Oct 2014

20. IBP - source 20Eye for Pharma Washington DC 14-15 Oct 2014

21. Spinal Cord Injury with Spasms – two part survey 21Eye for Pharma Washington DC 14-15 Oct 2014 Incomplete – awaiting prompt 2

22. Summary Patients are a repository of large quantities of data that is: –Relevant to the Real World –Not Recorded –And Valuable It is important we collect and evaluate this data and utilise it as a valuable source of RWD 26 22Eye for Pharma Washington DC 14-15 Oct 2014