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Patients The Key to Real World Data?

Patients The Key to Real World Data?. Alan G. Wade. Real World Data Sources. What is real world data?. Reflects the average patient in a real-world setting Demographics Sex Age Social status Education Diagnosis Co-morbidities Co-medications Medical system Social impact

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Patients The Key to Real World Data?

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  1. PatientsThe Key to Real World Data? Alan G. Wade

  2. Real World Data Sources

  3. What is real world data? Reflects the average patient in a real-world setting • Demographics • Sex • Age • Social status • Education • Diagnosis • Co-morbidities • Co-medications • Medical system • Social impact • Work impact • Family impact • Impact on Quality of Life

  4. Uses for “real world” data?

  5. Traditional data sources

  6. Hierarchies of evidence • Systematic reviews of randomised controlled trials (RCTs). • RCTs. • Controlled observational studies - cohort and case control studies. • Uncontrolled observational studies - case reports. • Expert opinion • ?KoL

  7. Randomised Controlled Trials • “the gold standard” for demonstrating (or refuting) the benefits of a particular intervention. • Important limitations

  8. Limitations of RCT’s Patients • Age - Effectiveness in younger or older patients • Sex • Severity - effectiveness in mild or severe • Risk factors - effectiveness in patients with risk factors for the condition (eg, smokers) • Co morbidities - Influence of other conditions • Ethnicity - effectiveness in other ethnic groups • Socioeconomic status - effectiveness in disadvantaged patients Treatment • Dose - high dose used in RCTs? • Timing of administration Influence on adherence (compliance) to treatment regimens • Duration of therapy - effectiveness during long-term use • Co medication - adverse interactions • Comparative effectiveness - in comparison with other products used for the same indication Setting • Quality of care • Prescription and monitoring by less specialist (expert) healthcare providers • Care pathway

  9. Effectiveness and efficiency Efficacy Effectiveness Efficiency Does it work in Does it work in Does it contribute to a clinical trials? real life? more efficient use of resources?  COST-EFFECTIVENESS

  10. Post Marketing Surveillance To assess performance of drug in real world setting • Large numbers • Off-label prescribing • Comorbidities • Concomitant medications • Speed of reporting Do we achieve this with formal PMS?

  11. Hierarchies of evidence • Systematic reviews of randomised controlled trials (RCTs). • RCTs. • Controlled observational studies - cohort and case control studies. • Uncontrolled observational studies - case reports. • Expert opinion • ?KoL 6. Patient reports ?

  12. Focus groups • Small numbers • Representative? • Skill of co-ordinator/observers

  13. Who in the world most influences the pharmaceutical industry? • Barack Obama 2. Michael Rawlins

  14. Harveian Oration Hierarchies of evidence should be replaced by accepting—indeed embracing—a diversity of approaches. The Lancet Vol 372 December 20/27, 2008

  15. NICE Patient and public involvement The views of patients, carers and the public matter to NICE. We want to involve them, as well as doctors, nurses, other healthcare professionals and managers in our work. http://www.nice.org.uk – accessed 22 06 09

  16. EMA The assessment of the benefit-risk balance should be based on the available tests and trials, which are designed to determine the efficacy and safety of the product under normal conditions of use (Directive 2001/83), and which are generally performed under ideal conditions. It is important to be explicit about the perspectives of different stakeholders that are taken into account in the assessment of the benefit-risk balance,in particular the perspectives of patients and treating physicians. Considerations about how the treatment is expected to perform under real conditions of use are relevant in the context of pharmacovigilance activities, for example, to take into account any available information on misuse and abuse of medicinal products which may have an impact on the evaluation of their benefits and risks (Directive 2001/83).

  17. Patient groups - the patient? EPF is the umbrella organisation of pan-European patient organisations active in the field of European public health and health advocacy. The European Patients’ Forum (EPF) currently represents 57 patient organisations and an estimated 150 million patients across the 27 Member States throughout Europe.

  18. Patient organisations EU drugs agency working with patient groups bankrolled by big pharma 23.04.10 @ 19:17

  19. Harveian Oration But equally, we have a right and responsibility to look at the interests of other patients who use the healthcare system. What I am critical of, however, is patient organisations that are acting on behalf of pharma companies. I am not alone in complaining about them. The Lancet Vol 372 December 20/27, 2008

  20. “Big Data”

  21. Big Data - definition No single agreed definition but The bottom line: whatever the disagreements over the definition, everybody agrees on one thing: big data is a big deal, and will lead to huge new opportunities in the coming years. http://timoelliott.com/blog/2013/07/7-definitions-of-big-data-you-should-know-about.html Accessed 27 01 14

  22. Big Data – Pharma??? Registries Claims Databases (US) e - Medical Records Also Pharmacy databases Specific hospital databases Specific disease or procedure databases

  23. What do you want from a registry? • Large numbers • Patient level data • Immediacy of data • Longitudinal data • Representative population • Presence of YOUR required data • Linkage of data fields of interest • Confirmation of diagnosis • Standardised measurement • Validated PRO’s

  24. Existing registries Strengths • Large numbers • Immediate access • Longitudinal data • Prescribing data Weaknesses • Inherent biases • Representative population • Diagnostic drift • Patient level data • Surrogate outcomes • Completeness of data • Family social and work history To effectively use any registry it is important to understand how it has been developed and its strengths and weaknesses

  25. Patient Reported Outcomes Why & How? • Some treatment effects only known to patient • Pts provide unique perspective on treatment • Provide information on QoL, work, social and family • Formal assessment may be more reliable than informal interview

  26. Addressing registry weaknesses What the Patient Knows What the Patient Shares Ask the patient – but how? What the physician understands What the Physician records Big Data

  27. Real World Data Sources

  28. Conclusion Collect data directly from patients Patient Reported Outcomes PRO’s

  29. Patient Reported Outcomes - Definition • “any aspect of patients health status that comes directly from patient” - FDA • “insight into way patients perceive their health & impact treatments or adjustments to lifestyle have on their quality of life” –DH

  30. FDA Guideline report Dec 09

  31. Patient Reported Outcomes – concerns • Pt recording versus doctor • Pt understanding of question/form • Validity of question (in that format, pt popn) • Reliability of question • Ability of question to detect change • For licensing – need set as per RCT

  32. Definition of “Real Data from Real Patients” • Collecting data from patients receiving “routine care” BUT • Not affecting their prescribers/ healthcare professionals behaviour

  33. Methodology PROBE Patient Reported Outcome Based Evaluation

  34. Process set-up • Define question - protocol • Define patient group of interest • Determine location of group e.g. Specialist unit, community setting, geography • Review options for accessing patient group • Orphan indications • Design questionnaires and reports • development and testing • Structure customised database

  35. Structured patient registries • Bespoke – ask required questions • Innovative • Prospective • Hosted on Patients Direct site • Interactive • Global Coverage

  36. Problems • Is on-line collection satisfactory? • Will patients cooperate • How do you recruit? • Methodology • Achieving a representative population

  37. www.InternetWorldStats.com 2009

  38. On-line reporting • Age? • Education? • Social class? • Carer reporting? • AlzDis • children

  39. Age and Social Media

  40. Conclusion Generally require alternative data collection routes Nurse manned telephone

  41. Why do people participate? ...the benefits and attraction to each individual will differ but we believe the main reasons are : • 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

  42. Will patients cooperate? 7. REPORT CONCLUSIONS. The above report has shown the public’s enthusiasm towards a system that would let consumers report adverse drug reactions through the Internet. The findings of the survey carried out by us revealed to us this enthusiasm. The report has further shown that health professionals have a positive opinion towards such a system, which works in favour of the overall mission of Patients Direct. Not only has the report articulated the publicity campaign that Patients Direct can carry out to raise its awareness, but also examples of different medicine inserts has been provided with reasoning behind them that could be used by the company to make itself different from its competition. Patients Directs corporate identity is important to begin the process of publicity. IT has been noted that establishing a user friendly website that is easily navigable is fundamentalto setting the pace for a strong brand and image that will appeal to Patient’s Direct customers. EDGE Survey - Market Research, 2008

  43. Recruitment ...tailored to attract patients of interest with a conscious effort to reduce bias and population selection issues Core recruitment methods include : • social networking sites • search engine and website optimisation • public affairs articles and traditional methods of publicity e.g. Newsprint/TV • Healthcare professionals –Doctors, pharmacists • wholesalers, distributors • Clinical trial participants • patient groups Special focus can be given to groups of interest such as children if appropriate and recruitment monitored to ensure sufficient numbers in each cohort.

  44. Recruitment Examples • General – statins • Pharmacy/wholesale distributor • Advertising - Google • Vaccines • Direct at vaccination • Families and children • Appropriate web-sites • Specialist product – home delivery • Invitation with delivery • OTC – Strepsils • General advertising • Pharmacy • Package wrap

  45. Inflammatory Back Pain3rd February 2014

  46. Recruitment • Recruitment Method - Facebook only • First Participant Recruited – 22nd Dec 2013

  47. Facebook Advert Example

  48. Facebook Advert Example

  49. Landing Page

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