The Patient Reported Outcomes, Burdens, and Experiences (PROBE) Study Phase 1 Methodology and Feasibility

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1 The Patient Reported Outcomes, Burdens, and Experiences (PROBE) Study Phase 1 Methodology and Feasibility HTAi 2016 Annual Meeting 13 May 2016 Tokyo, Japan Enhancing the Direct Patient Voice in Health care Decision Making

2 What is Hemophilia? Incidence 1 in 10,000 (X-linked recessive) Deficiency of clotting factor protein Blood does not clot properly Severe patients (<1% clotting factor) at most risk Clinical manifestations Internal bleeding into joints, soft tissues Significant morbidity and mortality SOA treatment (There is no cure, at least yet) Prophylaxis (factor concentrate replacement) Integrated disease management

3 Answering Unanswered Questions World Federation of Hemophilia (WFH) Global Survey shows: DX: How many PWH are diagnosed (prevalence vs expected incidence) TX: How much treatment is available (per capita or for a typical PWH) Mortality: If PWH are surviving into adulthood (ratio children : adults) What we don t understand well is how the typical PWH is fairing in their daily life (Quality of life) How are various dimensions of living impacted? What interventions influence them? The goal of treatment is not simply to add years to a PWH s life, but to add life to their years!

4 Increasing the Value of the Patient Voice Moving advocacy arguments beyond emotion and anecdote to evidence-based arguments grounded in real-world patient outcomes Providing a validated foundation for decision making in Health Technology Assessments and comparative effectiveness research Raising awareness of the impact of living with hemophilia, treatment and effective prevention from the patient perspective Patients have a unique perspective and consider issues differently than regulators, manufacturers, scientists, clinicians and payers.

5 Gather & Interpret Patient-Centered Experiential Data Evidence-based arguments grounded in real-world patient experiences

6 Multi-phase Project Phase 1 ( ) Feasibility assessment Develop / Test understanding of questionnaire content and clarity Assess methodology and in-country implementation feasibility Establish core analytics Phase 2 (2016) Reproducibility assessment Pilot web platform Validate reproducibility (individual and population level) Establish analytic controls / population comparators Phase 3 (2017-beyond) Global Implementation

7 Deliverables A self-reported outcomes data framework with metrics to: Perform reliable assessments Within a country or between countries over time To the general population and other diseases Easy to administer, low resource burden, global Usable by patient organizations worldwide (multi-lingual) Both paper (Phase 1) and web-based (Phase 2) Designed to complement and enhance the utility of national patient registries and the WFH Global Survey

8 Participating Countries Phase 1 Feasibility Assessment Argentina (Cordoba Chapter), Australia, Brazil, Canada, France, Germany, Hungary, Ireland, Italy, Japan, Mexico, Netherlands, New Zealand, Spain, United Kingdom, United States, and Venezuela

9 Results Phase 1 Feasibility Assessment Field work complete 704 responses (117% of objective) 379 FVIII PWH, 86 FIX PWH, 212 not affected by a bleeding disorder 27 other / unknown bleeding disorder Preliminary analysis indicates study methodology is feasible 71.3% met study objectives as to time to completion (0-15 min) Minimal to acceptable staff time required For one cycle of administration: 2-40 hours (median 9 hours)

10 Patient-Centered Outcomes On patients: Directly probe outcomes relevant to patients life Impact on daily living pain, independence, educational attainment, employment, family life, mobility, Current health status (EQ-5D-5L VAS) Demographics On healthy controls : Build a benchmark comparator Applicable questions asked also to non-hemophilia subjects On patients, it also collects explanatory variables: Treatment history disease severity, treatment regimen, bleeds, joint impairment / interventions,

11 The Concept of Comparison Dimension Country Control Mild Mod Severe EQ5D-5L A (some prophylaxis) B (no prophylaxis) Chronic pain A (some prophylaxis) 28% 53% 68% 78% B (no prophylaxis) 30% 56% 70% 86% +56% PROBE Investigators unpublished data not for citation

12 Preliminary Observations Preliminary data analysis also indicates variable correlation between patient responses to PROBE study questions using standardized clinical definitions when compared with responses to questions using patient-developed definitions. The variation highlights the importance of continued research to assess whether clinical trial end-points are the appropriate measures to evaluate real-world patient outcomes.

13 Acknowledgments Investigators Principal Investigator Mark Skinner JD, Institute for Policy Advancement Ltd. (US) Co-Investigators: Randall Curtis MBA, Factor VIII Computing (US) Neil Frick MS, National Hemophilia Foundation (US) Alfonso Iorio MD Ph.D. FRCPC, McMaster University, Department of Clinical Epidemiology and Biostatistics (Canada) Michael Nichol Ph.D., University of Southern California, School of Policy and Planning Development (US) Declan Noone, Irish Haemophilia Society (Ireland) Brian O Mahony, Irish Haemophilia Society, Trinity College Dublin (Ireland) David Page, Canadian Hemophilia Society (Canada) Jeff Stonebraker Ph.D., North Carolina State University, Poole College of Management (US) Funders PROBE is an independent investigator led research project supported with unrestricted grants from: Baxalta USA Inc. Bayer Healthcare Biogen Novo Nordisk HERO Sobi Administrative support provided by the US National Hemophilia Foundation Collaborating Patient Organizations Fundación de la Hemofilia (Argentina) Cordoba Chapter Hemophilia Foundation Australia Federaçao Brasileira de Hemofilia (Brazil) Canadian Hemophilia Society Association Française des Hémophiles (France) Deutsche Hämophiliegesellschaft (Germany) Magyar Hemofilia Egyesulet (Hungary), Irish Haemophilia Society Federazione delle Associazioni Emofilici (Italy) National Hemophilia Network of Japan Federación de Hemofilia de la República Mexicana (Mexico) Nederlandse Vereniging van Hemofilie- Patiënten (The Netherlands) Haemophilia Foundation of New Zealand, Federación Española de Hemofilia (Spain The Haemophilia Society (United Kingdom) National Hemophilia Foundation (US) Asociación Venezolana para la Hemofilia (Venezuela)

14 For More Information If you have questions or would like more information about PROBE you may directly contact the PROBE investigator team: Mark W. Skinner, Principal Investigator Phone: Web: