1. The Role of Patients in Defining “value” and Engaging in Drug Development from Bench to Bedside 1 CADTH Conference Concurrent Session A5 April 13, 2015 Saskatoon, SK

2. Session Overview Canadian HTA agencies are accepting formal patient input in their review process. The need for patient involvement in the design of effective value measures throughout the drug development cycle to support stronger reimbursement and policy decisions is increasing. – What key questions should be asked to measure the impact of a medication on the life of the patient? – How should the information required to answer these questions be collected? – How should this information used to make decisions about funding 2

3. The Panel Lineup Deborah Marshall - Associate Professor and Canada Research Chair, Health Services and Systems Research, University of Calgary Louise Binder - Cofounder of the Canadian Treatment Action Council and Project Manager for Carcinoid NeuroEndocrine Tumour Society (CNETS) Pauline McNulty - Vice President, Global Market Access and Commercial Strategy Operations at Janssen Pharmaceutical Companies of Johnson & Johnson. Ken Bond - Director, Strategic Initiatives CADTH Laura Faye - Manager (Acting), JRMS Department, Acute and Ambulatory Care Information Services at the Canadian Institute for Health Information. 3

4. Defining Value from the Patient’s Perspective 4 Informing Relevant Value Measurement Deborah Marshall, PhD University of Calgary CADTH Conference April 13, 2015

5. Outline 1)Alternative Approaches to Patient Engagement 2)Redesigning Drug Development to be Patient-Centered 3)Introducing Patient Engagement Researchers (PERs) as an approach to relevant value mesurement 5

6. Methods of Engagement Across International Association for Public Participation (IAP2) Spectrum 6 - Kovacs Burns et al. BMC Health Serv Res 2014, 14:175

7. Theme Definition of Patient Engagement Citizen engagement, consumer engagement, meaningful patient involvement, participation, patient and public engagement, public involvement, patient centred care and patient involvement Stakeholder Roles and Expectations Lack of consensus and understanding about terminology, goals and expectations and roles and responsibilities as barriers to meaningful and successful patient engagement Meaningful and Appropriate Engagement Desire to ensure patient perspectives to design and improve health services, but not easy (25% want involvement in healthcare decision making, but <10% want involvement in funding decisions) Models of Engagement 19 different models of engagement across different levels of involvement (IAP2) Benefits/Barriers to Patient Engagement PE generally considered beneficial, but lack of research on this and which methods most effective (Cochrane review ) Evaluation of Patient Engagement Variety of toolkits and guides, but a lack of consensus on best approach to evaluate Engagement Resourcing Important to ensure adequate resourcing for PE including time, resources and capacity 7 Scoping Review: PE in Healthcare Decisions N=74 published and N=193 unpublished articles/items - Kovacs Burns et al. BMC Health Serv Res 2014, 14:175

8. Multidimensional Framework for Patient Engagement in Health and Health Care 8 - Carman KL et al. Health Affairs 2013; 32: 223–31

9. CADTH Patient Input Process Review, April 2013 9 - https://www.cadth.ca/sites/default/files/pdf/2012_SECOR_Patient-Input-Review_e.pdf

10. Submission Length – 8 pages Section 1 – General Information Section 2 – Condition and Current Therapy Related Information Section 3 – Related Information about the Drug Being Reviewed Section 4 – Additional Information 10 - https://www.cadth.ca/sites/default/files/pcodr/pCODR%27s%20Drug%20Review %20Process/pcodr-patient-engagement-guide.pdfhttps://www.cadth.ca/sites/default/files/pcodr/pCODR%27s%20Drug%20Review A guide to completing the pCODR Patient Advocacy Group Input on a Drug Review Template

11. 11 “Patient Centered Outcomes” Engage Patients to Define the Disease, Define Relevant Measures and Identify the Patients for Treatment 2000 Patient Reported Outcomes (PRO) 2009 Clinician Reported Outcomes (ClinRO) Observer Reported Outcomes (ObsRO) 2011, FDA Workshop Clinician Outcome Assessments (COAs) Treatment benefit demonstrated by positive impact on survival or on how patients feel or function in daily life

12. Why Patient Engagement? Who is the Expert: patients and families with direct experience What is Right: morally, patients and families are the ones who affected directly What is Relevant: measure what is meaningful What Makes a Difference: better self-care, improve quality of care, better compliance, and better outcomes, how patients feel and function predicts use of health resources Patient Values ≠ System Values

13. Institute of Medicine: 10 Rules for Redesigning Health Care 1.Care based on continuous healing relationships  Care whenever it’s needed, not just face-to-face 2.Customization based on patient needs and values 3.The patient as the source of control 4.Shared knowledge and free flow of information 5.Evidence-based decision making - Institute of Medicine. Crossing the Quality Chasm: A New Health System for the 21st Century, 2001.

14. Proposition: Towards the Science of Effective Patient Engagement Include the patient perspective in each stage of the lifecycle… – from clinical development, regulatory review, HTA evaluation, funding decisions and real world evidence; ‘Patient Reported Outcomes’ – don’t always reflect issues that are most important to patients – need to be informed and developed with patients – and then tested and validated as outcome measures; Embed meaningful and relevant patient measures in the value equation – Weighting of evidence from patients. 14

15. Patient and community engagement researchers (PACERs) are people with various health conditions, trained to design and conduct health research, using specific adapted methods of qualitative inquiry. PACER graduates work in collaboration with health professionals and researchers to: Patient and Community Engagement Research - Marlett N, Shklarov S, Marshall DA, et al. Building New Relationships in Research: A Model of Patient Engagement Research. Qual Life Res 2014; Doi: 10.1007/s11136-014-0845-y

16. The simple answer is that patients are fully engaged in: Choosing research questions important to patients and their families and communities. Making decisions about how to collect and analyze information. Making decisions on how to communicate findings to other patients, professionals and the public. Rigorous training involves mastering specific adapted methods of qualitative research: focus groups, field observation, questionnaires, and narrative interviewing. A year-long training program = 120 hours in-class instruction plus an internship. How does Patient Engagement Research differ from more traditional methods?

17. The PACER Research Method A Collaborative Framework for Engaging Patients in Research - N Marlett and C Emes. Grey Matters. A Guide to Collaborative Research with Seniors. University of Calgary Press, 2010.

18. “ Part of the Team”: Building New Patient Roles and Relationships in Health Research and Planning 18 - Marlett N, Shklarov S, Marshall DA, et al. Building New Relationships in Research: A Model of Patient Engagement Research. Qual Life Res 2014; Doi: 10.1007/s11136-014-0845-y Model: Co-creation of PER role Results: 3 major areas of impact: 1) increased capacity of patients to engage in healthcare research and planning, 2) effective uptake of new patient roles in health care planning – impacting attitudes and practices, and 3) introduction and acceptance of new, collaborative roles for patients in research. Interpretation: Process of merging two distinct roles of patient and researcher unleashes a force for fundamental cultural change, and a way to embed and measure patient value.

19. 19 Thank you ! damarsha@ucalgary.ca 403 210 6377