Transcript Louise Binder

The Role of Patients in Defining
“value” and Engaging in Drug
Development from Bench to
Bedside
CADTH Conference
Concurrent Session A5
April 13, 2015
Saskatoon, SK
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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
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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
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Information.
Defining Value from the
Patient’s Perspective
Informing Relevant Value Measurement
Deborah Marshall, PhD
University of Calgary
CADTH Conference April 13, 2015
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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
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Methods of Engagement Across
International Association for Public
Participation (IAP2) Spectrum
- Kovacs Burns et al. BMC Health Serv Res 2014, 14:175
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Scoping Review: PE in Healthcare Decisions
N=74 published and N=193 unpublished articles/items
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
- Kovacs Burns et al. BMC Health Serv Res 2014, 14:175
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Multidimensional Framework for Patient
Engagement in Health and Health Care
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- Carman KL et al. Health Affairs 2013; 32: 223–31
CADTH Patient Input Process
Review, April 2013
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- https://www.cadth.ca/sites/default/files/pdf/2012_SECOR_Patient-Input-Review_e.pdf
A guide to completing the pCODR
Patient Advocacy Group Input on
a Drug Review Template
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
- https://www.cadth.ca/sites/default/files/pcodr/pCODR%27s%20Drug%20Review
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%20Process/pcodr-patient-engagement-guide.pdf
“Patient Centered Outcomes”
Engage Patients to Define the Disease,
Define Relevant Measures and Identify
the Patients for Treatment
Basic
Research /
Discovery
Translational
Research
Investigational
New Drug
Treatment benefit
demonstrated by
positive impact on
survival or on how
patients feel or
function in daily life
Clinical
Research
Phase I, II, II
Post
Marketing
Surveillance
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2011, FDA Workshop
Clinician Outcome Assessments (COAs)
2000
Patient Reported
Outcomes (PRO)
2009
Clinician Reported Outcomes (ClinRO)
Observer Reported Outcomes (ObsRO)
Patient Values ≠ System Values
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
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.
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.
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Patient and Community
Engagement Research
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:
Formulate
research agendas
Include patient
perspectives in
grants and
proposals
Conduct
research
with
patients
Share research
with patients
and
communities
- 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
How does Patient Engagement Research differ
from more traditional methods?
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.
The PACER Research Method
A Collaborative Framework for Engaging Patients in
Research
Collect
Set
the direction
of the study
data using: Focus
groups ; Narrative
interviews;
Observation;
Questionnaires
Reflect
on the findings
- N Marlett and C Emes. Grey Matters. A Guide to Collaborative Research with Seniors.
University of Calgary Press, 2010.
“Part of the Team”: Building New Patient Roles and
Relationships in Health Research and Planning
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.
- Marlett N, Shklarov S, Marshall DA, et al. Building New Relationships in Research: A Model
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of Patient Engagement Research. Qual Life Res 2014; Doi: 10.1007/s11136-014-0845-y
Thank you !
[email protected]
403 210 6377
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