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Transformation of Patient Involvement in Healthcare: Enablers
Social ConnectivitySocial media & data
sharing, peer reviews, peer pacts, wellness
tracking, games
Data Sensor & monitoring devices, algorithms,
visualization, risk identifiers
Consumer OfferingsHealth information websites & apps, provider ratings, comparison tools
Cost Management More coverage options,
chronic care tools, HTAs, co-pay trade-offs,
wellness programs
Drug ApprovalBurden of proof
requirements, benefit-risk, PROs
Outcomes MetricsValue-based pricing,
standard performance measures, alternative
care delivery
Communication Platforms
Real time physician access, remote visits,
retail clinics
Information SystemsCloud based records, secure collaboration networks, patient-
friendly records
Technology Advancements Driving Information Access
Demands on Access and Reimbursement
The Empowered Patient and Patient Organizations
Is The Patient’s Voice Missing In Drug Development?
• Are we asking the right questions? Of the right people?
• Is there an opportunity to push for more with Health Authorities and other decision makers?
• Where do patients want to have a voice in drug development (e.g., in clinical trial design/ protocols, endpoints, risk benefit assessments)?
• How can patients help address challenges with enrollment and retention in clinical trials?
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Patients Want A Greater Voice In Drug DevelopmentInput on… Requiring…
Deciding what is effective Shaping regulations and our current practice
What evidence is required to show effectiveness
Influencing the existing environment and ongoing collaborative efforts (e.g. PCORI)
Identifying important questions for clinical trials
Education for patients and opening our mindset
Contributing to clinical trial endpoints
Education for patients and opportunities for meaningful dialogue
Regulatory processes, including risk/benefit
New approaches from regulators and industry
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Need for A Greater Patient Voice in Drug Development
“Why can researchers or clinicians alone not provide the patient perspective? Experiences and training determine a person’s worldview, and patient-centeredness is at its heart a question of including the worldview of patients. Because of their training and orientation, researchers and clinicians can be at a disadvantage for representing the patient perspective. Patients—individuals whose worldview about health is centered on the experience of healthcare—may more accurately and comprehensively capture the patient perspective than those whose worldview centers on inquiry about health or health care. Getting the patient view directly from patients also reduces the risk of representing only those on the upside of the power asymmetry inherent in interactions between patients and researchers or patients and clinicians.”
Frank L, Basch E, Selby JV, The PCORI Perspective on Patient-Centered Outcomes Research, JAMA October 15 2014 Volume 312, Number 15
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I´m worriedand concerned
GI symptoms bother me!
I can not bendover or exercise
My whole lifeIs affected
Heartburndisturbs my sleep
I can not eat anddrink whatever
I like
PROs Represent The Voice of The Patient
The purpose of a Patient Reported Outcome measure is to capture the patient’s experience
• “A PRO is a measurement of any aspect of a patient’s health status that comes directly from the patient (i.e. without the interpretation of the patient’s responses by a physician or anyone else).”
• A PRO instrument is a means to systematically capture data from patients (from questionnaires, diaries, interviews)
• In clinical trials, a PRO instrument can be used to determine whether or not a drug has been shown to provide benefit to patients in terms of how they feel and/ or function
FDA PRO guidance: http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf
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Uses of PROs• Most common use of PROs is in clinical research to measure
symptoms, functional status, and health-related quality of life– In clinical trials, a PRO instrument can be used to measure the
effect of a treatment intervention on symptoms and/ or functional status
– Findings can be used to support a claim in product labeling
• With the increasing focus on patient-centeredness there is a growing interest in using PROs in clinical practice and assessment of quality of care– Increasing integration into registries and EHRs– Assessing patient experiences with services and care
(satisfaction)– Payers/HTAs increasingly interested in patient symptoms and
functional status• Associated with use of services and compliance• Essential part of understanding overall value