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The Patient Perspective In Drug Development

Pauline McNulty

April 13th, 2015

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The Healthcare World is Changing

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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

Example of Findings from Rheumatoid Arthritis Trials: Improvement in Daily Activities

*DMARD: disease-modifying antirheumatic drug†TNF: tumor necrosis factorMTX: Methotrexate http://www.orencia.bmscustomerconnect.com/How-It-May-Help#clinical-trial-results

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