This document discusses the increasing role of patients in drug development and healthcare decision making. It notes that patients want a greater voice in deciding what evidence is required to show drug effectiveness, influencing clinical trial design, and assessing risk/benefit for regulatory approval. PROs (patient-reported outcome measures) are highlighted as a way to systematically capture the patient perspective on symptoms, functioning and quality of life in clinical trials and practice. The example shows how PRO data from rheumatoid arthritis trials demonstrated improvement in daily activities with certain drug treatments.

1. 0
The Patient Perspective In Drug Development
Pauline McNulty
April 13th, 2015

2. 1
The Healthcare World is Changing

3. 2
Transformation of Patient Involvement in
Healthcare: Enablers
Social Connectivity
Social media & data
sharing, peer reviews,
peer pacts, wellness
tracking, games
Data
Sensor & monitoring
devices, algorithms,
visualization, risk
identifiers
Consumer Offerings
Health information
websites & apps,
provider ratings,
comparison tools
Cost Management
More coverage options,
chronic care tools,
HTAs, co-pay trade-
offs, wellness programs
Drug Approval
Burden of proof
requirements, benefit-
risk, PROs
Outcomes Metrics
Value-based pricing,
standard performance
measures, alternative
care delivery
Communication
Platforms
Real time physician
access, remote visits,
retail clinics
Information Systems
Cloud based records,
secure collaboration
networks, patient-
friendly records
Technology Advancements
Driving Information Access
Demands on Access and
Reimbursement
The Empowered Patient and Patient Organizations

4. 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?
3

5. Patients Want A Greater Voice In Drug Development
Input 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
4

6. 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
5

7. 6
I´m worried
and concerned
GI symptoms
bother me!
I can not bend
over or exercise
My whole life
Is affected
Heartburn
disturbs my sleep
I can not eat and
drink whatever
I like
PROs Represent The Voice of The Patient

8. 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
7

9. 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
8

10. Example of Findings from Rheumatoid Arthritis Trials:
Improvement in Daily Activities
*DMARD: disease-modifying antirheumatic drug
†TNF: tumor necrosis factor
MTX: Methotrexate
http://www.orencia.bmscustomerconnect.com/How-It-May-Help#clinical-trial-results
9