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Rules of Engagement in Drug Development: Activating the Patient Voice
November 14, 2016
Eleanor M. Perfetto, PhD, MSSenior Vice President Strategic Initiatives
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Who is the Patient?
Those having or at risk of having a medical
condition(s) whether or not they currently
receive medicines or vaccines to prevent or
treat a disease, and…
• Family
• Caregivers (typically unpaid)
• Patient advocates
• Patient advocacy groups
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Patient Engagement
Bi-directional relationship between patient and
other stakeholder(s)
Principles of Engagement:
• Reciprocal relationships
• Co-learning
• Partnership
• Trust
• Transparency
• Honesty
Concannon et al., JGIM, 2014 &
Frank et al, Qual Life Res, 2015
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• Impact of disease on health and daily life
• Journey over time
• Outcomes of importance
• Goals and needs
• Preferences
• Experiences with care
• Satisfaction with care
• Quality of life
Patients Are Experts on Their Disease
Evolution of PDUFA
PDUFA V: FDASIA
Rare Diseases
Biomarkers/ PROs
Benefit-Risk Framework
Patient-Focused Drug Development
Initial patient engagement
Shift to consumer engagement
Patient groups re-engage
PDUFA III:
Bioterrorism Preparedness
PDUFA IV: FDAAA
Unintended Consequences
Process for Engagement
PDUFA I
Backlog Reduction
PDUFA II:
Reduce Review Times
1993 -2003
2003 -2013
2013 -2018 2014 2015 2016
2018 -2023
PDUFA VI
Additional Draft FDA Guidance
Biomarkers/ PROs
Adaptive Clinical Trial Designs
Combination Products
FDA Hiring
PFDD:
NHC Patient Community Stratification Tool
Solidifying ongoing patient engagement
PFDD:
NHC/GA Patient Engagement Dialogue at FDA
PFDD:
First Draft FDA Guidance for Patient Engagement
PDUFA VI
Burden of Disease
Holistic Sets of Impacts
Measures for
Identified Impacts
Clinical Outcomes
Assessments
Patient Engagement Guidances
FY 2018 FY 2020FY 2019 FY 2021
The Role of Patients In Drug Development
Patient
Express
Past Future
The Role of Patients In Drug Development
• Continuous throughout the entire product
lifecycle
• Understanding of natural history of disease
• Clinical development plan:
o Select outcomes patient care about
o Select comparators actually used
o Design trials patients will enroll in
• Aid data interpretation
• Aid communicating results
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Not all patient-REPORTED outcomes
are patient CENTERED.
Not all patient-CENTERED outcomes
are patient REPORTED.
Don’t fall into the trap….
Levels of Engagement
Forsythe, et al. JGIM, 2015
Perfetto, ISPOR 2015 Annual Meeting
Stakeholder-
Directed
Collaboration
ConsultationStudy
participant in
an RCT
o Stakeholder-Directed• Patient/Patient group led
o Partnership• Investigator/Co-investigator
o Collaboration• Advisory committee member
o Consultation• Consultant
• Interviews
• Focus groups
• Surveys
o Informal • Unstructured discussions
o Study participant
Patient Engagement in the Healthcare Ecosystem
Eleanor Perfetto, PhDSenior Vice President, Strategic Initiatives
National Health Council
