This document discusses patient engagement in drug development. It defines patients as those with or at risk of a medical condition, along with their families and caregivers. Patient engagement is described as a bi-directional relationship built on principles like partnership and transparency. The document outlines how patients are experts on their disease based on factors like symptoms, quality of life, and treatment experiences. It traces the evolution of patient engagement through different versions of PDUFA (Prescription Drug User Fee Act) and guidance documents from the FDA. The role of patients is described as continuous throughout the drug development lifecycle, from helping design clinical trials to aiding in data interpretation. Different levels of engagement are identified, from being a study participant to leading research

1. Rules of Engagement in Drug Development:
Activating the Patient Voice
November 14, 2016
Eleanor M. Perfetto, PhD, MS
Senior Vice President Strategic Initiatives

3. 3
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

4. 4
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

5. 5
• 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

6. 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

7. 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

8. The Role of Patients In Drug Development
Patient
Express
Past Future

9. 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

10. 10
Not all patient-REPORTED outcomes
are patient CENTERED.
Not all patient-CENTERED outcomes
are patient REPORTED.
Don’t fall into the trap….

11. Levels of Engagement
Forsythe, et al. JGIM, 2015
Perfetto, ISPOR 2015 Annual Meeting
Stakeholder-
Directed
Collaboration
Consultation
Study
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

12. Patient Engagement in the Healthcare Ecosystem

13. Eleanor Perfetto, PhD
Senior Vice President, Strategic Initiatives
National Health Council
eperfetto@nhcouncil.org