AGES: Activating and guiding the engagement of seniors Intervention Training.
Rules of Engagement in Drug Development: Activating the Patient Voice
-
Upload
national-alopecia-areata-foundation -
Category
Health & Medicine
-
view
19 -
download
1
Transcript of Rules of Engagement in Drug Development: Activating the Patient Voice
![Page 1: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/1.jpg)
Rules of Engagement in Drug Development: Activating the Patient Voice
November 14, 2016
Eleanor M. Perfetto, PhD, MSSenior Vice President Strategic Initiatives
![Page 2: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/2.jpg)
![Page 3: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/3.jpg)
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
![Page 4: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/4.jpg)
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
![Page 5: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/5.jpg)
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
![Page 6: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/6.jpg)
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
![Page 7: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/7.jpg)
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
![Page 8: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/8.jpg)
The Role of Patients In Drug Development
Patient
Express
Past Future
![Page 9: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/9.jpg)
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
![Page 10: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/10.jpg)
10
Not all patient-REPORTED outcomes
are patient CENTERED.
Not all patient-CENTERED outcomes
are patient REPORTED.
Don’t fall into the trap….
![Page 11: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/11.jpg)
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
![Page 12: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/12.jpg)
Patient Engagement in the Healthcare Ecosystem
![Page 13: Rules of Engagement in Drug Development: Activating the Patient Voice](https://reader034.fdocuments.us/reader034/viewer/2022042722/58a2a3151a28ab0d0a8b57e3/html5/thumbnails/13.jpg)
Eleanor Perfetto, PhDSenior Vice President, Strategic Initiatives
National Health Council