1. FDA’s Center for Devices and Radiological Health: Partner with Patients Strategic Priority
Jeff Shuren, MD, JD Center for Devices and Radiological Health U.S. Food and Drug Administration

2. Patients are at the Heart of What We Do
CDRH Vision: Patients in the U.S. have access to high-quality, safe, and effective medical devices of public health importance first in the world

3. Evolution of the Role of the Patient
Traditional Medicine:
Provider-led treatment decision-making
Emerging Diseases:
Patient advocacy for availability of and access to new treatments
The Internet:
Patient empowerment through information
The Future Today:
Patient-Provider partnership in shared decision-making

4. Shared Goal Improve the health and quality of life of patients by better understanding their needs, experiences and preferences
Art of Patient Engagement
Science of Patient Input
Patient-Centric Health Care

5. Patient Perspectives from Innovation to Evaluation to Access
Patient-Informed Needs
Patient Preference Benefit-Risk Information
Patient-Centered Outcomes
Patient-Informed
Clinical Trial Design,
Patient Reported Outcomes
Communicating
Benefit-Risk Information
to Patients 5

6. CDRH Strategic Priority 2016 – 2017 Partner with Patients
We must interact with patients as partners and work together to advance the development and evaluation of innovative devices, and monitor the performance of marketed devices.
Promote a culture of meaningful patient engagement by facilitating CDRH interaction with patients.
Increase use and transparency of patient input as evidence in our decision making.

7. Patient Engagement in the medical device arena

8. Promote a culture of meaningful patient engagement
by facilitating CDRH interaction with patients
Interaction
Meaningful Engagement
Partnership

9. Science of Patient Input: PRO + PPI

10. Regulatory Decision Making
BENEFITS
RISKS
???

11. What can PROs and PPI tell us?
Patient Reported Outcomes (PRO)
Endpoints in regulatory studies
Outcomes to monitor postmarket
Interest to payers, providers, regulators, patients
Patient Preference Information (PPI)
Inform endpoints or effect size for regulatory studies
Inform subgroup considerations
Labeling changes / expanded indications
Inform clinical trial design

12. PROs are Ubiquitous in Premarket Review
~Half of device clinical trial protocols collect PROs
~Half of PMAs received in recent yrs contain PROs
Observed a >500% increase in premarket submissions with PRO endpoints
Identified over 600+ premarket submissions containing PROs from CY *
Use of PROM in Device Submissions*
Based on search for PROs in CDRH’s
historical submission archives

13. The value of patient preference information as a function of benefit and risk

14. CDRH Patient Preference Initiative
Talk about guidance…you’ll learn more as training comes

15. Examples and Resources
Guidance: Factors to Consider for Benefit – Risk Determinations in Medical Device Premarket Approval and De Novo Classifications (2012)
CDRH Patient Preference Obesity Study
M. Ho, M. Gonzalez, H. Lerner, C. Neuland, J. Whang, M. McMurry-Heath, A. Hauber, and T. Irony. "Incorporating patient-preference evidence into regulatory decision making." Surgical endoscopy.29(10): ; 2015
Patient Engagement Advisory Committee Announced (2015)
Guidance: Patient Preference Information – Voluntary Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in Decision Summaries and Device Labeling on Patient Preference Information (2016)
MDIC Patient-Centered Benefit Risk Project: A Framework for Incorporating Information on Patient Preferences Regarding Benefit and Risk into Regulatory Assessments of New Medical Technology (2015) (

16. Methods Included in MDIC Catalog
Group
Method
Structured-weighting
Simple direct weighting
Ranking exercises
Swing weighting
Point allocation
Analytic hierarchy process
Outranking methods
Health-state utility
Time tradeoff
Standard gamble
Stated-preference
Direct-assessment questions
Threshold technique
Conjoint analysis and discrete-choice experiments
Best-worst scaling exercises
Revealed-preference
Patient-preference trials
Direct questions in clinical trials
Grouping scheme meant only to facilitate discussion of methods
Not intended to preclude other grouping schemes
Some methods could be assigned to multiple groups

17. What is a Favorable Benefit-Risk Tradeoff?
Risks
↓ Benefit
↓ Risk
New Device
Weight Loss

18. Partner with Patients progress

19. Partner with Patients Targets
Promote a culture of meaningful patient engagement by facilitating CDRH interaction with patients
Establish one or more new mechanisms for CDRH employees to obtain patient input on key pre- and postmarket issues facing CDRH and foster participation of 10 patient groups to participate.
December 31, 2016
Foster participation of 20 patient groups to participate in these mechanisms.
December 31, 2017
50 percent of CDRH employees will interact with patients as part of their job duties.
90 percent of CDRH employees will interact with patients as part of their job duties.
Increase use and transparency of patient input as evidence in our decision making
50 percent of PMA, de novo and HDE decisions will include a public summary of available and relevant patient perspective data considered.
September 30, 2016
100 percent of PMA, de novo and HDE decisions will include a public summary of available and relevant patient perspective data considered.
September 30, 2017
Increase the number of patient perspective studies (e.g., evaluating patient reported outcomes or patient preferences) used in support of premarket and postmarket regulatory decisions. (compared to FY 2015 baseline)
Increase the number of Expedited Access Pathway data development plans or regulatory submissions that consider patient perspectives. (Compared to FY 2015 baseline)

20. Major Milestones & Accomplishments
Sep-2016
Dec-2016
Sep-2017
Dec-2017
10 new pt. groups
1 or more new mechs
20 new pt. groups
1 PEAC
Goal 1
50% staff interactions
90% staff interactions
50% decision summaries
100% decision summaries
Increase Exp. Access pathway
Goal 2
Incr. in patient pers. studies
Partner with Patients
Patient
Interact.
PRO/PPI Training
Outreach
Education
PPI Rollout
Establish PwP Program
Program

21. 30+ patient groups 63 PERCENT Partner with Patients
Promote a culture of meaningful patient engagement by facilitating CDRH interaction with patients
63 PERCENT
Target:* 50 percent by 12/31/16
Participation by staff across All offices in the Center
Facilitated numerous engagement opportunities to meet different needs
*50 percent of CDRH employees will interact with patients as part of their job duties.
30+ patient groups
Target:* 10 groups by12/31/16
Patient groups provide access to patients with a variety of clinical diseases and conditions.
Establishing PE mechanisms makes it easier for staff to get patient input as part of routine business
*Establish one or more new mechanisms for CDRH employees to obtain patient input on key pre- and postmarket issues facing CDRH and foster participation of 10 patient groups to participate.

22. Patient Engagement Feedback from Staff

23. Final PPI guidance document Implemented and in Effect
Partner with Patients
Increase use and transparency of patient input as evidence in our decision making
59 PERCENT
Target:* 50 percent by12/31/16
Revised Template, Staff Training, & oversight ensure consistency and transparency for patient perspective information
*50 percent of PMA, de novo and HDE decisions will include a public summary of available and relevant patient perspective data considered.
↑ PPI & PRO studies
in FY16   
Target:* Increase
Final PPI guidance document Implemented and in Effect
PRO effort will focus on identifying obstacles to use in decision-making
*Increase the number of patient perspective studies (e.g., evaluating patient reported outcomes or patient preferences) used in support of premarket and postmarket regulatory decisions. (compared to FY 2015 baseline)

24. MDUFA 4: Patient Engagement and the Science of Patient Input
Develop expertise and staff capacity to respond to submissions containing PPI or PROs
By the end of FY 2020 hold one or more public meetings to discuss and public findings on:
Approaches for incorporating PPI and PROs in submissions and ways to advance patient engagement
Ways to use patient input to inform clinical study design and conduct
Identify priority areas where decisions are preference-sensitive
Improve regulatory predictability and impact of PROs

25. Summary
Patients are at the heart of what we do. Patients have unique perspectives about the value of benefits and impact of risks of medical devices on their health and quality of life.
FDA’s sharpened focus on patient-centered technology development, evaluation, and use has already begun to positively affect the development and availability of innovative therapies and clinical solutions.
We invite the patient, industry, healthcare professional and academic communities to join in and help us advance this important work.

26. Thank You