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

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 www.fda.gov

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 www.fda.gov

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

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 www.fda.gov 5

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. www.fda.gov

Patient Engagement in the medical device arena

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

Science of Patient Input: PRO + PPI

Regulatory Decision Making BENEFITS RISKS ???

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

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 CY2000-2014* Use of PROM in Device Submissions* Based on search for PROs in CDRH’s historical submission archives

The value of patient preference information as a function of benefit and risk www.fda.gov

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

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): 2984-2993; 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) (http://mdic.org/pcbr) www.fda.gov

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 www.fda.gov

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

Partner with Patients progress

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) www.fda.gov

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 www.fda.gov

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. www.fda.gov

Patient Engagement Feedback from Staff www.fda.gov

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) www.fda.gov

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 www.fda.gov

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.

Thank You www.fda.gov