1. FDA’s Center for Devices and Radiological Health’s State of the Union 2017
Jeff Shuren, MD, JD Center for Devices and Radiological Health U.S. Food and Drug Administration

2. Disclosure Statement of Financial Interest
I, Jeffrey Shuren, DO NOT have a financial interest/arrangement or affiliation with one or more organizations that could be perceived as a real or apparent conflict of interest in the context of the subject of this presentation.

3. In 2009, industry argued that FDA regulation hindered innovation and contributed to the growing number of device companies seeking marketing authorization for their devices abroad before introducing them in the United States and the increasing gap between when a device is approved in another country and when it is approved in the US. This reality, its adverse impact on patients, plus CDRH’s own awareness of our declining performance over almost a decade, led CDRH to implement new programmatic changes. These changes, along with increased user fee funding and changes in federal law, have helped us to change course, strengthen our performance, and better address the rapidly evolving field of medical device innovation.
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4. Novel Device Approvals

5. Examples
Early Feasibility Studies for Transcatheter Mitral Valve Replacement Devices: Study approvals are on par with other countries
Edwards Lifesciences SAPIEN XT/3 for Intermediate Risk Patients: From > 4 years for first generation device to 18 days plus “automatic” CMS coverage for new indication
Medtronic 670G hybrid closed loop system for people with type 1 diabetes (first generation “artificial pancreas”): First in the world

6. Vision We face a critical public health challenge
The U.S. regulatory standard for market approval protects patients by setting a high public health bar but imposes costs that make the U.S. marketplace less attractive for innovators thereby delaying patient access to important technologies
The solution is to reduce the time and cost of the total product life cycle…
device development, assessment, review, manufacturing, monitoring, and reimbursement – without compromising the reasonable assurance of safety and effectiveness standard
Vision
“Patients in the U.S. have access to high-quality, safe and effective medical devices of public health importance first in the world.”

7. Doing Business Better
Since late 2009, CDRH has continuously improved the way we do business through a series of culture, policy and process changes

8. Lower-Risk “Me-Too” Devices (510(k)s)

9. High-Risk Devices (PMAs)

10. Lower-Risk Innovative Devices (De Novos)

11. Clinical Trials (IDEs)

12. Doing Business Different
Since late 2009, CDRH has been transforming the way we do business through a series of culture, policy and process changes

13. Patient-Centered, TPLC Approach Benefit-Risk Tradeoffs
Flexible Regulatory Paradigms Applied Across the Total Product Life Cycle
CDRH Vision
Patient-Centered, TPLC Approach
Benefit-Risk Tradeoffs
PMA, De Novo Benefit-Risk Determination Framework Guidance (2012)
Postmarket Benefit-Risk Guidance (2016)
Evidence Generation
Premarket-Postmarket Balance
Science of Patient Input
Clinical Trials
Regulatory Science
Real-World Evidence
Early Feasibility Study Paradigm Guidance (2013)
Bayesian Statistics (2010), Adaptive Trial Design (2016) Guidances
RWE Draft Guidance (2016)
Expedited Access Pathway Program (2015)
Patient Preference Information Guidance (2016)
IDE Benefit-Risk Determination Framework Guidance (2017)
Unique Device Identification Final Rule (2013)
Balancing Premarket and Postmarket Data Collection Guidance (2015)
Medical Device Innovation Consortium (MDIC) Patient Centered Benefit-Risk Project
MDDT Pilot Program
NEST
MDIC

14. The Value Proposition for NEST
Patients would have more timely access to safer, more effective devices and better information to make decisions
Clinicians would have better information about the use of a given device in practice
Hospitals, clinical practices, and integrated health systems would benefit from improved quality, reliable assurances of safety, and, possibly, relief from multiple reporting requirements
Payers would benefit from access to high-quality evidence on device performance in clinical practice, either alone or compared with other therapies

15. The Value Proposition for NEST
Device manufacturers would be able to provide high-quality evidence at lower cost and in less time to support premarket approval, clearance, and payer coverage, coverage with evidence development and reimbursement decisions and to meet or reduce the need for postmarket study and adverse event reporting requirements
In cases where the potential public health value of the device is high, some data that would otherwise be collected in the premarket setting could be responsibly collected after market entry instead, owing to strong assurances that additional postmarket data would be generated
The system may obviate the need for FDA premarket review of some device modifications because more timely and informative evaluations of the impact of those changes would occur in the course of routine data collection

16. Key Features of NEST
Purpose: To drive down the time and cost and increase the value and use of real-world data to meet the needs of medical device ecosystem stakeholders through a market-driven, collective buying power approach and using a neural network data model:
Independent Coordinating Center responsible for driving standardization of core data elements, data quality, use of common definitions, linkages between data sources, development of advanced analytics, and creating data use agreements as conditions of data sources participating in NEST
Overseen by a Governing Board comprised of representatives of ecosystem stakeholders that is responsible for establishing NEST’s policies and procedures, setting strategic direction and priorities, and directing investments by the Coordinating Center
Business Model:
Base funding from private and public sector sources
NEST users pay for access to and analysis of data from participating NEST data sources
Success requires demonstrating ROI, e.g., less expensive, more timely to use NEST
“Minimally Viable Product” by December 31, 2019

17. Historical model of clinical research: Many recruitment sites and a coordinating center
Hub & spoke model
Top-down decision-making
Sites operated independently 17

18. Neural Network Share Sites and Data
Node
Nodes are clusters of data that we are accessing

19. Neural Network Share Sites and Data
NEST
Coordinating Center

20. Proof of Concept Premarket Examples
Data from the Society of Thoracic Surgeons and American College of Cardiology’s Transcatheter Valve Therapy Registry used to support approval of an expanded indication for transcatheter aortic valve replacement devices
Data from a company’s registry and the Society for Vascular Surgery’s registry used to support approval of a new indication for a drug-coated balloon for in-stent restenosis
Use of registry data as a control arm for pivotal clinical studies
Left ventricular assist devices
Stent grafts
Surgical mesh
Use of database data to demonstrate clinical validity
Next generation sequencing tests for cystic fibrosis

21. Proof of Concept Postmarket Examples
Post-approval study of TEVAR devices using the Vascular Quality Initiative Registry enrolled patients twice as fast as expected
Nesting of required postmarket studies in registries
40% to 60% cost savings
Pilot on using registry data in lieu of submitting Medical Device Reports for on-label adverse events

22. Where Do We Go From Here?

23. Thank You