1. The FDA Early Feasibility Study Pilot and the Innovation Pathway
Andrew Farb, MD and Dorothy Abel, BSBME
Division of Cardiovascular Devices
Center for Devices and Radiological Health (CDRH)
Medical Device Innovation 2013: The FDA Town Hall
CRT 2013
Washington, DC
February 26, 2013

2. I/we have no real or apparent conflicts of interest to report.
Andrew Farb, MD
I/we have no real or apparent conflicts of interest to report.

3. Climate Change at FDA FDA recognizes:
Advantages for the device development process with US clinical studies (including FIH and early feasibility studies)
Opportunity for close contact between developers & investigators
Early access to promising novel technology
Continuity of clinical investigations
Human costs associated with delaying access to new technology, in the context of the limitations of current treatment alternatives
A need for a cultural change to shift the focus from not only protecting public health but also to promoting public health

4. Words into Deeds
The core principle in meeting these priorities is the application of benefit/risk principles throughout regulatory decision-making
The Innovation Program
Priority review program for innovative devices with promising and potentially far reaching benefits
Entrepreneurs-in-Residence Program to enhance regulatory science
Early Feasibility Study Guidance
Use of benefit/risk principles to justify study initiation

5. Benefit-Risk Principles
Allow regulators to consider:
The totality of the benefit/risk profile for the device, for example:
Disease condition (e.g., life-limiting, life-threatening)
Limitations of and risks associated with currently available therapies
Patient tolerance for risk & perspective on benefits
Risk mitigation strategies when balancing risks and benefits

7. The Innovation Pathway
Priority review program launched February 2011 intended to:
Shorten the total time to market
Transform the FDA/innovator experience
Targeted for pioneering technologies that address unmet clinical needs and have the potential to revolutionize patient care or health care delivery
Early and intensive investment of resources
Sponsor/FDA collaboration early in the device development process to identify the appropriate and least burdensome pathway to market

8. End Stage Renal Disease Innovation Challenge
Innovation Pathway 2.0:
End Stage Renal Disease Innovation Challenge
(32 Applications Received, 3 Chosen)
UCSF bioimplantable artificial kidney
CreatiVasc
Hemoaccess
Valve System
BPTi
Wearable artificial kidney

9. Entrepreneurs-In-Residence (EIR) Program
Recruitment of entrepreneurs and innovators working with government employees
Time-limited (6 months) participation
Intended to deliver transformational change via principles of lean engineering in rapidly testing, validating and scaling new approaches

10. Current EIR Projects at CDRH
Streamlining clinical trials: Find ways to reduce the time and cost of clinical trials in support of FDA approval or clearance of medical devices
Streamlining the FDA approval-to-CMS coverage pathway: Find ways to reduce the inefficiencies and delays in data collection to support FDA approval and CMS coverage
Striking the right balance between pre- and post-market evidentiary requirements: Find ways to allow efficient use of pre-market studies to allow for timely approvals, with greater emphasis on capturing informative real-world data in post-market studies

11. EFS Pilot Program
Pilot program of 8 innovative device technologies is underway to implement and evaluate the draft EFS guidance
Purpose of the Pilot
Identify strengths and weaknesses of the guidance document
Assess whether adequate guidance is provided toward targeting IDE approval
Status of the Pilot
Pre-IDE and IDE FDA/sponsor interactions are ongoing
FDA
Based on feedback from pilot, the guidance has been modified to:
Improve the device evaluation strategy section;
Provide additional guidance on preparing the informed consent form for an EFS; and
Emphasize benefit/risk principles.
Sponsor

12. FDA EFS Guidance Revisions Based on Pilot Experience and Comments
Emphasized benefit/risk concepts throughout the document
Improved the Report of Prior Investigations section to clarify the information that will be useful to justify study initiation
Expanded on the Device Evaluation Strategy
Included device design information and clinical protocol risk mitigation strategies in the table to provide the appropriate context when considering benefit/risk
Added a Design Control section
Included more guidance on drafting the informed consent form for an EFS
Suggested pre-submission topics for discussion between the sponsor and FDA

13. FDA EFS Guidance Update
Although new pilot projects are not being accepted, EFS IDEs may be submitted prior to the publication of the final guidance.
Many sponsors have committed to conducting EFS studies in the US.
Publication is anticipated soon.

14. Parting Thoughts
Early and continuous interaction between FDA and sponsors will be instrumental in FDA’s efforts to facilitate innovation
Accelerating innovation involves getting to the answers quicker and not just shortening the time to market or reducing regulatory requirements

15. Contacts Early Feasibility Study Guidance Innovation Pathway
Innovation Pathway
Associate Director for Technology & Innovation, CDRH