1. The CRT of EFS Where We’ve Been and Where We’re Going
Andrew Farb, MD and Dorothy Abel, BSBME
and
Division of Cardiovascular Devices
Center for Devices and Radiological Health (CDRH)
Food and Drug Administration
FDA Town Hall
CRT 2016
Washington, DC
February 23, 2015

2. Conflict of Interest
No conflicts of interest to report.

3. Considering the past
Recent efforts
To the future

4. EFS @ CRT in Review 2012 Draft EFS guidance
Introduction of the EFS pilot program
2013
Just-in-Time testing and the Device Evaluation Strategy (DES) in depth
Initial EFS lessons learned
Revisions to the draft guidance
2014
Finalized guidance
CDRH EFS Strategic Priority
Progress (approved IDEs) and challenges
2015
EFS Program update: Process and resources

5. From the Beginning
Efforts focused on device innovation and bringing potentially beneficial devices to the US earlier
High interest level
Interaction is critical
Constantly refining the process: Learn-as-you-go

6. EFS IDE Progress CDRH Office of Device Evaluation
* Through 6/30 of each FY

7. The CDRH EFS Program is under the direction of Owen Faris, Clinical Trials Director
Carla Wiese, Policy Analyst in the Clinical Trials Program Focusing on EFS, is the primary point of contact

8. Recent Efforts CDRH Learn Modules available
Device Evaluation Strategy module coming soon; others to follow
CDRH teams established to discuss EFS non- clinical cross-cutting issues
Biocompatibility
Sterility
Animal studies
Outreach to CMS to discuss Category A/B IDE studies

9. CMS
Category A Device: Experimental
Category B Device: Non-experimental/investigational
Open to discussions regarding the appropriate coverage, particularly for studies addressing unmet clinical needs
For Category A EFS IDE devices, may consider coverage reasonable and necessary study costs
But does not include coverage for the cost of the device
Category A (Experimental) device refers to a device for which “absolute risk” of the device type has not been established (that is, initial questions of safety and effectiveness have not been resolved) and the FDA is unsure whether the device type can be safe and effective.
Category B (Non-experimental/investigational) device refers to a device for which the incremental risk is the primary risk in question (that is, initial questions of safety and effectiveness of that device type have been resolved), or it is known that the device type can be safe and effective because, for example, other manufacturers have obtained FDA premarket approval or clearance for that device type.

10. Division of Cardiovascular Devices (DCD) EFS Plans
Translate EFS successes (particularly for trans-catheter valves and aortic endografts) into processes across DCD
Continue internal and external DCD training

11. Translating Success Effective interactions
Communicate justification for study initiation through use of:
DES
Just-in-time testing
Understanding the use of appropriate leveraging from:
Information in the public domain
Testing done on prototype devices

12. Broadening Our Thinking
The links between compassionate use and EFS

13. Compassionate Use (or Single Patient/Small Group Access)
Access to an investigational device under or outside of an IDE (Expanded Access provisions)
Criteria:
Serious disease or condition
No alternative
Prior FDA approval required before compassionate use can occur

14. Justification for Compassionate Use (1)
A description of the patient's condition and the circumstances necessitating treatment
A discussion of:
Why alternatives therapies are unsatisfactory; and
Why the probable risk of using the investigational device is no greater than the probable risk from the disease or condition

15. Justification for Compassionate Use (2)
Describe patient protection measures, for example:
informed consent
concurrence of IRB chairperson
clearance from the institution
independent assessment from uninvolved physician
authorization from the IDE sponsor (if applicable)
If applicable, identify any deviations in the approved clinical protocol that may be needed to treat the patient

16. From Compassionate Use (CU) to EFS
CU can be viewed as belonging to the continuum of expanded access for a small select group of no-option patients to potentially beneficial devices considering (like EFS):
the clinical context;
addressing unmet needs; and
benefit-risk 
CU incorporates additional patient protection measures as compared to other investigational device uses.
This approach is also used in EFS IDEs via the inclusion of enhanced risk mitigation strategies.

17. From CU to EFS
If information is adequate to support CU, there could be adequate evidence to build a case for an EFS
Ideally, efforts to pursue initial compassionate uses to help individual patients should be done in parallel with developing an EFS that builds the foundation to serve larger groups of patients.
However, a series of CU cases should not be used by either sponsors or FDA as a substitute to an EFS, which have the rigor of a clinical investigation.

18. We Still Need Your Input
Please share:
Your DCD EFS experiences (good and bad)
Training needs
Comments and questions
EFS contacts for the Division of Cardiovascular Devices: Dorothy Abel and Andrew Farb