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FDA/CDRH Webinar

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Passcode: PW1052941 1 
Carla M. Wiese

Policy Analyst for the Early Feasibility Program

Office of Device Evaluation

Center for Devices and Radiological Health

Early Feasibility Study (EFS) 

Investigational Device 

Exemptions

 

A Valuable Regulatory Tool for 

Medical Device Development

2 
Agenda

• What an Early Feasibility Study is 

How it can benefit sponsors

• Key Elements of the EFS Guidance Document 

What does doing the “right testing at the right time” mean? 

• What a successful pathway to an EFS IDE approval looks 

like

• Common questions/tips

• Helpful links

3 
What an EFS IDE is

IDE - Investigational Device Exemption

• An IDE submission allows an investigational device to be used in a 

clinical study in order to collect safety and effectiveness data. 

EFS IDE - A standard IDE except...

• There are significant unknowns about how the device will perform

 Device is generally early in development or 

 Device has a new intended use

• Small number of subjects in the clinical investigation

 Initial evaluation of safety and/or effectiveness

 Proof of concept

* EFS is an 

informal 

designation 4 
How Conducting an EFS in 

the US Benefit Sponsors

Permits A More Efficient Pathway to US Commercialization

• FDA feedback early in product development may...

 Help the sponsor improve their development strategy and 

reduce the chances that unnecessary testing is completed.

 Increase the predictability of data requirements for a 

future study or commercialization needs. 

• Data collection in the US patient population may be easier to 

leverage to support later studies.

5 
How Conducting an EFS in the 

US Benefit Sponsors

Additional Benefits

• Assurance of patient protection under the IDE 

regulations 

• Have better access to technical experts and Key 

Opinion Leaders in the US

• Logistical advantage and proximity to US innovation 

centers 

• Allows for device iteration, including during the EFS 

study, which may result in high quality products

6 
Some Types of IDE studies

EFS Feasibility Pivotal

Small number of patients, < 15 

(approximate)

More patients than EFS Number of patients 

determined by statistical 

needs

 There are fundamental questions 

about device performance & safety 

 Device design may change.

 There may be limited nonclinical 

data available

Enough is known about 

the design, procedure or 

indication to justify 

clinical studies with more 

patients than EFS

Device is the final design 

and there is significant 

information known about 

the design, procedure and 

indication.

Purpose of study can be...

 to demonstrate a proof of concept

 determine what design or procedure 

changes could optimize the therapy

 And more...

Purpose of study can be...

 capture preliminary 

safety and 

effectiveness 

information and to 

adequately plan an 

appropriate pivotal 

study

Purpose of study can be...

 Demonstrate safety and 

effectiveness to support 

a marketing application

*note: not all of these phases are required for market approval

7 
Key Elements of the EFS 

Guidance

• Doing the “Right Testing at the Right Time”

 Comprehensive testing during early phases of device 

development may add cost without significant return (some 

testing may be deferred) 

 EFS is not to take the place of informative nonclinical testing 

• Unknowns and risk can be addressed by...

 Using clinical mitigations to provide patients with extra 

protection 

 The use of more frequent/detailed reporting

 Informed consent recommendations

8 
Key Elements of the EFS 

Guidance continued...

• Allows for timely device and clinical protocol 

changes 

 More changes can be made during the study through 5-day 

notification rather than FDA approval

 Contingent approval: approval of anticipated or proposed 

device changes can be obtained contingent on the completion 

of an agreed upon test plan and acceptance criteria

• Recommendations on pre-submission contents is 

provided

 An example risk assessment method is provided 9 
What Does Doing the 

“Right Testing at the 

Right Time” Mean?

10
• Different test methods for small batches

 E.g. – Single lot Ethylene Oxide sterilization versus full Ethylene Oxide 

sterility validation

• Some test data could be leveraged. Examples...

 Some biocompatibility endpoints could be leveraged from an animal 

study if one is conducted.

 Some test data could be leveraged from a previous version of the 

device. 

FDA Recognizes the Value of 

Alternative Nonclinical Test 

Methods and Leveraging Data

11
Risk presented to patient (after clinical mitigations are 

considered) versus potential benefit.

• Is the probability of failure or patient harm understood and can this 

be mitigated? 

(e.g. risk of irritation to a material, possible mitigation = timely 

clinical assessments/interventions)

• Can a potential failure/harm be detected and mitigated? (e.g. risk of 

patient pain, possible mitigation = titrating therapy, ability to revert to 

standard of care)

FDA Recognizes That Some 

Nonclinical Testing Could be 

Deferred

12
• Can the clinical study be controlled to further protect 

patients? 

(e.g. limiting use of a device to the hospital instead of the 

home, where it may eventually be used, may change EMC 

testing needs)

• Is the clinical situation emergent and/or are there are no 

alternatives available? 

(e.g. long term durability testing deferred due to the 

criticality of short term benefit) 

Considerations for Deferral of 

Nonclinical Testing Continued...

13
Considerations for Deferral of 

Nonclinical Testing Continued...

• Will the nonclinical test data provide valuable information on how 

the device will perform in the proposed clinical study? 

(E.g. If test data will not inform the clinical study today but will 

characterize the device and will be important for developing 

specifications prior to a marketing approval, data could be gathered in 

parallel with the clinical study and submission of this data to FDA 

could be deferred.)

Note: If the clinical situation is non-emergent and there are therapeutic 

alternatives, the amount of nonclinical testing may need to be comparable 

to other available therapies.

14
Understanding and Explaining 

the Utility of the Nonclinical 

Tests is Important

• If it is an animal study, which device performance data will inform 

the human clinical study?

• Is the test conservative or not?

• Is the test validated? 

• Does the test have historical value?

• Will the data be used for quality control in the future?

 What will the data tell us? Are there options to protect patients 

when nonclinical testing has limited utility? 15
What a Successful Pathway 

to an EFS IDE Approval 

Looks Like

16
Recommendation #1

Sponsor is Well Prepared

• Sponsor knows what information they want to learn 

from the EFS

• Sponsor uses their resources: FDA guidance 

documents and recognized standards, CDRH Learn 

Modules, external experts

• Sponsor has reached out to an EFS representative to 

discuss their submission strategy

17
Recommendation #2

Submissions are well planned

• Informational meeting may be useful, for novel ideas in 

particular

• Initial pre-sub includes all the information described in the 

guidance (Goal: agree upon the risks and test plan)

• Additional pre-subs as needed (ex: if test requirements are 

uncertain/discuss clinical protocol)

• IDE submission contains all required information

18
Note: 

The use of pre-submissions to discuss the test plan and the 

clinical protocol... 

• Can be useful when the nonclinical testing needed is unclear, 

can be used to agree upon the test plan that will support an IDE 

submission with FDA

• May avoid the need to re-do expensive and time consuming 

testing

• May help determine appropriate clinical mitigations, reporting 

requirements and the patient population for whom the benefitrisk profile supports inclusion into the EFS

Highly Recommended 19
Submissions are high quality

• Contain enough information for FDA to provide 

valuable feedback.

 Reference the EFS Guidance and IDE required elements (links are 

located at the end of this presentation)

• Contents are well organized and navigable.

• High quality scientific discussion and evidence is 

provided. 

Recommendation #3

20
Recommendation #4

The sponsor is able to describe why additional nonclinical 

testing will not be informative and that a human clinical study 

is appropriate.

• There is a clear identification of potential risks & how they will 

be addressed

 Nonclinical testing, clinical mitigations, reporting

• Explanation is provided for why the plan is sufficient: 

 Explain what can/cannot be learned from bench tests/animal models and 

why any information to be leveraged is directly applicable to the study

• List which tests will be done to support the EFS versus which 

will be done to support a later study if applicable

21
Common Questions

22
Question #1

Is EFS for Novel Technology Only?

NO: EFS are just small studies used to gather information when 

there are significant unknowns.

EFS May be Used for a Variety of Reasons

• To study a novel device

• To study an expanded access (e.g. for devices used for compassionate 

use or emergency use cases) 

• To support new indications for a marketed device

23
When is a good time to talk to 

FDA about an EFS?

After...

• You have established your general device design, intended use 

and what information you would like to gather from the EFS

Before...

• Expensive and time consuming nonclinical testing has been 

started

 It is recommended to communicate with FDA informally 

throughout the development process to optimize submission 

efficiency 

24
The guidance document contains an 

optional risk assessment template. 

When/How is this used?

Called a “Device Evaluation Strategy (DES) Table”

• Can be helpful if you do not currently use another 

method for assessing risk (e.g. ISO standard).

• Should contain a high level description of risks, not as 

detailed as an FMEA. More from a clinician’s perspective.

• Intent is to link primary risks together with risk 

mitigations.

25
Tips

26
1. If you are iterating your device, keep samples of previous 

generations.

• They may be useful in the future for establishing 

biocompatibility equivalence, for example.

2. Keep clear and detailed records of the testing completed 

with each device iteration.

• Ensure that a detailed description of the device iteration is 

included in protocols.

• This may help leverage information in future submissions.

Tips

27
3. If you would like to use test results that were not obtained per 

standard FDA recommendations, we recommend that you 

provide an explanation for why the data is sufficient.

• E.g. If your animal study is intended to support device safety and 

deviates from 21 CFR 58 (Good Laboratory Practices) we 

recommend that you tabulate each part of the regulations, list how 

the study deviates and how you will ensure data integrity and 

minimize bias.

Tips

28

Note: Only animal studies intended to support device safety need to address 21 CFR 58. 

Reference FDA guidance “The Applicability of Good Laboratory Practice in Premarket Device 

Submissions: Questions & Answers” for further information.
Tips

If your animal study is intended to support device safety and 

deviates from GLP, FDA recommends that it include...

• Protocol signed/dated by all key parties prior to initiation of the study

Including objectives, acceptance criteria, and detailed procedures

Include IACUC protocol with amendments 

Clear description of the animals enrolled in the study and their 

final designation

• Quality measures and an explanation of how data integrity is ensured. 

QA personnel to monitor the study - may be in the same company but 

organizationally separate and independent of those engaged in the 

study. 

• Animal facility licenses, accreditations, and assurances

29 Note: Reference FDA guidance on EFS and the draft guidance “General Considerations for 

Animal Studies for Medical Devices” for further information.
4. Understand that FDA feedback is not a directive. It is 

information for your consideration and to assist with 

further discussion.

Tips

30
Helpful Links

• Early Feasibility Study Guidance

http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceD

ocuments/UCM279103.pdf2 

• EFS CDRH Learn Modules

http://www.accessdata.fda.gov/cdrh_docs/presentations/EFS/story.html 

• Pre-Submission Guidance 

http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDo

cuments/UCM311176.pdf 

• IDE Submission Information

http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevi

ce/InvestigationalDeviceExemptionIDE/ucm046706.htm#reqele

• Design Controls Guidance

http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/u

cm070627.htm

• Electronic Submissions Guidance

http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceD

ocuments/UCM313794.pdf 

 

31
Carla Wiese Carla.Wiese@fda.hhs.gov

Andrew Farb, M.D. Andrew.Farb@fda.hhs.gov

Joel Anderson, Ph.D. Joel.Anderson@fda.hhs.gov

Joy Samuels-Reid, M.D. (pediatric 

specialist) Joy.Sameuls-Reid@fda.hhs.gov

Hiren Mistry (ICDB, PIDB, VSDB) Hiren.Mistry@fda.hhs.gov

Rob Kazmierski (CDDB, CEDB, IEDB) Robert.Kazmierski@fda.hhs.gov

Katie Wallon (CSDB, SHDB) Katherine.Wallon@fda.hhs.gov

Erin Keegan Erin.Keegan@fda.hhs.gov

Devjani Saha, Ph.D. Devjani.Saha@fda.hhs.gov

Jemin Dedania Jemin.Dedania@fda.hhs.gov

Charles Warner Charles.Warner@fda.hhs.gov

Krutanjali Shah Krutanjali.Shah@fda.hhs.gov

Tieuvi Nguyen, Ph.D. Tieuvi.Nguyen@fda.hhs.gov

Angelo Green, Ph.D., DABT Angelo.Green@fda.hhs.gov

Andrew Fu, Ph.D. Andrew.Fu@fda.hhs.gov

David Birsen David.Birsen@fda.hhs.gov

Thomas Claiborne, Ph.D. (Ted) Thomas.Claiborne@fda.hhs.gov

Maureen Dreher, Ph.D. Maureen.Dreher@fda.hhs.gov

Christopher Scully, Ph.D. Christopher.Scully@fda.hhs.gov

EFS Representatives

32
Questions? 

Want to share your EFS experience & thoughts for 

Improvement? Contact Carla Wiese, Policy Analyst for the 

Early Feasibility Program - 301-796-0627 or 

Carla.wiese@fda.hhs.gov

General questions about early feasibility studies? Contact 

CDRH's Division of Industry and Consumer Education (DICE) 

at dice@fda.hhs.gov, 1-800-638-2041, or 301-796-7100 

Slide Presentation, Transcript and Webinar Recording will be 

available at:

http://www.fda.gov/training/cdrhlearn

Under “How to Study and Market Your Device” Heading

33