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FDA’s Early Feasibility Study Program
J. Angelo Green, Ph.D., DABTBranch Chief (Acting)
Contact Lens and Retinal Devices BranchDivision of Ophthalmic and Ear, Nose and Throat Devices
Center for Devices and Radiological Health/FDA
Agenda
• What is an Early Feasibility Study (EFS)
• Provisions of the FDA EFS guidance
• EFS process
• Recommendations for a successful EFS Submission
• How conducting an EFS in the US can benefit sponsors
• Helpful links/Additional information
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What is an EFS?
It is a clinical study that…
• Includes a small number of study subjects (~15)
• Involves use of device design that may be early in development or new intended use for a device already in clinical use
• Is designed to gain initial clinical insights or proof of concept when additional nonclinical testing cannot provide the information needed to advance device development
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• Any type of device may be evaluated in a small clinical study
• EFS May be Used for a Variety of Reasons➢ To study a novel device
➢ To support new indications for a marketed device
➢ To study an expanded access (e.g., for devices used for compassionate use or emergency use cases)
What Types of Devices May be Evaluated in an EFS?
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EFS
• Device design may not be final, with changes anticipated
• Less nonclinical data available for the study device with potentially more reliance on device design and leveraged information
• Intended to provide initial insights
Traditional Feasibility
• Device design may be final or near-final
• Generally supported by more nonclinical or prior clinical data
• Intended to capture preliminary safety and effectiveness information and to adequately plan an appropriate pivotal study
Pivotal
• Final device design
• Clinical feasibility established and all IDE-level nonclinical data completed
• Intended to capture safety and effectiveness data to support a marketing application
Types of Clinical Studies
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Early Feasibility Study Traditional Feasibility Study
Applicability Most useful for novel technology or intended use
Generally useful
Purpose Obtain initial insights Capture preliminary safety and effectiveness information and to plan an appropriate pivotal study
Justification for study initiation
Potentially more reliance on device design, leveraged information, and clinical mitigation strategies
Generally supported by more nonclinical (or prior clinical) data
Stability of device design Changes may be anticipated
Near final or final design
# of subjects ~ 15 Varied, but can be large (100)
Types of Clinical Studiescontinued…
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EFS Program
• Voluntary program that allows devices in an early stage of development be evaluated in a small human clinical study in the US.
• Goals: To provide patients in the US access to technology that may benefit them & to encourage US innovation
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DOED1 EFS Submissions(cumulative)
3
7
15
38
42
2014 2015 2016 2017 20181DOED: Division of Ophthalmic and Ear, Nose and Throat Devices
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Key Elements of the EFS Guidance
• Provides a regulatory toolkit that enables sponsors and regulators to think in new ways about device development
• 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
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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
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EFS Process
The EFS guidance recommends and encourages early and frequent interactions between FDA and sponsors to enhance the predictability of the EFS IDE review process
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What a Successful Pathway to an EFS IDE Approval Looks Like
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Recommendation #1
Preparation
• 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
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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
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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 benefit-risk
profile supports inclusion into the EFS
Highly Recommended
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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
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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
Recommendation #4
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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
Communication with FDA throughout the development process is recommended to optimize submission efficiency
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How Conducting an EFS in the US Benefits 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 US studies
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Helpful Links
• Early Feasibility Study Guidance
http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM279103.pdf2
• CDRH Learn Modules
https://www.fda.gov/Training/CDRHLearn/default.htm
• Pre-Submission Guidance http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM311176.pdf
• IDE Submission Information
http://www.fda.gov/MedicalDevices/DeviceRegulationandGuidance/HowtoMarketYourDevice/InvestigationalDeviceExemptionIDE/ucm046706.htm#reqele
• E-Copy Submission Guidance
http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM313794.pdf
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DOED’s Commitment to Outstanding Customer Service
• Response to queries from external stakeholders within two (2) business days
• If immediate definitive answer not possible, acknowledgement with target response date provided
• DOED Contact: [email protected]
