FDA-Regulated Research
Food and Drug Administration (FDA) Resource Links by Topic [PDF]
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| Investigational Drugs | Medical Devices |
What is a drug?
“...articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease…” and “...articles (other than food) intended to affect the structure or any function of the body of man or other animals.” 21 U.S.C. 321(g)(1)
What is a drug clinical investigation?
“...any experiment in which a drug is administered or dispensed to, or used involving one or more human subjects...” – “...an experiment is any use of a drug except for the use of a marketed drug in the course of medical practice...”
Clinical investigations may involve testing (collecting safety or effectiveness data) on a drug* including an FDA approved drug; unapproved use of an FDA approved drug; and/or an investigational drug?
What is a biologic?
A wide range of products (not chemically synthesized) such as virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component...allergic product, protein applicable to the diagnosis, prevention, treatment, or cure of a disease or condition of humans.
Investigational biologics fall under the FDA Investigational New Drug (IND) regulations
When is a dietary supplement considered to be a drug?
Dietary supplements are regulated as drugs when intended to test their ability to diagnosis, cure, mitigation, treatment, or prevention of disease.
For more detail, see the ORI Dietary Supplement Study FAQ. [PDF]
What guidance does FDA provide on other unusual products that may be considered as investigational test articles depending on use in research? (e.g., Cellular Based Tissue Products, Fecal Transplant, Tobacco, Items Generally Recognized as Safe)
Unusual Products That May Be Investigational Test Articles Depending On Use In Research [PDF]
What training does the UK IRB require for a Sponsor-Investigator holding an IND?
FDA IND Responsibilities [HTML]
IRB policy requires mandatory training for investigators who are also serving as sponsors of an IND. [HTML] This involves completion of the Good Clinical Practice Course for Clinical Trials Involving Investigational Drugs on CITI. The ReGARDD training below may be substituted for the CITI GCP course upon request, by providing completion documentation to ORI (HSPTrainingSupport@uky.edu).
ReGARDD Training Modules on IND Sponsor-Investigator Responsibilities [HTML]
What resources are available to help with submitting an IND to FDA?
- FDA Investigator Initiated Investigational New Drug Applications webpage https://www.fda.gov/drugs/investigational-new-drug-ind-application/investigator-initiated-investigational-new-drug-ind-applications
- FDA Guidance for Industry: Investigational New Drug Applications Prepared and Submitted by Sponsor-Investigators http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM446695.pdf
- FDA IND Forms [HTML]
- FDA IND Pilot Portal (NextGen IND Portal designed to automate and ease application process) [HTML]
- ReGARDD IND Template [HTML]
How do you maintain an IND?
ReGARDD IND Maintenance [HTML]
What guidance is available for a Phase I or first-in-man study?
ORI Guide for IRB Review of Phase I Drug Trials [PDF]
What is the procedure to request Emergency Use of an investigational drug for a patient?
If time is sufficient, contact the UK Office of Research Integrity (ORI) at (859) 257-9428 to reach an IRB Chair for confirmation that use meets FDA criteria. Follow the steps in the Emergency Use SOP and use Checklist for documentation.
- Emergency Use SOP [PDF]
- Emergency Use Checklist [PDF]
- FDA Emergency Use of an Investigational Drug or Biologic [HTML]
- FDA's Emergency Call Center [HTML]
What is the procedure to gain Expanded Access to an investigational drug for a patient with an immediately life-threatening condition where no comparable alternative is available?
The FDA Expanded Access program provides a pathway for investigational treatment outside of clinical trials for life-threatening conditions or serious disease with no comparable or satisfactory therapy options. Expanded Access requires FDA approval, IRB approval, and the company’s agreement to provide the investigational product.
- FDA Expanded Access Website [HTML]
- ORI Emergency and Early/Expanded Access Program for Drugs and Devices [PDF]
- Individual Patient Expanded Access Applications: Form FDA 3926 [HTML]
- Expanded Access Program Search Navigator [HTML]
- FDA Expanded Access Instructional Videos [HTML]
- FDA Expanded Access Requests for Investigational Oncology Products [HTML]
Where can I find resources for conducting investigator-initiated studies on COVID-19 treatment?
FDA Drug Development Inquiries for Drugs to Address the COVID-19 Public Health Emergency
CDER is committed to supporting the development of novel drugs, and the potential repurposing of existing therapies, to address the Coronavirus Disease 2019 (COVID-19) public health emergency. The Office of New Drugs (OND) within CDER is expediting this effort, working with potential drug sponsors to rapidly move products into clinical trials, while assuring that the trials are properly designed and are appropriately safe.
What is a medical device?
An instrument, apparatus, implement, machine, contrivance, implant, in vitro reagent, or other similar or related article, including a component part, or accessory which is:
- intended for use in the diagnosis of disease or other conditions, or in the cure, mitigation, treatment, or prevention of disease, in man or other animals, or
- intended to affect the structure or any function of the body of man or other animals, and which does not achieve primary intended purposes through chemical action within or on the body of man or other animals and which is not dependent upon being metabolized for the achievement of any of its primary intended purposes.
What is a device clinical investigation?
A clinical investigation or research involving one or more subjects to determine the safety or effectiveness of a device. 21 CFR 812.3
When is a mobile app or software considered to be a mobile medical app (MMA) or software as a medical device (SaMD)?
App or software used as an accessory to a regulated medical device; or used to transform a mobile platform into a regulated medical device (e.g., control the function of operation of a medical device, used in active patient monitoring to analyze patient-specific medical device data).
See the FDA Policy [HTML] for examples of software or apps that are not considered medical devices, those that are devices but FDA will not enforce regulations because they are low-risk, and those that FDA will regulate.
See the FDA Digital Health Policy Navigator Tool [HTML] designed to help product developers understand whether a software function is potentially subject to or the focus of the FDA's regulatory oversight as a device, and if so, the considerations that may assist in determining the applicable FDA-specific legal and regulatory requirements and recommendations.
See the FDA Infographic to determine whether Clinical Decision Software is a medical device.
Is your device clinical investigation exempt from IDE requirements?
Check the flow chart on the ReGARDD IDE website [HTML]. If still uncertain, you may seek regulatory guidance from the FDA Device Advice Website [HTML] or email DICE@fda.hhs.gov.
Is your study a significant-risk (SR) device trial requiring an Investigational Device Exemption (IDE) application to FDA?
For studies that are not exempt, sponsors are responsible for making the initial risk determination (SR or NSR) and presenting it to the Institutional Review Board (IRB). A device is significant-risk (SR), if use in the study presents potential for serious risk to health, safety, or welfare of a subject, particularly if it
- is an implant;
- is used to support or sustain life;
- is used to diagnose, cure, mitigate, or treat disease, or otherwise prevent impairment of human health; or
- otherwise presents a potential for serious risk to health, safety, or welfare of the subject.
21 CFR 812.32
An SR device study requires an IDE be submitted to FDA and full IDE requirements apply.
- FDA SR/NSR Guidance [PDF]
If the IRB does not concur with the sponsor SR/NSR designation, the FDA serves as the final arbiter. To obtain a written risk determination from FDA submit correspondence labeled “Study Determination” in triplicate to USFDA, CDRH, Document Mail Center – WO66-G609, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002.
- ReGARDD Sample FDA Risk Determination Request [HTML]
Is your study a nonsignificant risk (NSR) device trial?
A device is NSR, if use in the study does NOT meet above SR definition as assessed by sponsor-investigator and confirmed by IRB.
An NSR device study has Abbreviated IDE requirements and is conducted under the purview of the IRB.
If the IRB does not concur with the sponsor SR/NSR designation, the FDA serves as the final arbiter. To obtain a written risk determination from FDA submit correspondence labeled “Study Determination” in triplicate to USFDA, CDRH, Document Mail Center – WO66-G609, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002.
- ReGARDD Sample FDA Risk Determination Request [HTML]
What training does the UK IRB require for a Sponsor-Investigator holding an IDE or conducting a NSR devices study?
FDA IDE Responsibilities for sponsor-investigators of SR Device Studies [HTML]
IRB policy requires mandatory training for investigators holding IDEs or serving as sponsor-investigator for SR or NSR device trials. [HTML] This involves completion of the Good Clinical Practice Course for Clinical Trials Involving Investigational Medical Devices on CITI. The ReGARDD training below may be substituted for the CITI GCP course upon request, by providing completion documentation to ORI (HSPTrainingSupport@uky.edu).
ReGARDD Training Modules on IDE Sponsor-Investigator Responsibilities [HTML]
How do you conduct a SR trial in compliance with IDE Responsibilities?
See FDA IDE Responsibilities for sponsor-investigators of SR Device Studies [HTML]
What is the procedure to request Emergency Use of an investigational medical device for a patient?
If time is sufficient, contact the UK Office of Research Integrity (ORI) main line to reach an IRB Chair for confirmation that use meets FDA criteria. Follow steps in the Emergency Use SOP and use Checklist for documentation.
- ORI Emergency Use SOP [PDF]
- ORI Emergency Use Checklist [PDF]
- FDA Emergency Use of an Investigational Device [HTML]
- FDA's Emergency Call Center [HTML]
What is the procedure to gain Compassionate Use of an investigational device for a patient with an immediately life-threatening condition where no comparable alternative is available?
The compassionate use provision provides a path to accessing investigational devices where a patient has a life-threatening or serious disease or condition; there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition; and potential patient benefit justifies the potential risks of the investigational device. Compassionate use requires FDA approval, IRB approval, and manufacturer agreement to provide the investigational device.
How do I find out if the software/application/algorithm I’m creating is a Medical Device?
Consult the FDA Digital Center for Excellence and use the Digital Health Policy Navigator to determine if subject to FDA oversight and applicable FDA legal and regulatory requirements
How do I find out if the Clinical Decision Support (CDS) software/application/algorithm I’m creating is a Medical Device?
Consult the FDA website and Graphic, Final FDA Guidance, and/or consult the Digital Center for Excellence with questions.
- CLINICAL DECISION SUPPORT TOOL: Flowchart for Determining when FDA Regulations apply [PDF]
- FDA Final Clinical Decision Support (CDS) Software Guidance
- Determine if Your Clinical Decision Support (CDS) Software is a Medical Device Infographic
I’ve used the FDA Digital Health Policy Navigator tool and found that the (software, algorithm, app) I’m developing may be subject to FDA oversight as a medical device. How do I classify or engage FDA to determine procedure for clearance or approval?
For formal classification of a product, a 513(g) request for information with the applicable Office of Health Technology (OHT). You may also engage FDA or request feedback through the Pre-Submission program.
How is FDA regulating the clearance and approval of Artificial Intelligence medical devices that use Machine Learning?
FDA Guidance Marketing Submission Recommendations for a Predetermined Change Control Plan for Artificial Intelligence/Machine Learning (AI/ML)-Enabled Device Software Functions [HTML]
Is there an FDA list of AI/ML Enabled Medical Devices?
FDA List of Artificial Intelligence and Machine Learning (AI/ML)-Enabled Medical Devices [HTML]
How do I determine what other laws and regulations apply to the software or mobile application I’m developing?
Mobile Health App Interactive Tool for determining which laws and rules may apply – Federal Trade Commission [HTML]
