Research with Drug Products
Any use of a drug product is considered to be an experiment, excepting the use of a marketed drug in the course of standard medical practice, according to FDA regulations. Likewise, any experiment in which a drug is administered, dispensed to, or used, involving one or more human subjects, is considered by the FDA to be a “clinical investigation.” Clinical investigations are considered to be subject to FDA regulations.
Definition of a Drug
A drug product achieves its primary intended purposes through chemical action within or on the body AND is dependent upon being metabolized for the achievement of any of its primary intended purposes.
- Intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease in man or other animal; OR
- Intended to affect the structure or any function of the body of man or other animals (other than food); OR
- Recognized in the official US Pharmacopoeia, official Homoeopathic Pharmacopoeia of the United States, or official National Formulary
When is a clinical investigation with a drug product subject to IND regulations? (i.e., When is an IND required?)
It must be assessed whether the clinical investigation meets the criteria for exemption from IND regulations. Investigators at Penn are not permitted to self-determine exemption from IND regulations.
- Product Marketing Status
Is the product approved by FDA or marketed in some way? (NOTE: Unapproved drugs are never exempt from IND regulations).
- Type of Product
What is the type of product being used? There are different exemption criteria depending on the type of product (e.g., drug versus dietary supplement).
Requesting an IND Exemption Determination
Penn Medicine Office of Clinical Research Regulatory Services
Penn Medicine Principal Investigators are recommended to request an exemption determination from the Office of Clinical Research Regulatory Services. OCR Regulatory will conduct exemption determinations for any product type.
Non-Penn Medicine Principal Investigators may request an exemption determination from the IRB. In order to request an IND exemption determination, submit the Research with Drugs Form with your initial application.
Department of Radiology
Penn Medicine Principal Investigators using imaging agents (including Gadolinium) may request an exemption determination from the Department of Radiology.
Penn Medicine RRSC
Penn Medicine Principal Investigators using Nuclear Medicine Agents (radiopharmaceuticals) should seek an exemption determination from Penn Medicine RRSC.
Any Principal Investigators may seek an exemption determination directly from the FDA.
Requirements for Conducting Clinical Investigations
PSOM Policies and Procedures
Perelman School of Medicine policies and procedures apply to research with drugs, when the research involves Penn Medicine patients and / or PSOM faculty. This includes research that is led by Faculty from non-Penn Medicine schools such as School of Nursing, SAS, Engineering, etc. Questions about these policies and procedures can be directed to the Office of Clinical Research.
PI Acknowledgement of Responsibilities in Biomedical Research
The Principal Investigator is responsible for:
- Ensuring that an investigation is conducted according to the signed investigator statement, the investigational plan, and applicable regulations;
- Protecting the rights, safety, and welfare of subjects under the investigator’s care; and
- The control of drug(s) and device(s) used in the investigation.
The Principal Investigator’s Acknowledgement of Responsibilities document has been developed as a reminder of the responsibilities of the principal investigator in a clinical investigation.
Information about IND Submissions
Submitting an IND Application to the FDA
FDA’s guidance is linked below. For further guidance on submitting an IND to the FDA, please contact the PSOM Office of Clinical Research, Regulatory Support Team. FDA Guidance Sponsor Investigator IND Applications
Treatment of a Patient with an Unapproved Product
An unapproved product can be administered for treatment purposes under the FDA’s expanded access program, if certain criteria are met. For more information, please click below.