Frequently Asked Questions

This page includes general frequently asked questions (FAQs) related to the Institutional Review Board and how it functions. Additional FAQs will be added to this page over time to provide investigators to find answers to commonly asked questions posed to the IRB and the IRB Office.

Where are FAQs related to specific topics located?

In addition to the more general FAQs located in the accordions (the green bars below) on this page, many other FAQs pages can be found located at the bottom of IRB-related webpages. These FAQs address commonly asked questions relevant to information and content discussed on the pages on which they reside.

  1. An external IRB is the IRB of record for my study. Do I need to submit anything in eIRB, and if so, why?

    Yes. Even though the CHOP IRB may not have oversight over the study, CHOP’s Human Subjects Protection Program (HRPP) still needs to know and track what human subjects research activities go on at the institution. In addition, the CHOP IRB must confirm that the consent form aligns with CHOP's requirements (e.g. HIPAA and injury compensation language), and CHOP Ancillary Committees may need to review and approve the study (e.g. the Investigational Pharmacy, Contracting, IBC, Conflict of Interest Committee, Medical Device Committee, etc.).

  2. Once I have submitted in eIRB and indicated that an external IRB is the reviewing IRB, do I have to wait for an acknowledgement before I can start my research, and if so, why?

    Yes. The CHOP IRB must confirm that the consent form aligns with CHOP's requirements (e.g. HIPAA and injury compensation language), and CHOP Ancillary Committees may need to review and approve the study (e.g. the Investigational Pharmacy, Contracting, IBC, Conflict of Interest Committee, Medical Device Committee, etc.). Once all the institutional requirements are met, the CHOP IRB will send a letter to accept the reliance on the outside IRB. Human subjects research at CHOP for that particular study cannot start until that letter has been issued and reliance documentation has been executed.

  3. If CHOP is relying on another IRB for my study, do I have to submit updates in eIRB, and if so, why?

    Yes. CHOP’s Human Subjects Protection Program (HRPP) still needs to track what human subjects research activities go on at the institution. In addition, CHOP Ancillary Committees, such as the Investigational Pharmacy, need access to currently approved documents (such as the Investigator’s Brochure).

  4. How do I submit Cooperative Updates?

    In the main study workspace for the study in eIRB, under the ‘Create’ header on the left side of the workspace, find the “New Cooperative Update” button. Clicking on it will open a new smartform. After the smartform has been completed by the investigator, using the “Submit Cooperative Update” activity will send the submission to the IRB. The IRB has created a Job Aid (under 'eIRB Help') to facilitate the submission process.

    Note: Multiple, separate Cooperative Updates can be active at once.

  5. Once I submit a cooperative update, do I have to wait for an acknowledgement before I can implement e.g. amendment changes?

    No. Once CHOP has accepted the reliance on an outside IRB, amendment changes can be implemented as soon as the reviewing IRB has approved the amendment.

  6. I’m writing a grant for a multi-center study and would like CHOP to be the Reviewing IRB. What is the process?

    Contact the IRB Director to discuss the grant requirements and sIRB logistical issues.

  7. What is CHIRP?

    CHIRP is the CHOP IRB's tool that allows investigators from external institutions who are relying on the CHOP IRB for IRB review to communicate directly with the CHOP IRB. See Single IRBs and IRB Reliance Agreements for more information.

  8. The relying PI for my study has received an email from CHIRP, but the link states 'The invite token is expired'! What do I do?

    This error can be resolved by having the external PI navigate to the CHIRP homepage, and request a password reset. Note: Each time a password reset is requested, a new automated email is issued. The new email will invalidate any prior emails, so the relying investigator should be sure to always use the most recent email.

  9. Do relying sites need to update their CHIRP application with investigators or their training(s)?

    Other than the relying PI and Study Coordinator, no. The CHIRP application asks relying sites to “Confirm that all study team members are in compliance with local institutional training requirements for human subjects research. (e.g. CITI training and/or other institution specific training)”. Otherwise, any relying personnel-specific changes are the responsibility of the relying institution to manage.

  1. What is an IRB?

    IRB is an abbreviation for the Institutional Review Board. The IRB is an administrative body established to protect the rights and welfare of human research subjects recruited to participate in research activities conducted under the auspices of the institution with which it is affiliated. The IRB has the authority to approve, require modifications in, or disapprove all research activities that fall within its jurisdiction as specified by both the federal regulations and local institutional policy.
    The official name of the IRB committees at CHOP is The Committees for the Protection of Human Subjects. For more information about the CHOP IRB, its membership and structure, go to CHOP Institutional Review Board.

  2. How do I contact the IRB?

    For IRB contact information, please go to Contact the IRB.

  3. What is an FWA number and what is CHOP’s FWA number?

    FWA stands for Federal Wide Assurance, which must be submitted to the Office of Human Research Protections (OHRP) in order for an IRB to review research that receives federal funding. An FWA is a document that assures an institution's commitment to comply with the federal research regulations, with national and international ethical principles, and with federal, state and local laws related to the protection of human research subjects. CHOP's FWA number is FWA00000459. For more information about FWAs, go to Federal Wide Assurance (FWA).

  4. What is an Institutional Official and who is that at CHOP?

    The Institutional Official (IO) is responsible for ensuring that the Human Research Protection Program (HRPP) functions effectively and that the institution provides the resources and support necessary to comply with all requirements applicable to research involving human subjects. The IO represents the institution named in the Federalwide Assurance (FWA). The IO is the individual who is legally authorized to act for the institution and, on behalf of the institution, obligates the institution to the Terms of the Assurance. At CHOP, the Institutional Official is Matthew Hodgson, MA, MS, CHRC, CCRP.

  5. What is the difference between laws, regulations and IRB policies/procedures?

    There are a variety of federal regulations and state laws that apply to human subjects research. In addition, the Office of Human Research Protections (OHRP) and the US Food and Drug Administration (FDA) have issued guidance documents and other materials that reflect their agency's current thinking.
    The IRB's Standard Operating Procedures (SOPs) provide the institution's standard approach to the interpretation of the federal regulations, best practice standards and state and local law. The SOPs serve as a reference for the IRB membership, IRB Office staff, and the CHOP community of investigators. Regulations are necessarily grey and their interpretation can evolve over time. The IRB SOPs, therefore reflect our current understanding of the Office of Human Research Protections (OHRP) and the FDA's interpretation of the regulations. In addition, the IRB has identified a number of ways to implement additional flexibility in its SOPs for research that is neither federally funded nor federally regulated. For more information go to Regulations and IRB policies.

  1. Who reviews my study/application?

    When an application is received by the IRB, it is triaged (based on information in the application) to be reviewed either by the convened board or, the IRB chair or a designee of the chair (vice chairs, the IRB director, assistant director or a senior Analyst). Before being forwarded to the reviewer, the application is pre-reviewed by an Analyst to ensure that it is ready to be reviewed (e.g. scientific review has been conducted, the protocol and consent form are attached, as applicable).

  2. I have submitted an application in eIRB. How do I know where it is in the review process?

    The current study state (i.e. where the submission is in the review process) is always indicated in the red bar in the top left hand corner in your eIRB workspace. Examples of common states your submission may be in are:

    • “In Department/Division Review” (the study awaits review by your Department Chair or Division Chief and has therefore not yet been submitted to the IRB);
    • “In Pre-Review” or “In Review” (it is being reviewed by the IRB);
    • “Changes Requested” (it is with you to respond to stipulations); or
    • “Ancillary Approval Pending” (all IRB issues have been addressed but approval from one or more ancillary committees, such as Conflict of Interest, Technology Transfer etc., may be outstanding).

    If the approval of ancillary committees is pending, you can find which ones are outstanding and which approvals have already been received by looking at the information at the top of your study workspace (the section where e.g. the study name, PI, study coordinator, and funder are listed) under "Ancillary Review Status".

  3. Who should I contact with questions about the stipulations sent to me?

    The first contact for questions about stipulations sent to you is the Analyst assigned to the study (either by email, phone, office hours or one-on-one meeting as necessary). S/he will then either be able to provide you with the information needed or, if necessary, will contact the reviewer to set up a teleconference or meeting between the PI and the reviewer. In general, questions about the eIRB application and consent form should be directed at the Analyst for the study first. Questions regarding the protocol, reasons for deferral, risk/benefit or waiver determinations should be directed to the reviewer.

  4. Are the same Analyst and reviewer looking at all amendments to my study?

    While it is the IRB’s goal to maintain consistency and keep the same Analyst/reviewer pair on a study throughout its duration, this has to be balanced by the necessity of a timely flow of submissions through the office. Depending on the nature of the amendment (staff change, change to recruitment materials versus substantial changes to the study design) and overall workload (full board meetings, high submission volume, vacation schedules), amendments will also be assigned to Analysts/reviewers who have not reviewed the study before.

  5. My submission is time sensitive. How do I let the IRB know?

    If a submission is time sensitive, (e.g. due to a just-in-time grant funding), please indicate this at the time of submission (as a comment in the workspace). Upon receipt of a JIT notice, please promptly contact the IRB. This allows the IRB to triage accordingly. As the IRB will have to prioritizie such submissions over those of other investigators, please attach the correspondence (from e.g. the funding agency) indicating the deadline for approval with the IRB submission.

  1. Who can make the determination that an activity is (or is not) human subjects research?

    At CHOP, the investigator is permitted to make the determination that what they are doing does not meet the definition of human subjects research. Only activities that meet the definition of human subjects research require submission to the IRB for a review and approval or for a determination of exemption. However, there are many situations where it may be unclear whether or not the proposed activities meet the definition of research and if it does, whether or not it involves human subjects. Requests for the IRB to make a determination as whether or not an activity meets the definition of human subjects research should be submitted via the eIRB system. Examples:

    A QI may produce as a byproduct information that is worth sharing. Depending on the design of the project, it might or might not be human subjects research.

    Receipt of a large Medicaid data set containing hundreds of thousands of records might contain birth dates and dates of service. If the investigator is uncertain, the IRB would need to determine if the individuals would be readily identifiable or not.

  2. Is innovative care designed to enhance the well-being of an individual using an FDA approved drug or device research?

    No. The FDA and the IRB do not regulate the practice of medicine. Since the drugs, biologic or device is approved and the intent is to provide care for an individual patient and not to produce generalizable knowledge, this is not human subjects research.

  3. Is a database (registry) designed for use in future research considered research?

    Yes. If the database/registry is being designed and used for the purposes of future research then even though no research activities are planned per se, this is considered research and also falls under the requirements of HIPAA. If the database will be used primarily for clinical care or hospital operations purposes, then prior IRB review is not required. However, any research that intends to use the information from the database would require IRB review.

  4. Does a case report require prior IRB review?

    No. The CHOP IRB does not consider retrospective case reports to meet the definition of research. However, prospective research involving just a single subject do not qualify as case reports just because the research is limited to one individual.

  5. What about a case series?

    It depends; a determination from the IRB may be necessary. A case series can be several notable cases reported at the same time. The IRB does not consider this to be research. In IRB SOP 407: Determining When a Proposal Meets the Definition of Human Subjects Research, the IRB excludes case series of 5 or fewer patients from review. Case series with 6 or more cases must be submitted to the IRB as research or for a determination that they do not meet the definition of research. The rationale for this requirement is that case series involving greater than 5 records frequently involve dozens of records and could be more appropriately classified as descriptive or observational research. See the IRB's webpage on Study Design.

  6. We are conducting a Quality Improvement/Quality Assurance project and have no plans to publish; do we need IRB review?

    Maybe. Publication is a consideration but it does not distinguish research from non-research QI/QA projects. The intent of QI/QA is to improve the care of patients at CHOP, often using proven care strategies and methods and most are not research. The project may still produce generalizable knowledge that clinicians would wish to share with others. If the QI/QA project intends to test an as yet unproven care strategy using a standard research methodology, then it is likely that the project is research. For more information, see the OHRP Quality Improvement Activities Frequently Asked Questions website, Quality Improvement vs Research, or the CHOP Screening Checklist for Quality Improvement (QI) Projects.

  7. What about receipt of deidentified datasets or biospecimens?

    No. Use of de-identified datasets or biospecimens is not human subjects research. In order for the data/biospecimens to be de-identified, there can be no identifiers and there can be no link to identifiers. A coded specimen that can be linked back to individuals is not considered de-identified. If the providers have access to the key but will not provide the recipient with any PHI, then from the recipients’ perspective the data or specimens are de-identified. The provider of the data/specimens must either provide a letter stating that they will not provide any identifiers, or have policies which clearly state that they will not provide identifiers. For details of the requirements for data sharing, see Guidance on Engagement of Institutions in Human Subjects Research

  8. Can I share de-identified data or biospecimens with another investigator?

    Yes. Use of deidentified data or specimens does not constitute human subjects research. However, the investigators must adhere to both the limitations (if any) specified in the consent form used to obtain the specimens and the subject's expressed preferences with regard to future use. If re-use of study data or biospecimens for future research is a possibility, then the consent form must include information about future use. For most studies, subjects should be able to opt-in or opt-out without affecting their ability to enroll in the main study. The IRB's Consent Form Templates include example language for optional future use. The subjects' preferences should be noted as part of the study record to ensure that future use of data and specimens matches each subject's decision.

  9. If CHOP is not enrolling subjects in the research but is functioning as the data coordinating center (DCC) does this require review by the IRB?

    It depends. If the research is funded by the DHHS or if the DCC will receive identifiable subject information, then the CHOP is engaged in the research and the IRB must review the activities of the DCC. If the DCC will only receive de-identified data and the funding is not from DHHS, then there is no obligation to obtain IRB approval. Having monitors (CRAs) from the DCC perform source-data verification during a site visit does not engage the DCC in Human Subjects Research unless it receives PHI from the site.

  1. What types of devices need to be included in the eIRB application?

    (1) Unapproved devices, (2) all investigational devices, meaning all those being studied for their safety or effectiveness regardless of whether they are FDA-approved/cleared or not, and (3) FDA-approved/cleared devices that are not approved for use for clinical use at CHOP.

  2. How do I include a device in the eIRB application?

    Check the "Device" box in Section 5.04 (2.0). This will then require completion of Section 8.02. The information about the device - e.g. brochures, manuals, 510(K) clearance approval letters - should all be attached in Section 12.02 (3.0). Risk information should be included in the protocol, consent form and in eIRB Section 11.01 (1.0).

  3. The device is not a physical instrument, it's medical software, a diagnostic test/assay, an MRI sequence, or a medical app. What type of storage plan is required?

    There needs to be a plan that explains how the use of the device is only used for this research study and that will limit access to members of the investigative team. For example, there needs to be a plan to limit the use of unapproved MRI sequences so that they are not used for clinical care.

  4. Who decides whether a device is non-significant risk or significant risk?

    The sponsor is responsible for making the initial NSR-SR determination. The IRB is responsible for reviewing and concurring with or disagreeing with, the sponsor's assessment. If the sponsor has a letter from the FDA indicating that the device is NSR, the IRB must accept that decision (the FDA's determination is final). Otherwise, if the IRB disagrees with the sponsor's determination that the device is NSR, the sponsor must request that the FDA issue its opinion.

  5. If the IRB has determined that the device meets the definition of an NSR device, do I need to get an IDE from the FDA?

    No. If the IRB concurs with the sponsor's NSR determination, then it is effectively issuing an abbreviated IDE to conduct the study. NSR device studies must follow the abbreviated requirements under 21 CFR 812.2(b) and the FDA's regulations for Human Subjects Protections and the IRB operations (21 CFR 50 and 56).

  6. My sponsor initially determined my device is NSR but the IRB determined it was SR. What do I need to do?

    The sponsor must obtain one of the following from the FDA: (1) a determination that the device is an NSR device or (2) an IDE (in the event the FDA agrees that the device is an SR device).

  7. My sponsor initially determined the device is an SR device. Can I submit the study to the IRB for review if the FDA hasn't issued the IDE yet?

    Yes. However, the IRB will not release its final approval until an IDE has been issued and documentation, including the IDE number, has been provided to the IRB.

  1. I want to use a marketed drug off label in clinic. Do I need to submit to the IRB or the FDA?

    Good medical practice and the best interests of the patient require that physicians use legally available drugs, biologics and devices according to their best knowledge and judgement. If physicians use a product for an indication not in the approved labeling, they have the responsibility to be well informed about the product, to base its use on firm scientific rationale and on sound medical evidence, and to maintain records of the product's use and effects. Use of a marketed product in this manner when the intent is the "practice of medicine" does not require the submission of an Investigational New Drug Application (IND), Investigational Device Exemption (IDE) or review by an Institutional Review Board (IRB). However, other institutional oversight requirements (e.g. Therapeutic Standards Committee approval) may apply.
    See FDA Guidance on "Off-Label" and Investigational Use Of Marketed Drugs, Biologics, and Medical Devices.

  2. I want to conduct a clinical investigation using a marketed drug off label. Do I need to submit an IND application to the FDA?

    Whether an IND is needed to conduct a clinical investigation of a marketed drug primarily depends on the intent of the investigation and the degree of risk associated with the use of the drug in the investigation. A clinical investigation of a marketed drug is exempt from the IND requirements if all of the criteria for an exemption in § 312.2(b) are met:

    • The drug product is lawfully marketed in the United States.
    • The investigation is not intended to be reported to FDA as a well-controlled study in support of a new indication and there is no intent to use it to support any other significant change in the labeling of the drug.
    • In the case of a prescription drug, the investigation is not intended to support a significant change in the advertising for the drug.
    • The investigation does not involve a route of administration, dose, patient population, or other factor that significantly increases the risk (or decreases the acceptability of the risk) associated with the use of the drug product (21 CFR 312.2(b)(1)(iii)).
    • The investigation is conducted in compliance with the requirements for review by an IRB (21 CFR part 56) and with the requirements for informed consent (21 CFR part 50).
    • The investigation is conducted in compliance with the requirements of § 312.7 (i.e., the investigation is not intended to promote or commercialize the drug product).

    See FDA Guidance on Determining Whether Human Research Studies Can Be Conducted Without an IND. and IND Exemptions for Studies of Lawfully Marketed Drug or Biological Products for the Treatment of Cancer

  3. What are the IRB’s responsibilities in the selection of clinical investigators and research sites, and determining if an investigational new drug application (IND) or investigational device exemption (IDE) is required?

    The regulations at 21 CFR 56.107(a) require that an IRB " able to ascertain the acceptability of the proposed research in terms of institutional commitments and regulations, applicable law, and standards of professional conduct and practice..." In addition, the regulations at 21 CFR 56.111 require that an IRB determine that the proposed research satisfies the criteria for approval, including that "...risks to subjects are minimized...[and] reasonable in relation to anticipated benefits, if any, to subjects...".

    Qualification of investigators

    The IRB needs information about the qualifications of the investigator(s) to conduct and supervise the proposed research. The IRB may also need to assess the investigator’s training and experience specifically related to the proposed study, particularly if the proposed research involves higher risks, vulnerable subjects, or novel technologies. In its assessment, the IRB may also rely on the clinical investigator’s medical department/division for information about the clinical investigator’s qualifications, or require a statement regarding the investigator’s qualifications from the chair of the investigator’s department/division.

    Adequacy of the research site

    The IRB needs to assess the site where the proposed research will take place to ensure it can adequately execute the protocol requirements. Depending upon the nature and risks of the proposed research and the IRB's prior knowledge of, or relationship to, the institution or other site at which the research will take place, this may be relatively simple and straightforward or it may entail a more involved assessment.

    Verifying the determination of whether an IND or IDE is required for an FDA-regulated investigation

    It is the sponsor’s responsibility to make an initial determination on whether a clinical investigation meets the criteria for an IND exemption. At CHOP, the IND-IDE Support Program can help a CHOP sponsor determine whether a specific study meets these criteria. The sponsor needs to include their determination (and the basis for that determination, including any supporting documentation) to the IRB with their protocol submission in eIRB. The IRB will, based on the scientific literature and generally known clinical experience (as presented by the investigator), either agree or disagree that there is no significant increase in the risk (or decrease in the acceptability of the risk) associated with the use of the drug product.
    If the IRB agrees with the sponsor’s determination, no IND submission to the FDA is necessary.
    If the IRB disagrees or has concerns that cannot be addressed by the sponsor, the sponsor will need to submit to the FDA. The FDA will then either issue an exempt determination or decide that an IND is required. The FDA’s decision is final.
    See FDA Guidance on IRB Responsibilities for Reviewing the Qualifications of Investigators, Adequacy of Research Sites, and the Determination of Whether an IND/IDE is Needed.

  4. I want to study a dietary supplement in a clinical investigation. Do I need to submit an IND application to the FDA?

    It depends.
    Under the Dietary Supplement Health and Education Act of 1994 (DSHEA), a dietary supplement is defined, in part, as a product taken by mouth that is intended to supplement the diet and that contains one or more dietary ingredients.
    Under DSHEA, a dietary supplement is not considered a drug if the intended use for which it is marketed is only to affect the structure or any function of the body (i.e., not intended to be used for a therapeutic purpose).
    Similarly, whether an IND is needed for a clinical investigation evaluating a dietary supplement is determined by the intent of the clinical investigation. If the clinical investigation is intended only to evaluate the dietary supplement’s effect on the structure or function of the body, an IND is not required.
    However, if the clinical investigation is intended to evaluate the dietary supplement’s ability to diagnose, cure, mitigate, treat, or prevent a disease, an IND is required under 21 CFR 312.
    See FDA Guidance on Determining Whether Human Research Studies Can Be Conducted Without an IND.

  5. I am the sponsor. Can I charge for an investigational drug?

    A sponsor can only recover the direct costs of making a drug available to subjects in a clinical trial — that is, those costs that are specifically and exclusively attributable to providing the drug to clinical trial subjects (21 CFR 312.8(d)(1)). These include costs to manufacture the drug in the quantity needed to conduct the clinical trial for which charging has been authorized or costs to acquire the drug from another source, including costs to ship and handle (e.g., store) the drug.
    Under 21 CFR 312.8(d)(3), to support its calculation of recoverable costs, a sponsor must provide documentation to the FDA describing recovery of direct costs and, if applicable, describing certain additional costs that may be recovered for intermediate-size patient population expanded access uses or treatment INDs or protocols. This documentation must be accompanied by a statement that an independent, certified public accountant has reviewed and approved the calculations (21 CFR 312.8(d)(3)).
    Once an authorization to charge has been obtained from the FDA, it needs to be attached to the eIRB application for the respective study. The consent form should reflect that the sponsor(-investigator) intends to charge for the drug.
    See FDA Guidance on Charging for Investigational Drugs Under an IND — Questions and Answers.

  6. What is expanded access and how is it different from clinical trials?

    Expanded access refers to the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient’s disease or condition.
    The terms expanded access, access, and treatment use are used interchangeably. The terms compassionate use and preapproval access are also occasionally used in the context of the use of an investigational drug to treat a patient. Although these terms have been used informally in the United States and are used outside the United States, they are not defined or described in FDA regulations.
    The main distinction between expanded access and the use of an investigational drug in the usual studies covered under an IND is that expanded access uses are not primarily intended to obtain information about the safety or effectiveness of a drug. Expanded access to an investigational drug can only be provided under a treatment IND or protocol if the sponsor is actively pursuing, with due diligence, marketing approval of the drug for the expanded access use.
    Under FDA’s current regulations, there are three categories of expanded access:

    • Expanded access for individual patients, including for emergency use (21 CFR 312.310).
    • Expanded access for intermediate-size patient populations (generally smaller than those typical of a treatment IND or treatment protocol — a treatment protocol is submitted as a protocol to an existing IND by the sponsor of the existing IND) (21 CFR 312.315).
    • Expanded access for widespread treatment use through a treatment IND or treatment protocol (designed for use in larger patient populations) (21 CFR 312.320).

    See FDA Guidance on Expanded Access to Investigational Drugs for Treatment Use — Questions and Answers.

  7. I want to treat one of my patients with an investigational drug. I need to do this as soon as possible. Do I need to submit to the IRB or FDA?

    Except for emergency expanded access use when there is not sufficient time to secure prospective IRB review, an investigator treating a patient with an investigational drug under expanded access is responsible for obtaining IRB review and approval consistent with 21 CFR part 56 before treatment with the investigational drug may begin.
    Generally, the investigator needs to contact the IRB to find out whether the IRB has sufficient time to review. If there is insufficient time for the IRB to review, FDA authorization is still required (21 CFR 312.310(d)), but it is not necessary to wait for IRB approval to begin treatment. However, the IRB must be notified of the emergency expanded access use within 5 working days of emergency use (21 CFR 56.104(c)).
    For further guidance, go to Single Patient Expanded Access or Emergency Use and contact CHOP’s IND/IDE Support Program.
    Also see the FDA Guidance on Expanded Access to Investigational Drugs for Treatment Use — Questions and Answers.

  8. Do I need to obtain consent from a subject who receives an investigational drug through expanded access?

    Expanded access to an investigational drug for treatment use, including emergency use, requires informed consent as described in 21 CFR part 50. Investigators treating a patient(s) with an investigational drug under expanded access are responsible for ensuring that the informed consent requirements of part 50 are met (21 CFR 312.305(c)(4)). One of the purposes of informed consent is to ensure that the patient is informed that he/she will be treated with an investigational product and that there may be uncertainty about the safety and effectiveness of the product.
    See FDA Guidance on Expanded Access to Investigational Drugs for Treatment Use — Questions and Answers.

  9. I am using a placebo in a clinical investigation. Does the placebo need an IND?

    Usually not.
    If the placebo consists of inactive ingredients (e.g. excipients considered to be generally recognized as safe (GRAS)), an IND is not required. A clinical investigation involving use of a placebo is exempt from the requirements of this part if the investigation does not otherwise require submission of an IND (21 CFR 312.2(b)(5)).
    See FDA Guidance on Exploratory IND Studies.