Regulatory Support

The Regulatory and Medical Affairs Office (RMA) is comprised of three cores: Regulatory, Biologics/Small Molecules, and Medical.

• Regulatory Core:
  • Supports research teams throughout the life cycle of their drug and device clinical trial through preparation, submission, and maintenance of regulatory applications to the FDA.
  • Liaises between the FDA and NINDS for communications pertaining to FDA-regulated clinical trials, FDA pre-submissions and meetings (Q-Sub, pre-IND), ensuring adherence to NIH and IC policy, as well as applicable federal regulations.
  • Provides device (NSR) determinations on behalf of NINDS as Sponsor
  • Assists investigators with Individual Patient IND/Expanded Access IND services.
• Biologics/Small Molecules Core:
  • Works with research teams to increase patient access to high quality biotherapeutics through FDA-regulated gene targeted and small molecule therapy clinical trials.
• Medical Core:
  • Provides medical monitor support, including training of the Independent Medical Monitors (IMM), and medical safety oversight, including reviewing adverse and serious adverse events for drug and device clinical trials and assisting research teams with the preparation of IND safety reports.

Helpful Reference Links:

Expanded Access/Compassionate Use INDs (Single Patient, Intermediate)

Emergency IND

IND (21 CFR 312)

IDE (21 CFR 812)

NIH Vendor Qualification:

If your clinical trial is being conducted at NIH/NINDS and your study uses an investigational drug manufactured by an external vendor, the vendor must be reviewed and approved by the Sterile Products for Human Administration (SPHA) office.  Please contact the NINDS Regulatory and Medical Affairs Office for more information at ninds-sar@ninds.nih.gov.

Frequently Asked Regulatory Questions (FARQs)

1. Who should be listed on the FDA Form 1572?
   A sub-investigator, per FDA, includes any individual member of the research team who assists the investigator and makes a direct and significant contribution to the data.  In general, if an individual is directly involved in the performance of study-specific procedures that make a significant contribution to the data and/or the collection of data, that person should be listed on the 1572. Anyone who documents results or findings on a study-specific form always should be listed on the Form 1572.
   Examples:
   Research interviewers who collect data from subjects should be listed.
   Statisticians who contribute to the design & analysis of data should be listed.
   Mid-level practitioners who perform procedures required by the protocol and collect data from those procedures should be listed. 
   Infusion nurses who administer the investigational product & collect data required by the protocol should be listed.
   Specialists, such as surgeons who collect tissue, would need to be listed if performing protocol specific procedures that make a significant contribution to the data.
    
2. Research Nurses & Coordinators who perform critical study functions and make direct and significant contributions to the data should be listed.  For example, a research coordinator who recruits subjects, collects and evaluates study data, or maintains study records should be included on the 1572.
    
3. Who should be listed on the Delegation of Authority Log?
   All persons listed on Form FDA 1572 should be listed on the Delegation of Authority log.  They should also be trained on the protocol and about their responsibilities.  This training must be documented by a training log/record that includes the training topics (e.g., protocol and investigator’s brochure) and name of trainer.
    
4. Can an investigational drug be used outside of a clinical trial?
   Yes, Expanded Access regulations provide a mechanism to use or administer an investigational drug outside of a clinical trial.  FDA regulations use the term “Expanded Access” although you may also see the terms “compassionate use” or ‘"treatment use”. Expanded access for drugs permits the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition for whom there are no comparable or satisfactory alternative treatment options.
    
5. Is Emergency Use the same as Expanded Access? 
   No, they are not synonymous terms.  An Emergency Use Authorization is defined by the FDA as the use of an investigational drug or biological product with a human subject in a life-threatening situation in which no standard acceptable treatment is available and in which there is not sufficient time to obtain IRB approval.  A physician who treats a patient under emergency use provisions must justify the treatment according to strict criteria, consult with the IRB, and fulfill specific follow-up/reporting responsibilities in a timely manner, as described in detail below.
   Note: Emergency use is emergency clinical care and does not meet the DHHS definition of research. IRB agreement that a particular case meets FDA criteria for emergency use applies to the treatment of one patient only and is not the same as IRB approval to conduct a research study.
    
6. Can a physician use an unapproved device in an emergency? 
   In general, an unapproved medical device may be used only on human subjects when the device is under clinical investigation and when used by investigators participating in a clinical trial.  However, there may be circumstances under which a health care provider may wish to use an unapproved device to save the life of a patient or to prevent irreversible morbidity when there exists no other alternative therapy. For investigational devices under an IDE, the IDE regulation permits deviations from the investigational plan without prior approval when necessary to protect the life or physical well-being of a subject in an emergency.
    
7. What if the situation is not an emergency? Can a patient with a serious illness or condition have access to an investigational device outside a study?
   Yes, FDA recognizes that there are circumstances in which an investigational device is the only option available for a patient faced with a serious or life-threatening condition (hereinafter referred to as "compassionate use"). Unlike emergency use of an unapproved device discussed above, prior FDA approval is needed before compassionate use occurs. In order to obtain agency approval, the sponsor should submit an IDE supplement requesting approval for a protocol deviation in order to treat the patient.

Forms (provided upon request)

• FDA Form 3926
• Letter of Authorization
• Clinical Summary
• MedWatch Mandatory Reporting Form 3500A
• Link to Clinical Trials
• Link to CTU SOPs
• IND Training
• NSR Training