When reviewing drugs for expanded access, IRBs face four challenges:
- To speed up the review process for patients who truly are almost out of time.
- To ensure that the experimental medicine will not cause undue harm to the patients involved.
- To ensure that patients make informed decisions about using the experimental medicine.
- To avoid jeopardizing drug approval for future.
WCGF has identified the following four-step process to support IRBs and desperately ill patients. A PDF version can be downloaded here.
Streamlining Procedures for Single Patient Expanded Access Requests Involving Investigational New Drugs
- FDA Form 3926
- Investigator brochure or another source describing risks and potential benefits of the treatment
- Draft consent document
- As of October 3, 2017, FDA permits the IRB chair or designee to review and concur with the expanded access request.
- Confirm the medical evaluation of the patient’s condition based on the information provided by the treating physician. This individual must be able to confirm or deny the claim that there is no comparable or satisfactory alternative available.
- Ensure that informed consent or appropriate permissions will be obtained and documented. Given the compassionate nature of the request and FDA’s involvement, consent documents should meet the requirements listed in 21 CFR 50.25, using plain language that is specifically aimed at “patients” who expect direct benefit, as opposed to “subjects” who may not expect benefit.
- Receive documentation that FDA has made its determinations regarding safety and effectiveness and has given clearance for the use. Request the IND number and any documentation from FDA.
- Review the physician’s treatment plan (or sponsor-provided treatment protocol) and determine that it makes adequate provision for ensuring the safety of the patient including adequate monitoring (timing and type of tests/exams, etc.) and appropriate plans for collecting and reporting the data.
The treating physician is the link between desperately ill patients and three key players in the expanded access process: the drug manufacturer, the Food and Drug Administration (FDA), and the institutional review board (IRB). Once approval is obtained, the physician also administers the experimental medicine and monitors the patient’s response.
Because many physicians are not familiar with the process, WCGF has summed it up below. A PDF version of the information can be downloaded here.
Responsibilities of a Sponsor-Investigator (Treating Physician):
- Apply to and obtain approval from FDA and an IRB prior to administering the investigational product.
- Seek IRB approval for continuing review if the treatment use extends longer than one year or a second dose is needed.
- Obtain and document appropriate informed consent from the patient or legally authorized representative prior to treatment.
- Maintain accurate case history records and observations related to provision of product, including adverse events.
- Report adverse events as required by FDA.
- Maintain accurate documentation of the disposition of investigational product, including dates, quantity, and use.
- Adhere to reporting obligations of IRB, FDA, and sponsor.
- Prepare and send summary report of treatment use to sponsor and FDA.
- Maintain confidentiality of the information both about the patient and the condition.
- Comply with applicable local laws and institutional policies.
Our IRB Partners
Below is a list of organizations whose IRBs are working with WCGF to streamline IRB review procedures. Since organizations continue to sign on, please check back frequently for the most recent list.
- University of Colorado Denver
- University of Pennsylvania
- University of Southern California
- UT Health, University of Texas Health Science Center at Houston
- Vanderbilt University Medical Center
In addition, WIRB, an independent IRB, has agreed to help support expanded access by providing IRB reviews free of charge in cases where the physician is not affiliated with an organization that has its own IRB.
We encourage all IRBs to join WCGF in transforming the review process so that every patient request for expanded access is approved quickly. Together, we can make a difficult time a little easier for desperately ill patients and their families.
For more information, including assistance with your IRB procedures, please contact WCGF at email@example.com or call 404.386.8982.
- Streamlining IRB Procedures
- Responsibilities of a Sponsor-Investigator (Treating Physician)
- FDA Guidance: Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers (October 2017)
- FDA Form 3926 *Right-click and select “save link” to save the file to your computer)
- FDA website
- Reagan-Udall Foundation Expanded Access Navigator