FDA provides expanded access options that enable more than one patient to try the same investigational product for the same indication. These multiple patient programs typically involve from several patients to up to thousands, although there is no hard and fast restriction on the number, since these programs are designed to allow flexibility.
For drugs and biologics, FDA offers intermediate-size patient population and treatment INDs. Expanded access to medical devices is available through small group access and treatment IDEs. FDA refers to this type of expanded access as “compassionate use.”
It’s important to note that considerable information—such as age, medical and treatment history, organ function, and medications—is considered in making case-by-case determinations about expanded access for individual (single) patients. As more patients are provided access under an IND or IDE, it’s likely that less will be known about each individual patient. That increases the need to have more safety information about the investigational product before extending treatment exposure to more individuals.
Therefore, FDA typically only makes multiple patient expanded access options available when there is sufficient evidence that the treatment is unlikely to cause harm or injury to the patient—and there is likelihood that the product will provide some level of benefit.
There is no “emergency use” component under these multiple patient programs. In immediately life-threatening situations, when administration of the investigational product is extremely time sensitive, a single patient emergency use IND or IDE should be used.
From Clinical Research Pathways
- Intermediate-size Patient Population Expanded Access Use
- Responsibilities of a Sponsor-Investigator (Treating Physician)
- How to Request Treatment Use via FDA’s Expanded Access Process (Non-Emergency)
- Points for Patients to Consider, PDF version, printed brochure, printing instructions
- The Right-to-Try Pathway
- Streamlining IRB Procedures
- FDA Guidance: Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers (October 2017)
- FDA Guidance on IDE Policies and Procedures, (January 20, 1998)
- FDA website
- Form FDA 1571
- Form FDA 1572
- For drug-related expanded access: FDA Division of Drug Information, 301-796-3400 or firstname.lastname@example.org
- For biologics-related expanded access: FDA Biologics Office of Communication, Outreach, and Development, 800-835-4709 or email@example.com
- For medical device-related expanded access (compassionate use): FDA Device Division of Industry and Consumer Education, 301-796-7100 or DICE@fda.hhs.gov
- For emergency requests after 4:30 p.m. weekdays and on weekends, for all medical products (drugs, biologics, and medical devices) contact FDA’s Emergency Call Center at 866-300-4374.