Individual (Single) Patients
Individual (single) patients who are desperately ill, have exhausted other treatment options, and are ineligible or otherwise unable to participate in a clinical trial, can request permission to try investigational (unapproved) drugs, biologics, and medical devices through two different pathways:
- Food and Drug Administration (FDA) expanded access, which is available to patients who are seriously ill or have life-threatening conditions. This process includes reviews by both FDA and an institutional review board (IRB) to help ensure that the investigational drug, biologic, or medical device does not expose the patient to undue risks. FDA usually completes its review within a few days. From 2010 through 2017, FDA approved 99 percent of individual (single) patient requests for expanded access.
- Right-to-Try, a new pathway made possible this year with the passage of federal right-to-try legislation. This pathway is available only to patients with life-threatening conditions and only applies to investigational drugs and biologics. It does not require FDA or IRB review and, therefore, does not provide the resulting extra level of protection for patients.
Under both pathways, the request must be made by a licensed physician on behalf of the patient. Also under both pathways, the product manufacturer is not required to grant the request.
Clinical Research Pathways assists patients, treating physicians, and IRBs with the expanded access process. We provide comprehensive information to help you understand and navigate expanded access:
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
- FDA Form 3926 *Right-click and select “save link” to save the file to your computer)
- 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: FDA Device Division of Industry and Consumer Education, 301-796-7100 or DICE@fda.hhs.gov