1. I have an emergency situation; access needed within hours or days. Who do I call once I have manufacturer agreement?
If your patient is in a situation where access is needed in a matter of hours or days, and a company is willing and able to provide the product needed, FDA can be contacted directly through its Division of Drug Information at 855-543-3784 or email@example.com for electronic or over-the-phone application processing. To request emergency access to investigational biological products regulated by CBER, call 240-402-8010 or 800-835-4709 or firstname.lastname@example.org.
2. Can patients or physicians apply for expanded access to investigational therapies directly from the Foundation site?
3. What factors do companies consider?
4. If a company lists its expanded access policy on this site, am I guaranteed it will accept my request?
5. If approved, is expanded access free to patients?
6. Are physicians compensated for the time involved in making a request?
7. I’m not eligible for a clinical trial of an investigational therapy, are there other options for expanded access?
8. Are companies required to offer Single-Patient Expanded Access?
9. Does FDA have the final say?
10. Identifying a rare disease often takes years; can I apply for expanded access without a diagnosis?
For single patient expanded access beyond trials, a criterion commonly required by companies is that the patient has exhausted standard treatments. While this may be difficult to demonstrate without a clear diagnosis, it is not impossible. Start with ClinicalTrials.gov and read the expanded access policies voluntarily supplied by companies on the Navigator’s Directory.
11. What counts as a serious or life-threatening disease, especially if it's rare or undiagnosed?
This is an important topic to discuss with your referring or treating physician because to apply for expanded access your disease or condition must be either serious or life-threatening. “Serious” is a clinical judgement based on many factors but can be summarized as having a substantial impact on day-to-day functioning. “Life-threatening” means there is a reasonable likelihood of death within months or prematurely without early treatment.
12. A majority rare diseases, 80%, are genetic; can I apply for expanded access for investigational gene therapies?
Yes, gene therapies and other biologics are types of investigational treatments available for patients with serious or life-threatening conditions, who are unable to participate in clinical trials and have exhausted other treatment options.
13. I want a drug that's been taken off the market for safety reasons; can I get it through expanded access?
According to FDA guidance updated in 2017, expanded access may also refer to (1) use in situations when a drug has been withdrawn for safety reasons, but there is a patient population for whom the benefits of the withdrawn drug continue to outweigh the risks; (2) use of a similar, but unapproved drug (e.g., foreign-approved drug product) to provide treatment during a shortage of the approved drug; (3) use of an approved drug where availability is limited by a risk evaluation and mitigation strategy (REMS) by patients who cannot obtain the drug under the REMS; or (4) use for other reasons. Expanded access is sometimes requested in other situations, for example when an approved drug exists for the adult population but is requested for pediatric use. Drug company approval of expanded access requests for approved drugs is less common than for investigational therapies and costs and reimbursements vary.
14. I want a drug that is already on the market but not approved for my disease; can I get it through EA?
The Navigator is designed to focus on expanded access – access to drugs not yet approved by FDA. This website does not provide guidance on access to “off-label” treatments, because only your physician can provide medical guidance about uses that are not approved by FDA. Your physician can prescribe medications that the physician and patient agree are appropriate, even if that use is “off-label." Patients and physicians must make these decisions based on each medical situation, taking account of all relevant factors for individual patients.