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Compassionate drug use means making a new, unapproved drug available to treat a seriously ill patient when no other treatments are available.
Drugs that are being tested but have not yet been approved by the US Food and Drug Administration (FDA) are called investigational drugs. These drugs are normally available only to people who are taking part in a clinical trial.
Being able to use one of these drugs when you are not in a clinical trial is most commonly referred to as compassionate drug use.
Compassionate drug use is legal, but it’s limited to people who meet certain conditions.
Patients with serious or life-threatening conditions who can't get treatment with an unapproved drug through a clinical trial might benefit from compassionate use, if it's available.
The usual way to get treatment with an unapproved drug is through a clinical trial. But many people with life-threatening diseases can't find clinical trials that they can be a part of. This could be because they live too far from locations where the studies are being done, or because they don't meet the requirements.
People who aren’t in clinical trials might be able to get access to an unapproved drug from the company that makes it in 2 ways:
Here are some important things to know about drugs that may be available through compassionate use:
The FDA accepts applications for expanded access every day of the week. It usually takes 4 days to process a non-emergency request, and less than one day to process an emergency request. Requests are made through your doctor. The FDA approves most of these requests, but often requires changes to the study protocol for the individual to assure safety. These may be changes in dose of a drug or changes to safety monitoring.
?In general, EAPs are for patients who meet all of these conditions:
A federal law passed in 2018 gave patients another path to access unapproved drugs, without needing the approval of the FDA. This pathway is commonly referred to as Right to Try. Right to Try laws do not replace EAPs but provide another way to access unapproved drugs.
It's important to understand that Right to Try does not actually give patients the right to try any unapproved drug they wish to try. Instead, it gives them the right to request access to an unapproved drug from the company that makes it, without having to go through the FDA.
Bypassing the FDA does not necessarily mean that such access will be granted. There are a couple of reason access may be denied, including:
Bypassing the FDA also means there may be less guidance for how the drug is being given.
To be eligible for Right to Try, a person must:
In addition, the drug itself must have already been through a phase I clinical trial. (This is the earliest phase of clinical trials, which is generally intended to start looking at the safety of the drug and the proper dose to use.)
To learn more, see
There are some differences between EAP and Right to Try pathways. Some of these might be good or bad, depending on the situation.
|
EAPs |
Right to Try |
Specific criteria are required for patients to get access to a drug being studied, such as:
|
Yes |
Yes |
FDA approval for access to a drug being studied is required. |
Yes |
No |
Request for drug access must be made by a licensed doctor. |
Yes |
Yes |
Includes some oversight about drug safety by the FDA and the treatment center’s institutional review board (IRB) |
Yes |
No |
Drug and clinical trial sponsors can decline requests for various reasons. |
Yes |
Yes |
If access is approved, insurance companies are required to pay the cost of drug and associated administration/treatment. |
No |
No |
Cost charged to a patient is limited to direct drug costs, although sometimes indirect charges to the patient are possible. |
Yes |
Yes |
Adverse events must be immediately reported to FDA. |
Yes |
No |
Here are some questions you might want to ask if you and your doctor are discussing compassionate drug use:
Your doctor or one of the office staff will work with you on this process. If you and your doctor find a drug you want to get access to through an EAP, your doctor will need to contact the drug company to see if they would be willing to provide the drug as part of your treatment. If so, the next step is to contact the FDA's Center for Drug Evaluation and Research (CDER) at 1-301-796-3400 or or the after-hours FDA Emergency Call Center at 1-866-300-4374 (toll free). See the to learn more.
For this pathway, your doctor will first need to contact the drug company to find out if you might be able to access the drug. (As a patient, you might be able to make the initial contact with the company to find out about your eligibility or if the drug is available, but the request for the drug will need to come from your doctor.) Your doctor or one of the office staff will work with you on this process. Then, your doctor will need to follow certain steps and work with you to get access if the drug is available.
The American Cancer Society medical and editorial content team
Our team is made up of doctors and oncology certified nurses with deep knowledge of cancer care as well as editors and translators with extensive experience in medical writing.
Congress.gov. S.204: Right to Try Act of 2017. https://www.congress.gov/bill/115th-congress/senate-bill/204.
Accessed August 3, 2020.
HHS.gov. About the Affordable Care Act. https://www.hhs.gov/healthcare/about-the-aca/index.html. Reviewed October 23, 2019. Accessed August 2, 2020.
Jarow JP, Lurie P, Ikenberry S, Lernery S. Overview of FDA’s expanded access program for investigational drugs. Ther Innov Regul Sci. 2017; 51(2): 177-179.
McKee AE, Markon AO, Chan-Tack KM, Lurie P. How often are drugs made available under the Food and Drug Administration’s expanded access process approved? J Clin Pharmacol. 2017;57(10):S136-S142.
U.S. Food and Drug Administration (FDA). Expanded access. https://www.fda.gov/news-events/public-health-focus/expanded-access. Updated April 27, 2020. Accessed August 3, 2020.
U.S. Food and Drug Administration (FDA). Expanded access to investigational drugs for treatment use: questions and answers. https://www.fda.gov/downloads/drugs/guidances/ucm351261.pdf. Updated October 2017. Accessed August 3, 2020.
U.S. Food and Drug Administration (FDA). Right to try. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try. Updated January 14, 2020. Accessed August 3, 2020.
Last Revised: September 1, 2020
American Cancer Society medical information is copyrighted material. For reprint requests, please see our Content Usage Policy.
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