Compassionate drug use, sometimes just called “compassionate use,” is the use of new and unapproved drugs to treat a seriously ill patient when there are no other treatments available. Normally, these drugs are in clinical trials. But being able to use these drugs outside of clinical trials is known as “compassionate use.” 

While compassionate use drugs are legal, to get approved for compassionate use of a drug, there are stringent criteria that must be met. According to the Mayo Clinic, these include: 

• Your disease is serious or immediately life-threatening.
• No treatment is available or you haven't been helped by approved treatments for your disease.
• You aren't eligible for clinical trials of the experimental drug.
• Your doctor agrees that you have no other options and the experimental treatment may help you.
• Your doctor feels the benefit justifies the potential risks of the treatment.
• The company that makes the drug agrees to provide it to you.

It’s important to note the following when considering compassionate use drugs: 

• Not all experimental drugs are available for compassionate use, and there is no requirement or law that says drug companies must supply their drugs for compassionate use.
• There may be very limited amounts of a drug available for compassionate use, or drug companies might only have enough of the drug for their clinical trials.
• While some drug companies will supply a drug through compassionate use for free, others might charge patients. In addition, most insurance companies will not pay for the costs of the investigational drugs themselves. 

People who are not in clinical trials may be able to access the drugs through two other pathways: Expanded Access Programs (EAPs) or via the “Right to Try.” 

There are three types of EAPs:

• Widespread EAPs: In these cases, a company sponsors an unapproved drug in the late stages of drug development and can offer expanded access for patients who are not able to enroll in a clinical trial. These EAPs are typically managed by the company sponsoring the drug.

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• Intermediate (medium-size) EAPs: A company that sponsors an unapproved drug may offer expanded access to groups of patients during early or late stages of drug development, and usually manages the EAP.

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• Individual use EAPs: If individual patients don’t qualify for an existing clinical trial or widespread or intermediate use EAPs, they may be able to apply for access to an unapproved drug on an individual basis. In this type of expanded access, the patient's doctor must communicate with the drug company to see whether the drug may be used for the patient and whether the drug company will agree to supply it. If the company agrees, the doctor works together with the drug company to ask the FDA to approve the drug for use by this one patient. The FDA will require the doctor to send details about the patient, why the request is being made, and the proposed treatment plan, as well as obtain a signed, informed consent from the patient. 

“Right to Try” is a different avenue for compassionate use. “Right to Try” is the result of a 2018 federal law that allows patients to access unapproved drugs by bypassing the FDA. “Right to Try” laws are another pathway for compassionate use that exist alongside EAPs without replacing them. 

“Right to Try” laws do not mean that individual patients can try whatever unapproved drugs they wish; they simply mean that the individual patient can request access to an unapproved drug from the company directly, rather than involving the FDA. Bypassing the FDA does not mean an automatic approval for compassionate use. Patients may still be denied if the patient does not meet the criteria spelled out in the law or if the drug company refuses to supply the drug for any reason, including limited amounts of the drug. 

To be eligible for compassionate use drugs under “Right to Try” laws, the patient must fit the following criteria: 

• Be diagnosed with a life-threatening disease or condition
• Have tried all approved treatment options for the disease or condition
• Be certified by a doctor that they are unable to participate in a clinical trial for the investigational drug
• Give written informed consent that they understand the risks of taking the investigational drug

The drug must also already have gone through its Phase I clinical trials (these are the earliest phase of clinical trials and look at the safety of the drug and what dosage to use). 

Both EAPs and “Right to Try” require specific criteria to be met; a request must be made by a licensed doctor; drug trial sponsors may decline the request for any reason; and the cost charged to the patient is limited to the prescription drug costs themselves, though insurance may not be required to cover these costs. 

However, in the “Right to Try” pathway, FDA approval for access to the drug is not required, and there is no oversight regarding drug safety by the FDA and the treatment center’s Institutional Review Board (IRB). Also in the “Right to Try” pathway, adverse events do not need to be reported to the FDA. 

Compassionate use pathways for drug access exist to give terminally ill patients the ability to receive drugs that may prolong or even save their life. For more information, check out the FDA’s information about EAPs or ask your doctor about the “Right to Try” pathway.