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