MCNA will be approved as Investigational Drug What is compassionate drug use?
Medical professionals use the term “compassionate use” to refer to the treatment of a seriously ill patient using a new, unapproved drug 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 generally available only to people who are taking part in a clinical trial (a research study that is testing the drug). Being able to use one of these drugs when you are not in a clinical trial has many names, but is most commonly referred to as compassionate use.
Is compassionate drug use legal?
Compassionate drug use is legal, but it’s tightly restricted to people who meet certain conditions. The FDA first approved investigational drugs to be used in this way for critically ill patients in 1987. For people who aren’t in clinical trials, there are 2 ways a drug company can allow use of their unapproved drug: through expanded access programs (EAPs) or through single patient access.
Expanded access
A company sponsoring a drug in the late stages of drug development, such as Phase III clinical trials, can offer expanded access programs for patients who are not able to enroll in a clinical trial. (See Clinical Trials: What You Need to Know to learn more about the phases of clinical trials.)
The FDA generally approves these EAPs if the drug has shown that it works at least somewhat to treat cancer in the clinical trials that are being done. This can allow a lot of people access to the unapproved drug, as long as they meet the requirements of the EAP.
Single patient access
Patients who don’t qualify for either clinical trials or an expanded access program (if one exists) may be able to get the unapproved new drug by applying for single patient access.
In this case, the patient’s doctor must first ask the drug company if the drug can be used for the patient and see if the drug company will supply it. If the company agrees, the patient’s doctor works with the drug company to ask the FDA to approve the drug for use by this one patient.
The FDA requires the doctor to send information about the patient, why the request is being made, the proposed treatment plan, and a signed informed consent from the patient (see our document Informed Consent for more on this). The length of time it takes to get single patient access varies. But if it’s an emergency, the FDA can complete the paperwork in 24 hours.
Who might benefit from using unapproved drugs?
According to guidelines from the National Cancer Institute, most compassionate drug use is for patients who meet all of these conditions:
- Have advanced disease
- Have used standard treatments and they have not worked
- Are not eligible for any clinical trial that’s in progress
- Have no other treatment options
- Have a type of cancer for which there’s reason to expect the investigational drug will help
- Are likely to have benefits that outweigh the risks involved
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