Compassionate drug use means making a new, unapproved drug available to treat a seriously ill patient when no other treatments are available. People who aren’t enrolled in a clinical trial may get access to an unapproved drug through mechanisms of compassionate use, expanded access programs (EAPs) or the Right to Try Act.¹

In 1987 the FDA released Investigational New Drugs regulations which established an expanded access program by implicitly acknowledging treatment use in individual patients and adding a provision for obtaining an investigational drug for treatment use in an emergency situation. However, said provision did not describe criteria or requirements that must be met to authorize individual patient treatment use.²

In the Food and Drug Administration Modernization Act of 1997, Congress included specific provisions concerning expanded access to investigational drugs for treatment use.²

The Right to Try Act is a federal law passed in 2018 that gave patients another avenue to access unapproved drugs, without needing the approval of the FDA.¹

Drugs that are being tested but have not yet been approved by FDA are investigational drugs, which are normally, only available to people who are taking part in a clinical trial. Being able to use investigational drugs when you are not in a clinical trial is vital and potentially lifesaving for patients who are terminally ill.

The FDA regulates EAPs and accepts requests for expanded access every day. An expanded access request is made through a doctor and the FDA usually takes 4 days to process a non-emergency request, and less than one day for an emergency request. EAPs are for patients who have a serious and life-threatening condition, who are ineligible for any current clinical trial of the drug, have no other comparable treatment options, and whose benefit will likely outweigh the risks. ¹

Eligibility for Right to Try is based on whether a person: is diagnosed with a life-threatening disease or condition, tried all the approved treatment options for the disease or condition, had a doctor certify that they are unable to participate in a clinical trial, and gave written informed consent that they understand the risks. In addition, the drug must have already been through a phase I clinical trial. Not all drugs are available for compassionate use, and drug companies aren't required to supply their drugs through compassionate use. Each drug company has different policies and processes, but they are required to publicly post contact information for compassionate use requests.¹

The US Food and Drug Administration (FDA) granted more than 99% of requests for patients to access unapproved drugs and biologics in fiscal year 2016.³

Regardless of whether a compassionate use request for a drug or treatment is through an EAPs or the Right to Try Act, it must be made by a physician on behalf of the patient.¹

1. Compassionate Drug Use. Cancer.org. https://www.cancer.org/treatment/treatments-and-side-effects/clinical-trials/compassionate-drug-use.html.

2. Food and Drug Administration. Expanded Access To Investigational Drugs For Treatment Use. Silver Spring, MD: Food and Drug Administration; 2009.

3. Becker's Hospital Review. FDA granted 99% of applications for access to unapproved drugs in FY 2016.https://www.beckershospitalreview.com/supply-chain/fda-granted-99-of-applications-for-access-to-unapproved-drugs-in-fy-2016.html. Published 2017.