Expanded access

Expanded access (also known as compassionate use or "pre-approval access") refers to the use of an investigational new drug (IND) outside of a clinical trial by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress. Outside the US, such access is allowed through Named patient programs. In the US this type of access may be available, in accordance with United States Food and Drug Administration (FDA) regulations, when it is determined that people may benefit from the treatment, the therapy can be given safely outside the clinical trial setting, no other alternative therapy is available, and the drug developer agrees to provide access to the drug. The FDA refers to such a program as an expanded access program (EAP). EAPs can be leveraged in a wide range of therapeutic areas including HIV/AIDS and other infectious diseases, cancer, rare diseases, and cardiovascular diseases, to name a few..

There are several types of EAPs allowed in the United States. Treatment protocols and treatment INDs provide large numbers of people access to investigational drugs. A single-patient IND is a request from a physician to the FDA that an individual be allowed access to an investigational drug on an emergency or compassionate use basis. When the FDA receives a significant number of requests (~10 to 100) for individual expanded access to an investigational drug for the same use, they may ask the trial sponsor to consolidate these requests, creating an intermediate-size group. “Compassionate use” is a more colloquial term that is not generally used by the FDA. Individuals and their physicians can apply for use of an investigational drug using FDA Form 3926, an application form created by the agency in June 2016.

Since 1987, the FDA has had rules in place that have enabled people, under specific circumstances, to access drugs or biologics that are still in development for treatment purposes. These expanded access program rules were amended in 2009 by the FDA to ensure “broad and equitable access to investigational drugs for treatment.”

The regulations include the following:

The regulations also include general criteria for granting expanded access:

Despite the updated regulations, debate remains over key elements of expanded access:

A number of challenges can exist when people seek access to investigational drugs:

As of September 2016, 32 states have passed right-to-try laws that permit manufacturers to provide experimental medicines to terminally ill people without US FDA authorization. Legal, medical, and bioethics scholars, including Jonathan Darrow and Arthur Caplan, have argued that these state laws have little practical significance because people can already obtain pre-approval access through the FDA's expanded access program, and because the FDA is generally not the limiting factor in obtaining pre-approval access.

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