In recent years, much attention has been focused on the pharmaceutical industry and its use-and non-use-of the US Food and Drug Administration's (FDA) expanded access program. But how much success has it had?

Background

That program, sometimes referred to as the "compassionate use" program, allows patients to access experimental treatments they might otherwise not be eligible to receive.

For a full explanation of the program, please see our Regulatory Explainer on the compassionate use program.

Expanded access works, in general, in one of two ways: Either a company with an experimental product creates a new clinical trial for a patient through the use of an investigational new drug application (IND), or it amends an existing clinical trial to add new types of participants through the use of a "protocol amendment."

Once a company determines which approach it wants to take, it then needs to decide on how many patients it is willing to accommodate. There are four general types of expanded access INDs and protocols:

• Single Patient (Emergency Access): Used to grant access to a single patient who does not have time to obtain written permission from FDA
• Single Patient (Regular Access): Used to allow a single patient access to a trial
• Intermediate Size: Used for intermediate-sized patient populations
• Treatment: Used for large patient populations (i.e. widespread use).

Those four types are found within both INDs and protocols, resulting in the following matrix:

More information can be found at 21 CFR 312 Subpart I.

Measures of Success

By some accounts, the success of the expanded access program can be measured by the amount of access it has provided over the years.  So how many INDs and protocols has FDA approved as of late?

According to new data released by FDA this week, it approves quite a few each year.

Some interesting observations from the data:

• FDA has approved fewer expanded access INDs in the last two years (2012-2013) than in the two years prior (2010-2011), but more expanded access protocols.
• Protocols are almost never used in emergency use cases (five times in four years). INDs, however, are used frequently for emergency use cases (more than 2,000 times in four years).
• The use of single patient protocols has increased dramatically in recent years, from just 16 in 2010 to 89, 121 and 62 in the three subsequent years.
• Treatment INDs have been sought (and granted) just once in the last four years. The preferred method of access appears to be treatment protocols, which have been sought and granted 40 times in the last four years.

And for all the flak FDA gets for its involvement as a gatekeeper in expanded access cases, it has proven remarkably willing to grant access when companies ask for it. In the last four years, it has denied a proposed IND just 24 times, and just eight times in the last three years.

It has not denied a single proposed protocol in the last four years.

FDA, it's worth noting, can only approve expanded access at the request of a company. It cannot order (though it can negotiate with or subtly pressure) a company to do so.

FDA Data on Expanded Access INDs and Protocols