Welcome to our new website! If this is the first time you are logging in on the new site, you will need to reset your password. Please contact us at email@example.com if you need assistance.
Your membership opens the door to free learning resources on demand. Check out the Member Knowledge Center for free webcasts, publications and online courses.
Our new book is a comprehensive look at a vital part of medicines development and regulatory affairs. Grab your copy today!
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
| 26 March 2014 | By Alexander Gaffney, RAC,
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?
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:
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.
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:
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
Tags: 21 CFR 312, INDs, Expanded Access, Compassionate Use, Protocols, Latest News