Posted 23 January 2015
By Alexander Gaffney, RAC
Last year, the US Food and Drug Administration (FDA) received—and approved—substantially more requests for "compassionate" access to potentially life-saving treatments than any time since at least 2010, according to new data provided by the regulator.
The data relate to FDA's "expanded access" program—sometimes called its "compassionate use" program—which was created to allow a pathway for terminally or critically ill patients to obtain access to drugs they would otherwise not be eligible to receive.
Only patients with serious or immediately life-threatening diseases with no comparable or satisfactory therapeutic alternatives are eligible to receive a product through FDA's expanded access program. Even then, the company that produces the drug they wish to access must agree to provide the drug to the patient and obtain FDA approval under one of several types of clinical trial applications, known as investigational new drug applications (INDs).
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 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:
|Expanded Access IND||Expanded Access Protocol|
|Single Patient Emergency IND||Single Patient Emergency Protocol|
|Single Patient IND||Single Patient Protocol|
|Intermediate Size IND||Intermediate Size Protocol|
|Treatment IND||Treatment Protocol|
More information can be found at 21 CFR 312 Subpart I.
While FDA cannot compel a company to give a patient an experimental drug, it is required to sign off on all requests to obtain experimental treatments through a clinical trial to ensure the patient understands the probable risks of the trial, and that the trial is more likely to help than harm the patient.
For a thorough explanation of FDA's expanded access/compassionate use programs, please see our comprehensive explainer article here.
FDA Approvals at All-Time-High
In fiscal year 2014, FDA approved far more of those requests than the year prior, or any year since the agency began publishing statistics on expanded access request approvals in 2010.
A complete breakdown of FDA's approvals is as follows:
|Expanded Access Requests Accepted by FDA|
|Expanded Access IND|
|Single Patient Emergency IND||500||442||287||313||1066|
|Single Patient IND||484||652||496||550||692|
|Intermediate Size IND||2||0||14||27||50|
|Expanded Access Protocol|
|Single Patient Emergency Protocol||0||3||0||2||9|
|Single Patient Protocol||16||89||121||62||35|
|Intermediate Size Protocol||5||1||8||8||9|
FDA's data show the overwhelming increase in its applications came from single patients seeking access through Single Patient Emergency INDs, which increased from 313 in 2013 to 1,066 in 2014—more than a 300% increase, and more than twice the previous record in 2010. Both Single Patient INDs and Intermediate Size INDs also saw large proportionate increases as well.
Rejections Remain Low
FDA's data also show the agency to reject relatively few expanded access applications each year.
|Expanded Access Requests Rejected by FDA|
|Expanded Access IND|
|Single Patient Emergency IND||16||1||2||2||3|
|Single Patient IND|| || ||2|| ||4|
|Intermediate Size IND|| || || ||1||2|
|Treatment IND|| || || || || |
The agency has not denied an expanded access protocol since it began keeping records in 2010.
FDA's new data comes just as many states are trying to make it easier for patients to obtain compassionate use access to drugs. Five states have since enacted "right to try" provisions, and more than a dozen others have introduced similar pieces of legislation.