Posted 19 June 2014
By Alexander Gaffney, RAC
Three Republican legislators are pressing the US Food and Drug Administration (FDA) to provide them with information about its application of its expanded access regulations, saying they are in the midst of performing "oversight" over the program.
FDA's expanded access regulations, sometimes referred to as its "compassionate use" program, allow patients to access clinical trials they otherwise might not be eligible for.
The program is meant to offer hope and perhaps a treatment of last resort for desperate patients who are otherwise out of treatment options and are running out of time.
While companies must ultimately decide whether to allow a patient to enroll in a study, and hold absolute veto power over the process, patients must also obtain FDA approval to enroll in the study through the submission of an expanded access investigational new drug (IND) application.
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).
For more—much more—on the expanded access program, please see our extensive Regulatory Explainer on the subject.
The use of the program has received extensive media attention in recent years, as increasing numbers of desperate patients turn to social media to pressure companies into granting access to clinical trials, and as states have sought to pass "Right to Try" laws which would allow patients to obtain access to any medicine that has successfully passed a Phase I (safety) clinical trial and is registered with FDA.
One argument advanced by advocates of the Right to Try legislation is that FDA is a hurdle for compassionate use access, and that it is difficult to obtain approval.
FDA, in turn, has provided data indicating quite the opposite: that it approves the overwhelming majority of all expanded access requests, and has consistently done so for years.
|Expanded Access Requests Accepted by FDA|| || || || |
|Expanded Access IND|| || || || |
|Single Patient Emergency IND||313||287||442||500|
|Single Patient IND||550||496||652||484|
|Intermediate Size IND||27||14||0||2|
|Expanded Access Protocol|| || || || |
|Single Patient Emergency Protocol||2||0||3||0|
|Single Patient Protocol||62||121||89||16|
|Intermediate Size Protocol||8||8||1||5|
Data provided by FDA indicate that just 24 expanded access INDs have been declined in the four years for which it provided data (2013-2010), and 16 of those occurred in 2010.
But legislative interest in the program seems to have spiked in recent months, and now three legislators—Sens. Lamar Alexander (R-TN), Tom Coburn (R-OK) and Richard Burr (R-NC)—have all written to FDA Commissioner Margaret Hamburg, calling on her agency to answer more detailed questions about the performance of the compassionate use program.
The legislators said they wanted data on the number of requests for INDs since 2009—odd, since that information is already publicly available—but also for information on the time it took for FDA to approve or deny IND requests, the conditions for which it approved an expanded access request, and how the agency uses data collected during the treatment of compassionate use patients.
The legislators also brought up information contained within one of FDA's guidance documents, a May 2013
Question and Answerdocument which stated, in part, that the agency admitted that "other options may be needed … [to] facilitate individual patient expanded access while providing appropriate ethical oversight." At present, that oversight is provided by Institutional Review Boards (IRBs), which some have said can act as a stumbling block to patient access.
Legislators said they wanted an explanation of what options FDA was aware of, and which of those were under active consideration and when they might be implemented.
The legislators also asked Hamburg to explain when the May 2013 draft guidance might be made final, and to summarize the feedback it has received to date.
Another point raised in the letter by the senators was the agency's interaction with drug manufacturers. "Please outline any applicable steps FDA could take to minimize the risks and burdens for drug manufacturers when they supply product as part of expanded access INDs and protocols," they wrote.
FDA has been asked to respond to the letter within 30 days.
Senate Letter to FDA