Responsiveness to Emergency Access INDs

Ever since I began my career at FDA in 1985 and straight through until today, I have heard the Agency criticized for failing to respond to patients needs regarding access to new life saving treatments.   I remember being locked in the Parklawn building during one particularly lively demonstration relative to the AIDS crisis.  I remember the loud voices of cancer patients seeking access for experimental cancer treatments as their last hope.  The FDA is always in a no win situation regarding the speed of drug approvals.  Critics say if FDA makes too fast of a decision, they are killing people, and, if they go too slowly, they are killing people.

There are new initiatives being discussed relative to patient’s ability to access new and unapproved treatments for serious, life threatening conditions, but the same arguments of ethics still hang visibly on each side of the issue.  It is my view that the Agency has listened to the patients and feels their agony and frustration as well and, as a result, has become more willing to go out on a limb and risk criticism.  Over the weekend, I stumbled across something I had not seen before on the CDER web page (lots of good information there; I wish there was ample time to take it all in).  It was a link to the Expanded Access Submission Receipts Report (here).  This report identifies the type of expanded access IND type, the number of IND requests for each type, and the number of grants for the INDs.  The link has reports from years 2010 through 2013.  I was actually surprised when I saw the figures for the 2013 report (here). For FY 2013, the Agency received a total of 977 Expanded Access INDs and Protocols and allowed 974 of them to proceed, which is 99.9%.  That is awfully close to 100% and represents a lot of work for the Agency.  FDA permitted all of the 550 single patient INDs to proceed, and all but 2 of the single patient emergency use INDs to proceed.  They also disallowed only 1 of the 28 intermediate size INDs to proceed (96.4%).   To perhaps my naïve eye, that seems like one heck of a good record in terms of access of patients to new treatments.

These findings are consistent with the statistics in the other three reports for 2010-2012, with the perhaps the “worst” record (if you can call it that) for 2010 when a total of 1030 Expanded Access INDs and Protocols were received and 1014 (or 98.4%) of those were permitted.

So FDA-good work!  Of course there are stories of individual patients failing to obtain certain treatments but, remember that the FDA is there to protect the patient and assure safety and effectiveness of the treatments it permits and the products it approves.  Will the Agency always get it right? – Of course not, but the numbers seem like they are providing the American public with the best anyone can expect.