The U.S. Food and Drug Administration (FDA) has proposed simplifying the procedures by which seriously ill patients can be allowed “compassionate use” of experimental drugs. The agency on February 4 issued a draft guidance document entitled Individual Patient Expanded Access Applications: Form FDA 3926, and will take public comments for 60 days.
Saying it wants to create a “streamlined alternative” to its current process of applying for what the agency calls “expanded access” to investigational drugs (which are being reviewed but have not yet been approved by the FDA). The agency’s Expanded Access Program aims to provide patients with serious or immediately life-threatening conditions for which there are no other comparable or satisfactory treatments an alternative avenue for getting access to drugs that may help.
The FDA says it already approves nearly all the expanded access applications it receives – last year, for instance, it rejected only nine of the 1,873 applications it received, and agency records show the FDA turned down just 33 (or about one-half of one percent) of the 5,995 expanded access applications it got over the last five years.
But the agency has been criticized for how complex its expanded access applications process is. The current system requires all expanded access submissions to file a summary form used for investigational new drug applications (IND 1571), which pharmaceutical firms submit when seeking FDA approval to run clinical trials for a drug under development. Since the IND 1571 is designed for drug developers and summarizes extensive information – one senior agency official says it calls for 26 distinct categories of information and seven attachments — the FDA estimates it takes 100 hours to complete.
But the overwhelming share of expanded access applications come not from drug makers trying to expand clinical trials, but from physicians filing on behalf of a single patient. In fact, 97% of expanded access applications the agency received in 2014 covered either an emergency or regular single-patient use. The FDA says it’s concerned the daunting form and its administrative burdens may be too much in such cases. In fact, unwieldy FDA expanded access procedures may have helped fuel the “right to try” movement, which has already led several states to enact laws allowing patients to take non-FDA approved drugs.
The agency’s proposed solution is to create the new Form 3926, for use by doctors seeking to gain access to an experimental drug for a single patient. The FDA says this simplified form could be completed in less than an hour, not the 100 hours for an IND 1571.
The new form would ask for eight fairly straightforward data categories. These include: the patient (for privacy reasons, only the patient’s initials are used), plus: the patient’s clinical data; treatment data on the drug and planned usage; a letter from the drug maker authorizing product use; information on the supervising physician and his or her qualifications; and an agreement that, absent an emergency triggering different procedures, the expended access treatment won’t start for 30 days – the period FDA has to review INDs).
As at present, under the new guidance a physician seeking emergency approval for expanded access could call FDA for authorization to start treatments; in such cases, a written form must be filed within 15 days of getting authorization.
Besides the new guidance, the FDA has recently created a new webpage to summarize for patients their expanded access options.