At NORD’s Rare Disease and Orphan Drug Breakthrough Summit held in Washington D.C., October 15-16, 2017, we talked with Lucas Kempf, MD, of the Center for Drug Evaluation and Research (CDER) at the US Food and Drug Administration (FDA).
What is expanded access?
Expanded access is the FDA's compassionate use program in which we allow an experimental drug that's in development to be used for treatment rather than research for patients.
What is the expanded access navigator?
The expanded access navigator is a website that's been designed by the Reagan-Udall Foundation which has links for patients and physicians to reach companies who want to release drugs for expanded access. They may not know exactly how to do that so the Navigator details how to contact the FDA, how to research what drugs may be in development for the disorder through the website clinicaltrials.gov.
Every company has to register at clinicaltrials.gov so it details how you search that for your indication. It's a been a good resource for folks who don't know where to go, it's sort of like the first step.
How does a patient qualify for expanded access?
In order to be eligible for expanded access, you first have to not qualify for any known drug treatment or current study. So you work with your physician and you fill out about a 2-page form—it takes about 45 minutes to fill it out— you can send it in and you also have to request if you know of a drug that's in development. You send a request to the company which then allows you access to the drug.
A lot of drugs in development are only made in small batches and so there may not be availability of the drug at that time. Sometimes they do make extras and they'll make it available to patients who don't fit the strict criteria for their clinical trials. Other times companies will open their own expanded access INDs – for example, the company has submitted the documents for reviewing the drug to be out on the market but there's a time lag between the period of time they submitted it and before it goes on the market. So frequently companies have an expanded access IND during that period in which they enroll patients. If you contact the FDA, we'll let you know whether or not that form is appropriate and the drug company can send you the drug safely.