Idaho’s Epidiolex Expanded Access Program hit a new double-digit milestone recently. 15 children are now enrolled and taking part in the program, and another 7 are scheduled for screenings.
A little history…
The governor’s office authorized the establishment of an FDA-approved Epidiolex Expanded Access program for children with treatment-resistant epilepsy in April 2015 and directed the Department of Health and Welfare to oversee it. So we’ve been working with a doctor, the drug company, FDA and the Drug Enforcement Agency to create the program and get it up and running. Dr. Robert Wechsler, who is running the program, began screening and enrolling children in December.
What is Epidiolex? Are the kids in this program getting high?
Epidiolex is purified oil from the marijuana plant that has been approved for study by the FDA. The THC, or the chemical that produces the high that pot is known for, has been removed, so they are not getting high.
What are the requirements for children to be enrolled?
The criteria to participate is strict, by design. This program is for children with severe epilepsy who are not doing well after having tried several medications and treatment protocols. We have room for 25 children in the program.
Why only 25?
The number of children we can serve is limited by the FDA and GW Pharmaceuticals, the drug company that makes Epidiolex. The FDA sets a total limit of the amount of drug that can be distributed for compassionate use programs like this one. The purpose of the strict limit is that the drug should not be widely used before it can be shown to be safe and effective. The drug company is allowing 25 Idaho children to participate in our program. So far, based on referrals from pediatric neurologists who treat children with severe epilepsy, the doctor has been able to evaluate all referrals for inclusion.
What happens if you have more than 25 eligible children?
If we have more than 25 eligible children, we will ask the drug company and the FDA for more spots, but it’s important to remember that this is a drug that is being studied. We don’t know yet what the effects will be. There is a risk involved in doing this study, and we want to minimize that risk as much as possible.
What happens next?
The program will continue until the drug is made available by prescription, or the drug is pulled by the manufacturer from further study, for example, if it doesn’t appear to be effective. All data collected will be shared with GW Pharmaceuticals and the FDA. Assuming it proves to be safe and effective, it can still take years to get a drug through clinical trials and approved for production. But we are hopeful that if we do get good results, that timeline will go fast.
Any initial outcomes or impressions of how the drug is working?
It’s really too early to tell. We hope to know more in a few months.
- Idaho’s Expanded Access Program