Explaining the numbers behind Idaho’s expanded access drug program for severe pediatric epilepsy

Dr. Christine Hahn M.D., the Idaho Department of Health and Welfare’s Public Health Medical Director, recently reported to the State Health Care Task Force on development of an Idaho program to allow children with severe epileptic seizures to receive an experimental drug derived from the cannabis plant, called Epidiolex.

The newly expanded access program often is referred to as a “compassionate use” program. It is regulated by the U.S. Food and Drug Administration, or FDA, which limits participation. Idaho is allowed up to 25 children for its program.

Dr. Hahn said Idaho does not track Idaho children with severe epileptic seizures so it is difficult to tell how many might be eligible to apply for the program. The number of children with intractable epilepsy was put at 1,500 repeatedly in testimony during legislative hearings last session.

However, beyond intractable epilepsy, the expanded access program requires that participating children meet other conditions, including Idaho residency and a lack of success after trying at least several other medications. Dr. Hahn said all those criteria make estimating the number of children who might apply almost impossible.

Utah also initially estimated up to 1,500 children there had intractable epilepsy when it passed legislation in 2014 to allow parents of children with intractable epilepsy to register for use of cannabidiol. However, only 84 parents had registered for the Utah program by June 2015.

The FDA limits the number of patients eligible for expanded access programs because, in the agency’s view, patients or parents likely would choose that process over participating in a clinical trial such as the one now under way on the use of Epidiolex for epilepsy patients. In an expanded-access program, all patients receive the experimental drug. In a clinical trial, some patients receive the drug, but others are administered a placebo.

The FDA limit protects the validity of the clinical trial, which is extremely important in determining the drug’s efficacy and its possible licensing for all patients with intractable epilepsy.

In Idaho, our expanded access program is still being developed. If it results in an outpouring of applicants, Health and Welfare will approach the pharmaceutical company, the FDA and our legislators about expanding the program. Check here again for any updates.

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