An FDA advisory panel recommended on Thursday that the federal government approve a nearly-injection option for pediatric patients with epilepsy that involves a 10 milliliter vial of the muscle-relaxing drug Phenergan.
The group’s unanimous vote backed the request of the American Epilepsy Society and followed months of intense lobbying on Capitol Hill.
The thought of the FDA approving the needle-free injection of this so-called adult drug, which has a high public health profile, has shaken many people who have learned their children have epilepsy or another childhood seizure disorder.
The future of Epidiolex, the new drug now before the FDA, has sparked intense debate here. For years, many have felt that such a life-changing medication is not the best use of taxpayer dollars. While children in particular have been able to benefit, advocates for those with the disorder argue that the price has only gotten higher.
There are as many as 4.5 million Americans with epilepsy who require medication to help control seizures, according to the Epilepsy Foundation. Some medications, such as Tylenol, use potent drugs to offset the euphoric effects of seizure drugs. Some other treatments simply start with the drugs. But many can cause massive, prolonged and frequently tragic seizures.
Epidiolex can be used to deliver a liquid, either by mouth or pill, directly into the brain. Instead of administering an injection, this drug is administered by a syringe to the head of the afflicted child, or in the case of severe epilepsy, to the eye.
Phenergan, like many popular seizures medicines, is the most affordable option. Doctors can pay $100 for a bottle and up. Patients use ointments for less often, paying about $30 to $40 for a tablet. A more potent medication, with no appetite suppressant effect, is more expensive. It may cost up to $1,000 for two syringes and the whole bottle of ointments and medication, according to studies.
But when price isn’t a factor, it is not clear why some pills and injections would seem easier or safer for children with epilepsy. The drug’s approval does not mean it is safe, or should be mandated. But it was considered a positive development for that smaller group of children with less severe types of the disorder.
“It takes a lot of muscle for kids to cough or swallow a liquid, so it does mean that children have to be much more careful,” said Donald Ebert, director of the University of Washington Epilepsy Center. “This is still a very medication, highly-concentrated epilepsy medicine.”
But he also stressed the risks that still accompany it. “You still have a child who is going to have some of the major depressive symptoms, emotional difficulties, anxiety that we all have,” he said. “It’s just not the standard of care anymore.”
Dr. Paul C. Rosenthal, a pediatric neurologist at Johns Hopkins Children’s Center, said that new drugs have improved by leaps and bounds over previous generation of medications. But Rosenthal said children with epilepsy should receive the most effective treatment that a doctor determines to be effective.
“We are happy to have another treatment,” he said. “That said, it still does have risks and side effects.”
Parents of patients have been the most vocal. Members of the House are looking at a 60-day study to see how the medicine fits in a “transitory” medicine package.
“The Board is committed to ensuring access to the medicines families need, even if they make high-priced drugs like this difficult to find,” the FDA said. “We remain committed to continuing to work with patients, parents, prescribers, and payers to enhance access.”
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