Families frustrated over not being given access to epilepsy medication

Families calling for a greater access to a medication that helps manage a severe form of epilepsy have been left frustrated and disappointed after being told that it has not been recommended for addition to the Pharmaceutical Benefits Scheme. Brooke Wrigley, mother-of-three of three, said her youngest daughter, Everly, was just four months old when she had her first seizure. Now she is two, and Ms Wrigley estimates Everly has likely had thousands of seizures. The bright and lively toddler has Dravet syndrome, a severe and rare form of epilepsy that starts in childhood and has no known cure. Everly's seizures can be so severe that she stops breathing and turns blue. Her seizure lasted about two hours and lasted two minutes. Despite Everly having six different anti-epileptic drugs every 12 hours, there was little to no control over her seizure episodes. When everly was prescribed the medication fenfluramine, as part of a free clinical trial through Melbourne's Austin Health hospital, it was like a miracle. Wrigley said Everly sank 90 days without a seizure. In contrast, fenfluramine is prohibitively expensive and costs $10,000 every 60 days, putting it out of reach of most patients. Why are so many families hoping that it will be added to the PBS? Paediatric neurologist Ingrid Scheffer has been leading fenfluramine trials at Melbourne's Austin hospital, saying it would be a big step forward for Australian patients with Dravet syndrome if the drug was added to the PBS. It might go from having two or three convulsive seizures a week to one in six months, or maybe even none, which is what we're all aiming for. She said most patients were cost-prohibitive, which was harrowing for families. I have had one family who've mortgaged their house to try to get access to the medication, Dr. Scheffer said. I've got other ones where the grandparents have helped. Dravet syndrome has a 17. percent mortality rate by 20 years of age, but Scheffer said the death risk might be reduced with greater control of seizures. My parents were afraid of it, and that's exactly what happened. The parents stayed up at night and then fell asleep in the middle of the night, and the child died within half an hour. The effectiveness of fenfluramine in reducing seizures in people with Dravet syndrome has been increasing in recent studies. In 2019, an Indian study found that fenfluramine reduced seizures by 54 per cent, while a study out of Belgium and the United States the same year found a 74.9 per cent decrease in seizures. The U.S. Food and Drug Administration approved fenfluramine in 2020 to treat Dravet syndrome in patients as young as two. As of today, Wrigley's petition calling for the federal government to add fenfluramine to the PBS has drew more than 12,000 signatures. The Federal Health Minister Mark Butler responded to the petition and said the government could not list a new medicine on the PBS unless recommended by the Pharmaceutical Benefits Advisory Committee. The PBAC has not received any applications seeking the listing of fenfluramine on the PBS for Dravet syndrome, the two-page response reads. In the PBS, medicines are not generally made available to treat conditions for which they have not been registered by the Therapeutic Goods Administration in the Australian register of Therapeutic Goods. There are no fenfluramine medicines registered in the ARTG. The health minister's response to the petition was disappointing and she will keep pushing until fenfluramine is approved, Ms Wrigley said. It shouldn't be so hard, she said. If you have to jump through hoops to get it on the PBS, you have to depend on the hospital system to provide it. Wrigley said children living with Dravet syndrome and their families needed the government to support them. It affects the entire family, she said. It's not a clear route of how families can get this medicine without having to spend the expensive $10,000 every 60 days. She said she knew of families considering moving overseas to access fenfluramine. She cried with relief when Everly was selected to test fenfluramine on a week-long basis, Ms Wrigley said. Her daughter's seizures were reduced just two weeks after starting the trial, she said. Everly's seizures improved as time went on, he said. We were averaging one every two days, and the ambulance was coming to our house three or four times a week, she said. It has been a relief for her family and had a huge effect on Everly, who will still be able to take the medication under the trial until October. She's been trying to stand and take steps now that she has fewer seizures.