Some turn to clinical trials to access breakthrough drugs
By Cindy Krischer Goodman | cgoodman@sunsentinel.com | South Florida Sun Sentinel
UPDATED: December 11, 2024 at 8:00 AM EST
The federal Food & Drug Administration has approved two new Alzheimer’s medications that can offer people more time to live normally, but accessing them in Florida is complicated.
Hold-ups related to insurance coverage, diagnostic test requirements, eligibility exclusions, and long waits for neurologist appointments can become stumbling blocks for whether and how quickly someone can get the medications.
The first step is to get a PET scan or spinal tap to confirm an early stage of Alzheimer’s, which could require seeing a primary care physician, neurologist, and neuropsychiatric and playing the waiting game for an appointment. After a diagnosis, a patient must meet eligibility requirements for the newly approved monoclonal antibody infusions for slowing memory and thinking loss.
The FDA approved Lecanemab (Leqembi) in July 2023. Its main risks are brain swelling and bleeding, so people who take blood thinners or carry a gene that puts them at risk for bleeds are advised against using it. This summer, the FDA approved Kisunla (Donanemab), adding another treatment option for the devastating disease. Donanemab carries a similar risk for brain bleeding and swelling.
“Once you get the diagnosis, it’s like going through a maze,” said Keith Gibson, research champion for the Florida Alzheimer’s Association. “The aim becomes to improve or maintain the quality of life for someone living with it.”
How much are you willing to pay?
Cost is one of the biggest obstacles for Floridians who want these new drugs. Leqembi carries a price tag of $26,500 a year. Kisunla is more expensive at $32,000 a year, but the amount can vary by how long someone stays on the drug. With Kisunla, treatment stops once amyloid plaques are reduced to a certain level. Regular MRI scans to monitor for side effects can also be costly if not covered.
Medicare will cover 80% of the cost for patients who meet the strict eligibility requirements. Patients, however, could still be on the hook for between $5,000 and $6,600 in co-payments. Medicare Advantage and commercial insurers are not required to cover the cost of the monoclonal antibody infusions for Alzheimer’s.
John Miller of Singer Island says he is fighting with his Medicare Advantage provider to get it to cover the cost of Leqembi for his wife, Cheryl, who participated in the drugmaker’s clinical trial.
“If she was doing good on it and the drug was approved, why won’t insurance cover it?” Miller said. “Why won’t the drug companies even give me a discount since she was a guinea pig for them? Why not help the ones who helped you?”
Miller said he called President Biden’s office to express his frustration. He did not get a response.
Gibson said he often hears similar complaints from frustrated Floridians.
“This is where people need to pay attention to their Medicare plans,” he said. “If you run into a situation with a plan that won’t cover the cost, I would encourage you to enroll in a clinical trial.”
Clinical trials offer access to drugs at no cost
Florida’s aging and diverse population attracts drug manufacturers with new therapies in research stages.The state has the second-highest number of Alzheimer’s patients over 65 after California.
In Miami-Dade, Broward, and Palm Beach counties, clinical trials are underway at private research centers, specialized hospital clinics, and local public and private universities. Most drug research focuses on ways to treat the disease as early as possible when a person has only mild symptoms or even before any emerge, particularly if they have a genetic predisposition to the disease.
At any given time, Dr. Mark Goldstein at JEM Research in Atlantis has patients enrolled in dozens of trials for Alzheimer’s or dementia. Some trials last up to five years, while others wrap up in six months.
“The length can’t always be predicted. We may need to wait until a certain number of subjects are entered before we have enough to determine whether the patients who got the medications do better than those who got the placebo,” he said. “Some studies go into an extended phase where all participants receive the drug.”
Goldstein said researchers screen potential participants to determine whether a clinical trial is an option and, if so, which is the best fit.
His center is currently conducting trials for a medication to prevent Alzheimer’s in those with a genetic disposition. JEM is also conducting a trial for an Alzheimer’s vaccine but is no longer enrolling participants.
The advancements in Alzheimer’s therapies can’t come fast enough: An estimated 6.7 million people in the U.S. ages 65 and older are living with Alzheimer’s, according to the Alzheimer’s Association. The association projects that number to double by 2060.
Getting into a trial doesn’t guarantee someone will get a treatment that works or even a medication at all. For participants, the trials are a gamble; those with placebos may continue to see their disease progress. Those who experience a slowdown in disease progression can lose access to the medication when the trial ends.
The future of drug trials
Going forward, Goldstein said participants in Alzheimer’s treatment trials will most likely get an approved drug plus the new one. “We know one drug has some effectiveness, so we will look at whether adding another would be better or cause more problems.”
At least two trials underway in South Florida are examining whether semaglutide (the ingredient in Ozempic) can improve memory and thinking abilities in individuals with Alzheimer’s disease. One drug company is even studying medication that would reverse symptoms in later-stage patients.
For those interested, the Florida Alzheimer’s Association can help match patients with ongoing trials.
“We have amazing research going on in our own backyard,” Gibson said. “All the most promising studies pretty much have a tie to Florida.”
Got comment, tips or experiences to share? South Florida Sun Sentinel health reporter Cindy Goodman can be reached at cgoodman@sunsentinel.com.
Originally Published: December 11, 2024 at 7:30 AM EST