Phil Galewitz, KFF Health News
Serenity Cole loved Christmas final month stress-free along with her household close to her St. Louis house, making crafts and visiting associates.
It was a distinction to how Cole, 18, spent a part of the 2024 vacation season. She was within the hospital — a frequent prevalence with sickle cell illness, a genetic situation that damages oxygen-carrying crimson blood cells and for years triggered debilitating ache in her legs and arms. Flare-ups usually would pressure her to cancel plans or miss faculty.
“With sickle cell it hurts every day,” she stated. “It might be more tolerable some days, but it’s a constant thing.”
In May, Cole accomplished a several-months-long gene therapy treatment that helps reprogram the physique’s stem cells to provide wholesome crimson blood cells.
She was one of many first Medicaid enrollees nationally to profit from a new payment model by which the federal authorities negotiates the price of a cell or gene remedy with pharmaceutical corporations on behalf of state Medicaid applications — after which holds them accountable for the remedy’s success.
Under the settlement, collaborating states will obtain “discounts and rebates” from the drugmakers if the therapies don’t work as promised, in response to the Centers for Medicare & Medicaid Services.
That’s a stark distinction from how Medicaid and different well being plans sometimes pay for medicine and therapies — the invoice often will get paid whatever the therapies’ advantages for sufferers. But CMS has not disclosed the complete phrases of the contract, together with how a lot the drug corporations will repay if the remedy doesn’t work.
The remedy Cole obtained affords a possible remedy for lots of the 100,000 primarily Black Americans with sickle cell illness, which is estimated to shorten lifespans by greater than twenty years. But the remedy’s price presents a steep monetary problem for Medicaid, the joint state-federal authorities insurer for individuals with low incomes or disabilities. Medicaid covers roughly half of Americans with the situation.
There are two gene therapies accepted by the Food and Drug Administration available on the market, one costing $2.2 million per affected person and the opposite $3.1 million, with neither price together with the expense of the lengthy hospital keep.
The CMS program is among the uncommon well being initiatives began beneath President Joe Biden and continued through the Trump administration. The Biden administration signed the deal with the 2 producers, Vertex Pharmaceuticals and Bluebird Bio, in December 2024, opening the door for states to affix voluntarily.
“This model is a game changer,” Mehmet Oz, the CMS administrator, stated in a July statement asserting that 33 states, Washington, D.C., and Puerto Rico had signed onto the initiative.
Asked for additional particulars on the contracts, Catherine Howden, a CMS spokesperson, stated in an announcement that the phrases of the agreements are “confidential and have only been disclosed to state Medicaid agencies.”
“Tackling the high cost of drugs in the United States is a priority of the current administration,” the assertion stated.
Citing confidentiality, two state Medicaid administrators and the 2 producers declined to disclose the monetary phrases of agreements.
New Therapies
The gene therapies, accepted in December 2023 for individuals 12 or older with sickle cell illness, provide an opportunity to reside with out ache and problems, which might embrace strokes and organ injury, and keep away from hospitalizations, emergency room visits, and different pricey care. The Biden administration estimated that sickle cell care already prices the well being system virtually $3 billion a yr.
With many costlier gene therapies on the horizon, the price of the sickle cell therapies presages monetary challenges for Medicaid. Hundreds of cell and gene therapies are in scientific trials, and dozens may get federal approval within the subsequent few years.
If the sickle cell fee mannequin works, it can in all probability result in related preparations for different expensive therapies, significantly for people who deal with uncommon ailments, stated Sarah Emond, president and CEO of the Institute for Clinical and Economic Review, an unbiased analysis institute that evaluates new medical therapies. “This is a worthy experiment,” she stated.
Setting up fee for medicine based mostly on outcomes is sensible when coping with excessive remedy prices and uncertainty about their long-term advantages, Emond stated.
“The juice has to be worth the squeeze,” she stated.
Clinical trials for the gene therapies included fewer than 100 sufferers and adopted them for less than two years, leaving some state Medicaid officers anticipating reassurance they had been getting a very good deal.
“What we care about is whether services actually improve health,” stated Djinge Lindsay, chief medical officer for the Maryland Department of Health, which runs the state’s Medicaid program. Maryland is predicted to start accepting sufferers for the brand new sickle cell program this month.
Medicaid is already required to cowl virtually all FDA-approved medicine and therapies, however states have leeway to restrict entry by limiting which sufferers are eligible, organising a prolonged prior authorization course of, or requiring enrollees to first endure different therapies.
While the gene remedy therapies are restricted to sure hospitals across the nation, state Medicaid officers say the federal mannequin means extra enrollees can have entry to the therapies with out different restrictions.
The producers additionally pay for fertility preservation reminiscent of freezing reproductive cells, which might be broken by chemotherapy through the remedy. Typically, Medicaid doesn’t cowl that price, stated Margaret Scott, a principal with the consulting agency Avalere Health.
Emond stated pharmaceutical corporations had been within the federal deal as a result of it may result in faster acceptance of the remedy by Medicaid, in contrast with signing particular person contracts with every state.
States are interested in the federal program as a result of it affords assist monitoring sufferers along with negotiating the fee, she stated. Despite some secrecy across the new mannequin, Emond stated she expects a federally funded analysis will monitor the variety of sufferers in this system and their outcomes, permitting states to hunt rebates if the remedy will not be working.
The program may run for so long as 11 years, in response to CMS.
“This therapy can benefit many sickle cell patients,” stated Edward Donnell Ivy, chief medical officer for the Sickle Cell Disease Association of America.
He stated the federal mannequin will assist extra sufferers entry the remedy, although he famous utilization will rely partly on the restricted variety of hospitals that provide the multimonth remedy.
Hope for Sickle Cell Patients
Before gene remedy, the one potential remedy for sickle cell sufferers was a bone marrow transplant — an choice accessible solely to those that may discover a appropriate donor, about 25% of sufferers, Ivy stated. For others, lifelong administration consists of medicines to scale back the illness’s results and handle ache, in addition to blood transfusions.
About 30 of Missouri’s 1,000 Medicaid enrollees with sickle cell illness will get the remedy within the first three years, stated Josh Moore, director of the state’s Medicaid program. So far, fewer than 10 enrollees have obtained it because the state started providing it in 2025, he stated.
Less than a yr into the federal program, Moore stated it’s too early to inform its fee of success — outlined as an absence of painful episodes that result in a hospital go to. But he hopes it will likely be near the 90% fee seen over the course of a few years in scientific trials.
Moore stated the federal program based mostly on how nicely the remedy works was most well-liked over reducing charges for a brand new and promising remedy, which might put the producers’ potential to develop new medicine in danger. “We want to be good stewards of taxpayer dollars,” he stated.
He declined to touch upon how a lot the state could save from the association or disclose different particulars, reminiscent of how a lot the drug corporations may need to pay again, citing confidentiality of the contracts.
Lately Cole, who underwent gene remedy at St. Louis Children’s Hospital, has been capable of give attention to her hobbies — taking part in video video games, drawing, and portray – and incomes her highschool diploma.
She stated she was glad to get the remedy. The worst half was the chemotherapy, she stated, which left her unable to speak or eat — and entailed getting caught with needles.
She stated that her situation is “way better” and that she has had no ache episodes resulting in a hospital keep since finishing the remedy final spring. “I’m just grateful I was able to get it.”