The announcement that an immunotherapy treatment using engineered versions of patients’ own cells — recently approved by the Food and Drug Administration (FDA) — has been heralded as a real breakthrough in cancer treatment.
But amid the celebration is the concern that the cost for the drug — ringing in at $475,000 — could be financially damaging for patients who pursue this treatment.
There is also a worry that the new drug could usher in an unsustainable rise in cancer treatment costs.
The drug is called Kymriah, and it was developed by Novartis.
Kymriah uses a patient’s own immune cells and reengineers them to fight cancer. It helps patients fight a kind of leukemia called B-cell acute lymphoblastic leukemia (ALL).
The Leukemia and Lymphoma Society (LLS) declared the FDA approval of Kymriah a “new era” in cancer treatment.
“This is truly an exciting new day for cancer patients,” Louis J. DeGennaro, PhD, LLS president and chief executive officer, said in a statement.
The LLS noted they have spent $20 million in funding CAR-T cell therapy research that helped contribute to Kymriah.
Every year an estimated 3,100 young people are diagnosed with ALL.
While children with the disease have an overall survival rate of 85 percent with traditional chemotherapy treatments, those who don’t respond or relapse face a poor outlook.
Kymriah reengineers a patient’s cells through a process called CAR-T cell (chimeric antigen receptor T-cell) therapy so they are primed to attack the cancer.
In a clinical trial, people with ALL who had relapsed and then given Kymriah showed an 83 percent remission rate.
The treatment has only been approved for children and young adults under the age of 25.
Dr. Gwen Nichols, the chief medical officer at the Leukemia and Lymphoma Society, said she is concerned about the growing costs for cancer treatment.
However, she said that Kymriah does not cost significantly more than a bone marrow transplant or intense hospitalization for multiple chemotherapy treatments.
“While this is an expensive therapy, so would be the treatment these children would be getting if they didn’t get this therapy,” she said. “The cost for just keeping them alive is exorbitant.”
Nichols said there needs to be more attention paid to rising cancer treatment costs in general, but Kymriah — despite its price tag — is different.
“The idea of being able to give a child their life back, the value here is so great, I think that focusing just on [the cost] isn’t focusing on the bigger problems,” she said.
Taking aim at pharma costs
However, some patient groups disagree that the drug’s price is acceptable.
Will Holley, spokesman at Campaign for Sustainable Rx Pricing, said the treatment is absolutely a breakthrough, but its pricing highlights how drug and medical treatment costs have escalated in recent years.
“Ten years ago, we had the thousand-dollar-a-month drug and their jaw dropped,” Holley told Healthline. “Now, half-a-million dollars for a drug. It's this ever-increasing trend.”
Holley said if costs don’t come under control, people will be unable to access these breakthrough therapies.
“At a certain point, access becomes just as large an issue as innovation,” he said. “If the patients who need these therapies don't have access to them... then what have we ended up with?”
David Mitchell, founder and president of Patients for Affordable Drugs, said in a statement that price tags perpetuate a cycle that hurts patients.
“While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive,” Mitchell said in a statement. “Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more.”
Mitchell said $200 million in funding from the National Institutes of Health has gone to CAR-T cell research that helped lead to Kymriah.
However, Novartis Chief Executive Officer, Joseph Jimenez, told Forbes that number was overblown and that only $16 million had been put towards CAR-T cell funding when they came onboard.
Jimenez said Novartis has spent $1 billion bringing the drug to market.
Novartis officials said in a statement they are working with the Centers for Medicare and Medicaid Services, and the company will only get reimbursed by the centers if patients show a response to the drug.
This means even if a patient gets the full treatment, there will be no cost if they do not go into remission.
In addition, Novartis has created Kymriah Cares to help patients afford the drug. The company has pledged to offer an access program for eligible uninsured and underinsured patients.
“This program includes copay assistance as well as travel assistance for eligible patients,” Novartis officials said. “The travel assistance program will arrange and cover air and ground transportation, accommodations, and meals for the eligible patient and up to two caregivers.”
How costs affect doctors
For those in the field, the increasing cost of drugs has taken a toll in how they work with patients.
Dr. Craig Devoe, acting chief at the Don Monti Division of Medical Oncology & Hematology at Northwell Health, said pharmaceutical costs have been increasing, even for therapies that have been around for years.
He estimated he’s seen costs go up by around 10 percent year over year for many cancer treatments.
As a result, Devoe said he’s had more and more issues keeping patients protected from financial strain while getting treatment.
“There's a whole other toxicity now called financial toxicity,” Devoe said. “[Patients] are struggling with out-of-pocket expenditures.”
He said they now have “teams of people” to help patients get through the maze of copayment assistance programs and insurance authorization.
In regard to Kymriah, Devoe acknowledged that this is a big step in a new kind of treatment, but he is concerned about what it means for cancer treatment in the future.
“This is a breakthrough, it's important and everybody gets that,” he said. “The issue is that overall this is just emblematic of skyrocketing costs of cancer drugs in general.”
Devoe said it’s true that Kymriah might cost as much as a bone marrow transplant, but he worries that some people may eventually relapse and then need a transplant later as well.
“There has to be more of a reasonable and fair profit that pharma can derive from these kinds of treatments,” he said. “That word reasonable is where the gray is and where the debate lies.”