Novartis's groundbreaking CAR-T cell therapy Kymriah has received FDA approval for treating B-cell acute lymphoblastic leukemia (ALL) in children and young adults under 25, marking a significant advancement in cancer immunotherapy. However, its $475,000 price tag has sparked intense debate about the economics of innovative treatments in oncology.
The therapy, which engineers a patient's own immune cells to fight cancer, demonstrated an 83% remission rate in clinical trials for patients who had relapsed after conventional treatments. This remarkable efficacy represents hope for the approximately 3,100 young people diagnosed with ALL annually, particularly the 15% who don't respond to traditional chemotherapy.
Revolutionary Treatment Approach
Kymriah utilizes CAR-T cell (chimeric antigen receptor T-cell) therapy, a process that extracts a patient's T-cells, genetically modifies them to target cancer cells, and reinfuses them into the patient. The Leukemia and Lymphoma Society (LLS), which contributed $20 million to CAR-T research, called the approval "a new era" in cancer treatment.
"This is truly an exciting new day for cancer patients," said Louis J. DeGennaro, PhD, LLS president and chief executive officer.
Dr. Gwen Nichols, chief medical officer at the Leukemia and Lymphoma Society, emphasized the therapy's transformative potential: "The idea of being able to give a child their life back, the value here is so great."
Pricing Controversy
The $475,000 price has drawn both criticism and defense from various stakeholders. Novartis CEO Joseph Jimenez told Forbes the company has invested approximately $1 billion bringing the drug to market, while acknowledging the contribution of publicly funded research.
Some experts argue the price is justified when compared to alternatives. "While this is an expensive therapy, so would be the treatment these children would be getting if they didn't get this therapy," Dr. Nichols noted. "The cost for just keeping them alive is exorbitant."
Critics, however, see the price as excessive. David Mitchell, founder of Patients for Affordable Drugs, stated: "While Novartis' decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive. Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more."
Will Holley, spokesman at Campaign for Sustainable Rx Pricing, expressed concern about the trajectory of drug pricing: "Ten years ago, we had the thousand-dollar-a-month drug and their jaw dropped. Now, half-a-million dollars for a drug. It's this ever-increasing trend."
Innovative Pricing Models
Two aspects of Novartis's pricing strategy represent potential shifts toward value-based pricing in pharmaceuticals:
Outcomes-Based Contracts
In collaboration with the Centers for Medicare and Medicaid Services, Novartis announced it will only receive payment if patients respond to treatment within 30 days. This approach directly links price to clinical benefit, though some experts question whether the 30-day timeframe is sufficient, noting that 25% of initial responders in the pivotal trial experienced disease progression by six months.
Indication-Specific Pricing
Novartis has indicated that future approved indications for Kymriah may carry different price points, acknowledging that a drug's value can vary based on its effectiveness across different conditions. This approach has gained traction in both public and private sectors, with pharmacy benefit managers like Express Scripts and CVS already implementing similar models for oral anti-cancer drugs.
Access Programs
To address affordability concerns, Novartis has established "Kymriah Cares," which includes copay assistance and travel support for eligible patients. The program covers transportation, accommodations, and meals for patients and up to two caregivers.
"This program includes copay assistance as well as travel assistance for eligible patients," Novartis officials stated.
Broader Implications for Cancer Treatment
Dr. Craig Devoe, acting chief at the Don Monti Division of Medical Oncology & Hematology at Northwell Health, expressed concern about the broader trend of rising cancer treatment costs: "This is a breakthrough, it's important and everybody gets that. The issue is that overall this is just emblematic of skyrocketing costs of cancer drugs in general."
He noted that while Kymriah might cost as much as a bone marrow transplant, some patients may eventually relapse and require additional treatments, potentially including transplants.
The Institute for Clinical and Economic Review is expected to release an independent assessment of CAR-T therapy pricing in March 2018, which may provide more context for evaluating Kymriah's cost-effectiveness.
As more cell and gene therapies approach market approval, the debate around Kymriah's pricing model will likely influence how future breakthrough treatments are valued and priced. The tension between innovation, access, and sustainability remains at the heart of this discussion, with stakeholders across healthcare seeking a balance that rewards scientific advancement while ensuring patients can benefit from these potentially life-saving therapies.
