Game-changing gene therapies present cost problems for health execs


CAR-T therapies have high price tags so it’s important to understand your patient population and budget impact.

The recent approval of two cell-based gene therapies has healthcare executives scratching their heads about ways to manage these anticipated costly drugs, say experts.

In August, the FDA approved Novartis’s tisagenlecleucel (Kymriah), the first chimeric antigen receptor (CAR) T-cell therapy for the treatment of certain pediatric and young adult patients with a form of acute lymphoblastic leukemia. The second CAR-T approved by the FDA is Gilead’s axicabtagene ciloleucel (Yescarta), developed by Kite Pharma, the first for certain types of non-Hodgkin lymphoma.

This technology, through specific and unique molecular biology cellular manipulations, changes the patient T-cells so most of them, if not all, recognize the cancers for destruction when infused back into the patient, according to Gerry Messerschmidt, MD, FACP, chief medical officer, Precision Oncology.

“The CAR-T cells target a very specific ‘flag’ on the malignant cells [CD-19] and have demonstrated dramatically improved tumor cell killing compared to prior therapy,” says Messerschmidt. “It is this rapid and efficient CAR-T cell therapy localization of the ‘flagged’ cells and immediate killing of these cells that leads to the cancer cells spilling their contents and resulting in significant toxic reactions, most commonly referred to as cytokine storm that requires specific training by physicians, nurses and other healthcare providers.” 

The price tag and implications

The cost of these drugs for one injection will be several hundreds of thousands of dollars-$373,000 for Yescarta and $475,000 for the Kymriah. Novartis announced a money-back guarantee program to help minimize risk in the event of treatment failure. At press time, Kite Pharma has not announced such a program for its drug.


Kymriah’s price came in lower than many analysts expected, according to Jeremy Schafer, PharmD, MBA, senior vice president, director, payer access solutions, Precision for Value. “However, it is still a significant cost for payers and managed care executives will need to think about the budget impact but also on how payment models can evolve to accommodate these therapies,” he says 

Jake Velie, CEO of Rx-Precision, a prescription risk management company, shares a similar viewpoint. “Treatment plans informed by precision medicine tools and protocols are inherently unique because they are leveraging the new frontier of individualized data to match the patient to the best possible treatment options,” he says. “Treatment plans not based on an individual’s genomic data may simply not work or at worst can harm the patient long-term.”

“However, because the U.S. healthcare marketplace is serviced by many payers, managed healthcare executives are often focused on short-term value and cost minimization since patients may not be a member of the same plan for more than a few years,” says Schafer. “This makes supporting drugs with high upfront costs like Yescarta and Kymriah difficult since the payer that reaps the rewards of the ‘cured’ patient may not be the same payer that funded the treatment. This issue was seen in HCV as well when the newest agents launched.”


Rebecca Sutphen, MD, FACMG, president and chief medical officer at InformedDNA, predicts that associated medical costs are expected to be significant. "Pretreatment and allotransplantation costs have been estimated to be around $200,000," she says. "Beyond these known associated costs, many patients experience a life-threatening complication called 'cytokine storm' that often requires intensive care unit treatment. Neurologic complications and serious infections also occur. Additionally, patients will need to travel large distances in some cases to receive treatment.

"Some other things to consider from a patient perspective is that they may already be very ill any may not survive the time needed to manufacture the cells for treatment, and remission rates in the real world may not be as high as in the limited clinical trials," Sutphen adds.

Despite these issues, the efficacy of the drugs and dire diagnosis means that there will be demand for these agents so payers need to understand their patient population and potential impact on budget, according to Schafer. “In addition, managed care executives need to understand the total cost of care beyond just the cost of the CAR-T therapy,” he says.

Schafer notes that Kaiser Health News reported that the total costs of Kymriah and the 21 similiar therapies, will be far higher than many have imagined, reaching $1 million or more per patient. 


“However, hospitalization, cell collection procedures, preparative therapies prior to the re-infusion of the manufactured cells, significant toxicities that can often require ICU stays and intensive nursing and patient care are all very likely with either drug,” says Gerry Messerschmidt, MD, FACP, chief medical officer, Precision Oncology. “Despite these considerations, the patient benefit on average is outstanding and could lead to long-term survival in many of these subjects. Weighing these risks, benefits and costs will be a challenge from many standpoints for healthcare executives, particularly payers.”

Remission can be achieved more quickly with gene-specific therapies, Velie says.


“In fact, a patient can potentially enter remission after one course of treatment," he says. "Whereas if they hadn’t had a therapy matched to their cancer type or their individual genome and genetic variants, they may need more courses. This saves tens or even hundreds of thousands of dollars in treatments. Many patients experience treatment ‘shot gunning’ if their genetic information and cancer typing isn’t part of the treatment planning process, resulting in long treatment processes with potentially several periods of remission and relapse.”




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