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First gene therapy for cancer approved: What health execs should know


The first therapy based on gene transfer has been approved by the FDA. What’s next for healthcare executives?

The approval of the first gene therapy marks what likely will be considered the next new major era of drug development in the U.S., according to experts.

The FDA approved the first chimeric antigen receptor T cell (CAR-T) therapy, Kymriah (tisagenlecleucel)-formerly CTL019-for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), ushering in a new approach for cancer treatment and other serious and life-threatening diseases. It is approved for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

According to the American Cancer Society, ALL is a cancer that starts from the early version of white blood cells called lymphocytesin, the bone marrow (the soft inner part of the bones, where new blood cells are made). The disease progresses quickly and is the most common childhood cancer in the U.S. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year.

Fighting from within

Kymriah is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer, according to the manufacturer, Novartis. It is the first therapy based on gene transfer approved by the FDA.

Each dose of Kymriah is customized, using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte, according to the FDA. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein that directs the T-cells to target and kill leukemia cells that have a specific antigen on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

“Personalized therapies like this, that harness the power one’s own immune system-the killing power of the T-cell in particular-represent enormous potential in treating a wide array of cancer types at various stages of progression,” says Managed Healthcare Executive Editorial Advisor David Calabrese, senior vice president, chief pharmacy officer at OptumRx.

“While extremely encouraging and exciting, this excitement must also be properly tapered by an appreciation of the fact that we still have a substantial amount of challenges to overcome and questions to answer in ascertaining the true clinical value of these therapies, particularly in solid tumors, and overcoming the magnitude of toxicity and the unprecedented costs that come along with therapies of this nature,” Calabrese says.

Next: Challenges to watch



Unique challenges

The advent of “one and done” gene therapy treatments presents a significant challenge, says Jeremy Schafer, PharmD, MBA, senior vice president, director, Payer Access Solutions at Precision for Value.

“In the U.S. healthcare system, people have many options for healthcare coverage and in the commercial world, often change insurers every two to three years,” Schafer says. “For U.S. payers, this means a focus on short-term value and return since the long-term benefits of a therapy are unlikely to be realized by the payer that initially funded them.”

This structure presents a challenge to therapies that promise to alleviate long-term costs with significant up front investment, says Schafer. “In the hepatitis C world, there was significant resistance to funding expensive therapy on the front end, despite the promise of avoiding costly complications later, because payers knew that those benefits were unlikely to be realized by the payers that funded the treatment in the first place,” he says. “Even in Europe, with its single-payer approach to healthcare, gene therapies have had minimal success because of the significant up front cost.”

Kymriah, expected to cost around $475,000, may find a similar struggle, Schafer adds.  


Still, Kymriah presents opportunity to innovate on the traditional payment system, says Schafer. “First, payers should understand their true risk and exposure to a product like Kymriah,” he says. “While the price is significant, the incidence of the disease is quite low. Secondly, payers should consider the clinical benefit. With a remission rate of 83%, Kymriah seems quite efficacious albeit payers should press for more information on long-term survival and durability of remission. Finally, payers should investigate ways to support gene therapies while also achieving their goals and providing cost-effective care.

“The gene therapy revolution isn’t going away and we can probably expect gene therapies for such ailments as blindness and hemophilia in the next decade,” Schafer says. “Payers will need to find a way to cover some of these therapies in an effective way as patients will increasingly demand them. Manufacturers will need to consider how to work with payers in this regard as a high-priced product with no coverage isn’t a revenue opportunity.”

Schafer believes Novartis seems to understand some of the U.S. payer complexities of the gene therapy space. In a press release upon approval, Novartis announced a collaboration with CMS on efforts to make Kymriah accessible, including an outcomes-based arrangement that looked to refund Kymriah’s cost if a patient does not respond in the first month.

FDA has approved a Risk Evaluation and Mitigation Strategy for Kymriah. To support safe patient access, Novartis is establishing a network of certified treatment centers throughout the country which will be fully trained on using Kymriah and appropriate patient care. Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah to further evaluate the long-term safety. 

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