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July 13, 2017

'A new era': FDA panel backs first-ever gene therapy drug

Daily Briefing

Read Advisory Board's take on the implications for providers.

An FDA panel on Wednesday unanimously recommended approval of a treatment developed by Novartis and the University of Pennsylvania that reengineers a patient's own cells to combat a type of leukemia. If approved, it would be the first-ever gene therapy to come to the U.S. market.

FDA is not required to follow advisory panels' recommendations, although it typically does so, the Washington Post's "To Your Health" reports.

About the treatment

The treatment, a type of CAR-T cell immunotherapy, entails extracting immune system T-cells from each patient's blood. The cells are then frozen and shipped to a Novartis facility, where they are genetically modified to attack patients' healthy and cancerous B-cells that have the CD-19 protein. Physicians then infuse the T-cells back into patients, at which point they begin fighting the cancer.

University of Pennsylvania's Carl June, who helped develop the treatment, described the modified cells as "serial killers," as just one can kill up to 100,000 cancerous cells.

Novartis' treatment, known as tisagenlecleucel, is meant to treat people ages 3 to 25 who have relapsed or resisted standard treatment for B-cell acute lymphoblastic leukemia (ALL), which amounts to about 600 U.S. patients per year. Of the 63 patients in a Novartis trial who received the treatment, 52 of them (82.5 percent) went into remission, while 11 died.

Most patients in the study had a side effect called cytokine release syndrome (CRS), which occurs when a patient's immune system attacks their vital organs. Physicians were able to manage CRS, and it caused no deaths in the study. Some patients also experienced neurological issues such as delirium and seizures, although there were no instances of fatal brain swelling in the study.

FDA panelists raised questions about potential long-term side effects of the drugs and about how Novartis would ensure quality control. Novartis plans to limit the therapy's use to 30 to 35 medical centers, whose staff will undergo extensive training and to post company employees at those hospitals. The company also would track patients for 15 years.

Novartis has declined to estimate the price of the one-time treatment. Analysts told the New York Times and NPR it could cost about $300,000 to $500,000. Denise Grady writes for the Times that "although the figure may seem high, people with cancer often endure years of expensive treatment and repeat hospital stays that can ultimately cost even more."

Patients who undergo the Novartis treatment also need an injection of immunoglobulin every three to four weeks to strengthen their immune systems, which can cost $1,000 to $10,000 per injection, depending on a patient's weight, Bloomberg reports.

'A new era' with similar treatments expected

The Associated Press reports that after "decades of setbacks and disappointments in efforts to fix, replace, or change genes to cure diseases, several companies are near the finish line in a race to bring CAR-T and other types of gene therapy to patients."

The FDA panel's decision on the Novartis treatment "opened a new era in medicine," the New York Times reports.

Timothy Cripe, a panelist and Nationwide Children's Hospital oncologist, described the treatment as the "most exciting thing I've seen in my lifetime."

Gwen Nichols, CMO of the Leukemia and Lymphoma Society, which helped fund some of the research, said researches would now try to determine whether the treatment can be combined and used to "treat patients with less disease, so that the immune system is in better shape and really able to fight," adding, "This is the beginning of something big" (Grady, New York Times, 7/12; Stein, "Shots," NPR, 7/12; McGinley, "To Your Health," Washington Post, 7/12; Cortez, Bloomberg, 7/13; Johnson, AP/ABC News, 7/12; Clarke, Reuters, 7/12; Chustecka, Medscape, 7/12).

What the news means for providers: Advisory Board's take

Deirdre Saulet, Oncology Roundtable

The FDA panel's recommendation to approve CAR T-cell therapy marks an exciting step forward for cancer treatment. The immediate impact of an approval would be limited to a small number of patients receiving care at select organizations, but cancer providers, researchers, drug manufacturers, and patients across the country are betting big on the promise of these new therapies, particularly immunotherapy and targeted treatments.

Because of the excitement around these innovations, providers across the country are understandably eager to deliver them to their patients. However, leaders need to be strategic in their investments, as the field is rapidly evolving and there are still many unknowns. To help physicians put precision medicine into practice, leaders need to invest in clinical decision support tools and ongoing opportunities for education and expert collaboration, such as molecular tumor boards.

In addition, many of these new therapies come with severe side effects that are not yet well understood. Another CAR T-cell therapy trial was halted last year due to five patient deaths, and studies have shown that 30 percent of patients on checkpoint inhibitors (a more common form of immunotherapy) experience "interesting, rare, or unexpected side effects." In response, we're seeing progressive organizations, such as Avera Cancer Institute and Intermountain Healthcare, develop dedicated precision medicine clinics. These serve as on-demand resources for patients who are interested in learning more about their treatment options and for providers who benefit from ongoing symptom management support for patients on these new therapies.  

To learn more, check out our top resources on personalized medicine strategy, including our Precision Medicine Investment Playbook and Precision Medicine Business Plan Template.

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