Photo/IllutrationKymriah (Provided by Novartis Pharmaceuticals Corp.)

A health ministry panel on May 15 approved expanding the public health insurance program to cover an individualized drug for leukemia and lymphoma patients, the most expensive new medicine ever offered in Japan.

Hopes are running high for the Kymriah for Chimeric antigen receptor T cell (CAR-T cell) therapy, developed by Novartis Pharmaceuticals Corp., based in Switzerland, which is the first product to use gene engineering technologies for leukemia and lymphoma patients.

The therapy is likely to start in this summer at the earliest with the official price of the drug set at about 33.49 million yen ($306,000). As it can cause serious side effects in patients, however, doctors will be required to use precaution in prescribing the treatment.

The Central Social Insurance Medical Council, an advisory council for the health minister, decided in a general meeting to cover Kymriah under the government-sponsored health insurance program.

In the Kymriah therapy, T cells that recognize foreign substances in human bodies and fight them are removed from patients, and then are genetically engineered so that they attack cancer cells.

White blood corpuscles will be first taken from patients at hospitals in Japan. Then, T cells will be separated from the corpuscles and frozen at facilities in those hospitals and sent to a Novartis facility in New Jersey in the United States.

At the Novartis site, the T cells will be genetically engineered so that they attack cancer cells. After that, the genetically engineered cells will be cultivated to become the pharmaceutical product Kymriah.

The drug will be sent back to the Japanese hospitals and put intravenously into the patients from whom the white blood corpuscles were taken out. The entire process will take about two months.

The medicine will be used for patients of leukemia or lymphoma who cannot be successfully treated by conventional therapies. Novartis estimates that the new drug will be used for up to 216 patients annually in Japan.

In the case of leukemia, Kymriah will be used for patients whose B cells, a kind of blood cells, have become cancerous. The patients comprise most of those with acute lymphocytic leukemia. In addition, patients who receive the medicine must be 25 or younger.

In treating lymphoma, the drug will be used for patients of any age whose disease was also caused by B cells. They account for about one-third of the lymphoma patients.

In the future, clinical trials will be also held for patients of different blood cancers whose B cells have become cancerous, and for patients aged 26 or older. If beneficial effects and safety are confirmed in the trials, those patients’ use of the medicine will also be covered by the health insurance program.

In clinical trials conducted so far, the survival rate of lymphoma patients after a year of therapy rose to 50 percent from about 20 percent of conventional treatments. In the case of leukemia patients, the corresponding rate jumped to 80 percent from 20 percent.

However, treatment using Kymriah poses serious side effects. In clinical trials, 80 percent of leukemia patients and 60 percent of lymphoma patients suffered from cytokine release syndrome, which could cause respiratory failure.

Some of those patients also experienced consciousness disorders and hallucinations. In addition, 20 percent of leukemia patients suffered from encephalopathy.

Therefore, the therapy cannot be used by all the patients who might benefit from the medicine.

“T cells must be sufficiently harvested. In addition, it is indispensable for patients to maintain a stable physical condition for the two months that are necessary to produce the drug,” said Hidefumi Hiramatsu, a lecturer at Kyoto University.

The health ministry attached conditions for approving Novartis’ production and sales of Kymriah. One is that hospitals have emergency rooms or similar facilities that can sufficiently deal with emergency situations.

Another is that the medicine is used by doctors who have sufficient knowledge and experience with transplants of hematopoietic stem cells. According to the health ministry, about 170 hospitals meet the conditions.

From now, Novartis will look into facilities and staff of those hospitals, offer training to doctors and some other staff members and certify the hospitals that can use Kymriah.

Some hospitals have already started preparations to offer the new therapy. They include the National Cancer Center Hospital, Kyushu University Hospital, Hokkaido University Hospital, Kyoto University Hospital and Nagoya University Hospital.

“This therapy is an epoch-making one that offers hope. But it could cause disease complications. Therefore, doctors should use it cautiously by obtaining cooperation from experts of many fields,” said Koji Kato, an associate professor at Kyushu University.

Patients welcomed the decision to cover Kymriah treatments with the health insurance program.

“It is a good news for patients who cannot be cured by conventional treatments. I believe that the new therapy will lead to the development of the next therapy,” said Akiko Hashimoto, head of “Ketsueki Joho Hiroba Tsubasa" (Blood information square Wing), a support group for blood cancer patients. Hashimoto lost her eldest son to leukemia.

In principle, patients shoulder 10 to 30 percent of fees for medical treatments, including those for medicines. Depending on their ages and incomes, however, upper limits are imposed on the amounts of fees that are actually covered by patients.

For example, if the fee for the new therapy that uses Kymriah is 34 million yen, including the technical fee for drip infusion, the actual charged amount will be about 400,000 yen if patients are younger than 70 and their annual income is between 3.7 million yen and 7.7 million yen.

The amount over 400,000 yen will be covered by premiums paid for the health insurance program or by public funds. Subsidizing the costs of the expensive medicine is expected to worsen the financial situation of the health insurance program.

Subsequently, the health ministry made attempts to lower the official price of Kymriah. As a result, the price was set at about 33.49 million yen based on data submitted by Novartis.

In other countries such as the United States, Britain and Germany, the price for the treatment ranges between 40 million yen and more than 60 million yen.