Gene therapy drug for infants priced at 170 million yen : The Asahi Shimbun – Asahi Shimbun

The price for a gene therapy drug to treat an intractable disease in toddlerswas set at about 167 million yen ($1.56 million) per patient, the countrys most expensive medicine covered by national health insurance.

The application for Zolgensma was approved at the Central Social Insurance Medical Council, an advisory council for the health minister, on May 13 and will go into effect on May 20.

The drug is for children under the age of 2 with spinal muscular atrophy,in which the poor functioning of motor nerves causes muscle weakness.

Zolgensma was developed to replace the function of a certain gene that is not working properly with the injection of a normal gene.

Patients can receive a single infusion of Zolgensma.

The price of the drug was determined based on that for Spinraza, an existing drug. While Spinraza costs about 9.5 million yen per dosage and needs to be injected repeatedly for a certain period of time, Zolgensma is believed to have a long-lasting benefit through a single injection.

The price of Zolgensma was first set at about 100 million yen, the estimated cost of using Spinraza for several years, and was then increased to about 167 million yen by taking into account the high therapeutic effects and other benefits of Zolgensma.

The amount that users of Zolgensma need to pay will be much lower than the price since they can use the government-sponsored reimbursement system for high-cost medical care, which caps the amount of out-of-pocket medical costs. In addition, many municipalities subsidize all the out-of-pocket expenses if the patients are children.

Only about 25 patients are expected to use Zolgensma annually, so the high cost of the drug will likely have only a limited impact on the nation's overall medical expenses.

Kymriah, which was approved for treatment of leukemia in Japan last year, is priced at 33.49 million yen, the highest price in the country at the time. As technology for developing new drugs advances, more drugs with expensive price tags are expected to be developed in the future. That could put a heavy burden on health insurance associations.

Each association may need to bear a huge financial burden, said a member of the Central Social Insurance Medical Council.

Ataru Igarashi, an associate professor of pharmacoeconomics at Yokohama City University School of Medicine, said, Manufacturers and the authorities are required to fulfill their responsibilities more than ever for explaining whether the value of a drug is truly worth its price.

(This article was written by Tamura Kenji, a senior staff writer, and Ryuichi Hisanaga.)

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Gene therapy drug for infants priced at 170 million yen : The Asahi Shimbun - Asahi Shimbun