TOKYO – Japan's Ono Pharmaceutical Co. Ltd. recently got into a public debate at home about the price of its cancer drug, Opdivo (nivolumab), which critics argue could bring too great a burden on the national welfare system. The company, however, said an analyst's estimates as to the amount of people that will use the anti-PD-1 drug and the length of time for which they will use it are both exaggerated.

Opdivo is a revolutionary drug that helps the body combat cancer by keeping T cells, which can fight tumors, active despite attacks from the tumor. "By canceling out the cancer's influence, the drug allows patients to reactivate their immune systems and fight the cancer themselves," said Yukio Tani, senior director of corporate communications at Ono.

The drug, approved in Japan in mid-2014, is often used as a last resort for patients for whom chemotherapy has not worked and do not have the opportunity to go into surgery. Its price, however, is prohibitively expensive, costing ¥730,000 (US$7,081) for a 100-mg bottle, or about ¥2.6 million a month for a 60-kg patient. (See BioWorld Today, July 9, 2014.)

Earlier this year, an analyst presented estimates to the government that the drug would be used by 50,000 patients for a period of one year at the cost of more than ¥1.5 trillion. According to the Ministry of Health, Labor and Welfare, Japan's total annual medical bill currently stands at around ¥40 trillion, or about $38.5 billion.

"The analyst and our company have completely different expectations on the number of patients that would be prescribed the drug and the dosages they would receive," Tani told BioWorld Today. While the analyst estimates it could be used for 50,000 patients, Ono said it believes the number will stand at around 15,000; it also believes patients will use the drug for three and a half months, rather than a full year.

A number of factors have led to that difference of opinion, including estimates on required dosages and the requirements needed to be given the drug. Ono argued that as it is given to patients for whom more traditional treatments have failed, the analyst's estimate is far off the mark.

The company hopes, however, to expand the drug's use to a greater variety of cancers. "We have approval for using the drug to treat malignant melanoma and non-small-cell lung cancer, but we are also currently seeking approval for its use in Japan to treat a variety of other types of the disease," Tani said. "Opdivo is currently in clinical trials for use in more than 20 different types of cancer. We expect that a lot of cancer patients will come to use the drug in the future."

If the drug's usage is approved for more types of cancer, in a country where more than 880,000 incidences of the disease were recorded in 2014, according to the Cancer Information Service, then the cost to the nation would likely rise.

However, the drug is a one-of-a-kind treatment and costs around ¥560,000 in production and distribution fees and taxes. Tani also pointed out that the company did not decide the cost of Opdivo. "I acknowledge there is a standpoint that the drug is too expensive, but it was priced at this level by a committee at the Ministry of Health, Labour and Welfare, so I do not share that view," he said.

Ono also has received approval for the drug to treat renal cell carcinoma and non-small-cell lung cancer in Europe. The company is awaiting approval for the drug's use in the treatment of Hodgkin's lymphoma in the U.S. and Europe. Ono sells Opdivo in Japan, Taiwan and South Korea, while partner Bristol-Myers Squibb Co. handles sales for the West.

A number of other drugs are in development at Ono, to treat diseases ranging from Alzheimer's to rheumatoid arthritis and Parkinson's, and this week inked a preclinical T-cell immunotherapy deal with Celyad SA. (See BioWorld Today, July 12, 2016.)

The company also recently reorganized its structure, opening a new research facility and restructuring to speed up drug development and get products to market faster. "We reorganized our structure about three months ago, and I feel we have gotten off to a favorable start," Tani said. "We recently completed a new research and development facility in Minase, Kyoto Prefecture, which focuses on compound synthesis and analysis. We are also working to speed up our oncology research and development, directly under the president."

For the fiscal year ended March 31, the company earned revenue of ¥160.28 billion and a profit of ¥24.97 billion. Its plans for the future include strengthening its overseas infrastructure for business development.

The company's ambitions are far-reaching, Tani said. "I think biotechnology is going to become a new pillar of Japanese industry."