"2" new drugs have attracted the most attention: the approval of anti-tumor drugs has been accelerated, and rare disease drugs have been included in medical insurance

Anti-tumor drugs and rare disease drugs are the two types of new drugs that have attracted the most attention. Beijing News Drafting/Xu Xiao

Under the dual "spawning" of policies and industries, anti-tumor drugs and rare disease drugs continue to emerge. Among the 78 new drugs that have been approved for marketing by the State Food and Drug Administration this year, 22 are anti-tumor drugs and 11 are rare disease drugs, accounting for more than 42% of the two.

A variety of oncology drugs have created the first in China, and large investments have been made in research and development

The 22 anti-tumor drugs approved for listing this year have indications for lung cancer, liver cancer and so on. For example, CStone's PhuketHua (pratinib capsules) was approved in March this year for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have previously received platinum-containing chemotherapy and have been positive for transfection rearrangement (RET) gene fusion, which is the first selective RET inhibitor approved for marketing in China. In December, Asunyped's drug orebatinib was listed, becoming the first third-generation BCR-ABL inhibitor approved for marketing in China for the treatment of any tyrosine kinase inhibitor resistance, and using a fully validated detection method to diagnose adult patients with chronic myeloid leukemia with T315I mutation in the chronic or accelerated stage.

On June 9 this year, Rongchang Bio's vidicetizumab and tetacilex were conditionally approved for listing. This is the first ADC (antibody conjugate) drug approved for marketing through independent research and development, and the approved indications are for advanced or metastatic gastric cancer (including gastroesophageal-conjugate adenocarcinoma). From the acceptance of the listing application in August 2020 to the conditional approval on June 9, 2021, the approval of vidicetizumab for listing took only 10 months.

The increase in the speed of approval has also made innovative biomedical companies attract the attention of the capital market. Rongchang Biotech, which landed on the Hong Kong Stock Exchange on November 9, 2020, set a record for the highest global biotech IPO fundraising in 2020 with an actual fundraising amount of US$590 million. On May 10, 2021, Rongchang Biotech announced its plan to go public on the Science and Technology Innovation Board, and successfully passed the meeting on November 11 this year. This time, Rongchang Bio plans to raise 4 billion yuan for the research and development of new drugs such as new drug industrialization and anti-tumor antibody new drugs.

Behind the frequent emergence of new drugs is a large amount of investment in research and development. CStone's R&D investment in 2020 was 1.405 billion yuan, exceeding the revenue scale of 1.039 billion yuan in the same period, and the R&D investment in the first half of 2021 was 513 million yuan. Yasheng Pharmaceutical's R&D expenditure in 2020 reached 565 million yuan, and the R&D investment in the first half of 2021 was 318 million yuan, achieving a year-on-year increase again; Nuocheng Jianhua's R&D expenditure in 2020 was 403 million yuan, nearly doubling that of 2019, and the R&D investment in the first half of 2021 was 185 million yuan. Junshi Bio invested 1.778 billion yuan in research and development expenses in 2020, an increase of 87.93% year-on-year, and R&D investment in the first half of 2021 was 947 million yuan, an increase of 33.62% year-on-year.

At the same time as the "new forces" are rising, established companies are also participating in the competition. Hengrui Pharmaceutical's R&D investment has increased year by year, from 1.18 billion yuan in 2016 to 4.989 billion yuan in 2020, and the R&D investment in the first three quarters of this year has exceeded 4 billion yuan.

At the same time, the policy level is guiding the research and development of tumor drugs to become rational. In July this year, the Drug Evaluation Center (CDE) of the State Food and Drug Administration issued the Guiding Principles for Clinical Research and Development of Antineoplastic Drugs Oriented by Clinical Value (Draft for Comment). The "Guiding Principles" pointed out that the research and development of anti-tumor drugs in China is in a stage of rapid development; the research and development of anti-tumor drugs, from determining the direction of research and development to carrying out clinical trials, should implement the concept of clinical demand as the core and carry out the research and development of anti-tumor drugs oriented by clinical value.

Rare disease drugs are included in medicament, with an average reduction of 65%

In addition to anti-tumor drugs, rare disease drugs are also widely concerned. Statistics from the Pain Challenge Foundation show that since the release of the first rare disease catalogue in May 2018, a total of 34 rare disease drugs have been approved for marketing. Especially this year, 11 rare disease drugs have been approved.

On December 21 this year, Novartis Pharmaceuticals' rare disease drug ofatumab injection was approved for the treatment of recurrent multiple sclerosis in adults, becoming the 11th rare disease drug approved for marketing this year.

Offaldumab was approved for marketing in the United States in October 2009 for the treatment of chronic lymphocytic leukemia. Since then, Novartis pharmaceuticals has launched a new development project to study the therapeutic effect of offalumab on relapsed multiple sclerosis, and in August 2020, it was approved by the US Food and Drug Administration (FDA) as a subcutaneous drug for the treatment of recurrent multiple sclerosis in adults. Just one year later, the drug was approved in China for the treatment of the rare diseases mentioned above.

In addition to offalumab, rare disease drugs approved this year include brosoyomab of Concord Fermented Kirin, Takeda's Etibant acetate and veravosidase α, and Baekje's introduction of stuximab.

Judging from the above approvals, rare disease drugs mainly rely on overseas imports. However, with the continuous introduction of incentive policies, the number of enterprises in the field of rare diseases has gradually increased. For example, the domestic enterprises Maibo Pharmaceutical and Chia Tai Tianqing are laying out mepolitizumab biosimilars. Among them, Chia Tai Tianqing submitted a clinical trial application in October this year and was accepted.

Frost & Sullivan expects China's rare disease drug market to increase from $1.3 billion in 2020 to $25.9 billion in 2030, with a compound annual growth rate of 34.5%. With the introduction of more innovative drugs and the increase in availability/affordability, China's rare disease drug market will account for 6.8% of the global rare disease market in 2030. On December 3, Chinese patients with spinal muscular atrophy (SMA) and their families ushered in the long-awaited good news: the world's first drug for precisely targeted treatment of SMA, Nocinasin Sodium Injection, was included in the new version of the medical insurance catalogue. When Nocinason Sodium was originally approved for listing in China, it was priced at 699,700 yuan per unit, and it was concerned by public opinion because of price issues during the medical insurance negotiations in 2019. Since then, although the price has been reduced to 550,000 yuan, it was still pushed on the hot search again in September this year.

Up to now, more than 60 rare disease drugs in China have been approved for listing, of which more than 40 have been included in the national medical insurance directory, involving 25 diseases. Among them, a total of 7 rare disease drugs have been successfully negotiated this year, with an average reduction of 65%.

In January this year, the National Medical Insurance Bureau and the Ministry of Finance issued the "National Medical Security Treatment List (2020 Edition)", and local rare disease protection policies may face clean-up. In this regard, Guo Jinchuan, director of information research at the Pain Challenge Foundation, pointed out that although there are more innovative payment models to consider, such as exploring payment by efficacy and exploring medical loans, "to ensure the accessibility of rare disease drugs, the most fundamental thing is the need for medical insurance support." ”

This year, the National Medical Insurance Bureau mentioned in reply to the rare disease-related suggestions submitted by Liu Xianxiang, a deputy to the National People's Congress, that for special rare diseases with particularly high costs, the National Medical Insurance Bureau will explore a rare disease drug guarantee mechanism with relevant departments and strive to reduce the burden on patients. "This has released a more positive signal that the National Health Insurance Bureau may try to solve the problem of high-value drugs for rare diseases through multi-sectoral alliances." Guo Jinchuan said.

Beijing News reporter Wang Kara Zhang Xiulan

Proofread by Zhao Lin