2019 Approvals For New Drugs: China Has Been Awarded The Same Number Of Treatments In The United States.

Over the past few years, the biopharmaceutical industry has flourished, largely thanks to the increasingly cooperative and flexible regulatory system of drug regulatory agencies in various countries. Every year, how many new drugs have been released by the watchdog to enter the market has become a guide for the next step of drug companies and investors.

In 2019, the trend continued in recent years, and regulators around the world still gave positive signals to innovative drugs, and more innovative drugs were on the market. Not only in the field of cancer, but also in rare diseases and more yuan diseases, including biological analogues, it has been approved frequently.

In January 6, 2020, the global regulatory agency's wind vane, FDA, CDER released the annual new drug approval report and analyzed the importance of innovation for regulation and medicine.

In 2019, CDER approved 48 new drugs, including small molecule drugs and biological agents. "New drugs are usually innovative products that can meet previously unmet medical needs or significantly promote patient treatment in other areas. One or more active ingredients in new drugs have never been approved in the United States. "

China is shoulder to shoulder with the United States and is catching up with others. Since 2017, the NMPA (former CFDA) has made a more active attempt to speed up the review and approval, leading to the development of domestic innovative pharmaceutical enterprises and the innovation input and transformation of traditional pharmaceutical enterprises.

As of December 31, 2019, according to incomplete statistics of twenty-first Century economic report, NMPA approved more than 50 new drugs listed, of which more than ten domestic new drugs accounted for more than 20%: local innovative enterprises are becoming the new force in the field of Biopharmaceutics in China.

In January 1, 2020, the official website of the State Administration of drug administration released the new year address of the party secretary Li Li and Secretary Jiao Hong. "Over the past year, the dividend of drug regulatory reform has been continuously released. The review and approval process has been further optimized. The 10 1 categories of new drugs, 16 new clinical urgently needed new drugs, 19 innovative medical devices have been approved, and 10 priority medical devices have been listed successfully. 　　

Looking back at the second ten years of twenty-first Century, the position taken by various regulatory bodies on new drugs plays an important role in leading the direction of R & D and establishing confidence in the field of biopharmaceutical industry.

Diversity of diseases

In 2018, the number of new drugs approved in the past 20 years was the largest in the past two years. In 2018, FDA approved 59 new drugs listed at a record high. In 2019, FDA did not exceed this figure, ending with 48 new drugs, the second highest figure in ten years.

In the second ten years of twenty-first Century, from 2010 to 2018, CDER approved about 37 new drugs per year.

Innovation is the key word. In its 2018 annual report, FDA defines its work as "one year of innovation, efficiency and new progress", and the 2019 summary is "promoting health through innovation". According to CDER, 20 (42%) of the 48 new drugs approved in 2019 were first-rate, and these drugs usually have different mechanisms of action from existing therapies.

In the speech, Janet Woodcock, director of the center for drug evaluation and research, pointed out that the innovation here is not only the "new" of drugs, but also the innovation of approval methods, "improving the efficiency and speeding up the key regulatory tools for application for examination and approval". In addition, the approval of new drugs also includes many expanded drugs, such as children's indications.

One of the characteristics of this year is the emphasis on the approval of drugs for different diseases. Although anticancer drugs may be the most popular area of concern, CDER did not regard it as the first part of the discussion.

The first is the rare disease drugs. Of the 59 new drugs approved by FDA in 2018, 34 (58%) were approved for the treatment of rare diseases, affecting the number of orphans with a history of 200 thousand or less.

In 2019, 21 of the 48 new drugs (44%) were approved for the treatment of rare diseases. CDER lists the approval of a variety of rare drugs, including cystic fibrosis. Scenesse implants can increase the painless exposure of patients with photosensitivity (sensitized to sunlight) caused by erythrocyte protoporphyria. This is a rare disease. Turalio capsule is suitable for some adult patients with giant cell tumor of tendon sheath. This rare disease involves non neoplastic tumors around the joint area. The rest includes the first treatment of spectrum disorders of the optic neuromyelitis, new drugs for the treatment of sickle cell disease, and a new therapy that can slow down the rate of pulmonary function decline in patients with systemic sclerosis associated interstitial lung disease.

Behind these awkward diseases and drug names are the growing importance of R & D, regulation and business companies to minority diseases.

In addition, new drugs are listed in neurological and psychiatric diseases, infectious diseases, heart, lung, circulatory and endocrine diseases, autoimmune diseases, specific health problems of women and men, and cancer and blood diseases.

CDER also mentioned in particular the biological analogues approved in 2019. "Biological products are very complex and are often used to treat patients with serious life-threatening diseases. FDA approves biological analogues designed to create competition, increase patient utilization and reduce the cost of important therapies. "

In 2019, CDER approved ten new biosimilars, involving Humira, Rituxan, Enbrel, Herceptin, Avastin, Remicade and Neulasta. The fourth biological analogues, including Avsola, three of them were Inflectra, Ixifi and Renflexis, respectively, which included the treatment of Crohn's disease. Rheumatoid arthritis, Eticovo, is the second similar drug of etanercept. There are also fourth and fifth biological analogues of adalimumab. Third, fourth and fifth biological analogues of Herceptin are used to treat patients with HER2 mutation breast cancer or metastatic gastric cancer.

Policy superposition guidelines

In January 7th, the State Administration of food and Drug Administration issued the first article in 2020: "Circular on the guiding principles (Trial Implementation) of supporting real world evidence to support drug research and development (Trial Implementation)" (2020 first).

In the past, randomized controlled trials (RCT) were generally considered to be the gold standard for evaluating the safety and efficacy of drugs. However, their conclusions may be challenged in clinical practice, or there may be problems in traditional drug clinical trials that may be difficult to implement or require high time. Real world data (RWD) can be used not only to support drug research and development, but also to other scientific purposes. It is a powerful supplement to accelerate the development of pharmaceutical products. How to use real world evidence to evaluate the effectiveness and safety of drugs has become a hot topic in drug research and development and regulatory decision-making at home and abroad.

Regulatory agencies around the world such as FDA, the European Union drug administration EMA, and the Japanese drug and medical devices Administration (PMDA) have accumulated rich experience in the use of real world data for the safety evaluation of medical products. China's systematic use of real-world evidence to support drug regulatory decision-making is still in its infancy. However, the publication of this paper means that after China's accession to ICH, China's regulatory authorities are further in line with global standards.

The approval of new drugs in China in 2019 was "more blooming". In the field of treatment, in addition to cancer drugs, the new drugs approved by NMPA have covered rare diseases, diabetes, antiviral drugs such as hepatitis C, influenza, immunology, nervous system, urinary system, skin diseases and so on.

In addition to speeding up the introduction of new drugs into China by foreign pharmaceutical companies, local enterprises have also been approved in many diseases. Hausen pharmaceutical and Heng Rui medicine both "Mei Kai two degrees", in 2019, two new drugs were approved. Hausen approved drugs. First, 1 kinds of innovative drugs, pegylated LnCP, were listed for adults to improve blood glucose control in patients with type 2 diabetes. Two is the treatment of chronic myeloid leukemia with fluamitinib mesylate, which is used to treat adult chronic myeloid leukemia patients with chronic Philadelphia chromosome positive.

In May, the PD-1 drug clalion bead monoclonal antibody was approved to become the fifth entry in the domestic PD-1 market. At the end of the year, the injection of RMA was used for sedation in routine gastroscopy. With the approval of ralezumab in December 27th, the domestic PD-1 approved 6 drugs. In addition to foreign capital Opdivo and Keytruda, the four domestic companies, except for Jun Shi's approved indications for melanoma, XinDa, Heng Rui and Baiji are all Hodgkin's lymphoma.

In addition, the first PD-L1 drug was also introduced in 2019. In December 10th, AstraZeneca PD-L1 drug Imfinzi, commonly known as "I", was approved to be marketed for the treatment of non resectable and III stage non-small cell lung cancer after concurrent chemoradiotherapy. AstraZeneca has recently announced another new oral potassium lowering drug, Zr sodium silicate, which has been approved for sale and is used to treat hyperkalemia in adults. "Hyperkalemia therapy has gone through a blank period of nearly 60 years, and is the first innovative drug in China."

The innovation of pharmaceutical enterprises in 2019 is another major achievement of the company's "sea going". Its self-developed anti-cancer drug BTK inhibitor, zerbu tinie (English Name: BRUKINSA zanubrutinib, English Name: zanubrutinib) was approved by the US FDA for the treatment of patients with mantle cell lymphoma who had received at least one therapy before, so as to achieve the "zero breakthrough" of China's original research and development of new drugs and become one of the 48 new drugs approved by FDA.

The new drug approved by local pharmaceutical companies and the "Ben Wei Mo" cream of Guan Hao bio were approved to be marketed for topical treatment of mild to moderate stable psoriasis vulgaris in adults. According to the three quarterly report of Guan Hao biology, the new drug Ben mod cream achieved 3 million 19 thousand and 100 yuan in the reporting period.

Of all approved domestic innovative drugs in 2019, the most controversial issue was the mango sodium capsule of Green Valley ("nine phase I"), which was used for mild to moderate Alzheimer's disease and improved cognitive function. But the doubt from the medical profession has been constantly involved, including its clinical trial design, principles and mechanisms, test data, and Green Valley historical products. In December 29th, the nine phase was officially launched, priced at 895 yuan per box. According to the drug instructions, the monthly cost is about 3580 yuan, which is higher than that of donepezil and memantine.

In 2018, the total value of China's pharmaceutical market reached US $130 billion, still the second largest pharmaceutical market in the world after the United States. According to IQVIA data, in 2018, the total sales volume of China's hospital drug market reached 771 billion 800 million yuan, an annual growth rate of 3.3%. The annual sales of multinational pharmaceutical companies and local pharmaceutical enterprises were 208 billion 400 million and 563 billion 400 million yuan respectively.

In 2018, global cancer drug expenditure nearly $150 billion, an increase of 12.9% over the past 5 years, showing a two digit growth rate. The cost of cancer drugs in the United States has doubled since 2013, and spent more than 56 billion dollars in 2018. China is still the leader in spending and growth in emerging markets. In 2018, the scale of China's oncology treatment market was as high as about 9 billion US dollars, with an annual growth rate of 11.1%. Cancer drugs increased by 23.6% to $6 billion 300 million.

But at the same time, China's pharmaceutical industry has come to a new crossroad.

In 2019, the Chinese market was still regarded as the engine of growth by the major pharmaceutical companies. It is a year of great change in China's health industry. It is a year when the policy continues to continue to radiate, and it is also a year of continuous transformation in the industry.

From the beginning of the survey of health products, the consistency evaluation of generic drugs, the expansion of quantity purchase, the monitoring of auxiliary drugs to the new round of medical insurance directory adjustment and negotiation, the impact of the heavy policy superimposed on drug and control fee strictness, medical insurance payment reform and drug proportion control has been restricted. Every ring in the medical industry from manufacturing enterprises, circulation enterprises to medical institutions, medical insurance regulators, doctors and patients are faced with different transformation needs.

Transformation is not easy. No ability to participate in the volume of procurement enterprises, no funds to do generic drug consistency evaluation of enterprises, lack of money and lack of ability to do innovative transformation of enterprises, get together to do the same kind of "innovative medicine" enterprises, control fees policy leading to the main business profitability decline in enterprises, compliance management, lack of operation capacity of enterprises...... In this round of transformation tide will face "cold winter".