2024Rare Disease Industry Trend Observation Report Release Conference
2024year2month28day,2024As the International Rare Diseases Day approaches in 2019, Frost & SullivanFrost & SullivanFrost & Sullivan, in collaboration with the Beijing Pain Relief Challenge Public Welfare Foundation (referred to as the 'Pain Relief Challenge Foundation'), jointly organized the2024The release conference of 'China Rare Diseases Industry Trend Observation Report' (hereinafter referred to as the 'Report') was successfully held online.
"The2024'Trend Observation Report on the Rare Diseases Industry in China' follows the release of the joint report by Frost & Sullivan and the PATH Foundation titled2023China Rare Diseases Industry Trend Observation Report》 and2022The third annual trend observation report following the 'China Rare Disease Industry Trend Observation Report'.
The Report aims to review and sort out China2023The new progress in the field of rare diseases in China in 2021, including policy support, diagnosis and treatment, special medical foods, drug research and development, investment and mergers & acquisitions, and future prospects, is comprehensively outlined in this section.2023The progress made over the year, the industry challenges faced, and prospects for China's comprehensive rare disease service system are outlined. It provides a panoramic overview of the industry to all sectors of society, with the hope of benefiting policymakers, advocates, practitioners from relevant institutions, patients and their families dealing with rare diseases, and other stakeholders in this field.
2018year5month11day,2023year9month18On the same day, ministries and commissions including the National Health Commission issued the 'First Batch of Rare Diseases Catalogue' and the 'Second Batch of Rare Diseases Catalogue', which include various rare diseases.207Variety (First Batch121kind, second batch86The development of the 'Rare Diseases Catalogue' has provided an important basis for the formulation of various rare disease policies in China. Ma Tao, Secretary-General of the Pain Challenge Foundation, delivered an opening speech. It has been ten years since the Ice Bucket Challenge, and rare diseases are increasingly recognized by more people. In recent years, the rare disease industry in China has developed rapidly. The patient community has made unprecedented progress in diagnosis, treatment, and medication security. He called on all sectors of society to join hands to promote the development of China's rare disease industry. Li Qian, Consulting Director of Frost & Sullivan's Life Sciences Business Unit in Greater China, attended the press conference and shared insights on the current development status and future trends of the rare disease industry.
The accessibility of drugs for patients with rare diseases has been further improved, with multiple parties contributing to the diagnosis and treatment of rare diseases.
At the press conference, Guo Jinchuan, Information Research Director at the Foundation for the Fight Against Illnesses, interpreted the Report from the perspective of policy development.2023In [year], China released the 'Second Batch of Rare Diseases Catalogue', bringing the total number of rare disease catalogues in China to207Based on the 'First Batch of Rare Diseases Catalogue' and the 'Second Batch of Rare Diseases Catalogue', the number of rare disease drugs that have been launched in China and included in medical insurance has reached112plant, cover64The introduction of this rare disease has greatly improved the accessibility to medication for patients with rare diseases.
2023In the same year, several policies were introduced domestically to promote the research and development, imitation of drugs for rare diseases, facilitate drug market launch, and accelerate the use of new rare disease treatment drugs by enabling patients to access them through filing in specific regions. Under the new policy environment, diverse social forces participate in ensuring access to medical treatment and drugs for rare diseases. Local governments such as Shenzhen actively explore upgrades to benefit-sharing insurance, and public welfare and charitable organizations represented by the Pain Challenge Foundation also actively participate. Through the 'Rare Disease Medical Assistance Program', they help ensure that patients with rare diseases have access to medication. In addition, pharmaceutical companies have also carried outPAP(Patient Assistance Program) andTPAThird-party service projects have also alleviated the medication burden on patients to a certain extent. The new policy, combined with diverse social forces, is actively accelerating the construction of multi-level protection for rare diseases.
The R&D and launch of rare disease drugs in China are experiencing new growth, with multiple trends emerging in the field of rare diseases.
Li Qian, consulting director of the Life Sciences Practice Group at Frost & Sullivan Greater China, said that China is actively constructing a multi-level rare disease drug development strategy to promote the market launch of rare disease drugs.2023A total of [X] companies were listed in China in [Year]45Rare disease medication, among which15The product was listed through the priority review and approval process. Currently, there is also such a product in China.27Rare disease drugs are in the process of applying for marketing authorization, and the number of rare disease drug pipelines that have entered clinical trials has also reached252items, with the number of pipelines entering Phase III clinical trials reaching70item. And2022Year-on-year,2023In the field of rare diseases, drug research and development cover a wider range of diseases and involve more drugs.
Globally, the rare disease sector is also showing a diversified development trend. On one hand, the global rare disease capital market has continued to heat up, whether it is Amgen278Yi acquired Horizon, or Merck & Co. did108Yi's AcquisitionPrometheus Biosciences, and the amount is in2023The biopharmaceutical industry ranks among the top in the country, and many multinational companies have established drug research and development cooperation models in the rare disease field, including Novartis, Sanofi, AstraZeneca, etc., with capital driving development in this area; on the other hand, in terms of technological innovation, whether it is small nucleic acid drugs or gene therapy drugs, they have all developed well in the rare disease field. Currently, there are marketed small nucleic acid drugs including73%The indication is for a rare disease; at the same time, many Chinese companies have also deployed innovative therapies in the field of rare diseases, with some drug pipelines entering phase III clinical trials. It is believed that with the entry of Chinese companies, the accessibility of rare disease drugs in China will be further improved, and there is hope that it will reduce the medication burden on patients with rare diseases in the future.
Li Qian stated that the research and development of rare disease drugs has become one of the important sectors in China's biopharmaceutical research and development. In the future, Frost & Sullivan will continue to pay attention to the field of rare diseases, keep abreast of industry trends, continuously publish blue books on biopharmaceuticals and the healthcare industry, and continue to work with various parties to help the biotechnology industry develop healthily and rapidly.
Multiple forces converge to jointly explore new trends in policies and industrial development in the field of rare diseases
2023In [year], the release of the 'Second Batch of Rare Diseases Catalogue' and a series of national policies have effectively promoted the development of the rare diseases field in China. At the press conference, Wang Chenguang, former dean of the Law School at Tsinghua University, Zhang Xue, academician of the Chinese Academy of Engineering and secretary of the Party Committee of Harbin Medical University, Zhao Kun, researcher at the National Health Commission's Comprehensive Evaluation Center for Drugs and Health Technologies, Yang Yue, dean of the Institute of Pharmaceutical Regulatory Science at Tsinghua University, and Mao Ningying, deputy dean of the International Business School of Pharmacy at China Pharmaceutical University, discussed “2024Where is the direction of rare disease policy going? A dialogue was held, chaired by Wang Yigou, founder and vice-chairman of the Pain Challenge Foundation. The panelists believe that China will further improve relevant regulations and policies in the field of rare diseases, introduce further promotional policies, accelerate drug research and development in the rare disease sector, and further protect the rights and interests of patients with rare diseases.
Regarding the development of the rare disease industry, Zhang Min, Vice President of Takeda China, Zhu Jingyan, Vice President of BeiGene, Yu Lei, Head of Rare Diseases and Hematology Business at Sanofi (China), and Shenxi Fusheng Biopharmaceutical Co., Ltd.CEOSheng Jian and Fan Jing, Head of Digital Marketing at JD Health's Healthcare Business Unit, were hosted by Feng Lan, Secretary-General of the China Healthcare Innovation Promotion Association, to discuss “2024Where is the rare disease industry heading? ” A dialogue was held. Industry representatives and platform leaders from overseas and domestic regions shared their views.2024In 2023, China's rare disease industry will further develop, and specific views and opinions on related trends will be exchanged.
Finally, all the participating guests expressed their expectations and hopes for the 'Report'. They believe that there have been new trends and changes in the field of rare diseases every year in recent years. The 'Report' can present an intuitive, continuous, and comprehensive overview of the overall situation, reflecting policy trends and market development. It is conducive to increasing the attention of all sectors of society to the field of rare diseases and ultimately benefiting more patients with rare diseases.
Dr. Wang Xin, Global Partner and Chairman of Frost & Sullivan Greater China, said, "We aim to continuously promote this research, leverage our expertise, and work with all parties to improve the healthcare environment for patients with rare diseases, enhance their quality of life, and contribute to and drive the development of the rare disease industry in China." As a global leading growth consulting firm, Frost & Sullivan is always actively fulfilling its corporate social responsibility of "making the world a better place through growth," continuously paying attention to the rare disease community, deeply studying its policies and industry progress, and actively advocating for more people of vision to join public welfare initiatives, so as to contribute a modest effort to a better society.
