On February 27th, as the 15th International Rare Diseases Day approached, Frost & Sullivan, a global growth consulting firm dedicated to 'making the world a better place through growth' (hereinafter referred to as 'Frost & Sullivan'), joined hands with the Beijing Pain Challenge Public Welfare Foundation (hereinafter referred to as 'Pain Challenge Foundation') to officially release the '2022 China Rare Diseases Industry Trend Observation Report' (hereinafter referred to as 'the Report').
According to statistics, there are currently more than 7,000 known rare diseases globally, with the number of patients suffering from rare diseases exceeding 300 million. In China alone, there are approximately nearly 20 million patients fighting against rare diseases.
Source: Official website of International Rare Diseases Day
Rare diseases, despite having a low incidence rate, carry a heavy burden on patients. Patients often face many difficulties. The first is 'difficulty in diagnosis', with a tortuous medical process and the disease being hard to confirm; the second is 'difficulty in treatment', as only 5% of rare diseases globally have effective treatment methods; and finally, there is 'difficulty in medication'. Even after an accurate diagnosis and treatment, patients may not be able to afford or obtain medications, placing a heavy burden on patients and their families.
In recent times, the rare disease community has increasingly received attention from the state and all sectors of society. Multiple departments including national health, drug supervision, medical insurance, and technology have collaborated to support rare disease diagnosis and treatment through measures such as establishing a rare disease catalog, setting up a national rare disease diagnosis and treatment collaboration network, encouraging and supporting the research and development of rare disease treatment drugs, prioritizing the review and approval of rare disease drugs, and promoting the inclusion of rare disease drugs in medical insurance. In addition, social entities such as patient organizations, commercial insurance companies, charitable enterprises, and philanthropic institutions are also working together to explore multi-party co-payment models, alleviate the treatment burden on rare disease patients, and improve the accessibility of drugs (especially high-value ones).
At the end of 2021, the 'astronomically expensive life-saving drug' nusinersen injection, priced at '700,000 per injection,' was included in medical insurance, once again drawing public attention to the rare disease community. With the Chinese government's determination and efforts not to abandon any medication group and to serve patients, remarkable progress has been made in the diagnosis, treatment, and protection of rare diseases in China in recent years; however, overall, there are still diverse unmet needs in aspects such as diagnosis, treatment, protection, rehabilitation, and social integration for the rare disease community.
Against this backdrop, the '2022 China Rare Disease Industry Trend Observation Report' was born. It aims to review and sort out the development of rare diseases in China in terms of diagnosis and treatment, medication, support, and innovative services. It outlines the problems faced and progress made in this field over the past, and offers prospects for the comprehensive service system for rare diseases in China. It is hoped that it will be beneficial to policymakers, advocates, relevant institutions and practitioners, patients and their families, as well as the general public concerned about rare diseases in China.
On February 27th, Mr. Mao Hua, Partner and Managing Director of Frost & Sullivan Greater China, was invited to deliver a keynote speech at the event marking the first anniversary of the launch of the Rare Disease Care Center at JD Pharmacy.
Mr. Mao Hua pointed out that in recent years, the number of rare disease drugs approved has increased. In China, there are 87 drugs with clearly specified indications for rare diseases, covering 43 types of rare diseases. Among them, as of the end of 2021, after the national medical insurance negotiations, 58 drugs have been included in the national medical insurance, covering 29 types of rare diseases. The dawn of medication access for Chinese patients has already appeared.
In January 2022, the National Medical Products Administration issued the 'Technical Guidelines for Clinical Research and Development of Drugs for Rare Diseases', which relatively relaxed the requirements for clinical trials.Positive policies will continue to promote pharmaceutical companies in developing rare disease drugs;In addition, the success rate of orphan drug clinical research and development is higher than that of drugs for other diseases. Looking at the entire cycle from research and development to market launch, the success rate from Phase I clinical trials to approval for orphan drugs is 17.0%, while the success rate for all disease drugs is only 7.9%, which is half that of orphan drugs.Higher R&D success rates will also boost the confidence of pharmaceutical companies in R&D, motivating them to carry out research and development of rare disease drugs.
On the other hand, Mr. Mao Hua believes that China can draw on foreign regulatory systems to gradually improve its rare disease drug research and development (R&D) and support system. Although the state's attention to the rare disease community is increasing, and a series of supportive policies have been introduced, such as encouraging R&D of rare disease drugs, prioritizing their review and approval, and providing tax incentives, overall, there is currently no programmatic legislation similar to the Orphan Drug Act in China.Programmatic laws can systematically standardize the definitions of concepts related to rare disease drugs, as well as drug research and development, introduction, production, supply, assurance, market supervision, etc., further promoting the innovative research and development and introduction of orphan drugs for rare diseases.
In the National Medical Insurance Catalogue for 2021, seven new drugs for rare diseases were added. Notably, the high-value rare disease drugs Nusinersen Sodium Injection and Agatrozyme & Alpha Injection are included in the medical insurance, reflecting the country's respect for the life and health of every patient. While taking into account patients' needs and the affordability of medical insurance funds, the coverage has been expanded to be more inclusive. According to a Frost & Sullivan survey, China is currently gradually constructingLed by the government and centered on the national basic medical insurance, multiple-party protection systems have been established in various regions such as Shanghai, Zhejiang, Shandong, Foshan and Guangzhou in Guangdong Province.
Currently, multiple social forces are continuously promoting the guarantee of rare disease medications. For example, charitable organizations drive more social forces to participate in 'multi-party payment'; innovative payment platforms start from the perspective of patients, creating multi-level patient services and security systems; commercial insurance is also constantly exploring ways to pay for rare disease medications; caring enterprises fill the gaps in security costs, sharing risks to assist in medication security.
In addition, large pharmaceutical companies are also actively deploying in the field of rare diseases. For example, Takeda Pharmaceutical spent up to $65 billion acquiring the British pharmaceutical company Shire. In recent years, capital has continuously flowed into the research and development of rare disease drugs, innovative biotechnology platforms, internet technology, and innovative healthcare services, promoting the construction of a rare disease ecosystem. Resources are collaborating with each other, injecting vitality into the rare disease industry chain.
Mr. Mao Hua said that in the future, Frost & Sullivan will continue to keep track of the latest industry trends, continuously release white papers on biomedicine and healthcare industries, and contribute to the healthy and rapid development of the industry.
At the press conference, Wei Xing, CTO of Huanuo Omics, proposed that policy support is at the core of the development of the rare disease industry, while technological innovation helps with early screening, prevention, diagnosis, and treatment of rare diseases. Chen Jun, COO of Weisheng Pharmaceutical, stated that solving rare diseases is a long-term battle. As a responsible innovative pharmaceutical company, Weisheng Pharmaceutical is willing to contribute to the accessibility, treatability, and availability of rare disease patients in China.
(From left to right: Wu Daling, Head of Prescription Drug Business Unit at JD Health Medicine Business Department; Wei Xing, CTO at Huanuo Aomei; Chen Jun, CCO at Weisheng Pharmaceutical; Guo Jinchuan, Director of Information Research at the Pain Challenge Foundation; Mao Hua, Partner and Managing Director of Frost & Sullivan Greater China)
It is worth mentioning that the report innovatively includes:(1) Promote legislation related to 'rare diseases' and 'rare disease drugs', (2) Establish a systematic top-level design and coordination mechanism for rare diseases, (3) Establish a comprehensive support system for rare disease screening, diagnosis, treatment, management, and rehabilitation, (4) Establish a comprehensive value evaluation system for rare disease drugs and services, (5) Prioritize the inclusion of rare disease medications in medical insurance, establish a national special medical insurance system for rare diseases, encourage local pilots, (6) Government guidance, explore mechanisms for diverse social forces to participate, (7) Explore diversified innovative payment strategies for medical security and services, (8) Establish patient participation guidelines for promoting drug research and development and access standards, (9) Special policy support for special medical foods, (10) Encourage the development of rare disease medical innovation service platformsTen aspects were discussed, offering pertinent suggestions and earnest prospects for the comprehensive rare disease service system in China.
This report has also received joint recommendations and messages from experts from industry, academia, and research institutions, including: Wang Chenguang, a director of the Pain Challenge Foundation and former dean of the Law School at Tsinghua University; Shi Luowen, director of the International Research Center for Pharmaceutical Management at Peking University; Xu Kaifeng, professor at the Department of Respiratory and Critical Care Medicine at Peking Union Medical College Hospital and chairman of the Respiratory Disease Branch of the China Rare Diseases Alliance; Wang Yigou, founder and vice-chairman of the Pain Challenge Foundation; Dong Dong, head of the Rare Diseases Real-world Data Research Experiment at the Shenzhen Research Institute of The Chinese University of Hong Kong; Wei Xing, CTO of Huanuo Aome; Jin Enlin, CEO of JD Health; Lu Anbang, CEO and director of Visen Pharmaceuticals, etc. These recommendations and messages are highly valuable for industry reference.
As Wang Chenguang said: 'Rare diseases are not just a matter for patients, their families, a few medical staff, and pharmaceutical companies. They are a major issue concerning the entire society. Because caring for the minority reflects great love, working together can promote social civilization, and common prosperity can leverage institutional advantages.'The '2022 China Rare Diseases Industry Trend Observation Report' not only reveals the current challenges posed by rare diseases to society as a whole but also calls for the conscience and participation of the entire society. It advocates for the establishment of an all-round system to address rare diseases, which will strongly promote the development of the Healthy China initiative."
Frost & Sullivan sincerely looks forward to working with all sectors to pay attention and jointly ensure the well-being of people living with rare diseases, striving to help them enjoy a colorful life. We will also continue our commitment to research in the field of rare diseases, actively fulfill our social responsibilities, and continuously contribute to the construction of 'Healthy China 2030'!
