Article from PhIRDA｜Bi Jingquan: To encourage the R&D of drugs for rare diseases, we need to study and formulate special policies that conform to the characteristics of rare diseases
China Pharmaceutical Innovation and Research Development Association (PhIRDA) Published in Beijing on October 31, 2022 at 09:32
“China Rare Disease Conference 2022” sponsored by China Alliance for Rare Diseases (CHARD) and China Pharmaceutical Innovation and Research Development Association (PhIRDA) was held in Beijing on October 29, 2022. At the opening ceremony, Comrade Bi Jingquan, deputy director of the Economic Affairs Committee of the Chinese People’s Political Consultative Conference and executive vice president of China Center for International Economic Exchanges, delivered a speech and put forward nine suggestions on the policies for approval and reimbursement of drugs for rare diseases.
Bi Jingquan pointed out, in recent years, the Party and the state have attached great importance to patients with rare diseases, and introduced a number of policies to ensure the diagnosis and treatment of rare diseases and the R&D of orphan drugs, and in this way, China has made great progress in the diagnosis and treatment of rare diseases. Since 2015, 35 drugs for 17 rare diseases have been approved for marketing in China, and 67% of the marketed drugs for rare diseases have been included in the national reimbursement drug list, greatly reducing the financial burden of patients for drug use. The 20th National Congress of the Communist Party of China further defined the strategic goals of China’s development, the task of building a healthy China, the strategy for rejuvenating the country through science and education, and the core position of scientific and technological innovation in the overall situation of China’s modernization. It can significantly guide the study and discussion of the diagnosis and treatment of rare diseases.
Bi Jingquan pointed out, the R&D of drugs for rare diseases is basically not financially feasible because it faces difficulty in patient recruitment, high development risks, small market sizes and other problems. The R&D of drugs for rare diseases is scientifically meaningful and needed in the market, but actually hard to bring profits; therefore, we should study and formulate special policies that conform to the characteristics of rare diseases. Encouraging enterprises to develop drugs for rare diseases is the key to meeting the need. To this end, Bi Jingquan specially put forward the following suggestions:
- We should attach great importance to the R&D of drugs for rare diseases. The treatment of rare diseases concerns more than 100 million persons. We should address the need for drugs for rare diseases from the perspective of “winning people’s hearts” and “ensuring people’s health”, and by implementing the strategy for scientific and technological innovation.
- We should stabilize enterprises’ market expectation of developing drugs for rare diseases.Both patent system and data protection system are designed to protect intellectual property rights, and essentially to reward innovators with market exclusivity. Such market exclusivity must include independent pricing. Without independent pricing, market exclusivity is meaningless, and patent and data protection lose their value. For innovative drugs with full market competition, we should give full play to the decisive role of the market in resource allocation, instead of price control by the government.
III. Efforts should be made to reduce enterprises’ R&D costs. On the basis of scientificity, the number of subjects in clinical trials of drugs for rare diseases should be appropriately reduced. In case of overseas approved drugs for the treatment of rare diseases in children, overseas declared clinical data should be allowed to be used for marketing in China. If there is a lack of racial difference data, clinical trials with a small number of samples can be conducted to accelerate the approval of marketing.
- We should take multiple measures to reduce patient burden. Patients’ right to pay medical expenses from medical insurance as the insurance applicants should not be deprived of just because some drugs for rare diseases have high prices. It is suggested to study the change in the fixed proportion of payment from medical insurance and reduce the proportion of medical insurance payment for some expensive drugs for rare diseases so as to strike a balance between encouraging the R&D of drugs for rare diseases and the pressure on payment from medical insurance fund. Patients with rare diseases should not be excluded from payment of medical expenses from medical insurance. For too expensive drugs, it is suggested to study the upper limit of payment of medical expenses from medical insurance: The part within the limit is to be paid from medical insurance according to regulations, and the part exceeding the limit is to be shared by patients, commercial insurance, social charity, fund subsidies and government assistance.
- We should extend the period of market exclusivity of drugs for rare diseases. At present, the data protection period for drugs containing new chemical components is six years. It is suggested to extend the data protection period for drugs for rare diseases to ten years in view of the practices of Japan and the European Union.
- We should exempt the income tax on enterprises’ income from sales of drugs for rare diseases. The financial authority has launched preferential policies such as 3% value-added tax on drugs for rare diseases and income tax offsetting/exemption due to R&D expenses. We should further study the proposal that the income from sales of drugs for rare diseases shall be exempted from corporate income tax.
VII. It is necessary to establish funds for R&D of drugs for rare diseases. It is suggested that governments at all levels should set up funds for R&D of drugs for rare diseases, and subsidize projects of R&D of drugs for rare diseases. Non-government capital should be encouraged to set up funds for rare diseases to fund the development of drugs for rare diseases.
VIII. The list of rare diseases should be updated and published regularly. Diagnosis of rare diseases is an important symbol of medical progress. We should publish the list of rare diseases regularly and add newly discovered rare diseases to the list in time according to medical progress, guide medical staff in improving their ability of early detection and treatment, and guide scientific researchers in R&D of diagnostic reagents and therapeutic drugs.
IX.We should study and formulate laws related to rare diseases and protect the rights and interests of patients with rare diseases through legislation.
About CANbridge Pharmaceuticals Inc.
CANbridge Pharmaceuticals Inc. (HKEX:1228) is a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 11 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. CANbridge is also building next-generation gene therapy development capability through a combination of collaboration with world-leading researchers and biotech companies and internal capacity. CANbridge’s global partners include Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, the University of Washington School of Medicine and the UMass Chan Medical School.
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