Kinase modulators approved by FDA in 2022 / 药学学报

The FDA approved a total of 37 new drugs in 2022, including 22 new molecular entities and 15 new biological products. This is the year with the lowest number of new drugs approved by the FDA since 2017. Among these approved drugs, 21 new drugs belong to the "first-in-class" category, accounting for 56% of the total approved drugs, which is the highest ratio in the past 10 years. Among the drugs approved in 2022, there are 5 small molecule kinase modulators, including the tyrosine kinase 2 (TYK2) allosteric inhibitor deucravacitinib, the first oral pyruvate kinase (PK) activator mitapivat, the Janus kinase 1 (JAK1) selective inhibitor abcrocitinib, the JAK2 selective inhibitor pacritinib and the broad-spectrum fibroblast growth factor receptor (FGFR) inhibitor futibatinib. This review briefly describes the discovery background, research and development process, synthesis routes and clinical efficacy and safety of small molecule kinase modulators approved by the FDA in 2022, hoping to provide ideas and methods for further research on kinase modulators.

The Development of Chinese Herbal Formulae for Non-severe COVID-19 Based on Artificial Intelligence Technology and Investigation of Its Action Mechanisms / 中医杂志

ObjectiveTo develop traditional Chinese medicine （TCM） formulae for the treatment of nonsevere coronavirus disease 2019 （COVID-19） and to explore its anti-inflammatory mechanism. MethodsThe dysregulated signaling pathways were determined in macrophages from bronchoalveolar lavage fluid of COVID-19 patients and in lung epithelial cells infected with SARS-CoV-2 in vitro based on transcriptome analysis. A total of 102 TCM formulae for the clinical treatment of nonsevere COVID-19 were collected through literature. The pathway-reversing rates of these formulae in macrophages and lung epithelial cells were evaluated based on signature signaling pathways， and the basic formula was determined in conjunction with TCM theory. The commonly used Chinese materia medica for nonsevere COVID-19 were summarized from the 102 TCM formulae as abovementioned. And together with the screening results from the Pharmacopoeia of the People's Republic of China， a “Chinese materia medica pool” was esta-blished for the development of TCM formulae for COVID-19. The regulatory effects of each herb on signaling pathways were obtained based on targeted transcriptome analysis. Oriented at reversing dysregulated signaling pathways of COVID-19， the calculation was carried out， and the artificial intelligent methods for compositing formulae， that are exhaustive method and parallel computing， were used to obtain candidate compound formulas. Finally， with reference to professional experience， an innovative formula for the treatment of nonsevere COVID-19 was developed. The ethanol extract of the formula was evaluated for its anti-inflammatory effects by detecting the mRNA expression of interleukin 1b （Il1b）， C-X-C motif chemokine ligand 2 （Cxcl2）， C-X-C motif chemokine ligand 10 （Cxcl10）， C-C motif chemokine ligand 2 （Ccl2）， nitric oxide synthase 2 （Nos2）， and prostaglandin-endoperoxide synthase 2 （Ptgs2） using reverse transcription-quantitative polymerase chain reaction （RT-qPCR） in RAW264.7 cells treated with lipopolysaccharide （LPS）. ResultsIn macrophages and lung epithelial cells， 34 dysregulated signaling pathways associated with COVID-19 were identified respectively. The effects of the 102 formulae for clinical treatment of nonsevere COVID-19 were evaluated based on the dysregulated signaling pathways and targeted transcriptome， and the result showed that Yinqiao Powder and Pingwei Powder （银翘散合平胃散， YQPWP） ranked first， reversing 91.18% of the dysregulated signaling pathways in macrophages and 100% of the dysregulated signaling pathways in lung epithelial cells. Additionally， YQPWP had the function of scattering wind and clearing heat， resolving toxins and removing dampness in accordance with the pathogenesis of wind-heat with dampness in COVID-19. It was selected as the basic formula， and was further modified and optimized to develop an innovative fomula Qiaobang Zhupi Yin （翘蒡术皮饮， QBZPY） based on expert experience and artificial intelligence in composing formulae. QBZPY can reverse all the dysregulated signaling pathways associated with COVID-19 in macrophages and lung epithelial cells， with the reversing rates of 100%. The chief medicinal of QBZPY， including Lianqiao （Fructus Forsythiae）， Xixiancao （Herba Siegesbeckiae） and Niubangzi （Fructus Arctii）， can down-regulate multiple signaling pathways related with virus infection， immune response， and epithelial damage. RT-qPCR results indicated that compared with the model group， the QBZPY group down-regulated the mRNA expression of Il1b， tumor necrosis factor （Tnf）， Cxcl2， Cxcl10， Ccl2， Nos2 and Ptgs2 induced by LPS in RAW264.7 cells （P＜0.05 or P＜0.01）. ConclusionBased on targeted transcriptome analysis， expert experience in TCM and artificial intelligence， QBZPY has been developed for the treatment of nonsevere COVID-19. The ethanol extract of QBZPY has been found to inhibit mRNA expression of several pro-inflammatory genes in a cellular inflammation model.

The application of positron emission tomography in the research and development of central nervous system drugs / 中国临床药理学与治疗学

Positron emission tomography (PET) now plays an important role in the research and development (R&D) of central nervous system (CNS) drugs. PET could characterize the biodistribution, pharmacokinetics, and receptor binding of CNS drugs quantitatively. The present review summarized the quantitative methods of PET used in the pharmacokinetics and receptor occupancy analysis of CNS drugs. Moreover, the present review listed various applications of PET supporting R&D of CNS drugs, which could provide a new direction for the R&D of CNS drugs.

Analysis and suggestions on the development trend of drug innovation in China / 中国药科大学学报

@#In order to comprehensivehy evaluate the stage of China in the global drug innovation, to further optimize the environment for drug innovation in China, and to unleash the vitality of drug innovation, this article mainly analyzes through comparison China''s situation of drug innovation in global competition from such perspectives as the current situation of the global drug R&D innovation market, R&D investment, product pipeline, policy support, and development trends, combined with the characteristics of China''s drug innovation development.It can be seen that China''s pharmaceutical innovation is faced with such practical problems as lagging behind some developed countries in terms of innovation development, companies bunching into research and development innovation, sudden research and development rush, and excessive dependence on capital markets for pharmaceutical innovation.Accordingly, this paper puts forward suggestions on continuously improving China''s new drug innovation environment, rationally selecting differentiated competition and new tracks, reasonably formulating drug innovation development strategies, guiding capital to return to innovative research and development, and constructing a "double cycle" strategy for drug innovation.

Reflections on Research and Development Institutions Becoming Medical Device Registrants / 中国医疗器械杂志

As the special subject of the applicant for registration of medical device, the research and development institutions have insufficient conditions and abilities to become medical device registrants, and there are certain difficulties in the actual registration application process, such as not clearing the certification path for the research and development institutions to hold the certificate. In view of the existing problems, by comparing the path of medicine research and development institutions to become medical device registrants and combining with the actual medical device industry to give relevant suggestions, including improving quality management over the whole life cycle of medical devices, quality and safety responsibility ability of research and development institutions, establishing the registration and certification path of research and development institutions, supporting laws and regulations, etc., so as to ensure that the research and development institutions become medical device registrants successfully.

Current status and new progress of drug therapy for premature ejaculation / 现代泌尿外科杂志

Premature ejaculation (PE) is the most common male sexual dysfunction with a high incidence, which seriously affects the relationship between a husband and wife and family harmony. Drug therapy is a first-line treatment for PE patients with premature ejaculation, and has achieved good efficacy, but the clinically available drugs are single and the abandonment rate is high. Coupled with the ineffective treatment of some patients, new drug research and development is imminent. This paper systematically reviews the current status of drug treatment for premature ejaculation, focusing on the research and development of new drugs and research progress in order to provide a reference for clinicians.

Advances in COVID-19 vaccine development and clinical application / 中国药理学通报

Severe acute respiratory syndrome coronavirus-2 (SARS-COV-2) has spread to more than 220 countries, including China. With the deepening of research on the viral molecular structure and pathogenesis of SARS-COV-2, various prevention strategies and treatment plans for coronavirus disease 2019 (COVID-19) are being actively explored. Pharmaceutical companies and scientific research institutions in many countries or regions are also actively engaged in the research and development of COVID-19 vaccines. More than 300 COVID-19 vaccine candidates are currently under study globally, and more than 10 have entered phase III clinical trials or are authorized for emergency use. Five COVID-19 vaccines have been approved for use in China, including three inactivated vaccines, one type 5 adenovirus vector vaccine and one recombinant protein vaccine (from CHO cell). The COVID-19 vaccines developed by Oxford University/Astrazeneca and Johnson & Johnson are adenovirus vector vaccines. Emergency licensing of mRNA vaccines developed by Pfizer and BioNTech, and mRNA vaccines developed by Moderna also offer potential prospects for COVID-19 prevention and control. This article reviews the development and clinical application of COVID-19 vaccines.

Metabolomics in drug research and development: The recent advances in technologies and applications

Emerging evidence has demonstrated the vital role of metabolism in various diseases or disorders. Metabolomics provides a comprehensive understanding of metabolism in biological systems. With advanced analytical techniques, metabolomics exhibits unprecedented significant value in basic drug research, including understanding disease mechanisms, identifying drug targets, and elucidating the mode of action of drugs. More importantly, metabolomics greatly accelerates the drug development process by predicting pharmacokinetics, pharmacodynamics, and drug response. In addition, metabolomics facilitates the exploration of drug repurposing and drug-drug interactions, as well as the development of personalized treatment strategies. Here, we briefly review the recent advances in technologies in metabolomics and update our knowledge of the applications of metabolomics in drug research and development.

Application progress of radiolabeled drugs in new drug research and development in China / 药学学报

The metabolism study of radiolabeled drugs plays an important role in the development of new drugs. It provides information on drug absorption, metabolism, tissue distribution and excretion, and plays an irreplaceable role in the metabolite safety evaluation and mass balance of new drugs. The new guidance draft on clinical trials of radiolabeled drugs recently released by the US FDA puts forward higher standards and has been widely concerned by the industry. In recent years, in the research and development of new drugs in China, 14C labeled drugs have been used to carry out clinical metabolism studies, which has overcome key technical bottlenecks and accumulated experience. This paper summarizes the above research progress, analyzes the existing problems, and preliminarily looks forward to the future technological development and application.

Application and development of systems biology in computer-aided drug design / 中国中药杂志

With the advances in medicine, people have deeply understood the complex pathogenesis of diseases. Revealing the mechanism of action and therapeutic effect of drugs from an overall perspective has become the top priority of drug design. However, the traditional drug design methods cannot meet the current needs. In recent years, with the rapid development of systems biology, a variety of new technologies including metabolomics, genomics, and proteomics have been used in drug research and development. As a bridge between traditional pharmaceutical theory and modern science, computer-aided drug design(CADD) can shorten the drug development cycle and improve the success rate of drug design. The application of systems biology and CADD provides a methodological basis and direction for revealing the mechanism and action of drugs from an overall perspective. This paper introduces the research and application of systems biology in CADD from different perspectives and proposes the development direction, providing reference for promoting the application.

ETCM v2.0: An update with comprehensive resource and rich annotations for traditional Chinese medicine

Existing traditional Chinese medicine (TCM)-related databases are still insufficient in data standardization, integrity and precision, and need to be updated urgently. Herein, an Encyclopedia of Traditional Chinese Medicine version 2.0 (ETCM v2.0, http://www.tcmip.cn/ETCM2/front/#/) was constructed as the latest curated database hosting 48,442 TCM formulas recorded by ancient Chinese medical books, 9872 Chinese patent drugs, 2079 Chinese medicinal materials and 38,298 ingredients. To facilitate the mechanistic research and new drug discovery, we improved the target identification method based on a two-dimensional ligand similarity search module, which provides the confirmed and/or potential targets of each ingredient, as well as their binding activities. Importantly, five TCM formulas/Chinese patent drugs/herbs/ingredients with the highest Jaccard similarity scores to the submitted drugs are offered in ETCM v2.0, which may be of significance to identify prescriptions/herbs/ingredients with similar clinical efficacy, to summarize the rules of prescription use, and to find alternative drugs for endangered Chinese medicinal materials. Moreover, ETCM v2.0 provides an enhanced JavaScript-based network visualization tool for creating, modifying and exploring multi-scale biological networks. ETCM v2.0 may be a major data warehouse for the quality marker identification of TCMs, the TCM-derived drug discovery and repurposing, and the pharmacological mechanism investigation of TCMs against various human diseases.

Research and development practice of traditional Chinese medicine based on network target theory and technology / 中国中药杂志

Network targets theory and technology have transcended the limitations of the "single gene, single target" model, aiming to decipher the mechanisms of traditional Chinese medicine(TCM) based on biological network from the perspective of informatics and system. As the core of TCM network pharmacology, with the development of computer science and high-throughput experimental techniques, the network target theory and technology are beginning to exhibit a trend of organic integration with artificial intelligence technology and high-throughput multi-modal multi-omics experimental techniques. Taking the network target analysis of TCM like Yinqiao Qingre Tablets as a typical case, network target theory and technology have achieved the systematic construction, in-depth analysis, and high-throughput multi-modal multi-omics validation of multi-level biological networks spanning from traditional Chinese and Western phenotypes to tissues, cells, molecules, and traditional Chinese and Western medicines. This development helps to address critical issues in the analysis of mechanisms of TCM, including the discovery of key targets, identification of functional components, discovery of synergistic effects among compound ingredients, and elucidation of the regulatory mechanisms of formulae. It provides powerful theoretical and technological support for advancing clinical precision diagnosis and treatment, precise positioning of TCM, and precise research and development of TCM. Thus, a new paradigm of TCM research gradually emerges, combining big data and artificial intelligence(AI) with the integration of human experience and scientific evidence.

Research advances on the role of epigenetic modifications in the long-term progression of alcoholic liver disease / 药学学报

There are more than 60 million alcoholic liver disease (ALD) patients in China, which has become a public health problem that cannot be ignored. Moreover, the social problem of "alcohol culture" is still hardly to solve, so that safe and effective prevention and treatment for ALD are in urgent need clinically. Previous studies on ALD have focused on the direct damaging effects of alcohol and its toxic metabolites, while recent studies have shown that the pathogenesis of ALD also include alcohol metabolic reprogramming and endogenous metabolites disorder. Although the endogenous metabolites have no direct toxicity, its long-term effect should not be ignored. These endogenous metabolites could change epigenetic modifications, cause widespread and persistent abnormal gene expression and signal pathway activation abnormally to promote metabolic reprogramming and stamp it as "metabolic memory", which manifest pathological changes and promote ALD, especially liver fibrosis/cirrhosis and liver cancer. Based on this, the article reviews the important epigenetic modifications caused by related metabolites in ALD and their associated effects. The role of traditional Chinese medicine (TCM) and its active ingredients in regulating epigenetics was also analyzed. The results suggest that regulation of epigenetics and alteration of "metabolic memory" may be a novel mechanism of TCM in the prevention and treatment of ALD.

Deep learning-based de novo drug design: principles, tools and practice / 药学学报

Design of structurally-novel drug molecules with deep learning can overcome the technical bottleneck of classical computer-aided drug design. It has become the frontier of new technique research on drug design, and has shown great potential in drug research and development practice. This review starts from the basic principles of deep learning-driven de novo drug design, goes on with the brief introduction to deep molecular generation techniques as well as computational tools and the analysis on representative successful cases, and eventually provides our perspective for future direction and application prospect about this technique. This review will provide ideas on new technique research and references for new drug research and development practice to which this technique is applied.

Bibliometrics of Sudanese scientific publications: Subjects, institutions, collaboration, citation and recommendations / Bibliometría de publicaciones científicas sudanesas: temas, instituciones, colaboración, citas y recomendaciones

Abstract Introduction: Sudan is the third largest country in Africa and has rich reserves of petroleum and other ground resources, but its per capita Gross Domestic Product is only $808 and researchers work in insufficient institutional facilities and with little funding. Previous studies about its scientific productivity have been limited to specific subjects and relatively short periods, with no large analyses until now. Objective: To analyze the scientific output of Sudan in depth, considering all research areas and several decades of scientific activity. Methods: We retrieved the documents with "Sudan" in field country in the Science Citation Index Expanded for the period 1900-2019. Results: We retrieved over 9 000 publications and found that most were articles; that citation was higher for review articles and book chapters, and that this index mostly covered articles in English. Beginning in 1972, the number of publications in this database has increased rapidly. The citation lifespan indicates slow growth in the Sudanese scientific literature, and collaboration is frequent both nationally and internationally, possibly because the scarce resources make collaboration almost compulsory. Most external collaboration is done with Saudi Arabia but citation is higher for articles resulting from international megaprojects, led by Europe and the USA, in which Sudanese researchers play secondary roles. Research focusses on applied technological subjects with little innovation value. Women play a smaller role in Sudanese science. Conclusions: Our recommendations for Sudanese science include increasing the number of women in leading research positions; providing funding directly to researchers (i.e., bypassing bureaucratic bodies); increasing basic research to avoid stagnation; training Sudanese researchers for leading positions; and identifying specific research areas where Sudan can lead in its region.

Resumen Introducción: Sudán es el tercer país más grande de África y tiene ricas reservas de petróleo y otros recursos terrestres, pero su Producto Interno Bruto per cápita es de solo $ 808 y los investigadores trabajan en instalaciones institucionales deficientes y con poca financiación. Los estudios previos sobre su productividad científica se han limitado a temas específicos y períodos relativamente cortos. Objetivo: Analizar la producción científica de Sudán en profundidad, considerando todas las áreas de investigación y varias décadas. Métodos: Recuperamos los documentos con "Sudán" como país de origen en el Science Citation Index Expanded para el período 1900-2019. Resultados: Hallamos más de 9 000 publicaciones y encontramos que la mayoría eran artículos; que fueron más citados los artículos de revisión y capítulos de libros, y que esta base de datos cubría principalmente artículos en inglés; desde 1972, el número de publicaciones en ella ha aumentado rápidamente. La vida útil de las citas indica un crecimiento lento en la literatura científica sudanesa, y la colaboración es frecuente tanto a nivel nacional como internacional, posiblemente porque los escasos recursos hacen que la colaboración sea casi obligatoria. La mayor parte de la colaboración externa se realiza con Arabia Saudita, pero hay más citas para los artículos resultantes de megaproyectos internacionales, dirigidos por Europa y Estados Unidos, en los cuales los investigadores sudaneses desempeñan papeles secundarios. La investigación se centra en temas de tecnología aplicada con poco valor de innovación. Conclusiones: Nuestras recomendaciones para la ciencia sudanesa incluyen aumentar el número de mujeres en altos puestos; proporcionar financiación directamente a los investigadores (sin pasar por organismos burocráticos); ir más allá de la investigación aplicada para evitar el estancamiento; capacitar al personal sudanés para puestos de liderazgo; e identificar áreas de investigación específicas donde Sudán puede liderar en su región.

Job burnout and associated influencing factors in employees of 7 research and development enterprises in Minhang District of Shanghai / 环境与职业医学

Background Job burnout is an early mental health condition caused by job stress and contributes to many negative effects on work and life. Employees of research and development (R&D) enterprises are exposed to constant pressure from innovation, production speed and sales expansion, and they are prone to burnout symptoms if such factors are not under effective control. Objective To evaluate the current situation of job burnout among employees of R&D enterprises in Minhang District of Shanghai and explore its influencing factors. Methods During November to December 2021, a cross-sectional study was developed and a convenient sampling method was used to enroll employees from 7 R&D enterprises in Minhang District of Shanghai. On the basis of voluntary participation with informed consent, a survey was conducted by using a self-made questionnaire (collecting data about general demographic characteristics, occupational characteristics, behavior and lifestyle), the Chinese version of the Concise Occupational Stress Questionnaire, and the Chinese version of the Maslach Burnout Inventory-General Survey. Occupational stress and its dimensions (job demand, job control, and social support) were divided into high, medium, and low levels according to tertiles. The positive rate of job burnout was reported according to score categorization (<1.5 refers to no job burnout, ≥1.5 refers to job burnout, where ≥1.5 and <3.5 refer to mild and moderate job burnout, and ≥3.5 refers to severe job burnout). Potential influencing factors of job burnout were evaluated by using one-way ANOVA, chi-square test, forward stepwise regression, and non-conditional binary logistic regression (α=0.05, two-sided test). Results A total of 3153 subjects were enrolled and 3014 samples were included in the analysis, with a valid response rate of 95.6%. Among the included subjects, 888 (29.46%) reported no job burnout, 1775 (58.89%) reported mild to moderate job burnout, and 351 (11.64%) reported severe job burnout. The mean of total job burnout score was 2.17±1.12, and the dimentional mean scores were 2.78±1.61 for emotional exhaustion, 1.60±1.60 for cynicism, and 4.05±1.57 for diminished personal accomplishment. Varied categories of sex, age, marital status, working position, sleep status, job demand, job control, and social support groups of workers resulted in significant differences in job burnout score. Compared with the low job demand group, the positive rate of job burnout was elevated in the medium and high job demand groups; the risk of job burnout in the medium job demand group was 1.42 (95%CI: 1.04-1.94) times higher, and that in the high job demand group was 2.64 (95% CI : 2.17-3.22) times higher versus the low job demand group. The risk of job burnout in the medium job control group was 1.35 (95%CI: 1.06-1.72) times higher versus the low job control group. Compared with the low social support group, job burnout was less reported in the other groups, and the OR (95%CI) values of the medium and high social support groups were 0.41 (0.31-0.53) and 0.15 (0.12-0.19) respectively. Conclusion The rate of reporting positive job burnout in R&D enterprises is high, which deserves sufficient attention. Relieving work pressure, increasing job control and social support, and maintaining adequate sleep are helpful to reduce job burnout.

Current Development Status and Consideration for Rare Hemorrhagic Disease Drugs / 罕见病研究

Hemophilia is the only rare hereditary hemorrhagic disorder included in the First Rare Diseases catalogue. However, rare bleeding diseases identified in the clinic are far more common than hemophilia. Most other rare hemorrhagic disorders have less effective treatment than hemophilia. Hemophilia has a history of successful drug development in rare hemorrhagic diseases, and the cycle between clinical research and drug development has been gradually realized. Drug research and pharmaceutical companies can refer to the drug research and development process in the field of hemophilia, learn from the experience of hemophilia drug research and develop treatments. The industry can increase drug development by strengthening basic research, focusing on the value of natural history research, the application of quantitative pharmacological tools and improving the efficiency of drug development to meet the urgent unmet medical needs of patients with rare hemorrhagic diseases.

Appraisal Concerns for the Clinical Research and Development of Drugs for Rare Diseases / 罕见病研究

The incidence of each of the rare disease is very low. The complexity and diagnosis difficulty of the rare disease lead to the difficulties in the clinical research and development (R&D) of drugs for rare diseases. There is an urgent clinical need for the drug development of rare diseases in China. Encouraging R&D of new drugs, particularly the innovative drugs with China's own independent intellectural property is the basis for solving the predicament in drug shortage in China.. In order to further improve the efficiency of clinical R&D of drugs for rare diseases, the National Medical Products Administration (NMPA), Center for Drug Evaluation (CDE) issued Technical Guidance for Clinical Research and Development of Drugs for Rare Diseases. This is the first guidance for rare diseases in China that is drafted from the standpoint of the clinical technology research and development.The guidance is the scientifitc thinking and framework for the drug developing enterprises to research and develop drugs for rare disease efficiently and appropriately by following drug developing protocols and relating to the special features of rare disease.This paper presents the concepts and rationale in the guidance for the appraisal of rare disease drug research and development.

Thoughts on the development of International Organization for Standardization standards for Traditional Chinese Medicine information models / 国际中医中药杂志

The development of the International Organization for Standardization (ISO) standard for Traditional Chinese Medicine (TCM) information model is needed to promote the coordinated development of TCM information research and application on a global scale, and it is of great significance to promote the use of TCM data. Based on the 13 years of experience in the development of the ISO standard for the TCM information model, we introduced and discussed the development background, development platform, internationality and classification of the standard development. We determined the necessity of the ISO standard of TCM information model to be independent of modern medical information model standard, the necessity of developing standards on a large scientific platform, and the necessity of health informatics experts participating in standard development. We summarized the main points of international and domestic coordination. We emphasized the balance between the overall layout and new areas development, and the importance of application and promotion.

Ethical Enlightenment of Foreign Incentive Mechanism for Orphan Drugs Research and Development to China / 中国医学伦理学

There are a large number of patients with rare diseases in China, and most of them are faced with problems such as no medicine to cure, and have drugs outside of China but not inside. This paper combed the incentive policies of orphan drugs in the United States and the European Union, including orphan drugs legislation and setting up special management institutions, orphan drugs qualification certification, government funding, tax reduction and agreement assistance in the research and development process, providing accelerated listing channels in the examination and approval process, giving market monopoly period in the circulation stage, and giving priority to review. On this basis, it also explored the incentive measures of non-profit organizations in the research and development of orphan drugs, including providing financial support, clinical research and so on. From the perspective of guaranteeing the right to life and health of patients with rare diseases, the fairness and accessibility of medication, and the subjects’ right to know and privacy in clinical trials, this paper provides reference for perfecting the incentive mechanism of orphan drugs research and development in China.