Release and impact of China's "Second List of Rare Diseases".

On September 18, 2023, the National Health Commission of China officially announced the "Second List of Rare Diseases". This list of 86 rare diseases, drafted in accordance with the "Working Procedures for Drafting the List of Rare Diseases", marks the second release of a rare disease list since the initial list was issued in May 2018. Following the release of the first batch, the Chinese Government introduced various policies to enhance the diagnosis and treatment of rare diseases, to promote the research on, development of, production of, and availability of rare disease medications in China, and to improve medication access for patients with rare diseases. Consequently, this has elevated the level of rare disease diagnosis and treatment, ensuring greater accessibility to treatment for affected individuals. The expansion of the rare disease list through the release of the "Second List of Rare Diseases" will further enhance rare disease management, increase awareness, improve diagnosis and treatment, facilitate the development and availability of more rare disease medications, establish a comprehensive support system for patients with rare diseases, and ultimately benefit a larger number of individuals affected by rare diseases. The definition of rare diseases in China should be refined by explicitly establishing corresponding criteria based on incidence, prevalence, or the number of affected individuals. Additionally, the mechanism for removal of diseases from rare disease lists should be enhanced, and prompt adjustments should be made regarding diseases that do not align with the selection principles of the list, taking into consideration environmental changes.

Nurses' practices and their influencing factors in palliative care.

Background: In 2017, the Chinese government launched a pilot project in palliative care, in which Shanghai was a pioneer. Nurses play a key role in palliative care services as they are the main providers improving the quality of services for patients and their families. However, little is known about practices and influencing factors in the field of palliative care from a nursing perspective in China. This is an original empirical study that has meticulously analyzed the interrelationship and intensity between practices and other factors among nurses in the initial stage of palliative care in primary healthcare institutions in Shanghai, China. Methods: A descriptive-correlational study design was used to sample 2,829 eligible palliative care nurses by purposive sampling survey in 225 healthcare institutions in Shanghai, China. Descriptive analyses were performed using IBM SPSS 24.0 software. Structural equation modeling was applied to analyze the data by AMOS 20.0. Data were collected using the well-designed Knowledge, Attitudes, and Practices of Hospice Care (KAPHC) scale. Results: The final model showed a good model fit. Self-efficacy directly influenced practices (ß = 0.506, P < 0.01) and indirectly influenced practices (ß = 0.028, P < 0.01) through intention. Subjective norm directly influenced practices (ß = 0.082, P < 0.01) and indirectly influenced practices (ß = 0.030, P < 0.01) through intention. Intention (ß = 0.152, P < 0.01) and knowledge (ß = 0.068, P < 0.01) directly influenced practices. Perceived susceptibility (ß = -0.027, P < 0.01), perceived benefits (ß = -0.017, P < 0.01), and perceived barriers (ß = -0.014, P < 0.01) indirectly influenced practices through intention. Conclusion: This study provided evidence of the associations of knowledge, perceived susceptibility, benefits, barriers, subjective norm, self-efficacy, intention, and practices among nurses concerning palliative care and interventions improving their actual work practices. Our findings revealed that self-efficacy, intention, and subjective norms greatly influenced practices. It is imperative to take interventions that focus precisely on self-efficacy, intention, and subjective norms to improve nurses' practices.

The initiation, exploration, and development of hospital-based health technology assessment in China: 2005 ‒ 2022.

A hospital-based health technology assessment (HB-HTA) can provide the evidence needed to inform clinical decisions at the administrative level. With the implementation of a new round of medical and health care system reforms in China, such as the abolition of medical mark-ups, adoption of modern hospital management systems, reform of diagnosis related groups (DRGs) payment, and performance evaluations for public hospitals, medical institutions increasingly need HB-HTA. The development of HB-HTA in China can be divided into three phases: An initiation phase (2005-2014), a preliminary exploratory phase (2015-2017), and a rapid development phase (2018-present). HB-HTA has been used to manage medical consumables, medical devices, and medicines, but there are still problems and challenges in terms of concept recognition, the mode of development, and limited professionals and data. To promote and use HB-HTA in developing countries, we have identifies the development paths and recommendations for implementation based on a case study in China, which can be summarized as follows: enhancing the top-level design of HB-HTA, formulating HB-HTA guidelines, further promoting the main ideas of HB-HTA, concentrating on the training of evaluation personnel, establishing an HB-HTA network and paying attention to the flexibility of HB-HTA in the application process, and multi- stakeholder participation.

The usage of enzyme replacement treatments, economic burden, and quality of life of patients with four lysosomal storage diseases in Shanghai, China.

Lysosomal storage diseases (LSDs) are a group of rare diseases that cause progressive physical dysfunction and organ failure, which significantly affected patients' quality of life. The objective of this study was to explore the characteristics and usage of Enzyme Replacement Treatments (ERTs), which is the only specific therapy for LSDs, of patients with the four different LSDs (Gaucher, Fabry, Pompe disease and Mucopolysaccharidosis) in Shanghai, and then evaluate the economic burden and quality of life of these patients. A total of 31patients, involving 5, 14, 4 and 8 patients with Gaucher, Fabry, Pompe disease and Mucopolysaccharidosis, respectively, were included in analysis. The result showed that only five Gaucher disease (GD) patients in Shanghai used Imiglucerase in 2019, while the other 26 patients with the other three LSDs did not receive ERTs. The total health expenditure of GD patients was 2,273,000CNY on average mainly resulted by the high cost of Imiglucerase. The total health expenditure of the other 26 patients was 37,765CNY on average. Though the cost-sharing mechanism between basic medical insurance, charity fund and patients had been explored for Gaucher disease in Shanghai, the out-of-pocket part, which was 164,301 CNY, still laid a heavy economic burden on the patients and their families. The mean EQ-VAS score of GD patients was 76.4 ± 15.5, which was higher than that of the other three LSDs. It is recommended that the scope of drug reimbursement list and the reimbursement level should be further expanded and raised to help improve the living conditions of patients with LSDs.

Sequential Multiple-Assignment Randomized Trials to Compare Antipsychotic Treatments (SMART-CAT) in first-episode schizophrenia patients: Rationale and trial design.

Accumulated studies have investigated pharmacological interventions for first-episode schizophrenia (FES) patients. However, studies on subsequent treatment steps, which are essential to guide clinicians, are largely missing. This Sequential Multiple-Assignment Randomized Trials comparing Antipsychotic Treatments (SMART-CAT) program intends to evaluate the effectiveness of commonly used antipsychotic drugs in FES patients. The major goals of this study are to examine: 1) what would be the optimal subsequent sequential treatment if the first antipsychotic drug failed; 2) whether clozapine could be used in those first-trial failed and have superior efficacy compared to other atypical antipsychotics. In this article we will report the detail protocol of SMART-CAT. The SMART-CAT is a randomized controlled clinical multicenter trial in which 9 institutions in China will participate. A total of 720 FES patients will be enrolled and followed up for 12 months in this study. The trial includes three treatment phases (each phase lasting for 8 weeks) and a naturalistic follow-up phase; participants who do well on an assigned treatment will remain on that treatment for the duration of the 12-month treatment period, while non-responders will move to the next phase of the study to receive a new treatment. Phase 1 is a randomized controlled trial; patients will be randomly assigned to one of the treatments with oral olanzapine, risperidone, amisulpride, aripiprazole or perphenazine. Subjects who fail to respond after 8 weeks will enter the phase 2 randomization. Phase 2 is an equipoise-stratified randomization trial, and patients will be randomly assigned to oral olanzapine, amisulpride or clozapine for 8 weeks. Subjects who fail to respond after phase 2 will enter an open label trial (phase 3); patients who receive clozapine in phase 2 and fail to respond will be assigned to an extended clozapine treatment or modified electroconvulsive therapy add-on therapy (Phase 3A). Patients who were not assigned to clozapine in phase 2 will be assigned to treatment with clozapine or another SGAs not previously used in phase 1 and 2 (Phase 3B). The primary outcome for the treatment phase is the treatment efficacy rate, which is defined as at least 40% reduction in Positive and Negative Syndrome Scale (PANSS) total score. We hypothesize that clozapine is more therapeutically effective than any other SGAs to patients who failed to meet efficacy criteria in Phase 1, and earlier treatment with clozapine can improve the functional outcomes of schizophrenia patients. As for the naturalistic follow-up phase, time to all-cause treatment failure, marked by its discontinuation is selected as the primary outcome, since it reflects both efficacy and side effects. The all-cause discontinuation is defined as discontinuing for any reasons, including poor efficacy, intolerance of adverse reactions, poor compliance and other reasons. The results of the SMART-CAT trial will provide evidence for the selection of antipsychotics in FES patients who fail to respond to the first trial of an antipsychotic drug. It will also provide evidence for the efficacy and safety of using clozapine in the early phase of schizophrenia treatment by comparing with other SGAs. The study is based on the combination of sequential therapy and dynamic therapy, which can be more suitable to assess the effectiveness of treatment options in the real-world clinical setting. As a result, we hope that this study can provide guidance for an optimal treatment algorithm in first-episode schizophrenia patients. Trial registration: ID NCT03510325 in ClinicalTrials.gov.

The impacts and unintended consequences of the nationwide pricing reform for drugs and medical services in the urban public hospitals in China.

BACKGROUND: Since 2015, China has been rolling out the pricing reform for drugs and medical services (PRDMS) in the urban public hospitals in order to reduce drug expenditures and to relieve financial burdens of patients. This study aims at evaluating the effectiveness of the reform and investigating its positive impacts and unintended consequences to provide evidence basis for further policy making. METHODS: The Difference-in-difference (DID) approach was employed to analyze the reform impacts on the 31 provincial administrative areas in China based on data abstracted from China Statistics Yearbooks and China Health Statistics Yearbooks from 2012 to 2018. RESULTS: The reform resulted in a decrease of 7.59% in drug cost per outpatient visit, a decrease of 5.73% in drug cost per inpatient admission, a decrease of 3.63% in total cost per outpatient visit and an increase of 9.10% in surgery cost per inpatient admission in the intervention group. However, no significant change in examination cost was found. The reduction in the medical cost per inpatient admission was not yet demonstrated, nor was that in the total outpatient/ inpatient expenses. The nationwide pricing reform for drugs and medical services in urban public hospitals (PRDMS-U) in China is demonstrated to be effective in cutting down the drug expenditures. However, the revealed unintended consequences indicate that there are still significant challenges for the reform to reach its ultimate goal of curbing the medical expenditures. CONCLUSION: We conclude that the pricing reform alone may not be enough to change the profit-driven behavior of medical service providers as the root cause lies in the unchanged incentive scheme for providers in the service delivery. This holds lessons for policy making of other low- and middle-income countries (LMICs) with similar health systems set up in the achievement of Universal Health Coverage (UHC).

Impact of DRGs-based inpatient service management on the performance of regional inpatient services in Shanghai, China: an interrupted time series study, 2013-2019.

BACKGROUND: The asymmetry of information brings difficulty for government to manage public hospitals. Therefore, Jiading District of Shanghai has been establishing DRGs-based inpatient service management system (ISMS) to effectively compare the output of different hospitals through DRGs, reward desired hospital performance and enhance inpatient service capacity. However, the impact of the implementation of DRGs-based inpatient service management (ISM) policy in Jiading district is still unknow. We therefore conducted this study to evaluate the impact of DRGs-based ISM policy on the performance of inpatient service since its implementation in Jiading District, Shanghai, China in 2017. METHODS: Using an interrupted time series design, we analyzed quarterly data of seven DRGs-based performance measures from the ISMS which covered all five public hospitals in Jiading District from 2013 to 2019. We utilized the segmented linear regression model to assess the change of level and trend of performance indicators before and after ISM policy. Dickey-Fuller test was used to examine the stationary of the data. Durbin-Watson test was performed to check the series autocorrelation of indicators. RESULTS: Significant changes in the following indicators were observed since the implementation of ISM policy. The case-mix index (CMI) level decreased by 0.103 (P < 0.05), the trend increased by 0.008 (P < 0.05). The total weight level decreased by 3719.05 (P < 0.05), and the trend increased by 250.13 (P < 0.05). The time efficiency index (TEI) level increased by 0.12 (P < 0.05), and the trend decreased by 0.01 (P < 0.05). The cost efficiency index (CEI) level increased by 0.31 (P < 0.05), and the trend decreased by 0.02 (P < 0.05). No significant difference was found in the change of DRGs number, inpatient mortality of low-risk group cases (IMLRG) and inpatient mortality of medium-to-low risk group cases (IMMLRG). CONCLUSIONS: Findings highlight the role of ISM policy in improving the capacity and efficiency of regional inpatient service. Three prerequisites, including a good information system, high-quality EMR data, and a management team, are needed for other countries to implement their own ISM policy to help government manage public hospitals and improve the performance of regional inpatient service.

Prospects for development of group purchasing organizations (GPOs) in China within the context of national centralized drug procurement.

Healthcare group purchasing organizations (GPOs) are considered to play an integral role in the healthcare supply chain by keeping prices low and helping all members of the healthcare system achieve their objectives. China has been exploring GPOs in the field of drug procurement since 2015, and there are currently three GPO models in Shanghai, Shenzhen, and Guangzhou. Although the three models operate differently and they have each been examined, they have all achieved certain results and demonstrated the ability to control drug expenditures. In 2018, the National Healthcare Security Administration implemented a national centralized drug procurement policy, also known as the 4 + 7 procurement policy ("4+7 Policy"). This policy context has also led to changes in the strategy for development of GPOs in China. GPOs need to explore strategies that do not overlap with the scope of 4 + 7 procurement, and they need to develop dynamic and personalized procurement plans that are more in line with first-line clinical practices to have a synergistic effect in combination with the "4+7 Policy." In the future, GPOs will grow rapidly in China. The number of GPOs need to be increased to prevent monopolies, and GPOs need to expand their diversified value-added services to perform more tasks in terms of supply chain management and data analysis.

An international comparison analysis of reserve and supply system for emergency medical supplies between China, the United States, Australia, and Canada.

Coronavirus disease 19 (COVID-19) has become a pandemic around the world. With the explosive growth of confirmed cases, emergency medical supplies are facing global shortage, which restricts the treatment of seriously ill patients and protection of medical staff. Taking China, the United States, Australia, and Canada as examples, this study compares and analyzes the reserve and supply systems of emergency medical supplies and problems exposed in response to the COVID-19 epidemic. Some common problems were found, such as insufficient types and quantities of emergency medical supplies in reserve, insufficient emergency production capacity, and imperfect command mechanism for emergency supplies deployment and transportation. A sound reserve system of emergency medical supplies is the basis and guarantee for dealing with public health emergencies such as major outbreaks. Based on the comparison of systems and practical experience, countries around the world should further improve the reserve and supply system of emergency medical supplies, and improve the coordination and cooperation mechanism for emergency supplies for international public health emergencies, so as to cope with increasingly severe public health emergencies in the context of globalization.

Introduction on collective quarantine of close contacts of patients with COVID-19 for medical observation in China: from the perspective of frontline staff.

The World Health Organization (WHO) has deemed coronavirus disease 2019 (COVID-19) to be a pandemic. The strict prevention and control measures taken by China have proven to be effective, creating a window of opportunity for other countries. The tracking and management of contacts of patients with COVID-19 are important components of prevention and control measures. This article briefly describes the placement of close contacts of patients with COVID-19 under collective quarantine for medical observation in China from the perspective of frontline staff. This article focuses on a community in the Jiading District of Shanghai to provide a reference for placement of close contacts of patients with COVID-19 under collective quarantine for medical observation in other countries and regions.

Challenges to the system of reserve medical supplies for public health emergencies: reflections on the outbreak of the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) epidemic in China.

On December 31, 2019, the Wuhan Municipal Health Commission announced an outbreak of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), China is now at a critical period in the control of the epidemic. The Chinese Government has been taking a series of rapid, comprehensive, and effective prevention and control measures. As the pandemic has developed, a fact has become apparent: there is a serious dearth of emergency medical supplies, and especially an extreme shortage of personal protective equipment such as masks and medical protective clothing. This is one of the major factors affecting the progress of epidemic prevention and control. Although China has made great efforts to strengthen the ability to quickly respond to public health emergencies since the SARS outbreak in 2003 and it has clarified requirements for emergency supplies through legislation, the emergency reserve supplies program has not been effectively implemented, and there are also deficiencies in the types, quantity, and availability of emergency medical supplies. A sound system of emergency reserve supplies is crucial to the management of public health emergencies. Based on international experiences with pandemic control, the world should emphasize improving the system of emergency reserve medical supplies in the process of establishing and improving public health emergency response systems, and it should promote the establishment of international cooperative programs to jointly deal with public health emergencies of international concern in the future.

Progress on drug pricing negotiations in China.

On November 28th, 2019, the National Healthcare Security Administration (NHSA) and the Ministry of Human Resources and Social Security (MOHRSS) of China announced the results of drug pricing negotiations. Seventy first-negotiated drugs with 60.7% average price decrease and twenty-seven re-negotiated medicines with 26.4% average price fall, involving 11 disease categories, were successfully incorporated into National Reimbursement Drug List (NRDL). Medicines that successfully get accessed to NRDL are mostly new listings with high clinical value, and more than half of them are manufactured by Chinese enterprises. Compared to the negotiated drug list of 2017, the biggest increase in western medicines is the digestive system medications (10 drugs added), and the traditional Chinese medicine is internal medicine (17 drugs added). The negotiation follows the process including preparation, examination, negotiation, and announcement. There are several innovations in the procedure, such as the parallel calculation of the floor price, the introduction to competitive negotiations, allowing companies to apply for price confidentiality, and increasing government-enterprise communication before negotiations. Incorporating patent drugs into NRDL by negotiation not only helps patients reduce the economic burden, but also encourages pharmaceutical companies to innovate.

Marketing of drugs for rare diseases is speeding up in China: Looking at the example of drugs for mucopolysaccharidosis.

In May 2019, China National Medical Products Administration approved the marketing of an elosulfase alfa injection (brand name: Vimizim) from BioMarin Pharmaceutical for the treatment of patients with mucopolysaccharidosis (MPS) type IVA. This is the first drug to treat MPS in China, and it has ended the "dearth of medicines" to treat MPS in China, a situation that has persisted for many years. One can reasonably say that the drug has benefited from the continuous reform of the drug review and approval system in China and the increasing attention paid to rare diseases. At present, China has implemented a series of preferential policies for the review and approval of drugs for rare diseases, mainly including priority review and approval, accelerated review and approval, special review and approval (mainly simplified review and approval), data protection, and communication. Moreover, China now has a specific reference for the review and approval of drugs for rare diseases with the creation of China's First List of Rare Diseases and the publication of two batches of the List of Overseas New Drugs Urgently Needed in Clinical Settings. Drug review and approval has been significantly accelerated, as has marketing. The two batches of lists of new drugs, issued in November 2018 and May 2019, include 43 drugs for rare diseases (58.1% of all drugs in the lists), 37 of which were included in China's First List of Rare Diseases. The lists also include three other drugs for MPS. As of July 1, 2019, four drugs for rare diseases from the first batch of new drugs have been approved for marketing. In order to further improve the review and approval of drugs for rare diseases in China, a special department should be established for the evaluation of drugs for rare diseases, research on and management of drugs in the post-approval phase should be enhanced, international cooperation in research on use of drugs to treat rare diseases should be enhanced, and the incentive policy for marketing drugs for rare diseases should be improved.

China has faster pace than Japan in population aging in next 25 years.

The aging of a population structure is an inevitable path of demographic transition, as an inescapable result of decline in fertility rate and extension in life expectancy. Although demographic transition occurred earlier in Japan than in China, the two countries had similar patterns, both of which took a much shorter period of time than Western countries to complete demographic transition, as well as have been aging at a rapid speed that has rarely been seen in the world. Japan has the highest level of population aging in the world, and China has been experiencing a very fast pace of the population aging process and has the largest older population. Drawing upon data from World Population Prospects (2019), this paper compares changes in population aging in both China and Japan. Findings show that Japan's aging process is 30 years ahead of China, but China has been changing in a similar way as Japan. To be specific, both countries experienced four phases of the population aging process: accelerated development period, rapid development period, slow down period and high-level maintained period. In addition, both countries had a quick growing rate of population aging. It will take China 23 years and 10 years respectively for the aging rate increasing from 7% to 14% and then to 20%, while Japan took 24 and 11 years respectively, which is much shorter than developed countries in the West. Furthermore, China has a faster pace than Japan in population aging in the next 25 years. We found that from 2019-2044, China's aging rate, elderly dependency ratio, oldest-old coefficient and median age of population will increase 13.24 percent points, 24.21 percent points, 8.33 percent points, and 8.47 years, while the four indicators of Japan will increase 8.38 percent points, 22.52 percent points, 8.29 percent points, and 6.20 years, respectively.

Overview on social security system of rare diseases in China.

China has initiated the special security project for rare diseases from the national strategic level since 2018. Chinese government has formulated China's First List of Rare Diseases (121 diseases included), established Expert Committee of Diagnosis, Treatment and Security of Rare Diseases and China Alliance of Rare Diseases. The government also encouraged all the provinces to include the security plan and strategies of rare diseases into key work and promoted the establishment of rare diseases clinical research center and the formulation of diagnosis and treatment guidelines. All these actions led to the further improvement of scientific research ability and diagnosis and treatment ability. In terms of the treatment drug of the 121 diseases in China's First List of Rare Diseases, 83 of them are on the market in China and 50 have been included in the national medical insurance accompanied with the tax reduction policy on rare diseases drug and several charity foundations and enterprise donation programs were developed so that drug availability and accessibility for patients are increased. However, there is no clear definition and corresponding coding of rare diseases; the economic burden of most rare diseases could not be calculated accurately because of the difficulty in diagnosis and misclassification. Regarding the service provision system, social security system of rare diseases in China needs further improvement because of the rather few kinds of rare diseases involved in the screening, low level of diagnostic ability, rather few rehabilitation programs and the lack of social caring and knowledge training. It is necessary for China to provide comprehensive and well-rounded health care and social caring for rare diseases patients. Since the formulation and implementation conditions of the policy are different among different regions, the pilot should be carried out first in regions with desired qualifications when China is formulating the rare diseases policy and then a mature national plan should be developed.

How the health institution combinative contracting mechanism influences community residents' patient experiences in Shanghai: A comparative study of data from two cross-sectional surveys.

OBJECTIVE: To understand the effect of the health institution combinative contracting mechanism (which make participating residents make a "combinative contracting" involving family doctor of community health center, one secondary hospital, and one tertiary hospital) on community residents' patient experiences in Shanghai, China. METHODS: We conducted two questionnaire surveys (2016 and 2018) on the patient experiences of 1200 permanent residents of 12 subdistricts of Shanghai, who were selected via stratified random sampling. Of these, 926 participants were included after propensity score matching. We compared five dimensions of patient experience-accessibility, environment and facilities, service attitude and emotional support, communication and patient engagement, and service integration-before and after implementation of the health institution combinative contracting mechanism in June 2016. Furthermore, logistic regression analysis was used to explore the factors related to residents' overall experience. RESULTS: The health institution combinative contracting mechanism influenced most dimensions of residents' patient experience, such as accessibility, service attitude and emotional support, communication and patient participation, and service integration. The mechanism in general helped contracted residents obtain a better patient experience than before its implementation. Referral had a significant effect on participants' overall experience. CONCLUSION: Contracted family doctors play active roles in improving nearly every dimension of residents' service experience, as well as their overall experience of services. The health institution combinative contracting mechanism not only increases interaction and strengthens trust between doctors and patients but also makes it possible for residents to obtain integrated health services.

Accessibility of drugs for rare diseases in China: Policies and current situation.

"Poor accessibility to drugs" is the most problematic issue for patients with rare diseases in China. In recent years, China has issued a number of policies, such as prioritizing speeding up the evaluation for rare disease drugs, publishing national rare disease lists and giving priority to treatments for severe diseases like rare diseases during annual adjustments of National Medical Insurance Medicine Catalogue to improve the accessibility of rare disease drugs. From the outcome perspective, the evaluation of rare disease drugs takes 3 months shorter than ordinary drugs, basic research projects have been started and the number of rare disease drugs included in National Medical Insurance Medicine Catalogue has increased to 50. However, the policies' effects on new drug research and development, rare disease diagnosis and treatment as well as drug pricing are limited. It is recommended to learn the tilt policy of research and development for rare disease drugs from foreign countries and the mechanism of medical insurance funding and patient co-payments. Thus it is important to improve the availability, accessibility and affordability of rare diseases drugs based on the Chinese context.

Incidence and prevalence of 121 rare diseases in China: Current status and challenges.

In order to ascertain the current status of and challenges posed by the incidence and prevalence of rare diseases in China, this study teases out data on the incidence and prevalence of 121 rare diseases listed in China's First List of Rare Disease to provide rationales and references for the development and promotion of rare-disease-related policies. The National Health Commission of the People's Republic of China issued the Rare Disease Diagnosis and Treatment Guide (2019) (denoted here as China's Rare Disease Diagnosis and Treatment Guide), which cited data on the incidence/prevalence of 21 rare diseases (21 of 121 rare diseases, 17.36%). Data on 68 diseases (56.20%) were found in monographs, literature databases, and official websites. Data on the incidence/prevalence of 70 diseases were compiled, though no data were available for the 51 remaining diseases. There are published data on the incidence/prevalence of only 14 diseases at the national level. Sources of data on the incidence and prevalence of rare diseases mainly include cases counts from hospitals (40.56%), other sources of data (24.48%), screening (20.98%), cross-sectional studies (8.39%), and estimates from models (7.69%). Data on the incidence/prevalence of rare diseases in China are limited and typically lack accuracy, uniformity, and timeliness. Epidemiological data at the national level are greatly lacking, and data are not amenable to comparison. China recently initiated epidemiological studies of rare diseases at the national and regional level. The country will continue to promote, use, and update its list of common rare diseases, actively encourage the coding and registration of cases of rare diseases, and take actions to collect, share, and use that information.

The progress of, challenges faced by, and future of rare disease patient organizations in China.

In addition to difficulties with treatment and expenses, patients with rare diseases in China greatly lack social support. In around 2000, Chinese patients with rare diseases and their families began to organize a series of charitable activities such as medical aid, public education, and policy advocacy. After nearly 20 years, organizations for Chinese patients with rare diseases have progressed. Many problems still remain, including a relatively small number of organizations, a low level of specialization, a lack of stability, limited social influence, and limited access to social resources. In order to spur the development of Chinese rare disease patient organizations, public education needs to be enhanced, policy support is needed, teams need to be created, and communication and cooperation need to be enhanced.

A brief introduction to China's new Drug Administration Law and its impact on medications for rare diseases.

The Drug Administration Law of the People's Republic of China (amended in August 2019) is a major piece of legislation governing drug administration in China. This law seeks to improve health legislation; to achieve this, it defines the concepts of fake drugs and inferior drugs and their differences, it emphasizes the regulation of reliability, it standardizes online drug sales, it encourages technological innovation, and it specifies legal liability and punishment through legislative amendments. The Drug Administration Law is characteristic of responsive legislation, and it guarantees a right to health for citizens. The law provides for priority review and approval of new drugs for diseases like rare diseases, it encourages the domestic manufacturing and development of new drugs for rare diseases, and it provides a firm legal basis for medications to treat rare diseases under the Healthy China strategy.