Protecting the socioeconomic rights and interests of patients with rare diseases based on an innovative payment mechanism.

The pathogenesis of diseases in the field of rare diseases is complex. Many rare diseases have yet to be conquered worldwide, and there are still no effective treatments for most rare diseases, resulting in limited accessibility to medications. Over the past few years, China has been committed to ensuring the availability of drugs for rare diseases, both at the national level and in all sectors of society.

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.

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.

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).

Quality and Performance Measurement in Primary Diabetes Care: A Qualitative Study in Urban China.

Background: Quality measurements in primary healthcare (PHC) have become an essential component for improving diabetes outcomes in many high-income countries. However, little is known about their implementation within the Chinese health-system context and how they are perceived by patients, physicians, and policy-makers. We examined stakeholders' perceptions of quality and performance measurements for primary diabetes care in Shanghai, China, and analyzed facilitators and barriers to implementation.

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.

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 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.

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.

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.

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.

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.

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.

"4+7" city drug volume-based purchasing and using pilot program in China and its impact.

In order to deepen the health system reform and improve the mechanism for the formation of drug prices, in January 2019, the General Office of the State Council of the People's Republic of China issued the "National centralized drug purchasing and using pilot program", selected 11 cities in mainland China to carry out "4+7" city drug volume based purchasing pilot work. This paper introduces the specific implementation plan, organizational structure and drug selection process of China's "4+7" city drug volume-based purchasing pilot work, and expounds the initial effects, existing problems and policy development after the implementation of the policy. After the implementation of the policy, the prices of 25 selected drugs were significantly lower, compared with the minimum purchase price of the same drugs in 11 pilot cities in 2017, the average drop was 52%.

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.

Progress on drug pricing negotiations in China.

On November 28, 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.

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.

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.

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.

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.

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.

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 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 (denoted here as ), which cited data on the incidence/prevalence of 21 rare diseases (21 of 121 rare diseases, 17.36%).

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.

System building and improvement for the diagnosis and treatment of rare diseases in Shanghai, China.

Shanghai has always attached importance to the prevention and treatment of rare diseases and has been at the forefront in China. The Shanghai Rare Diseases Diagnosis and Treatment Center, Shanghai Children's Rare Diseases Diagnosis and Treatment Center, and Shanghai Rare Diseases Specialist Clinic were established in February 2018. Moreover, with the development of clinical pathways for rare diseases and the provision of related services such as diagnosis, treatment, screening, information and training, the service system for diagnosis and treatment of rare diseases in Shanghai has formed, which greatly improves the accessibility of medical services for patients with rare diseases in Shanghai and surrounding areas, and is of great significance in reducing the burden on patients with rare diseases.