Objective:To explore the survival status and economic burden of disease for patients with Cryopyrin Associated Periodic Syndrome(CAPS)in China.Methods:From August 2023 to February 2024,a questionnaire survey was conducted on patients who volunteered to participate.The survey included patients'sociodemographic characteristics,current medical treatment status,disease economic burden and life quality.Results:A total of 35 valid questionnaires were collected.The average age of onset for the patients was 5.67 years,and the average duration from onset to confirmed diagnosis was 7.63 years.The average total medical cost per person in the past 12 months was 82 532.79 yuan,which is significantly higher than the national per capita disposable income of China in 2023.Conclusion:CAPS has an early onset and a long duration until diagnosis,with treatment primarily symptomatic,resulting in a heavy disease burden for patients and their families.

Objective To investigate the diagnosis,treatment and follow-up experience of patients with diffuse large B cell lymphoma,explore their preferences and needs,and improve their treatment experience and effectiveness.Methods From August to November 2023,17 patients and their family members were interviewed in one-hour semi-structured interviews,which were organized and analyzed by Excel.Results The preference,diagnosis stage and diagnosis time were mainly related to the complexity of symptoms and knowledge storage.Patients selected hospitals according to their medical habits and geographical location,and the departments were recommended by the guidance desk or judged by experience.In the treatment stage,the choice of hospital mainly considers the comprehensive strength,recommendation of relatives and geographical location,trust doctors and choose treatment plan according to their own conditions;In the follow-up stage,all patients were followed up according to the doctor's advice.About the need,diagnosis stage,hope to improve the level and speed of pathological diagnosis.During the treatment phase,patients and family members expressed their needs for drug policy assurance,accessibility and convenience of chemotherapy,and easy-to-communicate physician and patient education guidelines;In the follow-up stage,it hopes to have easy-to-use follow-up management tools.Conclusion It is suggested that the government should pay more attention to drug guarantee,issue patient education guidelines and pay more attention to popular science education of major diseases.It is recommended that hospitals improve the level and speed of pathological diagnosis,increase the opening rate of daytime chemotherapy area,use smart bed reservation system and actively popularize the latest research results of diseases.

OBJECTIVE To analyze the current status and challenges in importing rare disease drugs in China， providing references for optimizing the import process and improving relevant policies. METHODS Questionnaires and interviews were conducted with stakeholders involved in rare disease drug importation， including government departments， multinational pharmaceutical enterprises， healthcare institutions， and patient organizations. This explored the current situation and challenges encountered by each party. Expert opinions were synthesized to propose improvement suggestions. RESULTS A questionnaire survey of representatives from 25 multinational pharmaceutical companies in the rare disease field revealed that these companies had a strong willingness to import rare disease drugs， with 58.33% of them practicing diverse import models. However， significant challenges hindered this process， including unclear regulations （54.17%）， complex approval procedures （45.83%）， and excessively long approval cycles （41.67%）， negatively impacting their motivation. Meanwhile， interviews with 13 experts from government departments， healthcare institutions， pharmaceutical enterprises， and patient organizations identified deficiencies in policy design， approval processes， sampling inspection costs， and communication efficiency with regulators. Additionally， the drug import model in special medical zones also required improvement. CONCLUSIONS The importation of rare disease drugs in China faces challenges such as incomplete policies， inflexible regulatory mechanisms， and insufficient communication channels. It is recommended to enhance the rare disease definition criteria， optimize import incentive policies， and refine regulatory models， so as to further optimize the import process of rare disease drugs and improve relevant policies.

Objective To investigate the diagnosis,treatment and follow-up experience of patients with diffuse large B cell lymphoma,explore their preferences and needs,and improve their treatment experience and effectiveness.Methods From August to November 2023,17 patients and their family members were interviewed in one-hour semi-structured interviews,which were organized and analyzed by Excel.Results The preference,diagnosis stage and diagnosis time were mainly related to the complexity of symptoms and knowledge storage.Patients selected hospitals according to their medical habits and geographical location,and the departments were recommended by the guidance desk or judged by experience.In the treatment stage,the choice of hospital mainly considers the comprehensive strength,recommendation of relatives and geographical location,trust doctors and choose treatment plan according to their own conditions;In the follow-up stage,all patients were followed up according to the doctor's advice.About the need,diagnosis stage,hope to improve the level and speed of pathological diagnosis.During the treatment phase,patients and family members expressed their needs for drug policy assurance,accessibility and convenience of chemotherapy,and easy-to-communicate physician and patient education guidelines;In the follow-up stage,it hopes to have easy-to-use follow-up management tools.Conclusion It is suggested that the government should pay more attention to drug guarantee,issue patient education guidelines and pay more attention to popular science education of major diseases.It is recommended that hospitals improve the level and speed of pathological diagnosis,increase the opening rate of daytime chemotherapy area,use smart bed reservation system and actively popularize the latest research results of diseases.

Objective:To explore the survival status and economic burden of disease for patients with Cryopyrin Associated Periodic Syndrome(CAPS)in China.Methods:From August 2023 to February 2024,a questionnaire survey was conducted on patients who volunteered to participate.The survey included patients'sociodemographic characteristics,current medical treatment status,disease economic burden and life quality.Results:A total of 35 valid questionnaires were collected.The average age of onset for the patients was 5.67 years,and the average duration from onset to confirmed diagnosis was 7.63 years.The average total medical cost per person in the past 12 months was 82 532.79 yuan,which is significantly higher than the national per capita disposable income of China in 2023.Conclusion:CAPS has an early onset and a long duration until diagnosis,with treatment primarily symptomatic,resulting in a heavy disease burden for patients and their families.

OBJECTIVE To provide suggestions for improving the path and system construction of patient engagement in drug regulation in China.METHODS By reviewing initiatives and experiences from the United States(U.S.),European Union(EU),and Japan in promoting patient engagement,this study summarizes the roles and contributions of patients in the entire drug regulatory process internationally.Combining China's current progress and challenges in patient engagement,specific proposals are formulated to refine regulatory pathways and institutional systems.RESULTS&CONCLUSIONS With growing global emphasis on patient engagement as a regulatory strategy,countries or regions such as the U.S.,EU,and Japan have established clear policies,designated oversight agencies,and developed diversified pathways for patient engagement.Patients contribute to regulatory processes through advisory meetings,direct decision-making roles,and leveraging lived experiences and expertise to optimize drug evaluation and monitoring.In contrast,China's patient engagement remains primarily limited to clinical value-oriented drug development,lacking formal policy guidance.It is recommended that China,based on its existing policy system,further strengthen the construction of a safeguard system for patient engagement,improve the capacity building and pathway models for patient participation in pharmaceutical regulation,and promote the continuous development of patient engagement in pharmaceutical regulation in our country.

OBJECTIVE：To provide reference for accelerating the process of prescription outflow in China by using “Internet+ medicare”. METHODS ：Based on stakeholder perspective ，the stakeholder model of prescription outflow was constructed under the background of “Internet + ”. By analyzing stakeholders （hospital，patient，government，drugstore and third-part consultation platform）who impeded the prescription outflow and their demands ，the obstacles and causes of promoting prescription outflow under the background of “Internet+”were summarized to put forward relevant countermeasures and suggestions. RESULTS & CONCLUSIONS：“Internet+Medicare”accelerated the process of prescriptions outflow by improving the convenience in each links of medical treatment ，but there were still obstacles in prescription source ，patient flow ，supporting policies and system construction to continue promoting prescription outflow. The reason lied in the pressure of hospital operation and the diagnosis demand of doctors；the treatment demand of patients could not be satisfied by the receiving party of prescription outflow ；the difficulty of government supervision and system construction ；immature supervision mode of the third-party consultation platform and online pharmacies，and the platform technology research and development to be break through. To continue promoting prescription outflow under the background of “Internet + ”needs to strengthen top-level design ，establish supporting systems ，break the regional restrictions，build a unified and standardized prescription circulation information platform ；formulate the prescription outflow assessment standard and encouragement mechanism of hospital ，enhance the ability of the third-part platforms and pharmacies to undertake the prescription ，strengthen the publicity and education of patients ，enhance their channel awareness and risk awareness. The exploration mode of prescription outflow in which all stakeholders develop together and cooperate with each other is formed finally so as to promote the outflow of prescription.

OBJECTIVE：To put forward some suggestions for the implementation of OTC monograph in China. METHODS ： Literature research was used to comprehensively review the concept ，emergence and development ，function of OTC monograph in USA；referring to experience in USA ，the necessity and feasibility of monograph system for OTC registration and evaluation in China were explored ，and some suggestions were put forward to the implementation of OTC monograph in China. RESULTS & CONCLUSIONS：OTC monograph in USA refers to the regulatory standards that should be followed when selling OTC products not included in new drug applications. The monograph originates from the re-evaluation project of the effectiveness of OTC drugs ， which plays a special role in accelerating the drug marketing and promoting product innovation. The OTC monograph system is necessary to help to optimize the OTC registration and evaluation ，but at present ，the conditions to establish and implement OTC monograph are not yet ripe in China. Based on the optimization of resource allocation and infrastructure construction ，national OTC drug management experience ，OTC monograph can play a full part in OTC registration and evaluation ，in terms of carrying out in-depth research ，taking pilot test ，improving risk management system and enhancing drug review resource allocation ability.

OBJECTIVE:To recommend the development strategies for small and medium-size pharmaceutical commercial enterprises. METHODS: We analyzed the development and change of the macro-policies and industrial surroundings and discussed different strategies available for small and medium-size pharmaceutical commercial enterprises. RESULTS & CONCLUSIONS: Among those suitable strategies, which include scale enlargement, service differentiation, and countryside & community market expansion etc, small and medium enterprises should choose different kinds of strategies according to their special characteristics. Meanwhile they should incessantly enhance their real strength and spare no effort to cultivate their own competitive advantage to seek survival and development.

OBJECTIVE:To probe into the evaluation method on technological capability of pharmaceutical enterprises.METHODS:The connotation of pharmaceutical enterprises'technological capability was expounded;the characteristics of each evaluation index,i.e.output index and input index of technological capability of pharmaceutical enterprises were ana?lyzed.RESULTS:Detailed and complete information on the evaluation of pharmaceutical enterprises'technological innovation capability was unavailable from the output index and input index only.CONCLUSION:The evaluation on pharmaceutical enterprises'technological innovation capability should include more comprehensive,more dynamic and more integral evaluation indexes.