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Chronic obstructive pulmonary disease （COPD） is a common disease in clinical practice. It is associated with obvious exposure to toxic particles or gases and has become the leading cause of death and disability worldwide. The pathogenesis of COPD is complex， and the oxidative stress involved in COPD plays a crucial role in the pathological process of the disease. Patients with COPD usually have high levels of oxidative stress in the lungs， which will affect the whole body for a long time， causing a variety of complications and accelerating the development of the disease. On the one hand， oxidative stress can directly damage the airway and lung tissue. On the other hand， it also drives other pathological mechanisms to jointly promote the development of disease， such as participating in inflammatory reactions and protease/anti-protease imbalance， promoting mucus secretion， accelerating cellular senescence， causing autoimmunity， and involving in genetic regulatory pathways. At present， western medicine treatment is mostly based on conventional drug treatment， and antioxidant-targeted oxidative stress is adopted， but there are still some challenges in efficacy and safety. Traditional Chinese medicine has a long history of preventing and treating COPD. In particular， Chinese herbal medicine formulas have great potential to interfere with the oxidative stress of COPD. Whether it is the modified classical traditional Chinese medicine or the new formulation developed by modern doctors， the research results reflect the multi-target and multi-channel advantages of traditional Chinese medicine treatment， and their efficacy and safety are gradually verified. This paper reviewed the literature in recent years， starting with the basic and clinical research on the intervention of traditional Chinese herbal medicine formulas on oxidative stress of COPD， so as to provide further ideas for related research on the prevention and treatment of oxidative stress of COPD by traditional Chinese medicine.
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In recent years, the vigorous development of clinical researches carried out by medical schools is inseparable from the effective participation of medical students. However, as the number and categories of clinical research projects that medical students participate in increase, medical ethics related issues gradually occur. This article sorts out the ethical issues that arose in clinical researches, in which medical students participated, analyzes the underlying causes, and proposes solutions for the above-mentioned ethical issues, aiming to provide reference for medical ethics education and research project management for medical schools.
La participación efectiva de los estudiantes de medicina ha contribuido en gran medida al desarrollo exitoso de la investigación clínica en las escuelas de medicina de China en los últimos años. Sin embargo, con el creciente número y tipos de proyectos de investigación clínica en los que participan estudiantes de medicina, las cuestiones éticas se exponen gradualmente. Este trabajo enumera las cuestiones éticas que han surgido en la participación de los estudiantes de medicina en la investigación clínica en los últimos años, analiza las causas subyacentes y propone soluciones a las cuestiones éticas mencionadas anteriormente, con el objetivo de proporcionar referencia para la enseñanza de la ética médica y la gestión de proyectos de investigación para las escuelas de medicina.
A participação efetiva dos estudantes de medicina tem contribuído muito para o próspero desenvolvimento da pesquisa clínica nas escolas médicas da China nos últimos anos. No entanto, com o aumento do número e tipos de projetos de pesquisa clínica dos quais os estudantes de medicina participam, questões relacionadas à ética e à alfabetização são gradualmente expostas. Este artigo lista as questões éticas que surgiram na participação de estudantes de medicina em pesquisas clínicas nos últimos anos, analisa as causas subjacentes e propõe soluções para as questões éticas acima mencionadas, com o objetivo de fornecer referência para o ensino de ética médica e gestão de projetos de pesquisa para escolas médicas.
Subject(s)
Humans , Male , FemaleABSTRACT
Patients with rare diseases frequently face unmet medical needs due to the high costs, lengthy development times, and slow approval processes for new treatments. This case study discusses innovative access alternatives for rare diseases in Brazil, focusing on early access to pabinafusp-alfa for mucopolysaccharidosis type II (MPS-II), a rare genetic lysosomal storage disease characterized by a deficiency of the enzyme iduronate-2-sulfatase. From September 2018 to March 2023, 20 Brazilian MPS-II patients received pabinafusp-alfa through a clinical research protocol. This enzyme replacement therapy (ERT) crosses the blood-brain barrier to address central nervous system manifestations unmet by existing treatments. Patients' participation in the clinical study resulted in an estimated BRL 65 million in cost savings for the public healthcare system compared to conventional ERT with idursulfase-alfa and potentially better clinical outcomes. The case study underscores the importance of innovative mechanisms in addressing patients' medical needs. Early access alternatives include: a) clinical study access, with execution/development aligned with healthcare managers and linked to future access strategies; b) regulatory-level risk-sharing, considering effectiveness uncertainties and the possibility of market withdrawal and/or reimbursement in case of negative results; and c) drug pre-delivery, with payment contingent on positive phase III clinical study outcomes. Although public-private partnerships in clinical research are underused, they could benefit all stakeholders by accelerating drug development, facilitating early patient access to innovative medicines, and generating healthcare system savings, particularly for rare diseases.
Pacientes com doenças raras frequentemente enfrentam necessidades médicas não atendidas devido aos altos custos, longos tempos de desenvolvimento e processos de aprovação lentos para novos tratamentos. Este estudo de caso discute alternativas inovadoras de acesso para doenças raras no Brasil, com foco no acesso precoce ao alfapabinafuspe para mucopolissacaridose tipo II (MPS-II), uma doença lisossômica de armazenamento genético rara, caracterizada por uma deficiência da enzima iduronato-2-sulfatase. De setembro de 2018 a março de 2023, 20 pacientes brasileiros com MPS-II receberam alfapabinafuspe por meio de pesquisa clínica. Essa terapia de reposição enzimática (TRE) atravessa a barreira hematoencefálica para tratar manifestações do sistema nervoso central não atendidas pelos tratamentos existentes. A participação dos pacientes no estudo clínico resultou em uma economia estimada de 65 milhões de reais para o sistema público de saúde, em comparação com a TRE convencional com idursulfase alfa, além de potencialmente melhores resultados clínicos. O estudo de caso destaca a importância de mecanismos inovadores no atendimento das necessidades médicas dos pacientes. As alternativas de acesso precoce incluem: a) acesso por meio de estudos clínicos, com execução/desenvolvimento alinhada aos gestores de saúde e vinculada a estratégias futuras de acesso; b) compartilhamento de risco em nível regulatório, considerando as incertezas de eficácia e a possibilidade de retirada do mercado e reembolso em caso de resultados negativos; e c) pré-entrega do medicamento, com pagamento condicionado aos resultados positivos do estudo clínico de fase III. Embora as parcerias público-privadas em pesquisa clínica sejam subutilizadas, elas poderiam beneficiar todas as partes interessadas ao acelerar o desenvolvimento de medicamentos, facilitar o acesso precoce dos pacientes a medicamentos inovadores e gerar economias para o sistema de saúde, especialmente para doenças raras.
Subject(s)
Mucopolysaccharidosis II , Rare Diseases , Access to Essential Medicines and Health TechnologiesABSTRACT
OBJECTIVES@#Clinical research plays a vital role in disease research and population health. The public is the main source of clinical research volunteers. Understanding the public's cognition of clinical research plays a decisive role in the development of clinical research. This study aims to understand the Chinese public's cognition for clinical research and the influencing factors.@*METHODS@#The questionnaire based on Chinese-translated Public Awareness of Research for Therapeutic Advancements through Knowledge and Empowerment (PARTAKE) was used to investigate the public's cognition for clinical research.@*RESULTS@#Of the 2 513 valid respondents, 91.84% had heard of "clinical research", 91.76% of the respondents believed that clinical research was beneficial to society, 65.90% were willing to participate in clinical research, 87.50% believed that confidentiality was a very important thing, 73.70% believed that their personal information had been protected when participating in clinical research, and, 46.40% did not know whether volunteers participating in clinical research could receive adequate compensation. Educational levels, employment status, and annual income impacted in public perceptions of willingness to participate in clinical research, especially in privacy protection, informed consent, whether clinical research is intended for society, compensation for clinical research, and safety of clinical research (all P<0.01).@*CONCLUSIONS@#The Chinese public's cognition level for clinical research is acceptable, but there is still a lot of room for improvement in privacy protection, informed consent, and compensation. By designing a reasonable knowledge training program for clinical research and using the multimedia, improving access to the relevant knowledge, more public will know about clinical research recruitment information, which is of great significance for the development of clinical research in China.
Subject(s)
Humans , China , East Asian People , Educational Status , Surveys and Questionnaires , Public Opinion , Knowledge , Biomedical ResearchABSTRACT
ObjectiveThrough a randomized， double-blind， double-simulation， positive-control， multicenter design， this study aimed to analyze the relationship between the dosage， efficacy， and safety of Pudilan anti-inflammatory oral liquid in treating acute pharyngitis/tonsillitis in adults caused by bacterial infection and validate the regulatory effect of Pudilan anti-inflammatory oral liquid on inflammatory markers such as serum amyloid A （SAA）， C-reactive protein （CRP）， white blood cells （WBC）， neutrophil percentage （NE%）， and erythrocyte sedimentation rate （ESR）， thereby exploring the feasibility of using Pudilan anti-inflammatory oral liquid as a substitute for antibiotics in the treatment of infectious diseases and providing a basis for rational clinical medication. MethodUsing a stratified randomized， double-blind， double-simulation， positive-control， multicenter design， 220 participants were enrolled from nine centers. The participants were randomly divided into three groups at 1∶1∶1 — a Pudilan anti-inflammatory oral liquid 20 mL group （73 cases）， a Pudilan anti-inflammatory oral liquid 10 mL group （73 cases）， and a control group （amoxicillin group， 74 cases）. The treatment course was 7 days. The study observed parameters including the total effective rate of sore throat， onset and disappearance time of sore throat， health status score， treatment time， and inflammation markers. Result①Dataset division： The 211 cases were included in the full analysis dataset （FAS）， 208 cases were included in the per-protocol dataset （PPS）， and 218 cases were included in the safety dataset （SS）. ② Efficacy evaluation： There were statistically significant differences （P<0.05） in the comparison of the three groups regarding the total effective rate of sore throat， disappearance time of sore throat， and health status. Both the 20 mL and 10 mL groups were non-inferior to the control group， and there was a statistically significant difference between the 20 mL and 10 mL dosage groups （P<0.05）. There was no statistically significant difference in the comparison of onset time of sore throat among the groups. CRP， WBC， and NE% of patients in all three groups significantly decreased on the 7th day of treatment compared with those before treatment （P<0.01）. ③Safety evaluation： Adverse events mainly occurred in various examination indicators. There were no statistically significant differences in the comparison between groups， and no adverse reactions or serious adverse events occurred. ④Economic evaluation： The increased cost of the 10 mL and 20 mL dosage groups was entirely justified as compared with that in the control group. When comparing the 10 mL and 20 mL dosage groups， the 10 mL dosage group was deemed less advantageous. ConclusionPudilan anti-inflammatory oral liquid can be used alone as an alternative to antibiotics in the treatment of acute pharyngitis/tonsillitis caused by bacterial infection. It demonstrates good safety and can lower inflammation markers such as CRP， WBC， and NE%， suggesting its potential to reduce the body's inflammatory response. Its mechanism of action may be related to its multi-target regulatory mechanism.
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Spinal cord injury leads to extremely high mortality and disability rates and its treatment has always been a global challenge. The survival of patients with spinal cord injury is partially lengthened with the development of medical treatments, but the clinical outcome is still not satisfactory. Some important progress has been made in the basic researches over spinal cord injury, such as analysis of repair mechanism of spinal cord injury and development of cellular therapies and biological scaffolds of spinal cord injury in China. However, some basic researches show insufficient understanding of the microenvironment and animal model of spinal cord injury and lack support from clinical problems, leading to too simplistic or contradictory conclusions. There is an urgent need to reexamine the research methods and carry out basic and clinical translational researches. Therefore, the authors discuss the key problems and difficulties in basic researches over spinal cord injury and propose improvement suggestions, aiming to provide a reference for conducting basic researches correctly and accelerating clinical innovational transformation.
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Under the background of "Medical Education Synergy", the clinical practice ability of postgraduates has been significantly improved, and the post competency has been enhanced. However, the "cultivation goal orientation" focuses on clinical practice, the education management department has weakened the cultivation of scientific research literacy, and the postgraduate tutors have not paid enough attention. As a result, the cultivation of scientific research literacy of professional degree postgraduates is seriously affected, and their scientific research ability is obviously weak. Taking gastroenterology as an example, by optimizing the course setting and rotation plan arrangement, attaching importance to the management of the graduate management department and the tutor responsibility system, and strengthening the application of interdisciplinary in the innovation and development of disciplines, we have explored an educational plan for cultivating professional degree postgraduates. The clinical practice and clinical research capabilities of postgraduates majoring in gastroenterology have been synergistically developed with remarkable results.
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Due to the physiological peculiarities of pregnant women, general clinical studies exclude pregnant women. Therefore, there is a lack of evidence of precise medication for pregnant women with diseases worldwide, which poses a significant risk for them to use medication during pregnancy. Whether to include pregnant women as subjects in clinical research has always been a focus of ethical discussion. By providing a broad overview of pregnant women’s participation in clinical research from an ethical perspective, this paper explored the risks and challenges faced by pregnant women’s participation in clinical research, and provided theoretical basis and thinking paths for how to fairly and effectively include pregnant women as subjects and promoting clinical research on pregnant women.
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With the rapid development of healthcare big data and artificial intelligence technology, how to utilize the massive medical data generated based on clinical diagnosis and treatment has become an important issue to be solved in the field of clinical research. Clinical diagnosis and treatment data is an essential part of healthcare big data, and also the main field of healthcare big data research. With the continuous deepening and extensive development of informatization, hospitals have accumulated a large number of patient-centered clinical diagnosis and treatment data. Deeply mining and analyzing these data through big data technology can provide reference for precise diagnosis and treatment, and standardized prevention and control of diseases. However, conducting relevant research still faces many difficulties and blockages, such as the increased risk of data leakage or abuse, and the difficulty in implementing informed consent. To safely, legally and efficiently utilize clinical diagnosis and treatment data to conduct clinical research and fully tap into the value of these precious medical resources, a tertiary hospital in Beijing has built a research big data platform and developed relevant systems to effectively solve the problems of blockages and difficulties in the application of rich clinical resources to clinical research, and improve the service quality of medical institutions and the conversion rate of scientific research achievements. By introducing the key points and management methods in the implementation of clinical research based on the scientific research big data platform, analyzing and exploring the existing problems and improvement measures, this paper aimed to provide theoretical basis and system reference for high-quality and efficient health and medical big data clinical research, inspire and promote the continuous improvement of medical research management, and promote the development of medical and health science and technology innovation.
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Under the background of scientific and technological innovation in the new era, ethical review of traditional Chinese medicine (TCM) is facing new opportunities and challenges. Focusing on how to maintain integrity and innovation in the ethical review work of TCM clinical research in TCM medical institutions, starting from the characteristics of innovation and inheritance of TCM clinical research, this paper discussed the construction of the ethical review system of TCM clinical research, key elements of attention, shortcomings and deficiencies, training and communication, etc. It is also proposed that the joint construction of a Chinese characteristic scientific and technological ethics governance system should consider the unique requirements of modern technological means for the ethical review of scientific and technological projects such as dialectical treatment, re-evaluation of prescription projects, and expansion of indications in clinical research of TCM; innovate the form of review and strengthen the process management of ethical review; scientifically, objectively and impartially evaluate the scientific nature and social value of clinical research projects in TCM; pay attention to the toxic side effects of TCM, especially compound preparations. So as to further improve the procedural norms and quality of ethical review of clinical research of TCM, protect the legitimate rights and interests and safety of subjects, and promote the development of medicine in China.
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According to the Ethical Review Measures for Life Sciences and Medical Research Involving Humans jointly issued by the National Health Commission, the Ministry of Education, the Ministry of Science and Technology and the State Administration of Traditional Chinese Medicine in 2023, to optimize the ethical review process and reduce the burden on clinical researchers, it is proposed that some eligible situations can be "exempted from ethical review". This is a breakthrough progress in China’s ethical review management measures that firstly aimed at "exemption from ethical review". This paper reviewed and sorted out the relevant situations about exemption from review at home and abroad, focused on analyzing and exploring the four situations of exemption from review, especially discussed and analyzed the understanding of anonymization and personal sensitive information in exemption from review, and proposed practical suggestions for the four situations. Based on the actual situation of ethical review work, this paper also explored the establishment of practical standards and processes for exemption from review, providing reference for other medical institutions to implement the exemption from ethical review process.
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At present, China is still in the exploratory stage in the field of electronic informed consent (eIC), and relevant policies, regulations and application guidelines have not yet been established and improved. While the traditional informed consent supervision system is difficult to meet the needs of the innovative development of eIC, such as subject privacy and data security. Through sorting out and analyzing the legal norms and construction system of eIC supervision in Europe and the United States, combined with the current development status, problems, and challenges of eIC in China, this paper targeted proposed the path to construct the supervision of eIC in clinical research in China from the aspects of restricting the signing form and process of eIC, adjusting the ethical review paradigm of eIC, enhancing the strength of eIC ethical review, improving the construction of eIC legal system, and strengthening the training of relevant researchers.
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Currently, the number of clinical research projects continues to grow. Both sponsors and researchers hope to accelerate medical ethical review efficiency, and the regulatory agencies strengthen the control over the ethical review quality. The ethics committee (EC) offices of medical institutions are relatively insufficient in terms of human resource allocation and archiving space. Combined with the development goals of the EC and the requirements of the homogeneity construction of ethical review, it was urgent to optimize the ethics review process and accelerate the efficiency of ethics review through informatization construction. Through informatization construction, the process management of ethical review could be strengthened, the work steps could be simplified, the ethical review level could be improved, and the supervision ability and efficacy of EC on clinical research could be strengthened, which may provide continuous quality improvement strategies and specific optimization measures for the operation and management of the EC, so as to effectively protect the safety, the rights and interests of subjects.
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Strengthening the capacity building of clinical researchers is an important way to achieve high-quality clinical research, ethics training is an important part of it. However, laws and regulations do not specify the requirements for ethical training, many researchers’ cognition of ethics often remain in the form of "passing ethical review", and the content of ethical training often lacks ethical principles, ethical considerations and choices, etc. In view of this, this paper proposed to build a clinical research ethics training system based on the researchers’ ethical decision ability, design and implement ethical concepts, ethical principles grasp, and ethical decision practices from each link of the whole process ethical review, organically integrate them into the training system of clinical research institutions, and promote the conscious infiltration of bioethical awareness that focused on the subjects’ health rights and interests to internalize into the behavior of researchers, accumulate to the ethical decision ability of researchers.
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The electronic informed consent (eIC) system is a product of modernization development of electronic and intelligent technology. In the context of COVID-19, the eIC system can adapt to the epidemic prevention and control requirements, showing its time-space advantages. By introducing the concept, form and the use of eIC system, this paper analyzed the challenges of acceptance, understanding, consent and information security faced by the eIC system. Based on this, some suggestions were put forward, including strengthening the training of the relevant personnel involved in the eIC system, enhancing and improving the functions of the eIC system, and perfecting the relevant laws and regulations of the eIC system, so as to provide reference for the future research and application of eIC.
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Syndrome differentiation and treatment is a unique mode of diagnosis in traditional Chinese medicine （TCM）. The establishment of scientific and standardized syndrome diagnosis standards is an important link to evaluate the clinical efficacy of TCM objectively and systematically， and also a prerequisite for the promotion and development of TCM to obtain international recognition. This article reviewed the basic modes and existed problems of the current syndrome diagnosis criteria， and proposed to construct a multidimensional core information set integrating the minimized core symptoms， the artificial intelligence signs， the multi-modal laboratory indicators， and multi-omics specific markers， so as to present syndrome characteristics from multiple perspectives systematically. This paper also described the basic mode， constructure， as well as the process and methodology to be adopted in the establishment of the standardized diagnostic research method. The core information set of diagnostic symptoms not only took into account the specificity of the disease， but also improved the inconsistency due to the complexity and subjectivity of the syndrome differentiation， thereby providing a methodological basis for the standardization of TCM syndrome differentiation in clinical research.
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Statins are a class of hydroxymethylglutaryl-CoA reductase inhibitors (HMG-CoA reductase inhibitors), which are widely used to reduce blood lipid in clinic, and are especially important for the prevention and treatment of cardiovascular diseases. In recent years, many studies at home and abroad believe that statins have a unique role in tumor prevention and treatment, and have been widely concerned. In terms of epigenetic regulation mechanism, statins mainly affect the progress of tumor through DNA methylation, histone modification and miRNA regulation. In addition, statins can also achieve their anti-tumor effects by promoting tumor cell autophagy, regulating tumor cell cycle, and promoting tumor cell apoptosis and other signaling pathways. Therefore, the research on statins provides ideas for the discovery of new anti-tumor treatments. In this paper, the role of statins in the prevention and treatment of common tumors is reviewed, including its mechanism of inhibiting the occurrence and development of tumors through epigenetic regulation and other related mechanisms, as well as its clinical research status.
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With the continuous progress of research methodology in the real world and the growing maturity of artificial intelligence technology， a method for conducting “quantitative” research to guide clinical practice based on traditional Chinese medicine （TCM） diagnosis and treatment data was gradually developed. However， there is still a need for further improvements in the overall design of studies and the transformation of findings into clinical practice. Based on this， we put forward a comprehensive overall design concept and application approach for real-world study and artificial intelligence research based on clinical diagnosis and treatment data of TCM. This approach consists of five steps： Constructing a research-based database with a large sample size and high data quality； Mining and classification of core prescriptions； Conducting cohort studies to evaluate the effectiveness of core prescriptions； Utilizing case-control studies to clarify the dominant population； Establishing predictive models to achieve precision medicine. Additionally， it is imperative for researchers to establish a standardized system for collecting TCM variables and processing data， optimize the determination and measurement methods of confounding factors， further improve and promote methodologies， and strengthen the training of interdisciplinary talents. By following this research method， we anticipate that the clinical translation of research findings will be facilitated， leading to advancements in TCM precision medicine. Real-world study and artificial intelligence research share similar research foundations， and clinical applications complement each other. In the future， the two will merge together.
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@#At the end of 2022, the National Medical Products Administration (NMPA), in conjunction with the National Cancer Center, Cancer Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College raised for the first time the important issue of clinical research globally: whether the source of the death time of clinical trials based on the simple follow-up records is credible, and proposed a consensus document on the source of the death time of clinical trials. The results were published in The Lancet Regional Health-Western Pacific, which attracted wide attention and recognition from the international industry. This is the first time that the China consensus on quality standards for clinical research has been ahead of the U.S. Food and Drug Administration and other international colleagues. The NMPA has been leading China in promoting the scientific development of clinical research, so as to constantly establish and improve the scientific regulatory system and ecological system, and promote China's full integration into the global pharmaceutical research and development system. China clinical research institutions and the whole industry are also gradually from standardized development to scientific development, high-quality development process. In this study, we summarized the scientific and subject-oriented development of China clinical research industry in recent years, and continuously strengthened the international competitiveness of China pharmaceutical industry. It is suggested that scientific thinking model should be used to deal with the normative problems in clinical research and promote the development of medical model to scientific model.
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@#Telephone follow-up is one of the important ways to follow up patients. High-quality follow-up can benefit both doctors and patients. However, clinical research-related follow-up is often faced with problems such as time-consuming, laborious and poor patient compliance. The authors belong to a team that has been committed to the study of patient-reported outcomes for a long time. The team has carried out long-term follow-up of symptoms, daily function and postoperative complications of more than 1 000 patients after lung cancer surgery, and accumulated certain experience. In this paper, the experience of telephone follow-up was summarized and discussed with relevant literatures from the aspects of clarifying the purpose of clinical research follow-up, understanding the needs of patients in follow-up, and using follow-up skills.