Rapid development of recently emerging precision medicine techniques represented by gene therapy has brought hope for the treatment of rare blinding eye diseases such as inherited retinal diseases (IRDs) for which there was no effective treatment previously. Although the globally growth of clinical trials for IRDs has increased rapidly over the past decade, due to the highly genetic and clinical phenotypic heterogeneity, as well as limited data on epidemiology and natural history of the disease, along with severe loss of vision function of majority patients for which the established measurements may not be appropriate, such studies lack standard outcome measurements and endpoints to asses clinical meaningful effectiveness, posing great challenges in terms of study design and evaluation of treatment efficacy, as well as clinical practice application. At present, there is no systematic nor standardized guidance on safety measures, clinical outcomes and endpoints of visual function for clinical trial design in IRDs. Therefore, in order to standardize the validated evaluation of IRDs clinical efficacy outcome measurements and endpoints, the Fundus Disease Group of Chinese Medical Association Ophthalmology Branch and Fundus Disease Committee of Chinese Medical Doctor Association Ophthalmology Branch organized domestic experts to put forward consensus and recommendations on standardizing outcome measurements and endpoints for clinical study design in IRDs, aiming to advance the study design of IRDs natural history research and clinical trials and to effectively evaluate disease progression and intervention efficacy. Along with the development of medical science and clinical trials, relevant content will be improved and updated accordingly.
Inherited retinal diseases (IRDs) are a group of severe retinal degenerative diseases leading to permanent visual impairment. IRDs are the major cause of irreversible blindness in children and working age groups. Gene therapy is a new clinical treatment method and currently the only clear and effective treatment for IRDs, while, there are still risks in clinical research and application. How to standardize perioperative management and reduce the potential risks of treatment is one of the keys to ensure the safety and effectiveness of treatment. However, there is no systematic and standardized guidance on the perioperative management for IRDs gene therapy. Therefore, in order to standardize the perioperative management, the Fundus Disease Group of Ophthalmology Society of Chinese Medical Association and Chinese Medical Doctor Association organized domestic experts to put forward standardized opinions on the perioperative management of IRDs gene therapy in China after repeated discussion and combined with domestic and foreign research experience, so as to provide clinicians with reference and application in clinical research and practice.
Every country has its own clinical guideline to standardize medical care, and China is developing guidehnes too. Viewing the guidehne＇s usage in the world over many years, we find that it hasn＇t achieved good effect. The development of evidence-based medicine brings a primal change to guideline development. The evidence-based clinical guidehnes are arising in the world. We summarized the principle of developing evidence-based guideline according to the experience of Scotland to provide reference for domestic peers.
Based on new clinical evidence, the National Comprehensive Cancer Network (NCCN) annually updates and releases the "NCCN Guidelines for the Clinical Diagnosis and Treatment of Non-Small Cell Lung Cancer" which has become the reference for clinical diagnosis and treatment approved and complied by clinicians worldwide. On November 25, 2020, the latest 2021 V1 version of "NCCN Clinical Diagnosis and Treatment Guidelines for Non-Small Cell Lung Cancer" (hereinafter referred to as "Guidelines") was released. Compared with the 8th edition of the "Guidelines" in 2020, many updates focused on the progress of targeted and immunotherapy. This article will provide the interpretations of the updated therapy content of this edition of the guidelines.
Breast cancer, the most common malignancy in the world, also causes the most death cases of women among malignancies. Breast cancer risk reduction guidelines (version 2023) was updated by National Comprehensive Cancer Network (NCCN). Based on high-level evidences from evidence-based medicine and the latest research progress, the guidelines provided standardized guidance for breast cancer risk assessment and risk reduction strategies for individuals without a history of invasive breast cancer or ductal carcinoma in situ, which has attracted widespread attention from clinicians worldwide. Breast cancer is also the most common malignancy in Chinese women, and the number of newly diagnosed breast cancer cases each year in China ranks first in the world due to the large population, so the breast cancer prevention has become a major public health challenge in China. Aimed to provide reference for breast cancer prevention in China, this article interpreted the guidelines (the new version) based on the characteristics of breast structure in Asian women and the epidemiological characteristics of breast cancer in China.
The National Comprehensive Cancer Network (NCCN) updates the "NCCN clinical practice guidelines in oncology: Lung cancer screening" annually, and the second edition of 2024 was released in October 2023. The 2024 edition of the guidelines builds on the 2023 edition with some updates on description and assessment of risk factors for lung cancer, evaluation and follow-up of lung nodules found during initial and subsequent screening, and low-dose CT screening protocols and imaging modalities. In this article, we will introduce the above updates and provide reference for lung cancer screening in China by combining the relevant guidelines and consensus in China.
Surrogate endpoints, defined as biomarkers or intermediate outcomes utilized in clinical trials to replace the ultimate targeted outcomes, have witnessed a growing prevalence in both clinical trials and drug-device approvals in recent years. To standardize the application and reporting of surrogate endpoints in clinical trial protocols and associated studies, relevant scholars published the SPIRIT-Surrogate and CONSORT-Surrogate reporting guidelines in the BMJ in July 2024. This article provides an interpretation of these guidelines in conjunction with published case studies, with the aim of offering references for domestic researchers, elevating the overall quality of related clinical trials, and eventually facilitating the enhancement of domestic healthcare level.
Objective To evaluate the methodological and reporting quality of clinical guidelines and consensus for adult AIDS. Methods Databases including PubMed, EMbase, Web of Science, CBM, WanFang Data and CNKI were electronically searched and major guideline websites such as GIN, NICE, NGC and Yimaitong were also searched to collect guidelines and consensus for adult AIDS from inception to December 2021. Two researchers independently screened the literature and extracted data according to the inclusion and exclusion criteria. Four reviewers evaluated the methodological and reporting quality of the included guidelines and consensus by using AGREE Ⅱ and RIGHT, respectively. Results A total of 17 adult AIDS guidelines and consensus were included. The average scores of AGREE Ⅱ in various domains were 59.48% for scope and purpose, 37.17% for stakeholder involvement, 30.76% for rigor of development, 74.75% for clarity of presentation, 35.54% for applicability, and 50.49% for editorial independence. The items with the highest reporting rate among the RIGHT evaluation items were 1a, 1b and 1c (100.00%), followed by 3 and 4 (94.12%), 13a and 13b (88.24%), 7b and 11a (76.47%), and 5 (64.71%), and the remaining items were all reported below 60%. Results of subgroup analysis showed that the clarity of presentation, applicability and editorial independence of the guidelines for adult AIDS expressed in AGREE Ⅱ and the average score of RIGHT were higher than those of the consensuses for adult AIDS; the average scores of guidelines and consensuses based on evidence-based medicine in five domains of AGREE Ⅱ (scope and purpose, stakeholder involvement, rigor of development, clarity of presentation and applicability) and RIGHT were higher than those based on expert opinions or reviews. The foreign guidelines and consensus had higher average scores in the six domains of AGREE Ⅱ and the RIGHT score than the domestic guidelines. Conclusion The methodological quality and reporting quality of the published clinical guidelines and consensuses for adult AIDS is low; in particular, there is a certain gap between the national and international guidelines and consensuses. It is suggested that future guideline developers should refer to international standards, such as AGREE Ⅱ and RIGHT, formulate high-quality guidelines and promote their application to better regulate the diagnosis and treatment of adult AIDS.