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.
Objective To systematically review the requirements of patient participation in clinical practice guidelines (CPGs) in Chinese and foreign guideline development manuals. Methods Thirty-six authoritative society websites and guideline databases and 5 commonly used databases were searched online. Relevant information on patients’ participation in the guideline manuals was collected, summarized, and analyzed. Results A total of 37 manuals (33 foreign and 4 Chinese) were included. The requirements for the number of patients, the right to speak, status equality, and the right to vote in the guideline development manual accounted for 35.1%, 13.5%, 8.1%, and 5.4%, respectively. The requirements for participants’ mode of participation were not mentioned in the guideline development manuals from 2000 to 2010. There were 6 (16.2%) in 2011–2015 and 12 (32.4%) in 2016–2022. The comprehensive guidelines for multiple disease types accounted for 35.7%, 28.6%, and 57.1%, respectively, in terms of requirements for participants’ knowledge or experience, management of specialized personnel, and training support. The specific guidelines for a certain type of disease or drug accounted for 21.7%, 4.3%, and 17.4%, respectively; fifteen (40.5%) guideline development manuals mentioned the specific collection forms of patients’ values and preferences in guideline development. Conclusion Given changes to medical models and the emphasis on patients’ rights and interests, an increasing number of manuals have proposed requirements that consider the expression of patients’ values and preferences in manual development, and the dimensions of manual development are constantly enriched. However, manuals outlining the requirements of patient participation are still not comprehensive and can continue to improve.
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.
Chinese medicine (CM) has significant clinical effects in the treatment of tracheal-bronchitis. It is of important clinical significance to formulate guidelines for the diagnosis and treatment of tracheal-bronchitis based on the characteristics of TCM syndrome differentiation. The Respiratory Disease Branch of China Association of Chinese Medicine and Respiratory Disease Branch of China Medical Association of Minorities organized and established a multi-disciplinary background working group, based on the World Health Organization Handbook for Guideline Development and the Guideline Development Handbook for Diagnosis and Therapy of Integrative Medicine to develop this guideline. It was developed through technical links such as clinical problem investigation, evidence collection and evaluation, Delphi consultation, and expert consensus meetings. Based on the current best evidence, CM intervention costs and expert experience, 25 recommendations were established to standardize the etiology and pathogenesis of tracheal-bronchitis, syndrome differentiation and treatment, prevention, and care, etc., which can be used by physicians at different levels of medical institutions.
Objectives To assess the quality of clinical practice guidelines for primary hepatic carcinoma published in 2016 and 2017 in China. Methods CNKI, WanFang Data, CBM and VIP databases were searched for clinical practice guidelines for primary hepatic carcinoma in China. The search date was from Jan. 1st, 2016 to Jan. 1st, 2018. Four researchers independently selected literatures and extracted data according to the inclusion and exclusion criteria. The Appraisal of Guidelines for Research and Evaluation Ⅱ (AGREE Ⅱ) was utilized to assess the methodological quality of the guidelines. Results A total of 7 guidelines were included. The average scores of six domains for these guidelines were: 65.1% for scope and purpose, 39.4% for stakeholders’ involvement, 64.3% for rigor of development, 55.6% for clarity of presentation, 61.8% for applicability and 6.1% for editorial independence. Conclusions The quality of clinical practice guidelines for primary hepatic carcinoma in China is relative high, of which the recommendations are of great value in clinical practice, yet still required to be improved in some ways.
This paper introduces the development and changes of clinical practice guidelines based on the enlightenment of the Reporting Items for Practice Guidelines in Health Care (RIGHT), and provides policy recommendations.