ObjectiveTo explore the parameter selection of different sample size estimation methods and the differences in estimation results in single-group target value clinical trials with rate as the outcome evaluation index. MethodsWe conducted a literature review to assess the method of target value selection for single-group target value clinical trials. Then, different values of target value (P0), clinical expected value (P1), and class II error level (β) were set through numerical simulation. Sample size results estimated using different sample size estimation methods were obtained using PASS software. The coefficient of variation, range/mean, analysis of variance and other methods were used to compare the differences between different methods. ResultsAnalysis of the data simulation results showed: when the expected value P1 was fixed, the sample size first decreased rapidly and then decreased slowly along with the increase or decrease of the targeted value P0 on both sides of the sample size limit value. When the difference between P0 and P1 was within 0.15, the ratio before and after correction could be controlled within 0.9. When the difference between P0 and P1 was more than 0.6, the ratio before and after correction approached 0.5. When P0+P1≈1, the ratio of different standard error choices (Sp0 or Sp1) to the estimated sample size was close to 1. When 0.65<P0+P1<1.35, the ratio of different standard error choices (Sp0 or Sp1) to the estimated sample size was about 3:1. When the confidence was 0.8, P0 and P1 were between 0.25 and 0.75 and between 0.20 and 0.80, respectively. We found little difference among the sample sizes estimated using these five methods (CV<0.10, range/mean<0.2). ConclusionThere are some differences among different sample size estimation methods, however, when P0 and P1 values are around 0.5, the differences between different methods are small, suggesting that appropriate methods should be selected for sample size estimation.
Objective To understand the feasibility of application of objective performance criteria single arm trial in clinical efficacy evaluation of traditional Chinese medicine, and put forward theoretical countermeasures for the difficulties in application, so as to provide references for promoting the application of objective performance criteria single arm trial in clinical efficacy evaluation of traditional Chinese medicine in the future. Methods Semi-structured qualitative interviews were conducted among clinical experts and methodologists who were involved in objective performance criteria single arm trials using purposive sampling combined with snowball sampling and a preset interview outline. To explore the application situations, influencing factors and difficulties in the application process of objective performance criteria single arm trial in the clinical efficacy evaluation of traditional Chinese medicine, and put forward theoretical countermeasures. Results A total of 12 respondents were interviewed, including 7 clinical experts and 5 methodologists. The interview content was summarized and formed into 216 codes. After category analysis, 5 categories were formed, including the applicable situations, advantages, limitations, difficulties in the application process and theoretical countermeasures. The respondents believed that objective performance criteria single arm trial could be used as an alternative plan when controlled trials could not be carried out, or as a preliminary exploration or evidence supplement. It had the advantages of reducing sample size, good patient compliance, saving resource input, and relatively rigorous design. However, its application was limited due to low awareness and unclear level of evidence. The application difficulties included the setting of objective performance criteria, sample size calculation, quality control and statistical analysis. It was suggested to form a standard to promote its application in the efficacy evaluation of traditional Chinese medicine. Conclusion The design of objective performance criteria single arm trial is consistent with the characteristics of clinical research of traditional Chinese medicine. However, because there is no control group, the validity of objective performance criteria single arm trial is challenged, which hinders its further application in the clinical efficacy evaluation of traditional Chinese medicine. By standardizing the selection of objective performance criteria, calculation of sample size, statistical analysis methods and research implementation steps, the strength of its demonstration and the recognition can be improved to a certain extent, but attention should be paid to avoid its misuse. In the future, the objective performance criteria single arm trial may be used as a basis to further explore the new direction of scientific research methods for clinical efficacy evaluation of traditional Chinese medicine.
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.
In the formulation of the clinical question of traditional Chinese medicine clinical practice guidelines, even if the intervention elements (intervention or control) have an appropriate scope, guideline developers are still faced with a variety of interventions. By analyzing the difficulty and necessity of priority selection of intervention interventions, we propose the approach of extending expert evidence to the process of priority selection of intervention interventions, and further provide the methodology of expert evidence data collection table design, application, data presentation and expert decision-making method to provide references and guidance for guideline developers.
Clinical practice guidelines will be incorporated in different evidence sources and evidence types, the research and exploration of multiple sources of evidence at China and foreign, this paper advocates the construction of both characteristics of multi-source evidence of traditional Chinese medicine, by combing 70 guide manuals, analyze the necessity and advantages of multi-source evidence construction, and further from the multi-source evidence source and evidence logic theory interpretation and application, in order to provide methodological references for the formulation of evidence-based clinical practice guidelines of traditional Chinese medicine.
Currently, the recommendations of the clinical practice guidelines related to acupuncture in China and abroad are opaque to the source of the acupuncture prescription, there is a lack of comprehensive evaluation of the rationality of the acupuncture prescription, and the standards for the selection of the acupuncture prescription are opaque and nonstandard, and the writing and reporting details are insufficient, thus affecting the clinical applicability of the guidelines. To a certain extent, the utilization rate of the recommendations of the guidelines is low. This paper discusses the origin, rationality comprehensive evaluation, priority selection, writing and reporting of acupuncture prescriptions, and puts forward detailed methodological suggestions, to provide guidance makers of methodological optimization thoughts and suggestions for the evaluation, selection and writing of acupuncture prescriptions in the recommendations.
After the completion of a clinical trial, its conclusion generally depends on the results of statistical analysis of the main outcome, that is, whether the P-value in the hypothesis test is less than the α level of the hypothesis test, usually α=0.05. The size of the P-value indicates the sufficient degree of reason for making the hypothesis judgment, and can be interpreted as to determine whether a conclusion is statistically significant but does not involve the difference in the degree of drug effects or other effects. Fragility index, which is, the minimum number of patients required to change the occurrence of a target outcome event to a non-target outcome event from a statistically significant outcome to a non-significant outcome, can be used to assist in understanding of clinical trial statistical inference results and assisting in clinical decision making This paper discusses the concept, calculation method and clinical application of the fragility index, and recommends that the fragility index be routinely reported in all future randomized controlled trials to help patient clinicians and policymakers make appropriate and optimal decisions.