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Experimental Research Design 2
This is a mind map about experimental research design 2, including types of clinical trial comparisons, Statistical analysis of data sets, estimation of sample size in experimental studies, etc.
Edited at 2024-01-20 12:38:56- bacteria
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Experimental Research Design 2
- bacteria
This is a mind map about bacteria, and its main contents include: overview, morphology, types, structure, reproduction, distribution, application, and expansion. The summary is comprehensive and meticulous, suitable as review materials.
- Plant asexual reproduction
This is a mind map about plant asexual reproduction, and its main contents include: concept, spore reproduction, vegetative reproduction, tissue culture, and buds. The summary is comprehensive and meticulous, suitable as review materials.
- Reproductive development of animals
This is a mind map about the reproductive development of animals, and its main contents include: insects, frogs, birds, sexual reproduction, and asexual reproduction. The summary is comprehensive and meticulous, suitable as review materials.
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- Outline
experimental research design
16.4 Types of clinical trial comparisons
Superiority trial: proves that the therapeutic effect of the experimental group is better than that of the control group. The control group can be divided into positive control or placebo control.
Non-inferiority trial: There is not much difference in efficacy between the experimental group and the positive control group. To a certain extent, it can be considered that there is no essential clinically meaningful difference in their efficacy, proving that the experimental group is the same as the positive control group. The difference in therapeutic effect between the two groups does not exceed the clinically acceptable range, that is, the experimental group may be slightly worse than the control group in terms of therapeutic effect, but the difference does not reach the critical value.
Equivalence trial: proves that the therapeutic effects of the experimental group and the control group are equivalent, that is, whether the therapeutic effect of the experimental group is slightly better or slightly worse than that of the control group, the difference in therapeutic effect does not exceed the clinical boundary value
The control group must choose a positive control
The selection of clinical cutoff value Δ in non-inferiority and equivalence trials is very important. The confidence interval control method is used to determine the results: superiority trials compare the difference in efficacy with 0, and non-inferiority trials and equivalence trials compare the difference in efficacy with clinical Boundary value Δ comparison
16.5 Statistical analysis of data sets
full analysis set (FAS)
The data set obtained after the least and most fair elimination of the data of all randomized subjects according to the intention-to-treat analysis principle
Should include all subjects who have used study drugs after randomization and have partial efficacy evaluation data
For the main efficacy indicator, if the case data of the entire treatment process cannot be observed, the last observed result can be carried forward to the end of the trial (last observation carry forward, LOCF). The number of subjects in each group who evaluates the efficacy at the end point. Be consistent with the start of the trial
Intention-to-treat analysis (ITT): All patients who have been randomized should be followed up, evaluated and analyzed according to the assigned treatment group, regardless of whether they comply with the planned treatment process, not according to the actual treatment group. Accepted treatment groups for analysis to maintain randomization results
Per-protocol set (PPS): all cases that can complete the study according to the trial protocol. Cases that seriously violate the trial protocol or drop out midway, such as poor compliance, lost to follow-up, use of prohibited drugs, seriously exceeding the visit window, not meeting the inclusion criteria, etc., will not be included
Safety analysis data set (safety set, SS): All subjects who participate in randomization, have used the experimental drug at least once, and have some safety evaluation data are included in the safety analysis set. Safety analysis data set is used for safety evaluation
16.6 Estimating sample size in experimental studies
Considerations
The estimated value of the primary endpoint indicator and its standard deviation: query the literature or estimate based on the results of previous studies and preliminary experiments. Quantitative - collect the expected mean and standard deviation; Qualitative - collect the expected incidence rate or percentage
Clinical boundary value: Δ is the difference in efficacy between the test group and the control group. It cannot exceed the maximum clinically acceptable difference range and should be smaller than the difference that can be observed in the superiority test of the positive control drug over the placebo. It should be determined by Given by a clinician or expert in the field
Test level: Type I error probability α, the probability of incorrectly rejecting the null hypothesis
Test efficacy: Power, when the alternative hypothesis is correct, the possibility of accepting it through statistical inference. When the experimental group is truly effective, the probability that the experimental group can be concluded to be valid through one sampling. 1-β should be at least 80%
16.1.1 Calculation method of sample size for superiority trial
Classification indicators
Metrics
16.6.2 Calculation method of sample size for non-inferiority trials
Classification indicators
Metrics
16.7 Design and evaluation of questionnaires and case report forms
Questionnaire, also known as questionnaire: a tool used to collect information in medical research. It is in the form of a carefully designed question form, used to evaluate different characteristics of the research object, and measure the attitude, behavior, and Psychological and other characteristics
Case report form (CRF): A document designed in accordance with the trial protocol and used to record the data of each subject during the trial.
16.7.1 The role of questionnaire/CRF
An important part of ensuring complete and accurate data collection and quality of clinical research
It is an important means of obtaining research data in clinical research and a carrier for collecting, recording and preserving experimental data; it can be used as an original record for scientific research archive preservation; it can provide original data to facilitate statistical analysis; it can facilitate verification by the health administration department
16.7.2 Design principles of questionnaire/CRF
Follow clinical study protocols
Comprehensive and complete, concise and to the point
Easy to understand and easy to fill in
Facilitates data entry and statistical analysis
16.7.3 Things to note in questionnaire/CRF design
The order of questions should be logical
The setting of options should be comprehensive and reflect the purpose of the research
Related question settings
Coding settings should be unified
Please indicate how to handle unknown situations when filling out the form.