Demographic subgroups

The subjects enrolled in chnical studies for small molecule and biologic products must be tabulated within relevant demographic subgroups (e.g., age, gender, and race) in annual IND reports and must be included in all BLAs. 

The extent of exposure to the drug should be described in tables. This should include the number of patients exposed to the drug for specific periods, for example, 1 day or less, more than 1 day to 1 week, more than 1 week to 1 month, or more than 1 month. The numbers should be broken down by sex and other relevant demographic subgroups. Patients included in more than one study should be counted only once. If it cannot be determined whether the same patient appears in more than one study, this should be indicated. 

The adequate representation of women and other demographic subgroups... 

Through the National Center for Health Statistics, data are available on the prevalence of numerous disease conditions in various demographic subgroups of the population of the United States. In Table IX, the prevalences of some of the diseases affecting these major organ systems are shown by age subgroups. 

The following is a table specification, or table shell, for the summary of adverse events by body system, preferred term, and maximum severity. As a rule for this summary, a patient should be counted only once at maximum severity within each subgrouping. Denominators should be calculated as the sum of all patients who had the given treatment in the demographics file. 

The report of results should include data, analysis, and presentation. Specifically, the data report should include the description of the flow of the patients through the study, as well as the number of patients included in each stage of the analysis and/or subgroup examined. The data report should provide distributions (including missing values) of basic demographic characteristics (e.g., sex and age), standard (disease-specific) prognostic... 

This broad demographic, trip planning, localised and satisfaction information on these three self-drive market subgroups helps interpret the motivational profile responses for the three groups. This material is presented in Table 3.11. 

If the size of the study permits, comparative efficacy within relevant baseline or demographic value-defined subgroups should be examined. Only relevant significant findings must be presented, and substantial elements of the inference can rely on descriptive statistics. If relevant, dose-response relationships should also be discussed. 

In multiple tables with similar data, present the data in the same order as much as possible. If the first column always has the active drug and the second column the placebo or comparative agent, then keep this order throughout the tables. In the analysis of data by demographic or disease subgroup, it is helpful to keep the subgroup of concern (i.e., women, the elderly, racial subgroups, impaired renal function) in the same column in each table. 

As we saw in the last chapter, an important goal of statistical analysis is to find an additive scale a system of measurement which has the property that treatment effects so defined are constant from patient to patient. Note that this does not require that patients be homogenous with respect to demographic characteristics, but merely that they react to treatment in the same way given some suitable scale of measurement. We cannot, however, expect that our efforts will always be successful in this respect and therefore a particular worry in drug development is that there may be subgroups of patients for whom the treatment will have quite different effects. 

Common factors that have shown to contribute to subgroup differences in safety include demographics, comorbidity, concomitant medications, disease stage, culture, ethnic factor, genetic factor, and metabolism factor. We will give some examples on these factors in this section. 

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