Improve clinical study design and conduct to better identify and evaluate possible sex differences related to FDA-regulated products.
Research in this area should help develop and promote use of better methodologies for identifying and evaluating sex differences and best practices for recruitment and retention of women. The identification of sex differences in clinical study data should help determine areas needing further exploration. For example, we need a better understanding of the biological basis of sex differences and how differences can affect the toxicity or the safety and efficacy of FDA-regulated products. Improvements to clinical study designs to facilitate identification of sex differences will complement other efforts to break down any remaining barriers to including women in clinical trials and increase the recruitment and retention of diverse populations of women in trials.
Collecting sufficient, high-quality data on women during clinical studies will help enable better analyses for identifying disparities.
2.1 Develop and promote use of clinical study designs (e.g., adaptive designs, observational studies, registries) and statistical methods (Bayesian methods, meta-analyses) to evaluate sex differences and perform other subset analyses with regard to the toxicity or the safety and efficacy (or effectiveness) of FDA-regulated products, including, but not limited to the following
- Determine how to properly incorporate and interpret data generated in clinical trials conducted in other countries specific to women or sex differences that may not be consistent with U.S. demographic profiles, especially when U.S. data are not available or are incomplete
- Develop methods for adaptive and enrichment studies to support the effective use of small sample sizes in clinical trials and studies
- Develop analytical methods for interpreting and using data on sex differences from trials and studies with small sample sizes
- Determine how best to incorporate electronic health records with other trial data when evaluating pragmatic clinical trials36
2.2 Identify and evaluate best practices for the recruitment and retention of women in clinical trials (e.g., new strategies, approaches)
2.3 Identify appropriate endpoints and outcome measures, including patient-reported outcome measures, for diseases or medical products that may affect women differently from men (e.g., certain types of cardiovascular disease present differently in men than in women).
2.4 Determine how best to define the appropriate representation of women in a given trial or study
- Identify standards such as background information needed to develop metrics
- Develop and evaluate metrics to measure the level of representation of demographic subgroups in clinical trials supporting FDA-regulated product approvals and marketing authorizations
- Evaluate to what extent the definition of the appropriate representation will affect the ability to identify sex differences (i.e., does your metric result in a sufficient number of women being included to reveal possible sex differences?)
2.5 Develop approaches and tools to track the inclusion of women in clinical trials, their demographic profiles, and the extent of subset analysis
36 Pragmatic trials or studies refer to trials or studies carried out as part of routine clinical practice, rather than as trials or studies performed under strictly controlled conditions.