FDA Guidance Advises Industry To Monitor Gender Equality In Clinical Trials

By Nick Otto

The FDA recently released final guidance advising device makers performing clinical trials to consider the gender distribution of study participants throughout various points of a study.

In order to provide better and more consistent information regarding the performance of medical devices in both sexes, the guidance recommends device makers to track differences in gender participation at various key points in a trial — such as the screening stage, after the evaluation of study inclusion/exclusion criteria, following consent, and at various follow-up stages.

Differences between men and women range from the obvious — sexual organs and body fat distribution — to some less obvious differences, such as bone density and blood viscosity, the guidance notes. Genetic gender difference can affect all levels of biological organization including cell, organ, organ system, and organism, and it can influence susceptibilities to disease.

To understand potential gender differences that may be relevant to a device’s clinical evaluation, the FDA asks investigators to consider whether gender differences may or may not exist for the disease or condition that a device is intended to treat/diagnose, such as:

• Gender-specific prevalence
• Gender-specific diagnosis and treatment patterns
• Identification of proportions of women included in past studies for the target indication
• Identification of any known clinically meaningful sex differences in outcomes related to either safety or effectiveness

The FDA recommends that sponsors include their conclusions about risk-benefit analysis both in the study protocol and the investigator training materials. Sponsors of completed studies should include any meaningful gender-based differences in outcomes in their marketing applications and in any postmarket interim reports.

Additionally, the agency also offers some recommendations for enrolling and tracking adequate proportions of men and women in clinical studies. For example, sponsors of postmarket studies could maintain patient interest by presenting the benefits of the study to participants — perhaps by sending out informational newsletters.

The guidance notes clinical investigators should also reinforce to patients the importance of trial follow-up, advising that sponsors attempt to contact patients who fail to maintain follow-up appointments.

The Regulatory Affairs Professionals Society notes that the guidance’s recommendations will apply to “ongoing, completed and post-market research . . . and trials should be designed to accommodate outstanding questions and in particular sex-specific differences.”