FDA's Gottlieb urges more clinical trial participation by women

Female Factor

In a speech acknowledging National Women’s Health Week, Food and Drug Commissioner Scott Gottlieb reiterated the “importance of making sure women are appropriately represented in clinical trials,” according to a blog by Michael Causey on the Association of Clinical Research Professionals (ACRP) website. He commended the U.S. Food and Drug Administration’s (FDA’s) Office of Women’s Health (OWH) and acknowledged that it provided more than $40 million for 371 projects to “inform the FDA’s work about issues specific to women’s health and sex differences” at an agency event May 16 on “The Great Debate: What is Enough…Women in Clinical Trials?”

According to Gottlieb, “We recognize that there are meaningful differences between men and women when it comes to disease, and the role that sex plays in the onset of illness and the maintenance of health. This wasn’t always the case. And years of neglect of these issues, and at times ignorance of them, have left us with disparities when it comes to the delivery of healthcare. This is reflected in many places across our system. One is the way that we enroll clinical trials. And make sure women are represented in appropriate numbers in studies. Another is the manner in which we focus our efforts to develop new medical innovations, and how much of our emphasis is focused on diseases that affect women disproportionately. Or on medical conditions that are experienced only by women.”

Gottlieb said that there have been some very positive results over the past few years. For instance, an analysis by the OWH of people enrolled in late-phase clinical trials supporting new molecular entity submissions determined that 52 percent were women in 2015, up from 39 percent in 2000.

He also discussed some of the findings of the FDA-led Decadal Review, “a study that analyzed clinical trial safety and efficacy by sex across 34 drugs and five cardiovascular disease indications from 2005 to 2015,” Causey reported. The study also did an exploratory analysis of the “inclusion and criteria” for five of the clinical trials. The objective was to figure out whether the parameters influenced patient enrollment.

As Gottlieb said, “The study found minimal gender differences in drug efficacy and safety profiles. It also found that women were well represented in trials of drugs for hypertension and atrial fibrillation, and overrepresented for pulmonary arterial hypertension. On the other end of the spectrum, women were underrepresented in trials studying therapies for heart failure, coronary artery disease, and acute coronary syndrome. In short, we still have more work to do.”

In addition, Gottlieb challenged clinical investigators: “More work is needed to identify factors leading to under-participation of women in cardiovascular clinical trials in certain areas, notably heart failure, coronary artery disease, and acute coronary syndrome.”

Gottlieb stressed that FDA is trying to do its part in improving the participation of women in clinical trials. As he said, “To advance all of our shared goals, the FDA and its Office of Women’s Health are leading a number of other initiatives…Among the steps we’re taking are our Diverse Women in Clinical Trials Initiative, which involves a consumer awareness campaign, as well as resources and workshops for health professionals and researchers. We’re also organizing training webinars with national organizations on recruitment and retention of women in clinical trials – nationally and globally; and we’re funding and conducting research initiatives that facilitate the FDA’s regulatory decision making to advance the understanding of sex differences. We’re also disseminating free FDA health educational materials through outreach activities and collaborative partnerships.”