FDA Publishes Updated ‘Roadmap’ For Advancing Women’s Health

The Food and Drug Administration’s Office of Women’s Health (OWH) has updated its research roadmap to reflect key areas where new or further research is needed concerning women’s health, as well as priorities and regulatory questions for the office. Device- and diagnostic-related priorities discussed in the roadmap include biocompatibility testing for medical device materials, the identification of biomarkers related to women’s health, and using data mining and other tactics to identify postmarket safety issues.

The OWH, which was established in 1994, says it has funded more than 400 research projects aimed at spurring innovation “in scientific knowledge, resulting in the discovery of novel methodologies and technologies, and improvements in the diagnoses and treatment of health conditions that impact women.”

There has been a push recently on the federal level to bring women’s health issues front and center. Last year for instance, the Biden administration tapped Jill Biden to spearhead a White House effort to find new ways for the federal government to boost women’s health. (Also see "First Lady To Lead First Ever Initiative On Women’s Health" - Medtech Insight, 14 Nov, 2023.)

And in September, the FDA issued a mandate requiring providers inform patients about their breast density on mammogram reports and any additional screenings they might need. (Also see "Mammograms Should Inform Women About Breast Density, FDA Says" - Medtech Insight, 13 Sep, 2024.)

Breast density, according to the American Cancer Society, presents an intrinsic elevated risk of developing cancer and can often mask tumors during routine screenings.

The roadmap will serve as a catalyst for research collaborations both internal and external to the FDA.

“Although many critical women’s health issues warrant further examination, future OWH-funded research should focus on areas where advancements will be directly relevant to FDA as it makes regulatory decisions,” the agency says. The roadmap creates strategic direction for OWH to help maximize the impact of OWH initiatives and ultimately promote optimal health for women.”

The FDA stresses the importance of focusing on women’s health because research on topics specific to women have been traditionally underfunded despite women comprising the majority of the US population.

Priorities

A top priority for the office emphasized in the updated roadmap is advancing the safety and efficacy of FDA-regulated therapeutic and diagnostic products used by women. Here, the FDA wants to explore how sex differences — and differences among diverse populations of women — may affect regulatory decision-making around medical product safety and effectiveness or play a role in the use of other regulated products, such as cosmetics.

The agency says it wants to also focus on expanding its capacity to effectively evaluate FDA-regulated products used by women “throughout all life and disease stages.”

Another updated priority is improving clinical study and design analysis to better identify and evaluate sex differences and gender influences related to FDA-regulated products. The update includes proposals to enhance best practices for recruitment and retention of diverse populations of women in clinical studies as well as considerations for social and behavioral research studies, such as patient-reported outcomes, to improve FDA-regulated therapeutic products.

As example of a clinical trial targeted specifically to women is one sponsored by Shockwave Medical that began last year.

The EMPOWER CAD (coronary artery disease) enrolled hundreds of women — and only women —across the US and UK to determine whether positive results in both sexes from earlier studies with Shockwave’s C2 coronary IVL catheter can be replicated in female patients undergoing a percutaneous coronary intervention. (Also see "Shockwave Begins First All-Female Study Of Coronary Interventions" - Medtech Insight, 26 Jul, 2023.)

As noted in that study, heart disease kills more women in the US than any other disease — a fact not even many women are aware of. In 2020, heart disease accounted for one in five female deaths, according to the Centers for Disease Control, yet only 56% of women realize it’s their number one killer. And often, symptoms present differently in women than in men, resulting in missed diagnoses.

Diabetes is another common condition that can affect women differently than men include diabetes, while autoimmune diseases, migraines, osteoporosis, and various cancers impact women disproportionately.

The roadmap also lists pain as potentially influenced by sex and states more research is needed in this area. For example, the perception of pain is influenced by sex hormones and their interaction with endogenous opioid function, while coping strategies to deal with pain vary depending on sex.

“Therefore, both gender and sex can lead to differences in the way pain is reported, treated, and managed,” the roadmap says.

OWH, as highlighted in the updated roadmap, also wants to develop tools and methods that can “identify, develop, and evaluate” biomarkers in nonclinical and clinical studies that can better measure and predict the safety and effectiveness of FDA-regulated products in women.

Additionally, the office wants to expand the agency’s knowledge of diagnostic, prognostic, and predictive biomarkers that can detect and predict the severity and progression of conditions in women and identify patients at risk for adverse events.

“Emergency preparedness and response plans should include studies of sex and gender differences to better understand the needs of women, such as the impact on pregnant and lactating women.” — US FDA Office of Women’s Health

Other priorities outlined in the update include expanding data sources to enhance techniques for data mining, data linkage, and large data set analysis that can be used to assess the post-market safety and effectiveness of FDA-regulated products used by women as well as providing additional tools to help identify adverse events in women.

The office also wants to improve health communications to foster the creation of easily accessible, clear, and useful information to help women and health care professionals make informed health-related decisions while promoting emerging technologies to address women’s health conditions.

And lastly, the OWH seeks to combat emerging threats to women’s health.

In this research area, the office wants to focus on identifying threats before they become crises and ensuring medical countermeasures — such as drugs, vaccines, and diagnostic tests —include women during development and include an evaluation of sex differences.

For example, the roadmap cites the biocompatibility of materials related to medical devices implanted in women, the effects of inks and colors absorbed through activities such as tattooing or microblading on diverse populations of women, and sex differences in the adverse effects related to use of electronic nicotine delivery systems as areas of concern specific to women.

Further, emergency preparedness and response plans should include studies of sex and gender differences to better understand the needs of women, such as the impact on pregnant and lactating women.

The agency should also explore, according to the roadmap, how FDA-regulated therapeutic products can counter growing threats to women’s health, such as maternal mortality and morbidity, congenital syphilis, and mental health disorders.

As noted in the roadmap, the FDA, through OWH, seeks to fund research “that will expand our understanding of the science of women’s health, close knowledge gaps, and contribute to policy, outreach, and education initiatives important for the health of women.”

The lack of research and data on women’s health, according to OWL, contributes to suboptimal medical care and health outcomes for women in the US. As an example, the roadmap cites data showing the US has the worst health outcomes in the world among high-income nations and the American women have the highest rate of maternal mortality among high-income countries.

Maternal mortality increased from 17.4 deaths per 100,000 live births in 2018 to 32.9 deaths in 2021, according to US government statistics, with women of color disproportionately affected.