By Susan Blumenthal, M.D. and Sarah Colon
As recently as two decades ago, women's health was very much neglected in the halls of public policy, at the research bench, and in clinical settings. In 1990, a GAO Report revealed that only 13 percent of the National Institutes of Health (NIH) budget was spent on women's health research and that data was not being adequately analyzed for sex and gender differences. These startling findings led to the passage of congressional legislation in 1993 requiring that women and minorities be included in clinical trials unless the study was sex specific (e.g., prostate cancer), and that studies be analyzed for sex differences. In that same year, a Deputy Assistant Secretary for Women's Health position was established within the U.S. Department of Health and Human Services (HHS) to help rectify inequities in women's health and to develop new cross-cutting initiatives across its agencies including NIH, CDC and FDA. I am honored to have served in this position and to have established programs including the National Centers of Excellence in Women's Health program at academic centers around the country to foster interdisciplinary research, training, and education as well as promote the career development of women in medicine, the National Women's Health Information Center (an information resource hub accessible at womenshealth.gov and through a toll-free telephone number), and to have appointed regional women's health coordinators across the country, among other initiatives.
Why does a focus on women's health matter? More than 51 percent of the U.S. population is female and women make 80 percent of medical decisions in families. Women use more medications and have higher rates of reported side effects than do men. They also seek more medical care, use more health services, spend more on medications than do men, and suffer greater disability from chronic disease. In addition, research has found that illnesses including lung cancer, heart disease, AIDS and Alzheimer's, among others, may affect men and women differently, as do some medications. Genetic, biological, socioeconomic, and environmental factors contribute to these differences for men and women's health.
Yet, despite the progress that has been in made in advancing women's health over the past two decades, some critical gaps in knowledge remain. In October, 2015, the Government Accountability Office (GAO) released a report that evaluated the current status of women's participation in clinical trials supported by NIH since the 1993 legislation was enacted. The NIH had a budget of $30 billion in FY15 and funds research at more than 2,500 different institutions, supporting over 300,000 researchers. The good news: The GAO report found that the majority of participants in clinical trials were now women (57 percent) who had been underrepresented in these studies in the past. However, the agency still does not provide data on the percentages of women and men participating in studies of specific diseases (e.g., AIDS, heart disease, cancer, and depression) or by Institutes (e.g., National Cancer Institute, National Institute of Allergies and Infectious Diseases, National Institute on Mental Health, National Heart, Lung and Blood Institute). Additionally, NIH does not provide public information about whether data from the research it has supported was analyzed for possible sex differences as required by the 1993 law. As a result, according to the GAO report, "NIH is limited in its ability to identify whether women are sufficiently represented in studies in specific areas -- such as cardiovascular disease -- or if the agency-wide data inadvertently mask enrollment challenges. By not examining more detailed data on enrollment below the aggregate level, NIH cannot know whether it is adequately including women in all of the research it supports, in a manner consistent with its Inclusion Policy. Further, NIH's reporting and monitoring in this area is inconsistent with federal internal control standards, which call for agencies to have controls to help ensure effective information flow and effective monitoring of agency activities." This omission was once again underscored in a report this week from the American Heart Association that women may have a different symptom presentation of heart attacks as compared to men and that the disease may be deadlier for females in part because they are underdiagnosed and undertreated. Yet, data about how many women participate in heart disease clinical trials supported by the NIH is not readily available.
In addition, until recently, there were no requirements in place for inclusion of female animals and cells in basic science studies. The NIH is finally taking steps to rectify this serious omission. In June 2015, a new requirement was put in place that went into effect this month (January 2016) ensuring that sex and gender differences be addressed in basic and preclinical biomedical research. In the past, the majority of basic science studies had included only male animals and cells, potentially obscuring understanding of critical sex influences in health and disease. The new NIH guidance requires that all basic science research studies supported by the agency include both male and female animals and cells, unless there is a compelling reason for using only one sex, such as research on a sex specific disease like ovarian or prostate cancer. This new policy will help to identify sex-specific differences that shape hypotheses before human clinical trials are conducted.
Even as these issues are addressed at the NIH and other government agencies, it should be remembered that these regulations and recommendations do not necessarily apply to research studies conducted by private industry and other organizations that do not receive federal funds. Pharmaceutical companies, for example, are major supporters of Phase 3 clinical trials. In medical journals, sex differences are rarely reported in publications unless they are the explicit focus of the study. This must change.
Madame Curie, who was never admitted into the all-male French Academy of Sciences even after she won an unprecedented second Nobel Prize, once said, "I never see what has been done. I only see what remains to be done." While much progress has been made to advance women's health over the past 20 years, clearly more remains to be done. While women now represent the majority of participants in clinical trials supported by the NIH, this alone will not be not sufficient to advance women's health. Data obtained from clinical and basic science studies must be disaggregated to examine potential sex differences in the causes, treatment and prevention of various diseases. The percentages of females and males participating in research studies by disease and importantly any results about sex differences about specific illnesses should be reported to the scientific community and the public in the annual reports issued by the NIH Office of Research on Women's Health and by each of the NIH Institutes. Ensuring this kind of data transparency by disease and Institute will help researchers, clinicians and the public better understand sex differences and put these findings into clinical practice to the benefit of both women and men's health.
Rear Admiral Susan Blumenthal, M.D., M.P.A. (ret.) is the Public Health Editor of The Huffington Post. She is a Senior Fellow in Health Policy at New America and a Clinical Professor at Tufts and Georgetown University Schools of Medicine. She is also Senior Policy and Medical Advisor at amfAR, The Foundation for AIDS Research. Dr. Blumenthal served for more than 20 years in senior health leadership positions in the federal government in the Administrations of four U.S. presidents including as Assistant Surgeon General of the United States, the first Deputy Assistant Secretary of Women's Health, and as Senior Global Health Advisor in the U.S. Department of Health and Human Services. She also served as a White House advisor on health. She provided pioneering leadership in applying information technology to health, establishing one of the first health websites in the government (womenshealth.gov) and the "Missiles to Mammogram" Initiative that transferred CIA, DOD and NASA imaging technology to improve the early detection of breast and other cancers. Prior to these positions, Dr. Blumenthal was Chief of the Behavioral Medicine and Basic Prevention Research Branch, Head of the Suicide Research Unit, and Chair of the Health and Behavior Coordinating Committee at the National Institutes of Health. She has chaired many national and global commissions and conferences and is the author of many scientific publications. Admiral Blumenthal has received numerous awards including honorary doctorates and has been decorated with the highest medals of the U.S. Public Health Service for her pioneering leadership and significant contributions to advancing health in the United States and worldwide. Named by the New York Times, the National Library of Medicine and the Medical Herald as one of the most influential women in medicine, Dr. Blumenthal was named the Health Leader of the Year by the Commissioned Officers Association and as a Rock Star of Science by the Geoffrey Beene Foundation. She is the recipient of the Dr. Rosalind Franklin Centennial Life in Discovery Award.
Sarah Colon is a junior at Dartmouth College majoring in Biological Sciences with a minor in International Studies. She served as a Health Policy Intern at New America.