Why Is the Women’s Health Initiative So Important?

One of my favorite parts of my job is sharing the research regarding women’s hormonal health with my patients. I enjoy talking about the details of the data and how that data informs why I think a given treatment option is best for them. But often during those conversations, we come to topics where there isn’t a lot of data to support our decision-making.

We don’t know what causes PCOS, or why women get dementia more often than men, or the part different hormones play in women’s sex drive and sexual function. Understanding some workings of the human body has been particularly elusive, but in many cases it is a lack of research that is limiting our understanding. Issues affecting women’s bodies are more likely to fall into the latter category.

That lack of research into issues that affect women’s health is why so many of my colleagues and I were distraught and angry a couple of weeks ago when it was announced that funding for the landmark Women’s Health Initiative (WHI) study was being cut. Since that time, the administration has changed course, but it is still important to understand why studies like the WHI are so critical and why funding from the government is key to getting those studies done.

But first, some background

The history of medical research includes some dark moments when women and people of color were treated appallingly by researchers. At the same time, women were often excluded from research exploring problems and treatments that affected both men and women. Researchers worried that women’s menstrual cycles would create undesirable variability in the data, making it difficult to interpret. And they worried that women would become pregnant during clinical trials.

In 1977 the U.S. Food and Drug Administration recommended excluding women of childbearing potential from early-phase clinical trials even if they were using effective contraception, were not sexually active, or their husbands had had vasectomies. While in theory this policy protected any baby resulting from an unexpected pregnancy from exposure to experimental drugs, the real impact was failing to test how those drugs worked in women.

As a result, any drug approved in the U.S. before 1993 — a list that includes everything from the migraine medicine sumatriptan to beta blockers to Ambien — were never tested in women. In 1993 the government reversed course and began requiring that women and minorities be included in any research that receives funding from the National Institutes of Health (NIH). That same year, the WHI started enrolling participants.

What is the Women’s Health Initiative?

The WHI began as two similar randomized controlled trials trying to determine if hormone replacement therapy prevented cardiovascular disease and osteoporosis, or caused cancer, in menopausal women. One arm of the study enrolled women with an intact uterus and compared Prempro — a pill containing conjugated equine estrogens (CEEs) and medroxyprogesterone — with a placebo. The other enrolled women who had had hysterectomies and compared only oral CEEs with a placebo. There was also an observational study and a community prevention study that were rolled into the original WHI design.

Both arms of the clinical trial were stopped — meaning participants stopped taking the Prempro, CEEs, or placebo — earlier than planned due to increased rates of cardiovascular disease and breast cancer in the treatment groups. In hindsight, both the design of the study — women were enrolled at any age, and many had been in menopause for more than a decade — and the specific forms of estrogen and progesterone used were likely responsible for the negative results. When the results were published, the media coverage was fierce and, in many cases, misleading. The effects on research regarding women’s hormone health were chilling.

But one really positive thing happened when the initial trials were stopped. The National Heart, Lung, and Blood Institute, a division of the NIH, funded extension studies. The extension studies include more than 161,000 participants and collect data annually, including information on heart disease, stroke, osteoporosis, cancer, and other metrics of aging. More than 500 ancillary studies have been conducted and more than 2,400 papers have been published using data from the WHI. In short, much of what we know about health and aging in menopausal women comes from the data collected by the WHI.

Why is government funding so important for medical research?

To understand the role of government funding in medical research, it is helpful to understand all the ways studies get paid for. There are three main sources of funding for medical research: pharmaceutical companies, private grants, and government grants. Together, pharmaceutical companies and government grants make up more than 90% of the funding for medical research.

The original WHI studies were funded largely by the pharmaceutical company Wyeth, which manufactured the hormone pills used in the trials. In general, studies of specific drugs are funded by the pharmaceutical companies that manufacture those drugs. Usually they are randomized clinical trials that the companies are required to do before they can apply to the FDA to get the drug approved, or studies collecting data on patients taking the medication after a drug is approved, often because the FDA is requiring them to.

The other time pharmaceutical companies fund research is to try to expand the number of people who take a drug. With the WHI, Wyeth was looking to show that Prempro didn’t just treat perimenopausal symptoms but also prevented cardiovascular disease and osteoporosis. That, of course, didn’t pan out. Typically the goal of a study funded by a pharmaceutical company is to help them sell more of a drug.

Private grants are awarded by foundations and are usually related to specific areas of medicine. When I was in medical school, the lab I worked in received a grant from the Michael J. Fox Foundation specifically to explore how the channel the lab studied might be involved in Parkinson’s disease. Private foundations tend to be similarly narrowly focused, and the grants they can make are small.

Government funding pays for pretty much all the other medical research that gets done. Most of those grants are awarded by the federal government. State governments do provide some research funding, but the size of these awards pale in comparison to federal government grants. And government grants are particularly important because they fund areas of research that would otherwise not get studied, including diseases that disproportionately affect women and people of color, and “orphan diseases” that affect too few people to attract investment from pharmaceutical companies.

Closing thoughts

In addition to the WHI, there are other longitudinal studies helping us understand women’s health and how women age. I cite them frequently, so their names may be familiar: the Study of Women’s Health Across the Nation, the Seattle Midlife Women’s Health Study, and the Nurses’ Health Study are a few. The primary funding source of each of those studies is the federal government.

If studying women’s health is not a financial priority of the federal government, the funding for these other studies may also be in jeopardy, and important new research may never get off the ground. As it stands, diseases affecting men receive about twice the funding from the federal government as those affecting women. As a result, if we want to increase our understanding of how women’s bodies work and how best to keep women healthy, we need to continue funding the important studies that are already being done and expand to fund new studies.