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Discovery & Development Clinical Trials

Women on the Verge of a Clinical Breakthrough

Traditionally, women – and particularly women from ethnic minorities – have been under-represented in clinical trials. Until relatively recently, the prevailing belief was that a medicine could be tested on white men and would work similarly in everyone else. We now know that is not the case, and despite efforts from regulators and advocacy groups over the past decade, change has been slow.

We are really only now beginning to understand some of the differences between men and women, and a lot of the findings are still from basic research – differences in neurology or gastroenterology. Right now, we have the most information on cardiovascular disease but even in this field there are still many unanswered questions, such as why certain treatments work better or worse in women, or why women suffer more in certain aspects of cardiovascular disease than men. We know that there are differences in many areas in terms of prevalence and symptoms, but in most cases we don’t know why. Does a specific treatment work in one sex and not the other? Do men and women require a different dosage? We seem to have many more questions than answers. We need to bring research to the bedside and test hypotheses.

Does a specific treatment work in one sex and not the other? Do men and women require a different dosage? We seem to have many more questions than answers.

For years, my colleagues and I at the Society for Women’s Health Research have been calling for more women to be included in Phase I and II trials. Women are all too often only included in Phase III trials, after initial safety and efficacy analyses have been completed. There are barriers; for example, women still take on the majority of child rearing and caring for elderly relatives, making long stays or frequent trips to trial centers challenging. And, there are a range of social and cultural issues that further reduce participation from ethnic minorities. There is still mistrust among the African-American community after infamous incidents like the Tuskegee trials, where life-saving treatment was deliberately withheld from African-American trial participants at the cost of many lives.

The aim is not to increase participation of women to 50 percent in all trials, but to achieve statistically significant, clinically balanced representation. If you’re designing a trial for Lupus, which is eight times more prevalent in women than in men, then ideally your study population should be predominantly women.

Recruitment is just the beginning; you also have to analyze your results to look for sex or ethnicity differences. Why is there a 40 percent failure rate and, crucially, in which patients? Hypotheses about the impact of sex and ethnicity need to be considered at the beginning of the trial (not afterwards) to ensure that analyses are statistically significant. The FDA has been active in encouraging this approach. Its new Drug Trials Snapshots provide information on age, race, and sex of clinical trial participants for a drug, and will put more pressure on companies to make sure they have the right data.

It’s been a long time coming, but we’ve certainly seen some major progress in the last year.

It’s not just clinical trials where we need to see more female representation. Beginning this year, all preclinical studies funded by the National Institutes of Health are required to take account of sex differences in the cells or animals being used. It’s a big change in thinking for a lot of researchers and it will introduce some extra costs and complexity, so it’s going to be a slow process. But ultimately it’s the only way we can unravel the full complexity of sex differences, “from womb to tomb”.

It’s been a long time coming, but we’ve certainly seen some major progress in the last year. I believe that if regulators continue to make trial participation more transparent, the public is going to demand greater inclusion of women and minorities in clinical trials. The test for pharmaceutical companies will be how they respond to that demand. Doing what we have always done is no longer an option – we must close the gaps in our knowledge. We must achieve the levels of participation in clinical trials that are necessary to understand the biomedical differences between men and women. This will truly lead to our overarching goal – ensuring that the right patient, receives the right treatment at the right time.

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