Factor Replacement Trials Must Include Women

Dear pharmaceutical companies, 

I am speaking to every one of you who makes a factor replacement product for hemophilia. 

Thank you for developing more options and safer products for our community. Because of your research, we have products that greatly improve our lives. We see the difference. Many of our young people live “normal” lives with hemophilia, which is so different from past generations.

But, where is the clinical research on women? To the best of my knowledge, there have been no clinical factor replacement trials for women with bleeding disorders.

I find this disturbing. Part of acknowledging bleeding issues in women is conducting research on the efficacy of factor replacement products to control their bleeding.

Recently, I was at a national hemophilia gathering. I spent time with pharmaceutical company representatives and asked each one about the research on women. My favorite response came from the representative of a company that is in the spotlight for a life-altering product.

“We cannot easily conduct research on women with hemophilia as they do not bleed like men,” he said. “This means we have no way to really measure effectiveness.” I told him there are many ways to measure bleeding in women. There are women with mild, moderate, and severe hemophilia — with levels identical to men — who could benefit from female-specific research. And women bleed every month. It would be easy to measure factor effectiveness by testing it on women with horrendous menstrual blood loss. The rep looked surprised as he said, “I had never thought of that.”

Women bleed every month. Bleeding issues can impact their lives. Some women use desmopressin, tranexamic acid, hormones, or IUDs to control bleeding. Others need factor replacement products to minimize blood loss. Still others struggle with joint bleeds — large and micro — that require factor products to promote healing.

Women do bleed differently than men. Women are using products off label because clinical trials on them do not exist. I hope to see this change.

As more women with bleeding disorders unite for better care, their voices grow louder. Women are demanding appropriate diagnosis and treatment that will lead to a better quality of life. The first company to conduct a clinical trial for women with hemophilia will open the floodgates for women to receive treatment they have too often been denied.

Pharmaceutical companies, wake up. Notice the hurting women in your midst. Break into a market that has consumers begging for treatment. The time is now. Will your company be the one to capture the market and improve the quality of life for women diagnosed with hemophilia and as symptomatic carriers? How long will we have to wait to find out?  

Eagerly waiting,

Shellye (and women with hemophilia everywhere)

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