Factor Replacement Trials Must Include Women

Shellye Horowitz avatar

by Shellye Horowitz |

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Regeneron, Intellia

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)


Note: Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Hemophilia News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to hemophilia.


Paul Clement avatar

Paul Clement

There have been several clinical trials of factor replacement therapy involving women with bleeding disorders. One phase I trial currently recruiting patients involves the use of Eloctate or Alprolix to control heavy menstrual bleeding https://clinicaltrials.gov/ct2/show/NCT03272568?term=advate%2C+female&cond=Hemophilia&rank=4.
There is also a clinical trial of Emicizumab (trade name Hemlibra) open to women which is currently recruiting. Several earlier trials were also open to women, but required them (as well as men) to have severe or moderate hemophilia, which eliminates most women. And for earlier trials which included only men, the product indications were for "patients with hemophilia" which does not exclude women. Rather than ask for new clinical trials with women (of already marketed products), it would be better to ask pharmaceutical companies to submit for an "additional indication," which would them to specifically state the product could be used to control bleeding in women, and would not involve the very costly and lengthy clinical trial process. An additional indication for women would make it easier for doctors to prescribe the drug for women and also make it harder for insurance companies to deny coverage for the drug due to a lack of indication for women.

Shellye Horowitz avatar

Shellye Horowitz

Thank you, Paul - this is really good information. I like the point you make re: lobbying for "additional indication" for women to use factor products. Do you have the link to the Emicizumab trial that is open to men and women?
Thank you, again!

Paul Clement avatar

Paul Clement

Hi Shellye,

Here is a link to two emicizumab clinical trials open to women: one has reached its participant goal and is currently active and not recruiting, the other is still open, but has more restrictive inclusion criteria (participants must be planning to have minor surgery within 60 days of enrollment).
https://clinicaltrials.gov/ct2/results?term=female%2C+emicizumab&cond=Hemophilia&rank=1#rowId0 (if link has expired, search the site for hemophilia, female, emicizumab).

Shellye Horowitz avatar

Shellye Horowitz

Thank you!!!


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