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Business & Regulation Business Practice, Standards & Regulation

Focus on Bleeding Disorders

Healthy People 2030 (HP2030), the US Department of Health and Human Services’ latest report outlining its goals to improve health outcomes for the American public, has identified bleeding disorders as a pressing public health issue alongside diabetes, cancer, and heart disease. Though these disorders are rare, they are often complex and chronic. But are the pharmaceutical interventions currently available for treatment effective?

“In my 30-year career, I’ve seen numerous advancements in the treatment of bleeding disorders,” says Amy Shapiro, Medical Director and CEO of the Indiana Hemophilia and Thrombosis Center in Indianapolis, and National Hemophilia Program Coordinating Center advisor. “Both factor and non-factor therapies are available to address the physiologic mechanism of blood clotting and researchers are exploring the potential of gene therapies for conditions like hemophilia.”

But questions arise as to whether this is enough. Shapiro acknowledges that every patient responds to treatment differently, which makes a holistic approach to treatment essential. She says, “Pharmaceutical and biotech companies are critical partners when it comes to treatment innovation for people with bleeding disorders – and a comprehensive care model involving the support of multidisciplinary teams is important for preventative care.”

Shapiro says regulators have a part to play, too. For example, further guidance is needed to develop drugs for ultra-rare clotting factor deficiencies. So what steps can the industry take next to address these issues? Shapiro points to HP2030 as a source of guidance. The report highlights one area of particular importance – the need to enhance existing data and collect more information about people with bleeding disorders to strengthen access and treatment programs.

“The fact that bleeding disorders are included in HP2030 is an accomplishment for members of the community and shows a commitment to enriching patient lives,” Shapiro says. “It will certainly help in addressing barriers that exist today and build on the work already happening across the industry.”

Find the full report here:

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About the Author

Maryam Mahdi

Associate Editor

After finishing my degree, I envisioned a career in science communications. However, life took an unexpected turn and I ended up teaching abroad. Though the experience was amazing and I learned a great deal from it, I jumped at the opportunity to work for Texere. I'm excited to see where this new journey takes me!

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