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Diversity Gaps in U.S. Rare Disease Trials and the Inequalities in Hemophilia Clinical Studies
Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, spoke to MHE about the current disparities in diversity and ethnicity in rare disease clinical trials in the U.S., as well as what he's seeing in clinical trials for diseases like hemophilia, in specific.
Managed Healthcare Executive spoke with Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, about how the disparity in diversity and ethnicity in rare disease clinical trials in the U.S. has led to gaps in understanding diseases and conditions, jeopardizing universal health, and increasing the economic burden of healthcare.
Dr. Rajasimha also shared some examples of rare diseases that have been affected most by the lack of diversity in clinical trials, but even some strategies or initiatives that are being implemented to address the diversity gap in rare disease clinical trials.
All rare diseases or most of them are most affected by the lack of diversity represented in their clinical trials, Rajasimha said.
"If I start with ALS, or Lou Gehrig's disease, (they're) more prevalent or better diagnosed in the western world than in the rest of the world. But even here, many patients suffer from lack of diagnosis or timely diagnosis fast enough. As with most diseases, early diagnosis is so critical for better patient outcomes and prognosis," he said.
Cystic Fibrosis and ALS are examples of diseases that are more diagnosed or prevalent in the U.S. and Europe. Other examples include sickle cell disease, thalassemia, hemophilia, blood related disorders, amino acid deficiencies, or leukodystrophies.
Hemgenix Gene Therapy Shows Long-Term Efficacy, Safety in Hemophilia B Patients
December 21st 2023Men with severe or moderately severe hemophilia B continue to display low bleeding rates and very little use of replacement therapies for at least three years following a single dose of the approved gene therapy hemgenix.
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Bridging the Diversity Gap in Rare Disease Clinical Trials with Harsha Rajasimha of IndoUSrare
November 8th 2023Briana Contreras, an editor with Managed Healthcare Executive, spoke with Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, in this month's episode of Tuning in to the C-Suite podcast. The conversation was about how the disparity in diversity and ethnicity in rare disease clinical trials in the U.S. has led to gaps in understanding diseases and conditions, jeopardizing universal health, and increasing the economic burden of healthcare.
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FDA Approves Adzynma for Rare Blood Clotting Disorder cTTP
November 27th 2023CTTP is rare hereditary blood clotting disorder stemming from a disease-causing mutation in the ADAMTS13 gene. This gene plays a crucial role in producing the ADAMTS13 enzyme, responsible for regulating blood clotting.
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