BLUE BELL, Pa., July 10, 2025 (GLOBE NEWSWIRE) — Slipstream, a trusted technology partner serving the Pharmaceutical and Biopharmaceutical industry, is proud to announce its co-authorship of a groundbreaking study on Hereditary Hemorrhagic Telangiectasia (HHT), recently published in the American Journal of Hematology.
The study, “Characterizing the Healthcare Utilization and Costs of Hereditary Hemorrhagic Telangiectasia”, is the largest study of patients with HHT ever conducted, including over 24,000 patients living with this rare, inherited bleeding disorder. The research reveals that, within this sample, the direct medical costs for HHT exceeded $450 million annually in the United States, driven primarily by the treatment of bleeding-related complications, including anemia.
“By leveraging Komodo Health’s Healthcare Map®, which includes data from over 330 million US patients, we have been able to characterize a rare disease whose natural history and burden have never been well-defined or fully understood,” said Tracy J. Mayne, PhD, Senior Vice President of Regulatory and Life Science Research at Slipstream. “We now know that nearly 60% of patients diagnosed with HHT have bleeding so severe that it causes anemia, and a significant portion of those patients receive frequent IV iron and/or red cell transfusions. The burden for both patients and the healthcare system is enormous, with significant implications for advocacy and drug development.”
The research team, which includes collaborators from Massachusetts General Hospital, Cure HHT, and Diagonal Therapeutics, utilized Komodo real-world claims data to evaluate the economic burden of disease in a cohort of more than 24,000 patients in 2022 and 2023.
Key findings from the publication include:
- Per patient per year (PPPY) costs for people living with HHT are comparable to or surpass those of other rare and resource-intensive diseases.
- Bleeding and its consequences were identified as the primary drivers of healthcare costs.
- Mean PPPY costs for all HHT patients were >$19,000 across 2022 and 2023, about 20% higher than those for sickle cell disease.
- For HHT patients with anemia, the mean PPPY costs were approximately $27,000
- Patients with HHT receiving hematologic support (iron infusions and/or red blood cell transfusions) had mean PPPY costs of approximately $40,000
- HHT patients with anemia, while accounting for nearly 60% of the HHT patient population, were responsible for approximately 80% of the direct medical costs.
- Notably, the prevalence of liver transplantation among HHT patients, arising from complications of liver AVMs, was 40 times greater than in the general U.S. population.
“Slipstream was founded on the belief that technology should empower the life sciences industry to move faster, work smarter, and operate more efficiently,” said Brandon McKay, Chief Executive Officer at Slipstream. “Studies like this one validate how the right data, paired with the right expertise, can redefine what is possible in healthcare. This collaboration is exactly the kind of work we strive to do partnering with innovators across the ecosystem to unlock the power of real-world data and digital platforms. Our goal is always to accelerate impact where it matters most, improving patient outcomes.”
This landmark study provides critical evidence of the high level of unmet need for patients living with HHT, who have few treatment options and no approved drugs that modify disease or are able to slow or halt progression. This study strengthens the case for future therapeutic development and is an example of the value real-world evidence and the Komodo health database bring to the rare disease space.
The full article is available online through the American Journal of Hematology: https://doi.org/10.1002/ajh.27756
About Slipstream
Slipstream is a trusted technology partner exclusively serving the Life Sciences industry. Our global team brings deep domain expertise and pragmatic, end-to-end solutions that simplify complex challenges across the entire product lifecycle. We deliver with speed, compliance, and foresight— empowering clients to accelerate research, optimize time to market, and improve patient outcomes, ensuring technology keeps pace with their life-changing mission.
About Slipstream’s Digital CRO Practice
Slipstream’s Digital CRO Practice is advancing drug development by utilizing real-world data and current data science to create external/synthetic controls and real-world/placebo hybrids as primary basis of approval for drugs developed to treat rare diseases. As a market leader in digital pharmaceutical digital platforms, Slipstream combines deep therapeutic expertise with real-world regulatory experience and industry-leading technology to help rare disease companies achieve approval faster and at significantly reduced cost. Using large national databases, Slipstream also assists in site identification and recruitment for rare disease trials.
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