Takeda to Present Rare Bleeding Disorders Research Highlighting Need for Real-World Evidence at NHF Bleeding Disorders Conference 2020

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”), today presented three abstracts at the 72^nd National Hemophilia Foundation’s Bleeding Disorders Conference, underscoring the need for real-world evidence (RWE) to advance patient-centric treatment in bleeding disorders.

RWE from these studies highlight the ongoing unmet clinical need for diagnosis, personalization and treatment optimization in patients with hemophilia and von Willebrand’s disease (VWD). Takeda’s presence at NHF Bleeding Disorders Conference includes the following presentations, which are intended for scientific discussion only:

Hemophilia A and B

• Adding the patient’s voice to a hemophilia specific goal menu to facilitate Goal Attainment Scaling: a qualitative study
  • Many outcome measures in hemophilia lack sensitivity, and few are patient centric. A refined Goal-Hēm hemophilia-specific goal menu was evaluated by patients with hemophilia to determine relevancy to the challenges they face. Participants endorsed the clarity and relevance of the revised menu and expressed enthusiasm for further interaction with the tool. Patient-centered tools such as this have the potential to detect clinically meaningful changes for both clinicians and individuals affected by hemophilia, while improving patient outcomes.¹
• Incidence and prevalence of diagnosed and undiagnosed hemophilia A and hemophilia B in the USA
  • This study highlights the thousands of patients remaining undiagnosed in the US and the need for improvement in diagnosis and/or reporting.²

Von Willebrand Disease

• Analysis of Bleeding and Treatment Patterns in Children and Adolescents before and after von Willebrand Disease Diagnosis Using Data from a US Medical Claims Database
  • This abstract underscores US medical claims data that characterize the diagnosis, bleeding and treatment patterns in children and adolescents with VWD and points to the need for improved treatment and care of this patient population.³

“Real-world evidence plays a crucial role in understanding patterns of care and the patient’s holistic experience outside of rigorous clinical studies,” said Michael Denne, VP of Medical Affairs, Hematology Rare Disease Unit at Takeda Pharmaceuticals. “Takeda is a passionate patient advocate and is working to provide critically-important personalized support that helps ensure that patients find, and remain on, the right therapy. These data highlight the gaps in diagnosis and treatment still prevalent in the bleeding disorders community today, and spotlight opportunities for Takeda to better support these patients.”

About Hemophilia

Hemophilia is a challenging chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.⁴ Hemophilia A is more common than hemophilia B; hemophilia A affects about 158,225 people, whereas hemophilia B affects about 31,247 people worldwide.⁵

People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.⁴ Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.^4,5

About von Willebrand disease (VWD)

VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population. VWD is caused by a deficiency or dysfunction of von Willebrand factor (VWF), one of several types of proteins in the blood that are needed to facilitate proper blood clotting.⁶ Due to this defect or deficiency in VWF, blood is not able to clot effectively in people with VWD, which may result in heavy menstrual periods, easy bruising or frequent nose bleeds. Bleeding caused by VWD varies greatly among patients with this disease.⁷

About Takeda Hematology

Takeda is a leader in hemophilia with the longest heritage and a market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years of experience driving innovation for patients and a broad portfolio of 11 products across multiple bleeding disorders. Our experience as leaders in hematology means we are well prepared to meet today’s needs as we pursue future developments in the treatment of bleeding disorders. Together with the hematology community, we are committed to raising expectations for the future, including earlier diagnosis, earlier and better protection against bleeds, and more personalized patient care.

About Takeda Pharmaceutical Company

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries.

For more information, visit https://www.takeda.com.

References

1. Roberts, J. C. et al. (2019). Adding the patient's voice to a hemophilia-specific goal menu to facilitate Goal Attainment Scaling: a qualitative study.
2. Gretchen R. Chiu, MS1 et al. (2019). Incidence and Prevalence of Diagnosed and Undiagnosed Hemophilia A and Hemophilia B in the USA.
3. Roberts, J. C. et al. (2019). Analysis of Bleeding and Treatment Patterns in Children and Adolescents before and after Von Willebrand Disease Diagnosis Using Data from a US Medical Claims Database.
4. World Federation of Hemophilia. “What is hemophilia?” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646. Last Accessed April 2019.
5. World Federation of Hemophilia. Report on the Annual Global Survey 2017. World Federation of Hemophilia website. http://www1.wfh.org/publications/files/pdf-1714.pdf Last Accessed April 2019.
6. National Hemophilia Foundation. "Von Willebrand Disease." National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease. Last accessed January 2020.
7. National Hemophilia Foundation. "VWD Summit Highlights." HEMAWARE website. https://hemaware.org/story/vwd-summit-highights . Last accessed January 2020.
8. Takeda Website. Rare Diseases. Website: https://www.takeda.com/what-we-do/areas-of-focus/rare-diseases/ Last Accessed June 2020.

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