Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK)
(“Takeda”), today presented three abstracts at the 72nd 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
- 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.
- Gretchen R. Chiu, MS1 et al. (2019). Incidence and Prevalence
of Diagnosed and Undiagnosed Hemophilia A and Hemophilia B in the
USA.
- 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.
- 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.
- 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.
- 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.
- National Hemophilia Foundation. "VWD Summit Highlights."
HEMAWARE website. https://hemaware.org/story/vwd-summit-highights .
Last accessed January 2020.
- 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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Takeda – For Media Outside of Japan David Murdoch
david.murdoch@takeda.com +1 781-421-1741