Baxalta Supports Research to Raise Awareness of Treatment Burden on Patients during the Sixth Annual World Primary Immunodeficiency Week

  • Research by Sheffield University, partnering with leading primary
    immunodeficiency (PI) organizations, aims to characterize the burden
    of treatment for patients with PI, with a focus on mode of treatment
    and factors affecting burden of care
  • Baxalta reaffirms its ongoing commitment to improving the lives of
    people living with PI by delivering innovative treatments and a
    variety of support services

BANNOCKBURN, Ill.–(BUSINESS WIRE)–Baxalta Incorporated (NYSE:BXLT), a global biopharmaceutical leader
dedicated to delivering transformative therapies to patients with orphan
diseases and underserved conditions, today celebrates progress and
reaffirms its commitment to supporting the global primary
immunodeficiency (PI) community during the sixth annual World PI Week,
April 22-29, 2016. As part of the company’s ongoing efforts to deliver
new solutions to help improve the lives of people with PI, the company
is supporting a new research initiative that will assess the major
factors contributing to the burden of treatment for patients managing
these chronic immune disorders.

“Baxalta is committed to making a meaningful difference for patients
with immune disorders,” said Jacopo Leonardi, executive vice president
and president, Immunology, Baxalta. “It is critical that we better
understand the burden of treatment for both patients and caregivers.
This includes the time and effort expended in treating and managing the
disease. We actively support the development and distribution of this
important study, and look forward to how the results will help advance
the well-being of PI patients worldwide.”

The health economic study is being launched through a partnership
between Sheffield University and the International Patient Organisation
for Primary Immunodeficiencies (IPOPI), along with support from the
Immune Deficiency Foundation in the United States. The program aims to
characterize the burden of treatment for PI based on mode of treatment
and factors that affect the burden of care. The first phase of the study
will be conducted among PI patients in 11 countries, including Canada,
Denmark, France, Germany, Italy, the Netherlands, Norway, Poland,
Sweden, the United Kingdom and the United States.

Results from the study will be used to educate and raise awareness among
policy makers, healthcare professionals, healthcare providers and
patients about the burden of treatment for PI, and to identify means to
help reduce treatment burden. Initial results will be reported later
this year.

As part of this study, Sheffield University is designing a PI specific
validated instrument that is intended to be used to assess the burden of
treatment associated with the most common types of immunoglobulin
therapy. This pioneering effort aims to produce a consistent methodology
through which physicians and care providers can assess treatment burden
variations and thus help clinicians identify the right treatment options
that match the personal needs of individual patients.

“We believe that choosing the right treatment for a person living with
PI has a major impact on quality of life,” said Johan Prevot, executive
director, IPOPI. “We recognize the importance of better understanding
treatment burden which includes the time, physical and psychological
effort of those living with PI and their caregivers expend in treating
and managing their condition. Our hope is that this study will shed
light on these issues and provide us with robust data to advocate for
better access to individualized treatments for PI patients.”

World PI Week is led by a partnership of clinical societies, patient
organizations and research foundations from around the world, aiming to
raise awareness and improve the diagnosis and treatment for people with
PI. World PI Week offers an opportunity to inform and educate medical
professionals, researchers, health policy-makers, schools and families,
and the general public about PI. Through events and activities promoting
disease awareness of PI, the global PI community is making positive
changes around the world in support of people living with PI. For more
information, please visit

About Primary Immunodeficiency

Primary immunodeficiencies (PI) are a group of nearly 300 disorders in
which part of the body’s immune system is missing or does not function
properly.1 Normally, the immune system protects the body from
pathogenic microorganisms like bacteria, viruses, and fungi, which can
cause infectious diseases. When any part of a person’s immune system is
absent or dysfunctional, they are susceptible to infections and may take
longer to recover from infections. When a defect in the immune system is
inherited, it is called primary immune deficiency.2 It is
estimated that as many as six million children and adults may be
affected by PI worldwide.3

About Baxalta

Baxalta Incorporated (NYSE: BXLT) is a global biopharmaceutical leader
developing, manufacturing and commercializing therapies for orphan
diseases and underserved conditions in hematology, immunology and
oncology. Driven by passion to make a meaningful impact on patients’
lives, Baxalta’s broad and diverse pipeline includes biologics with
novel mechanisms and advanced technology platforms such as gene therapy.
Launched in 2015 following separation from Baxter International,
Baxalta’s heritage in biopharmaceuticals spans decades. Baxalta’s
therapies are available in more than 100 countries and it has advanced
biological manufacturing operations across 12 facilities, including
state-of-the-art recombinant production and plasma fractionation.
Headquartered in Northern Illinois, with its Global Innovation Center in
Cambridge, Mass., Baxalta employs 17,000 employees worldwide.


  1. Bousfiha A, Jeddane I, Al-Herz W, et al. The 2015 IUIS phenotypic
    classification for primary immunodeficiencies. J Clin Immunol. 2015;
    35(8): 727-738.
  2. Blaese RM, Bonilla FA, Stiehm ER, Younger ME, eds. Patient & Family
    Handbook for Primary Immunodeficiency Diseases. 5th ed. Towson, MD:
    Immune Deficiency Foundation; 2013.
  3. Bousfiha AA, Jeddane L, Ailal F, et al. Primary immunodeficiency
    diseases worldwide: more common than generally thought. J Clin
    Immunol. 2013;33(1):1-7.


Baxalta Media Relations
Kellie Hotz, +1-224-940-2202,
Investor Relations

Mary Kay Ladone, +1-224-940-3371,