Baxalta Supports Research To Raise Awareness Of Treatment Burden On Patients During The Sixth Annual World Primary Immunodeficiency Week

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.

Back to news