Worldwide Primary Immunodeficiency Statistics: Patients, Trends, Market

Worldwide Primary Immunodeficiency Statistics: Patients, Trends, Market

Primary immunodeficiencies (PI) are defects of the immune system that cause severe, sometimes life threatening, infections if not diagnosed and treated appropriately. Many patients with PI are undiagnosed, underdiagnosed, or misdiagnosed. There are at least 300 genetically defined single-gene inborn errors of immunity. Recent studies have shown that PI may be more common than previously estimated and that as many as 1–2% of the population may be affected with a PI when all types and varieties are considered. Over the last decade, improvements in molecular diagnosis, genetic sequencing, and cutting edge research and treatments have led to a better understanding of the immune system, as well as improved quality of life for those living with PI. However, awareness of PI among physicians and the general public remains challenging, and there continues to be a need for improved and timely management of these conditions.

The global primary immunodeficiency diseases market by disease type (Antibody deficiency – Agammaglobulinaemia, Common variable immune deficiency, IgG subclass deficiency, SIgAD; Cellular immunodeficiency – Ataxia Telangiectasia, DiGeorge syndrome, Hyper IgM syndromes, Wiskott-Aldrich syndrome; innate immune disorders – Complement deficiencies, and Hyper IgE syndrome), test type (blood and prenatal testing), treatment modality (immunoglobulin replacement therapy, antibiotics therapy, stem cell and gene therapy, etc.), and by geography (regional and country based) into North America (U.S., Canada), Latin America (Brazil, Mexico, Rest of LA), Europe (U.K., Germany, France, Italy, Spain, Rest of EU), Asia Pacific (Japan, China, India, Rest of APAC), and Rest of the World. The global primary immunodeficiency diseases market report also provides the detailed market landscape, market drivers, restraints, opportunities), market attractiveness analysis and profiles of major competitors in the global market (Baxter International, Inc., Bio Products Laboratory, Biotest AG, CSL Behring LLC, Grifols S.A., Kedrion S.p.A., LFB S.A., and Octapharma AG.) including company overview, financial snapshot, key products, technologies and services offered, and recent developments.

Global Patient Surveillance 2015

In 2015, the distribution of patients diagnosed with PI based on the categories defined by the IUIS Expert Committee for the Classification of PI, predominantly Antibody Deficiencies are reported by physicians to be 63.4% in the US, 47.7% internationally, and 53% globally. Well-defined Syndromes with Immunodeficiency was 16. % in the US, 11.2% internationally, and 12.9% globally. As a percentage of all patients identified with a specific defect, Antibody Deficiencies account for 57 % of all patients identified with a specific defect.

Based on the 15 most prevalent PI defects and percentage of each defect by region, Selective IgA deficiency is the most prevalent with 16.4% in the US, 13.0% internationally, and 14.1% globally. Common variable immunodeficiency (CVID) showed a prevalence of 15.4% in the US, 11.2% internationally, and 12.6% globally. It is noteworthy that Canada and Australia had 25.5 and 39.5% prevalence for CVID, respectively. The Middle East reported 30.5% Familial Mediterranean fever compared to 3.3% globally. Africa reports 6.5% Ataxia telangiectasia (A-T) compared to 2.6% globally, and 8.7% other combined immunodeficiencies compared to 1.5% globally.

With respect to gender and age, male patients accounted for 58.3% globally, while female patients accounted for 41.7%. In the US, 55.7% of the patients were male and 44.3% of patients were female. Globally, 63 % of the patients were 19 years of age or younger, while 37% were 20 years of age or older.

Treatment Modalities 2015

There was a 19% overall increase in patients receiving IgG. There was an increase of 7% in all patients receiving intravenous immunoglobulin therapy (IVIG) and a 100% increase in patients receiving subcutaneous immunoglobulin (SCIG). Of all patients diagnosed with a PI defect, 24.3% are on immunoglobulin replacement therapy. As to all patients with an Antibody Deficiency, 42.9% are treated with IVIG/SCIG. It is noteworthy that there was a 27% increase in the number of patients receiving IVIG in the hospital or clinic. There was a decrease of 49% in the number of patients receiving IVIG at home. This decrease was more than offset by an increase of nearly 100% in patients receiving SCIG. Latin America reported 87% of their patients receiving IgG are treated in the clinic or hospital compared to 55 % globally. In the US, 30% of patients receiving IgG are treated at home compared to 14% globally. Western Europe reported 46% of their patients requiring IgG receive SCIG compared to 28 % globally.

There were commensurate increases in patients receiving other treatments for PI as well. The number of patients treated with PEG-ADA increased by 44%. There was a 22% overall increase in patients treated by HSCT or thymus transplantation, with the number of patients receiving matched donor transplants, matched unrelated donor transplants, mismatched unrelated donor transplants, and parental haplo transplants increasing by 28.6, 17.6, 5.5, and 15.6%, respectively. Use of bone marrow as the source of stem cells increased by 17.4%, while cord blood as the stem cell source increased by 66%. While there was a 22% increase in the number of patients having received HSCT, specific stem cell sources varied significantly by region and source. Latin America reported 74% of the transplants were bone marrow, as opposed to 61% globally. Middle East reported 35% of the transplants were peripheral stem cells as opposed to 22% globally. There was a 65% overall increase in cord blood transplants in the past 2 years. Asia reported 42% of the transplants were cord blood, compared to 16% globally.

Salient Features of the report (Primary Immunodeficiency Diseases Market):

1. Botttom-up research methodology (assumptions) for market size estimation and forecast
2. Global Number of patient population for PID (subtypes) by country, 2013 & 2015
3. Global Number of patient population for treatment or therapy by country, 2013 & 2015
4. Global Number of PID Patients, by 15 Most Commonly Identified PID, 2013 & 2015
5. PID Patient’s Gender and Age – Global Comparative Analysis, 2013 & 2015

2017-01-02T11:57:02+00:00 Categories: Analyst Insights|