In the coming decade, it is expected that more European patients will need access to plasma-derived medicines.
Every year, more patients across the EU are diagnosed with life-threatening plasma protein-related disorders, such as immune deficiencies, immune-mediated peripheral neuropathies, hereditary angioedema, alpha-1 antitrypsin deficiencies, haemophilia, and other bleeding disorders.
In many cases, plasma-derived medicines are the only option to treat these rare diseases. New indications, improved diagnostic techniques, greater use in Europe, and increased use in in cancer treatment-induced secondary immunodeficiency contribute to a growing clinical need.
Over the past 10 years, the use of immunoglobulins – some of the most used plasma-derived medicines – has almost doubled. With a yearly increase of about 8%, immunoglobulin usage increased from 32 tonnes in 2009 to 60 tonnes in 2019 (Marketing Research Bureau, 2019). This increase is driven by: innovations in medical research; improved and early diagnosis; extended patients’ lifespans due to timely treatments; and efforts to increase immunoglobulin usage in the Eastern EU countries, which is currently behind usage in the Western countries.
Today, almost 40% (5.15 million litres) of the plasma used to manufacture plasma-derived medicines for EU patients comes from the U.S. (Marketing Research Bureau, 2021).
European patients in need of plasma protein therapies (estimates)*
Primary Immunodeficiency Diseases (PIDs)
- Caused by missing or malfunctioning immunoglobulins (antibodies)
- Antibodies controle the immune system and prevent illness
- Patients are chronically ill from severe, persistent, recurrent infections
- Caused by missing or malfunctioning clotting factor protein
- Clotting factors control bleeding
- Patients cannot regulate bleeding (joint damage is common)
- can be fatal if bleeding occurs in brain or vital organs
Alpha-1 Antitrypsin Deficiency
- Caused by missing or malfunctioning Alpha-1 Proteinase inhibitor
- Alpha-1 Proteinase inhibitor protects the lungs
- Patients have chronic emphysema and live damage
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Cause not certain; immune system attacks nerve coating
- Messages from the brain aren't delivered to the body if nerve coating is damaged
- Patients experience progressive weakness; loss of limb function, and disability
- Caused by missing or malfunctioning C1 esterase inhibitor protein (C1-INH)
- C1-INH helps control inflammation
- Patients have oedema (severe swelling)
- Can be fatal if airway obstructed
These therapies are unique, biologic medicines derived from human plasma. They treat millions of patients worldwide who live with a variety of rare, life-threatening, chronic, genetic diseases, such as primary immunodeficiencies and haemophilia, and are used for medical emergencies, such as shock and burns.
The Plasma Protein Therapeutics Association (PPTA) is the global industry trade association representing the private sector manufacturers of plasma-derived and recombinant analogue therapies, collectively known as plasma protein therapies; and the collectors of source plasma used for fractionation to make plasma-derived medicines. PPTA also administers standards and programmes that help ensure the quality and safety of plasma protein therapies, donors, and patients.