Plasma-derived medicines are unique

Treatments for patients with rare diseases in Europe

Plasma-derived medicines are unique biologic medicines that treat people with plasma protein deficiencies and dysfunctions. These disorders occur in a very small patient population and belong to a group of rare diseases. In the European Union, a disease is considered rare if it affects 1 individual per 2,000.

The unique value chain for producing plasma-derived medicines

It always starts with a person’s donation of plasma. The process of making specialized plasma-derived medicines takes 7-12 months from the time of plasma donation until the medicine is ready for use by patients. In contrast, it takes one month to produce a small molecule pharmaceutical medicine.

Plasma donation & collection

Plasma-derived medicines are unique. The source material for manufacturing these medicines comes only from human donations. For most of the conditions that plasma-derived medicines treat, patients have no alternative treatment.

Plasma is collected from healthy donors through a process called plasmapheresis which removes only the plasma and returns the remaining blood components to the donor. Therefore, a plasma donation can be made more frequently than a blood donation. Today, PPTA members operate around 160 plasma donation centres in the European Union. After collection, the plasma donation is frozen and shipped to state-of-the-art facilities for manufacture into lifesaving plasma-derived medicines.

Plasma-derived medicines are not interchangeable

Each plasma-derived medicine is unique, due to the pharmacological and manufacturing differences across different brands and to each patient’s unique response to the treatments. As different brands of plasma-derived medicines are not bioequivalent, these therapies are non-interchangeable, sole-source biologics, so it is essential that patients have continued access to their specific therapy. One-size-fits-all policies are not suitable for plasma-derived medicines and endanger patient health.

Council of Europe

“[...] Take into account that human normal immunoglobulin therapeutic products differ from one another in terms of production processes, which might have an impact on specifications
and clinical performance.”

Council of Europe, Resolution CM/Res(2015)2 on principles concerning human normal immunoglobulin therapies for immunodeficiency and other diseases (Adopted by the Committee of Ministers on 15 April 2015 at the 1225th meeting of the Ministers’ Deputies)
Iris – Association des patients déficit immunitaires primitifs, France

“Immunoglobulins are not interchangeable with each other, they are not generic medicines, prescribers must have long-lasting and readily available preparations.”

Immuno-déficience primitive, Recherche, Information, Soutien (I.R.I.S.) Position Statement Summary
IPOPI – International Patient Organization for Primary Immunodeficiencies

“It is important to realize that there is no single immunoglobulin (Ig) product or method of administration that is suitable for all PID patients. [...] All countries and immunodeficiency centres should have access to a wide spectrum of Ig products, to provide optimal treatment for all immunodeficient patients.”

Frontiers in IMMUNOLOGY, Primary Immune Deficiencies – Principles of care; Hypothesis and Theory Article, published: 15 December 2014, doi: 10.3389/fimmu.2014.00627
UK Department of Health

“Although the active ingredient in IVIg — purified immunoglobulin — is the same from brand to brand, there are considerable differences in the manufacturing processes used. This results in individual products that cannot be used interchangeably.”

Clinical Guidelines for Immunoglobulin Use; the UK Department of Health, 2008, p. 15