Rare Diseases: Towards a patient-centred EU Blood Directive

For rare disease patients who rely on plasma-derived medicinal products, supply has become the major safety issue
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By Johan Prevot

Johan Prevot is the Executive Director of the International Patient Organisation for Primary Immunodeficiencies

24 Feb 2022

In her statement on World Blood Donors Day 2021, EU Commissioner Stella Kyriakides highlighted the vital role of plasma in the production of life-saving medicines for rare diseases, such as immune deficiencies. She even encouraged EU citizens to become blood and plasma donors.

IPOPI welcomes her support for increasing plasma donation to develop plasma-derived medicinal products (PDMPs) such as immunoglobulin (IG) therapies. Patients living with a primary immunodeficiency (PID) need IG therapies throughout their lives to fight life-impairing or life-threatening infections. No alternative treatments are available for PID patients, nor are they expected in the coming decade.

READ MORE: This article is published as part of our special supplement looking at how Europe can come together to find solutions for people suffering from a rare disease. To read the full supplement click here. 

Plasma can be collected through whole blood donations (‘recovered plasma’, from blood donors) or through plasmapheresis (‘source’ or ‘apheresis’ plasma) from plasma donors. Patients are grateful to all donors for this gift of life. The majority of the world’s plasma to manufacture medicines is collected from plasma donors, who can donate more plasma much more frequently than blood donors, as plasma regenerates in the human body more quickly than blood. Today, unfortunately not enough plasma is collected to meet the needs of EU patients.


To collect more plasma and ensure patients can continue to access their life saving medicines, the EU must revisit its strategies and explore best practices and new ideas

Demand for IG therapies is growing annually at 6-8%, and the EU is short of approximately 30% of the plasma needed to manufacture PDMPs for European patients. The EU relies on plasma imported from the US, the largest plasma collecting country in the world. And the pandemic resulted in a 20% decline in donations compared to 2019 collection levels. For patients who rely on PDMPs, ensuring we collect more plasma is vital.

EU Blood, Tissues and Cells (BTC) legislation is currently undergoing revision. IPOPI, working hand in hand with other patient organisations under the umbrella Plasma Protein Users Group (PLUS), calls for a more patient-centred Blood Directive. To collect more plasma and ensure patients can continue to access their life saving medicines, the EU must revisit its strategies and explore best practices and new ideas.

For patients, supply is the major safety issue. Four countries collect three times more plasma volume than any other European country: Germany, Austria, Hungary, Czechia. Together, they account for about 40% of EU plasma for fractionation as these countries enable both public and private sectors to collect plasma, dramatically increasing plasmapheresis, thanks to dedicated plasma donors.

IPOPI and PLUS call on policymakers to introduce a range of measures including more accurate definitions in the Blood Directive to remove ambiguities and ensure that differences between blood and plasma and their derivatives are clearer. We also need to learn from Member States that collect more plasma, and ensure that legislation does not promote ‘one-size-fits-all’ approaches, but gives countries flexibility to adapt and optimise their collection systems. Overall, the goal should not be achieving European sufficiency, but global sufficiency based on more regionally balanced plasma collection. We also believe the new BTC legislation should contribute to increased supply and free movement of safe and efficacious PDMPs and avoid wastage of plasma and encourage plasmapheresis collection whenever possible.

We are grateful to MEPs who support efforts to collect more plasma in the EU and to the European Commission for involving patients treated with PDMPs meaningfully in discussions on the legislation. We hope our efforts will result in a more patient-centred approach to EU plasma collection. After all, the ultimate goal of this legislation is to ensure patients in the EU can access their life saving treatments.