There is a rapidly growing “unmet need” for more plasma donations for people suffering from debilitating and possibly life-threatening conditions who rely on plasma-derived medicines (PDMPs).
Only a “good supply” of plasma can offer such people a healthy, normal life.
This was one of the keynote messages to emerge from a high level debate on plasma donation and current EU legislation on the issue in the European Parliament on Monday.
Plasma, which makes up 55% of our blood and carries water, salt and proteins, around the body, is used in the treatment of patients with serious burns, blood disorders and compromised immune systems.
Attendees at the event, online and in person, were told that, every year, 15 million people in Europe donate their blood, while there are 4.6 million patients who receive blood transfusions.
But, in the EU, there is currently an estimated shortfall of over 5 million litres of plasma needed to manufacture plasma-derived medicines .
Donors, in plasma donation centres, undergo plasmapheresis, a process, that removes plasma from the donor’s blood and returns the remaining cellular components to them. Plasma is an essential raw material for a number of drugs used in the treatment of diseases such as haemophilia, as well as severe immuno-deficiency.
Approximately 300,000 people with a chronic condition across the EU depend on plasma-derived medicines for which there is often no alternative treatment available. In addition, PDMPs are used as well for treating other conditions such as liver diseases, pregnant women suffering from serological conflict, as support therapy for patient suffering from secondary immune deficiency due to cancer treatment, in emergency and critical-care situations.
At the event, it was underscored that certain medical interventions are only possible thanks to donations or transplants of substances of human origin (SoHO), such as plasma, skin, embryo, blood, sperm, microbiota or corneas.
EU regulation
The two hour debate was timely, as EU policymakers are currently debating a proposal for a Regulation on Substances of Human Origin (SoHO).
The proposal, the audience heard, has posed several key questions, including: how can the Regulation become a framework that would encourage Member States to collect more plasma, benefit donors and improve access to life-saving plasma-derived therapies?
The event, called “Substances of Human Origin Regulation: a step forward or backwards for patient access to plasma-derived medicines?”, was divided into three main, key themes: patient access to plasma-derived medicines (PDMPs), the safety of plasma donation and the ethical principles of compensating donors in the context of the SoHO Regulation.
Listeners heard that the Regulation may be an opportunity to meet patients’ needs across the EU for blood, plasma, tissues and cell products, without compromising donor safety.
Key industry experts and decision-makers shared their insights and, in the opening session, Giles Platford, Chair of the PPTA Global Executive Board and President of the Plasma-Derived Therapies Business Unit at Takeda, said his “key message” to policymakers was to emphasise the need for a final legislative text that “promotes collaboration between the private and public sectors, along with stakeholders” in order “to find a solution to increase plasma collection in the EU and decrease the reliance on the US, which is around 40 percent”.
He stressed how many patients depend on plasma-derived treatments, adding that, in Europe, millions of plasma donations are required per year to help sustain patient treatment, one reason why plasma is vital and why plasma donation is so crucial.
He said, “We need to work to get together to support a reliable supply of plasma and we are calling on EU member states to collect more plasma because there is an unmet patient need and this is increasing all the time.”
He reminded the audience that some 44 percent of all plasma needed for treatments in Europe comes from just 4 member states, saying “this is why we need to ensure that the EU has legislation that guarantees collaboration and regular supply”.
He said, “It is important the EU is reviewing the current legislation and we welcome this as an industry, but there are areas where we see an opportunity for more improvement, for example, in the area of compensation.
“Plasma donors should be compensated for their expenses and inconveniences related to the donation.
“We also need more recognition that donation is safe. It is indeed safe, but the current text implies there is significant risk and this is not the case.”
He said that “greater assurance of the reliability of plasma supply is also critical to patients”. “Eventually Member States decide how to implement an efficient plasma collection system, and we remain available to support this”.
Patient voice needed
Another speaker, Martine Pergent, President of the International Patient Organisation for Primary Immunodeficiencies, said, “Patients must be given their voice on all this because they need treatment and if they lack it they are in big trouble and may even die. If properly treated they can lead a normal life and have a family. This is invaluable.”
She was asked how the EU draft text might help boost plasma collection.
She said, “At the moment, the real risk is about supply, so the value of this Regulation is that it gives really important momentum to this issue.”
One of the biggest misconceptions about plasma donation was that many are not aware of the good it can do.
“This is because donating plasma is not as common as blood donation and this needs to be highlighted. I know of many people who are keen to donate and this is about saving lives, because without treatment, people could die.”
A second session considered safety issues and whether plasma donation is safe.
Svenja Tatjana Barckhausen, Executive Board Member at Haema AG, pointed out that there are differences between blood and plasma donation, for example in the number of times per year one can donate.
Donations for blood do not take as long as for plasma donation and not every blood donor will necessarily be a plasma donor.
“It is not the same donor base.”
She said, “The public perception of blood donation is far more common than for plasma donation, but this is not because one is no more safe than the other. Plasma donation is safe and I don’t understand why plasma donation is considered high risk by some.”
On the compensation issue, a thorny issue for MEPs, she said the “motivation of the donor” was the primary concern, “not how much they are compensated”.
She added, “The biggest motivation is to know someone who has a disease that can be treated with PDMPs. We find that people are also motivated to do something good and be proud of that and not by how much they are compensated. It is breath-taking how they are often touched by helping someone.”
Her message to policymakers is: “You have to differentiate between blood and plasma and compensation or non-compensation does not affect plasma quality. Compensation should be just for expenses and inconveniences.”
Also speaking, Dr. Behrouz Mansouri, Medical Director at PlasmaVita Magdeburg, said, “People should donate plasma because plasma-derived medicines are essential for people who may be severely ill and in a life-threatening condition. That is why we are asking for people to come forward for donation.”
Asked about the safety of plasma donation, he said, “I am convinced there is no significant risk to donors. Plasma donation is based on a very well-established procedure and, also, the frequency of donation is not associated with safety risks. This has been shown by several studies.”
His key message was, “There is no evidence for classifying plasma donation as unsafe. It is not dangerous.”
Ethical and philosophical considerations
A third and final session looked at the ethical implications of the EU Regulation and compensation.
One key question is: could compensation be seen as exploiting people for their plasma and is it harder to get someone to donate without offering compensation?
Begoña Roman Maestre, Professor of Ethics and Bioethics at the University of Barcelona, addressed the difference between compensation and incentivisation. She said, “Compensation constitutes a gesture of gratitude for time and inconvenience, whereas for incentivization the main goal is reward. Nevertheless, compensation must not be the main reason to donate, that must be done out of a sense of duty.
“There is an argument that compensation can be fairer if it is the same for everyone, but monetary compensation is an equaliser and we should trust the donor when it comes to the way compensation best suits them.” She also called for a system of reasonable and proportionate compensation that would fit everybody. “Students would for example not be eligible for compensation in the form of days-off-work or tax advantages”.
Asked about any changes to the EU draft legislation, particularly regarding compensation and quantifiable losses, she said, “the word ‘losses’ sounds too negative, as if something truly has been lost’. In the end, the patient and society wins (from plasma donation).”
Over the past 20 years, growth in plasma collection in the EU was achieved largely by the four countries (Germany, Austria, the Czech Republic and Hungary) that allow both the public and private sectors to collect plasma and centre-right MEP Stelios Kympouropoulos used the event to call for greater access to medicines, including PDMPs, and for all involved to raise awareness about plasma donation.
He said, “This debate is timely and this is a crucial issue. In the legislation, we have got to find the right balance and recognise the value of donations and of donors who give their time and effort. We also must not continue to simply import plasma from other countries outside the EU, but create conditions to collect more from here, in the EU. This means creating more incentives in the EU, so that we are independent of third countries for donations. We also need to strengthen the role of EMA and achieve better coordination with the other EU agencies.”
The event heard that lessons should be drawn from the COVID-19 pandemic, which saw unprecedented levels of public-private partnerships to tackle a priority global health issue.
There is also a purposeful goal to reduce Europe’s reliance on imported plasma to produce these life-saving medicines - just under half of the plasma currently used in Europe comes from abroad – in particular from the United States.
But the overarching message to emerge from the event was a hope that whatever finally comes out of the legislative loop will benefit patients and address the issues addressed by the speakers.
The event was organised by The Brussels Times with the support of the Plasma Protein Therapeutics Association (PPTA), the trade association representing the manufacturers of plasma-derived medicinal products (PDMPs) and the private sector plasma collectors, which includes more than 160 donation centres in Europe.
