Plasma Protein Therapeutics Association Europe, international Association without lucrative purpose
PPTA Europe aisbl
The PPTA represents private manufacturers of plasma protein therapies and plasma collection centers.
Focus Areas
2024-2025
2022-2024
2016-2022
ID: 687559214721-11
Lobbying Activity
Meeting with Olga Solomon (Head of Unit Health and Food Safety) and European Federation of Pharmaceutical Industries and Associations and
3 Oct 2025 · The discussion focused on simplifying Annex II to foster innovation while aligning with ICH guidelines, with ideas for legacy products to gradually transition.
Meeting with András Tivadar Kulja (Member of the European Parliament, Shadow rapporteur for opinion)
17 Sept 2025 · CMA
Response to Critical Medicines Act
4 Jul 2025
Plasma Protein Therapeutics Association (PPTA), representing leading manufacturers of life-saving plasma-derived medicinal products (PDMPs) and companies collecting plasma for fractionation, welcomes the Commission's efforts to address supply chain vulnerabilities to strengthen the availability of critical medicines. To effectively meet this objective, the Critical Medicines Act must account for the vast diversity and complexity of pharmaceutical supply chains. PPTA calls for tailored approaches, especially to plasma-derived medicines (PDMPs), and emphasises the importance of continuous dialogue with the industry to tackle the underlying root causes of availability for plasma-derived medicines. Based on the following facts: 1. Plasma is recognised as a critical substance of human origin in the SoHO Regulation 2. The Critical Medicines Act applies to all medicines from the Union list of critical medicinal products 3. The majority of PDMPs are included in the Union's list of critical medicinal products 4. The production of PDMPs is dependent on the voluntary donation of human plasma, a starting material in limited supply 5. Plasma for fractionation cannot be synthesised in a factory or laboratory 6. PDMPs are not interchangeable due to natural variations in plasma and differences in their production processes 7. The patient need for PDMPs is not seasonal but has been steadily growing 8. PDMPs have a short shelf life 9. Manufacturing of PDMPs cannot be easily scaled up 10. Manufacturing of PDMPs is a lengthy process, taking up to 12 months PPTA recommends that policy makers incorporate the following elements into the Act: A. Recognise the distinctive characteristics of specific medicinal supply chains, especially medicinal products derived from substances of human origin. Differentiation is particularly relevant for Article 2, and Recital 15 to support appropriate, effective implementation. B. Guarantee sustained dialogue with pharmaceutical manufacturers of different classes of medicines included in the Unions list. Given the wide scope of the Act it is essential that the Critical Medicines Group includes and regularly consults with industry representatives of different classes of critical medicines. C. Prioritise patient access over rigid national stockpiling requirements, recognising that medicines, such as PDMPs, have unique characteristics. These include: non-seasonal demand; production dependent on voluntarily donated human plasma in limited availability, short shelf life, lengthy manufacturing timelines that cannot be easily scaled up. While PPTA acknowledges the importance of appropriate inventory/stockpiles, distinct limitations must be accounted for. D. Encourage Member States to establish procurement programmes as well as pricing and reimbursement frameworks tailored to the distinct nature of the PDMP supply chain. Cost containment measures, including price and reimbursement capping should consider exempting PDMPs given their unique characteristics. Countries like Belgium or Romania recognise the uniqueness of plasma-derived medicinal products and have moved to exempt PDMPs from cost containment measures such as clawbacks and paybacks. At the same time, Portugal applies a PDMP-specific tax at 2.5%, significantly lower than all other medicines at 14.3%. In Greece, immunoglobulins and albumins are exempt from the clawback tax. E. Maintain structured dialogue and collaboration with the PDMPs industry as the Critical Medicines Act will be implemented, with an emphasis on prioritising uninterrupted patients' access to these life-saving therapies. PPTA looks forward to collaborating with all stakeholders to strengthen the frameworks of sustainable and resilient supply chains for plasma-derived medicines.
Read full responseMeeting with Patricia Reilly (Cabinet of President Ursula von der Leyen)
22 May 2025 · exchange of views on plasma protein industry
Meeting with Patricia Reilly (Cabinet of President Ursula von der Leyen)
22 May 2025 · to follow
Response to EU Strategy on medical countermeasures
8 May 2025
As the largest association representing manufacturers of life-saving plasma-derived medicines (PDMPs) and private plasma collectors, the Plasma Protein Therapeutics Association (PPTA) is actively involved in the development of all initiatives related to supporting medical countermeasures (MCMs) against public health threats across the EU. Since the strategy will cover MCMs, including substances of human origin and blood products, PPTA calls for tailored approaches to PDMPs. This is due to the unique global supply chain of PDMPs and our industry's role and obligations as a global supplier. PPTA is open to discussing in detail all EU proposals to be put forward in the context of MCMs. PPTA calls for: 1. Clear differentiation between blood products for transfusion and plasma for manufacturing in EU policies. This distinction is fundamental for increasing plasma availability. Plasma, while derived from blood, differs significantly in composition, collection process, and donation frequency. Plasma donation is more complex and time-intensive, with different regulatory needs due to its use in pharmaceutical production. Finally, the manufacturing of PDMPs is extremely complex and lengthy, lasting up to 12 months. 2. Addressing the root cause of challenges of PDMPs shortages: the limited availability of plasma for fractionation. Currently, manufacturing is constrained due to the scarcity of donated human plasma. The EU is reliant on US plasma for fractionation for nearly 40% of its needs. 3. Expanding source plasma collection with high quality standards within the EU. Implementing the SoHO Regulation, Member States should devise clear strategies to ensure plasma supply continuity, with the active involvement of the private plasma collectors. The private sector has demonstrated efficiency, safety and high quality in collecting plasma in the EU despite operating in just 4 countries (AT, CZ, DE, HU). In 2023, 9.3 million litres of plasma for fractionation were collected in the EU50% by private centres in these 4 Member States and the remaining 50% by public sector in 27 countries. 4. Prioritising patients' access to PDMPs over stockpiling and avoiding a one-size-fits-all approach. Differentiation between specific groups of medicines is essential. PPTA calls to avoid national stockpiling of PDMPs and to use EU-level stockpiling as an extraordinary tool, only for some PDMPs and in case of major crisis. Unlike API-based medicines, PDMPs require human plasma for fractionation, which cannot be synthetically produced. Due to the long and complex manufacturing process of PDMPs, limited availability of starting material(voluntarily donated human plasma), short shelf-life, and lack of seasonality in demand, the scaling up of PDMP production upon a sudden increase in stockpiling demand cannot be done immediately. 5. Enhanced pharmaceutical supply chain security: Aligned with the Draghi report, PPTA urges the EU to further bolster PDMP manufacturing as a strategic asset of the EU. European plasma fractionation throughput accounts for 37% of global capacity (MRB, 2018) and adheres to stringent safety, quality and regulatory standards. At the same time, PPTA calls on the EU to recognise the importance of ensuring the economic viability of PDMPs production. Procurement requirements should recognise the uniqueness of the supply chain of critical medicines manufactured from substances of human origin with limited availability, in particular human plasma, and exempt them from national cost-containment measures. 6. Strengthening international partnerships by expanding and implementing international agreements such as the Mutual Recognition Agreement with the US to facilitate access to source plasma and to ensure less burdensome requirements for GMP inspections for third-country plasma fractionation programmes. 7. Harmonization of international regulatory processes to facilitate plasma availability and enable movement of PDMPs within Europe.
Read full responseResponse to Communication on the EU Stockpiling Strategy
8 May 2025
As the largest association representing both manufacturers of life-saving plasma-derived medicines (PDMPs) and private plasma collectors, Plasma Protein Therapeutics Association (PPTA) is actively involved in all initiatives related to solving shortages across the EU. PPTA calls for tailored approaches to PDMPs and continuous dialogue with the industry due to PDMPs unique and global supply chain and our industry's role as a global supplier. The industry is open to discussing in detail all EU proposals to be put forward in the context of stockpiling. Although national and EU-level stockpiling has been considered as a possible solution, stockpiling alone cannot address the challenges of PDMPs' availability due to its complex and unique supply chain. Therefore, PPTA strongly recommends: 1. Avoiding a one-size-fits-all approach regarding stockpiling of medicines. Differentiation between specific groups of medicines is essential. We call on the EU Commission to ensure thoughtfully tailored policies for different medicines supply chains, recognising their unique challenges and root causes of shortages. Unlike API-based medicines, PDMPs require human plasma for fractionation, which cannot be synthetically produced. Due to the long and complex manufacturing process of PDMPs, limited availability of starting material (i.e. voluntarily donated human plasma), short shelf-life, and lack of seasonality in demand - the scaling up of PDMPs production upon a sudden increase in stockpiling demand cannot be done immediately. 2. Prioritising patients' access to PDMPs over stockpiling. Timely patient access to PDMPs is a core priority for the PPTA and its member companies. Stockpiling could be a temporary solution to address a sudden, short-term surge in demand for certain medicines during, for example, outbreaks or pandemics. In the case of PDMPs, their need continues to increase steadily each year, and stockpiling would not address this challenge. Consequently, to ensure long-term solutions, policymakers should focus on continuous dialogue with the industry to address the root causes of challenges, particularly the limited availability of plasma. 3. Coordination and Solidarity: National stockpiling would require a strong European Commission oversight to ensure that EU countries do not excessively stockpile medicines based on unilateral decisions and grow inequities. The EU should also avoid mandating stockpiles of medicines that have the same obligations at the Member State level, as it would risk solely introducing an additional layer of complexity and further disrupting the supply of these medicinal products. 4. Expanding source plasma collection within the EU with high-quality standards: European plasma fractionation throughput accounts for 37% of global capacity (MRB, 2018). Aligned with the Draghi report, which calls for enhanced pharmaceutical supply chain security, PPTA urges the EU to further bolster both plasma collection through plasmapheresis and the PDMP manufacturing as strategic assets of the EU. To ensure the availability of PDMPs, it is necessary to strengthen the entire supply chain, especially through increasing the EU collection of plasma. As the SoHO Regulation moves towards implementation, Member States should develop clear strategies for ensuring plasma supply continuity, with the active involvement of the private sector. The private sector has demonstrated efficiency, safety and high quality in collecting plasma in the EU despite operating in just four countries (AT, CZ, DE, HU). In 2023, 9.3 million litres of plasma for fractionation were collected in the EU50% by private centres in these four Member States and the remaining 50% by public sector across 27 countries. 5. Systemic streamlining of regulatory procedures, as well as greater flexibility for product labelling and redistribution of stocks.
Read full responsePlasma industry warns against one-size-fits-all EU medicine rules
26 Feb 2025
Message — The PPTA requests a tailored regulatory approach that recognizes the unique, non-synthetic nature of plasma-derived medicines. They oppose mandatory national stockpiling, arguing it disrupts regional supply and worsens shortages for patients in other countries. They also call for streamlined reporting systems and expanded private sector plasma collection across the EU.123
Why — Tailored rules would lower production costs and prevent logistical disruptions from mandatory stockpiling.45
Response to Revision of the variation framework for medicines
29 Feb 2024
PPTA welcomes the revision of the Variations Regulation (EC) 1234/2008 and recognises the importance of streamlined and agile provisions for medicines lifecycle management and post-authorisation changes in the EU. The amendments to the Regulation aimed at reducing administrative burden and increasing efficiency are crucial for optimising processes that require disproportionate resources from both the competent authorities and marketing authorisations holders. We believe that an updated variation framework should reflect scientific progress to improve the availability of plasma-derived medicinal products (PDMPs) for patients in the EU. Based on the experience of PPTA member companies in submitting variations to marketing authorisation and Plasma Master File (PMF) dossiers, some of the EC proposed amendments aimed at streamlining and simplifying post-marketing lifecycle management will optimise processes and allow better allocation of resources. In particular, PPTA welcomes the amendment removing the requirement to classify some of the changes related to biological medicinal products as major variations by default and allowing a risk-based approach. However, further changes to the guideline are needed to enable a risk-based approach to classifying changes for plasma-derived therapies. Currently, the guideline requires upgrading Type IA variations to Type IB when certain conditions are not met for biological/immunological medicinal products, including PDMPs. In addition, given that the majority of PDMPs are nationally authorised (MRP/DCP), the current processes for Type IB variations are unjustifiably lengthy, often impacting and prolonging the availability of the final plasma-derived medicinal product through the supply chain. The revision of the Regulation and the upcoming revision of the Variation Classification Guideline will be important to 1) facilitate further streamlining of the variations framework for the PMF changes, reducing the administrative burden and accelerating the notification process, for example, in cases of plasma centre relocation and/or change in center ownership, particularly, for centres already included in the PMF; 2) the current PMF 2nd step procedure represents a purely administrative task on a national level after a centralised evaluation of the PMF by the EMA. This lengthy and resource-intensive process proved to be a major barrier for PMF updates outside the annual update procedure. Changes to the requirement for the 2nd step procedure will be key in improving the life-cycle management for plasma-derived medicinal products without any impact on the product quality, safety or efficacy. PPTA welcomes more frequent updates to the classification guideline. To keep pace with scientific developments and cover cases not captured in the classification guideline, periodic updates based on the latest evidence and MAHs experiences are crucial. Further, the super-grouping of variations is an important and welcomed step towards harmonising dossiers currently registered across different EU Member States, reducing the time required for review and approval of the variation and its implementation, particularly for changes affecting several products. Nevertheless, whilst allowing the submission of Type IA variations as the annual update is encouraging, PPTA believes that it is important to retain the flexibility for immediate submissions, allowing to accommodate the submission requirements in countries outside the EU. Also, the lack of such flexibility could more severely impact the supply in cases where a minor change is upgraded to Type IB. PPTA supports the implementation of the ICH Q12 guideline to facilitate the management of post-approval for CMC changes, thus would welcome further amendments. PPTA and member companies remain committed to working with the EC and relevant stakeholders in ensuring that the new variation framework is fit for purpose, allowing swifter delivery of therapies to patients in the EU.
Read full responseMeeting with Andreas Glück (Member of the European Parliament, Shadow rapporteur)
31 Jan 2024 · SoHOs
Plasma industry urges tailored rules to protect medicine supplies
29 Oct 2023
Message — The group requests maintaining a shorter two-month notification period for medicine shortages. They advocate for streamlining regulatory processes and aligning with international standards.12
Why — Reducing administrative red tape would lower operational costs and accelerate supply chains.3
Impact — Patients in some EU countries could lose access if stockpiling requirements divert supplies.4
Plasma industry group urges regulatory reform for essential medicines
29 Oct 2023
Topic — This consultation evaluates the revision of the EU general pharmaceutical legislation framework.
Message — The group requests a clear definition of plasma for fractionation to facilitate global regulatory alignment. They also advocate for risk-based manufacturing inspections and the removal of redundant administrative steps.12
Why — Streamlined regulations would lower operational costs and improve the industry's global competitiveness.3
Impact — Patients in some regions may lose access if mandatory stockpiling diverts supplies.4
Plasma industry group urges simpler rules to speed supply
26 Sept 2023
Message — The group wants to remove redundant administrative steps for plasma collection. They advocate for reclassifying notifications to a less burdensome category.12
Why — These changes would lower administrative costs and prevent delays in plasma production.3
Impact — National regulators would lose their role in performing secondary administrative reviews of plasma files.4
Meeting with Tiemo Wölken (Member of the European Parliament, Rapporteur)
17 Jul 2023 · Revision of the Pharmaceutical Legislation (staff level)
Meeting with Nicolás González Casares (Member of the European Parliament, Shadow rapporteur)
12 Jul 2023 · SoHO
Meeting with Tiemo Wölken (Member of the European Parliament)
20 Feb 2023 · Verordnung über Qualitäts- und Sicherheitsstandards für zur Verwendung beim Menschen bestimmte Substanzen menschlichen Ursprungs (staff level)
Meeting with Elina Melngaile (Cabinet of Executive Vice-President Valdis Dombrovskis) and Takeda Pharmaceuticals International AG
20 Jan 2023 · EU-US Mutual Recognition Agreement on Pharmaceutical Good Manufacturing Practices
Meeting with Nathalie Colin-Oesterlé (Member of the European Parliament, Rapporteur) and Grifols, S.A.
12 Oct 2022 · SoHO regulation
Meeting with Romana Jerković (Member of the European Parliament)
5 Oct 2022 · Substances of human origin
Response to Revision of the Union legislation on blood, tissues and cells
7 Sept 2022
PPTA feedback on EU Commission proposal for a EU Regulation on standards of quality and safety for substances of human origin (SoHO)
The EU Commission proposal for a SoHO Regulation is an opportunity to help ensure broad and reliable patient access to life-saving plasma-derived medicinal products (PDMPs) and the health and the safety of plasma donors. Legislation can help address challenges posed by the growing clinical need for PDMPs and the EU’s reliance for almost 40% on US plasma.
The safety of plasma donors is paramount for PPTA
PPTA supports measures and standards that help ensure donor safety which are based on scientific evidence. For example PPTA members implement voluntary industry standards for assessing Donor Adverse Events, which provide common definitions to assess and monitor adverse occurrences in plasma collection facilities. Therefore we welcome the proposal requiring SoHO entities to collect donor recruitment and collection data in a centralized EU SoHO Platform.
However, PPTA is concerned that the Regulation states that “donations of plasma imply a significant risk”. This statement has no scientific basis. A recent PPTA study of more than 12 million plasma donations has shown that adverse events related to plasma donations are very rare (around 16 events per 10,000 donations), and 90% are mild in nature. These results are confirmed by many other scientific studies. Overall, the adverse events rate in plasma donors is comparable to that of whole blood donors.
Differentiation between plasma and blood
PPTA welcomes the differentiation of plasma from blood components for transfusion, including with new definitions. Recognizing these differences lays the foundation for policies to support increased availability of plasma in Europe, such as different donor deferral criteria for plasma donors, as highlighted in the text.
The “fixed-rate allowance” concept with regard to donor compensation
PPTA welcomes the clarification that a “fixed rate allowance” is compatible with the principle of “Voluntary Unpaid Donations”. PPTA however believes keeping in the SoHo Regulation the wording of the EU Tissues and Cells Directive which clarifies that “donors may receive compensation, making good the expenses and inconveniences related to the donation” as the inconvenience between different SoHO donations can be significant.
Proactive national plans can help to avoid emergency situations related to SoHO supply
The Commission study supporting the impact assessment highlights that monitoring the availability of PDMPs alone will not solve the EU’s reliance on US plasma. The Regulation should make Member States aware of the importance of developing proactive plans, in addition to emergency plans to support the collection of critical SoHo such as plasma. For example, the Commission recognizes that plasmapheresis is the most efficient method to collect plasma, but currently not all EU Member States have dedicated plasmapheresis programmes in place.
Need for clear collaborative dialogue between industry experts and technical expert bodies.
The new SoHO Coordination Board and the enhanced role of ECDC and EDQM require a collaborative and open dialogue with industry experts. We look forward to sharing our expertise with these bodies in full transparency to ensure that provisions for plasma take into account the substantial differences, compared with transfusable components, including in the areas of donor deferrals, mandatory testing, manufacturing, and storage. EMA’s current stakeholder collaboration model could serve as a good practice example.
PPTA looks forward to a continuous engagement with and calls on EU and national policymakers to ensure that the final SoHO Regulation addresses the mentioned challenges, to preserve the health and safety of donors, increase EU plasma collection, and improve EU’s patient access to PDMPs.
Read full responseMeeting with Karolina Herbout-Borczak (Cabinet of Commissioner Stella Kyriakides), Ralf Kuhne (Cabinet of Commissioner Stella Kyriakides)
17 Mar 2022 · VTC Meeting on Blood, Tissues and Cells legislation and availability of Plasma-Derived Medicinal Products
Response to Evaluation and revision of the general pharmaceutical legislation
26 Apr 2021
The Plasma Protein Therapeutics Association (PPTA) promotes the availability of, and access to, safe and effective plasma-derived medicinal products (PDMPs) which treat a variety of rare, chronic, and potentially life-threatening conditions. 300,000 European patients rely on these essential medicines, without which they might not survive or would have a substantially diminished quality of life.
PPTA supports the European Commission’s revision of the EU pharmaceutical legislation to address the need for equal access to safe, state-of-the-art and affordable medicines to diagnose, treat and prevent diseases, and the need to support and foster innovation in the EU.
The EU Pharmaceutical legislation is key in supporting the EU pharmaceutical industry’s competitiveness, innovation and sustainability. The existing incentives support a favourable R&D environment; therefore, any revision must further allow for and support existing measures. Any unfavourable changes to existing incentives may impact Europe’s attractiveness for innovation and hamper existing efforts in promoting R&D and technological advancements, which could delay or curtail development of medicines, especially those for rare diseases and/or paediatric indications, such as PDMPs.
A key component to meet the clinical needs of EU patients who rely on PDMPs is assuring sufficient plasma collection in Europe. Human donated plasma (plasma for manufacturing) serves as the essential biological starting material for PDMP production. The current plasma volumes collected in the EU are insufficient; they only fulfil around 62% of the clinical need for PDMPs, 38% of plasma is imported from the United States (U.S). In addition, the clinical need for PDMPs will increase over the next decades due to better diagnosis, treatments and new indications Hence, PDMPs should be specifically considered when addressing uncertainty and any steps regarding supply continuity and availability of existing products and products in development. This should be streamlined with other activities, such as the ongoing revision of the BTC legislation, and through multi-stakeholder engagement and multidisciplinary input and dialogue to better understand and address the multifactorial and often differing elements of ‘unmet needs’ and access inequality to PDMPs. The identification of root causes of supply constraints is crucial, for instance, through outcomes of the ongoing “Structured Dialogue”.
Global context is key when addressing revisions needed to current legislation: Since plasma is collected and manufactured globally, concrete steps are needed to streamline and harmonise regulatory and pharmaceutical quality standards and requirements for plasma and PDMPs, including the addition of plasma for manufacturing and PDMPs to the mutual recognition agreement (MRA) on GMP inspections between the U.S and the EU and need for an extensive MRA for GMP conformity with the UK.
PPTA welcomes the proposal to simplify legislation and create regulatory attractiveness: This revision presents an opportunity to refine and accelerate regulatory standard activities, based on ‘lessons learned’ from the COVID-19 pandemic, and implementing these as part of post-pandemic preparedness and standard practice. Reduction of unnecessary administrative burden is paramount (removal of 2nd step EMA Plasma Master File (PMF) approval, modifications to PMF requirements for new and existing plasma centres, modifications to GMP inspections). Focus should further be put on improving interaction between regulators and industry, shortening of time between scientific advice, clinical trials and marketing authorization application/submission, including acceptance of new methods of evidence generation and assessment as well as streamlining of regulatory and market access related activities, all of which would further improve timely access to medicines for patients.
Read full responseMeeting with Margaritis Schinas (Vice-President) and
8 Apr 2021 · Health Union
Response to Revision of the EU legislation on medicines for children and rare diseases
6 Jan 2021
The Plasma Protein Therapeutics Association (PPTA) promotes the availability of, and access to, safe and effective plasma-derived medicinal products (PDMPs) which treat a variety of rare, chronic, and potentially life-threatening conditions. 300,000 European patients rely on these essential medicines, without which they might not survive or would have a substantially diminished quality of life.
PPTA supports the European Commission’s (EC) goal to equal access to safe, state-of-the-art and affordable medicines to diagnose, treat and prevent diseases. We commend the EC’s recognition of the importance of the EU pharmaceutical sector in bringing in innovation into the EU, which has been acknowledged in the ‘Pharmaceutical strategy for Europe’.
PPTA is of the opinion that the orphan and paediatric regulation is a key component of supporting the pharmaceutical industry’s competitiveness, innovation and sustainability in Europe. Since its introduction, the regulation, and in particular the incentives, created a favourable R&D environment, which is essential for making progress in preventing and treating diseases. Hence the revised legislation must further allow for and support any existing measures.
We believe that unfavourable changes to existing incentives, such as abolishment of the 10-year market exclusivity period may impact Europe’s attractiveness for innovation and hamper existing efforts in promoting R&D and technological advancements in Europe. Therefore, the new incentives should be an add-on to existing exclusivity measures.
The current criterion for orphan disease designation (ODD) should be retained. Reducing the existing prevalence criterion or changing it with the incidence criterion could delay or curtail the development of innovative medicines in many existing rare diseases. However, a revision should further clarify the ODD significant benefit criteria, especially based on an assumption of a major contribution to patient care.
PPTA would encourage additional investigation, including additional stakeholder engagement and multidisciplinary input, to better understand the elements of ‘unmet needs’ as well as access inequality across the EU. These will not be addressed through changing incentives as they are multifactorial and variable across the different EU Member States (differences in national pricing and reimbursement, national access timelines, national regulatory and HTA/payers requirements). Cooperation and dialogue among EU Member States, a multi-stakeholder engagement and holistic approach in the development, authorisation and access to medicines and improved availability and affordability should thus be incorporated into any future legislative proposal.
The proposal should consider improving and streamlining regulatory and HTA procedures, in the context of new methods of evidence generation and assessment, such as choice of biomarkers, analysis of big and real-world data to support the development. Plasma-derived products and innovative medicines such as gene therapies and other advanced therapy medicinal products (ATMPs) require different regulatory approaches and therefore, the proposal should also address the concept of similarity of medicinal products to ensure that similar medicinal products with significant benefit to patients are not hampered.
Finally, the proposal should consider that a key component to meet the clinical needs of EU rare disease patients who rely on plasma-derived therapies is assuring sufficient plasma collection in Europe. The current plasma volumes collected in the EU are insufficient; they only fulfil around 62% of the clinical need for PDMPs to treat rare diseases, 38% of plasma is imported from the United States.
Read full responseResponse to Revision of the Union legislation on blood, tissues and cells
14 Dec 2020
The Plasma Protein Therapeutics Association (PPTA) is steadfast in its mission to promote the availability of, and access to, safe and effective plasma-derived medicinal products (PDMPs). 300,000 European patients rely on essential PDMPs to treat a variety of rare, chronic, and potentially life-threatening conditions. Without these treatments, many patients might not survive or would have a substantially diminished quality of life.
PPTA supports the European Commission’s goal to build a stronger European Health Union. We therefore call for “more Europe” to meet the growing need for plasma as a critical resource for securing patient access to PDMPs. The current plasma collection landscape is acknowledged throughout the Evaluation of the EU blood, tissues, cells (BTC) legislation and the Inception Impact Assessment (IIA) as “inadequate in protecting EU patients” to ensure access to PDMPs. The COVID-19 pandemic has exacerbated the existing situation and emphasized the need to take prompt action.
Plasma volumes currently collected in Europe fulfil only around 62% of the clinical need. 38% of plasma is imported from the United States. To decrease dependency from third countries, the EU has an important role to play in supporting Member States in their efforts to collect more plasma through legislative or non-legislative means and by increasing regulatory flexibility. However, PPTA is concerned that the proposed policy options do not target a solution to increase plasma collection. We believe that a more ambitious objective around increasing the collection of plasma can be achieved while maintaining a high level of donor safety. The industry has been leading on for decades.
It has been proven that incentives are effective in supporting a greater level of plasma sufficiency and the revised legislation must allow and support these measures. For example, monetary compensation has been successful in four European countries whose collections account for more than 55% of source plasma collected in Europe. In these countries, four times more plasma per capita is collected. Compensating donors can be considered as a voluntary unpaid donation (VUD) and is referenced in the EU Tissue and Cells Directive. This is also reflected in the 2016 EC Report on the VUD Principle, and recognized by the Council of Europe Bioethics Committee in 2018. In addition, Member States make use of different types of incentives and compensation such as tax benefits.
To optimize plasma collections in Europe, the revision should also consider more regulatory flexibility. Especially in developing an efficient framework for plasma center inspection procedures by expanding the concept of Mutual Recognition Agreements with the US to the plasma collection sector, in addition to re-assessing donor eligibility requirements, and increasing donation frequency based on science and current practices.
Whilst we welcome the focus on policy options to keep technical requirements up to date, the knowledge and expertise of plasma collectors and fractionators must be taken into account. Quality and safety requirements in the plasma collection process, whether developed by the European Commission, EU agencies or third party experts, should be established in collaboration with competent experts and in a robust, transparent and inclusive stakeholder consultation framework with clear processes.
A revised Blood Directive should address the differences between collecting whole blood for transfusion and plasma collection for manufacturing PDMPs. The revision should at least include definitions of plasma for fractionation, the Voluntary Unpaid Donation concept, and donor compensation. PPTA members play an important role today in the collection of plasma alongside national services. The revision should explore all options to increase plasma collection, including promoting co-existence between these actors by ensuring all parties are at liberty to provide public health services.
Read full responseResponse to Pharmaceutical Strategy - Timely patient access to affordable medicines
6 Jul 2020
The Plasma Protein Therapeutics Association (PPTA) welcomes the Commission’s initiative to help ensure EU’s supply of safe and affordable medicines to meet patients’ needs and reinforce EU’s pharmaceutical industry competitiveness. PPTA is the global industry trade association with a European presence representing the private sector manufacturers of plasma-derived medicinal products (PDMPs), and privately-owned plasma donation centers.
PDMPs, derived from human plasma from healthy donors, are essential for around 300,000 European patients who rely on these therapies to treat a variety of rare, chronic, and potentially life-threatening conditions, which are often genetic in origin. Without these treatments, many patients would either not be able to survive or would have a substantially diminished quality of life.
However, the PDMP Ecosystem is in a fragile balance as it depends on often uncertain plasma donation volumes, complex regulations, strict safety procedures, a lengthy and costly manufacturing process. European policies must therefore recognize the unique nature of PDMPs and their ecosystem if we are to cope with the growing clinical need for PDMPs in Europe, of Europe’s reliance on plasma imports, and of ensuring greater access to these products throughout Europe.
The COVID-19 pandemic further highlighted this fragile balance and the need to prepare for future pandemics during which plasma collection must be maintained and the therapeutic potential of convalescent plasma should be explored.
We need to work together to address current restrictive plasma donation policies in Europe and to establish a stronger plasma collection infrastructure.
PPTA would like to address the following issues raised in the Roadmap:
1. Addressing the EU’s reliance on imports of medicines and starting materials
Despite the plasma sector’s strong European manufacturing footprint, there is a growing reliance on plasma from the US as a starting material for PDMPs, as recognised by the Commission’s Report on the Evaluation of EU’s legislation on blood, tissues and cells .
Today only four countries (Germany, Austria, Czechia, Hungary) allow privately-owned plasma collection centers to contribute to the collection of source plasma in the EU, while 37% of the plasma is imported from the US.
Europe therefore needs to collect more plasma to increase its contribution to the global supply. This would strengthen the resilience of national healthcare systems to also meet the growing clinical demand of PDMPs and support the EU’s future pandemic preparedness.
In PPTA’s view, European policies must therefore facilitate the collection of more plasma in the EU.
2. Ensuring greater access and availability of pharmaceuticals to patients
In some EU countries, PDMPs are either not reimbursed at all, or only for narrowly defined eligible patient populations. This results in great inequalities for patients across the EU whilst it has been shown that the socio-economic benefit of treating patients with PDMPs is significant .
PDMPs often face additional economic challenges from inappropriate cost-containment measures such as clawback or payback taxes. However, several countries have recognised the unique nature and value of PDMPs and have lifted, deferred or reduced such taxes for PDMPs .
In PPTA’s view, this approach can strengthen the resilience of healthcare systems in the EU and ultimately ensure increased patient access to PDMPs.
3. Enabling innovation to address unmet medical needs and respond to public health threats
In their timely response to COVID-19, regulators across the EU have introduced ‘regulatory flexibility’ and measures to improve the existing regulatory framework, which led to some reduction in regulatory burden without compromising safety.
To further support patient access to medicines, it is key to maintain and build on this towards more regulatory efficiency and enhanced dialogue between industry and regulators in the EU.
Read full responseMeeting with Vytenis Andriukaitis (Commissioner) and
15 Jun 2018 · HTA
8 Jan 2018
PPTA, the Plasma Protein Therapeutics Association, welcomes the opportunity to comment on the European Commission Roadmap regarding the Evaluation of the legislation on medicines for children and rare diseases which was published on 11 December 2017.
The Plasma Protein Therapeutics Association (PPTA) is the standard setting and global advocacy organization with a strong European presence representing private manufacturers of plasma-derived and recombinant analogue therapies, collectively known as plasma protein therapeutics (PPTs). PPTs are unique, biologic medicines to treat a variety of rare, life-threatening, chronic, genetic diseases, such as haemophilia or primary immunedeficiencies. Some of these PPT’s do have an orphan drug status according to the EU Regulation on Orphan Medicinal Products (OMP), other PPTs for the treatment of rare diseases have been developed before the OMP Regulation came into force.
In PPTA’s view, the planned Evaluation of the two legislations is a useful exercise but should duly consider the specifics of each legislation. Having experienced also other EU Commission Evaluations, we believe that such a complex exercise should foresee enough time for stakeholders to respond. Given that the Commission plans to conduct the Evaluation also during Q3 2018 which comprises the months of July and August, an adequate extension of the Evaluation period would be advisable to leave enough time to respond . We refer in this context to the 2017 EU Commission Evaluation of the Tissues and Cells and the Blood Directives which ran also over July and August, and which was extended.
PPTA is looking forward to actively participate in the forthcoming Commission Evaluation.
Read full responseResponse to REACH Regulation - Annex XIV
13 Oct 2016
The Plasma Protein Therapeutics Association (PPTA) is the international trade and standards-setting organization representing private plasmapheresis centers and manufacturers of plasma protein therapies (PPTs). PPTA represents global companies Biotest, BPL, CSL Behring, Grifols, Kedrion and Shire, which make 60+% of PPTs for the world. PPTA comments on the planned restriction of 2-[4-(2,4,4-trimethylpentan-2-yl)phenoxy]ethanol (Triton-X-100) and requests exemption of Triton-X-100 for manufacturing of PPTs for the following reasons: PPTs are unique: PPTs are produced from human plasma or recombinant mammalian cell lines. They are used to treat rare, life-threatening, chronic and genetic coagulation diseases, including haemophilia, Factors VIII/IX/XIII, von Willebrand Factor, Protein C, Antithrombin III, and alpha-1-antitrypsin deficiencies; primary immune deficiency, neurological, autoimmune disorders and other acute, severe conditions. Many products are Orphan Drugs. The diseases are often under-diagnosed and globally many patients do not have access to therapies. Triton-X 100 cannot be easily substituted: Effective virus inactivation/removal steps are required to ensure that PPTs are safe and viruses such as HIV, Hepatitis A/B/C, Parvovirus B19 are eliminated (EMA/CHMP/BWP/706271/2010, WHO TR series 924/2004). Triton-X-100 offers: 1. effective and fast virus reduction capacity: In conjunction with a solvent for Solvent/Detergent (S/D) treatment it is highly effective against all lipid-enveloped viruses [HIV, Hepatitis B/C viruses; West Nile virus, Yellow Fever and Japanese Encephalitis virus (Remington et al 2004; Kreil et al 2003)] as well as Zika virus (EMA/CHMP/BWP/596747/2016). S/D treatment is the ‘Gold Standard’ for inactivation of lipid-enveloped viruses (Bertolini 2013). While transmission of viruses by some PPTs occurred before introduction of S/D treatment, there was no reported transmission of HIV, Hepatitis B/C virus by S/D treated PPT with over 35 million doses administered (Horowitz B 1998); 2. non-toxicity: Traces in the final product cannot be fully eliminated; 3. biological PPT integrity and yield-some PPTs are sensitive to degradation; 4. uniform applicability: Triton-X-100 alternatives are restricted to purification of monoclonal antibodies-suitability is yet to be confirmed for more complex proteins (i.e. FVIII, vWF). At present there is no evidence that these alternatives are suitable for all therapeutic PPTs. Impact on manufacturing industry and PPT supply: Efficacy and safety of PPTs are intrinsically linked to the manufacturing process. Thus, the use of substitutes require repeating process development and clinical studies that are most time-consuming and costly in drug development. There is a serious risk of significant supply disruptions of PPTs to patients, given the proposed short transition time. Where PPTs are only used by a few patients, the burden of re-development may force some manufacturers to cease production. Amounts of Triton-X-100 used are small: 1kg to 10kg of Triton-X-100 per batch of PPT (S/D treatment with 1% Triton-X-100 ranges from 100L to 1000L). Used Triton-X-100 is discarded in liquid waste; residual Triton-X-100 enters the environment after extensive treatment to minute residual levels. Some manufacturers already implemented specific Triton-X-100 waste treatment and if not, it can easily be done to mitigate any residual environmental impact. PPTA considers that the overall burden, potential unwarranted consequences on valuable pharmaceutical processes and detrimental impact on the global supply of safe, affordable PPTs to patients due to Triton-X-100 restriction disproportionately outweigh the limited benefits. We urge the EU Commission to allow an exemption of Triton-X-100 for manufacturing of PPTs and conduct an appropriate impact assessment of the potential safety issues, supply disruptions and access for patients to essential medicines manufactured using Triton-X-100.
Read full response