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International Patient Organisation for Primary Immunodeficiencies

IPOPI

IPOPI is the Association of national patient organisations dedicated to improving awareness, access to early diagnosis and optimal treatments for primary immunodeficiency patients worldwide through global collaboration.

Lobbying Activity

Meeting with Ann-Sofie Ronnlund (Cabinet of Commissioner Ekaterina Zaharieva)

21 Nov 2025 · Life sciences

Response to EU’s next long-term budget (MFF) – EU funding for competitiveness

31 Oct 2025

Please find attached IPOPI's position on the document.
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Response to European Innovation Act

2 Oct 2025

The International Patient Organisation for Primary Immunodeficiencies (IPOPI) welcomes the opportunity to contribute evidence for the impact assessment on the proposed European Innovation Act. We provide our input from the perspective of patients living with primary immunodeficiencies (PIDs) across the European Union. Our hope for the forthcoming European Innovation Act is that it will bring about increased transparency, predictability, and simplification across the regulatory, reimbursement, and commercialisation pathways for therapies in the EU. Please find IPOPI's full feedback included in the attached document.
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Response to Critical Medicines Act

27 Feb 2025

Medicine shortages are a real challenge for patients requiring therapies for life, such as those living with primary immunodeficiencies (PIDs) and associated conditions. IPOPI, the International Patient Organisation for Primary Immunodeficiencies, welcomes the prioritisation of tackling medicines shortages in the EU and the intention of the new Commission to approach the topic in an expedite manner. However, we believe that such an important proposal should be accompanied by an adequate impact assessment and a round of consultations. We understand that part of the consultation process has been discussed by the Critical Medicines Alliance but that is not enough: this call for evidence comes when stakeholders have neither seen a draft proposal nor any EU Commission document or an impact assessment explaining how the proposal could potentially impact on patients relying on medicines in the EU. Patients with PIDs and associated conditions may require a broad range of therapies to treat their lifelong rare and chronic condition, depending on the specific PID they live with. These range from antibiotics to vaccines, immunoglobulin replacement therapies, targeted therapies or curative options such as gene therapy. Representing a patient community that requires such a vast array of therapies makes us understand that there are some common problems but that the different therapies face significant challenges that are routed in the different specificities of the therapy classes. For example, immunoglobulin replacement therapies are derived from human plasma. The intricate balance between the collection of human plasma and the production time of these therapies (6-12months) requires careful management and represents a unique supply chain that cannot be considered in the same fashion than synthetically produced pharmaceuticals . We therefore hope that the measures included in the Critical Medicines Act will take into consideration the different types of medicinal products and propose tailored solutions, thereby avoiding using a one-size-fits-all approach that would lead to counterproductive effects for the supply of some medicines. It would also be important that there is an alignment between the proposed Critical Medicines Act, recently adopted legislation (such as the Substances of Human Origin Regulation) and does not enter into potential conflict of legislation under discussion (as the pharmaceutical package legislation). In terms of procurement of medicines, we believe that patients are a central piece in the development an adequate tender for the different medicines. In that sense, a meaningful patient engagement that would allow to ensure that the call is fit for purpose, that is: that patients have access to the medicines prescribed by their treating physicians. The inclusion of additional elements for evaluation of joint procurement calls, such as the continuity of supply, so as to avoid having the price as the only deciding criteria, would also ensure that there are no situations of dumping the prices so as to be the only provider in a specific market, disloyally displacing any potential competitor, as it is the case with certain antibiotics in national markets. As the Critical Medicines Act will aim to tackle shortages of medicines to improve the lives of patients in the European Union, we believe that any of the measures should foresee the meaningful involvement of the patients from the definition of critical medicines to the assessment of public procurement criteria, as well as reporting of shortages.
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Meeting with Billy Kelleher (Member of the European Parliament)

9 Sept 2024 · Rare Disease Screening

Meeting with Stella Kyriakides (Commissioner) and

28 Sept 2023 · Meeting of Commissioner Kyriakides with newborn screening associations

Response to Evaluation and revision of the general pharmaceutical legislation

1 Aug 2023

The International Patient Organisation for Primary Immunodeficiencies (IPOPI) is the global patient organisation representing patients living with primary immunodeficiencies (PIDs). PIDs are genetic chronic and rare disorders (RD) in which the immune system does not work properly or at all. PID patients will need different therapies, ranging from antimicrobials, vaccines to immunoglobulin replacement therapies (Igs), biological and targeted therapies or curative therapies (including BMT, gene therapy or thymic transplant). IPOPI welcomes the opportunity to comment on the Commissions proposal. We would like to outline the following points of concern that should be addressed to ensure that it results in a truly patient-centred legislation: 1. Ensure faster & sustainable access to new ATMPs. Patient access to medicines is not a straightforward process. Securing marketing authorisation for any given medicine is only one step of the process, the healthcare systems sustainability and resilience, the manufacturers decision to market the medicine in a given country as well as the coverage and/or coverage of a medicine within national/regional schemes are factors that must be taken into consideration. - Recognise that highly specialised knowledge, infrastructure and follow-up are needed for complex therapies and that a pragmatic approach may be needed when it comes to the interesting proposal to launch new therapies simultaneously across all countries. - Seek pathways to overcome systemic challenges linked to incentives that lead to limited availability, delayed access to medicines and inadequate research and development of orphan medicines as well as potential market failures. 2. Tackle & prevent medicine shortages: shortages are becoming increasingly concerning for PID patients. The Commissions approach is welcomed given that it recognises the problems patients face in the EU, but should be further complemented: - Develop prevention plans, early warning systems and contingency plans that can support the EUs ability to overcome potential shortages in a timely fashion and across all diseases with specific regard for therapies, such as Igs, where the creation of contingency stocks may negatively impact under-pressure markets. - Take steps to efficiently assess and address the multifactorial causes of medicine shortages which can be directly caused by the scarcity of their source material. The production of Igs, for instance, is only possible thanks to plasma availability and blood donations. - Address systemic shortages and supply chain challenges that lead to medicine shortages through the coordinated efforts of European and national regulatory authorities, healthcare professionals, patient organisations, and industry stakeholders. 3. Smooth interplay between existing and future legislation: the pharmaceutical package is one of the primary legislative efforts envisaged by the European health policy ecosystem. Harmonising provisions across legislative dossiers and initiatives already in force or that are being negotiated is highly desirable. - Ensure that patients have clarity on diagnostics and treatment options they can access abroad by further exploring and defining interplay between the cross-border healthcare Directive (2011/24) and the Regulation on the coordination of social security systems (883/2004)). - Optimise the EUs public health ecosystem through regulatory clarity for SoHOs, such as Igs impacted by the future SoHO Regulation and the future pharmaceutical package. - Work towards the development of an EU Action Plan on RD that provides a holistic vision and comprehensive approach that supports people living with RD. 4. Patient involvement - The EUs positive approach has led to increased rates of representation across many European institutions such as ERNS and the EMA CHMP. European decision-makers must ensure patient participation in upcoming political discussions & public forums.
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Response to EU strategic Framework for Global Health

16 Sept 2022

The International Patient Organisation for Primary Immunodeficiencies (IPOPI) welcomes the Commission’s public consultation on the new EU strategy for global health. The European Union has been championing the field of rare diseases from a political, medical and research perspective for many years although the Union is now at the point in which ambitious and comprehensive action needs to be taken in order to ensure that it continues to be a strong ally of persons living with rare diseases. IPOPI considers that the European Union has a key role to play in the following aspects: - Reducing child mortality (identified as UN Sustainable Development Goal 4): the European Union is already promoting and leading on several activities aimed at reaching this ambitious goal. One of them should be to provide a pool of expertise and information so as to ensure equity in access to newborn screening practices for treatable rare diseases. The creation of such platform to favour scientific, medical and administrative evidence on newborn screening programmes could really benefit EU citizens, but also be helpful to those countries elsewhere that are considering strengthening their newborn screening practices. - Universal Health Coverage is another aspect in which the EU has been a strong advocate at international level. Health security systems have managed to ensure a high level of social protection and social inclusion that could be explored by third countries. Examples of this could be the Strategy for the Rights of Persons with Disabilities. In the field of primary immunodeficiencies (PIDs) this would mean that patients in need can have access to their diagnosis, treatment and care in the right and prescribed manner without any financial hardship. - Funding and innovation in the field of rare diseases, by programmes such as the EU Framework Programme for Research and Innovation but also any other funding mechanism that allow for the development of innovative therapies from horizon scanning to the patient bedside. And this last step in the area of gene therapies and ATMPs in general is key: especially in a context where we see that many some companies are withdrawing EU authorised products for rare diseases from the market as they are no profitable due to reimbursement issues. More needs to be done to ensure that patients in need can continue accessing the therapies they need including implementing innovative approaches in terms of reimbursement of expensive but often curative ATMPs. - International collaboration in the field of health research should be strengthened, as citizens around the world are in deer need of new antimicrobials that can be for the new and future resistant microorganisms.
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Response to Revision of the Union legislation on blood, tissues and cells

8 Sept 2022

The International Patient Organisation for Primary Immunodeficiencies (IPOPI) welcomes the European Commission proposal for a Regulation on standards of quality and safety for substances of human origin (SoHO) intended for human application published in July 2022. IPOPI considers that this proposal as a good basis to help tackling some of the shortcomings of the current system and will work with the EU institutions to ensure that the final legislative text is patient centred while ensuring the protection of donors. Over the last years the demand for immunoglobulin replacement (Ig) therapies has been growing at a rate of about 8% per annum and Europe has increasingly been relying on US plasma to meet patient needs. This has prompted IPOPI and other relevant patient organisations to call for increased plasma collection in Europe and more regionally balanced plasma collection worldwide to attain global sufficiency in Plasma Derived Medicinal Products. This much awaited proposal is therefore key to ensure that EU patients with primary immunodeficiencies (PIDs) and many other patients relying on plasma-derived medicinal products, can ultimately and sustainably access an appropriate supply of high quality and safe medicines. Ig therapy is a life-saving treatment for the majority of people with PIDs as it offers protection against life-threatening infections and reduces autoimmune symptoms. There is no alternative treatment for these patients. IPOPI is pleased that this proposal effectively recognises patients and aims to ensure that they are protected and receive high quality and safe medicines. Additionally, we consider a positive step to recognise the importance of ensuring the availability of SoHOs to ensure optimal patient access and sufficiency of supply. At IPOPI we recognise and are grateful to both blood and plasma donors, for their gift of life. We consider it a good step forward that this proposal recognises donor compensation as compatible with the standards concerning voluntary and unpaid nature of SoHO donations. In short, IPOPI considers the Commission proposal as an encouraging starting point to ensure that patients, such as patients with PIDs, can have access to their life-saving therapies in a safe and continued manner. IPOPI is committed to engage with policymakers in the next months to identify areas in the proposal which could be further improved and provide an optimal framework for the collection of life-saving plasma in Europe.
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Meeting with Margaritis Schinas (Vice-President) and

8 Apr 2021 · Health Union

Response to Revision of the Union legislation on blood, tissues and cells

14 Dec 2020

IPOPI represents patients with primary immunodeficiencies (PIDs), chronic and rare conditions in which the immune system does not work appropriately or at all. Around 60% of PID patients need immunoglobulin replacement therapies (Igs) throughout their lives to keep the levels of antibodies within a “normal” threshold to fight infections. No alternative treatments are available for these patients. IPOPI welcomes the Commission’s Inception Impact Assessment and the considerations made to ensure that patients dependent of plasma derived medicinal products (PDMPs) have safe products with high quality and in sufficient quantities. IPOPI agrees that the COVID-19 pandemic has highlighted the risks for supply interruptions, the EU reliance on third countries for plasma and that this underlines the need for timely action. PID patients in the EU are vulnerable to interruptions in the Ig supply. Importantly Igs are not generic medicines and will be tolerated differently by patients. Gold standard care necessitates personalised treatment approaches with the best suited Ig for a given patient based on a physician-patient decision making process, meaning that in every country a panel of different Ig should be available. Some countries have had shortages or severe tensions requiring patients to be switched to whatever Ig available so as not discontinuing their treatment but leading for some to an impaired quality of life. In the most dramatic cases, it has led to interruptions in their treatment with some patients going without their therapy for longer periods. We welcome the idea of increasing monitoring activities to anticipate potential shortages or supply disruptions. This would be helpful in helping anticipating EU supply interruptions. However, the reality for Igs is that potential shortages, potential supply interruptions, are linked to the fact that there is not enough plasma to meet the demands of patients relying on Igs. We welcome the intention of the Commission to clarify certain definitions and concepts. The need to differentiate between blood/blood products and plasma for manufacturing would be important. The EU needs to ensure that countries collect more. Availability of Ig medicines is, for EU PID patients, the most important problem at present. The EU is highly dependent on imports from US plasma. Only few EU countries collect high volumes of plasma. They should be analysed and promoted to make sure that this can also happen in other Member States. Plasma donors can make their donations more often than blood donors, require more time and the procedure is quite different from blood donation. It is time to take different approaches in the promotion of these two types of donations and encourage more plasmapheresis collection programmes across the EU. All countries report respecting the VUD principle, it is time to look into what works in the countries, identify best practise and encourage it adaptation across all EU countries. Increased collection of plasma in one country should be accompanied by measures to ensure that the plasma collected in that particular country could benefit part of its population. Citizens relying on Igs and other PDMPs require that their needs are considered front and centre by their governments, including providing a range of Igs to ensure gold standard care. Patients should be able to access these medicines, with no financial hardship, no matter where they are produced (as long as safe and efficacious). This will be possible if and when countries are able to collect more plasma, ensuring that the EU contributes more towards global sufficiency in PDMPs. Regionally balanced plasma collection where each world region shares its responsibility towards global sufficiency is the way to reach optimal patient care and deal more equitably with potential shortages. The new BTC legislation should not be defined with a view to isolate the EU but within the global context.
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