European Haemophilia Consortium
EHC
The European Haemophilia Consortium (EHC) is an international non-profit organisation representing 48 national patient organisations of people with rare bleeding disorders from 27 Member States of the European Union (EU) and most Member States of the Council of Europe.
ID: 786550013705-85
Lobbying Activity
Meeting with Tiemo Wölken (Member of the European Parliament, Shadow rapporteur) and BGP Products Operations GmbH – a Viatris Company
10 Apr 2025 · Critical Medicines Act
Meeting with Vytenis Povilas Andriukaitis (Member of the European Parliament)
10 Dec 2024 · EU health policy
Response to Revision of the Union legislation on blood, tissues and cells
8 Sept 2022
The European Haemophilia Consortium (EHC) welcomes the European Commission’s proposal for a regulation on quality and safety standards for substances of human origin.
The EHC represents European people with rare congenital bleeding disorders, including haemophilia and von Willebrand Disease. For some of these conditions, plasma-derived medicinal products (PDMP) remain the primary source of treatment. These include, for example, human-derived coagulation and von Willebrand factors. To this end, it is critical that our members have timely access to safe and efficacious medicines. As this legislation impacts the collection of plasma for manufacturing such medicines, it is of direct relevance to patients as end-users of those therapies.
The EHC welcomes the above-mentioned Commission proposal as its objectives align with the EHC’s access and safety objectives. In particular, we appreciate the introduction of requirements for further harmonisation of collection establishments and monitoring by competent authorities, as well as requirements for transparency and avoidance of conflict of interests. We believe that such a framework will help improve the confidence of donors and recipients alike in substances of human origin.
On safety, the EHC welcomes the additions of the European Centre for Disease Prevention and Control (ECDC) and the European Directorate for the Quality of Medicines and Healthcare (EDQM) to set safety standards for the protection of donors and recipients. Both are scientific bodies with the mission to protect human health and have great expertise on topics such as blood-borne pathogen transmission and the preparation for blood and blood components. Their role in harmonising safety requirements will further increase the confidence and safety of donors and recipients alike.
We also agree with the ‘Explanatory Memorandum’ that the Blood Directive 2002/98/EC led to safer blood, blood components, and blood-derived medicines in Europe. However, since its implementation, and despite European and national campaigns and efforts to increase blood and plasma collection, the EU has still not become self-sufficient in plasma collection to manufacture PDMP. Patients in the EU and globally remain reliant on US plasma, which leaves them vulnerable to potential supply disruptions, as most recently witnessed during the COVID-19 pandemic when US blood and plasma collection fell by 10% and 20% over the two years, respectively. However, we believe that this topic extends beyond this legislation. As PDMPs are medicinal products, we would welcome seeing them further included in the general European pharmaceutical strategy on shortage prevention.
Regarding article 54 in the legislation, we believe that blood donation should be voluntary and unpaid and that donors’ health must be protected. However, the requirement for plasma for fractionation or further manufacturing can not be met from recovered plasma from whole blood donors alone. If Europe is to significantly increase plasma collection, there will be a requirement for plasmapheresis programmes where donors would be asked to donate much more frequently than blood donors. In most countries, this will require compensating plasma donors for the time and inconvenience of donating so regularly. Currently, countries with a mixed system of public and private collectors are allowed to provide compensation for the time and efforts of donors, resulting in higher volumes of plasma collection. We welcome the proposal that the legislation will allow the Member States to choose how to thank donors for their donations.
For references, please see the attached document.
Read full responseResponse to Revision of the EU legislation on medicines for children and rare diseases
6 Jan 2021
The European Haemophilia Consortium (EHC) welcomes the revision of paediatric and orphan medicinal products' regulations.
In haemophilia, the OMPR has brought much innovation in the past decade; however, we are disappointed to see that the uptake from the Member States has been variable. Although we understand that the issue of access to novel therapies and technologies, in terms of uptake from a national healthcare system, remains a national competence, we welcome the plan of the European Commission laid in its pharmaceutical strategy to understand causes for lack of uptake and shortages.
We understand that one of the main issues that the Commission wishes to tackle through the revision of this legislation is the lack of medicines in areas of unmet need such as rare diseases, and lack of medicines for children, who make up most of the rare disease population. We understand from the inception impact assessment that new approaches are being considered to define and quantify the concept of rare disease. This could potentially alter the incentives given for product development. In particular, the Commission wishes to use measures of incidence instead of prevalence. From a rare bleeding disorders’ perspective, this could be problematic as we know that often there is a significant delay in diagnosis for some rare bleeding disorders or certain patient populations (e.g. women) suffering from rare bleeding disorders. We would like to take the example of Von Willebrand Disease (VWD), a rare bleeding disorder linked to the lack- or malfunctioning of- von Willebrand Factor. According to two large epidemiological studies(1), up to 1% of a predominantly paediatric population has been found to manifest symptoms and laboratory signs of VWD. This makes VWD one of the more common rare bleeding disorders. However, people affected by VWD have traditionally lacked adequate treatment options compared to rarer bleeding disorders such as haemophilia A and B. Also, in a recent survey conducted by the EHC, which is being submitted for publication, we note that the age of diagnosis in VWD patients ranges from +/- 5.6 years (for the more severe type 3 VWD) to +/- 29.8 years (for VWD type unknown). Although we see encouraging earlier diagnosis in younger patients, which is increasing the incidence slowly, this does not mean patients have adequate treatment options. We understand that the Commission wishes to find ways to incentivise areas of unmet need, but we would like to note that even in more prevalent and well-known rare diseases, patients still lack adequate treatment.
Given the above, we would like to suggest that the Commission consider adding additional criteria alongside prevalence, such as availability of adequate therapeutic options, to identify and potentially layer associated benefits in accordance with true unmet need. As pointed out by our colleagues from EURORDIS: “Even with no objective definition of unmet medical need, regulators, clinicians and patients have no problem identifying them (patients with unmet medical need). A legally binding definition could raise more problems than it would solve, leading potentially to long discussions to the detriment of the populations intended to be served.”
(1) Rodeghiero F, Castaman G, Dini E. Epidemiological investigation of the prevalence of von Willebrand’s disease. Blood 1987; 69:454-459, and
Werner EJ, Broxson EH, Tucker EL, Giroux DS, Schults J, Abshire TC. Prevalence of von Willebrand disease in children: A multiethnic study. J Pediatr 1993; 123:893-898.
Read full responseResponse to Revision of the Union legislation on blood, tissues and cells
14 Dec 2020
The EHC is pleased to see that the revision of the legislation maintains its original objectives, i.e. to ensure safety and quality of blood components as well as to safeguard the health of patients and donors. This is paramount to the rare bleeding disorders community, which in the past has experienced first-hand devastating effects of the lack of provisions ensuring safety and quality of plasma-derived therapies, which has resulted in the contamination of thousands of patients with HIV and hepatitis C.
The EHC believes that the second objective of ensuring access and avoiding shortages is key as, at the moment, many patients in Europe rely on these therapies for living a normal life, and any supply disruptions can put their health at great risk. Finally, the field of blood collection and safety is continuously evolving and facing new challenges, such as emerging pathogens. Therefore the flexibility and ability to quickly keep up to date with technological and epidemiological developments are essential to meet the first two objectives.
The EHC prefers the second policy option laid out in the document. Both the EDQM and ECDC are technical bodies whose work is guided by science and not politics and whose primary role is to give sound scientific advice to both European Institutions and the Council of Europe. The ECDC is already tasked with issuing epidemiological reports and their impact on the blood supply as well as guidance on safety measures to address these risks, while the EDQM is already producing guidance material on the quality of blood, tissues and cells. Also, although the EDQM is not part of the European institutions, it is already well versed in working with such institutions through its work on the European pharmacopoeia, in the context of the pharma code. Option 2 has the advantage of building on existing work, which will not require for these agencies to increase their workforce significantly. Therefore we can predict that the budgetary impact on the collection of BTC will be minimal. These options will help to ensure further that the quality of BTC will be the same across Europe, hence improving patients’ safety. Besides, we would hope that a more official role for the EDQM in setting standards of quality for BTC will further encourage countries outside the EU to adopt these measures and further improve the quality of their BTC. Finally, from a policy and legislative perspective, this option seems to allow for the flexibility required to adapt to technological and epidemiological changes quickly.
However, it is not yet clear to us how this second policy option would further promote the collection of BTC and support the second objective of the revision. It is our understanding that, at the moment, a majority of plasma-derived treatment used in Europe is made from US plasma, this is due to legislative differences in the US, which allow more donations per donor per year. We understand that for the past decades, Member States have been trying to reach self-sufficiency in Europe for blood and blood components but that this, however, has not yet been achieved. We also understand that the need for blood components for transfusion has been declining due to medical developments, while the need for plasma for fractionation has continued to increase. We would like to know whether the Commission has identified policies and initiatives implemented in the Member States that have allowed for an increase in plasma collection while maintaining the safety of both donors and patients. Also, seeing the Commission has laid out in its pharmaceutical strategy several objectives regarding a better understanding of pharmaceutical supply chains, we think that this may also be an area worthy of work as to perhaps identify ways to increase plasma collection in Europe further while maintaining the safety of both donors and patients.
Read full responseResponse to Pharmaceutical Strategy - Timely patient access to affordable medicines
6 Jul 2020
The European Haemophilia Consortium (EHC) welcomes the European Commission’s (EC) proposed Pharmaceutical Strategy (PS) and agrees with its objectives.
The EC rightly points out in its Roadmap that there is unequal access to medicines in Europe, especially for innovative therapies. The EHC would like to confirm that this is also the case in the disease area we represent. Therefore, EHC supports the objective of the PS to ensure greater access to affordable medicines.
However, we believe that getting medicines through regulatory approval for marketing in Europe is only half of the challenge. In fact, in our particular disease area, we know that limitations to access novel and existing therapies are due primarily to economic reasons. Thus, we believe that the EC should spearhead new economic models and purchasing approaches to make innovative treatments truly available and affordable to all European Member States (MS).
Integrating novel therapies into national healthcare systems will also require MS preparedness, and we think that this should be coordinated at European level in particular when talking about highly innovative and disruptive treatments like gene therapy. Such therapies will require a high level of education for both healthcare professionals and patients, and we think that they should only be given in expert centre hubs. This is because of the unknowns on the long-term effects of these medicines, not only regarding their efficacy but also concerning their safety. This is why we believe that any novel therapies coming to Europe should be adequately monitored for their efficacy and safety for a period of time that allows generating statistically meaningful data to assert these properties. We recognise that regulatory provisions are already in place to this end, however, for innovative therapies, which are irreversible and have the potential to last a lifetime, these should be extended, perhaps by collecting this data in existing registries.
On the note of registries, we believe that these are important and powerful tools to monitor medicines but also to help develop new medical technologies and we believe that the PS should address their importance in medicines development and ideally find ways to provide them with resources and support. This could be perhaps done via ERNs.
The COVID-19 pandemic has disrupted access to regular care and sometimes medicines. In the haemophilia community delivery of treatment at home was already an established practice in some countries, but it was taken up by other countries during this time to avoid unnecessary hospital visits. The PS should also put forward models for safe delivery of hospital-distributed medicines in times of global health crisis so that we can ensure that all patients continue to have timely access to their treatment during these difficult times. This should go in par with the use of telemedicine for continued access to care and digital reporting of potential side-effects. Another disruption brought by this pandemic has been the decrease in blood and plasma donations, which are used to make plasma-derived medicines. While the EU should find solutions to reduce its dependence on plasma import and increase blood and plasma supply within its borders, efforts should also be made on improving the supply of innovative products as a way to reduce reliance on blood components.
Finally, the EHC would like to see the inclusion of patient representatives at each step of medicines development, purchase and distribution processes. This is a model that is successfully used in our disease area. Furthermore, an EHC 2014 survey demonstrated that cost-savings were made in countries that formally involved patient representatives in their medicines purchasing processes. We hope that this can be included in the PS. In attachment our full response and references.
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