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EUROPEAN ORGANISATION FOR RARE DISEASES

EURORDIS

EURORDIS-Rare Diseases Europe is a patient-driven alliance representing 30 million people with rare diseases across Europe.

Lobbying Activity

Rare disease alliance urges dedicated health funding in EU budget

10 Nov 2025
Topic — EU's long-term budget 2028-2034 and proposed European Competitiveness Fund structure
Message — The organization requests a ring-fenced health program within the EU budget with dedicated funding for rare diseases. They call for a European Action Plan, sustained support for European Reference Networks, joint procurement mechanisms for therapies, and continued operating grants for patient organizations.123
Why — This would maintain visibility and stable funding for their network infrastructure and policy participation.45
Impact — The 30 million Europeans with rare diseases lose coordinated care if health priorities are subordinated to market goals.67
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Meeting with Nicolás González Casares (Member of the European Parliament, Rapporteur)

6 Nov 2025 · Rare Diseases

Meeting with Olivér Várhelyi (Commissioner) and

2 Oct 2025 · All pressing portfolio topics

Meeting with Maya Matthews (Head of Unit Health and Food Safety)

29 Sept 2025 · Involvement of individual experts, including patients, taking part in joint clinical assessments and scientific consultations under the EU HTA Regulation, as well as capacity-building activities funded by the EU4Health

Meeting with Olivér Várhelyi (Commissioner) and

15 Jul 2025 · Ongoing policy developments

Response to Establishment of an expert panel on orphan and paediatric devices

16 Jun 2025

The proposal to establish an expert panel dedicated to orphan and paediatric medical devices is strongly supported. Currently, there are very few devices specifically developed for rare diseases and children, yet these devices are essential for patients, carers, and healthcare professionals managing complex and rare conditions. Our membersrare disease patient organisationsreport significant unmet needs and growing difficulties in accessing necessary medical equipment. For example, healthcare professionals are finding it increasingly hard to obtain disposables and instruments for operations. Manufacturers are withdrawing products from the market rather than navigating new certification processes, especially since the small patient populations make these markets less attractive. In countries with tax-financed healthcare, political decisions often result in the withdrawal or increased cost of essential aids, leaving vulnerable patients without support. Patients and carers also highlight a lack of information about new developments in medical devices. Many feel excluded from the innovation process and report that once they receive a device, they are not informed about new options that might become available. The main sources of information remain treating physicians, physiotherapists, device technicians, patient communities, and quite often only the internet. To address these challenges, it is crucial that the expert panels include patient representatives to ensure that the voices and experiences of those directly affected are central to decision-making. Furthermore, these panels should be closely linked with initiatives funded by the EU4Health programme, such as the DeCODe project, to leverage existing expertise and foster collaboration. In summary, the expert panel should: Provide scientific, technical, and clinical guidance to improve access and innovation for orphan and paediatric devices. Enhance transparency and communication about new and existing medical devices. Include patient representatives to reflect real-world needs and experiences. Collaborate with EU-funded projects to maximise impact and ensure sustainable solutions for rare disease and paediatric patients. This approach will help address current gaps and improve outcomes for patients across Europe.
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EURORDIS urges dedicated Rare Disease Cluster in Biotech Act

11 Jun 2025
Topic — The European Commission consults on legislative measures to accelerate biotechnology market transition.
Message — EURORDIS proposes a Rare Disease Cluster to consolidate fragmented efforts and strengthen competitiveness. They recommend adaptive approval pathways and greater acceptance of non-traditional evidence like surrogate endpoints. The group calls for strategic investment tailored to academic institutions and smaller developers.123
Why — Regulatory alignment would reduce the high development costs currently hindering smaller developers.4
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EURORDIS urges EU to prioritize rare diseases in life sciences

17 Apr 2025
Topic — A new European strategy to boost competitiveness and innovation in life sciences.
Message — The organization calls for prioritizing rare diseases by streamlining regulations and increasing research funding. They request that patient organizations be funded as equal partners throughout the research chain.123
Why — This would secure sustainable funding and higher political priority for rare disease patient groups.45
Impact — Drug manufacturers may face reduced profit margins from collective EU pricing and procurement strategies.6
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Meeting with Donata Meroni (Head of Unit Health and Food Safety)

26 Mar 2025 · Exchange of views on Cross-Border Healthcare and on patient organisations, EURORDIS and ERNs

Response to EU rules on medical devices and in vitro diagnostics - targeted evaluation

21 Mar 2025

EURORDIS-Rare Diseases EURORDIS Rare Diseases Europe is a non-profit alliance of over 1100 rare disease patient organisations from 75 countries that work together to improve the lives of over 30 million people living with a rare disease in Europe. By connecting patients, families and patient groups, as well as by bringing together all stakeholders and mobilising the rare disease community, EURORDIS strengthens the patient voice and shapes research, policies and patient services. EURORDIS wishes to contribute to this consultation by sharing again the attached letter, dated December 22, 2023, co-signed with the European Society for Human Genetics (ESHG) and European Reference Networks (ERNs) Coordinators Group, addressing the exemption of rare diseases diagnostics from the In Vitro Diagnostic Medical Devices Regulation (EU) 2017/746 (IVDR). The issues raised there remain highly relevant and are reiterated -in light of extensive evidence gathered by the ESHG recent survey- in the contribution of the ESHG to this consultation, which EURORDIS endorses. Our concerns, as illustrated in the attached letter, persist: the IVDR in its current form would result in a shortage of crucially important genetics diagnostics, and therefore lead to missed, delayed or inaccurate diagnoses. This would not only hinder innovation of rare disease diagnostics, but would also roll back advances made in the last decade in the field of rare disease diagnostics and challenge the substantial improvements towards shortening of the diagnostic odyssey of people with rare diseases. Challenges for rare disease diagnostics include increased costs and administrative burdens, requirements for large cohorts that are unavailable for most rare diseases, potential hindrances to indicated tests for economic reasons. There is a serious risk of shortages of devices, especially class D devices, which may hinder the availability of in vitro devices in the EU. These issues may lead to missed, delayed, or inaccurate diagnoses, potentially rolling back advances made in rare disease diagnostics over the last decade. Therefore, we collectively called for an exemption of rare diseases/rare cancers diagnostics (or, as we propose, orphan diagnostics) from IVDR regulation in Article 5.5; and all necessary measures, including those of legislative nature where appropriate, to reduce or avoid shortages of classes of devices that are essential for patients. We kindly request your attention to these crucial matters and remain available for further discussion.
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Meeting with Donata Meroni (Head of Unit Health and Food Safety)

5 Mar 2025 · Exchange of views on the role of patient organizations with the European Reference Networks.

Meeting with Isabel Wiseler-Lima (Member of the European Parliament) and ALAN asbl-Maladies Rares Luxembourg

5 Mar 2025 · Rare Disease Day

Meeting with Vlad Vasile-Voiculescu (Member of the European Parliament) and European Cancer Organisation and European Society for Paediatric Oncology

15 Jan 2025 · Intergroup on cancer and rare diseases

Meeting with Stine Bosse (Member of the European Parliament) and DSW (Deutsche Stiftung Weltbevoelkerung)

5 Dec 2024 · European health policy

Meeting with Lynn Boylan (Member of the European Parliament)

20 Nov 2024 · Rare Diseases Week

Meeting with Olivier Chastel (Member of the European Parliament)

20 Nov 2024 · Politique de santé de l Union européenne

Meeting with Margarita De La Pisa Carrión (Member of the European Parliament)

20 Nov 2024 · Enfermedades Raras

Meeting with Pál Szekeres (Member of the European Parliament)

19 Nov 2024 · Rare Disease Week 2024

Meeting with Dolors Montserrat (Member of the European Parliament)

19 Nov 2024 · Rare Diseases incidence in Europe

Meeting with Estelle Ceulemans (Member of the European Parliament)

19 Nov 2024 · Dossier SANT à venir

Meeting with Vytenis Povilas Andriukaitis (Member of the European Parliament) and FGS Global (Europe) GmbH

19 Nov 2024 · EU health policy

Meeting with Tilly Metz (Member of the European Parliament)

19 Nov 2024 · Rare diseases

Meeting with Billy Kelleher (Member of the European Parliament)

19 Nov 2024 · Rare disease policy

Meeting with Radan Kanev (Member of the European Parliament)

19 Nov 2024 · EU Policy - Rare Diseases

Meeting with Tomislav Sokol (Member of the European Parliament) and MedTech Europe and

19 Nov 2024 · Health policy

Meeting with Ingeborg Ter Laak (Member of the European Parliament)

19 Nov 2024 · Rare diseases

Meeting with Elena Nevado Del Campo (Member of the European Parliament)

19 Nov 2024 · Rare Diseases in Europe

Meeting with Stine Bosse (Member of the European Parliament, Committee chair)

8 Oct 2024 · European health policy

Meeting with Vlad Vasile-Voiculescu (Member of the European Parliament)

17 Sept 2024 · Introductory Meeting

Meeting with Tomislav Sokol (Member of the European Parliament) and MEDICINES FOR EUROPE

17 Sept 2024 · Health policy

Meeting with Stella Kyriakides (Commissioner) and

23 Jul 2024 · Meeting with EURORDIS to discuss the Commission’s actions on rare diseases

Response to Health technology assessment – Procedural rules for the assessment and management of conflicts of interest in joint wo

26 Jun 2024

The European Organisation for Rare Diseases (EURORDIS) welcomes the opportunity to comment on the draft Implementing Act on the management of conflict of interest. EURORDIS welcomes the attention paid to ensuring all stakeholders taking part into the European health technology framework are free of conflict of interest and would like to highlight some conditions and key considerations for the success of the upcoming activities on health technology assessments at the European level: 1. Timeline and delays are long and may significantly delay the HTA procedure. In the scenario where a DoI is submitted and requires further information to be provided, it would take a maximum of 10 days for the representative to submit additional information (Article 4), 15 working days for the European Commission to complete the assessment if no further information is needed from the submission of the DoI (Article 4), and 14 days for the representative to provide requested information should the European Commission become aware of inconsistent information with the information provided in the DoI (Article 10). This brings the total possible maximum number of days to 39 to, potentially, decline a representative participation in joint work. If the whole procedure has to be started again to find a second and alternative expert free of conflict of interest, it significantly delays their participation in joint work, which will have already started long before the start of the appointed representatives participation. Shorter delays should be envisaged for actions linked to the submission of information and the evaluation of these information, as suggested in our specific comments on articles 4 and 10. 2. As risks and implications relating to potential conflicts of interest vary from participation in Joint Clinical Assessments to Joint Scientific Consultation, different and distinct measures should be stated to manage conflicts of interest based on which procedures representatives will be invited to participate in. 3. The definition of representative should encompass carer, relative, friend, staff of a patient group, trusted person. Their contributions, as well as patients contributions, must be as visible and transparent as any other experts contributions. 4. Vagueness of the implementing act should be avoided. Further definitions and examples are needed to define what a situation of appearance of a conflict of interest are. Further clarification is also needed on the measures about conflict or potential conflict of interest of the representatives. 5.Confidentiality Undertaking and Insider Trading are missing from the annex. We thus suggest the Declaration of Interest should include these aspects 6. It would be relevant to determine which financial support or percentage of financial support from an industry to a patient organisation would represent a problematic conflict of interest and hamper independence. Some patient organisations, especially in rare diseases, would have to list all industrials partially financing their structure and activities, although some of these industrial supports do, not represent a percentage high enough to hamper impartiality on the part of patients. Not taking account of this aspect would hamper participation of valuable experts to the detriment of the quality of patient participation, mostly in rare diseases where expertise is scarce and precious to different stakeholders, often without inducing conflict of interest. 7.The challenge with Implementing Act is that it is difficult to modify these afterwards, which does not apply to guidelines. EURORDIS therefore suggests that the annexes of this Implementing Act (for instance, on the Declaration of Interest) be included in a document distinct from the Implementing Act, as to allow for possible modifications based on experience and practice later on.
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EURORDIS urges realistic deadlines and deeper patient involvement

28 Mar 2024
Topic — New rules establishing a framework for joint clinical assessments of medicinal products.
Message — EURORDIS requests realistic assessment scopes based on medical knowledge and longer deadlines for data submission. They also call for meaningful, predictable involvement of patient experts throughout the evaluation.12
Why — Realistic timelines would ensure high-quality assessments and faster access to rare disease treatments.3
Impact — National health systems lose if tight deadlines result in poor quality or inconclusive clinical reports.45
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Meeting with Frédérique Ries (Member of the European Parliament, Shadow rapporteur) and Acumen Public Affairs

7 Mar 2024 · Revision of the Pharmaceutical Legislation

Meeting with Stelios Kympouropoulos (Member of the European Parliament)

21 Feb 2024 · rare diseases

Meeting with Helena Dalli (Commissioner)

21 Feb 2024 · Meeting with National Alliance for Rare Diseases Support Malta to discuss women's health matters.

Meeting with Tilly Metz (Member of the European Parliament, Shadow rapporteur)

8 Feb 2024 · Pharmaceutical legislation

Meeting with Frédérique Ries (Member of the European Parliament, Shadow rapporteur)

7 Feb 2024 · Revision of the Pharmaceutical Legislation

Meeting with Stella Kyriakides (Commissioner) and

29 Jan 2024 · Exchange of views on the ongoing EU actions on rare diseases

Meeting with Pernille Weiss-Ehler (Member of the European Parliament, Rapporteur)

11 Jan 2024 · Directive on Medicinal products for human use

Response to Interim evaluation of the EU4Health Programme 2021-2027

9 Jan 2024

EURORDISRare Diseases Europe is a non-profit alliance of over 1000 rare disease (RDs) patient organisations working together to improve the lives of 30 million people living with a RDs in Europe. A EU public health priority due to the unmet needs of RD population, added value of the EU action, RDs have been consistently supported by EU funding programmes, thus helping improve lives of people with RDs, advance European research and innovation in a cutting-edge area and strengthened healthcare systems. EURORDIS is thankful for the renewed support of the EU4Health (EU4H) to RDs and the RD community at large. For the upcoming years, actions susceptible to impact lives of people with RDs, in line with the EU4H objectives and the ongoing EU policy agenda, are listed in the attached document. EU4H is a welcome opportunity to build better resourced, equal and resilient health systems and we acknowledge the unprecedented amount of funding associated with the Programme and its ambitions. We trust that this level of financial support is maintained and reinforced in the long-term as increased investments are needed to keep Europeans healthy and Europe prosper. Areas to improve the Programme include: 1.Meaningful involvement of civil society in EU Health Programmes. To achieve its objectives, to improve all Europeans health, responding to their needs, it is crucial to fully involve health civil society for ex. by consulting them on strategic priorities of annual work programmes to ensure these address real needs for a more efficient use of the budget, leaving no disease areas nor population groups behind. More meaningful involvement of civil society organisations (CSOs) is needed to truly comply with the Better Regulation guidelines. For ex. the short time provided, over the winter holidays, for this Interim Evaluation of a programme of this size, is regrettable as extremely limited. 2.Continued support to CSOs through Operating Grants within a multi-annual framework: OGs are a financing mechanism enabling CSOs to support the achievement of many EU4H objectives as independent and expert partners, reflecting real-life perspectives of patients and citizens. Multi-annual frameworks for OGs to CSOs are crucial to provide CSOs a more stable and secure framework to better serve the citizens needs. Year-to-year funding perpetuates precarity for CSOs, impedes long-term strategic planning, compels applicants and the EC to spend additional time and resources each year in evaluation and reviewing processes. 3. 60% funding ceiling for action grants is challenging: While reasonable for initiatives requiring reallocation of staff to new actions, this funding level is financially burdensome CSOs where the nature of the work involves significant direct costs, such as attendee travel or engaging contractors. This appears penalising, if compared to other programmes (e.g. CERV 90% cofunding for action grants). A reassessment is needed to rapidly alleviate this financial burden. Exceptions to the 60% funding rule could be envisaged based on the typology of partners (patients organisations vs. public bodies) and to the typology of the activities (e.g. training should be 100% funded). 4.Awarding procedure. The decision to award two consortiums for the same EU4HEALTH Call (EU4H-2022-PJ-04, HTA patient training) was sub-optimal. It is not uncommon to have multiple training programmes on the same subject, yet, despite the general encouragement for collaboration between the two successful projects, the existing framework of our respective consortium agreements makes it difficult to achieve meaningful cooperation, creates an unclear environment for those who are looking for trusted sources of trainings. A more proactive approach e.g. consulting both consortiums earlier in the process, inviting them to revise their proposals during the contract stage would have prevented such complications, facilitated synergy between the selected projects from the outset.
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Meeting with Pernille Weiss-Ehler (Member of the European Parliament, Rapporteur) and Novo Nordisk A/S

30 Nov 2023 · Directive on Medicinal products for human use

Meeting with Frédérique Ries (Member of the European Parliament, Shadow rapporteur)

28 Nov 2023 · Revision of the Pharmaceutical Legislation

Meeting with Ville Niinistö (Member of the European Parliament, Shadow rapporteur for opinion)

22 Nov 2023 · Reform of the EU pharmaceutical legislation

Response to European Disability Card

10 Nov 2023

EURORDIS-Rare Diseases Europe welcomes the proposal for a Directive establishing the European Disability Card and the European Parking Card for persons with disabilities, as well as the complementary proposal extending the Directive to third country nationals legally residing in a Member State. The proposed Directive is an important step to support free movement of persons with disabilities within the European Union. Both cards will reduce barriers to tourism and travelling, promoting the participation of persons with disabilities in society and reducing discrimination. They will also be particularly helpful for people with invisible or cumulative disabilities, who face additional attitudinal barriers. EURORDIS has done a first analysis of the proposal, from the perspective of people living with rare diseases, the majority of whom live with disabilities. Based on this analysis, we would like to highlight some positive elements of the proposal and some areas where the proposal could be strengthened. We applaud that the proposal: Is based on binding legislation. Has a broad scope, applying to all public and private services, activities and facilities which offer preferential conditions (except social security benefits, social protection, or social assistance). Reflects many principles highlighted by the disability community. To guarantee the success of the proposed Directive, and that people living with rare diseases and others with complex disabilities are not left behind, we recommend: 1. Ensuring that the Directive is clear on eligibility to the European Disability Card being granted to all persons with disability status, irrespective of the severity of disability. 2. Recognising that the improvement of national disability assessment and recognition frameworks is essential to guarantee adequate eligibility to the European Disability Card; and further assisting Member States in establishing and sharing good practices on disability assessment. 3. Supporting a greater harmonisation of disability assessment principles and tools across Member States, to prevent inequalities in accessing the European Disability Card. A detailed analysis and justifications on these three points are provided in the attached document. We further call on the EU institutions to move quickly to improve the proposal and adopt it as soon as possible. We stand ready to engage with all policy makers who wish to have further information.
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Meeting with Catherine Amalric (Member of the European Parliament, Shadow rapporteur)

26 Oct 2023 · Reform of the EU pharmaceutical legislation

Meeting with Laura Ballarín Cereza (Member of the European Parliament, Shadow rapporteur for opinion)

13 Oct 2023 · Pharmaceutical Package

Meeting with Pernille Weiss-Ehler (Member of the European Parliament, Rapporteur) and Gilead Sciences

5 Oct 2023 · Directive on Medicinal products for human use

Meeting with Stella Kyriakides (Commissioner) and

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

Meeting with Deirdre Clune (Member of the European Parliament)

28 Jun 2023 · Rare Diseases

Meeting with Giorgos Rossides (Cabinet of Commissioner Stella Kyriakides), Karolina Herbout-Borczak (Cabinet of Commissioner Stella Kyriakides)

31 May 2023 · Exchange of views on the pharmaceutical reform

Meeting with Deirdre Clune (Member of the European Parliament)

31 May 2023 · Rare Diseases & Neonatal Screening

EURORDIS urges EU recognition of rare disease mental health crisis

14 Feb 2023
Topic — This consultation addresses the development of a comprehensive EU strategy for mental health.
Message — EURORDIS requests that the EU recognize rare disease patients as a vulnerable population. They call for mental health screening to be integrated into the diagnostic pathway for families.12
Why — Recognition would secure specialized care and help reduce the significant socioeconomic burden.34
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Meeting with Tilly Metz (Member of the European Parliament)

8 Feb 2023 · rare Diseases

Meeting with Andrey Kovatchev (Member of the European Parliament, Rapporteur for opinion)

7 Feb 2023 · European Health Data Space and Rare disease patients' concerns

Meeting with Deirdre Clune (Member of the European Parliament)

7 Feb 2023 · Rare Disease Week

Meeting with Dolors Montserrat (Member of the European Parliament, Rapporteur)

11 Jan 2023 · Lessons learned on impact of COVID-19 pandemic

Meeting with Stella Kyriakides (Commissioner) and

6 Dec 2022 · Meeting with representatives of Eurordis

Meeting with Andrey Kovatchev (Member of the European Parliament, Rapporteur for opinion)

25 Oct 2022 · European Health Data Space and Rare disease patients' concerns

Meeting with Stella Kyriakides (Commissioner) and

21 Sept 2022 · Rare diseases

Rare Disease Patient Group Calls for Patient-Centred EU Health Data Space

27 Jul 2022
Topic — EU regulation establishing a European Health Data Space for safe data exchange and research.
Message — The organisation requests that electronic health records use ICD-11 and ORPHA rare disease codes, implement strong data governance allowing patients to control their data, and establish the European Reference Networks as a testing hub for data governance models.123
Why — This would enable rare disease patients to share health information with specialists across borders more easily.4
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Meeting with Sirpa Pietikäinen (Member of the European Parliament, Rapporteur)

10 Mar 2022 · EU Care Strategy

Meeting with Helena Dalli (Commissioner) and

26 Jan 2022 · Discussion on the Commissioner work programme related to rare diseases as well as the continued work of EURODIS, in particular its collaborative efforts with the EU's institutions.

Meeting with Margaritis Schinas (Vice-President) and

2 Dec 2021 · Rare diseases

Meeting with Giorgos Rossides (Cabinet of Commissioner Stella Kyriakides) and European Patients' Forum (EPF) and

25 Nov 2021 · Follow up to Cssr Kyriakides’ meeting with CSOs on 25/10/2021 on civil society funding through EU4Health

Meeting with Stella Kyriakides (Commissioner) and European Patients' Forum (EPF) and

25 Oct 2021 · VTC Meeting: EU4Health Work Programme 2022 and operating grants.

Meeting with Stella Kyriakides (Commissioner) and European Patients' Forum (EPF) and

30 Jul 2021 · Meeting with civil society representatives regarding the EU4health programme.

Meeting with Karolina Herbout-Borczak (Cabinet of Commissioner Stella Kyriakides)

17 Jun 2021 · Exchange of views on rare diseases in the context of the pharmaceutical strategy for Europe and the presentation of EURORDIS Recommendations of the Foresight Study on rare diseases policy

Rare disease advocates call for EU-wide cooperation on innovation and access

27 Apr 2021
Topic — Revision of EU pharmaceutical legislation to improve medicine access and foster innovation.
Message — The organization requests multi-stakeholder early dialogue on unmet medical needs, including patient representatives, and stronger EU cooperation on pricing negotiations. They emphasize the need for definitions of unmet needs that include disease severity and patient-reported metrics.12
Why — This would give rare disease patients earlier influence over drug development and more coordinated access across EU countries.34
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Response to Evaluation of patient rights in cross-border healthcare

11 Feb 2021

EURORDIS-Rare Diseases Europe welcomes the undergoing evaluation of the Directive on patient rights in cross-border healthcare. Rare diseases (RD) are disabling and degenerative conditions, most of them with no cure, that affect 30 million people in Europe that seldom have access to the right expertise and diagnostic infrastructure where they live. The needs of these extremely rare conditions are of such magnitude that they require greater cross-border healthcare collaboration to meet them. Ten years after its entry into force, the evaluation of the Directive is timely as the latter fails short of its ambition and, specifically for the RD population, it leaves many of its needs not addressed. We share the conclusions of the 2019 report of the European Court of Auditors on the Directive which, whilst praising its ambitions, noted there is still a long way to go in implementing it well, as the community of people with RD knows all too well. 1. ERNs. Key findings from a survey on RD patients experience of medical care (Jan. 2021), give us a positive albeit small glimpse into the experience of care provided by the expert centres in the ERNs. Respondents indicated that they waited for a shorter amount of time to get a confirmed diagnosis of a rare or a complex disease, have a better experience with their care and are significantly more satisfied with their care. Yet, ERNs’ full potential is not yet unlocked and the consolidation of this new structure needs to be secured into our national health systems (see attached document for more details). ERNs do not yet fully contribute to meet the healthcare needs of the RD patient population: although the ‘infrastructure’ is established, patients have limited access to it, hence cannot enforce their rights to cross-border healthcare. Now, at a time when greater EU cooperation in health is essential, we also need to carefully plan the next steps to reach the ERNs’ true potential and integrate this new structure into our national health systems. 2.Measures to ensure patients’ rights to cross-border health care. People living with RD often have little information available about their diseases and very few treatment options. This is why, while they usually prefer to be treated near their home, they are also more willing to travel to another country to receive care or treatment than the general population. EURORDIS’ recent findings (see enclosed document) confirm this disposition. Every person living with a RD in Europe has the right to access to the highest possible quality of diagnosis, care and treatment without unnecessary delay or physical, social or financial burden. Where this right cannot be met within the country of residence, national competent authorities ensure it is met on a cross-border basis, in a timely manner. It is the Directive’s ambition to secure this right for those who seek care across borders with adequate measures. Now, the Directive fails short of its ambition. As a matter of fact, people living with RDs do not travel under the Directive but continue to use the “Social Security” Reg. 883/2004, as a result of a number of barriers they face when trying to use the Directive’s measures instead. Barriers and suggestions are detailed in the attached document, and include: -addressing upfront payments that are burdensome and most patients; -linking prior authorisation entities and RD Centres of Expertise and ERNs to help taking well-informed decisions by the former, with consequent benefit for patients. Prior authorisations for RD patients should be based on the opinion of the relevant experts; -better information of patients, health professionals and stronger NCP with link to RD expert centres and bodies for information relevant to RDs. Overall, EURORDIS calls for an overall review and redesign, where necessary, of the policy and legal framework for cross border access to healthcare, which is essential to truly leave no one behind.
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Response to Legislative framework for the governance of common European data spaces

8 Feb 2021

EURORDIS represents 30 million people living in Europe with one or more of over 6,000 identified rare diseases. We welcome the opportunity to submit our observations to the Data Governance Act (DGA) as we believe that it should contribute to derive value from health data to improve healthcare services, research and policymaking while protecting people’s privacy and respecting their preferences. As their member, EURORDIS endorses the European Patient Forum’s response to the consultation, but would like to highlight some additional aspects that are relevant for our community. The regulatory and non-regulatory initiatives that will form the EHDS will have to respect and build on the definitions and provisions of the future DGA. We are concerned that some of these are not sufficiently clear and might be difficult to enforce in the health sector. We would like draw attention to the definition of the term ‘general interest’ and the implications of the artificial distinction being made between data sharing in exchange of a reward and data sharing for the public good, leading to two different regulatory schemes. Clarify “general interest”. This term is used extensively in the draft DGA and is also used to define concepts such as data altruism. However, no clear definition of the term is provided. We invite the European Commission (EC) to consider providing further criteria to clarify what activities might qualify as being of general interest, and whether the organisations undertaking those activities need to meet any further criteria. Re-assess the rationale for having 2 different sets of rules for data sharing and data altruism. We urge the EC to clarify the rationale for having two different sets of conditions and requirements for health data sharing activities. The DGA should provide further clarification on the definitions of data sharing and data altruism, and the rationale for having two different legal frameworks for data intermediaries and data altruism organisations and services. Ultimately, both terms refer to the same activity, namely allowing the use and re-use of one’s personal data for specific purposes. Some uses will have a commercial purpose and others not, sometimes there might be a direct reward, other times not. Yet, this distinction does not appear to justify having different regulatory oversight, compliance monitoring mechanisms, governance arrangements and standards for the organisations offering these data “brokering” services, at least not in the health sector. Also, the conditions for providing any type of health data sharing service for any purpose, including for the common good, should be the same. We also have concerns with the use of the term “altruism” as it evokes an emotional approach that springs from personal inner values and refers to “why” people provide their data for further use and not the action on itself. Purpose should not trigger a different regulatory framework or level of scrutiny for the organisations dealing with personal data sharing. The use of this term could also give raise to prejudices against people who might not feel comfortable sharing their data for whichever reason.Certainly, health sector-specific legislation will be needed to complement the DGA, but it will not be possible to include derogations from the provisions of the DGA unless this possibility is specifically foreseen in the DGA. As a horizontal framework, it is key that the DGA provisions and principles can work properly across different sectors without creating further confusion and fragmentation. Therefore, we urge the EC to re-assess, together with the relevant stakeholders, how and to what extent the DGA can function properly as a horizontal framework in the health sector. EURORDIS looks forward to continue collaborating with the EC to bring clarity on how the horizontal and sector-specific legislation will complement each other. Attached our response to the Digital Health Data and Services Roadmap Consultation
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European rare disease alliance urges multi-stakeholder health data governance

3 Feb 2021
Topic — Proposal for EU regulation establishing a European Health Data Space.
Message — EURORDIS requests a well-structured multi-stakeholder dialogue to co-create health data guidance and rules. They want ERNs used as testing environments for new governance models. They call for an EU-level agency to coordinate cross-border health data sharing with national agencies.123
Why — This would help them pool scarce expertise and data across borders for rare diseases.45
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Patient alliance urges seamless EU rare disease therapy pathway

6 Jan 2021
Topic — Revising EU regulations on medicines for rare diseases and children.
Message — EURORDIS calls for early multi-stakeholder dialogue on unmet needs, graduated incentives rewarding earliest engagement, strengthened regulatory committee mandates, and a common European negotiation table for timely equitable access across member states.1234
Why — This would accelerate development and approval of treatments for their 30 million constituents.56
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Response to Revision of the Union legislation on blood, tissues and cells

14 Dec 2020

EURORDIS acknowledges the need to revise legislations for Blood, Tissue and Cell derived products. Current legislations and rules did not suffice to ensure EU self-sufficiency for these products, and their safety might still be a concern. Furthermore, important disparities exist in the EU, between Member States where these products are available and used, and other Member States where they are too difficult to obtain. Another concern is the waste of some blood-derived products, as large amounts of coagulation factors VIII or IX are left unused. While the collection and the use of blood remain in the public sector, plasma-derived products, e.g. immunoglobulins are processed and commercialised by the private sector. This creates administrative complexity for care centres. Considerations on the three options and preferred scenario Option 2 is our preferred scenario, with a revision of EU legislations, new principles and the implementation of technical rules by both the ECDC and the EDQM. These organisations proved to be science driven, with the EDQM reaching out to non-EU countries as part of the Council of Europe. Some of these countries use blood-derived products more than new technologies such as recombinant coagulation factors. EURORDIS notices the European Medicines Agency’s role is not mentioned in the inception impact assessment. The distinction between BT derived products and medicines is not always clear, and the revised legislation should clarify precisely when hey are a medicine regulated by EMA, or by other agencies. One important role that needs to be conferred to the ECDC and the EDQM is the conduct of campaigns to invite citizens to donate their blood, plasma tissue, cells. EURORDIS is convinced that more could be done in this respect, relying on the altruism of EU citizens. When support programmes are in place, with volunteers who accompany and encourage donors, managed by organisations that collect blood and plasma such as the Red Cross, then more donors are inclined to donate, and more frequently. Possibly some coordination with international bodies should be envisaged, as done for medicines regulatory agencies with scientific and technical discussions as part of the International Coalition of Medicines Regulatory Authorities (ICMRA) from different world regions. Different methods are used to attract donors for blood, plasma, tissue and cells, and an abundant scientific literature exists, that compare different strategies and approaches, analysing their consequences on the quantities of donations, and also on safety, quality and costs. EURORDIS proposes the European Commission to conduct a meta-analysis of the published literature in this field, for example by commissioning one of the European Health Technology Assessment bodies specialised in monitoring meta-analysis of the scientific literature. Option 1 is not attractive as tensions persisted over the years between the public and the private sector, which could impair the adoption of common rules. EURORDIS believes a consensus could be difficult to obtain between these different actors. Furthermore, as expressed to the European Commission in its letter back in 2009, EURORDIS considers that, in order to ensure highest safety standards, it would be reasonable to separate strictly the role of plasma collector from the role of manufacturer of blood and plasma ‐derived medicinal products, as during periods when fewer donors donate blood manufacturers may be tempted to collect blood from donors they would usually exclude from donation. Option 3 could introduce a too complex legislative process. Technical rules will need to evolve, as this field is highly innovative and new challenges often arise. There is a risk that the legislation will not keep up with new challenges, or that it could block the system if one of the legal measures turns to be counter-productive and no legal change can be made easily.
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Response to Union of Equality: European Disability Rights Strategy

10 Nov 2020

EURORDIS-Rare Diseases Europe is a non-profit alliance of over 900 member organisations from more than 70 countries working together to improve the lives of the 30 million people living with a rare disease in Europe. Our ambition is to see people living with a rare disease integrated in a society that indeed leaves no one behind. Evidence demonstrates that people living with a rare disease and their families continue to face serious every day and social inclusion challenges, to a large extent linked to disability. As such, EURORDIS welcomes the roadmap of the European Commission for the EU Strategy on the Rights of Persons with Disabilities 2021-2030. In order to further strengthen such roadmap and ensure the inclusion of all voices of the disability community in the future EU Strategy, EURORDIS would like to offer inputs and evidence on two key areas: 1) disability assessment; and 2) work-life balance and employment. Both points are in line with the EESC opinion: Shaping the EU agenda for disability rights 2020-2030, points 4.6.1.3 and 4.4.3.4 respectively. In the attached document we detail the rationale behind our key recommendations for the European Strategy on the Rights of Persons with Disabilities 2021-2030 , which include: - Providing guidance to Member States on disability assessment procedures to ensure persons with all types of disability, including persons with rare conditions or multiple impairments, are not overlooked and are provided with adequate levels of disability allowance, social protection schemes, community-services and independent living arrangements. - Providing guidelines for Member States on how to ensure reasonable accommodation for persons with disabilities in the workplace, in order to correctly transpose Article 5 of Directive 2000/78/EC. The guidelines should encourage Member States to entitle persons with disabilities with adequate leave of absence and flexible work arrangements, in line with the provisions offered to parents and carers within the Directive (EU) 2019/1158 on work-life balance. - Providing Member States with the necessary support to ensure the full implementation of Directive (EU) 2019/1158 on work-life balance for parents and carers of persons with disabilities.
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Response to Legislative framework for the governance of common European data spaces

31 Jul 2020

EURORDIS welcomes the opportunity to comment on this proposal for the governance of common European data spaces. The rare disease community is acutely aware of the importance of pooling and sharing data to advance research and improve health outcomes. People living with a rare disease are willing to share their health data to foster research and improve healthcare, as long as they remain in control of their data throughout the data cycle and their choices and needs for information regarding the use of their data are respected. There are certain particular aspects that refer to three of the four thematic clusters identified in the Roadmap that we would like to comment on: I. Unlock value from data held by public sector bodies the use of which is conditional on the respect of rights of others. An “open data” approach to enable the re-use of health data stored in public databases, requires a sound understanding of the expectations and incentives for citizens to share their health data for secondary purposes. Although we understand the potential benefits of having a legal instrument to govern cross-domain aspects, we believe that a domain-specific framework will be required for the health sector. For example, in the case of people living with a rare disease, beyond ensuring full respect of data protection rights, the main incentives to participate in such initiatives are the possibility to discuss and learn information about the disease and the research project. Therefore, any “open data” initiative for health data should allocate adequate financial resources to set up mechanisms to inform about the purpose and outcomes of data sharing initiatives on a regular basis. II. Support the use of data that individuals or companies voluntarily contribute to the wider public good 1. The differences between data altruism and data sharing are unclear. We welcome the fact that the planned legislative framework will support the use of data voluntarily shared not only by individuals, but also by companies that want to contribute data to the wider public good. However further clarification of the term “data altruism”, widely agreed by all relevant stakeholders, and how this concept differs from “data sharing” is necessary before moving forward with any health data altruism initiative. The distinction is relevant in the case of health data to understand if we need a different ethical and legal framework to govern health data altruism that is different from the principles and rules that apply to health data sharing. 2. Health data "altruism" must be organised in a way that is safe and ethical. If such an opt-in system is put in place, it should be accompanied by inclusive governance structures, dynamic systems to restore data subjects’ autonomy, education to enable informed choices for patients to share or not to share their health-related data, transparency on the use of the data but also the insight derived from the data and adequate incentives. There should also be proportionate safeguards in place to protect the data subjects’ interests and redress mechanisms. Health data altruism at scale will require clear accountability and some kind of redress mechanism where people would not be required people to prove that a specific action or omission by a specific data user caused the harm, but just show that the harm experienced is plausibly connected to the data use. III. Lower transactions costs in data sharing. In the health sector, rather than commercial intermediaries, we believe that patient organisations and other non-for profit intermediaries could have a greater impact if they would play an intermediary role. Patients’ level of trust in not-for-profit stakeholders (medical doctors and other health professionals, researchers from non-profit organisations, patient organisations) to handle and use their health information is considerably higher than trust in for profit stakeholders (see evidence here: https://tinyurl.com/wyxa8l6).
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Rare disease group EURORDIS urges patient-centred EU pharmaceutical reforms

8 Jul 2020
Topic — The European Commission is consulting on a strategy for affordable, safe medicines.
Message — EURORDIS wants to establish a European Table of Negotiations for medicines. They seek a patient-centred strategy across the whole product life-cycle. The group also proposes a fund to track how medicines work after approval.123
Why — This approach would help rare disease patients access innovative treatments sustainably.45
Impact — Pharmaceutical companies could face reduced profits due to cost-based pricing models.6
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Meeting with Olympia Neocleous (Cabinet of Commissioner Stella Kyriakides)

14 Feb 2020 · Health Technology Assessment, Rare Diseases

Meeting with Stella Kyriakides (Commissioner) and

20 Jan 2020 · Discussion on cancer

Meeting with Anne Bucher (Director-General Health and Food Safety)

21 Oct 2019 · the Regulation for a European Cooperation on Health Technology Assessment.

Meeting with Anne Bucher (Director-General Health and Food Safety)

14 Feb 2019 · courtesy visit, presentation of activities

Response to European Electronic Health Record (EHR) Exchange Format

20 Dec 2018

EURORDIS – Rare Diseases Europe representing the rare disease patient community welcomes the announcement by the European Commission of the adoption of a Commission Recommendation to establish a Format for a European Electronic Health Record (EHR) Exchange and appreciates the opportunity to give feedback on the Roadmap. Having an EHR data exchange format has the potential to improve the quality and safety of cross-border healthcare services, accelerate research and, ultimately, contribute to better health outcomes and patient well-being, specifically for people with rare diseases. However, the actions outlined in the Roadmap will not achieve by themselves this wider impact if other factors are not in place. These include addressing the bottlenecks that are preventing patients’ access to cross-border healthcare, such as the lack of clarity regarding prior-authorisation requirements, the lack of awareness among healthcare providers and patients of their rights, or the financial burden that the current reimbursement procedure imposes on patients who seek cross-border healthcare services. The ability to share and pool data is particularly important in the rare diseases field and in all fields requiring a specific concentration of expertise: only through data aggregation can one attain a critical mass, which generates valuable knowledge and drives forwards improvements in research and healthcare. A year ago, the European Reference Networks (ERNs) were launched to pool expertise on rare and complex conditions as well as data and resources for medical but also for research purposes. Data sharing is at the foundation of the successful functioning of ERNs. This EU cross-border healthcare initiative is still early in its development, but ERNs have already defined a basic clinical data set for the provision of cross-border virtual consultations among experts on the most complex cases. Each Network is now developing an extended data set, relevant to a specific group of diseases and they are also working on data quality and validation. Certainly, the work done by the Networks in this domain must be re-used and incorporated into the Recommendation and could serve as the basis for the definition of a RD patient summary. Equally important is to consider the work developed by other data-related initiatives in the rare disease field at EU level, such as the Joint Research Centre’s set of common data elements for rare disease registration developed for the European Platform for Rare Disease. Also national initiatives, like the French minimum data set for rare diseases. EURORDIS welcomes the Commission’s intent to facilitate with this Recommendation the flow of health data across borders to increase its availability for research purposes, rather than limiting the scope of the initiative to the exchange of EHR for medical purposes. Indeed, the scope of the Recommendation should be extended to fully address the opportunities of secondary use of the data stored in EHRs. Although, the main purpose of EHRs is to support care delivery we should not oversee the potential that these EHRs databases have to support other purposes thanks to big data analytics and artificial intelligence. We welcome that the Commission will propose new ways of working between the Member States, relevant stakeholders and the European Commission. However, we invite the Commission to think more broadly about how to organise such a collaborative working mechanism and expand its scope beyond the development and implementation of the EHR exchange data format. The discussion on EHRs standards and data formats should not be happening in isolation. Developing a European data ecosystem requires setting up a strong and dynamic coordination mechanism for broad and open discussion of all aspects of digital health and health data sharing. Detailed comments are enclosed.
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Meeting with Vytenis Andriukaitis (Commissioner) and

15 May 2018 · HTA

Response to EU cooperation on Health Technology Assessment

30 Mar 2018

Transparency and Health Technology Assessment cooperation as proposed by the Regulation are the only real antidote to secrecy and political games Why EURORDIS supports the proposal for a Regulation on Health Technology Assessment (HTA) Cooperation in Europe 30 March 2018, Paris – EURORDIS-Rare Diseases Europe, an alliance of 798 rare disease patient organisations from 69 countries, calls on the European Parliament and Member States to adopt the European Commission’s (EC) proposal for a future European cooperation on Health Technology Assessment (HTA). - Only a permanent structure can guarantee long-term cooperation and organise sharing of scientific expertise and methods across all EU HTA agencies. - Joint HTA reports represent a major progress towards high quality, transparent and timely information necessary for the subsequent coverage/ reimbursement decisions made on a national level. - With this proposal, patients will be better equipped to understand the scientific rational behind the assessment of the added value of health technologies, everywhere in the EU and for the first time in all Member States. - In 2018, 24 years after the first EU project on HTA, it is time to create a more efficient process, with no replication in 28 Member States of the assessment of the added value when diseases and patients are the same across the EU. - This is particularly needed when diseases are rare or technologies complex. The EC proposal calls for the mandatory uptake of joint HTA assessments. EURORDIS welcomes this as the only guarantee that the future cooperation will achieve its goals (no duplication, high quality assessment, timely production of the reports). The Statement (to be downloaded): 1. Details why patients need decision-making to be based on scientific and medical evidence, transparent processes and facts, everywhere in the EU 2. Reminds that Solidarity between Member States is a funding principle of the European Union 3. Explains that HTA cooperation on a voluntary basis has its limits 4. Comments on fairness, equity, high scientific standards and efficiency in the decision-making process, in the interest of all patients, as provided for by the proposal 5. Welcomes the synergie with the European policy to create and develop European Reference Networks
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Meeting with Vytenis Andriukaitis (Commissioner) and

28 Feb 2018 · European Reference Networks

Meeting with Vytenis Andriukaitis (Commissioner) and

24 Feb 2016 · Improving access to orphan medicinal products and other rare disease therapies; European Reference Networks

Meeting with Annika Nowak (Cabinet of Commissioner Vytenis Andriukaitis), Paula Duarte Gaspar (Cabinet of Commissioner Vytenis Andriukaitis)

9 Dec 2015 · European Reference Networks in the field of rare diseases; improving access to orphan drugs; platform on rare disease registration

Meeting with Xavier Prats Monné (Director-General Health and Food Safety)

8 Oct 2015 · Patient perspective and involvement

Meeting with Vytenis Andriukaitis (Commissioner) and

24 Feb 2015 · Action on rare diseases, Bridging Inequalities & Giving Patients with Rare Diseases same type of access to treatment and care