Lobbying by European Hematology Association

European Hematology Association

EHA

The European Hematology Association (EHA) is a non-governmental and not-for-profit membership organization that is guided by its mission to promote excellence in patient care, research, and education in hematology.

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ID: 91666778136-14

Lobbying Activity

Response to Biotech Act

10 Jun 2025

The European Hematology Association (EHA) is the leading organization representing hematology professionals in Europe. As a not-for-profit, public-benefit association with over 10,000 members and an annual congress attracting around 18,000 stakeholders, EHA supports career development, research, harmonized education, and advocates for equitable access to innovative therapies for patients with hematological diseases across Europe. EHA welcomes the Commissions announcement on the Biotech Act as timely and essential. Biotechnological products offer transformative potential for diagnostics and treatments, improving patient outcomes. From precision diagnostics to advanced therapies, biotech is reshaping healthcare. However, timely access is critical. The Biotech Act must prioritize patients, ensuring equitable access to high-quality, safe, and effective biotechnologies across the EU. A simplified, harmonized regulatory framework is vital for the sectors growth. Current administrative burdens and national discrepancies hinder timely innovation. A streamlined, predictable, science-based EU-wide framework will accelerate biotech development and market entry, benefiting patients and healthcare systems. Over the past decade, medical and life sciences have evolved dramaticallyfrom CAR-T cell therapies to AI in diagnostics. The Biotech Act must support and adapt to such innovation. A future-proof regulatory framework, informed by scientific progress, will foster long-term investment, speed up treatment development, and benefit all stakeholders, especially patients. Public investment in biotech must yield public benefit. EHA calls for EU and national funding mechanisms that ensure transparency, affordability, and reinvestment in public health. Conditionalities should guarantee that publicly funded innovations are accessible and support sustainable healthcare. EHA strongly supports funding transparency. Manufacturers should disclose the full funding history of biotech products, including those acquired, regardless of origin. This transparency will aid Member States in negotiating fair prices and ensure public and non-profit investments are reflected in pricing and access strategies. Public investment should also promote genuine competition. Academic institutions and SMEs must receive support to contribute meaningfully to the sector. Legislation must address disparities in biotech access across Member States. EHA urges the inclusion of mechanisms to improve access, such as joint public procurement, as proposed in the Critical Medicines Act. Under-resourced health systems require targeted support. The Biotech Acts success should be measured by its impact on health outcomes. EHA recommends embedding health impact assessments in the legislative framework, with emphasis on patient-reported outcomes. EHA also proposes establishing a stakeholder forum, as seen in the HTA Regulation and European Health Data Space. This forum should include diverse stakeholdershealth professionals, patientsand enable regular, transparent dialogue. It could co-develop Key Performance Indicators (KPIs) to monitor implementation and ensure accountability. Governance structures at EU and national levels should also involve relevant health stakeholders. Finally, the Biotech Act must align with existing legislation, including the European Health Data Space (EHDS) and Medical Devices Regulations (IVDR and MDR). The Commissions proposal should address these interactions to ensure clarity and legal certainty.

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Response to Health technology assessment – Joint scientific consultations on medicinal products for human use

26 Oct 2024

The European Hematology Association (EHA) welcomes the European Commissions draft Implementing Act laying down procedural rules for the Joint Scientific Consultations (JSC) on medicinal products for human use under the HTA Regulation. EHA proposes stronger emphasis on the following key elements: Alignment and synergy with JCA To meet the aim of JSC, which as described in recital 2 is to facilitate the process of preparing joint clinical assessments for medicinal products, optimal alignment and synergy between the JSC and JCA is essential. This requires the coordinated finetuning and sequential complementarity of timelines and processes, as well as maximum convergence in evidence criteria. From the clinical perspective, it is important that there is room for adaptation of evidence requirements in response to shifts in standard of care, in case such shifts occur between the start of JSC and the start of JCA. Involvement of stakeholder organizations The European learned medical societies, such as EHA, can help identify and engage the appropriate experts across Europe, including and crucially in rare or ultra-rare diseases with often only a small number of experts available across the continent. EHA and other medical societies play a key role in ensuring that individual expertise is grounded in collective expertise, through their highly specialized clinical and scientific expert networks and development of consensus and evidence-based clinical practice guidelines. In line with our previously stated position that the involvement of the medical learned societies and patient organizations should be structural, timely and substantial, we propose the following changes to the draft text: - Art. 5.2: For the selection of individual experts, the HTA secretariat may consult one or more of the following sources (eg. members of the Stakeholder Network or the ERNs). This should be replaced by less tentative language: the HTA secretariat shall consult one or more of the following sources, as relevant and appropriate. - Art. 11.1: Here, similarly, the may should be replaced by shall: the HTA secretariat shall seek input on the disease and therapeutic area relevant for the medicinal product from patient organisations, healthcare professional organisations or clinical and learned societies via the members of the stakeholder network. Involvement of clinical experts The involvement of the best available clinical experts is crucial for high-quality, credible and impactful JSC and JCA. Given that highly-specialized experts are often few in numbers (especially in ATMPs and rare and ultra-rare diseases) and high in demand, it is of great importance that: - involvement opportunities are timely and substantial; - timelines are realistic and communicated as early as possible; - medical societies and patient organizations are given sufficient advantage notice of upcoming requests to help with the identification and engagement of experts; - conflicting or competing interests are assessed and managed in a transparent and pragmatic manner, taking into account the specific context, needs, and availability of expertise.

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Response to Health technology assessment – Cooperation with the European Medicines Agency

19 Jul 2024

The European Hematology Association (EHA) welcomes the draft Implementing Act laying down procedural rules for the exchange of information between the HTA Coordination Group, the European Commission and the European Medicines Agency. The timely and effective exchange of information with relevance for the Joint Clinical Assessments (JCA) and Joint Scientific Consultations (JSC) will be essential for implementation of the Regulation and contribute to achieving maximum synergy and complementarity between EMA and EU HTA. In particular, EHA: supports the exchange of information related to the identification of patients, clinicians and other individual experts to be involved in JSC and JCA; considers it important that the exchange of information between HTA Secretariat, HTA Coordination Group and EMA does not delay the sharing of timely information with stakeholder organizations (asked to assist with expert identification) and individual experts (asked to contribute to JSC and JCA); welcomes the explicit statement (recital 6) that experts' involvement in EMA evaluations does not preclude their involvement in JSC or JCA, and vice versa, as long as transparency of such engagement is ensured. Crucially, EHA urges the HTACG, Commission and EMA to agree on a common, pragmatic, transparent, needs-based approach to assessing Conflicts of Interest (CoI). As we have argued in our response to the consultation on CoI: Adoption of EMA procedures and templates for Declaration of Interest (DoI) would avoid duplication and help limit the administrative burden on clinical experts getting involved in JCA or JSC, in the face of short timelines and clinical and teaching duties. The quality and credibility of joint assessments and by extension, the uptake and impact on access decisions at the national level will depend to a large degree on the ability to involve the best available experts. This requires pragmatic and transparent management of competing interests, rather than rigid application of a free of CoI approach that would inevitably lead to the exclusion of vital expertise, particularly in (ultra) rare disease areas. In this context, we consider it imperative that the recent court rulings and the subsequent Commission Implementing Decision regarding EMAs handling of CoI do not lead to a move away from the pragmatic and balanced approach to COI developed by EMA over the years. To ensure access to vital expertise, the HTAR Implementing Acts should provide for effective and transparent management of competing interests by the Coordination Group and the Commission, in the interest of high-quality assessments and access decisions that serve patient, clinical and public health needs.

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

26 Jun 2024

The European Hematology Association (EHA) welcomes the European Commissions draft Implementing Act on the assessment and management of Conflicts of Interest (CoI) in the joint work to be performed under the EUs HTA Regulation. Successful implementation of the HTAR, to which EHA is strongly committed, will depend to a large degree on the involvement of the best available experts clinicians as well as patients. Their input is indispensable for ensuring that the joint assessment reports will have the quality and credibility needed for achieving what joint HTA is meant to do: improving and harmonizing Member States decision making on the cost-effectiveness, pricing and reimbursement of novel therapies, for the benefit of Europes patients and health systems. The formulation of clear CoI rules is essential for ensuring that the involvement of experts in EU HTA is based on principles of transparency, independence and impartiality. Overly strict interpretation and application of COI policies is however one of the major potential hurdles to the (effective) involvement of experts. This is especially the case in rare diseases, such as certain types of haemophilia where the number of key opinion leaders (KOLs) can be as low as 1-2 persons, who are, inevitably and necessarily, involved in advising stakeholders such as pharmaceutical companies or payers. EHA therefore calls for: 1. Clear and transparent principles and procedures for declaring and assessing CoI. 2. Pragmatic interpretation and application of CoI rules, on a case-by-case basis. Both the principles and their interpretation and application should be based on the recognition that avoiding CoI is neither realistic nor desirable. Rather than attempting to ban them, conflicting or competing interests should be assessed, weighed and managed in a transparent manner, taking into account the specific context, needs, and availability of expertise. 3. Structural and mandatory consultation of medical societies on expert selection. European medical societies such as EHA can help identify and engage the appropriate experts across Europe, including and crucially in rare or ultra-rare diseases with often only a small number of experts available across the continent. EHA and other medical societies play a key role in ensuring that individual expertise is grounded in collective expertise, through their clinical and scientific expert networks and development of consensus and evidence-based clinical practice guidelines and (through tools such as the MCBS:H) assessment of the clinical benefit of new drugs. 4. Alignment and synchronization of policies, methods and procedures for the declaration and assessment of CoI between EMA and EU HTA. EMA has well-established, time-tested approaches to expert involvement and CoI management. Adoption of EMA templates for Declaration of Interest (DoI) would avoid unnecessary duplication and helps to limit the administrative burden on clinical experts, many of whom will struggle to free themselves up amid short timelines and heavy clinical and teaching workloads. In this context, EHA and fellow medical societies (see also the opinion of the BioMed Alliance) are very concerned about the March 14 decision by the EUs Court of Justice , which ruled that EMA must ensure that the experts it consults do not have a conflict of interest. We call on all those formulating, interpreting and applying EU legislation with impact on how scientific advice, regulatory evaluations and clinical assessments are performed, to ensure that both EMA and EU HTA can benefit from the best available expertise. EU HTA is designed to improve affordable and equitable patient access to the best available health technologies. This goal will only be served if the best available clinical experts and patient experts are included rather than excluded, based on pragmatic and transparent management of competing interests.

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Response to Health technology assessment - Joint clinical assessments of medicinal products

2 Apr 2024

The European Hematology Association (EHA) welcomes the emphasis throughout the draft Implementing Act on Joint Clinical Assessments on involvement of clinicians, patients and other relevant experts. Getting such involvement right will be crucial to ensuring that EU HTA contributes to better, harmonized, evidence-based decision making on access to innovative therapies, for the benefit of patients and public health systems. EHAs recommendations focus on effective involvement of clinical experts, the role of medical societies, and what is needed for high-quality, evidence-based JCA. In summary: 1) The IA should provide more explicitly for involvement of clinical experts that is structural, timely, and substantial. To ensure that the assessment scope proposal is evidence-based, seeking input from clinical experts at the early drafting stage should be mandatory. 2) Consultation of medical societies should not be optional, but systematic, early, and substantial. The European medical societies can help identify the best available clinical experts across Europe and ensure that individual expertise is grounded in the collective expert networks that establish the standard of care and develop evidence-based guidelines. This is particularly important given the high pace of scientific and therapeutic innovation in areas such as hematology, and in view of the discrepancies in standards and access across Member States. Through their close links with national associations, the European medical societies can also contribute to connecting and aligning HTA work at the European and national levels. The IA should specify that European medical societies must be consulted on selection of experts and may proactively share new evidence or information on specific unmet needs in different Member States. 3) Effective expert involvement requires pragmatic management of Conflict of Interest, to be specified in the dedicated AI on COI, as well as a balanced and realistic approach to timelines. There must be room for flexibility when requested by stakeholders such as medical societies. Feasibility of timelines should be regularly assessed together with stakeholders. Provisions may be included for incentivising hospital and university employers to better facilitate HTA work by clinicians. 4) Balance is also needed regarding confidentiality requests for information deemed commercially sensitive by developers. From a scientific point of view, we support maximum transparency in all documents, especially on adverse events but also on patient-reported outcomes. We attach a more elaborate commentary on the draft IA on JCA with specific suggestions for enhancements.

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Response to Evaluation and revision of the general pharmaceutical legislation

8 Nov 2023

The European Hematology Association (EHA) is the leading representative of hematology and hematology professionals in Europe. A not-for-profit, public-benefit organization with nearly 7,000 members, EHA supports career development and research, develops and harmonizes hematology education, and advocates for enhanced, affordable and equitable access to innovative therapies for patients with hematological disease across Europe. As part of its advocacy agenda, EHA focuses specifically on the access challenges around precision diagnostics, personalized medicine, rare hematological diseases, and the need for more innovative and less bureaucratic clinical trials. EHA actively engages with regulators and stakeholders to find ways of harmonizing and improving evidence generation in support of decision making on clinical trials, market authorization, treatment optimization, health technology assessment, pricing and reimbursement. Our official position on the Proposal for a regulation - COM(2023)193 can be found enclosed.

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Response to Evaluation and revision of the general pharmaceutical legislation

8 Nov 2023

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

8 Sept 2022

The European Hematology Association (EHA) warmly welcomes the legislative proposal for an EU SoHO Regulation which represents a big step forward for protecting patient and donor safety, improving access to SoHOs and innovative SoHO-derived therapies, ensuring sustainability of supply, facilitating innovation, and for increasing efficiency and coherence of regulation and oversight. During the revision of the blood, tissues and cells legislation EHA has consistently flagged the need for (1) strengthening harmonization and supervision to ensure patient and donor safety; (2) a more flexible legislative network that could adapt to and facilitate scientific and medical innovation; (3) harmonized and efficient evidence-based implementation with a larger role for experts and expert bodies; and (4) concerted action to increase and sustain plasma supplies. We therefore particularly welcome the Regulation’s emphasis and provisions on the following aspects: Ad (1) safety and quality: - Common standards and guidelines, set mostly by expert bodies (ECDC, EDQM) - Coverage of (almost) all SoHOs applied to humans - Improved safety reporting - Training and exchange of Competent Authorities’ personnel - Possibility to introduce binding rules via Delegated Acts if needed for safety/quality Ad (2) facilitating innovation: - Risk-based authorization and evidence-based benefit assessment of SoHOs processed or applied in new ways - Creation of a registry of authorizations to reduce administrative burdens Ad (3) implementation: - Better linkage and complementarity with other EU legislation (pharmaceutical, clinical trials, devices/IVD) - Creation of a SoHO Coordination Board for more efficient and harmonized implementation and oversight (including advice on applicability of the SoHO Regulation in borderline cases) - Common platform for data sharing (EU SoHO Platform) - Clear division of roles/responsibilities between EU (overall regulatory and data framework, coordination, resilience, controls/audits, funding of collaborative work), expert bodies (esp. ECDC, EDQM – technical standards and guidelines) and Member States/NCAs (supervision of SoHO entities, ethics framework). Ad (4) access to and supply of plasma: - Strengthened oversight - Improved crisis preparedness, via central EU requirements for emergency response, supply monitoring, and mitigation of shortages More emphasis and/or stronger wording is needed to ensure that: (a) Involvement of experts/professionals and interaction with medical societies including EHA via the proposed expert group should be timely, structural and substantial (b) The EU SoHO Platform is set up in a way that serves data sharing and use by professionals as well as the interests of patients and donors, in addition to serving the needs of regulators, companies and institutions (c) With a view to secure and sustainable supply, especially of plasma: - Both healthcare professionals and patients should be involved in discussions on compensation of donors and their views must be taken into account in decision making on forms of compensation - In addition to mandating better monitoring and notification of shortages, Member States should be encouraged explicitly to take active measures to improve security of supply and agree on proactive, EU-level planning to reduce dependency on third countries - Measures to improve the collection of and access to plasma should be guided by the work of and involve the members of the SUPPLY consortium, led by the European Blood Alliance (EBA) and including the European Hematology Association (EHA) (d) The multiple references to “SoHOs prepared or used in new ways” are reworded to make clear that ‘new ways’ should imply ‘meaningful innovation’, especially in the section on Impact Assessments of the Explanatory Memorandum which states that “a clear and (risk-) proportionate pathway will facilitate access to SoHOs prepared or used in new ways”

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Response to Evaluation and revision of the general pharmaceutical legislation

27 Apr 2021

Hematology is at the forefront of medical and technological advances which have brought truly patient-centered, personalized medicine within reach. Clinicians, researchers, policymakers and regulators at the EU and national level, research funders and the pharmaceutical industry have a shared responsibility to ensure that innovative treatments are affordable and available for all patients in Europe. In the context of the EU’s proposed Pharmaceuticals Strategy, the European Hematology Association (EHA): - Welcomes the emphasis on affordable access, on unmet needs and on moving towards for adaptive, harmonized, bureaucracy-light regulatory frameworks. - Views as essential the strategy’s comprehensive lifecycle, interdisciplinary, multi-stakeholder approach. - Strongly agrees that the strategy must be patient-centered and strive for a better balance between clinical, public health and industry needs. Improving the accessibility, quality and safety of medicines should go hand in hand with boosting the sector’s global competitiveness. More in particular, EHA: - Supports the focus on the unmet needs of rare disease patients, with an important role for the ERNs. Multi-stakeholder partnership Rare Cancers Europe should be involved in all policy-making on rare cancers. Rare non-malignancies should not be forgotten. - Supports joint EU-level clinical assessments, as foreseen by the proposed HTA Regulation. Joint clinical assessments will contribute to improved access and affordability of therapies with proven added value for patients. They will enhance efficiency, reduce cost, ensure better allocation of public health funds, and improve alignment between national-level decision makers on pricing and reimbursement. - Welcomes the recognition that regulatory frameworks – for market authorization but importantly also for clinical trials, post-licensing evidence generation and HTA – need to be simplified, streamlined and more adaptive to scientific and technological innovation. - Welcomes the much-needed emphasis on affordable and equal access. To be successful, the temptation needs to be resisted to narrowly focus on specific elements of the broad and complex access challenges, such as generics and biosimilars or supply issues. Only a holistic approach that looks at the whole lifecycle and achieves a balance between the interests of developers, funders, patients and public health systems can be successful. EHA calls for: - An integral approach that looks not only at medicines but also at diagnostics, advanced therapies, medical devices and combination therapies. - Dedicated and harmonized regulatory frameworks that enable the uptake of precision diagnostics and personalized therapies. Specifically, EU-wide access to high-quality, reproducible, affordable precision diagnostics must be secured; a new group of ‘Personalized Medicine Trials’ centered around patients with an unmet medical need must be enabled; and evidence generation and assessment must be aligned across clinical trials, market approval, HTA and cost-benefit assessments. - More attention for the long-term sustainability of ERNs and their integration into national healthcare systems. - Sufficient attention for non-malignant (blood) diseases amid the hausse of anti-cancer policies. - Last but not least, the involvement of patients and healthcare professionals in the revision and implementation of the legislation, which is crucial for ensuring that the EU’s pharmaceutical strategy is truly focused on the (unmet) needs of patient and public health systems.

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