On January 21 2025, the Council of the European Union adopted the European Health Data Space (EHDS).
Background
This groundbreaking initiative, put forward by the Commission in May 2022, has two main aims:
- to place citizens at the centre of their healthcare, granting them full control over their data, to achieve better healthcare across the EU;
- to allow the use of health data for research and public health purposes, under strict conditions.
Thanks to the new rules, citizens will benefit from immediate and simple access to their digital health data when in the EU, regardless of their location. For instance, when a patient seeks healthcare abroad, healthcare professionals will be able, when necessary, to access key information from the patient’s home Member State. This will improve evidence-based decision-making, reduce repetition of tests and examinations and enhance patient care.
The EHDS also establishes a strong legal framework for the re-use of health data for research, innovation and public health purposes in full compliance with strict EU data security and access criteria, fundamental rights and cybersecurity rules. The data will help develop life-saving treatments and personalised medicines and improve European crisis preparedness.
Next Steps
The new European Health Data Space regulation will be published in the Official Journal. It will then become applicable in different stages according to the use case and data type.
Source: European Commission
HTA Regulation: A New Framework for Europe
The European Health Technology Assessment (HTA) Regulation 2021/2282 represents a fundamental shift in how medical technologies are evaluated across the European Union. Implemented in January 2025, this groundbreaking legislation establishes a permanent framework for joint assessments, marking a significant advancement in European healthcare policy harmonisation.
The regulation introduces a progressive implementation timeline: beginning with cancer treatments and advanced therapy medicinal products in 2025, extending to orphan medicines in 2027, and encompassing other new medicines by 2028. This phased approach allows for systematic adaptation while maintaining high-quality standards. For rare diseases like haemophilia, where evidence generation can be challenging, this coordinated approach is particularly significant.
The regulation’s core objective is to streamline evaluation processes while preserving member states’ authority over pricing and reimbursement decisions. This balance ensures national healthcare systems retain control over resource allocation while benefiting from shared expertise and standardised clinical evidence assessment.
Joint Scientific Consultations and Joint Clinical Assessments
The regulation introduces two key procedures: Joint Scientific Consultations (JSC) and Joint Clinical Assessments (JCA). Each serves distinct but complementary purposes in the evaluation process.
JSCs provide early dialogue between technology developers and HTA authorities before clinical trials commence. For bleeding disorders treatments, where trial design can be complex due to the rare nature of the disease, this early consultation is invaluable. Companies receive coordinated guidance on study design, comparators, endpoints, and evidence requirements, helping ensure their clinical development programs will generate appropriate evidence across all EU member states.
JCAs evaluate the relative clinical effectiveness of new health technologies compared to existing treatments. Multidisciplinary expert teams from member states collaboratively assess clinical evidence submitted by manufacturers. This process results in standardised reports that all EU countries incorporate into their national HTA processes. For haemophilia treatments, this harmonised approach could significantly reduce evaluation timelines and ensure consistent access to innovative therapies across Europe.
Patient and Patient Organisation Involvement
The regulation emphasises meaningful patient involvement throughout the evaluation process, recognising the unique insights patients bring to health technology assessment. This involvement is structured differently for JCAs and JSCs, with distinct roles for individual patients and patient organisations.
In JCAs, patient involvement occurs at multiple stages. Individual patients participate during the scoping phase, providing input on assessment proposals, and during the assessment phase, reviewing draft reports. Patient organisations, particularly those representing rare diseases like haemophilia, play a distinct role by providing collective insights on therapeutic areas without accessing confidential information about specific interventions.
For JSCs, while patient involvement is less explicitly detailed in the regulations, patients can participate in consultations about development plans, particularly regarding clinical study design. Their advisory role helps ensure that evidence generation will meet future JCA requirements and reflect real patient needs.
This structured patient involvement represents a significant advancement in recognising patient expertise in healthcare decision-making. For the haemophilia community, where patient experiences and needs are highly specific, this formal recognition of patient input could lead to more patient-centred assessments and ultimately better treatment access across Europe.
The Stakeholder Network, established by the regulation, provides a formal platform for patient organisation engagement. The European Haemophilia Consortium (EHC), as a network member, brings specialised expertise in rare bleeding disorders to the HTA process. Following Article 29, EHC must declare its membership, funding sources, and potential conflicts of interest. Through the network, EHC participates in annual meetings with the Coordination Group, contributes to discussions on joint work outputs, and provides expert input on assessment scopes within its domain.
On Monday, May 27, 2024, the European Council adopted new rules on substances of human origin (SoHO).
The regulation on substances of human origin (SoHO) will ensure better protection for donors and recipients, as well as for children born following medically assisted reproduction. The new rules aim to strengthen the existing legal framework while also increasing flexibility in order to keep up with scientific and technical developments.
Voluntary and unpaid donations
Member states will be able to compensate living SoHO donors, following the principle of voluntary and unpaid donation and based on transparent criteria, including fixed allowances or through non-financial forms of compensation. The conditions for such compensation will be established in national legislation.
Promotion and publicity activities in support of SoHO donations (for example, campaign billboards or posters, television, newspaper or magazine advertisements) must not refer to compensation. National authorities will therefore have to ensure that compensation is not used as an incentive to recruit donors.
Safeguarding supply
EU countries will establish and regularly update “national SoHO emergency plans”, setting out measures to ensure the security of supply for critical substances.
Next steps
The SoHO Regulation will come into force three years after its publication in the Official Journal of the European Union. The new rules will facilitate the cross-border exchange of SoHO in the EU, improve patients’ access to necessary treatments in all Member States, and promote greater cooperation between national public health authorities.
Useful links
Council of the EU Press Release – here.
Text of the Regulation – here.
EU Health Policy background information – here.
The European Commission released its pharma legislation proposal in 2023 to make access to medicines more secure and affordable, encourage innovation, and reduce the gaps.
One of the key changes it proposed was to shave two years off the amount of time new branded medicines have to themselves on the market before rivals launch, down from the current eight years to six.
The newly voted in the EP text bumps that up to seven and a half years of regulatory protection, with a cap of one extra year for those that qualify.
The Parliament’s directive proposal also includes new incentives not listed in the Commission’s original text: six more months of data protection for medicines in which “significant development, including clinical and preclinical” was carried out in the EU; or for where development was done in collaboration with university hospitals, centres of excellence, or bioclusters.
Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (Text with EEA relevance).