The 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 harmonization.
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 hemophilia, 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 standardized 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 standardized reports that all EU countries incorporate into their national HTA processes. For hemophilia treatments, this harmonized approach could significantly reduce evaluation timelines and ensure consistent access to innovative therapies across Europe.
Patient and Patient Organization Involvement
The regulation emphasizes meaningful patient involvement throughout the evaluation process, recognizing 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 organizations.
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 organizations, particularly those representing rare diseases like hemophilia, 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 recognizing patient expertise in healthcare decision-making. For the hemophilia community, where patient experiences and needs are highly specific, this formal recognition of patient input could lead to more patient-centered assessments and ultimately better treatment access across Europe.
The Stakeholder Network, established by the regulation, provides a formal platform for patient organization engagement. The European Haemophilia Consortium (EHC), as a network member, brings specialized 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 their domain.
