The European Medicines Agency (EMA) has launched a transformative Health Technology Assessment regulation (HTAR) that promises to reshape how new medical technologies are evaluated across the European Union. The regulation, which took effect on January 12, initially focuses on cancer therapies and advanced therapy medicinal products, marking a significant shift toward centralized assessment procedures.
Streamlined Assessment Process
The cornerstone of the new regulation is the introduction of Joint Clinical Assessments (JCAs), conducted by the HTA Coordination Group. These assessments will evaluate the clinical effectiveness and safety of new therapies compared to existing technologies. The EMA's executive director, Emer Cooke, emphasized that this collaboration across decision-makers will strengthen efforts to improve medicine access throughout the EU.
The regulation's scope will expand progressively, incorporating orphan medicinal products by January 2028 and extending to all medicinal products by 2030. This phased implementation represents a strategic approach to harmonizing assessment procedures across the EU's diverse healthcare landscape.
Impact on Market Access and Industry Operations
The new framework aims to address the historical fragmentation in HTA processes across EU Member States. Previously, countries like France and Germany operated with independent regulatory organizations, while others, such as Sweden's Dental and Pharmaceutical Benefits Agency (TLV), functioned as government agencies. This diversity in assessment approaches often led to varying timelines and requirements.
Dr. Gunnar Sachs, a partner at Clifford Chance, suggests that the harmonized assessments could significantly benefit the EU pharmaceutical industry through accelerated timelines. Stephen Reese, co-head of Healthcare and Life Sciences at Clifford Chance's London office, adds that the centralized process could deliver substantial cost savings for pharmaceutical companies.
Industry Concerns and Implementation Challenges
Despite the potential benefits, the industry has raised several concerns about the new regulation's implementation. The European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) has highlighted the limited availability of regulatory guidance and consultation opportunities. Of particular concern is the restricted number of joint scientific consultations scheduled for medicinal products, with only five to seven slots initially planned.
The regulation requires developers to address nine specific domains in their HTA preparations, including both clinical and non-clinical aspects. Industry stakeholders express uncertainty about compliance with "soft requirements" related to reimbursement standards across different Member States, an area where guidance remains limited.
Future Outlook and Market Implications
The centralized assessment process may encourage pharmaceutical companies to enter smaller European markets that were previously overlooked, potentially expanding access to innovative therapies across the EU. However, questions remain about Member States' adherence to the harmonized approach and the practical implementation of this compromise solution.
The European Federation of Pharmaceutical Industries and Associations (EFPIA) has acknowledged positive aspects of the regulation, such as the inclusion of patient-reported outcomes and flexibility in clinical trial outcome measurements. However, they have also voiced concerns about restrictions on the number of clinical trial outcomes that Member States can request from developers.
