EU HTA Regulation 2025: Preparing for Joint Clinical Assessments

The EU Health Technology Assessment (HTA) Regulation 2021/2282 enters its operational phase in 2025, fundamentally transforming how new medicines and medical devices are assessed across the European Union. This landmark regulation introduces mandatory joint clinical assessments (JCAs) and joint scientific consultations (JSCs), creating a harmonized approach to HTA across all 27 member states.

Understanding the EU HTA Regulation

Regulation Timeline and Scope

January 2025: Full implementation of joint clinical assessments for:

Oncology medicines (all cancer indications)
Advanced therapy medicinal products (ATMPs)
Orphan medicinal products

2028: Extension to all new medicines with new active substances

2030: Inclusion of certain high-risk medical devices (Class III implantables)

Key Regulatory Components

The regulation establishes three core processes:

Joint Clinical Assessments (JCA): Collaborative evaluation of clinical effectiveness and safety
Joint Scientific Consultations (JSC): Pre-submission advice on evidence generation
Emerging Health Technologies Identification: Early horizon scanning

Joint Clinical Assessment Process

JCA Scope and Deliverables

JCAs focus exclusively on clinical domains:

Clinical effectiveness: Comparative efficacy vs. relevant comparators
Safety profile: Adverse events, contraindications, drug interactions
Certainty of evidence: Quality and robustness of clinical data

Explicitly excluded: Economic evaluations, budget impact, organizational aspects (remain national competence)

JCA Timeline

The regulation mandates strict timelines:

Submission: Simultaneous with or shortly after EMA marketing authorization
Validation: 30 days for completeness check
Assessment: 280 days (extendable to 380 days with clock stops)
Finalization: JCA report published and distributed to member states

Stakeholder Involvement

The process includes:

Patient and clinical expert input
Manufacturer responses to preliminary assessments
Member state review and commenting
Public consultation on draft reports

Preparing for Joint Scientific Consultations

JSC Strategic Value

JSCs provide critical early guidance on:

Appropriate comparators for clinical trials
Relevant endpoints and outcome measures
Study design considerations
Evidence requirements for specific indications

JSC Application Process

Manufacturers should:

Apply 6-12 months before anticipated JCA submission
Prepare comprehensive briefing documents
Engage early with HTA bodies
Align JSC outcomes with regulatory strategy

Optimal Timing

Ideal JSC timing:

Phase II completion for oncology products
Before Phase III initiation for other products
Early enough to influence pivotal trial design
Coordinated with EMA scientific advice

Evidence Requirements and Best Practices

Clinical Evidence Standards

JCAs demand high-quality evidence:

Randomized Controlled Trials (RCTs):

Head-to-head comparisons with relevant alternatives
Appropriate patient populations reflecting clinical practice
Clinically meaningful endpoints
Adequate follow-up duration

Real-World Evidence (RWE):

Increasingly accepted for supportive evidence
Critical for rare diseases and ATMPs
Must meet methodological quality standards
Complementary to RCT data

Comparator Selection

Critical considerations:

Current standard of care in EU member states
Variation across countries and healthcare systems
Multiple comparators may be necessary
JSC guidance essential for alignment

Dossier Preparation Strategies

JCA Submission Requirements

Comprehensive dossiers must include:

Clinical efficacy data: All pivotal and supportive studies
Safety information: Comprehensive adverse event data
Quality of life: Patient-reported outcomes
Subgroup analyses: Relevant patient populations
Indirect comparisons: When head-to-head data unavailable

Documentation Excellence

Best practices:

Clear, structured presentation following templates
Transparent methodology and analysis
Comprehensive literature reviews
Explicit handling of uncertainties
Plain language summaries for stakeholders

Impact on Market Access Strategy

National HTA Processes

Post-JCA, member states conduct:

Economic evaluations (cost-effectiveness)
Budget impact assessments
Organizational and ethical considerations
Pricing and reimbursement negotiations

Strategic Implications

Manufacturers must:

Maintain parallel national HTA preparations
Adapt JCA outcomes to national contexts
Engage with national payers early
Develop country-specific value propositions

Medical Device Considerations

2030 Implementation for Devices

High-risk implantable devices (Class III) will require JCAs from 2030:

Cardiac implants (pacemakers, ICDs, valves)
Orthopedic implants (joint replacements)
Neurostimulation devices
Other Class III implantables

Device-Specific Challenges

Unique considerations:

Rapid technology evolution
Learning curve effects
Operator dependency
Limited RCT feasibility

Practical Preparation Steps

For 2025 Submissions

Immediate actions:

Identify products requiring JCA in 2025-2026
Review clinical development programs against JCA requirements
Engage in JSCs for products in development
Build internal HTA expertise and capabilities
Establish relationships with HTA bodies

Long-Term Strategy

Strategic planning:

Integrate HTA considerations into early development
Design trials with JCA requirements in mind
Develop comprehensive evidence generation plans
Build cross-functional teams (regulatory, medical, market access)
Invest in health economics and outcomes research

Challenges and Opportunities

Key Challenges

Timing coordination: Aligning JCA with regulatory approval and national processes
Resource intensity: Comprehensive dossier preparation requirements
Uncertainty: Evolving processes and precedents
Comparator complexity: Addressing diverse treatment landscapes

Strategic Opportunities

Streamlined assessments: Single clinical evaluation for all EU markets
Early engagement: JSCs enable proactive evidence planning
Transparency: Clear assessment criteria and processes
Harmonization: Reduced duplication across member states

Conclusion

The EU HTA Regulation represents the most significant change in European market access in decades. Success requires early preparation, robust clinical evidence, strategic engagement with HTA bodies, and seamless coordination between regulatory and market access functions.

Manufacturers who embrace these requirements, invest in comprehensive evidence generation, and engage proactively with the JCA process will be best positioned for successful EU market access in 2025 and beyond. The regulation, while demanding, ultimately creates a more transparent, efficient, and scientifically rigorous pathway to patient access across Europe.