A New Era for Access and Evidence Expectations in Europe
For years, Europe has been a place of scientific opportunity but operational complexity. Companies launching innovative therapies often found themselves navigating 27 different interpretations of the same evidence; different comparators, different endpoints, different standards of practice. The result wasn’t just administrative burden. This convoluted process delayed patient access to innovative drugs and created uncertainty around how value would be understood across markets.
As of 2025, that world has shifted.
The EU’s new Health Technology Assessment (HTA) Regulation introduces a single, collaborative evaluation of clinical effectiveness and safety: the Joint Clinical Assessment (JCA). It’s a change designed to reduce duplication of work, ensure high quality assessments, and create a more predictable, transparent assessment process across member states. But for biotechs preparing for European launch, it also tightens the timelines and demands earlier alignment across clinical, regulatory, and market access functions than ever before.
What This Guide Covers
This EU HTA Regulation guide explains the regulatory shift and how we support biotech teams in aligning evidence and operations early—from trial design through submission strategy.
In brief: From 2025, the EU HTA Regulation introduces a Joint Clinical Assessment (JCA) alongside EMA review for in-scope medicines. After PICO scoping, teams may have a tight window—often around 100 days—to submit a complete clinical value dossier. The shift isn’t just procedural; it changes when evidence decisions need to be made, and it rewards earlier alignment across clinical, regulatory, HEOR, and market access.
1. EU HTA Reform Explained: What It Is and Why It Was Needed
Health Technology Assessment has always played a critical role in European access. While the EMA focuses on benefit–risk, national HTA bodies assess whether a therapy offers meaningful clinical value compared with existing alternatives, strong enough to justify reimbursement.
The problem wasn’t HTA itself.
It was the fragmentation.
Each country developed its own methodologies, comparators, requirements, and submission timelines. A therapy embraced in Germany could be heavily restricted in France. Clinical evidence designed to satisfy the UK might miss key endpoints valued in Italy. Companies often repeated work, tailored evidence narratives multiple times, and managed parallel reviews across the continent.
Furthermore, the often stepwise timing of HTA applications across the EU led to unequal access for patients.
The Solution: Joint Clinical Assessment
The new regulation doesn't eliminate national HTA. But it lays the foundation for greater alignment by introducing a shared evaluation of a therapy's clinical benefit: the JCA. National bodies still have the responsibility for economic evaluations and reimbursement decisions, but the clinical assessment will be centralized.
It's a step toward coherence, and a shift that requires new levels of preparedness.
2. What’s Changing Under the EU HTA Regulation
The EU HTA Regulation began its phased rollout in early 2025. At the heart of the reform is the Joint Clinical Assessment, a single evaluation of a product's relative clinical effectiveness and safety across all EU member states.
Implementation Timeline
2025
Oncology & ATMPs
New oncology products and advanced therapy medicinal products (ATMPs)
2028
Orphan Medicines
Rare disease therapies with orphan designation
2030
All Centrally Authorized Medicines
All new centrally authorized medicines and new indications
Why Is This Different?
Instead of 27 separate clinical assessments, you will from now on produce one!
This streamlines parts of the process, but it also means:
- Comprehensive evidence requirements
- HTA evaluation in parallel with regulatory MAA procedure
- Evidence needs must be anticipated and addressed well in advance of regulatory submission
In summary, evidence expectations have increased and now apply much earlier in the product lifecycle.
3. The Joint Clinical Assessment (JCA)
The Process, the Pressure, and the Opportunity
The JCA introduces a new pace to European access — one many companies underestimate.
Once your Marketing Authorization Application (MAA) is validated by the EMA, HTA assessors will start the assessment scoping process, the PICO: Population. Intervention. Comparator(s). Outcomes.
This scope isn't advisory. It defines the content and structure of your entire clinical assessment and is designed to meet the evidence needs of all member states.
The 100-Day Window
From the moment you receive the PICO scope, you have 100 days to submit a complete JCA dossier. Those 100 days move quickly, so preparation can't begin at submission. It must begin long before.
Why the JCA timeline matters so much
The JCA is built to be efficient. But efficiency requires readiness. Companies who treat HTA as a late-stage activity often find themselves lacking the evidence needed to satisfy for European expectations.
Oncology, rare disease, and ATMP developers feel this most acutely.
Small populations, evolving standards of care, and surrogate endpoints already stretch evidence generation. The JCA amplifies those challenges by requiring a coherent, comparative narrative across multiple PICOs.
Where early preparation makes the difference
The companies who navigate the JCA successfully are those who understand:
- Comparator expectations reflect what clinicians actually use in routine health care, not what regulators expect.
- Subpopulations and varying standards of care across the EU can create multiple PICOs, each requiring its own evidence set.
- Indirect comparisons (ITCs) often become essential when head-to-head comparisons are not available to support all PICOs.
- Treatment landscapes shift quickly. Trial design must anticipate the future, not the past.
This is where we help organizations bring clarity to complexity.
4. The Joint Scientific Consultation (JSC): Shaping Evidence Before It’s Too Late
If the JCA is the exam, the JSC is the study session.
A Joint Scientific Consultation offers developers the rare opportunity to engage with HTA assessors before pivotal trial designs are finalized. It's a moment to ask critical questions:
- Is our comparator appropriate for EU clinical practice?
- Are our endpoints meaningful for HTA decision-makers?
- Can we use real-world evidence to meet some evidence needs?
- Are our subpopulations aligned with how the therapy is used in practice?
Not every company secures a JSC slot. Availability is limited, and the regulation prioritizes therapies with high unmet need or significant public health relevance. But for programs in oncology, rare disease, and advanced therapies, a well-timed JSC can prevent costly redesigns and future misalignment.
Why a JSC matters
A JSC doesn’t guarantee alignment, but it creates a shared understanding of expectations. It exposes risks early. It allows you to adjust trial design and plan additional evidence generation while there’s still time. And it builds a foundation for a stronger, more deliberate evidence generation strategy.
The preparation for a JSC often becomes as valuable as the consultation itself.
It forces teams to think deeply, to align cross-functionally, and to anticipate how European HTA demands are evolving in real time.
5. How US and EU Expectations Differ — and Why it Matters
US-based companies are often surprised by how differently Europe structures the assessment of clinical value: through a formal framework, applied earlier and with clearer expectations for evidence.
United States
Primary Driver: Evidence generation is guided primarily by FDA expectations.
Comparator choice: Grounded in scientific rationale and feasibility.
Endpoints focus: Designed to demonstrate benefit-risk for regulatory approval.
Value & Cost-effectiveness: Typically addressed after approval, often through payer-specific negotiations; sits outside the regulatory process.
European Union
Primary driver: Market access depends on regulatory approval and formal HTA evaluation.
Comparator choice: Anchored to what patients currently receive in routine care (standard of care).
Endpoints focus: Must support relative effectiveness and clinically meaningful improvement for HTA/JCA assessment.
Value & cost effectiveness: Considered as part of access decisions alongside the clinical package; relative value is central.
HTA bodies — and now the JCA — ask:
How does this therapy compare to what patients currently receive? Does it offer clinically meaningful improvement?
This means that:
- FDA-ready trials may not meet European comparator expectations.
- Endpoints valued by regulators may not satisfy HTA bodies.
- Standards of care differ across countries and shift quickly.
- Economic evaluations depend heavily on the strength of the clinical case.
The most common pitfalls we see
Companies tend to underestimate:
- How many PICOs their indication may generate
- Differences between European and US clinical practice
- How frequently comparators diverge between France, Germany, Spain, and Italy
- The level of evidence expected for ITCs and RWE
- How fast oncology landscapes move
These differences, if overlooked, can delay access and erode value. When addressed early, through integrated evidence planning, they’re entirely solvable.
6. How We Help Companies Get HTA-Ready
Our role isn’t just to interpret the regulation.
It’s to guide companies through the real-world decisions it demands; from trial design to submission strategy.
We support companies across four core areas:
HTA & Evidence Strategy
We help you anticipate how your evidence will be interpreted, and where gaps may exist. This includes:
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PICO scope scenario drafting
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Comparator landscape mapping
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Endpoint relevance assessments
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Prioritized integrated evidence generation plans
JCA Dossier Support
We help teams move from PICO scenarios to a finalized JCA dossier with a clear evidence strategy and defensible narrative. This includes:
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PICO scenarios & prioritization
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Scientific Advice strategy
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Evidence generation planning + oversight (ITCs, RWE)
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Dossier preparation & finalization (adapted to final PICO scope
JSC Preparation & Leadership
We guide teams through the entire process:
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Identifying when a JSC is valuable and feasible
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Crafting focused, high-impact questions
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Preparing briefing packages and applicant’s positions
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Translating advice into actionable changes (incl. alternatives when a JSC isn’t feasible: national HTA advice, advisory boards)
Organizational Readiness
We help build the workflows, governance, and capabilities to manage parallel MAA and JCA submissions without operational strain.
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Cross-functional operating model and workflows
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Governance and decision-making structure
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Capability building across key functions (clinical, medical, regulatory)
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Strategic Medical Office framework to enable integration
7. Real-World Scenarios: What This Looks Like in Practice
Oncology Developer Facing Rapid SOC Changes
An oncology team designs a trial around today’s standard of care (SOC), only to find that by the time of JCA, new therapies have shifted practice. Early PICO modeling helps anticipate this and reduce the risk of misalignment as SOC evolves.
Rare Disease Program with Limited Evidence
A company relying on a single-arm trial can strengthen its case through natural history data and thoughtful use of RWE, but only if planned early enough.
ATMP With Immature Durability Data
When long-term outcomes aren’t yet mature, thoughtful justification and post-market evidence strategies become essential.
US Biotech Entering Europe Late
When companies approach HTA after pivotal data are locked, we focus on how to address outstanding PICOs with indirect treatment comparisons and weaving a coherent narrative around the resulting evidence constraints.
These scenarios are common — and entirely manageable with the right preparation.
8. Resource Hub: What you need to know and where to start
Explore the EU HTA Regulation through our expert-led articles and webinars. We focus on what’s changing, where teams get caught off guard, and how to prepare early - across evidence strategy, operations, and execution.
Articles: Short reads to help you get oriented
Webinars: Watch the sessions or download the summaries
Preparing for the Future Starts Now
The EU HTA Regulation represents one of the most important shifts in European market access in decades. It’s a system designed to bring clarity and coherence, but it also requires companies to think differently, align earlier, and build evidence that truly reflects clinical practice. As a positive side-effect, this will improve drug development and the value generated across all geographies.
The organizations that succeed under this new framework are those that treat HTA not as an obstacle, but as a strategic lever. They anticipate PICO scenarios. They design trials that serve both regulatory and HTA expectations. They build cross-functional systems that can move quickly — without sacrificing quality or clarity.
We’re here to help you do exactly that.
