Shaping a competitive, patient-centered future for Europe: Insights from the EURORDIS Round Table of Companies

At a pivotal moment for Europe’s life sciences ecosystem, stakeholders gathered at the EURORDIS Round Table of Companies (ERTC) meeting to examine a critical question: 

How can Europe remain competitive in medicines innovation while ensuring people living with rare diseases gain faster access to new therapies? 

Bringing together representatives from the European Commission, the European Medicines Agency (EMA), patient leaders, and industry, the discussion highlighted both the urgency of reform and the opportunity to reshape Europe’s regulatory and innovation landscape. Here are the key themes and takeaways.

A new geopolitical reality: Competitiveness as a health imperative

The global context for medicines innovation has shifted dramatically, with competitiveness ultimately determining where innovation and trials occur—directly impacting patient access. 
Europe no longer operates in a purely collaborative global environment. Instead, it faces intensified competition from (1) The United States, which accounts for roughly half of the global medicines market   and remains highly attractive for investment and launch sequencing; (2) China, which has rapidly expanded its biotech and biomanufacturing capacity and is increasingly a source of pharmaceutical innovation. 

In today’s environment, competitiveness is inseparable from health security, and Europe’s ability to attract innovation will determine patient access for years to come. Competitiveness, therefore, must align with patient outcomes — not compete with them.

The EU pharmaceutical reform and Biotech Act: A once-in-a-generation opportunity

Speakers described the revision of EU pharmaceutical legislation and the proposed EU Biotech Act as a ‘once-in-a-lifetime’ opportunity to address fragmentation across Member States, slow clinical trial processes, declining global innovation share, complex regulatory pathways, and limited scalability for biotech start-ups. For rare disease development, these reforms could significantly influence where trials are conducted and how quickly innovative therapies reach patients.

The ambition: make Europe faster, simpler, and more coordinated — without compromising safety or scientific rigor.

Key regulatory priorities of those reforms include:

• Faster, more agile clinical trials through improved reliance mechanisms between Member States, with increased responsibility on lead Member States to reduce duplication and timelines—critical for rare diseases with small, geographically dispersed patient populations
• Regulatory sandboxes as a "safe playground" for genuinely innovative products requiring flexible, future-proofed approaches beyond existing frameworks
• Lifecycle coherence with greater alignment from early scientific advice through approval, HTA, and post-marketing evidence generation, emphasizing real-world evidence and patient experience data

While new frameworks were welcomed, stakeholders cautioned against creating additional layers of complexity. The overarching practical goal must be to accelerate innovation for rare disease therapies through:

• Simplification of existing processes
• Reduction of administrative burdens
• Improved coordination across legislative instruments (Pharma Reform, Biotech Act, HTA Regulation, Medical Devices Regulation)

Patient-centeredness: The ultimate test of reform

From a patient perspective, the evaluation criteria are simple:

Will a person living with a rare disease in Europe gain faster and more meaningful access to clinical trials and treatments? 

Speakers emphasized several non-negotiables around patient engagement and evidence generation.

Meaningful and early patient involvement

Patient engagement must move from consultation to co-creation and begin before trial design and endpoint selection.  Early patient involvement improves both trial feasibility and regulatory relevance which is particularly important for rare diseases where evidence generation is challenging.
There was strong support for preserving and strengthening structured patient involvement within the EMA, including voting roles in committees and integration of disease-specific expertise. No one is more expert than a patient or care partner living with the impact of their rare disease.

Proportionate evidence requirements

Rare and ultra-rare diseases require flexible trial designs, adaptive methodologies and acceptance of alternative evidence models, where appropriate.

Embedding elements like (1) patient experience data (beyond traditional patient-reported outcomes); (2) real-world evidence generation; (3) parallel scientific advice between regulators and HTA bodies throughout the product lifecycle — from development to post-marketing — will be critical to ensuring both approval and reimbursement decisions reflect what truly matters to patients. However, cultural alignment across institutions will take time.  

The regulatory system must recognize feasibility constraints — without lowering safety standards. To date, there has been an abundance of caution around alternative evidence models such as the appropriateness of external or synthetic control arms. While caution may well be warranted in some cases, we should not sacrifice the good for the perfect. 

The access gap: Approval is not the finish line

One of the most pressing concerns raised was the persistent gap between marketing authorization and actual patient access across Member States. Regulatory approval, while essential, represents only the first step in a patient's journey to treatment. Authorization alone does not guarantee that patients will receive the therapies they need. Challenges include:

• Fragmented pricing and reimbursement systems
• Delays in HTA decisions
• Sustainability concerns for ultra-rare, high-cost therapies

The example of ultra-rare gene therapies highlighted the inadequacy of traditional reimbursement models for one-time, potentially curative treatments. There was recognition that Europe may need new payment models and potentially new economic paradigms — including not-for-profit or hybrid approaches — for the smallest, rare disease populations.

Single market thinking: From fragmentation to scale

The rare disease ecosystem may offer a unique opportunity to ‘prototype’ a more integrated European Single Market approach. Greater coordination could specifically benefit rare disease trials and patient access. With small patient populations already requiring cross-border cooperation, rare diseases naturally demand:

• Multinational trials
• Shared data infrastructures
• Joint procurement or coordinated reimbursement mechanisms

The COVID-19 vaccine procurement model was cited as proof that collective European action can work when political will aligns. The question now is whether similar ambition can be applied to rare disease therapies.

Conclusion: From vision to delivery, with shared responsibility

The ERTC discussion made one thing clear: Europe stands at a crossroads and it can either: 

• Embrace competitiveness as a vehicle for patient benefit
• Build a coherent, lifecycle-based regulatory ecosystem
• Lead globally in rare disease innovation

Or it can remain fragmented and risk losing its innovation base — and its patients’ trust.

If Europe chooses to prototype a truly integrated, patient-centered Single Market anywhere, rare diseases may be the place to begin.

The rare disease community has long been a pioneer of collaboration, scientific ambition, and patient partnership. Rare disease patients are not passive stakeholders — they are system users and risk bearers. The ultimate metric is not regulatory reform on paper, but whether a child or adult living with a rare disease in Europe can access the right treatment, at the right time. That is the ecosystem worth building, with shared accountability embedded from the start. 

A clear message emerged: reform cannot be delivered by regulators alone. Success requires:

• Member States commitment to implementation
• Industry readiness to engage early and strategically
• Patient organizations adequately funded and supported to participate meaningfully
• Stronger collaboration across EU institutions

By aligning competitiveness, regulatory agility, and patient-centered design, Europe can do more than retain innovation—it can establish global leadership in rare disease development.

Parexel is a CRO of choice for many rare conditions, with profound expertise in precision medicine. Supported by a deep bench of former regulators, cross-functional experts, and patient insights, we’re ready to optimize your development program in Europe, providing practical guidance on how to translate the EU legislation changes into company and patient benefits.  

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