For biotech companies specializing in rare diseases, expanding in Europe offers an exciting opportunity to generate value, access new markets and deliver therapies to patients with high unmet medical needs. As we previously discussed in our two-part series about the benefits of entering the EU, diversifying the market helps reduce commercial and financial risks while also boosting clinical development. Indeed, engaging in early-stage communications with regulatory authorities can significantly improve the validity and accuracy of clinical data.
A complex and changing Europe
While all of this is true, opportunities also come with complexity. The European healthcare landscape is diverse and for U.S. start-ups the leap overseas can feel daunting. The European Union is home to 27 Member States, each with its own language, healthcare system and regulatory requirements.
Unlike the United States, in the EU there’s no unified framework for national access processeslike Pricing and Reimbursement negotiations. This fragmentation means that every country must be approached individually by paying careful attention to national timelines, assessment bodies and stakeholder dynamics.
Moreover, the European pharmaceutical environment is changing. In the coming years, the new EU pharmaceutical legislation and the implementation of the joint Health Technology Assessment (HTA) regulation will be effective for the rare diseases landscape as well.
- The new HTA regulation is estimated to come into force for rare diseases therapies in 2028 and will streamline clinical assessments at the EU level by introducing joint clinical assessments (JCAs) and focusing exclusively on clinical benefit, while economic evaluation, pricing and reimbursement will still be determined independently by each Member State.
- At the same time, the revision of the EU pharmaceutical legislation will redefine how incentives, access and regulatory processes work, particularly for orphan drugs. The proposals include linking market exclusivity to timely access across EU countries and optimizing procedures to reduce time for approval.
BlueDil International to help you navigate the EU context
In today’s heterogeneous and rapidly evolving European healthcare landscape, achieving a successful expansion demands scientific excellence, but more so a well-planned combination of strategic outlook, operational precision and a reliable partner capable of handling both. This is precisely where BlueDil International can step in to support your development goals.
Unlike conventional consultancy firms, BlueDil International serves as a functional and integrated ally acting as a virtual yet fully operational affiliate for startup companies aiming to enter or expand within the European rare disease market. We offer a unique model that combines senior-level strategic support and coordination with sharp national execution.
Our pan-European teams deliver customized support tailored to the specific needs of your product across its entire life cycle. Whether your focus is the launching strategy or the establishment of a professional network across hotpoints, our approach is agile and aligned with your goals.
Partnering with BlueDil International offers:
- Tailored strategic planning across the EU led by experienced industry professionals who understand both the macro and micro drivers of success.
- National-level execution of operational activities, whether related to market access, medical affairs, regulatory support, or stakeholder engagement, delivered by country teams with an in-depth understanding of national frameworks and key decision-makers.
- Fully customized support, adapted to the development stage of your product and aligned with your broader corporate strategy.
By working as an extension of your team, we ensure strategic continuity, operational alignment, and the flexibility to adapt to real-time needs. The result is a partnership that supports you in understanding the European landscape and positions you to thrive within it.
Turning potential into impact: a smarter path forward
As the rare disease field continues to evolve, so do the expectations of regulators, payers and patient communities. Therefore, it is vital to sustain promising therapy with the know-how to bring it to patients efficiently and compliantly in each individual country.
For biotech start-ups ready to bring their orphan drugs to Europe, BlueDil International stands as a strategic and operational bridge, helping them navigate complexity, reduce risk and ultimately deliver hope to those who need it most.