Expanding a rare disease therapy across Europe is a great business opportunity but at the same time a unique challenge to handle. Low prevalence, high unmet need, extremely specialized clinical networks and heterogeneity in regulatory and access frameworks among the 27 EU countries mean that success depends on how you build your strategy and not just what you launch on the market.
Here three fundamental factors to build a solid strategy:
Rare Disease in Europe: set up early multi-country insights
In rare diseases, even small differences in diagnostic pathways can change everything, from time to diagnosis, patient flow, to referral patterns. Relying only on EU-level data is risky without digging into country peculiarities. Therefore, you should start early in collecting and analysing country-specific insights across a mix of large and small markets, including epidemiology, diagnostic delays, centers of excellence and patient organization maturity.
Country-level evidence is a decisive driver when approaching pricing and reimbursement discussions. Bringing robust local data significantly increases the credibility of the value story in front of national HTA bodies and payers. It shows a deep understanding of the local healthcare environment, makes projections more realistic, and helps tailor the access ask to specific priorities and constraints.
In a nutshell, this can translate into smoother negotiations, shorter timelines, and stronger adoption post-launch.
Recently, we have supported a biotech developing a therapy for a rare disorder of bone mineralization. We convened a multidisciplinary Italian expert board composed of clinicians actively diagnosing and managing affected patients with the aim to:
- map real diagnostic pathways and barriers
- collect clinical cases to enrich understanding of the disease’s natural history
- identify the most impactful touchpoints to accelerate recognition and referral
These country-level insights helped refine the evidence and company strategy, ensuring that future engagement with Italian stakeholders and access authorities is grounded in credible, real-world knowledge.
Co-create value with local stakeholders
Engagement is not an afterthought: it is the strategy.
Clinicians, nurses, local patient groups, and reference centres hold unique knowledge of the real-world barriers patients face.
Co-developing education, care pathway materials, or early data initiatives ensures your solution is credible, relevant, and immediately useful as they are shaped by those who will deliver and experience care. This approach is fundamental because it helps build a network of people who can provide trusted feedback essential to improve the outcome.
Tailor your access strategy to national healthcare realities
European healthcare systems vary widely in how they evaluate new treatments, set priorities, and enable early access for high-need patients.
Some countries focus more on clinical outcomes, others on affordability or long-term sustainability, and others still on supporting innovation.
One of the most common mistakes is to assume that an access mechanism that worked in one market can simply be copied and pasted into another. Each country has its own formal regulations and laws, and the correct interpretation and practical application of these rules often require deep local expertise. Moreover, many nuances, “unwritten rules,” stakeholder expectations, and informal practices can only be navigated effectively by someone with on-the-ground knowledge.
A successful example of this approach is our collaboration with a company focused on an ultra-rare pediatric disease in the fields of endocrinology and neurology. We supported them in navigating early access pathways across multiple EU countries, ensuring timely treatment for the patients who needed it most. The company relied on the expertise of our local teams to operate in full compliance with national regulations, while guaranteeing a smooth execution of logistics and drug delivery.
That is why it is important to keep in mind that early access schemes, evidence requirements, pricing rules, and negotiation dynamics differ substantially across countries and evolve over time.
Thus, success requires:
- an accurate assessment of all available access routes in each priority market
- a clear understanding of risks, timelines, and evidence expectations
- a sequenced plan that balances speed of availability with long-term value sustainability.
Very importantly, a strong launch strategy aligns evidence generation, value communication, and sequencing with these national realities and not with a one-size-fits-all EU roadmap.
Europe rewards those who prepare early
Rare disease therapies have the potential to transform lives. Ensuring that patients across Europe can truly benefit requires understanding, engaging, and planning from day one.