The European market for orphan drugs presents many opportunities and challenges for pharma companies. With over 30 million people in the EU living with rare diseases, the demand for innovative therapies is high. However, achieving market access is complex with varied healthcare systems, strict pricing controls, and reimbursement hurdles to overcome. Rare disease treatments must navigate stringent orphan drug regulations, evidence requirements, and payer skepticism regarding cost-effectiveness.
In recognition of the challenges faced by pharma companies, the European Medicines Agency (EMA) launched a new joint health technology assessment (HTA) process in January 2025 to streamline and improve the evaluation of new medicines and health technologies. However, the reports generated through the joint HTA process aren’t legally binding and national HTAs will still be able to ask for additional clinical evidence to make their own judgement. In reality, it’s still very early days and we don’t yet fully understand how this will all work so it’s a good idea to stay up to date with both the old and new forms of assessment.
In this article, we look at common pitfalls in EU orphan drug launches and how can you avoid making them.
- Taking a ‘one-size fits all’ approach
While the EMA provides centralized marketing authorization, pricing and reimbursement decisions are made at a national level. Each country has its own HTA body, reimbursement criteria, and pricing negotiation process.
If you don’t take this into account your rare disease drug may gain approval but fail to secure reimbursement in key markets, delaying patient access and affecting revenue forecasts. That’s why it’s vital to develop an early pricing and reimbursement strategy that considers country-specific HTA requirements. In doing so, make sure to engage local payers and decision-makers early to understand their expectations and make use of early access programs (EAPs) and compassionate use programs to build real-world evidence.
- Delaying real-world data collection
Failing to collect real-world data (RWD) early in the drug development process for rare disease drugs is risky, as these treatments often face significant clinical trial challenges. This makes it difficult to gather comprehensive evidence on long-term efficacy and safety and, without it, health authorities may reject or delay reimbursement.
To avoid this, it’s wise to apply adaptive trial designs and gather real-world evidence (RWE) early to supplement data gaps. Patient registries and natural history studies can also strengthen the clinical justification for your product by providing a deeper understanding of disease progression. Exploring Managed Entry Agreements (MEAs), such as outcome-based pricing models, can enable you to share financial risks with payers, making it easier to address their concerns.
- Ignoring high pricing sensitivity and budget impact concerns
Orphan drugs are typically high cost, which can lead payers to reject or restrict coverage due to uncertainty around long-term value, high budget impact relative to the patient population size, and concerns about equity across therapeutic areas. Even if market access is secured, controls such as step therapy, restricted prescribing, or capped patient numbers can limit the drug’s sales potential.
Developing flexible pricing models such as risk-sharing or pay-for-performance agreements that address payers’ concerns can help. Collaborating with patient advocacy groups can also help you to demonstrate the unmet medical need and strengthen the drug’s value proposition. Country-specific affordability solutions, such as tiered pricing or volume-based discounts, can further ease budgetary concerns and improve access across different markets.
4. Failing to plan for delays in HTA and reimbursement decisions
Even after EMA approval, market access timelines tend to vary significantly by country. Reimbursement decisions can also be delayed by additional data requests, pricing disputes, or payers demanding additional evidence. This subsequently delays patient access and can have a detrimental impact on the drug’s commercial success.
It is essential to engage with HTA agencies early to understand their evidence requirements to support a smooth submission process. Developing a pan-European dossier with local economic models can help support negotiations across different markets, while Managed Access Programs (MAPs) enable early patient access and uptake, allowing the drug to reach the market sooner, even before full reimbursement is secured.
5. Relying solely on EU-wide incentives
A common mistake is relying on pan-EU initiatives which can lead to miscalculations in the financial viability of an orphan drug in specific markets. While incentives like protocol assistance and fee reductions are valuable, national policies and incentives vary, which can affect your market access.
It’s vital that you conduct a thorough analysis of country-specific orphan drug policies and incentives to optimize launch sequencing. Engaging local regulators and patient advocacy groups can help build a stronger case for funding and access in individual markets and parallel submissions can streamline the HTA and pricing approval process across multiple countries.
Conclusion
Launching a rare disease drug in Europe can be perilous, with many market access pitfalls that can delay patient access and impact financial returns. The fragmented reimbursement landscape, data limitations, payer resistance to high prices, and prolonged negotiations all present significant challenges.
Partnering with a specialist provider with deep expertise in the European market and varying regulations and has local experts on the ground, is essential for successful EU market entry and long-term growth. By harnessing your partner’s infrastructure and local knowledge you can navigate regulatory and cultural differences across Europe. This can reduce costs and accelerate market access by avoiding delays and missteps. It can also help you build a strong foundation in the region, providing valuable insights and networks that can support future independent expansion.
Sciensus
Sciensus is a leading European life sciences organisation with over 30 years of experience. We’re redefining patient-centric care, connecting patients with life-changing medicines while empowering pharmaceutical companies and healthcare professionals with the insights needed to improve people’s lives.
To learn more and get in touch, please visit: Rare and Orphan Diseases - Pharmaceutical Services | Sciensus or contact us at: [email protected]