EU As A Launch Pad

Think Global, Start European:
A Strategic Launchpad for Rare Disease Therapies

The global rare disease landscape is evolving rapidly, with increasing investment and a growing pipeline of innovative therapies. Yet, navigating the complex regulatory and market access pathways remains a significant challenge for biotech and pharmaceutical companies. In this context, Europe, with its robust regulatory framework and supportive policies, presents a compelling launchpad for rare disease therapies, offering a strategic advantage for companies aiming to make a global impact.

The European Medicines Agency (EMA)
A Supportive Framework for Rare Disease Innovation

The European Medicines Agency (EMA), the European Union’s (EU) regulatory body for medicines, plays a pivotal role in fostering rare disease innovation. The EMA’s commitment to rare diseases is evident in its dedicated programs, initiatives, and incentives aimed at accelerating the development and approval of therapies for these underserved patient populations.

Orphan Drug Designation (ODD)
A Cornerstone of EMA's Commitment

The EMA’s Orphan Drug Designation (ODD) program is a cornerstone of its rare disease strategy. This designation, granted to medicines intended for the diagnosis, prevention, or treatment of life-threatening or chronically debilitating conditions affecting a limited number of patients (no more than 5 in 10,000 people in the EU), offers a range of incentives to encourage investment in rare disease research and development. (EMA, 2023)

Key Benefits of EMA's Orphan Drug Designation

1. Market Exclusivity

ODD grants up to 10 years of market exclusivity upon approval, safeguarding investments and fostering a competitive advantage. (EMA, 2023)

2. Fee Reductions

The EMA significantly reduces regulatory fees for ODD-designated therapies, easing the financial burden on smaller companies and startups. (EMA, 2023)

3. Scientific Advice

The EMA provides scientific advice and protocol assistance, offering expert guidance on clinical trial design and regulatory strategy. (EMA, 2023)

4. Potential for Conditional Approval

In certain cases, promising early-stage data can lead to conditional approval, accelerating patient access while the company gathers additional evidence. (EMA, 2023)

European Success as a Global Catalyst
Leveraging EMA Approval for Worldwide Impact

A successful launch in Europe, facilitated by the EMA’s supportive framework, can propel rare disease therapies onto the global stage.

Key Advantages of a European Launch

Attracting Investment

A positive regulatory outcome in Europe and a successful market launch can attract significant investment from global investors. This influx of capital can fuel further research, development, and commercialization efforts, including expansion into other markets like the US.

Generating Robust Evidence

The EMA places a strong emphasis on real-world evidence (RWE) generation, including patient reported outcomes (PROs) and quality-of-life data. This focus aligns with the growing demands of global payers and health technology assessment (HTA) bodies, strengthening the therapy’s value proposition worldwide. (Eurordis, 2022)

Partner Rare
Your Strategic Partner for Navigating the European Rare Disease Landscape

Partner Rare is a team of seasoned experts with a deep understanding of the European rare disease ecosystem. We offer comprehensive support throughout the development and commercialization journey, helping you navigate the EMA’s regulatory landscape, design and execute clinical trials, and develop effective market access strategies.

Our Services Include

  • Orphan Drug Designation (ODD) Applications
  • Clinical Trial Design and Execution
  • Regulatory Strategy and Guidance
  • Market Access and Pricing
  • Value Proposition Development
  • Patient Advocacy Engagement

Conclusion

Europe’s commitment to rare disease innovation, coupled with the EMA’s supportive regulatory framework, presents a unique opportunity for companies developing rare disease therapies. By partnering with experienced consultants like Partner Rare, you can leverage this opportunity to accelerate your therapy’s global impact and ultimately bring life-changing treatments to patients worldwide.

If you are ready to unlock the vast potential of Europe for your rare disease therapy, contact Partner Rare today to schedule a meeting.

Let’s discuss your specific development and commercialization goals and explore how our expertise can propel your therapy towards success.

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