Empowering innovation: The future of rare disease therapies in Europe

With biotech companies playing a pivotal role in finding therapies for people living with rare diseases, Europe has the potential to lead the charge in revolutionising new treatments

By Claire Skentelbery

Director General for EuropaBio

23 Feb 2024

Rare diseases impact millions of Europeans and finding therapeutic solutions for rare diseases faces significant challenges. Despite these hurdles, notable progress has been made in the last two decades, with over 230 treatments developed for European patients driven by the Orphan Medicinal Products (OMP) Regulation. Biotechnologies including gene therapies, gene editing tools, RNA-based therapies, enzyme replacement therapies and monoclonal antibodies have played key roles in this progress. However, the persistence of untreated rare diseases emphasises the urgency for Europe to enhance its life science ecosystem to reward innovation generated by European countries and provide solutions for all patients. 

With diverse talent, advanced research facilities and supportive regulations, Europe fosters an ideal environment for biotech success

Europe, with its rich tapestry of academic research centres and small and larger biotech companies, has the potential to lead the charge in revolutionising rare disease treatments. As we continue to witness medical breakthroughs, it is imperative to recognise and prioritise the importance of biotech in reshaping the landscape of rare disease treatment. 

The European biotech ecosystem: an innovation hub for rare diseases 

Europe's biotech landscape, featuring both large and small companies, is a key innovation hub for rare disease therapies. The region boasts top-tier scientists producing outstanding research outputs, propelling innovation forward. A robust ecosystem is essential for ground-breaking research and delivering novel treatments to European patients. With diverse talent, advanced research facilities and supportive regulations, Europe fosters an ideal environment for biotech success. However, amid global competition, Europe must implement supportive policies to ensure the development and accessibility of rare disease therapies for its patients. 

Boosting investments: fuelling biotech innovation and growth 

The path from idea to transformative therapy is riddled with financial hurdles. Investments are pivotal for sustaining and expediting biotech innovation, allowing research and development (R&D) efforts to thrive. Europe's fragmented life science investment landscape faces challenges, representing only 14 per cent of venture capital deals in 2022, compared to 62 per cent for the United States and 21 per cent for China. Recognising biotech as a critical technology for Europe should translate to increased investments in R&D and clinical trials, and scaling up for European companies. Strategic investments from public and private entities into healthcare biotech have the potential to drive innovation, unlocking science for improved therapeutic solutions for rare disease patients. 

United in complexity: strengthening collaboration between large and small biotech 

Collaboration is crucial in rare diseases. Large biotech firms bring knowledge and resources, while small ones, innovative and nimble, operate at the cutting edge of science. A symbiotic relationship, driven by a shared commitment to bettering the lives of rare disease patients, accelerates therapy development from bench to patients. To sustain and nurture this collaboration, the right policies and incentives are vital for its growth, talent attraction and the delivery of groundbreaking innovations to European patients. 

Preserving innovation drivers: a necessity for biotech  

The 10-year orphan market exclusivity, coupled with orphan designation (OD), is crucial for incentivising biotech investment in rare disease research by providing the necessary predictability and risk mitigation. These incentives counterbalance a highly risky development, enabling companies to secure funds, develop therapies and recoup investments. For smaller innovators, who originated two-third of the OD medicines between 2018 and 2022, the OD is key to attract investments to fund clinical development.  

Breakthrough designation: paving the way for unprecedented progress 

To boost innovation in underserved areas, introducing a breakthrough designation alongside the existing orphan designation is crucial. This new designation, awarded to truly innovative orphan products meeting defined criteria, would provide predictability early in the development process. It would serve as a leverage point, encouraging biotech companies, particularly smaller ones relying on external investments, to pursue high-risk development through increased partnerships and investments, especially in clinical development. 

Read more about Rare Disease Day on our Rare Disease Hub or in our Rare Disease supplement.