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Rare diseases affect millions of Europeans and we face significant challenges in finding treatments for rare diseases. Despite these hurdles, significant progress has been made over the past two decades, with Orphan Medicinal Products (OMP) regulations leading to the development of more than 230 treatments for patients in Europe. Biotechnologies such as gene therapy, gene editing tools, RNA-based therapies, enzyme replacement therapies, and monoclonal antibodies have played an important role in this progress. However, the continued existence of untreated rare diseases highlights the urgency for Europe to strengthen its life sciences ecosystem to reward the innovations produced by European countries and provide solutions for all patients. There is.
With a diverse workforce, advanced research facilities and supportive regulations, Europe fosters an ideal environment for biotech success
Europe is rich in academic research centers and large and small biotech companies that have the potential to usher in a revolution in rare disease treatment. As we continue to witness advances in medicine, it is essential to recognize and prioritize the importance of biotechnology in reshaping the rare disease treatment landscape.
Europe’s biotech ecosystem: rare disease innovation hub
Europe’s biotech landscape, with its mix of large and small companies, is an important center of innovation in rare disease treatments. The region is home to top scientists who produce outstanding research results and drive innovation. A robust ecosystem is essential to conduct innovative research and bring new treatments to patients in Europe. With a diverse workforce, advanced research facilities, and supportive regulations, Europe fosters an ideal environment for biotechnology success. However, in the face of global competition, Europe needs to implement supportive policies to ensure rare disease treatment development and patient access.
Promoting Investment: Fostering Innovation and Growth in Biotechnology
The path from idea to innovative treatment is fraught with financial hurdles. Investment is critical to sustaining and promoting biotechnology innovation and ensuring successful research and development (R&D) efforts. Europe’s fragmented life sciences investment environment faces challenges, accounting for only 14% of venture capital deals in 2022, compared to 62% in the US and 21% in China. Recognizing biotechnology as an important technology for Europe should lead to increased investment in research and development and clinical trials, and the expansion of European companies. Strategic public and private investments in healthcare biotechnology have the potential to drive innovation and unlock science to improve treatments for patients with rare diseases.
Uniting in complexity: Strengthening collaboration between large-scale and small-scale biotechs
Collaboration is critical in rare diseases. Large biotech companies bring knowledge and resources, but small, innovative and agile companies operate at the cutting edge of science. A symbiotic relationship, driven by a shared commitment to improving the lives of people with rare diseases, accelerates the development of treatments from the clinic to the patient. To maintain and foster this collaboration, the right policies and incentives are essential for its growth, attracting talent and delivering breakthrough innovations to European patients.
Keeping the momentum for innovation: essential for biotechnology
Ten years of orphan market exclusivity and orphan designation (OD) are critical to facilitating biotechnology investment in rare disease research by providing the necessary predictability and risk mitigation. These incentives offset risky development, allowing companies to secure funding, develop treatments, and recover their investments. For small innovators, which developed two-thirds of OD medicines between 2018 and 2022, OD is key to attracting investment to fund clinical development.
Breakthrough Designation: Paving the Way to Unprecedented Progress
Introducing breakthrough designations in addition to existing orphan designations is critical to fostering innovation in underserved areas. This new designation is given to truly innovative orphan products that meet defined criteria, providing predictability early in the development process. This would act as a lever, encouraging biotech companies, especially smaller companies that rely on external investment, to pursue high-risk development through increased collaboration and investment, particularly in clinical development.
For more information about Rare Disease Day, please visit our Rare Disease Hub or our Rare Disease Supplement.
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