The Urgent Need for Rare Disease Research
Millions of individuals across Europe suffer from rare diseases, yet treatment options remain scarce. Out of the estimated 6,000 to 7,000 recognized rare diseases, a staggering 95% lack approved therapies, and most do not have ongoing research efforts. In response to this crisis, the Rare Disease Moonshot project aims to catalyze research and foster collaboration between public and private sectors, striving to expedite the development of effective treatments.
Overcoming Research Challenges
To achieve advancements in rare disease research, several major obstacles must be addressed, as highlighted by Magda Chlebus, Executive Director of Scientific and Regulatory Affairs at the European Federation of Pharmaceutical Industries and Associations (EFPIA). These challenges include enhancing scientific and translational research capabilities, navigating regulatory and access barriers, and establishing a supportive ecosystem of incentives. During a recent debate, experts discussed the importance of overcoming these hurdles to accelerate progress in treatment developments.
The Power of Collaboration
Daria Julkowska, Assistant Director at the French National Institute of Health and Medical Research (INSERM), emphasized the significance of fostering dialogue among diverse industry stakeholders. She asserted that collaboration is essential since no single entity possesses all the necessary resources to drive change. Magda Chlebus echoed this sentiment, highlighting the unique contributions each participant can offer, from scientific expertise to infrastructural support.
Breaking Down Industry Silos
Graham Slater, a Board Member of the European Organisation for Rare Diseases (EURORDIS), pointed out that some segments of the healthcare industry still operate within silos, which hampers progress. He emphasized the need for interdisciplinary collaboration to foster innovation, suggesting that gathering various stakeholders early in the process can prevent redundant efforts and streamline research initiatives.
Boosting Investment in Europe
Public-private partnerships play a crucial role in promoting competitiveness within Europe’s pharmaceutical sector, according to Vlad Voiculescu, Member of the European Parliament for Renew Europe. He argued that maintaining Europe’s leadership in pharmaceuticals and medical equipment hinges on supporting research and innovation. However, Daria Julkowska noted that funding for research remains insufficient, suggesting that actively optimizing public investments and promoting collaborative ventures could bridge existing gaps and enhance private sector involvement in rare disease treatment development.
SOURCE: Ref Image from Euronews
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