Over the last years, the number of Orphan Medicinal Products drugs being developed has rapidly increased, but most of the 6,000-8,000 rare diseases still lack proper diagnosis or treatment. However, new development opportunities stemming from the wealth of recent scientific discoveries, disease understanding and new potential targets are becoming available. This is the result of coordinated research investments and worldwide stakeholder collaborations initiated by the International Rare Diseases Research Consortium. In addition, the US Food and Drug Administration and the European Medicines Agency are stepping up their collaboration to boost the development of orphan medicines. This session aims to present opportunities for public funding and regulatory support for orphan drug development available in Europe and the US with testimonies from companies benefiting from such support and stakeholder collaboration.
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Session ID: 21837