The following statement is not my work but is a quote from another poster on an overseas forum.Very informative and to the point: 12 December 2024 2025 Outlook: Rare Diseases and Big Pharma: *Niche but Lucrative: Rare diseases, defined by their small patient populations but high unmet medical needs, are an increasingly attractive sector for investment.
*Orphan Drug Designations: Regulatory incentives, such as market exclusivity, tax credits, and expedited approval pathways, make rare disease therapies particularly appealing.
*High Margins and Differentiation: The ability to charge premium prices due to the limited competition and specialized nature of these treatments ensures robust returns.
*Expanding Therapeutic Areas: While traditionally focused on genetic and metabolic disorders, rare disease innovation is now branching into neurology, immunology, and hematology.The rare disease space is no longer just about one-off blockbuster therapies. Companies are building entire franchises around rare conditions, leveraging advanced gene and cell therapy technologies. Kens
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