Scientist experimenting in a pharma lab

Orphan drugs encounter challenges that extend from their initial discovery to eventual launch, primarily due to the restricted patient numbers and niche markets associated with therapies for rare diseases. In an August 2023 study involving close to 400 professionals within the orphan drugs sector, Renejix investigated the pivotal factors and obstacles spanning both the development and commercialization stages. Despite grappling with limitations in skills and resources, the visionary entities that confront these hurdles are frequently driven by a determination to aid patients who currently lack alternative solutions.


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