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An Evolving Regulatory Environment for Rare and Orphan Diseases

Advarra

The law established the Office of Orphan Products Development , providing financial incentives for pharmaceutical companies to develop orphan drugs and making it more viable to invest in orphan drug research and development. The impact was clear: Between 1983 and 2020, FDA approved 599 orphan drugs. A Lasting Impact.

Disease 52
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Deliberate Dysentery

Codon

We wouldn't do a challenge trial for Ebola, because the disease is extremely severe, and there are currently no licensed treatments or vaccines. You can do a challenge trial with deadly diseases, but you need a “rescue therapy.” There have been deaths in phase 1 medical and drug research among healthy participants.

Vaccine 111