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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.

Disease 52
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Deliberate Dysentery

Codon

In some cases, I’d argue that a malaria challenge trial is safer than a typical oncology phase 1 drug trial, where you're taking people who don't have cancer and testing the safety of a drug that has maybe been tested in mice but has never been used in humans. What is your pitch for them?

Vaccine 108