Every year biotechnology companies invest billions of dollars developing therapies for rare diseases. Brilliant scientists identify new genetic targets, sophisticated AI models analyze enormous amounts of data, and promising treatments produce remarkable results in laboratory studies.
Yet most of these therapies never become successful medicines.
The common explanation is that the clinical trial failed.
I believe the real answer begins much earlier.
Too often, our scientific models simply don't reflect the complexity of human biology.
A therapy may correct a genetic mutation in a laboratory. It may restore a missing protein in animal models. It may even improve biomarkers that everyone agrees should predict success.
Then the treatment reaches patients—and the expected clinical benefit simply isn't there.
That isn't necessarily because the drug failed.
It's because the understanding of the disease market was incomplete from the beginning.
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