The Real Reason So Many Rare Disease Therapies Never Reach Patients

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…