California Consortium Models Rare Diseases Readiness

By Gina Shaw“Rare diseases” aren’t actually all that rare in the aggregate: More than 30 million Americans live with one of roughly 7,000 known rare diseases, although barely 10% of those conditions have an FDA-approved therapy.Globally, spending on drugs for rare diseases reached $195 billion in 2024 and is projected to hit $375 billion by 2030, expanding at an 11.6% compound annual growth rate. Orphan indications now account for more than half of all new molecular entity appr