Rare disease launches that scale

Launching a therapy for a rare disease is both triumph and trial. The science is often dazzling, yet companies face the stark reality of vanishingly small patient pools, skeptical payers, and regulators demanding robust evidence from the sparsest of data.

Success requires deft strategy: finding patients early, designing trials that can pass scientific muster, and building outcomes-based frameworks that keep health systems on side.

Finding the patients