How Rare Diseases Are Reshaping Evidence Generation Strategies

How Rare Diseases Are Reshaping Evidence Generation Strategies

Defining “Rarity” in the Modern Evidence Landscape  Rare diseases collectively represent a significant public health challenge despite their individual low prevalence. In the United States, a rare disease is defined under the Orphan Drug Act as one affecting fewer than 200,000 individuals; definitions vary internationally. Although each condition affects relatively few patients, more than 10,000 rare diseases have been identified worldwide, toge…