Supporting the Development of Drugs for Rare Diseases — The Importance of Regulatory Transparency

More than 300 million people worldwide have a rare disease or condition, yet treatments approved by the U.S. Food and Drug Administration (FDA) are available for less than 5% of these diseases.¹ Legislation enacted in 2022 called on the FDA to task the National Academies of Sciences, Engineering, and Medicine (NASEM) with studying processes in the United States and the European Union for evaluating the safety and efficacy of drugs for rare diseases and conditions, nearly all of which are classified as orphan drugs because they are intended to treat a condition affecting fewer than 200,000 people in the United States.¹ A central theme of the resulting report’s conclusions was that inadequate regulatory transparency impedes the development of safe and...