The Success of the Orphan Drug Act

Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses the success of the Orphan Drug Act (ODA). This Act was first enacted in the United States in 1983 and set up to encourage the development of drugs for rare diseases. At that time, drug therapies for such diseases were rarely developed. Now, 35 years later, a growing proportion of industry research and development and regulatory drug approvals target diseases affecting fewer than 200,000 persons in the United States, the prevalence-based threshold of rare disease under the ODA.