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Our Work Consensus/Advisory Activity Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union

Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union

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In response to a Congressional request, an ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will conduct a study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union.

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To Increase Pace and Volume of Drug Approvals for Rare Diseases, Report Recommends FDA Enhance Information Sharing, Improve Collaboration

News Release

Rare diseases affect at least 300 million people around the world, yet less than 5 percent of these diseases have approved products on the market for treatment. A new report provides recommendations to increase the pace and volume at which drugs to treat rare diseases are developed and approved.

Sep 12, 2024

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Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union

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To Increase Pace and Volume of Drug Approvals for Rare Diseases, Report Recommends FDA Enhance Information Sharing, Improve Collaboration