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Cover of Regulatory Processes for Rare Disease Drugs in the United States and European Union

Regulatory Processes for Rare Disease Drugs in the United States and European Union

Flexibilities and Collaborative Opportunities

; Editors: Carolyn K. Shore, Tequam L. Worku, Carson W. Smith, and Jeffrey P. Kahn.

Washington (DC): National Academies Press (US); .
ISBN-13: 978-0-309-72655-9ISBN-10: 0-309-72655-7

Rare diseases, such as sickle cell disease and thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. Congress called on the U.S. Food and Drug Administration (FDA) to sponsor a National Academies study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union. The resulting report provides recommendations for enhancing and promoting rare disease drug development by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between FDA and the European Medicines Agency.

Contents

This activity was supported by a contract between the National Academy of Sciences and the U.S. Food and Drug Administration (75F40123C00077). Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project.

Suggested citation:

National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory processes for rare disease drugs in the United States and European Union: Flexibilities and collaborative opportunities. Washington, DC: The National Academies Press. https://doi.org/10.17226/27968.

Digital Object Identifier: https://doi.org/10.17226/27968

This publication is available from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu.

Printed in the United States of America.

Created: September 12, 2024; Last Update: October 30, 2024.

Copyright 2024 by the National Academy of Sciences. All rights reserved.
Bookshelf ID: NBK609392PMID: 39585949DOI: 10.17226/27968

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