Regulatory Processes for Rare Disease Drugs in the United States and European Union : xibilities and Collaborative Opportunities / National Academies of Sciences, Engineering, and Medicine.

Format:

Book

Author/Creator:

National Academies of Sciences, Engineering, and Medicine (U.S.), author, issuing body.

Contributor:

National Academies of Sciences, Engineering, and Medicine

Health and Medicine Division

Board on Health Sciences Policy

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

Carolyn K. Shore

Tequam L. Worku

Carson W. Smith

Jeffrey P. Kahn

Language:

English

Subjects (All):

Drug development.

Drugs--Effectiveness.

Drugs.

Rare diseases.

Physical Description:

1 online resource (xxii, 363 pages)

Edition:

1st ed.

Place of Publication:

Washington, D.C. : National Academies Press, 2024.

Summary:

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:

The National Academies of SCIENCES • ENGINEERING • MEDICINE

COMMITTEE ON PROCESSES TO EVALUATE THE SAFETY AND EFFICACY OF DRUGS FOR RARE DISEASES OR CONDITIONS IN THE UNITED STATES AND THE EUROPEAN UNION

Reviewers

Acknowledgments

Preface

Acronyms and Abbreviations

Summary

REGULATORY FLEXIBILITIES, AUTHORITIES, AND MECHANISMS

USE OF ALTERNATIVE AND CONFIRMATORY DATA

FDA AND EMA COLLABORATION

1. Introduction

CLINICAL TRIALS FOR RARE DISEASES AND CONDITIONS

CONTEXT FOR THIS STUDY

STUDY APPROACH

REFERENCES

2. FDA Flexibilities, Authorities, and Mechanisms

DRUG REVIEW AND APPROVAL

DESIGNATION FOR RARE DISEASE PRODUCTS

EXPEDITED REGULATORY PROGRAMS

Priority Review

Regenerative Medicine Advanced Therapy

Oncology Center of Excellence Programs

Orphan Drugs and Expedited Review

INCLUSION OF PEDIATRIC POPULATIONS

STAKEHOLDER ENGAGEMENT

SELECT RARE DISEASE PROGRAMS

TRANSPARENCY

SUMMARY OF CONCLUSIONS AND RECOMMENDATIONS

3. EMA Flexibilities, Authorities, and Mechanisms

ORPHAN MEDICINE DESIGNATION

RARE DISEASE INITIATIVES

4. Alternative and Confirmatory Data

GUIDANCE ON ALTERNATIVE AND CONFIRMATORY DATA

SOURCES OF ALTERNATIVE AND CONFIRMATORY DATA

TRENDS IN REGULATORY USE

Expanding the Use of Alternative and Confirmatory Data

NOVEL APPROACHES FOR DATA ANALYSIS

Bayesian Statistical Methods

Network Meta-Analysis

Randomization-Based Inference

Quantitative Systems Pharmacology

BIOMARKERS

OPPORTUNITIES TO ENHANCE INNOVATION

5. FDA and EMA Collaboration

SIMILARITIES AND DIFFERENCES BETWEEN FDA AND EMA

COLLABORATION BETWEEN REGULATORY AGENCIES

OPPORTUNITIES FOR ENHANCED COLLABORATION

Appendix A. Biographical Sketches of Committee Members and Staff

Appendix B. Disclosures of Unavoidable Conflicts of Interest

Appendix C. Public Meeting Agendas

Appendix D. Centre for Innovation in Regulatory Science Data Analysis Methodology

Appendix E. Qualitative Interview Summary and Methodology

Appendix F. Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023

Appendix G. List of Orphan Approvals by FDA or EMA Between 2018 and 2022

Appendix H. Select Examples of Rare Disease Drug Products

Appendix I. FDA and EMA Resources, Policies, and Programs Relevant for Drug Development for Rare Diseases and Conditions.

Notes:

Description based on publisher supplied metadata and other sources.

ISBN:

0-309-72656-5

OCLC:

1474611579