FDA ResourcesEMA Resources
Trial Design
  • Adaptive Designs for Clinical Trials of Drugs and Biologics—FINAL Guidance (FDA, 2019a)
  • Master Protocols: Efficient Clinical Trial Design Strategies to Expedite Development of Oncology Drugs and Biologics—FINAL Guidance (FDA, 2022d)
  • Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics—FINAL Guidance (FDA, 2024h)
Trial Design
  • Complex Clinical Trials—Questions and Answers (EMA, 2022a)
  • IHC-E20 Adaptive Clinical Trials—FINAL Guidance (ICH, 2019)
  • Concept Paper on Platform Trials (EMA, 2022b)
Decentralized Trials
  • E17 General Principles for Planning and Design of Multiregional Clinical Trials—FINAL Guidance (FDA, 2018e)
  • Decentralized Clinical Trials for Drugs, Biological Products, and Devices—DRAFT Guidance (FDA, 2023g)
Decentralized Trials
  • IHC-E17 General Principles for Planning and Design of Multi-regional Clinical Trials—FINAL Guidance (EMA, 2017b)
FDA ProgramsEMA Programs
  • Complex Innovative Trial Design Paired Meeting Program (FDA, 2023c)
    • Interacting with FDA on Complex Innovative Trial Designs for Drugs and Biological Products—FINAL Guidance (FDA, 2020b)
  • Model-Informed Drug Development Paired Meeting Program (FDA, 2024p)
  • Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program (FDA, 2024u)

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

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.
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; Shore CK, Worku TL, Smith CW, et al., editors.
Washington (DC): National Academies Press (US); 2024 Oct 30.
Copyright 2024 by the National Academy of Sciences. All rights reserved.

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