Regulatory Aspects and Approval of Biopharmaceuticals for Mucosal Delivery: Quality, Toxicology, and Clinical Aspects

  • Karen Brigitta Goetz
  • Yuansheng Sun
  • Katrin Féchir
  • Evelyne Kretzschmar
  • Isabel Buettel
Chapter
First Online:

Abstract

Regulation of products for mucosal application has many points in common with products for other routes but also some that are unique. Identifying regulatory pitfalls is of tremendous importance early in development as this saves time, money, and nerves. There is a huge amount of regulatory advice available from the different players in the field (governments and related organizations) that is partly very general or exceptionally specific. This chapter is meant to provide guidance on what to find, where and who to ask if certain questions arise. It also provides a description of common and known problems differentiated by the three major parts: quality, preclinical, and clinical development. Finally, special key points focusing on pediatric use, use of genetically modified organisms or products classified as advanced therapy medicinal products are discussed.

Keywords

Comparability Pharmacopoeia Genetically modified organism Advanced therapy medicinal products Toxicology First-in-human Effectiveness Safety Pediatric 

Abbreviations

BA

Bioavailability

BE

Bioequivalence

EMA

European Medical Agency

ERA

Environmental risk assessment

FDA/CBER

Food and Drug Agency/Center for Biologics Evaluation and Research

FBS

Fetal bovine serum

GMO

Genetically modified organism

ICH

International Conference of Harmonization

IMP

Investigational medicinal product

MABEL

Minimal anticipated biological effect level

NOAEL

No observed adverse effect level

PCR

Polymerase Chain Reaction

PD

Pharmacodynamics

PDCO

Pediatric Committee

Ph. Eur.

European Pharmacopoeia

PIP

Pediatric investigation plan

PK

Pharmacokinetics

USP

United States Pharmacopoeia

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Copyright information

© Springer Science+Business Media New York 2014

Authors and Affiliations

  • Karen Brigitta Goetz
    • 1
  • Yuansheng Sun
    • 1
  • Katrin Féchir
    • 1
  • Evelyne Kretzschmar
    • 1
  • Isabel Buettel
    • 1
  1. 1.Paul-Ehrlich-Institut, Federal Institute for Vaccines and BiomedicinesLangenGermany

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