1.1 Introduction

Preamble

The World Health Organization (WHO) defines a target product profile (TPP) as the desired ‘profile’ or characteristics of a target (pharmaceutical) product that is aimed at a particular disease or diseases. TPPs state intended use, target populations and other desired attributes of products, including safety and efficacy-related characteristics. Such profiles can guide product research and development (R&D):

• In industry, in-house target product profiles (TPPs) are used as planning tools that guide development towards desired characteristics.

• In the regulatory context, TPPs are considered as tools to frame development in relation to submission of product dossiers.

Introduction to a TPP

A Target Product Profile (TPP) is a key document which provides information to the regulatory agencies, such as the Food and Drug Administration (FDA), about the drug development process. The purpose of a TPP is to ensure that the drug development process of the manufacturer is efficient and provides all the required relevant medical, technical and scientific information for evaluating the commercial outcome of a drug. It provides the overall intent of the drug and gives the status of the drug at any given point of time in the development process.

The TPP consists of sections specific to drug labeling linking specific concepts to the drug development activities. TPP is a dynamic document and should be updated regularly to keep up with the new information and changes related to the clinical development of the drug.

TPPs may differ in their layout, technical specifications and other elements between "classical" small molecule drug candidates and advanced therapies, such as cellular and gene therapy products.