Skip to main content

ICH Q8 (R2) Pharmaceutical development (CHMP/ICH/167068/04)

 ICH Q8 (R2) is a guideline titled "Pharmaceutical Development" (CHMP/ICH/167068/04). This guideline is part of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) and provides recommendations for the pharmaceutical development of medicinal products. It offers a structured approach to the development of pharmaceutical products to ensure their quality, safety, and efficacy. Here's an elaboration of ICH Q8 (R2):


1. Purpose of ICH Q8 (R2):

The primary purpose of ICH Q8 (R2) is to provide a systematic and science-based approach to pharmaceutical development. The guideline aims to facilitate the design and development of high-quality pharmaceutical products that meet the needs of patients and regulatory authorities.

2. Scope:

ICH Q8 (R2) applies to the development of all types of pharmaceutical products, including small molecules, biotechnological products, and other complex medicinal products.

3. Pharmaceutical Development Principles:

The guideline emphasizes a quality-by-design (QbD) approach to pharmaceutical development. This means that product quality is built into the development process from the beginning, with a focus on understanding and controlling the critical quality attributes (CQAs) of the product.

4. Quality Target Product Profile (QTPP):

Manufacturers are encouraged to define a Quality Target Product Profile (QTPP) early in the development process. The QTPP outlines the desired quality characteristics of the product and serves as a guide throughout development.

5. Risk Assessment:

Risk assessment is a fundamental component of ICH Q8 (R2). Manufacturers should identify and assess potential risks to product quality, safety, and efficacy and develop strategies to mitigate these risks.

6. Pharmaceutical Development Studies:

The guideline emphasizes the need for systematic pharmaceutical development studies to understand and control the variables that can impact product quality. These studies may include formulation development, process development, and analytical method development.

7. Design of Experiments (DoE):

Design of Experiments (DoE) is a statistical approach recommended for optimizing and controlling processes. It allows for the systematic study of variables and their impact on product quality.

8. Lifecycle Approach:

ICH Q8 (R2) promotes a lifecycle approach to pharmaceutical development. This means that development activities continue throughout the product's lifecycle, including post-approval phases.

9. Regulatory Considerations:

Regulatory authorities encourage early dialogue between manufacturers and regulators to discuss development plans and strategies. Transparency and collaboration with regulatory agencies are important aspects of this guideline.

10. Control Strategy:

- Manufacturers should develop a control strategy that includes specifications for CQAs, critical process parameters (CPPs), and critical material attributes (CMAs). The control strategy ensures that the product consistently meets its intended quality attributes.

11. Regulatory Filing:

- The data generated during pharmaceutical development, including information on formulation, manufacturing processes, and analytical methods, should be included in regulatory submissions to support marketing authorization.

12. Post-Approval Changes:

- The guideline recognizes that post-approval changes to a product may occur and emphasizes the importance of assessing the impact of changes on product quality and regulatory requirements.


In summary, ICH Q8 (R2) provides a structured and science-based approach to pharmaceutical development, with an emphasis on quality by design, risk assessment, and a lifecycle perspective. Compliance with these principles helps ensure that pharmaceutical products are developed and manufactured with a strong focus on quality, safety, and efficacy, leading to better patient outcomes and regulatory approval.

Popular posts from this blog

Ago2 Immunoprecipitation for RISC-siRNA Quantitation

 Ago2 (Argonaute 2) immunoprecipitation (IP) is a technique used to isolate RNA-induced silencing complexes (RISC) from cell lysates. This method allows for the specific enrichment of active RISC complexes bound to small interfering RNA (siRNA) or microRNA (miRNA) within cells. By isolating these complexes, researchers can then quantify the siRNA associated with Ago2, which is an essential step in determining the efficacy of RISC loading and siRNA activity. Here’s a detailed overview of how Ago2 immunoprecipitation is performed for RISC-siRNA quantitation: Steps in Ago2 Immunoprecipitation for RISC-siRNA Quantitation Cell Lysis and Preparation of Lysate : Sample Preparation : Collect cells that have been treated with siRNA, then wash them with cold phosphate-buffered saline (PBS) to remove extracellular contaminants. Lysis : Lyse the cells in a gentle, RNA-preserving lysis buffer that typically includes detergents (e.g., NP-40 or Triton X-100), protease inhibitors, and RNase inhibi...
Read more

Guideline on development and manufacture of lentiviral vectors (CHMP/BWP/2458/03)

The guideline with the reference number "CHMP/BWP/2458/03" pertains to the "Guideline on Development and Manufacture of Lentiviral Vectors." This guideline was developed by the Committee for Medicinal Products for Human Use (CHMP) and the Biotechnology Working Party (BWP) of the European Medicines Agency (EMA). It provides recommendations and regulatory guidance for the development and manufacture of lentiviral vectors, which are widely used in gene therapy and cell therapy applications. Here's an overview of the key points covered in this guideline: 1. Introduction: The guideline begins with an introduction highlighting the increasing importance of lentiviral vectors in advanced therapies and the need for guidance on their development and manufacture. 2. Scope: It defines the scope of the guideline, which covers the development and manufacture of lentiviral vectors intended for use in gene therapy and cell therapy products for human use. 3. Quality and Characte...
Read more

Stem-Loop PCR for siRNA

 Stem-loop PCR is a method often used for detecting and quantifying small RNAs, such as siRNA or miRNA, which are typically difficult to amplify directly due to their short lengths. The method involves the design of a stem-loop reverse transcription (RT) primer, which enhances specificity and stability of the short RNA during the RT-PCR process, allowing for sensitive detection and quantification of the siRNA. Here’s a detailed guide to how stem-loop PCR can be applied to siRNA detection: Key Steps in Stem-Loop PCR for siRNA Designing the Stem-Loop RT Primer : Structure : The stem-loop RT primer consists of a loop region flanked by complementary sequences on either side (the "stem"), which will fold back on itself to form a hairpin structure. Specific Binding Region : A short sequence complementary to the 3’ end of the siRNA is added at the end of the stem-loop primer to ensure specific binding to the siRNA target. Stabilization : The loop structure helps prevent primer-dimer...
Read more

FDA Guidance on Studying Multiple Versions of Cellular or Gene Therapy Products in Early-Phase Clinical Trials

 The purpose of this guidance is to offer advice to sponsors interested in conducting early-phase clinical trials for a single disease involving multiple variations of a cellular or gene therapy product. Sponsors aim to gather preliminary safety and efficacy data for these product variations within a single clinical trial. It's important to note that even though multiple product versions are studied together, each version is distinct and typically requires a separate investigational new drug application (IND) submission to the FDA. The primary goal of these early-phase clinical studies is to inform decisions about which product version(s) should be advanced for further development in later-phase trials. As such, these studies are not designed to provide the main evidence of effectiveness needed for a marketing application. They are generally not statistically powered to demonstrate a significant difference in efficacy between the different study arms. In this guidance, the FDA prov...
Read more

Human Genome Editing: FDA Draft Guidance Summary

Consideration for Developing Gene Editing Product  1. Genome Editing Methods: Genome editing can be achieved through nuclease-dependent or nuclease-independent methods. Nuclease-dependent methods involve introducing site-specific breaks in DNA using technologies like zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), modified-homing endonucleases, and CRISPR-associated (Cas) nucleases. These breaks can lead to modification of the DNA sequence at the cleavage site. Nuclease-independent methods can change DNA sequences without cleaving the DNA and include techniques like base editing and synthetic triplex-forming peptide nucleic acids. The choice of GE technology should consider factors such as the mechanism of action, the ability to target specific DNA sequences, and the potential to optimize components for efficiency, specificity, or stability. 2. Type and Degree of Genomic Modification: Different GE approaches rely on DNA repair pathways such a...
Read more