Essential Information Before Requesting a Nonclinical Study

Types of Non-clinical Studies

In Japan, nonclinical studies are generally classified into three categories: pharmacodynamic studies, safety studies, and pharmacokinetic studies.

Pharmacodynamic studies are essential for demonstrating a drug’s intended therapeutic effect and can include sub-studies to identify efficacy and mechanism of action. Safety studies evaluate potential adverse effects and toxicity and are used to determine safe dosage levels. Pharmacokinetic studies investigate absorption, distribution, metabolism, and excretion (ADME) to understand how a drug behaves in the body.

These studies are fundamental to new drug development and regulatory approval.

Animal Models Used in Non-clinical Studies

In new drug development, nonclinical studies using animal models are highly important. Species are selected based on disease manifestations and ease of management/handling. Animal studies are conducted under applicable regulations, with attention to the experimental environment and ethical considerations. This framework enables high-precision evaluation of a new drug’s safety and efficacy. For example, mice and rats are widely used because they are comparatively easy to handle.

Compound Libraries for Identifying Candidate Compounds

Candidate compounds are identified in the early stages of drug development. From a compound library, compounds expected to act on disease-related targets are selected; after detailed evaluation and refinement, final drug candidates are chosen. Efficient, accurate evaluation across a large number of compounds is directly linked to the success of new drug development.

Disease Models Used in Nonclinical Studies

Disease models are animal models developed to reproduce a specific disease or condition. Using disease models in nonclinical efficacy evaluations enables experiments in animals that exhibit symptoms and disease states similar to those of patients. Such models allow a more realistic assessment of a new drug’s efficacy, improving the accuracy of treatment-effect prediction.

Path to New Drug Development and the Clinical Trial Process

New drug development involves a complex process. Clinical trials are typically divided into three phases and are conducted at medical institutions under strict criteria. With informed consent from participants, these trials play a vital role in confirming a new drug’s efficacy and safety. In general, more than 10 years are required from discovery of a candidate to new drug approval. This process is essential for ensuring quality and protecting patient safety.

What is GCP?

GCP establishes standards for the conduct of clinical trials following nonclinical studies and is designed to ensure ethical and scientifically appropriate trials. It specifies trial procedures, safety oversight, and documentation management. Compliance with GCP helps ensure the validity and reliability of clinical trials.

What is GLP?

GLP sets standards to ensure the reliability of nonclinical studies. From the standpoints of accuracy, comprehensiveness, and retention, GLP prescribes requirements for facility and equipment control, organizational structure, and record keeping at testing facilities. Observing GLP is important for global development and for facilitating efficient regulatory review of pharmaceuticals.