What is GCP?

GCP is a ministerial ordinance that sets standards for conducting clinical trials of pharmaceutical products. It establishes requirements for the proper conduct of clinical trials in Japan. Issued by the Ministry of Health, Labour and Welfare (MHLW), it compiles international rules for drug development and governs the development stage following nonclinical studies.

GCP and Clinical Trials

Article 2, Paragraph 6 of the Pharmaceutical Affairs Law defines “controlled medical devices.” These are devices that (1) are not classified as highly controlled medical devices, (2) may affect human life or health if adverse effects or functional failures occur, and (3) require appropriate management. The Minister of Health, Labour and Welfare designates such devices after hearing opinions from the Pharmaceutical Affairs and Food Sanitation Council.

Laws pertaining to Clinical Trials

Law on Controlled Medical Devices

Article 2, Paragraph 6 of the Pharmaceutical Affairs Law defines controlled medical devices. Controlled medical devices are medical devices with the following characteristics: "medical devices other than highly controlled medical devices," "have the potential to affect human life and health in the event of side effects or functional disorders," and "require appropriate management. The Minister of Health, Labor and Welfare designates them after hearing the opinions of the Pharmaceutical Affairs and Food Sanitation Council.

Although not as strictly regulated as highly controlled medical devices, these products present a certain level of risk and require appropriate management. The regulatory framework for ensuring the safety and effectiveness of such devices is established under Article 2, Paragraph 6 of the Pharmaceutical Affairs Law.

Law Concerning Approval for the Manufacture and Sale of Pharmaceuticals, Quasi-Drugs and Cosmetics

Article 14, Paragraph 3 of the Pharmaceutical Affairs Law stipulates that an applicant seeking approval to manufacture and sell a pharmaceutical, quasi-drug, or cosmetic must submit an application that includes materials specified by an MHLW ordinance, such as clinical trial results.

When the product is designated by an MHLW ordinance, the submitted materials must be collected and prepared in accordance with the standards specified by that ordinance.

Key Points for Clinical Trial Design in Compliance with GCP

GCP compliance improves the reliability of clinical trial results and facilitates regulatory approval. It also strengthens corporate credibility and can expand partnership opportunities. Below are particularly important points to review.

Data Quality Control

GCP compliance ensures the reliability of clinical trial data. Reliable data are essential to earn the trust of regulators and investors. High-quality data accurately assess the efficacy and safety of investigational drugs, help prevent unexpected issues, and reduce development risk in later stages.

Proper data management under GCP also improves trial efficiency and can reduce development costs—especially important for ventures operating with limited resources.

Ethical Consideration

GCP requires that the rights, safety, and welfare of individuals participating in clinical trials be given the highest priority. Informed consent must be obtained freely and voluntarily, ensuring respect for self-determination and the ethical conduct of the study.

GCP also requires strict protection of participants’ personal information. Protecting information is essential to maintain patient trust, and measures such as data anonymization are used.

Compliance with GCP ensures rigorous review by internal and external ethics committees, supporting both scientific validity and ethical legitimacy. GCP further requires proper recording and retention of clinical trial data and disclosure as necessary, which increases research transparency and helps prevent ethical issues.

Strategies by Study Phase

GCP requires an approach suited to each phase. In Phase I, participant safety is the primary focus. Because this is the first administration of an investigational drug to humans, safety and pharmacokinetics are studied in a small number of healthy participants. Compliance with GCP supports sound study design, accurate data collection, and rapid response to unexpected adverse events, with participant safety as the highest priority.

Phase II is the critical stage for exploring efficacy, confirming safety, and determining dose and regimen. Phase III verifies efficacy, safety, and dosing in a larger patient population.

A phase-appropriate strategy enables an efficient development process, strengthens data reliability, and facilitates communication with regulators—helping organizations achieve strong results with limited resources and maintain competitiveness.

Steps in Moving from Nonclinical to Clinical Trials

Role of Nonclinical Testing

This phase confirms an active ingredient’s safety and potential efficacy in animal models and in vitro. Based on these data, the drug’s efficacy is projected and the program advances to clinical trials. Toxicity and safety pharmacology studies are conducted to anticipate human safety, helping to minimize risk during clinical development.

Nonclinical studies also characterize the drug’s behavior in the body—absorption, distribution, metabolism, and excretion (ADME)—which informs appropriate dose selection and route of administration.

What to Address When Moving into Clinical Trials

When preparing a clinical trial plan based on GCP, it is necessary to have an appropriate data management and monitoring system in place. It is important to create a data management plan (DMP) and clearly define how data will be collected, processed, and stored. A quality management system (QMS) should also be in place and a risk-based approach should be used to identify processes and data that are critical to protecting human subjects and ensuring the reliability of study results. It is also important to develop a monitoring plan and implement a PDCA cycle to continuously evaluate and improve the quality of the study. Such a system will enable the conduct of high-quality clinical trials as required by the GCP Guidance.

Conducting Nonclinical Studies with an Understanding of GCP

GCP is the ministerial ordinance that defines standards for conducting clinical trials of pharmaceuticals. Understanding and following GCP expectations supports the safety of clinical trials and the accuracy of clinical data.