How New Drugs are Developed

New drugs, or " new drugs," are launched after a long period of research and development, and after the efficacy and safety of the new ingredients are confirmed, they are approved by the government.

In Japan, it is said that it takes approximately 9 to 16 years and a large amount of money to develop a single "new drug. Even with that much time and money spent, the success rate of new drug development is only about 1 in 31,000*. Pharmaceutical companies that develop new drugs are obliged to continue research and reconfirm the efficacy and safety of the drug after a certain period of time has elapsed since its launch (reexamination). Source: "Pharmaceutical Manufacturers' Association Guide 2025.

It takes about 9 to 16 years and a large amount of money to create a new drug.

Step 1 of new drug development Basic research (2-3 years)
Discovery and manufacture of a new substance that will become the basis of a drug

The development of a new drug begins with research to discover or chemically create a new substance (ingredient) that has the potential to become a drug in the future. The motto of a drug is discovered from natural materials (plants, animals, microorganisms, etc.), created by combining two or more substances, or by using various scientific techniques.
New substances are further examined and sifted over time to select only those with the potential to become drugs.

Drugs that reduce the burden on patients (kanja) and their families The development of new drugs reduces the physical and financial burden on patients and their families, as well as the labor required to care for them, and contributes to reducing medical costs.

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  The development of immunosuppressive drugs dramatically increased the success rate of organ transplants.
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  Drugs to slow the progression of Alzheimer's disease have been developed, reducing the labor and cost of nursing care.
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  The use of proteins produced by living organisms has led to new treatment options for rheumatoid arthritis and improved the quality of life (QOL*) of patients. QOL: Quality of Life

The source of a drug is discovered from natural materials such as plants and microorganisms, or created using scientific technology.

Step 2: Non-clinical studies (3-5 years)
Research on Efficacy and Safety of New Substances

Potential drug substances are tested on animals and cultured cells to study their efficacy and safety.
We also study the absorption, distribution, metabolism, and excretion processes of drug substances, as well as their quality and stability.

Japan ranks 6th in the world in new drug development The technology used to develop new drugs is so advanced and complex that there are only a few countries in the world that have created new drugs. Looking at the world's top 100 drugs in terms of sales by country of origin, Japan boasts the world's sixth largest drug discovery capability, with eight drugs born in Japan.

Japan is the 6th largest creator of new drugs in the world!

Source: Copyright&copy2025 IQVIA. IQVIA World Review Analyst, Data Period 2023, IQVIA Pipeline & New Product Intelligence, Evaluate Pharma® (as of November 2024) Prepared by the Pharmaceutical Industry Policy Institute based on Clarivate Cortellis Competitive Intelligence (All rights reserved).

Source: Policy Research Institute News No. 74 (March 2025), Pharmaceutical and Industrial Policy Research Institute

Note: Classification by nationality of companies to which patents belong.

The use of a drug substance in animals or cultured cells to test its efficacy and safety is called a non-clinical study, while the use of a drug in humans is called a clinical study.
For more information on clinical trials, please refer to Step (3) of New Drug Development.

Step 3 Clinical trials (3-7 years)
Efficacy and safety testing on human subjects

Clinical trials (Chiken) are the final confirmation that a drug candidate ( investigational new drug ) that has passed the necessary non-clinical trials is safe and actually effective in humans.
Clinical trials are conducted at various stages in accordance with laws and based on reviews and examinations by many experts.
Prior to a clinical trial, medical institutions are required to provide detailed explanations in writing to patients who are to cooperate in the trial, to ensure that they fully understand the details, and to obtain their written consent (called "informed consent"). (Informed Consent).

Clinical trials are important trials supported by the cooperation of patients. Clinical trials are divided into the following three stages and are conducted at hospitals and other medical institutions with the consent of healthy people and patients.

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  Target:
  A small number of healthy people / Safety of side effects, etc. will be checked
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  Target:
  A small number of patients / Confirmation of effective and safe dosage, administration method, etc.
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  Target:
  Many patients/comparison of efficacy and safety of the drug with standard drugs used up to now.

Clinical trials are conducted based on strict standards known as GCP (*). GCP: Good Clinical Practice (standards for conducting clinical trials of drugs)

Step 4 Application for approval and review (approx. 1 year)
Application for approval to the Ministry of Health, Labour and Welfare (MHLW) and review by experts

After efficacy, safety, quality, etc. are proven in various trials, notification is submitted to the Ministry of Health, Labour and Welfare to obtain permission to manufacture and sell the drug as a new drug.
The drug is then reviewed by the Pharmaceutical Affairs and Food Sanitation Council, a body composed of academic experts and others, and if it is approved as a "drug," it can be manufactured and sold.

Post-launch safety and usage checks As a result of the use of the drug by many patients at medical institutions, information on side effects and proper usage that could not be detected during the development stage is collected by medical representatives (MRs*) of pharmaceutical companies. In this way, various checks are obligatory even after a drug is put on the market. MR: Medical Representative

After a drug is put on the market, it undergoes various checks and improvements to make it safer and easier to use.