Japan’s new drug approval process is managed by the Pharmaceuticals and Medical Devices Agency (PMDA), which works under the Ministry of Health, Labour, and Welfare (MHLW). Here’s an overview of how a drug gets approved in Japan:

1. Preclinical Testing

Before human trials begin, the drug must undergo lab and animal tests. These studies help determine whether the drug is safe and shows potential effectiveness.

2. Clinical Trials

Clinical trials involve testing the drug on humans, which happens in three phases:

Phase I: Small studies on healthy volunteers to understand the drug’s safety and how the body processes it.

Phase II: Trials on a group of patients to see how well the drug works and to check for side effects.

Phase III: Large-scale trials with more patients to confirm the drug’s effectiveness and monitor for any additional risks.

3. New Drug Application (NDA)

Once the trials are successful, the pharmaceutical company submits an application (NDA) to the PMDA. This includes all research data, safety and efficacy results, proposed labels, and details on how the drug will be made.

4. Regulatory Review by PMDA

The PMDA then reviews the application thoroughly. They check:

• • Quality: Is the drug made consistently and up to standard?
  • Safety: What risks or side effects are involved?
  • Efficacy: Does it work as intended?
• The agency may also inspect the manufacturing facilities and consult with the company to ensure compliance with Japan’s standards.

5. Final Approval by MHLW

• If the PMDA gives the green light, the application is sent to the Ministry of Health, Labour, and Welfare (MHLW) for final approval. Once approved, the drug is listed under Japan’s National Health Insurance (NHI) system and given an official price.

6. Post-Marketing Surveillance

• After a drug is approved and available to the public, it’s still closely monitored for long-term safety and effectiveness through post-marketing studies. The PMDA collects ongoing safety data and any reports of adverse effects.

Fast-Track Approvals

• Japan also offers faster approval processes for certain drugs that address urgent health needs:
  • Sakigake Designation: A priority pathway for groundbreaking drugs.
  • Conditional Early Approval: Allows drugs to be approved early based on limited data in cases of severe need.
  • Orphan Drug Program: Special incentives for drugs that treat rare diseases, such as extended market exclusivity and financial benefits.