How orphan drug designation is given in Japan

In the previous articles we have studied orphan drugs and now we are going to learn about how orphan drug designation is given in Japan, we are going to discuss how and what are the laws guiding orphan drug designation in Japan.

Criteria for orphan drug designation in Japan:  In Japan orphan drug designation is given based on the prevalence of the disease like less than 50000 patients, and high medical need, even though other treatments are available, the significant benefit is considered.

Laws guiding orphan drug designation in Japan:  Article 77-2 of the Pharmaceutical Affairs Law Act governs the orphan drug designation for medical devices, regenerative and cellular therapy, gene therapy, and cosmetics. MHLW gives designation based on the opinion of PAFSC

Designation criteria in detail – Ministry of Health Labour and Welfare will give orphan drug status based on the following

1. Number of Patients – should be less than 50000 in Japan, estimation in Japan is done based on report of Health and labor science research or other reports
2. Medical need – seriousness of the condition, if no alternative present, high efficacy when compared to other products.
3. Possibility of development based on non-clinical and clinical data

How to Apply for Orphan Drug Designation:

Steps 1- Consultation phase

Step 2- Application

Step 3 -Evaluation and decision by MHLW

Consultation Phase:

1. Submission of consultation application to Pharmaceutical and Food Safety Bureau, MHLW
2. Consultation Date notified by MHLW
3. Consultation typically 30 min session

Designation Process

1. Application submission in original to evaluation and licensing division, Pharmaceutical & Food safety bureau, MHLW – application should be complete, incomplete and those which doesn’t meet criteria are not accepted and application must be submitted in Form 107
2. MHLW review for eligibility, preliminary evaluation by PMDA, further evaluation by pharmaceutical affairs and food sanitation council.
3. PAFSC sends opinion to MHLW
4. Final decision by MHLW and designation notice
5. Mere designation of orphan drug status does not automatically lead to marketing approval.
6. Once designation is granted MHLW publish it in govt gazette as MHLW ministerial notification.

Contents of Application:

1. Application
2. Data on number of patients
3. Data on medical needs
4. Rationale for use of drug or medical device
5. Related data available at time of application
6. Development plan
7. Summary of orphan drug or medical device