Gaining knowledge about the orphan dug regulations in Japan

On 1 October 1993, the Japanese government revised the pharmaceutical law by acquainting special provisions relative with research and improvement of orphan drugs.

Consistent with these new provisions, MHLW Orphan drug status might be conceded to a drug, given it fulfils the accompanying two criteria:

·         The disease for which use of the drug is guaranteed must be hopeless.

·         There must be no possible elective treatment; or the viability and wanted safety of the drug must be superb in comparison with other accessible drugs.

·         The amount of patients influenced by this disease in Japan must be less than 50 000 on the Japanese region.

·         This corresponds to a maximal frequency of four for every ten thousand.

·         There is a significant extent of multinational companies, including Japanese companies that market orphan drugs.

·         Yet small and medium companies represent the most critical a piece of suppliers.

As in the USA, the Japanese orphan drugs and CRO Regulatory system offered new opportunities both for multinational and small-size and medium-size companies. Unexpectedly, open institutes and universities, and biotechnology companies are less animated than in the USA.

Orphan drugs labelling and legitimate status

The MHLW Orphan drug status is conceded by the Ministry of Health, Labour and Welfare (MHLW). Scientific examination is in the hands of a subcommittee of the Medicinal Products Committee. Conclusions are sent to special trustees.

Keeping in mind the end goal to receive orphan designation, the sponsors must submit the accompanying information to the Authorities:

·         Estimated size of patient populace 

·         Non-clinical and early phase clinical study

·         Improvement convention

The orphan drug status that has been conceded may be withdrawn if the conditions of the licence are no more satisfied.

Incentives to orphan drugs providers in term of R&D, licensed innovation and promoting

Orphan drugs and CRO Regulator profit from a fast-track Marketing Authorisation method. Specifically, the law requires necessity of assessment of applications made for indications concerning uncommon diseases. Notwithstanding this crucial measure, the Organisation for Pharmaceutical Safety and Research provides pharmaceutical companies starting orphan drugs. They do this with a consultation on improvement protocols and some guidance concerning the arrangement of support applications.

Japan Orphan Medical Device/Drug Designation – Criteria

In Japan, medical devices and drugs can be chosen as orphan medical devices or drugs as per the Pharmaceutical Affairs Clause under Article 77-2(PDF: 87KB). The only condition is that it should be used in Japan on less than Fifty Thousand patients. In Japan Orphan designation of medical devices and drugs are done by MHLW based up on the view of PAFSC.

MHLW designates medical devices and drugs only after it thinks that they are applicable and match best with some underlined criteria. Once the applicants give an application for the orphan designation, they are designated. The MHLW Orphan designation based on following criteria:

(1)   How many patients? In Japan, the total number of availability of patients about to use the medical device or drug should not exceed 50,000. The estimation of total patients should be done on the basis of the data that has been published by credible scientific societies or the report derived from Health & Labor Science Research. It is more difficult to estimate total patient population than to treat them. Lack in research work leads to inaccurate calculation of the patient population. Thus, vivid statistical data are used for indicating that total patients are not more than 50,000.

(2)   Medical requirements – The medical devices or drugs should be designated for treating serious diseases, together with diseases that are not easy to treat. In addition to this, those orphan medical devices or drugs must be used only when there is need of high medication. The criteria are mentioned below:

·         There isn’t any alternative medical device/drug or treatment.

·         Safety or high efficacy is expected when compared with the existing products.

(3)   Development Possibility – As per the clause, the orphan medical device or drug should be used to cure the targeted disease when there is valid theoretical rationale and also there should be appropriate development plan.

Since 2006, applicants are eligible for applying for the orphan medical device/drug designation along with subsequent new drugs, only on the ground that the total patient using the drug or medical device is not more than 50,000 and comes under below mentioned situations.

·         If through normal vaccine life taking disease, that is reported only overseas or rarely found in Japan, cannot be prevented.

A normal vaccine unable to cure emerging infectious disease linked up with hereditary mutation, then in such case they are brought into use.