Rule relating to orphan drugs in Japan

Orphan drugs are those that are developed purely to treat rare diseases. The nature of orphan drugs is a little piquant: on the one hand, rare diseases affect very few people, but these cannot be ignored. On the other hand, orphan drugs, since they are so few in number, are not taken up usually by profit-driven organizations because they offer very less scope for large-scale production and monetization.

Different countries such as the US and Japan, and blocs such as the European Union have their own unique rules regarding orphan drugs. Rules relating to orphan drugs in Japan too, have their unique and salient features.

A look at the uniqueness of rules relating to orphan drugs in Japan

Orphan drugs in Japan are governed by five incentives:

• Subsidies
• Consultation
• Tax preferences
• Priority review
• Reexamination period review

Briefly, this is how each of these is carried out in practice:

Subsidies:The Japanese government has a fund of close to a billion yen that is used to subsidize orphan drug applicants. This grant, given through the National Institute of Biomedical Innovation (NIBIO), is aimed at helping manufacturers to ease the costs associated with development of orphan drugs in Japan.

Consultation:Under this system, the Japanese government offers priorities for manufacturers of orphan drugs in Japan. Called the Priority Consultation System; this process is offered in two ways:

One, when a sponsor of orphan drugs in Japan approaches the PMDA; the application is never rejected. Clinical data is discussed with each sponsor, at which all the aspects of orphan drugs in Japan are considered. Areas taken up for discussion include the number of patients, the intensity of the disease, the integrity and safety of the data, and so on.

Two, the fee category is also considerably lower for orphan drugs in Japan, with these manufacturers given about 25 percent discount. An application made for developing orphan drugs in Japan is put for consolation with a team of Technical Experts, who are selected from the Office of New Drug Review team, the Office of Cellular and Tissue-based Products Review team, and the Office of Medical Devices Review team. In addition, fee categories are drastically lower for orphan drugs in Japan when they are being developed by small companies or research institutes. These categories have a whopping 90 percent discount.

Tax incentive: Manufacturers involved in developing and manufacturing orphan drugs in Japan are given a 12 percent reduction as tax credits for expenses incurred during the NIBIO subsidy payment period.

Priority review:Manufacturers of orphan drugs in Japan are offered a priority review by the PMDA. While the median standard review time is one year for non-orphan drugs, that for orphan drugs in Japan is nine months.

Reexamination period review: The Reexamination period review for orphan drugs in Japan is also relaxed. It is fixed at eight years for orphan drugs in Japan, while this period is ten years for non-orphan drugs in Japan.

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