Orphan drugs in Japan
- Public policy on orphan drugs in Japan
- Orphan drugs labelling and legal status
- Incentives to orphan drugs providers in term of R&D, intellectual property and marketing
On 1 October 1993, the Japanese government revised the pharmaceutical law by introducing special provisions relative to research and development of orphan drugs.
According to these new provisions, orphan drug status can be granted to a drug, provided it fulfills the following two criteria :
- The disease for which use of the drug is claimed must be incurable. There must be no possible alternative treatment; or the efficacy and expected safety of the drug must be excellent in comparison with other available drugs.
- The number of patients affected by this disease in Japan must be less than 50 000 on the Japanese territory, which corresponds to a maximal incidence of four per ten thousand.
There is a significant proportion of multinational companies, including Japanese companies that market orphan drugs. Yet small and medium companies account for the most important part of suppliers.
As in the USA, the Japanese orphan drugs system offered new opportunities both for multinational and small-size and medium-size companies. On the contrary, public institutes and universities, and biotechnology companies are less active than in the USA.
Limit of incidence for a rare condition : 4 / 10 000
The orphan drug status is granted 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 a special committee.
In order to recieve orphan designation, the sponsors must submit the following data to the Authorities :
- Estimated size of patient population ;
- Non-clinical and early phase clinical study ;
- Development protocol.
The orphan drug status that has been granted may be withdrawn if the conditions of the licence are no longer fulfilled.
The Japanese government's support for research and development orphan drugs can be found at two levels :
- The administrative level
Orphan drugs benefit from a fast-track Marketing Authorisation procedure. In particular, the law requires priority of evaluation of applications made for indications concerning rare diseases.
In addition to this measure, the Organisation for Pharmaceutical Safety and Research provides pharmaceutical companies launching orphan drugs with a consultation on development protocols and some advice concerning the preparation of approval applications.
The registration validity period, which varies from four to six years for traditional drugs is extended to 10 years for orphan products.
- The financial level
Some government funds, such as the Drug Fund for Side-Effects Relief and Research Promotion, are available.
These funds guarantee financial assistance to cover a proportion of the expenditure devoted to research and development of orphan drugs.
Funding also covers scientific activities and the provision of advice in terms of development, notably concerning clinical trials.
The Japanese authorities reimburse up to 50% of the development costs. This measure costed to Y2bn from 1993 to 1997. The support for individual drugs ranged from Y4m to Y72m per compound, with an average of Y16m.
In addition, a 6% tax reduction for Research and Development expenses is granted, other than those coming from funding grants and within the limit of 10% of company tax.
Companies making profits on sales of orphan drugs must return a proportion of the subsidy granted as a contribution to these funds.
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