Download Legislation and Regulations defining "Rare Diseases"

Appendix 8.5.1
Legislation and Regulations defining “Rare Diseases” and “Orphan Drugs”
A. Japan
In 1993, the Japanese Ministry of Health and its Organization for Pharmaceutical Safety and
Research established regulations defining a “rare and serious disease” as one that affects fewer
than 50,000 individuals. It further defines an “orphan drug” as one that treats a “rare and
serious disease” and for which there is no alternate product or intervention.1
According to the Organization for Pharmaceutical Safety and Research, Japanese Ministry of
Health and Welfare, an “Orphan drug” must meet three conditions to be classified as such: (1)
intended to treat a rare and serious disease that affects fewer than 50,000 individuals; (2) there is
no alternative product or other intervention available or it is expected that the new product
would have a much higher efficacy or greater safety profile compared to existing drugs and
interventions; and (3) that drug has a high probability for successful development, at least on a
theoretical basis; that is it must appear feasible that the development plan, if followed, can lead
to an approved drug product.2
B. Australia
The Therapeutic Goods Administration, Department of Health and Aging, of the Australian
Government, has set the following regulations concerning “orphan drugs”:
Statutory Rules 1997 No. 399, Therapeutic Goods Regulations (Amendment):
"16H. (1) A drug, vaccine or in vivo diagnostic agent is an orphan drug if it complies with this
regulation.
"(2) It:
(a) must be intended to treat, prevent or diagnose a rare disease; or
(b) must not be commercially viable to supply to treat, prevent or diagnose another disease or
condition.
"(3) It is not an orphan drug if any of the following persons or bodies has refused to approve it
for use for the disease for a reason related to the drug's safety:
(a) the Secretary;
(b) the Food and Drug Administration of the United States of America;
(c) the Medicines Control Agency of the United Kingdom;
(d) the Bureau of Pharmaceutical Assessment of Canada;
(e) the Medical Products Agency of Sweden;
(f) the Medicines Evaluation Board of the Netherlands;
(g) the European Agency for the Evaluation of Medicinal Products.
"(4) It is not an orphan drug if it has been registered for use for the disease or condition before 1
January 1998.
"(5) However, it may be registered before 1 January 1998 for another use or indication.
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Appendix 8.5.1
C. European Union
Definition of “Orphan Medicinal Product”:
From the European Parliament and Counsel of 29 April 1999: European Agency for the
Evaluation of Medicinal Products, Committee for Orphan Medicinal Products. The regulations
state, “A medicinal product shall be designated as an orphan medicinal product if its sponsor
can establish: (a) that it is for the diagnosis, prevention, or treatment of a life threatening or
chronically debilitating condition affecting not more than 5 in 10,000 persons in the Community
when the application is made, or that it is intended for the diagnosis, prevention or treatment of
a life threatening seriously debilitating or serious and chronic condition in the Community and
that without incentives it is unlikely that the marketing of the medicinal product in the
Community would generate sufficient return to justify the necessary investment and (b) that
there exists no satisfactory method of diagnosis, prevention or treatment of the condition in
question that has been authorized in the Community of, if such method exists, that the
medicinal product will be of significant benefit to those affected by that condition.
D. Korea
The Korea Orphan Drug Center (KODC) is a non-profit organization established in accordance
with Pharmacy Act 72-12 of the Republic of Korea. The center was incepted with the Korean
government funding, after there was a growing the social consensus to increase the chance of
overcoming rare disease and to improve the quality of life. To achieve these goals, the center
supplies the medications needed for diagnosis and treatment to the patients suffering from rare
disease to ensure they have access to the best treatment possible. In addition, KODC collects the
latest information on rare disease and the medications for them, builds the database based on it
and provides the patients, the medical professionals the much-needed information and the
medicines for rare disease to contribute to the improvement of the healthcare to the public.
E. Singapore
The following is a statement of Singapore’s policy regarding “Rare Diseases” and “Orphan
Drugs”
From www.Orphanet.org:
The orphan drugs policy in Singapore is based upon a Medecine Order ("Orphan drugs
Exemption"). The legislation, which came into force at the end of 1991, gave a definition of
orphan drugs and of the legal framework for imports in Singapore.
A rare disease is defined as a life threatening and severely debilitating illness.
An orphan drug is a medicinal product which has been identified by any doctor or dentist as an
appropriate and essential remedy with no effective substitute for the treatment of a rare disease.
The product should not hold a previous product licence under the Medicine Act and should be
approved by the competent Health Authorities either from the country of origin or from any
other country where the orphan drug has been used.
Orphan drugs importers must maintain proper records, including:
The quantity imported or supplied
The date of reception or supply
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Appendix 8.5.1
The name and address of the person for whom the orphan drug is provided
In addition, any other drug imported shall be kept in a hospital and be under the charge and
control of a " custodian " who must be a physician, dentist or pharmacist appointed by the
hospital.
Any doctor or dentist who requires an orphan drug for the treatment of his/her patient who is
suffering from a rare disease may request the custodian to provide him with the drug.
So far, there has been no other incentive, such as marketing exclusivity or subsidies in the
orphan drug policy.
References
1
2
Report of Department of Health and Aged Care, Canberra, 12/2001, p.46.
Report of the Dept. of Health and Aged Care, Canberra, 12/2001, page 46
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