Rare Disease and Orphan Drug Act

Article 1

This Act is enacted for the prevention and treatment of rare diseases; for the early diagnosis of these diseases; for the intensive care of rare disease patients; for assisting patients gaining access to specific pharmaceuticals for the treatment of these diseases and to special nutrient foods essential to the maintenance of life; and for promoting and ensuring the supply, manufacturing, and research and development of such pharmaceuticals and foods.

For matters not regulated in this Act, regulations of other relevant Acts will apply.

 Article 2

The competent authorities stipulated in this Act shall be the Department of Health of the Executive Yuan at the central government level; the municipal governments at the municipality level; and the county and city governments at the county and city level.

Article 3
The rare diseases specified in this Act refer to diseases with prevalence lower than that formulated and publicly announced by the central competent authority, and recognized by the Committee specified in Article 4 of this Act; or diseases designated and publicly announced by the central competent authority under special circumstances.

Orphan drugs specified in this Act refer to pharmaceuticals with major indications for the prevention, diagnosis and treatment of rare diseases that have been duly submitted in accordance with this Act for application and have been publicly announced by the central competent authority.

Article 4
To review and evaluate matters related to rare diseases, the central competent authority shall set up a Committee for the Review and Examination of Rare Diseases and Orphan Drugs (hereafter referred to as the Committee). The organization and meetings of the Committee shall be regulated by the central competent authority.

Members of the Committee shall consist of representatives of government organizations, scholars and specialists in medical affairs, and impartial citizens. At least two-thirds of the members of the Committee shall be scholars and specialists in medical affairs.

Article 5
Missions of the Committee are:

        1. Identification and control of rare diseases;
        2. Review and approval of orphan drug application;
        3. Testing, marketing & registration of orphan drugs;
        4. Review and examination of funding, research and development of orphan drugs;
        5. Review and examination of the diagnosis, treatment, or international cooperation plans for rare               diseases, and issues concerning assistance and counseling;
        6. Review and examination of the use of non-orphan drugs for the treatment of some specific                       diseases; and
        7. Other relevant issues.

In the execution of the aforementioned missions, the Committee shall consult other relevant scholars, specialists and representatives of industry or patients of rare diseases for their opinions.

 Article 6
 The central competent authority may entrust institutions with projects concerning the control and  research of rare diseases.

   
 Article 7
 Medical personnel, upon identifying patients of such rare diseases specified in Article 3, or human  remains died of such diseases, shall report to the central competent authority.

 
Article 8
The central and the municipal competent authorities, upon receiving reports or on identification of persons with genetic anomalies of rare diseases, may, if need be, dispatch specialists from genetic health counseling centers for interviewing. They shall tell patients the relevant effects of the disease.

   
   Article 9

Organizations, institutions, and personnel authorized by this Act for the execution of functions specified in Article 8 and Article 9 shall be attentive to their attitude and methods of execution. They shall respect the will and dignity of the patients. They shall also protect the privacy and normal social functioning of the patients.

   Article 10

The central and the municipal competent authorities shall encourage medical care institutions and research institutions at all levels to conduct the prevention and control of rare diseases; and provide them funding for manpower development, research, and facilities.

Funds required for the aforementioned subsidies shall come from budgets of the central and the municipal competent authorities. Donations from organizations and groups concerned will also be solicited.

Article 11  
The central and the municipal competent authorities shall organize and hold educational activities for the prevention and control of rare diseases. These activities will be conducted with the assistance of organizations, schools, civil groups, and mass media.
 
 Article 12 
The central and the municipal competent authorities may entrust medical care institutions with the testing and treatment of rare diseases.
 
 Article 13  
The central competent authority may assist institutions, reviewed and approved by the Committee with the following certificates, in their international medical cooperation programs:

        1. certificates, applications, and medical care plans prepared by the medical care institutions or                  research institutions specified in Article 10 or Article 12 of this Act;

        2. certificates or letters of consent issued by other national hospitals or research institutions;
        3. letters of consent signed by patients;
        4. other necessary documents.

Items for the laboratory testing of the aforementioned medical cooperation programs may be done directly by the medical care institutions or research institutions specified in Article 10 or Article 12 of this Act. The central competent authority shall appropriately subsidize their costs.
 
 Article 14 
 Unless otherwise regulated by this Act, orphan drugs shall not be manufactured or imported without the registration, market approval, and permit licenses issued by the central competent authority.
 
 Article 15  
Pharmaceuticals meeting one of the following criteria may apply for registration and market approval as orphan drugs.

        1. The major indications of the medicinal products are for the prevention, diagnosis, and treatment              of rare diseases.
        2. Medicinal products that have been approved by other countries to have major indications for the             prevention, diagnosis, and treatment of rare diseases.

Documents required for registration and market approval, review procedures, and other relevant issues shall be regulated by the central competent authority.
 
 Article 16
In the application for registration and market approval of orphan drugs, the central competent authority may, if necessary, require the conduct of domestic clinical trials. Applicants shall adequately explain in public contents of the clinical trials and their results.
 
 Article 17
Permit licenses issued in accordance with the registration and market approval of this Act for orphan drugs are valid for ten years. During this period, the central competent authority will not accept applications for registration and market approval of pharmaceuticals of the same kind.

If the manufacturing or importation of the aforementioned orphan drugs need to be continued after the permit licenses expire in ten years, application for extension must be made in advance to the central competent authority. Each extension shall not exceed five years. During the extension period, medicinal products of the same kind may apply to the central competent authority for registration and market approval.

If orphan drugs which have been issued permit licenses through registration and market approval in accordance with this Act are publicly announced by the central competent authority to be no longer designated as orphan drugs, the extension of their permit licenses shall be processed in accordance with the regulations of the Pharmaceutical Affairs Acts.

If owners of licenses issued under Paragraph 1 decide to terminate the manufacturing or importation of orphan drugs within the valid period, they shall notify the central competent authority in writing six months prior to such termination.

 
 Article 18
Under either one of the following conditions, the central competent authority may, irrespective of the regulations of Paragraph 1 of Article 17, accept applications for registration and market approval of other pharmaceuticals of the same kind and issue them permit licenses.

        1. The new applicants have in their possession authorized consent from right-holders of the orphan             drugs duly issued through registration and market approval.

        2. New applications for orphan drugs of same indications and similar quality and their safety and                 efficacy have been proved to be superior to those of the orphan drugs already issued licenses.

        3. Permit license owners for orphan drugs fail to meet demands on the orphan drugs.
        4. Prices of the orphan drugs are considered unreasonable by the central competent authority.
            Regulations of the preceding Article will apply to permit licenses issued by the central competent             authority through registration and market approval under subparagraph 2 through subparagraph             4 of the preceding Paragraph.

    Article 19

Government organizations, medical care institutions, patients of rare diseases and their families, and relevant foundations, societies, and associations, may apply on ad hoc basis to the central competent authority for permission for orphan drugs not yet processed through registration and market approval, or meeting one of the conditions specified in subparagraph 3 and subparagraph 4 of Paragraph 1 of the preceding Article. These orphan drugs shall not be for profit making.

The central competent authority may, if necessary, entrust or designate relevant institutions or groups to process the aforementioned ad hoc applications.

Documents required for ad hoc applications specified in Paragraphs 1 and 2, their review procedures, and other relevant issues shall be regulated by the central competent authority.

   Article 20

When orphan drugs are proved to be unsafe or hazardous to health, the central competent authority may order pharmaceutical firms or ad hoc applicants to recall the orphan drugs in due time. If necessary, permits issued to the orphan drugs may be revoked.

   Article 21

The central competent authority shall compile annual report of orphan drugs approved by this Act for marketing or on ad hoc basis. The report shall contain information on the amount taken, number of patients, any adverse reactions, and other relevant matters.

Pharmaceutical firms and ad hoc applicants shall submit relevant information for the aforementioned report.

   Article 22

Regulations concerning registration and market approval and ad hoc application of this this Act shall apply to pharmaceuticals that are difficult to manufacture or import by regulations of the Pharmaceutical Affairs Act, but are reviewed and confirmed by the Committee to be beneficial to the medical care of certain specific diseases.

   Article 23

The central competent authority shall announce periodically the recognition, permission, revoking, and annulment of rare diseases and orphan drugs.

   Article 24

Fees for review, registration, or licenses shall be paid for applying for registration and market approval, conducting clinical trials, issuance and extension of permit licenses, and ad hoc applications, The fee schedule shall be determined by the central competent authority.

   Article 25

The central competent authority may promote the supply, manufacturing, and research and development of orphan drugs. Regulations governing the recipients of incentives, ways of promotion, and matters to be followed by the recipients shall be decided by the central competent authority.

   Article 26

Manufacturing and importation of orphan drugs without permission, or the sales, supply, dispensing, transportation, deposit, trading of stolen goods, transfer, or display for sale of orphan drugs known for not being permitted shall be punished according to the regulations of Article 82 and Article 83 of the Pharmaceutical Affairs Act.

   Article 27

Any violations of the regulations of Article 16 shall be fined NT$ 30,000 to NT$ 150,000. For serious violations, pharmaceutical firms shall be suspended from making application for registration and market approval for the said pharmaceutical for two years. Medical care institutions may be suspended for one month to one year.

   Article 28

In the case of submitting false documents for the application for registration and market approval of orphan drugs or extension of licenses for such orphan drugs, the applicants shall be fined NT$ 20,000 to NT$ 100,000, and shall be suspended from making application for registration and market approval of the said pharmaceutical for two years. Permit licenses already in the possession of the applicants shall be revoked. For their criminal responsibilities, they shall be sent to judicial organizations for further investigation.

   Article 29

Any violations of the regulations of Paragraph 1 of Article 19 to allow orphan drugs approved on ad hoc basis for profit-making, the applicants shall be fined NT$ 30,000 to NT$ 150,000; the profits thus made shall be confiscated; and the applicants shall be suspended from making any ad hoc application for orphan drugs for two years.

Article 30

If orphan drugs ordered to be recalled in due time by the central competent authority under regulations specified in Article 20 are not recalled, the pharmaceutical firms or ad hoc applicants shall be fined NT$ 30,000 to NT$ 150,000, consecutively until the orphan drugs are recalled.

Article 31

Pharmaceutical firms in violation of the regulations of Paragraph 2 of Article 21 shall be fined NT$ 10,000 to NT$ 50,000. Ad hoc applicants in violation of such regulations, their future applications for orphan drugs shall not be permitted by the central competent authority.

Article 32

Fines regulated in this Act shall be administered by the central and the municipal competent authorities.

The aforementioned fines should be paid in due time. If not, the cases shall be transferred to the judicial court for enforcement by law.

Article 33

The central competent authority shall allocate budget to subsidize costs for the diagnosis, treatment, pharmaceuticals, and special nutrient foods essential to the maintenance of life not reimbursable according to the regulations of the National Health Insurance Act. Ways of subsidies, their contents, and other relevant issues shall be determined by the central competent authority.

Funds for the aforementioned subsidies may also be solicited from relevant organizations and groups.

Article 34

Special nutrient foods essential to the maintenance of life of patients of rare diseases may be managed with reference to relevant regulations of this Act.

Article 35

The Enfercement Rules of this Act shall be formulated by the central competent authority.

Article 36

This Act shall become effective as of six months after the promulgation.

Article 37

The Executive Yuan shall review and amend this Act within six months after this Act is implemented for one year.