Unofficial translation
In accordance with paragraph 4 of Article 177 of the Code of the Republic of Kazakhstan "On Public Health and the Healthcare System" I HEREBY ORDER:
Footnote. The preamble is in the wording of the Order No. 108 of the Minister of Healthcare of the Republic of Kazakhstan dated 21.10.2025 (shall enter into force upon expiry of ten calendar days after its first official publication).1. To approve the Rules for formation of the list of orphan diseases and medicines for their treatment according to the Appendix to this order.
2. The Department for Organization of Medical Care of the Ministry of Health of the Republic of Kazakhstan, in accordance with the procedure, established by law, shall ensure:
1) state registration of this order with the Ministry of Justice of the Republic of Kazakhstan;
2) placement of this order on the Internet resource of the Ministry of Healthcare of the Republic of Kazakhstan after its official publication;
3) within ten working days after the state registration of this order, submission to the Legal Department of the Ministry of Healthcare of the Republic of Kazakhstan, of information about implementation of activities, stipulated by subclauses 1), 2) of this clause.
3. Control over execution of this order shall be entrusted to the supervising Vice-Minister of Healthcare of the Republic of Kazakhstan.
4. This order shall come into force upon expiry of ten calendar days after the date of its first official publication.
|
Minister of Healthcare of the Republic of Kazakhstan |
A. Tsoy |
| Appendix to the to the Order of the Minister of Healthcare of the Republic of Kazakhstan dated October 16, 2020 № ҚР ДСМ-135/2020 |
Rules for the formation of a list of orphan diseases and medicines for their treatment
Footnote. The rules are in the wording of the Order No. 108 of the Minister of Healthcare of the Republic of Kazakhstan dated 21.10.2025 (shall enter into force upon expiry of ten calendar days after its first official publication).
Chapter 1. General provisions
1. These Rules for the formation of a list of orphan diseases and medicines for their treatment (hereinafter – the Rules) have been developed in accordance with paragraph 4 of Article 177 of the Code of the Republic of Kazakhstan “On Public Health and the Healthcare System” (hereinafter – the Code) and shall establish the procedure for the formation of the list of orphan diseases and medicines for their treatment (hereinafter – orphan medicinal products).
2. The following terms shall be used for the purposes of these Rules:
1) an authorized body in the field of healthcare (hereinafter – the authorized body) means the central executive body responsible for leadership and intersectoral coordination in the field of the health protection of citizens of the Republic of Kazakhstan, medical and pharmaceutical science, medical and pharmaceutical education, sanitary and epidemiological welfare of the population, circulation of medicines and medical devices, and the quality of healthcare services (medical care);
2) orphan (rare) diseases mean rare severe diseases that are life-threatening or resulting in disability, the prevalence of which does not exceed the officially established threshold.
3) a working body means a healthcare organization responsible for organizational and methodological guidance, coordination, monitoring, and expert support in the field of diagnosis, treatment, prevention, and registration of orphan (rare) diseases within the territory of the Republic of Kazakhstan.
Chapter 2. Procedure for the formation of the list of orphan diseases
3. For the inclusion of a disease in the list of orphan diseases, healthcare entities, representatives of associations and public organizations, as well as individuals and legal entities, shall submit proposals to the Working body.
4. Proposals for the inclusion of a disease in the list of orphan diseases shall be reviewed by the commission of the Working body.
The commission shall be established by an order of the head of the Working body and shall consist of a chairperson, commission members (at least three specialized experts, including non-staff specialists in the field of healthcare), and a secretary.
The chairperson shall be elected from among the commission members by voting.
5. Within a period not exceeding 30 (thirty) working days from the date of receipt of the proposals, the commission shall conduct an analysis of the proposed diseases, including the following information:
1) general description of the disease or condition;
2) code in accordance with the International Classification of Diseases (ICD);
3) description of the etiology and symptoms;
4) prevalence of the orphan disease, calculated on the basis of statistical data of the Republic of Kazakhstan and/or international statistical data.
5) justification of the severe or disabling nature of the disease, based on objective and quantitative medical or epidemiological information;
6) justification of the life-threatening nature of the disease or condition, based on mortality rates and life expectancy indicators.
6. The commission's opinion shall be formalized in minutes, which shall contain the name of the medical organization, the date of the meeting, the list of members present, the list of matters discussed, the decision adopted, and the justification thereof.
The commission's opinion shall be submitted to the authorized body for consideration at a meeting of the Joint commission on the quality of medical services (hereinafter – the JCQMS), established in accordance with Article 15 of the Code.
7. The authorized body shall decide on the inclusion of a disease in the list of orphan diseases where there is a positive recommendation from the JCQMS and the disease meets criterion 1) and at least one of criteria 2), 3), 4) or 5) below:
1) prevalence in the Republic of Kazakhstan;
2) the need for systematic treatment for which medicinal products have been developed and registered in the territory of the Republic of Kazakhstan (treatable patients);
3) the need for systematic treatment using medicinal products that have been developed and are available internationally but are not registered for use in the territory of the Republic of Kazakhstan;
4) the existence of diseases for which no medicinal products have been developed anywhere in the world;
5) the need for palliative care due to the absence of curative therapy (incurable patients).
The prevalence of the disease in the Republic of Kazakhstan shall not exceed 10 cases per 100,000 population.
8. The list of orphan diseases shall be reviewed once a year and/or upon the identification of new confirmed cases.
Chapter 3. Procedure for the formation of the list of medicines for the treatment of orphan diseases
9. For the inclusion of medicinal products in the list of orphan medicines, proposals shall be submitted to the authorized body by entities involved in the circulation of medicines, representatives of pharmaceutical manufacturers, healthcare entities, representatives of associations and public organizations, as well as individuals and legal entities.
10. Within 3 (three) working days from the date of receipt, the authorized body shall forward the proposals to a subordinate organization of the authorized body whose competence includes health technology assessment matters (hereinafter – the Centre).
11. Within a period not exceeding 30 (thirty) working days from the date of receipt of the proposals, the Centre shall conduct an analysis of the proposed medicinal products and prepare an opinion containing the following information:
1) a general description of the medicinal product;
2) information on the registration of the medicinal product in the Republic of Kazakhstan with orphan medicinal product (technology) status, or a description of the reasons for the absence of such registration in the Republic of Kazakhstan;
3) a general description of the orphan disease included in the list of orphan diseases approved in accordance with paragraph 3 of Article 177 of the Code, or a more narrowly defined indication within such orphan disease for which the medicinal product is proposed to be used;
4) details of the regulatory orphan designation status of the medicinal product in foreign jurisdictions;
5) a review of the principal clinical studies;
6) a comparison with orphan technologies registered in the Republic of Kazakhstan, including confirmation of a significant clinical or economic benefit.
12. The results of the analysis, in the form of the Centre's opinion, shall be submitted to the authorized body for consideration at a meeting of the Formulary commission (hereinafter – the FC) in accordance with the Rules for the operation of the formulary system, approved by Order No. ҚР ДСМ-28 of the Minister of Healthcare of the Republic of Kazakhstan dated 6 April 2021 (registered in the Register of state registration of regulatory legal acts under No. 22513).
13. A medicinal product shall be included in the list of orphan medicinal products where there is a positive recommendation from the FC and the medicinal product meets the following criteria:
1) the medicinal product is intended for the diagnosis, prevention, or treatment of one or more orphan diseases included in the approved list of orphan diseases (as of the date of submission of the proposal);
2) the medicinal product is registered for use in the territory of the Republic of Kazakhstan with orphan medicinal product (technology) status or is not registered for use in the territory of the Republic of Kazakhstan;
3) the medicinal product is intended for patients suffering from a disease for which no satisfactory methods of diagnosis, prevention, or treatment exist, or, where such a method does exist, the medicinal product will provide a significantly greater benefit to patients suffering from that disease.
14. The list of orphan medicinal products shall be reviewed once a year and/or upon the emergence of new orphan medicinal products.
