MORE THAN 16.8 THOUSAND PATIENTS WITH ORPHAN DISEASES WERE PROVIDED WITH DRUGS IN THE FIRST HALF OF 2024

Orphan diseases are a global problem faced by many countries around the world. The main difficulty is that due to their rarity, diagnosis, treatment and prevention of such diseases are often difficult, and in some cases remain inaccessible.

According to GlobalGenes, more than 400 million people worldwide suffer from rare diseases, 80% of which are genetic in origin.

The most pressing problem in the treatment of these diseases is drug provision, since expensive drugs may be required to treat severe forms of rare diseases. And the production of orphan drugs may be unprofitable due to the low prevalence of these diseases.

According to the Code of the Republic of Kazakhstan "On Public Health and the Healthcare System", orphan diseases include rare severe diseases that threaten human life or lead to disability, the incidence of which does not exceed the officially defined level.

In Kazakhstan, the government is taking active steps to improve this situation.

Government measures are aimed at providing patients with the necessary drugs, despite the difficulties associated with their rarity and high cost of treatment.

The list of orphan diseases and drugs for their treatment, approved by the order of the Ministry of Health of the Republic of Kazakhstan (MH RK) dated October 20, 2020 No. ҚР ДСМ - 142/2020, has become an important step towards solving the problem of rare diseases. It is important to note that SK-Pharmacy LLP, as a Unified Distributor, carries out centralized procurement of drugs based on the List approved by the order of the MH RK dated August 24, 2021 No. ҚР ДСМ - 88, as well as on applications from medical institutions.

For example, for the first half of 2024, the Unified Distributor purchased 169 types of drugs for the treatment of orphan diseases.

For the first half of 2024, more than 16.8 thousand patients with orphan diseases were provided with drugs. The average cost of treatment per patient is 1.69 million tenge. The most costly orphan diseases include:

- mucopolysaccharidosis;

- spinal muscular atrophy;

- other sphingolipidoses;

- acute lymphoblastic leukemia, chronic myeloid leukemia;

- Duchenne muscular dystrophy.

You can see the list of orphan diseases and drugs for their treatment at the link: https://adilet.zan.kz/rus/docs/V2000021479