The Ministry of Health Has Proposed to Expand the Orphan Diseases List

On June 29, 2017, the Ministry of Health of Ukraine published the draft order of the Ministry of Health of Ukraine "On introducing changes to the list of rare (orphan) diseases leading to a reduction in the life expectancy of patients or their disability and for which there are recognized methods of treatment." on the website for public discussion.

The draft proposed to complete the current list of orphan diseases with new nosologies, and to expand the list of orphan diseases indices according to the Orphanet portal system for individual nosologies.

In particular, it was proposed to add to "Rare endocrine diseases, nutritional disorders and metabolic disorders" section the following items:

• impaired ornithine metabolism. Argininemia. Ornithine transcarbamylase deficiency;
• glycine imbalance;
• other disorders of lipid accumulation. Wolman's disease;
• hereditary erythropoietic porphyria;
• porphyria cutaneous tarda;
• other porphyria;
• lipomatosis, not elsewhere classified.

In addition, section IV "Rare diseases of the nervous system" is recommended to be completed with the following nosologies:

Encephalitis, myelitis and encephalomyelitis. Encephalitis, myelitis and encephalomyelitis in diseases classified elsewhere. Progressive encephalomyelitis with rigidity and myoclonus;

• secondary parkinsonism, caused by other external factors;
• other forms of secondary parkinsonism;
• blepharospasm;
• dystonia, unspecified;
• hereditary motor and sensory neuropathy.

Also, the Ministry of Health proposes to introduce a new section of orphan diseases, namely: Section XI "Rare diseases of sclera, cornea, iris and ciliary body", including Interstitial (stromal) and deep keratitis (Kogan syndrome).

Let us remind you that since January 1, 2015, after the introduction of amendments to the Fundamentals of Ukrainian legislation on healthcare in order to ensure the prevention and treatment of rare (orphan) diseases, the orphan patients have been given the right to continuous and free treatment of these diseases with the drugs according to their list and volumes, Approved by the Ministry of Health. The procedure for providing the citizens suffering from rare (orphan) diseases, witht the drugs and related products for special dietary consumption is approved by Cabinet of Ministers Resolution No. 160 dated March 31, 2015.