Drugs for people with orphan diseases not included in the national list

Life-saving drugs for people with rare, orphan diseases (among them, in cystic fibrosis), for some reason not included in the national list.The paradox of these drugs is to purchase for the funds of local budgets. But since they are not in the National list, buy life-saving drugs as a residual.

Today in Ukraine catastrophically heavy mortality statistics from orphan (rare) diseases.

Also read: KNOW MORE than FIVE THOUSAND HEREDITARY DISEASES! WHY SHOULD GENETIC TESTING IN NEWBORNS AND HOW IS THE SCREENING

One of clonicotonic diseases, cystic fibrosis hereditary disease characterized by malfunction of the exocrine glands and severe disorders of the respiratory system. The disease is inherited in an autosomal recessive manner. The incidence is 1 in every 2-2,5 thousand infants. Cause of cystic fibrosis is a genetic mutation, due to which there are abnormalities in the structure and functions of the MVTR (transmembrane conductance regulator cystic fibrosis). This protein is involved in water and electrolyte metabolism of the epithelium of the bronchopulmonary system, pancreas, liver, reproductive organs and gastrointestinal tract.

In Ukraine a lot of patients suffering from rare diseases. These diseases require not just expensive treatment, but first the timely and correct diagnosis, without which, alas, the patient is doomed. In science such diseases are called orphan, from the English word orphan is an orphan. And almost as orphans, the speakers of these terrible diseases in Ukraine are often left alone with their troubles.And without drugs a painful and lingering death.

At regional levels these patients can’t get treatment because it prevents it is the decision of the Cabinet No. 180, which States that any medication outside macspice can be procured after satisfying needs.

Cancer patients, diabetics, patients with renal insufficiency, a rare disease. All of these people daily have to fight with their illness and with the bureaucratic tumor. They come in the Cabinet, protest, assemble briefings to be heard. But pokerspelen.

Most rare diseases are hereditary, and many patients die in the first years of life without the right diagnosis.In European countries the average life expectancy of 60 years. in Ukraine 18.

Most from the shortage of medicines is affected orphan patients. Patients with hemophilia, cystic fibrosis… In Ukraine people with rare болезнями5%.

They suffer, go very hard, leave in full consciousness. And it’s just catastrophically difficult.

Orphan patients in Ukraine rarely become adults. Do not survive, because the state partially provides medication only to children under 16 years of age. Adults have to survive on their own.

Author: Love Doll