A rare case.The Ukrainian Relief Fund opens a program to help children with orphan (rare) diseases. We started with a diagnosis of "bullous epidermolysis". Such children have extremely vulnerable skin, they need special care, special bandages, so treatment is very expensive. Ufond correspondent Svetlana Khisamova met with Tatiana Zamorskaya, head of the Debra-Ukraine Center and vice president of the Dermatologists-Children International NGO, to find out how children with bullous epidermolysis live in Ukraine and what help orphans receive from the state.
In 2011, the Dermatologists-Children IOC established the Debra-Ukraine Center to help children suffering from bullous epidermolysis (BE). Debra-Ukraine has the status of a non-profit public organization, which is carried out by dermatologists, relatives of patients with BE and volunteers.
- What problems do orphan patients face in Ukraine? - The main problem is that they do not receive the necessary medical care in the required amount. The state does not have enough funds to purchase the necessary medicines, special medical equipment, and clinical nutrition.
In all countries, the treatment of orphan patients is expensive. That is why the state plays a key role here. The main costs in the EU and the US are borne by the state budget and insurance companies. Each state has its own system of providing orphan patients, and there is no single model that could be copied and implemented in Ukraine. But it is obvious that the problem needs to be solved systematically and at the central level. For example, to conduct state procurement of drugs through international organizations. In addition, as part of a policy of decentralization and strengthening the role of local budgets (including health contributions), orphan patients can and should receive assistance from regional authorities.
- What are the statistics of orphan diseases?- In total, according to statistics, there are about 8,000 species of rare (orphan) diseases in the world, of which 80% are genetic. Most of them are diagnosed in childhood, and more than 30% of children with rare diseases do not live to be five years old. There are about 30 million orphan patients in the European Union. This is about 8% of the population. There are no exact statistics in Ukraine, as the electronic state register of patients has not been implemented yet. According to our estimates, about 5% of the country’s population have orphan diseases.
Last year, the Center for Orphan Diseases was established on the basis of the National Children’s Specialized Hospital (NDSB) OKHMATDET. We expect that in 2016 the management of the Center will take concrete steps to create a register of patients with rare diseases. We understand how important it is to lay the foundation for a reliable and efficient system of registration of orphan patients and their lifelong provision with the necessary drugs, as required by current legislation. That is why we are ready to help and participate in this work on a voluntary basis.
- Not so long ago, a law was passed that regulates the provision of medical care to patients with orphan diseases. Did he somehow change the situation?- Law of Ukraine of April 15, 2014 № 1213-VII "On Amendments to the Fundamentals of Legislation of Ukraine on Health Care for the Prevention and Treatment of Rare (Orphan) Diseases" entered into force on January 1, 2015. However, in practice it still does not work: orphan patients do not receive adequate care from the state, although the document states that they must be provided with free, lifelong and uninterrupted medicines and special dietary food. Throughout 2015, we negotiated with colleagues from the Dermatologists-Children NGO and the public organization Orphan Diseases of Ukraine with the Ministry of Health (MOH), the Ministry of Finance and regional administrations and regional health authorities. As a result, the first registers of orphan patients appeared in the working group of the Ministry of Health, the calculation of the needs of patients,
Based on the data of the Ministry of Health and under pressure from public patient organizations and orphan patients, the Ministry of Finance has included in the Law on the State Budget for 2016 about UAH 500 million. for the needs of orphan patients. The Ministry of Health should use these funds to purchase medicines through international organizations. However, it is already mid-February, and work on procurement has not begun.
- This year Ufond opened a department to help children with orphan diseases. The first diagnosis is bullous epidermolysis. Children with this disease are also called "butterflies". How many such patients are there in Ukraine? What help do they get?- In Ukraine, there are about 200 patients with bullous epidermolysis, most of them - children under 18 years. As you rightly pointed out, such patients are called "butterflies" all over the world for their thin skin, which is injured by even the slightest touch. The disease affects not only the skin but also the internal organs: esophagus, stomach, intestines. Quite often at "butterflies" fingers grow together, there is a deformation of extremities. As a result, patients are able to move only in a wheelchair. Such patients need the help of a multidisciplinary team of doctors, ie not only a dermatologist, but also a pediatrician, surgeon, gastroenterologist, dentist and other specialists.
Diagnosis of PE patients has never been diagnosed in Ukraine, as there were no conditions for this. Parents with "butterfly" children turned to European centers for bullous epidermolysis (mainly in Austria and Germany) to determine the exact diagnosis and treatment of such pathology.
With the support of the leadership of NDSB OKHMATDET and sponsorship of the charity fund "Lions Club of Kiev" in September 2013, we opened the first in Ukraine specialized center for the treatment of patients with bullous epidermolysis - BE’s office at the hospital OKHMATDET. About a hundred patients have already received the necessary medical and counseling care.
Like other orphan patients, "butterflies" need constant medical care, but do not receive it due to lack of funding. Treatment of such patients is very expensive. The average monthly cost of critically needed medication for one child- "butterfly" is about 40-50 thousand. UAH., in particularly severe patients over the age of 18 costs can be about 100-120 thousand. UAH As all special skin care products are imported and have no analogues in Ukraine, prices for products for "butterflies" are rising literally every day due to the devaluation of the hryvnia.
Therefore, in addition to fundraising (collecting donations for medicines and bandages for "butterflies"), an important goal of our work is a systematic solution to the issue of providing patients with BE with the necessary drugs and care. We managed to include UAH 26 million. in the state budget for 2016 for the needs of patients with BE. We hope that the necessary plasters and silicone nets for open wounds, wound-healing ointments and creams will be purchased and given to our patients living in all corners of Ukraine. Although it is already clear that this funding will not be enough to cover all needs (since the calculations were made in early 2015, and since then the hryvnia has devalued and 20% VAT has been introduced on some drugs), we will turn to local budgets to lobby for additional support. patients.
06/10/2016