May 15 is Tuberous Sclerosis Complex Global Awareness Day. It is an orphan disease. There are a total of one million people with such diagnosis in the world. Early or even prenatal diagnosis may significantly improve the quality of life of a TSC patient. However, this is absolutely not affordable for such families, so state support is vital, said Iryna Hladun, head of the NGO “Ukrainian Association for support of patients with tuberous sclerosis” at a press briefing at Ukraine Crisis Media Center. “TSC requires constant medication, and parents cannot sustain it. State support for patients’ treatment, rehabilitation and socialization is needed. If we start it in time together with the state, we’ll give patients a chance to become fully functional citizens. Absence of medical support means fast disability and death,” she explained.
These are oncology and antiepilepsy drugs. “The antiepilepsy drug Vigabatrin is effective in 70 % or more cases. When seizures go away, children progress in their development. The sooner it is used, the smaller future mental impairment will be. When treatment continues for years, it’s difficult to do without state support,” said Olena Savchenko, head of Pediatric Neurology Department at National Special Pediatric Clinic “OKHMATDYT”. Nevertheless, this drug is not registered in Ukraine. Oncology drug Everolimus has side effects, but its efficacy outweighs these side effects. For instance, no one can make drug concentration in Kyiv today due to lack of funding. “Making drug concentration in Kyiv today is impossible. Shalimov National Institute of Surgery and Transplantology in Kyiv could do it with the medicine producers’ financial support. Today it is impossible, though necessary,” said Stella Kushnirenko, PhD in Medicine, lecturer of the Department of Nephrology at Shupyk National Medical Academy of Postgraduate Education.
The Law On life treatment of patients with rare diseases is in effect in Ukraine, which entitles such patients and their families to demand satisfaction of their requirements. “Orphan diseases should be a priority for the state, and orphan patients’ need should be taken into account in every reform,” believes Tetiana Kulesha, head of the Council of Public Union “Orphan diseases in Ukraine”. There are other draft bills that should enhance legal framework. “There are three draft bills at present which should reconsider philosophy of attitude towards people with such needs,” added the Member of Ukrainian Parliament Oksana Yurynets.
In addition to financial support of medical treatment, TSC patient need socialization and community involvement. “People with special needs should be integrated into society. You cannot hide them. You should keep them company. Society needs to see them. You’ll see that these people can join the others in singing or doing crafts,” said Eberhardine Seelig, President of David Deacon Charity Institution – Childhood Cancer Foundation Ukraine.