On the occasion of International Rare Disease Day, a panel discussion "Beyond the possible" was held in Kyiv. The event brought together representatives of state authorities, patient organizations, the expert community and international partners. The focus was on practical mechanisms for ensuring that patients with orphan diseases have access to the necessary treatment.
The State Expert Center of the Ministry of Health of Ukraine also joined the event. The discussions were attended by: Yevheniia Ishkova, Deputy Director for Health Technology Assessment and Clinical Trials; Oresta Piniazhko, Director of the HTA Department; Oksana Hulenko, Head of the Department for Standardization of Medical and Rehabilitation Care.
Within the discussion, attention was focused on practical tools for ensuring access to innovative therapy. These include conducting clinical trials, implementing state health technology assessment and systematically updating standards of medical care.
Clinical trials are an integral part of the system of access to treatment for rare diseases. This is not only an opportunity to receive an innovative medicinal product, but also early diagnosis, genetic testing and multidisciplinary support in accordance with international standards.
Clinical trials have already been conducted in Ukraine in the following areas: genetic, metabolic and neurological diseases, pulmonology, cardiology, etc. Today, the international multicentre phase II clinical trial SHIELD-DMD is ongoing for patients with Duchenne muscular dystrophy. More detailed information can be found in the section List of clinical trials in Ukraine https://clinicaltrials.dec.gov.ua/
Another important mechanism is health technology assessment (HTA). It enables the state to make informed decisions on the financing of innovative medicinal products based on evidence-based approaches, taking into account clinical effectiveness and economic feasibility.
As of 25 February 2026, the Center has issued 54 conclusions from state HTA for the treatment of rare diseases – this is 35% of all HTA conclusions, which emphasises the priority of this area.
In 2022–2025, on the basis of the Center's recommendations, managed access agreements were concluded for 14 innovative medicinal products. Thanks to this, patients with severe genetic and metabolic diseases gained access to modern therapy.
An important separate role is played by the updating of standards of medical care. The Center provides methodological support to about 30 multidisciplinary working groups in the field of orphan diseases. As of the end of 2025, 62 standards and clinical protocols have already been updated in the following fields: neurology, immunology, endocrinology, pulmonology, hematology, rheumatology. Work on new documents and the revision of existing ones continues in line with modern evidence-based medicine approaches.
Participants in the event noted that it is precisely the combination of patient experience, an expert approach and responsible state policy that makes it possible to build a sustainable system capable of providing people with rare diseases with the necessary treatment.
