Availability of Orphan drugs: Basic legal issues of development and financing

This paper critically analyzes the basic models for the development and financing of orphan drugs, i.e. medicines for the treatment of rare diseases, starting from the principle of equality in health care proclaimed within the framework of the World Health Organization and to the protection of the rights of vulnerable categories of the population as well. A comparative and normative method has been applied. On the basis of the regulations of the European Union and the national laws of the member states, the paper emphasizes the possible solutions for the development of domestic law in the context of ensuring access to patients and exercising the right to health care. The opinion of the European Court of Human Rights in the case of Nitecki v. Poland is, specifically, analyzed from the aspect of refunding the price of an orphan medicinal product and exercising the right to access to health care services for patients. The model of public-private partnership was identified as the most adequate one in the conditions of limited public resources, taking into account the interest of the state, and the need for expressing social responsibility considering the interest of the (pharmaceutical) companies. The model is based on the (bio)ethic principle of justice, conceptualized as assistance to those people who suffer from bad health condition and who are economically worse off.