EMA had a workshop on the theme: “Support for Development of Orphan Medicines” on November 30, 2020. The workshop underlined that patients with rare diseases, shall receive the same treatment quality as other patients. That is why it is necessary to stimulate pharmaceutical companies to research and produce proper drugs on the market. There is an innovative network of EU that strengthens the co-operation between innovative offices and EMA, supports the control to drugs producers on the national level and on the level of EU, aiming at attracting of producers innovators. Incentives are also granted to SME, the producers of rare drug diseases for the development of innovative drugs.  

   IRDiRC also supports development of the methods of rare disease treatment. The goal of IRDiRC in 2011 was appearance of 200 new treatment types till 2020, but this goal was achieved in 2017. The second aim of IRDiRC is approving of 1000 new treatment types especially of those diseases that have not any approved treatment.

   A lot of patients with rare diseases do not have or have unsatisfactory methods of treatment, but they shall receive profit from scientific progress and development of innovative drugs as soon as possible.

   The future of patients with pulmonary hypertension depends on increasing of the disease awareness, better diagnosis and development of new methods of treatment. Thus, to achieve these goals, it is necessary for scientists and production market to co-operate.

The photo was taken from EMA website.