CIS Orphan Consortium
Rare (orphan) diseases are diseases that occur in a very small number of people. Although each individual orphan disease affects a relatively small number of people, there are more than 7,000 such diseases in total, affecting millions of patients worldwide. Most orphan diseases are genetic in nature, manifest in childhood, and are often accompanied by serious health complications, making them a major challenge for healthcare systems.
The global epidemiology of orphan diseases indicates that about 300 million people worldwide suffer from these diseases, and this figure continues to grow as diagnostic methods improve. In the CIS countries, there has also been an increase in the detection of rare diseases, but statistics remain incomplete due to the lack of unified registration data and coordinated monitoring systems. This creates an additional challenge for healthcare systems, which must take into account the needs of these patients in the long term.
Challenges faced by patients with orphan diseases include late diagnosis, a shortage of specialized clinical specialists and healthcare facilities, and limited access to effective treatments. Orphan diseases are often understudied due to their rarity, resulting in a lack of scientific data to develop new treatments. Healthcare professionals often lack information and experience in diagnosing and managing these patients, which compounds the problem and leads to significant delays in treatment.
Orphan drugs are a specific category of medicines that are developed specifically to treat rare diseases. However, their development and availability face many obstacles. Developing drugs to treat orphan diseases requires significant financial investment, and the market potential for such drugs is limited. This means that pharmaceutical companies are often reluctant to invest in research aimed at such small patient groups. As a result, many patients suffering from rare diseases often face high prices for drugs or a complete lack of treatment.
In addition, many countries often lack mechanisms for rapid access to drugs for the treatment of orphan diseases, even if they are registered in other countries. This is due to difficulties in registration, pricing and budgetary constraints. Thus, patients are left without the necessary treatment, which negatively affects their quality of life and life expectancy.
The Orphan Consortium of the CIS Member States was created to unite the efforts of all stakeholders - doctors, geneticists, researchers, pharmaceutical companies, government agencies and patients. We strive to overcome barriers that exist in the diagnosis, treatment and access to orphan drugs in the CIS countries.
