Description of the Center and the Rationale of Establishment
Rare Diseases and Orphan Drugs Application and Research Center-ACURARE established in Acıbadem Mehmet Ali Aydınlar University in 2017. Aims of the Center are, to improve the diagnostic and treatment opportunities in the field of rare diseases and orphan medicines used in the treatment of these diseases, to increase scientific and clinical research, and to increase the quality of life of patients and their participation. It is also aimed to develop existing resources and use them more efficiently.
European Commission on Public Health defines rare diseases as "life-threatening or chronically debilitating diseases which are of such low prevalence (fewer than 1 in 2,000 people) that special combined efforts are needed to address them". The definitions used in the medical literature and by national health plans are similarly divided, with definitions ranging from 1/1,000 to 1/200,000. It is estimated that as many as 6,000 to 8,000 distinct rare diseases exist, and as much as 6% to 8% of the population is affected by one. Approximately 80 % of these diseases are of genetic origin. On the basis of this general ratio, it is estimated that at least 4-6 million people in Turkey are affected by a rare disease.
Rare diseases lead to significant social and national health problem in Turkey and in almost every country. Clinical diagnosis of these diseases is often difficult and many may not have any diagnostic laboratory tests. Therefore, rare diseases require specialized physicians, clinical centers and diagnostic laboratories in health care. Orphan drugs" are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare. These drugs are called “orphan” because under normal market conditions the pharmaceutical industry has little interest in developing and marketing products intended for only a small number of patients.Thus, these potent drugs are generally expensive and bringing a medicinal product to market has extremely high cost for drug companies. In order to solve these problems experienced by patients and their relatives, it is necessary to establish a reliable data collection and evaluation mechanism on rare diseases and to establish a national health service policy on rare diseases and orphan drugs in the general health system.
"ACURARE-Rare Diseases and Orphan Drugs Research and Application Center" aims to facilitate following issues in this important subjec;
- to improve diagnostic and treatment opportunities
- to increase scientific and clinical research capacities
- to improve the quality of life of patients and to ensure patient participation
- to enhance improvement and efficient use of existing resources
ACURARE aims to include all related stakeholders under te same roof. These stakeholders include asscoated clinicians, researchers, clinics, laboratories, hospitals and other healthcare organizations specializing in the diagnosis and treatment of rare diseases, patient organizations, professional organizations and associations, pharmaceutical companies working on rare diseases and orphan drugs. ACURARE will contribute to reveale following issues, which are essencial for our country on the issue,
- providing academic knowledge,
- conducting research on the subject,
- data collection for use of all stake holders
- coordination of interdisciplinary assistance setting for patients and their relatives
- coordination of multidisciplinary setting for researchers