One drug to treat many diseases: unlocking the economic trap of rare diseases

• Published in: Metabolic brain disease Vol. 35; no. 8; pp. 1237 - 1240
• Main Authors: Pierzynowska, Karolina, Kamińska, Teresa, Węgrzyn, Grzegorz
• Format: Journal Article
• Language: English
• Published: New York Springer US 01.12.2020; Springer Nature B.V
• Subjects: Biochemistry; Biomedical and Life Sciences; Biomedicine; Drug development; Drugs; Economics; Hypotheses; Literature reviews; Medical treatment; Metabolic Diseases; Neurodegeneration; Neurology; Neurosciences; Oncology; Rare diseases; Review; Review Article; Rare diseases; Orphan drugs; Neurodegenerative diseases; Hypothesis of one drug for many diseases; Economic trap
• ISSN: 0885-7490; 1573-7365
• DOI: 10.1007/s11011-020-00617-z

There are two major problems with the development of therapies for rare diseases. First, among over 7000 such diseases, the vast majority are caused by genetic defects and/or include neurodegeneration, making them very difficult to treat. Second, drugs for rare diseases, so-called orphan drugs, are extremely expensive, as only a small number of patients are interested in purchasing them. This results in the appearance of a specific economic trap of rare diseases; namely, despite high biomedical, pharmaceutical and technological potential, the development of new orphan drugs is blocked by the economic reality. The purpose of this work was to find a potential solution that might resolve this economic trap of rare diseases. A literature review was conducted, and a hypothesis was formulated assuming that the use of one drug for the treatment of many rare diseases might overcome the economic trap. We provide examples showing that finding such drugs is possible. Thus, a possible solution for the problem of developing orphan drugs is presented. Further preclinical and clinical studies, although neither easy nor inexpensive, should verify whether the hypothesis regarding the possibility of unlocking the economic trap of rare diseases is valid.