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.

罕见病疗法的开发存在两个主要问题。首先，在 7000 多种此类疾病中，绝大多数是由遗传缺陷和/或包括神经退行性疾病引起的，因此很难治疗。其次，罕见病药物，即所谓的孤儿药，价格极其昂贵，只有少数患者有兴趣购买。这导致罕见疾病的特定经济陷阱的出现；即，尽管具有很高的生物医学、制药和技术潜力，但新的孤儿药的开发受到经济现实的阻碍。这项工作的目的是寻找一种可能解决罕见疾病经济陷阱的潜在解决方案。进行了文献综述，并提出了一个假设，假设使用一种药物治疗许多罕见疾病可能会克服经济陷阱。我们提供了一些例子，表明找到这样的药物是可能的。因此，提出了开发孤儿药问题的可能解决方案。进一步的临床前和临床研究，虽然既不简单也不便宜，但应该验证有关解锁罕见疾病经济陷阱可能性的假设是否有效。