One drug to treat many diseases: unlocking the economic trap of rare diseases
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REVIEW ARTICLE
One drug to treat many diseases: unlocking the economic trap of rare diseases Karolina Pierzynowska 1
&
Teresa Kamińska 2
&
Grzegorz Węgrzyn 1
Received: 22 July 2020 / Accepted: 8 September 2020 / Published online: 14 September 2020 # The Author(s) 2020
Abstract 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. Keywords Rare diseases . Neurodegenerative diseases . Orphan drugs . Economic trap . Hypothesis of one drug for many diseases
Introduction There are approximately 7000 rare diseases, and the vast majority of them are particularly difficult to treat (Nguengang Wakap et al. 2020). Among rare diseases, genetic and neurodegenerative disorders are predominant. There are two problems with finding effective drugs for such diseases. First, genetic defects occur in all cells (or their progenitors) of patients; this problem is enhanced by the fact that the majority of these diseases are severe, with devastating symptoms that might be irreversible if the treatment is not started early (Zschocke 2008). In fact, their molecular mechanisms are usually complicated, and even in the case of monogenic disorders, pathomechanisms involve disturbances of many processes occurring at the cellular and organismal levels
* Karolina Pierzynowska [email protected] 1
Department of Molecular Biology, Faculty of Biology, University of Gdańsk, Wita Stwosza 59, 80-308 Gdańsk, Poland
2
Department of Microeconomy, Faculty of Economy, University of Gdańsk, Armii Krajowej 119/121, 81-824 Sopot, Poland
(Gaffke et al. 2020). However, currently available knowledge and technology allow for discoveries of various treatments that can be potentially effective in rare diseases (Ahmed et al. 2019). On the other hand, the second problem appears to be the major inhibitor of this process. Namely, since the number of patients suffering
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