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

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.

The national economic burden of rare disease in the United States in 2019

“…Historically, due to the small prevalence of individual RDs, and the heterogeneity in disease etiology, clinical presentation, healthcare needs, and cost implications across difference RDs, rare diseases were viewed as diseases that may be individually debilitating but with limited public health implications. Advances in science and research have now shown several commonalities across RDs: first, many RDs share the same genetic or environmental risk factors [ 2 ]; second, there is evidence that therapeutic approaches that work for multiple RDs are both feasible and cost-effective [ 10 ]; third, patients with different RDs often share similar clinical experiences and care journeys. For instance, the phenomenon often regarded as the extensive “diagnostic odyssey” is known to affect many RD communities and results in significant delay in appropriate treatment, emotional stress, and economic loss [ 11 ].…”

Medically important bacteria isolated from commercial herbal medicines in Kampala city indicate the need to enhance safety frameworks

“…On average therefore, each remedy was suggested to treat over 15 ailments. Though the “one drug for many diseases” approach may not only reduce the cost of treatment of multiple infections 41 , it can also increase the number of individuals (suffering from different ailments) who may consume the same drug, and hence get exposed to its associated risks such as pathogenic microbial contaminants.…”

Establishing a second-generation artificial intelligence-based system for improving diagnosis, treatment, and monitoring of patients with rare diseases

“…Third, a significant proportion of these patients do not respond to available therapies because of partial or complete loss of response (LOR). This results in the emergence of an economic trap of rare diseases; specifically, despite the high biomedical, pharmaceutical, and technological potential, the development of new drugs is blocked by the economic reality and lack of effectiveness of the drugs [10].…”