Neurological diseases are regarded as one of the most intractable disease category. Many neurological diseases are fatal without any effective drug therapeutics. As there are few effective surgical treatments applicable, the development of new drugs is needed. In this study, we analyzed the present status of drug development and marketing of drugs categorized in this field in Japan to show the strategies for developing the drugs efficiently.
At first, we analyzed the present status of the drug development for intractable neurological diseases specified by Japan intractable diseases information center. Developing and marketing status were investigated using commercial database (Bio Today, and estimated based on the sales data from IMS JAPAN, respectively). We referred to website of National Institute of Biomedical Innovation (NIBIO) to research compounds designated as orphan drug (OD).
Twenty-one neurological diseases were selected among 130 rare diseases (the third most disease category of 15 areas). There were 12 diseases which have therapeutic drugs as OD among the 21 diseases. Twenty compounds were indicated as OD and 12 compounds were approved. Among the 12 compounds, 4 compounds had OD indication after other indication, and 8 compounds had indicated just OD. There were 4 compounds ranked in top 500 sales in Japan. We also analyzed the price change and market possession of these drugs.
In neurological diseases, number of patients in each disease categorized in this field was smaller compared with cardiovascular, respiratory and gastrointestinal diseases. The first in class drugs were used for major population of patients in target disease in long period. Even it was not so much sales, it made steady sales in every year. Patients in lot of neurological diseases which have no remedy need a new drug development. Drug development without estimating future sales made a decrease in success probability for supply-side players in drug development (e.g. pharmaceutical industries, academia, bio-venture companies etc.).
From the result, we can declare that pharmaceutical-economic cross strategic development and marketing is indispensable for effective developing neurological drugs.