Development of Verification Method for The Effectiveness and Safety of Off-Label Drug Use

Abstract

ABSTRACT

Development of Verification Method for the Effectiveness and Safety of Off-label Drug Use

Young Mee Lee Laboratory of Computational Biology and Bioinformatics Graduated School of Public Health Seoul National University

Development of a new medication is not an easy task being not only financially costly but also very time consuming without any guarantee of success. Information about medications that has been developed and used until now is well accumulated due to technological advances, especially IT technology. This information has allowed more thorough inspection of safety of existing medications. Also, in case of any safety problem, certain medication could be withdrawn from the market. A newly magnified side effect can give a different perspective so that a change in indication leads to the use of new medication. Mechanism of action and target are not clearly known for all medications, but research on unknown medications is ongoing and those new information are being recorded as big data. In addition, gene information of organisms is continuously being added to the data base and offered to the public research for the health and prolonged life of humanity. Usage of thalidomide had once been stopped due to a risky side effect, however, the mechanism of action that caused certain side effect has been verified and it is now being used to treat multiple myeloma with a new indication. This was possible because there has been sufficient research on drug target and disease associated genes. This research will use bioinformatics to examine the effectiveness of pre-existing medications that are used for off-label indications. Under the current circumstance where reference is rare and clinical trials are not possible, this research will study the validity of off-label uses of many drugs to determine whether there are any safety issues based on the accumulated information. On the other hand, this research will give theoretical propriety to well-known off-label uses that pharmaceutical companies avoid due to lack of economic profit. Also, this research will suggest that the process of finding a new indication of a new substance can be developed from pre-existing medication using bioinformatics.

Keywords : Off-label use, Drug repositioning, New indication development, Gene information, Drug information