Orphan Drugs and The Role of Orphan Drugs In Novel Drug Development

Abstract

It has recently been observed that orphan drugs have a very important place in the researches carried out within the scope of new drug development studies. With the development of orphan drugs, it will be possible to obtain useful new drugs to be used in the treatment of rare diseases, and these drugs with proven effectiveness are expected to lead to the treatment of many diseases that are not yet curable. In this review study, it is aimed to research and evaluate the current status of orphan drugs. As a result of the literature review carried out within the scope of the study, the laws, and programs that encouraged research and development studies and the latest developments about orphan drugs are presented in detail. The numerical data presented on orphan drugs show the importance of these drugs in the development of new drugs.

Keywords

• Orphan Drug
• Orphan Drug Act
• Drug Development

Öksüz İlaçlar ve Öksüz İlaçların Yeni İlaç Geliştirilmesindeki Yeri

Abstract

Son yıllarda yeni ilaç geliştirme çalışmaları kapsamında yapılan araştırmalarda, öksüz ilaçların çok önemli bir yerinin olduğu görülmektedir. Öksüz ilaçların geliştirilmesiyle, nadir hastalıkların tedavisinde kullanılacak yararlı yeni ilaçların elde edilmesi mümkün olabileceği gibi, etkinliği kanıtlanmış bu ilaçların, henüz tedavisi mümkün olmayan pek çok hastalığın tedavisi için de öncü olması beklenmektedir. Bu çalışmada, öksüz ilaçların, mevcut durumuna ilişkin araştırma ve değerlendirme yapılması amaçlanmıştır. Çalışma kapsamında yapılan literatür taramaları sonucunda, öksüz ilaçlar hakkında yaşanan son gelişmeler ve bu ilaçlarla ilgili araştırma-geliştirme çalışmalarının artmasını sağlayan yasa ve programlar detaylı olarak sunulmuştur. Öksüz ilaçlarla ilgili sunulan sayısal veriler, bu ilaçların yeni ilaç geliştirilmesindeki yerinin önemini göstermektedir.

Keywords

• Öksüz İlaç
• Öksüz İlaç Yasası
• İlaç Geliştirme

Supporting Institution

Bu çalışma herhangi bir proje desteği olmadan Çukurova Üniversitesi Eczacılık Fakültesi bünyesinde gerçekleştirilmiştir.

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