Pharmaceutical companies – driven primarily by increased pressure from payers and regulators – now focus their attention more or less exclusively on areas with a high and clearly unmet medical need. 技术和
scientific advances within the field of genomics and precision medicine also means that the patient populations targeted by new therapeutics become smaller and more defined. 在一起, these trends have paved the way for a booming interest in the orphan- and rare diseases space – a development that is seen also in 日博备用网站.
This paper describes the orphan disease pipeline of Swedish companies and presents statistics over orphan drug designations granted to Swedish companies by the US Food and Drug Administration (FDA) and the 欧洲an Medicines Agency (EMA). 除了, five different perspectives on orphan drug development will be presented, 包括大型和小型专业公司, 一流的医科大学, a venture capital investor and a company that provides regulatory services and expertise.
报告强调
- Swedish companies have been granted 60 orphan designations in the EU and 47 in the US from January 2000 until December 2018.
- Oncology is the leading orphan drug therapy area accounting for about 38 percent of the total Swedish pipeline.
- 神经学, immunology/rheumatology and transplantation are the next leading therapy areas, 又占了26%.
- Sobi is the largest Swedish company active in this area with several approved orphan drugs.
- 日博体育备用孤儿药开发的观点由HealthCap提供, Immedica制药, 卡罗林斯卡医学院, NDA集团, 和艺人.
- 概述了日博备用网站孤儿病管道项目清单.
