Developing Drugs for Rare Diseases in the US— Emerging Trend

Posted 01 January 2011 | By Rumana Haque-Ahmed 

If you Google the term "rare diseases," you will discover a wide variety and significant number of organizations that are engaged in the innovation and advancement of drugs to treat these diseases (such as the US Food and Drug Administration (FDA) and its Office of Orphan Product Development (OOPD) and Center for Drug Evaluation and Research (CDER) Office of Rare Diseases; the National Institutes of Health (NIH), the National Organization for Rare Disorders (NORD), the Genetics Alliance, Pharmaceutical Research and Manufacturers of America (PhRMA) and numerous patient advocacy groups). You will also find announcements by large pharmaceutical companies about their investment in drugs to treat rare diseases. There is an increased awareness by all these groups that treatments for rare diseases are possible and sustainable and have the potential to address a significant public health need. This article reviews recent important milestones in the US and discusses some of the challenges that are unique to developing drugs for rare diseases.

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