Learn More About Medicines in Development for Rare Diseases
Medicines in Development for Rare Diseases
Rare diseases, taken together, aren’t "rare" at all. In fact, according to the National Institutes of Health, 25-30 million Americans have one of the nearly 7,000 diseases that are officially deemed “rare” because alone they each affect fewer than 200,000 people. Sometimes, only a few hundred Americans are known to have a particular rare disease.
Moreover, the Orphan Drug Act of 1983 provided incentives, for example a longer period of marketing exclusivity, for drugs that are not expected to recoup their development costs or that are targeted at diseases affecting fewer than 200,000 people. Under the Orphan Drug Act, 2,313 medicines have been designated orphan drugs by the U.S. Food and Drug Administration (FDA) as of January 24, 2011. These medicines are in all stages of development and some will eventually gain approval. Over the last few years, new medicines have been approved for Pompe disease, which causes among other symptoms an engorged heart. Other recently approved treatments are for rare cancers and myelodysplastic syndromes, which are pre-leukemia diseases affecting blood marrow.
Other examples include a medicine in development for epidermolysis bullosa, a group of inherited disorders where skin blisters develop in response to minor trauma, and one for Friedreich’s ataxia, a genetic disease that causes degenerative nerve damage in children.
With continued innovation, we hope that one day, whenever the question is asked, “How can we help them?” there always will be an encouraging answer.