Why High Molecular rx rare?
Answers
Answer: There is a pressing need for new medicines (new molecular entities; NMEs) for rare diseases as few of the 6800 rare diseases (according to the NIH) have approved treatments. Drug discovery strategies for the 102 orphan NMEs approved by the US FDA between 1999 and 2012 were analyzed to learn from past success: 46 NMEs were first in class; 51 were followers; and five were imaging agents. First-in-class medicines were discovered with phenotypic assays (15), target-based approaches (12) and biologic strategies (18). Identification of genetic causes in areas with more basic and translational research such as cancer and in-born errors in metabolism contributed to success regardless of discovery strategy. In conclusion, greater knowledge increases the chance of success and empirical solutions can be effective when knowledge is incomplete.
A disease is considered rare in the USA if it is believed to affect fewer than 200,000 individuals. There are approximately 6800 such diseases, according to the NIH. While each disease is rare, when considered together they affect nearly 30 million people or almost one in ten. In the EU, a disease is considered rare if it has a prevalence of fewer than five in 10,000 people. Some rare diseases have less than a dozen known cases, whereas others are more common, such as multiple sclerosis, cystic fibrosis and Duchenne muscular dystrophy. Collectively, these disorders affect 6–7% of the population in the developed world
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