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By Donna Kelly, Chief Consulting Officer and Alice Liftin, Senior Vice President
As opposed to the 20th century, when blockbuster agents were tweaked to produce many similar compounds (e.g., 13 angiotensin receptor blockers), product development in the 21st century will focus on more targeted and specialized agents with the goal of providing more effective treatment for smaller subsets of the population versus the scattershot approach of some of the previous century’s medications. For example:
- In 2009, the FDA approved 18 new molecular entities (NMEs) and 20 vaccines; in 2010, 21 NMEs and nine biologics license applications (BLAs) were approved, demonstrating the pharmaceutical industry R&D shift towards biologics, vaccines and monoclonal antibodies
- The European Medicines Agency has emphasized development of treatments for rare diseases. From 5,000 to 8,000 distinct rare diseases exist today, affecting 6 percent to 8 percent of the total population – in other words, from 27 million to 36 million people in the EU alone.
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