With the U.S. being the world leader in the area of life-sciences innovation, having created 57 percent of new chemical entities from 2001-2010, it is difficult to believe that 95 percent of the rare diseases in the U.S. do not have a single FDA-approved treatment; yet this is a reality that Stephen Ezell, vice president of global innovation policy with the Information Technology and Innovation Foundation (ITIF), is seeking to bring to light.
"There are a number of unique challenges when it comes to rare disease and innovation," Ezell told Patient Daily. "Rare diseases are diseases that affect populations of fewer than 200,000 individuals and, more than that, 80 percent of rare diseases are genetic in origin."
Because of these factors and the relatively small number of people facing these rare diseases, innovation is not in such high demand, Ezell said, especially when developing and testing new drugs is such an economically taxing task.
"It's a process that for biologics today (that) can be as much as $2.6 billion and 12 to 14 years of development," Ezell said, speaking of the investment, development and testing of a new drug. "There need to be mechanisms in place to encourage and incentivize companies to invest in these types of rare diseases that have small populations."
Ezell said one of the biggest influences in such innovation coming to pass is smart policy, referencing the Orphan Drug Act of 1983. Ezell said that before this act, the FDA had approved under 10 orphan drugs in the 1970s.
"We have the FDA approving over 500 orphan drugs since the act's passage in 1983," Ezell said. "As of December 2014, the FDA has granted the orphan drug designation to a total of 3,200 potential therapies."
ITIF believes that the OPEN Act, the Orphan Product Extensions Now Accelerating Cures and Treatments Act, which was passed by Congress has helped to increase incentivization, as well as the Orphan Drug Exclusivity Provision, which gives companies freedom to market their successful, FDA-approved medications for seven years.
"We believe that the Orphan Drug Exclusivity Program has been a very important mechanism to encourage investment in rare diseases because it really gives the innovators the ability and time to recoup their R&D inspection costs," Ezell said.
Ezell concluded by saying that it is deeply important that the U.S. continue investing heavily in life-sciences innovation, pointing out that over the past decade, there has been a 13 percent decrease in government funding of life-sciences research.
"It's important that we continue on a bipartisan basis to really embrace this framework that is robust private and public sector investment in life-sciences research," Ezell said.
