IOM report seeks to ramp up R&D war against rare diseases

The Institute of Medicine has sized up new efforts aimed at spurring greater research into therapies for rare diseases and pronounced itself dissatisfied by the progress that has been made so far.

The NIH has recently determined that it will get directly in the game of developing new therapies for rare diseases. And the FDA has set out to encourage more orphan drug filings. But to really ramp up activity in the R&D field IOM's experts say that animal models and biological data for rare diseases should be publicly pooled to rev up new programs. And they want a national task force formed to make sure that the NIH, FDA and patient advocacy groups-big players in this space-coordinate their work better.

The problem, says the IOM report, is that too many researchers and R&D advocates are working in isolation, a common issue in drug development across the board and particularly in rare disease work, where researchers stay fixed on individual disorders. The IOM wants to break down those barriers and it may get some help from Congress.

"We feel we already launched a war, and now it's time to escalate," Timothy Coté, director of the FDA's orphan products development office, tells the Wall Street Journal.

- read the WSJ article

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