Feds ramp up new efforts to spawn rare disease R&D

Faithful FierceBiotech readers should be acutely aware by now just how popular rare diseases have become in the drug development field. Big Pharma companies like GlaxoSmithKline, Novartis, Pfizer and Sanofi are jumping into the rare disease field, anxious to mimic the success of such biotechs as Genzyme, which found out years ago that small patient populations are capable of paying large sums for treatments. But there's a lot more going on behind the scenes to make the field even more popular in the biopharma world.

The AP tackles the story, first by suggesting that pharma companies haven't been all that interested in rare diseases. Many are now, as the story notes at a later point, but the interest is so new that we're still years away from seeing new therapeutics emerging from the initiatives. So government agencies and lawmakers are working to jump-start new drug programs.

The NIH's new early-stage research unit, slated to get under way later in the year, should help by ramping up programs that biopharma companies can pick up at a more mature stage of development. The FDA is bird-dogging older therapeutics that show promise in being repurposed for rare diseases. And then there's the "Creating Hope Act," which would offer developers the inside track on a faster review for one of their big market drugs in exchange for a new drug that treats rare or neglected diseases that threaten children.

There is a good development argument in favor of these programs. "Getting a home run with a rare disease sometimes points you in a direction that will be beneficial for common diseases," NIH chief Francis Collins told The Associated Press. But pharma companies also know that in a world with fewer blockbuster contenders, going rare makes good business sense.

"The industry as a whole has a pipeline problem. It's increasingly difficult to develop drugs for common diseases," says Pfizer's Ed Mascioli.

- here's the article from the AP

Special Report: Rare diseases: All the rage in Big Pharma

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