Victims of rare diseases often are left without options from the pharmaceutical industry, but a new federal government effort aims to change that.
As the Associated Press reports, the National Institutes of Health will open a center this fall to speed genetic research on potential drug therapies for rare diseases. The government work will focus on the trickier early-stage investigations, which have a high rate of failure, with the hope that drug companies will step in once the potential markets for some of the medicines becomes apparent.
The more customary scenario is that pharmaceutical companies, aiming to come up with a landmark medicine, will invest enormous sums in developing a drug. They do so knowing that they have the potential to earn fat profits if they succeed in coming up a medicine that will treat a large number of people.
Yet that calculus doesn’t work for victims of, say, Niemann-Pick Type 3, a fatal disease that afflicts only a few thousand people or less per year.
Still, the victims of rare diseases, collectively, add up to a huge number. More than 20 million Americans suffer from one of 7,000 rare diseases, which are defined as those with fewer than 200,000 victims, including many with far fewer victims.
Officials said that success on one rare disease can lead to spillovers in treating others, including common ailments. “Getting a home run with a rare disease sometimes points you in a direction that will be beneficial for common diseases,” Dr. Francis Collins, the director of NIH, told the AP.
Legislation recently introduced in Congress also seeks to give drug companies incentives to pursue treatments for rare diseases. The Creating Hope Act would offer pharmaceutical companies a voucher for faster evaluation of a potential blockbuster drug in exchange for a groundbreaking treatment for a rare disease affecting children.
“We have to give drug companies a reason to go into this market,” says Nancy Goodman of Kids v Cancer, a group pushing the bill.
