It takes years of testing and millions of dollars to get a new drug approved in the U.S., and that's for drugs that treat major illnesses. For rare conditions, the process of getting approval for a new drug can take much longer, because of the lack of financial incentives to develop them.
This week the the Food and Drug Administration was in Minneapolis to help companies, researchers and even patients cut through the bureaucratic process to get drugs for rare diseases approved more quickly.
The FDA has been offering incentives to companies that develop treatments for rare diseases since 1983, when the Orphan Drug Act became law.
But over the past 27 years, only 2,200 applicants have been granted the special designation. Of those, 358 have gone on to develop a marketable product.
The FDA's Dr. Timothy Cote thinks there are many more researchers and companies who could benefit from the orphan drug designation. But he says they're often overwhelmed by the regulatory process.
"We have an opacity problem at the FDA. It's a big black box. Nobody knows what they're thinking inside," said Cote. "And we're smashing that black box. We're making it transparent and we're showing people that what we're doing is reasonable, that we don't bite, and we are just as interested in getting new drugs for people with rare diseases as the sponsors are."
Rare conditions are often described as orphan diseases when there are very few treatment options, largely due to a lack of money for drug research. There are about 7,000 known orphan diseases in the U.S.
Cote, who directs the Office of Orphan Products and Development, says most of those diseases have no approved treatment. For that reason, he wants to double the number of orphan drug designations that his office approves in the next five to 10 years.
The designation is just the first step in the long process of getting drug approval. But for many companies it opens a door that otherwise would remain shut.
The designation comes with a 50 percent tax credit on any clinical trials that need to be conducted. Drug fees, which currently cost around $1.6 million for one drug application, are eliminated. And a company that succeeds in getting final FDA approval will be allowed exclusive drug marketing rights for seven years.
But more than that, Cote says the designation brings the new drug to the attention of investors.
"When you get orphan status designation, you go up on our website saying that the FDA has given a nod that this is promising for this rare disease," said Cote. "And then the venture capital rains down from the heavens. That is how it has tended to work for a lot of these companies."
Until that designation is secured, though, most companies prefer to keep a very tight lid on what they're developing.
Thirty-seven small businesses or research groups attended the Minneapolis workshop. Three groups were from Minnesota, but Cote said he couldn't identify them without their permission. In fact, most of the attendees only knew each other by their first names and last initials. That's all that was printed on their name tags.
But for Dr. Neera Gulati from Buffalo, New York, there was no financial or competitive reason to keep her application details secret.
"My son Suneel Ram has a fatal, devastating disease, Duchenne muscular dystrophy, and we're here to try to help save him," said Gulati.
Gulati and her husband Ken Manning have been searching for a promising drug to treat Suneel's condition for the past seven years. Gulati says her son is now 13 years old, and time is running out.
"Without it, boys die in their late teens, early 20s. So we're getting a little farther along," said Gulati.
Gulati and Manning created a foundation that is funding the work of an Australian researcher, who has developed a drug they hope will work for Suneel. Their next step is securing drug approval. That's what brought them to the FDA's workshop in Minneapolis.
Manning was expecting the application would take months or even a year to complete. But he and his wife finished their paperwork, with significant help from FDA staff, during their two days in Minneapolis.
"It was a little bit of a surprise to us to see the FDA reaching out to make it easier for people to develop disease designations. That's actually a big help," said Manning. "It's a big step forward," his wife Neera Gulati added.
The FDA says it will rule on all of the applications it received while in Minneapolis within 60 days. Typically, between 55 percent and 70 percent of orphan drug status applications are approved.