This Groundhog’s Day, Punxsutawney Phil saw his shadow, suggesting the unseasonably warm season might be taking a turn for the frigid. Nowhere has it been hotter this year than in the life science industry, which saw blazing Food and Drug Administration approvals of a handful of new, game-changing drugs. But unlikely the weather on the Eastern seaboard, it’s unlikely that the groundhog will bring a big chill to biopharma.
What made January extraordinary for drugmakers was not merely the speed with which the FDA acted (at least two were approved well ahead of the agency’s deadlines), but the fact that the highest–profile of the major approvals came in so-called orphan diseases, which affect less than 200,000 patients and can afford developers additional market exclusivity.
The trend of serving smaller patient populations with more targeted drugs is almost certainly going to increase, thanks to better technology and an better-understood business model for serving smaller groups. The best evidence for this the number of drugs for which orphan designation was granted. For years, this number hovered in the double-digits. Then, in 2004, 133 drugs were given orphan status. Last year, 199 made the cut. If the trend holds, we’ll top 200 in 2012. (See chart.)
And this is a leading indicator: many of these drugs are years from getting an FDA nod. But the number of approvals is rising, too. A record number of orphan indications — 26 — got FDA approval last year, and it’s not a stretch to think that that number will rise, too.
The race to explore orphan indications marks a fundamental shift in the way breakthrough therapies are introduced. The number of drugs that are aimed at populations of millions and millions are shrinking. And while we won’t see the end of primary-care-driven blockbusters, they will certainly become more rare. That’s going to mean a move for those in communication to get closer to the smaller patient and provider populations: less flashy celebs, fewer DTC ads, more emphasis on trade publications and narrow online communities.
It’s always dangerous to make large conclusions out of a few data points. Regression to the mean means that it will get cold again. And the FDA isn’t likely to have many weeks where they sign off on three new drugs. But the surging number of orphan designations means that — over the long term — the orphan drug climate is likely to stay hot, no matter what the groundhog says.