The pursuit of orphan drugs remains high and why not? The Orphan Drug Act, which encourages development of meds for diseases with small markets, gives drugmakers seven years in which to sell a product without competition for seven years and sometimes tax incentives, too. So it is not surprising that a record 323 requests for designation were submitted to the FDA last year.
And the agency's Office of Orphan Products Development obliged by designating 192 meds with orphan status. As a result, there are now 2,308 drugs with this designation, as noted by the FDA Law Blog, which scanned the FDA orphan drug database. However, the FDA approved only 14 orphan meds last year, down from 17 approvals in 2009 and down from a high of 25 back in 1996.
Why is there so much interest lately? Hard to say, although it is reasonable to speculate that FDA workshops garner attention (here's one) and, of course, the opportunity to get a lock on a market for several years is all-the-more tempting at a time when FDA scrutiny of new drugs is particularly high and drug development is so expensive.
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I think there are reasons, not very mysterious: 1/ Today's orphan drug approvals are most likely to match up with designations that are 2 to 6 years old. There is every reason to think this year's large number of designation will result in approval of more orphan drugs in a few years.. 2/ Science is creating an accelerating number of opportunities to attack rare conditions. 3/ Amidst a difficult IP environment, orphan market exclusivity is the one that can't be challenged. As a result, Wall Street has placed more value on orphans. 4/ As personalized medicine becomes closer to reality, it creates more drug products with populations under 200,000. 5/And yes, FDA deserves lots of praise for its outreach programs and that is generating designation requests. Steven Grossman sgrossman@fdamatters.com
Ed- I think it bears mentioning that another approach used by the FDA to encourage innovation in new product development was the creation of medical foods. This occurred under the same Orphan Drug Act. While these products are not listed as orphan "drugs" per se; medical foods are consistent with the new approach of medicine that is more "personalized". See the FDA definition below. Jim Currie jcurrie@pamlab.com
The term medical food, as defined in section 5(b) of the Orphan Drug Act (21 U.S.C. 360ee (b) (3)) is "a food which is formulated to be consumed or administered enterally under the supervision of a physician and which is intended for the specific dietary management of a disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation."
What am I missing?
There seem to be multiple companies sinking millions into pursuing clinical trials, with orphan drug designations, for drugs for the same disease with the same mechanism of action.
Searching the clinical database for something like "xyz inhibitor" turns up multiple candidates for the same treatment.
Why does a company two years behind the competition persevere?
I plead for a response to that, and also: how can Amarin take a medical food through Phase III trials as a drug?
They are proving fish oil, specifically EPA with a reduced amount of DHA, as a "drug" for lowering LDL.
They are conspicuously NOT going head to head with a wild-caught sockeye salmon sandwich.