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Orphan drugs may target small patient populations, but they continue to rack up big numbers at the Food and Drug Administration.

Last year, the agency issued a record-breaking 77 orphan drug approvals, far exceeding the previous annual high of 49 approvals notched in 2014. At the same time, the FDA agreed to award a stunning 476 orphan designations, well above the 355 designations made in 2015, which was the previous record total. And there were 526 designation requests last year, second only to the record of 582 made in 2016.


The increase in designations is likely due to the Orphan Drug Designation Modernization Plan, which the FDA used to eliminate a backlog of designation requests, according to the FDA Law Blog, which tallied the movement of orphan drug requests and applications through the agency. A designation means the FDA has decided a drug qualified for orphan status and occurs before a drug is approved.

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  • That is to be expected. They do not have any new drugs that are substantially better than the existing drugs and whatever they are bringing to the table are unaffordable. Question of survival and revenue generation is in their plate and they need decide how to proceed.

  • And Commission ScottG “questioned whether the financial incentives should be different for drugs that receive “secondary approvals.” ‘ How about, “Obviously, Commissioner.” But then again, he doesn’t write the laws, does he?

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