Regulatory Focus™ > News Articles > FDA Argues its NME Approval Record Holds Constant When Adjusted for Quality of Drugs Approved

FDA Argues its NME Approval Record Holds Constant When Adjusted for Quality of Drugs Approved

Posted 06 August 2013 | By Alexander Gaffney, RAC

Is there an "innovation gap"-that is, a slowdown in the development and approval of truly innovative pharmaceutical products reaching the market-affecting the pharmaceutical industry right now? While the much-discussed theory has more than a few proponents, the US Food and Drug Administration (FDA) isn't one of them, and in a new public missive attempts to pour cold water on something that has "generally been accepted as truth."


In recent years, FDA has been under fire from the pharmaceutical industry for approving fewer new molecular and chemical entities  (NME/NCE) than it did in previous years, such as during the 1990s when it approved a record-high number in 1996 (53).

That number, however-and we should note FDA's historical approval numbers are generally far lower than that, dropping to as low as five NMEs approved in 1969-has flagged in recent years, dropping as low as 17 NMEs approved in 2002, and hovering in the mid-to-low 20s for much of the 2000s before recovering into the 30s in 2011 and 2012.

Is the 'Conventional Wisdom' Right?

But as FDA's Mike Lanthier, operations research analyst at FDA's Office of Planning, argues, perhaps measuring NME and NDA approvals isn't the metric we should be looking at most.

"Conventional wisdom suggests that the pace of drug innovation should be measured by tallying the number of FDA-approved novel new medicines, known as NMEs," Lanthier writes in a new FDA Voice blog posting on the agency's website. "When the number of NME approvals increases from year-to-year, media reports generally proclaim that drug innovation is on the rise; when the number dips, concerns are often raised about FDA's drug review performance and the health of the industry as a whole."

Lanthier argues this view is misguided, because not all NMEs are created equal. That is, by focusing on the quantity of NMEs-what Lanthier calls a "one-size-fits-all approach"-analysts are missing out on the exceptional novelty of some NMEs.

Breaking Down NMEs

To advance his argument, Lanthier splits up NMEs into three classes: First-in-class NMEs representing drugs with novel, never-before-seen mechanisms of action; advance-in-class drugs exhibiting an improvement over existing products that are nevertheless similar to already-approved products; and addition-in-class drugs (sometimes referred to as "me-too" drugs) that are similar to existing drugs in both design and effect.

By breaking out all NMEs approved from 1987 through 2011, Lanthier said FDA "found that the number of NMEs approved every year is largely driven by changes in total approvals of drugs in the addition-to-class category."

"Indeed, a lot of the much-hyped decline in drug approvals from historic highs observed in the mid-1990s occurred because fewer of these addition-to-class drugs were being approved," Lanthier wrote. "In contrast, year in and year out, approvals of the crucial first-in-class drugs have remained essentially the same."

Other Questions Raised

This conclusion in some respects is intuitive, as FDA has long placed an emphasis in its review resources on first-in-class drugs intended to treat conditions for which there is no available cure. But it also raises a number of other questions. For example, if the number of approvals for first-in-class drugs has remained relatively constant, what's behind the variability of addition-to-class drugs? Does a lack of me-too drugs really indicate a lack of innovation or cause harm to patients? And is FDA or industry to blame for fewer approvals?

For now, these are all questions without answers, but if Lanthier's post illustrates anything, it's that FDA-and the Center for Drug Evaluation and Research (CDER) in particular-is of the opinion that, "It's not just about quantity of drugs, it's also about quality."

Lanthier's FDA Voice Post

Health Affairs Journal Article


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Tags: Approvals, NME

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