Innovative New Drugs Are Reaching Patients at a Fairly Constant Rate: New FDA Study Reports on 25-year record of approvals

By: Mike Lanthier

So much has been said and written about the supposed innovation gap in drug discovery that it’s generally been accepted as truth and a topic of deep angst for the pharmaceutical industry.

And yet, if you take a hard look at the data, as we did, you’ll find it isn’t true. The fact is, the way data is collected has been masking some important facts.

Conventional wisdom suggests that the pace of drug innovation should be measured by tallying the number of FDA-approved novel new medicines, known as new molecular entities (NMEs). 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.

However, while the number of NME approvals in a given year provides something of value regarding the output of novel new drugs, this perennial focus on the quantity of drug approvals may not be sufficient to provide a meaningful measure of “innovation.” This is true primarily because not all NMEs are equally innovative. For instance, one NME may offer an important new way to treat a disease, while another may work in a way that is similar to drugs already on the market.  

To help move beyond this “one-size-fits-all” approach and provide deeper insights into what trends in NME approvals can tell us about innovation, FDA examined NME approvals over the 25 years from 1987 to 2011. We identified three distinct subcategories of novel new drugs: 1) first-in-class, which represents novel drugs that use new mechanisms to treat or prevent disease 2) advance-in-class, drugs that work in ways similar to, but demonstrate significant advantages over, existing drugs, and 3) addition-in-class, essentially representing new drugs that work and perform similarly to ones we already have on the market. 

Using this measure, we found that the number of NME’s 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. In contrast, year in and year out, approvals of the crucial first-in-class drugs have remained essentially the same.  

In other words, if the focus is placed on the more innovative drugs, no evidence of an innovation gap in drug discovery exists, as explained in a paper I co-published with other FDA officials.

While these findings are heartening, there is still great need for further drug innovation.  Recently we’ve seen successful drug innovation in areas of special need, including the first-ever drug to treat the underlying cause of cystic fibrosis in certain patients; new and effective ways to treat various forms of cancer; and drugs to treat lupus and tuberculosis, conditions that until recently had not seen a new drug therapy approved in several decades. However, for many diseases there are simply not enough FDA – approved drug therapies – and for some diseases, none are available. 

FDA continues to work with patients and drug developers to help identify areas of unmet medical need, particularly from the patient perspective. Over the next five years, under the new Patient-Focused Drug Development initiative, FDA will hold public meetings on about 17 additional medical conditions to gain better understanding of patients’ perspectives on the severity of these diseases and their current treatment options. FDA also has a new designation called “Breakthrough Therapy” for new drugs that have the potential to offer a substantial improvement over existing therapies for patients with serious or life-threatening diseases in development. The intent is to provide timely and frequent communication with drug sponsors to help expedite the development and review of these innovative therapies. 

As always, FDA will continue to approve safe and effective new drugs as efficiently as possible, with an emphasis on products that have potential for the biggest beneficial impacts on U.S. public health. And when it comes to assessing the success of our efforts and drug innovation as a whole, one thing remains clear: It’s not just about quantity of drugs, it’s also about quality. 

Mike Lanthier is an Operations Research Analyst on the Economics Staff in FDA’s Office of Planning

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