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

Advancing “Breakthrough” Drug Therapies

By:  Janet Woodcock, M.D.

Thanks to a recent law that went into effect on July 9, 2012, FDA now has a new program to help expedite the development of new drugs that could potentially offer a substantial improvement over existing therapies for patients with serious or life-threatening diseases. The new law is designed to get “breakthrough” therapies developed as quickly and safely as possible so they can be available to treat the patients who need them. We’re excited about it!  In fact, although the law is only a few months old, we’re already putting it to use. Recently we identified the first therapy to receive this special designation. And it likely won’t be long before we have more. Several other drug developers have already made inquiries and there is lots of interest in the pharmaceutical industry in taking advantage of this new development tool.  

Janet Woodcock, M.D.The law is called the Food and Drug Administration Safety and Innovation Act, or FDASIA for short. It defines a “breakthrough” therapy as one that is “is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.” In other words, a breakthrough drug is one that may offer important new benefits for patients with serious or life-threatening disease who are especially in need of new safe and effective treatments. 

This new option will complement the three programs we have used for many years to help speed up the development and FDA review of especially important new drug therapies.  They’re called “expedited drug development and review” programs, named Fast Track, Priority Review and Accelerated Approval. Each one is different, but for simplicity, think of them as various ways of bringing potentially important new therapies to patients sooner.  These programs have been very successful and are part of the reason that FDA leads the world in first approvals of innovative new drugs.

We’re delighted now to have another tool to help expedite the development and approval of products with the “breakthrough” designation. We’ll continue to use our existing tools and the new “breakthrough” authority to make our expedited drug development process even more effective, with the ultimate goal of benefiting patients with unmet medical needs.

I’d like to assure the American public of one important aspect of all of FDA’s development and review programs and procedures. We always decide whether to approve a drug after evaluating whether its benefits outweigh its risks. Regardless of which development or review program we use, FDA never compromises its safety or efficacy standards in exchange for rapid approval. That means that those drugs approved under the new “breakthrough” designation will meet our usual rigorous standards for safety and effectiveness. We intend to continue to maximize the value of our new breakthrough therapy designation. So stay tuned!

 Janet Woodcock, M.D. is the Director of FDA’s Center for Drug Evaluation and Research