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.  

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