Despite the fact that the FDA continues to perform woefully relative to its statute-defined target review goals by PDUFA and MDUFA, its campaign of public relations continues unabated. It is truly unprecedented to see the FDA Commissioner and high ranking officials address the public as often as we have seen over the past 6 years.
Indeed, even as the outgoing Commissioner departed, the opportunity for more PR was taken. At least, there was a new talking point – “In the race for the newest treatment we must remember the point that innovation doesn’t matter if the product doesn’t work.”
And, this sentiment was expressed two weeks later by other FDA officials in an Op-Ed – “It is important to remember, however, that innovative therapies only save lives if they work properly. U.S. citizens rely on the FDA to ensure that the drugs they take are effective and that their benefits outweigh their risks. Improving a patient’s life or lifespan must be central to the concept of drug innovation.”
Unable to convince the Congress, which is pursuing the 21st Century Cures proposal by Reps. Fred Upton (R-Mich) and Dianne DeGette (D-Colo.) precisely because the FDA is not performing in compliance with the statute-defined performance goals, the FDA has switched tactics. Maybe not switched, rather added a tactic. Their preferred talking point for the past year was relative performance to other countries:
“As a result, the FDA consistently reviews new drugs faster than all other advanced regulatory authorities around the world, including the European Medicines Agency, and American patients are frequently the first in the world to receive new, innovative drugs.”
Well, the news is full of examples where drugs are approved in Europe and other countries first, so that message isn’t working. But more important is that even if the FDA performed as well or slightly better than Europe, is that cause for celebration? What if the US military performed as well as, or slightly better than the military forces of France and Britain in WWII? You get my point.
No, the real measure is the law and the law states that new drugs will be reviewed in 10 months. The FDA has been very deceptive by reporting days, not months. And, not calendar days; rather, review days, where the clock is stopped and started at FDA’s whim. Even with that, the best the FDA can say is that the median review time is 304 days, which means that the FDA is breaking the law (taking longer than 10 months) on half of all drug reviews.
Time for a new tactic.
The new approach is to attack the innovative products, themselves. The law says that new products must be safe and effective. All Americans want that, including those who are involved in the discovery, research, development, marketing, and dispensing of drugs, biologics, and medical devices. Developing new drugs is not easy – the industry stops the development of products that are not showing promise routinely, for example, the development of a drug to treat diabetic gastroparesis (sluggishness of the digestive system) was just terminated. The FDA is not alone in wanting safe and effective products.
Insidiously, however, the FDA is establishing a new standard – “saving lives and improve life or life span.” The law does not say this – rather, it says “safe and effective.” Many products are quite effective even if they are not shown to save lives or improve life in pre-approval clinical trials. One of the wonderful things about our scientific advances of late has been in diagnostics, which allow us to treat diseases earlier and use tests as measures of the effectiveness of new products. This is known as surrogate endpoints.
If we are now to permit the FDA to approve only products that “work” by their new definition, we will see medical innovation come to a grinding halt as development time and costs increase.
The FDA needs to follow the law, approve drugs that are safe and effective, and let doctors and patients use these drugs in the real world to determine which safe and effective drugs, biologics, and devices are the most beneficial for them. This is how the system was designed, for good reason.
Americans want new and innovative safe and effective products that may help. The FDA needs to listen and to stop putting the cart before the horse.
Gulfo is the executive director of The Rothman Institute of Innovation and Entrepreneurship at Fairleigh Dickinson University and author of INNOVATION BREAKDOWN: How the FDA and Wall Street Cripple Medical Advances (Post Hill Press). He has more than 25 years of experience in the biopharmaceutical and medical device industries and is former CEO of MELA Sciences.
This article originally appeared on www.thehill.com on April 22, 2015.