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STAT Pharmalot – Dear Mr. President: Your view of the FDA is misguided. Here’s why

STAT Pharmalot – Dear Mr. President: Your view of the FDA is misguided. Here’s why


Dear President Trump,


During your speech to Congress last week, you took a whack at the Food and Drug Administration for its “slow and burdensome” approach toward approving medicines. You then suggested that if “we slash the restraints” on the agency, Americans will be blessed with more miracle cures.


To be sure, the FDA is not infallible. Like any institution, the agency is a collection of people who sometimes make mistakes or exercise poor judgment. And the public is right to demand more new medicines more quickly.

But diminishing the FDA is wrongheaded. Why? The agency has largely performed well in recent years.


You’re a businessman, so let’s consider some numbers.


Overall, the median time it took the FDA to approve a new drug was 7.8 months in fiscal year 2016. That’s the fastest pace in more than 20 years. Meanwhile, 95 percent of novel new drugs approved last year were reviewed within the periods of time mandated by law. And while some complain that Europe moves faster, data show that, of all the novel drugs launched worldwide in 2015, 65 percent were approved first by the FDA.


During the past decade, the FDA has increasingly used various tactics to speed approvals of medicines that work in new ways. Last year, 73 percent of the so-called novel drugs were approved using programs such as fast track or breakthrough designations, up from 66 percent in 2014.


And here are more figures to consider: Between 2003 and 2010, 23 cancer drugs were approved by both the FDA and European Medicines Agency, but the median review time by the FDA was 182 days — compared with the 350 days taken by European regulators. And all the drugs that were approved by both regulators entered the US market first, according to Friends of Cancer Research.


This hardly appears to be the track record of a broken agency, Mr. President.


“Slow and burdensome implies that products are being held up by the process,” said Dr. Stephen Goldman, a former director of the FDA’s safety program, known as MedWatch, who is now a medical products safety consultant. “But that’s not the case.”


Yet during your speech, Mr. Trump, you pointed to a young woman in a wheelchair who suffers from a rare ailment called Pompe disease. As you directed members of Congress to look her way, you maintained that the FDA “keeps too many advances, like the one that saved [her] life, from reaching those in need.”


Sorry, but as former FDA Commissioner David Kessler pointed out in a widely read tweet that night, the drug in question was approved in nine months after a clinical trial involving just 39 patients. That’s hardly an example of slow and burdensome.


The young woman’s father, who happens to be the chief executive of a biotech called Amicus Therapeutics, has pointed to a drug that his own company developed for yet another rare disease as an example of FDA inertia. Last year, European regulators approved the treatment, but the FDA recently required additional testing for gastrointestinal symptoms, which means Americans will lack access to the drug for at least two more years.


Was the FDA wrong? After all, if the Europeans can endorse the medicine, why should the FDA not do so? These are fair questions. When the test results come in, we might get more clarity as to whether the FDA was right to slow things down. Whatever the results, this episode will likely be debated as an example of the need to overhaul the agency.


A recent study, however, suggests that Americans are actually not missing out on much.


The analysis found that the FDA moved more slowly than European and Canadian regulators to approve 110 medicines between 2001 and 2010. But only 10 of those 110 drugs, or 9 percent, represented a new way of treating diseases for which no alternative therapy was available in the US, according to the study, published in BMJ Open.


Yes, there were instances in which Americans did not benefit from a drug — or at least not as quickly — because the FDA did not reach the same decision as other regulators. But the numbers are small. Of course, for those patients and families who were affected, all that matters is one delay or rejection.


“This is one of the challenges in this area,” said Frank David, managing director of the Pharmagellan consulting firm and one of the report’s authors. “But at the end of the day, the problem with [Trump’s] view is it basically treats current regulations as pure bureaucracy without any value. This is hard stuff. Each patient matters, but the sound-bite answers about deregulating FDA aren’t every helpful.”


On a similar note, Mr. President, the idea of naming Jim O’Neill, a Peter Thielprotégé and former hedge fund manager with no medical background, as FDA commissioner would be reckless. Why? He has expressed interest in allowing drugs on the market before effectiveness is proven.


Your sound bites may appeal to anyone frustrated with bureaucracy. But if anything is burdensome, it is your misguided notion of improving public health.