D

ear 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.

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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.

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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 very helpful.”

On a similar note, Mr. President, the idea of naming Jim O’Neill, a Peter Thiel protégé 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.

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  • Why isn’t sodium citrate 4% (4% Sodium citrate anticoagulant USP) approved in the US as a lock flush solution to replace heparin? Sodium citrate 1.8 gm is in every unit of blood as an anticoagulant. It is used as a pH buffer in injectable products as an excipient. The FDA’s position is that sodium citrate as a lock flush solution needs to be approved as an NDA, then a PMA as a device. Is this the “least burdensome regulatory path”? How is risk benefit being applied compared to exposure to heparin? Just one example of FDA over regulation of a product that could benefit society. The sodium citrate 4% is approved for use in Canada and Europe as a device.

    • Actually “Hep lock” is a term of art. It hasn’t been used in flush solutions for years. It has been replaced by saline, plentiful, safe and cheap.

  • Ed, I agree with the points in your article. However it is only one half the story. The other half, which could speed things up is unwinding the increasingly burdensome regulations governing design and conduct of clinical trials. The “Bible” here is 21CFR12. Drugs for Human Use. Linked below is just one of the many newly bloated regs, on responsibilities of investigators (Form 1572). Now the FDA wants to know everything about any potential financial conflicts of interest by investigators. We have to hand hold a lot of investigators who think that if they own one share of some XYZ drug company in some ABC mutual fund then they must disqualify themselves.

    Just one example.

    http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=312.53

  • Ed, you actually seem a bit misguided. The FDA is a bloated, unelected, unaccountable group of federal leeches. I look forward to seeing significant downsizing of the administrative state. Thank you for opinion though.

  • Excellent commentary. I worked on the analytics for Myozyme, the Pompe’s drug that was approved so had a good view of that product coming to the market. I would rather see more people hired into the FDA and pay them better to attract very high quality talent to improve the entire process.

  • A lot of patient pressure on the FDA dates back to Pres. Obama’s 1st term. An FDA buoyed by a ” Big Govt. can fix all your problems mentality ” . More interested in ” Flexing It’s Regulatory Muscle with Big Pharma “, ( from the WSJ , 2011 ), than in saving patient lives . Delaying approval for drugs that cured end stage cancers – like Kadcyla and Iclusig . Patient activists fighting for the ” Right To Try ” exposed an FDA that delayed all compassionate use requests with hours and hours of bureaucratic paperwork . Pro – FDA writers always cite the ” months ” that the FDA takes to approve – that is only the end of a long, 20th century trial system- 7 to 10 years long, marked by companies fearing the FDA regulatory muscle – that can nitpick and delay approval for years – bankrupting companies , costing execs their jobs like the CEO of Ariad .
    The President is a shoot from the hip , a thin- skinned ” tweety ” type – but with FDA and the slow drug approval system – he is right – it needs a thorough over haul .
    Another note – comparing the FDA and European regulators is like comparing Slow with Slower . Big Govt. Bureaucracy over there is stifling innovation – few, if any , new drugs coming out of the UK and Europe.

    • Attract high quality people by paying more and hire more people to help speed the process. The FDA budget has been cut for years. Given the tons of data input for approval that must be reviewed, more people are needed. OR maybe have clinical trials require new drugs be compared to best current treatment instead of a placebo–would cut down on the number of approvals and me-too drugs that are not that efficacious.

  • The use of ‘burdensome’ in the closing paragraph is certainly a deft turn of phrase. One could think of more barbed phases that would indeed be appropriate but in deference to the office, the term used will serve. That said, do be aware of your surroundings, Mr Ed.

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