Mar 03 2017
During Trump’s recent address to Congress he referred to 20 year-old Megan Crowley who has Pompe’s disease.
“Megan’s story is about the unbounded power of a father’s love for a daughter,” Trump said. “But our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need.”
This statement, an unwarranted and factually-challenged attack on the FDA is all the more frightening when put into context. He has appointed to the FDA Jim O’Neill, who said in 2014:
“We should reform FDA so there is approving drugs after their sponsors have demonstrated safety—and let people start using them, at their own risk, but not much risk of safety. Let’s prove efficacy after they’ve been legalized.”
Not requiring evidence for efficacy for drugs would be an unmitigated disaster. It also makes not sense – the concept of “safety” cannot be entirely separated from efficacy. Drugs are evaluated and their use determined by risk vs benefit. You cannot do a risk vs benefit assessment if there is no data on benefit.
Also, how are you going to prove efficacy after a drug is already on the market? The incentive to invest in careful clinical research is now largely gone. Uncontrolled data is all but worthless in determining efficacy. We will go back to the patent medicine days where the public was subjected to countless dubious and largely worthless products with unfounded claims (basically the supplement industry of today).
Trump has indicated that he is serious about this. In a meeting with Pharmaceutical CEOs in January he said:
“We’re also going to be streamlining the process, so that from your standpoint, when you have a drug you can actually get it approved, instead of waiting for many, many years.”
“We’re going to get the approval process much faster. One thing that’s always disturbed me is that you come up with a new drug for a patient that’s terminal and FDA says you can’t have this drug used on this patient and patient will be dead… we don’t know if drug works or doesn’t work, but the patient’s not going to live for more than 4 weeks.”
It is no surprise that Trump’s ideological and simplistic view is at odds with reality.
First, do not assume that I am coming from a position of being pro-regulation or big government. I am not. I am for minimalist evidence-based regulations. The market needs regulations. But each regulation should be carefully constructed, evidence-based, and monitored for its actual effects in the real world (included unintended consequences).
Further, we need to consider the cumulative burden of all regulations. Even if each individual regulation is reasonable, in the aggregate they can represent an unreasonable burden.
So I get it – clearing the decks every now and then by seriously looking at regulations and pruning those that are not essential is necessary to keep the system from getting too sclerotic.
However, simplistically slashing needed regulations is reckless and dangerous.
It can be argued that the legislators and the FDA over the years has done a decent job of finding a near-optimal balance of protecting the public while streamlining the process (for drugs, not for supplements and certainly not for homeopathic products). If drugs are approved too quickly, then too many worthless or harmful drugs will make it to the market. If they are approved too slowly, then patients will miss out on needed medications. Creating the best outcome for the health of Americans is all about finding the best balance.
One way to assess the current balance of the FDA approval process is to compare it to other countries. A recent BMJ study did just that. They found:
From 2001 to 2010, 282 drugs were approved in the USA, Europe or Canada, including 172 (61%) first approved in the USA, 24 (9%) never approved in the USA, and 86 (30%) approved in the USA after Europe and/or Canada. Of the 110 new drugs approved in Europe and/or Canada before the USA, 37 (34%) had a novel mechanisms of action compared with drugs already approved by the FDA, but only 10 (9%) were for conditions lacking alternate available therapies in the USA at the time of ex-US approval—of which the majority (9/10; 90%) were indicated for rare diseases. 12 of the 37 agents with novel mechanisms of action approved first in Europe and/or Canada (32%) had their initial FDA submissions rejected for safety reasons—including 2 drugs that were ultimately withdrawn from the market in Europe due to safety concerns.
What this means is that, overall, the US is quicker to approve drugs than Canada and Europe. For those drugs in which the US was slower, or never approved the drug, many were unsafe and 2 were even withdrawn from the Market.
If you want a good historical example of this, think thalidomide, which was approved in Europe but never in the US because of safety concerns that eventually were vindicated.
So, on balance it looks like the FDA is more efficient than comparable agencies in Canada and Europe. American consumers get drugs faster, while simultaneously being protected from more harmful drugs, and only rarely have delayed access to useful drugs without existing alternatives.
Further, the FDA already has a program for expanded access (compassionate use). This allows terminal patients without options to gain access to experimental drugs before being approved. The FDA approves 99% of applications for expanded access.
So essentially, Trump is trying to fix a problem that does not exist. In the process he will destroy a system that is already near optimal.
Further still – even many pharmaceutical CEOs do not want deregulation. When an industry wants to be regulated, and is arguing against proposed deregulation, you have to take that seriously. (This also strikes a blow to common “Big Pharma” conspiracy theories.)
“People often argue that the FDA is too restrictive,” said Roger Perlmutter, head of research and development at Merck & Co Inc. “We have the sense that the balance is pretty right … you have to have a well-characterized risk/benefit profile.”
The pharmaceutical industry understands their customers, which are as much doctors and insurance companies as patients. They are concerned that doctors will not prescribe drugs, and insurance companies will not pay for them, if there is not adequate evidence for efficacy.
“Any change at the FDA that allows drugs to be tried out on patients without clinical evidence is a damaging approach,” said Jeremy Levin, chief executive officer at Ovid Therapeutics Inc., which is developing drugs for rare diseases.
Trump, however, apparently believes in the magic wand approach to complex policy. Just slash regulations and everything will miraculously work out.
It is especially concerning that Trump gives no evidence of listening to experts. In fact, he tends to disparage experts and expertise in favor of his preferred (and often simplistic) narrative. This approach is what has led him to claim for at least a decade that vaccines cause autism, despite being publicly corrected many times. That is another policy disaster in the making.
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