The FDA

The new “Right to Try” law is the motive for today’s post.

For a long time it has been an article of faith among many people that it is a responsibility of government to protect the consumer from rapacious and callous businesses.  And so we have had a multiplicity of agencies all intended to “protect” the regulate and restrict those businesses in the name of protection. It’s government paternalism at its finest.

I’m taking a look at the FDA here since that’s the agency with the most nearly universal support.  Who, after all, wouldn’t be in favor of ensuring that the medications people take are safe and effective? (Yes, I know you folk are out there, but you do recognize you’re in the distinct minority, right?)

It all at the turn of the 20th century.  Noted Socialist Upton Sinclair went “undercover” in the Chicago meat packing industry.  As a result of his experience he wrote his novel “The Jungle” as an expose, published in 1904.

Sinclair’s novel was intended to expose the terrible conditions of the workers in the interest of furthering socialism.  Instead the public grasped onto the unsanitary conditions which Sinclair presented in the novel as the way the meat packing was done.

This sparked an uproar that led to the Meat Packing Act and the Pure Food and Drug act of 1906.

There were some surprising allies in this crusade for government regulation of the food and drug industry.  Even before Sinclair’s novel, organizations like The Women’s Christian Temperance Union and the National Temperance Society had formed the National Pure Food and Drug Congress.  One of their targets was the plethora of patent medicines and nostrums which were largely alcohol–which explains the interest of the temperance people.  Another ally was the meat packing industry itself.  You see, although the meat packing industry was well aware that poisoning their customers was simply bad for business (despite what Sinclair had to say in his novel), one problem they were encountering was that foreign markets were blocking the import of US beef claiming that it was diseased.  If they could get the government to certify that it was disease free, and also to pay for the inspections, they believed they could get past that barrier and enter those markets.  Another ally was the pharmacological industry and its trade associations. This one wasn’t entirely economic.  They saw it as a positive public good to regulate the direct sale of ineffective “patent medicines” promising miraculous cures.  That they were in direct competition with pharmacists and the manufacturers of medicines was also a factor, but likely a smaller one.

The result, as I said, was the Food and Drug act of 1906.  This was largely limited to the inspection of foods and the labeling of patent medicines.  There was language subjecting prescription drugs to control but this power was not used immediately.

It was not until the Eixir Sulfanilamide disaster of 1937.  Sulfanilimide was generally taken as a capsule or used topically as a powder.  The elixir form was intended for people who were unable to take capsules.  Tragically, the solvent used was highly toxic to humans although this was not generally known at the time.  The result was a disaster which led to more than 100 deaths (including the chemist who developed the elixir–who committed suicide while awaiting trial for his part in the disaster).

This led to the Food, Drug, and Cosmetic act of 1938 requiring animal testing and approval by the FDA to ensure safety before they could be sold in interstate commerce.  The decision had to be made by the FDA within 180 days.

So things mostly stayed until the lat 50’s and thalidomide.  Primarily prescribed as a sedative or hypnotic, thalidomide also claimed to cure “anxiety, insomnia, gastritis, and tension”. Afterwards, it was used against nausea and to alleviate morning sickness in pregnant women.

It was this last used that led to tragic results.  Thalidomide is a powerful teratogen–interfering in the development of the fetus–and it crosses the placental barrier.  This meant that literally thousands of children were born with severe birth defects including malformed limbs, deformed eye and heart, alimentary and urinary tracks, and many other issues.  Only about 40% of the children survived.

As it happened, the FDA declined to permit thalidomide from being sold in the US.  Once the horrific results of its use in pregnant women became known, President John F. Kennedy awarded the official in charge of the investigation which blocked thalidomide, Frances Oldham Kelsey, the President’s Award for Distinguished Federal Civilian Service.

The result was yet further expansion of the FDA’s power and further requirements that new medicines had to meet before being permitted in the US.

So far, that sounds very much like a good thing.  We get safer drugs and avoid another Elixir Sulfanilamide disaster or another Thalidomide disaster.

But, as always, when one talks of safety the questions must be asked:  compared to what and at what cost.

Both of those disasters were extremely costly to the companies involved in terms of liability lawsuits as well as loss of business.  Now, I can hear the howls now: “How dare you compare the companies’ financial losses to the pain, suffering, and deaths caused by feeding people toxic chemicals and calling them medicine!” Well, it’s not my intent to compare the two.  I am not saying that the financial losses are somehow just compensation for the pain and suffering.  That is not my point.  My point is simply that even from the most coldly pragmatic point of view, companies have an extremely strong incentive to not let another elixir sulfanilamide, another thalidomide, get out the door.  Even without the government regulation simple good business sense and an interest in making money will lead companies to do extra testing in an attempt to ensure that their products are both safe and effective.

Would mistakes happen?  Yes.  But then, mistakes will happen with government regulation too.  And have.  Many times over the years the FDA, as well as regulatory bodies in other parts of the world have changed their minds and withdrew approval for previously approved drugs.  Oops.

So, there will still be “bad” drugs getting out with either a regulatory agency like the FDA overseeing things or if companies are allowed to rely on their own self-interest to encourage testing.

One might still argue that there’s the flip side of the issue.  There are two kinds of mistakes that an organization like the FDA can make.  The first is what we have discussed above:  approving a drug that proves to be excessively dangerous with unacceptable side effects.

The second is to not approve a drug that could alleviate suffering or even save lives.

Consider the class of medications called beta blockers.  One of the uses of beta blockers is that when administered after a heart attack they can dramatically reduce the chance of death–“Secondary prevention of coronary death after myocardial infarction”–to the extent of saving more than 10,000 lives per year.  The first major beta blocker, propranalol, was introduced (not in the US) in 1964.  Simply look at the data on the FDA’s own site on when propranalol was approved for use in the US and do the math.  How many people died because of that delay?  How many elixir sulfanilimide or thalidomide disasters to equal the death and suffering that was simply every day business over those years?

The problem is that if a government bureaucrat approves a drug and it proves to be disastrous, that bureaucrat will be vilified, wrecked professionally and personally, and ill go down in the history books as a great monster.  If, however, that bureaucrat denies approval of a drug that could alleviate pain and suffering, or even save lives, who would ever know?

The incentive is to reject.  If there is any doubt whatsoever, reject.  And even with that incentive mistakes still creep through.  But with that incentive many potentially useful and beneficial drugs are filtered out.  With the result that thousands, possibly even millions, of people suffer needlessly.

I cannot point to direct math calculating costs and benefits, although the case of the beta blockers is telling, but I am firmly convinced that the FDA does far more harm than good.  Most of the “good” it might accomplish is already present in the financial incentives of the companies developing new medications.  The bad is conveniently out of sight of suffering and death not relieved because of medications and treatments that were not made available.

That’s why Right to Try is so important.  It doesn’t go nearly far enough but it is at least a good start.