For a Taste of Government-Run Healthcare…

 

…look no further than the FDA, which is a bureaucratic behemoth that threatens public health, stifles drug innovation, and keeps costs high. As I argue in my column this week for Defining Ideas, if the Supreme Court upholds Obamacare, we can look to the FDA for our model of what government-run care will look like. 

In his recent article in National Affairs, “Changing the FDA’s Culture,” Dr. Scott Gottlieb rightly takes the FDA to task for its retrograde attitudes on the approval of new drugs for sale in the United States market. But he is far too kind to the FDA. Its culture is beyond repair by external exhortation. The FDA is a bureaucratic monopolist. Currently, in the name of protecting the American public, the FDA represents a major systematic threat to drug innovation and public health. It should be stripped of its power to block the entry of new drugs and medical devices into general use.

In making his case for a more modest response, Gottlieb begins with a story of the drug Elaprase, which has been used since 2006 to treat Hunter’s Syndrome, a horrific but rare children’s disease. The disease is caused by the want of a single enzyme needed to break down sugar molecules inside the body. As sugar molecules build up in concentration, the disease cripples and often kills children from a very young age.

As Gottlieb notes, progress against the disease was slow, and the first signs of real hope appeared in the early 2000s when researchers found a way to develop enzyme replacements in related enzyme deficiency diseases. When Elaprase first became available in 2004, the sensible response for the FDA would have been to ensure that the product did not kill patients at small dosages, and then to let it into the marketplace, with due notification of its experimental nature and any known side effects.

The logic behind this position is as follows: First, the deadly course of Hunter’s syndrome has been well documented; second, there is virtually no downside in giving the drug to afflicted children who have literally no other alternative. The desperate parents were not fools. They were likely advised by the finest doctors. They were able to network with other families to swap information about the course of the disease during treatment. They knew how to calculate the expected value of a new treatment. That ex-ante calculation was strongly positive even if the drug had adverse side effects and did not work in many cases. A ten percent chance of saving a child is greater than no chance at all. Get the drug out there, and if it does not work, it will be rejected by the market. If it does work, it will save dozens or hundreds of lives.

But the FDA marches to the beat of a different drummer. Its institutional blindness, masquerading as science, ultimately costs lives, as I explain further over at Defining Ideas.

There are 17 comments.

  1. Israel P. Inactive

    If bureaucrats say “yes,” they understand that to mean they are not necessary. Anyone can say “yes.”

    Bureaucracies are designed to start off with “no” with the burden of proof on the petitioner. No power, no sense of purpose.

    • #1
    • June 26, 2012, at 3:37 AM PDT
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  2. raycon and lindacon Inactive

    Bureaucratic leadership consists of aligning oneself to gain credit from success and immunity from failure. You are never wrong with a no since no action can be attached to your decision. A yes however carries with it the risk that the actor could fail and soil the bureaucrat’s position.

    Every government entity has this characteristic. 

    We weave the ropes of our own binding when we support big government.

    • #2
    • June 26, 2012, at 5:47 AM PDT
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  3. KarlUB Inactive

    I think you misread Gottlieb a little bit, in that you did not mention what I thought was his key recommendation: Separate within the FDA the functions of setting up the clinical requirements for approval, and signing off on the approval. This would be an improvement, I think.

    And one issue neither of you touch– which touches me every day– is the FDA approval that is necessary for drug marketing to healthcare professionals. While it is obviously true that pharmaceutical companies will try and put lipstick on a pig whenever they can, doctors are smart people.

    If the FDA would just loosen the reins in this regard the companies themselves would be freer to communicate both the benefits of their products, and the demerits of the products of their competitors. Currently, instead, the communications companies design for doctors are slow, boring, and pretty anodyne. The FDA expends tremendous effort in paralyzing the companies– and fining them huge sums of money– when it could just be concentrating on evaluating and approving drugs for use.

    Example: The FDA has been promising clear regulatory guidance on digital communications for physicians since the late nineties. We are still waiting for that guidance.

    • #3
    • June 26, 2012, at 5:50 AM PDT
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  4. KarlUB Inactive
    raycon: Bureaucratic leadership consists of aligning oneself to gain credit from success and immunity from failure. You are never wrong with a no since no action can be attached to your decision. A yes however carries with it the risk that the actor could fail and soil the bureaucrat’s position.

    Every government entity has this characteristic. 

    We weave the ropes of our own binding when we support big government. · 3 minutes ago

    To be fair, I think this is common in business as well. It is a huge problem when it comes to the marketing communications I have discussed above, as well:

    Nobody wants to be responsible for final sign-off for fear of being held responsible if some bureaucrat at the FDA (or the internal review team at the pharmaceutical company) thinks you are being deceptive by omitting a P-value somewhere.

    • #4
    • June 26, 2012, at 5:53 AM PDT
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  5. Bryan G. Stephens Thatcher

    This is why your entrepreneurial types take risks: They are not afraid to fail. That is where greatness comes from.

    I agree with taking down the FDA. We have a shortage of drugs because the FDA controls how much can be made. Does that even make sense?

    • #5
    • June 26, 2012, at 6:01 AM PDT
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  6. reidspoorhouse Inactive

    The system regardless is too slow. People die needlessly. My Grandmother died of TB in 1941, penicillin was discovered in 1927. Seems to me that there should be a clearer path for medications for the terminally I’ll.

    • #6
    • June 26, 2012, at 6:10 AM PDT
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  7. Profile Photo Member

    Could we limit the FDA’s role to only determine whether a product does no harm. The free speech stifling they do in the marketplace is also damaging to society. For example:

    I’m sure some of us remember the folks that wanted to save the rainforest make the arguement that certain flora or fauna found in it could cure diseases.

    One of the other problems with the FDA is that if they did find such a plant. The labeling of said product is not allowed to advertise as such.

    • #7
    • June 26, 2012, at 7:22 AM PDT
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  8. Viator Member

    There is one group of Americans who have secured a special carve out from FDA regulations:

    “HIV also has special status among regulators. America’s Food and Drug Administration (FDA) created a faster way to review HIV drugs, allowing them on the market before the most expensive stage of clinical trials.”

    further:

    “More than 60% of HIV drugs in America are bought with public money. Insurers give HIV special treatment: patients are rarely pressed to buy the cheapest pills, as they might be if they had another disease.”

    Which makes one ask, why don’t the rest of Americans have this special status?

    More people die horribly of pancreatic cancer annually (35,628 – 2009) than HIV (9,406 – 2009). Shouldn’t pancreatic cancer patients get equal treatment under the law?

    http://www.economist.com/node/21556275

    http://www.cdc.gov/nchs/data/dvs/deaths_2009_release.pdf

    • #8
    • June 26, 2012, at 7:26 AM PDT
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  9. Shane McGuire Member

    How about this—you have two classes of drugs, those with FDA approval, and those without. Kind of like the toothpastes that are endorsed by the ADA and you have others that aren’t. That way, the drugs are on the market, but the patient must be advised that they haven’t been approved by the FDA.

    This brings up some tort issues—-whether there’s contributory negligence on the part of a patient who knowingly takes a non-FDA approved drug, but those can be worked out.

    • #9
    • June 26, 2012, at 8:29 AM PDT
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  10. KarlUB Inactive
    Aelreth:

    I’m sure some of us remember the folks that wanted to save the rainforest make the arguement that certain flora or fauna found in it could cure diseases.

    One of the other problems with the FDA is that if they did find such a plant. The labeling of said product is not allowed to advertise as such. · 1 hour ago

    Well, interestingly, not quite. If said product was just a straight derivative of the plant, you can make all sorts of claims without the FDA coming down on you like a ton of bricks. Or, to be more accurate, you can imply all sorts of claims with vague, hedging language.

    The real force of the bureaucratic Gods does not apply to supplements and food in the same way that it applies to pharmaceuticals. Which is another thing that makes zero sense…

    • #10
    • June 26, 2012, at 10:03 AM PDT
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  11. Mendel Member

    Let me defend the FDA. 

    The process of determining drug efficacy – whether a drug is actually useful, and not simply non-toxic – is incredibly expensive and scientifically challenging. Due to the variable nature of reactions to medications, large numbers of patients from different groups must be recruited for double-blind studies (except for the rare really good drugs, for which anecdotal evidence may suffice).

    There is also a huge demand for a certifying agency from both doctors and patients. Doctors may be smart people, but they don’t have the time to individually evaluate each new medication. And even if they did, they would still need the large-scale surveys mentioned above.

    Without the FDA, I imagine that an independent verification agency (think UL) would spring up. However, the incredible cost of verifying a new medication (much more than testing an electrical product) would mean that the pharmaceutical companies would end up footing the bill, and we end up in a situation where Arthur Anderson is paid by the companies it audits.

    • #11
    • June 26, 2012, at 10:15 AM PDT
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  12. Mendel Member

    The other key point is that the FDA is necessary as long as government pays for a large share of drugs in the medical economy.

    As opposed to many other products, when a new drug comes out there is often a huge unmet need, and thus patients want to have the drug even if it is unproven. Since the elderly both take the most drugs and get the most subsidy from the government, an unregulated drug market would mean a deluge in consumption of useless pills.

    I assume that Mr. Epstein is also in favor of shifting the cost of drugs to patients, but it is dangerous to call for the FDA to be stripped of its gatekeeper function without first removing the government from drug purchasing all together.

    • #12
    • June 26, 2012, at 10:22 AM PDT
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  13. raycon and lindacon Inactive
    KarlUB
    raycon: Bureaucratic leadership consists of aligning oneself to gain credit from success and immunity from failure. You are never wrong with a no since no action can be attached to your decision. A yes however carries with it the risk that the actor could fail and soil the bureaucrat’s position.

    Every government entity has this characteristic. 

    We weave the ropes of our own binding when we support big government. · 3 minutes ago

    To be fair, I think this is common in business as well. It is a huge problem when it comes to the marketing communications I have discussed above, as well:

    Nobody wants to be responsible for final sign-off for fear of being held responsible if some bureaucrat at the FDA (or the internal review team at the pharmaceutical company) thinks you are being deceptive by omitting a P-value somewhere. · 7 hours ago

    The point I was making is that ALL buraucracies… that is, all big organizations, have the same characteristic. Pharamaceuticals have developed the defensive buraucracy required to deal with the FDA.

    Call it BURAUCRACY SQUARED. 

    Lucky us. 

    We get to experience the worst of all possible worlds.

    • #13
    • June 27, 2012, at 1:00 AM PDT
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  14. Caryn Member
    reidspoorhouse: The system regardless is too slow. People die needlessly. My Grandmother died of TB in 1941, penicillin was discovered in 1927. Seems to me that there should be a clearer path for medications for the terminally I’ll. · 7 hours ago

    At the risk of being pedantic (forgive me, but you’re on my home turf–dissertation topic): TB is not treatable with penicillin, never was. The first drug active against TB was streptomycin, discovered in 1944. It was also the subject of what’s widely regarded as the first real clinical trial (randomized, double-blind, placebo controlled). Interesting history, very interesting disease. 

    I have plenty of stories of the random silliness of FDA and the wasted millions of dollars spent complying with truly unnecessary hoops, but only 200 words. And I have to get back to work

    FDA does provide a necessary service, but they do it with typical bureaucratic means. Consequently, all of the problems noted above. We do need to know whether drugs are safe and effective–else we’re just back to patent medicines. Like all governmental agencies, they need a major shave and haircut.

    • #14
    • June 27, 2012, at 2:39 AM PDT
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  15. Caryn Member

    BTW, apropos of my last comment and reidspoorhouse’s. It’s commonly acknowledged by all who deal with the FDA that neither penicillin nor aspirin would get through the FDA approval process. Sure, they’re effective, but so many side effects and look at all the people who have adverse events (allergies?).

    • #15
    • June 27, 2012, at 2:42 AM PDT
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  16. KarlUB Inactive
    Mendel:

    I assume that Mr. Epstein is also in favor of shifting the cost of drugs to patients, but it is dangerous to call for the FDA to be stripped of its gatekeeper function without first removing the government from drug purchasing all together. · 2 hours ago

    I take a back seat to no man when it comes to maligning the FDA, but I still agree with you 100%. Anyone who has worked for– or with– a pharmaceutical company will charitably acknowledge that by the time recruitment for a Phase III study has begun they are under tremendous pressure to realize billions of dollars. A lot can go wrong in that environment.

    That’s why I liked Gottleib’s suggestion that the part of the FDA that does the study requirements and data evaluation should be separated from the part that does the approval. The latter should lie– only– with very senior people who have no dog in the fight when it comes to requirements and analysis. Only such people are equipped to make judgment calls when weighing risk and reward.

    • #16
    • June 27, 2012, at 12:41 PM PDT
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  17. raycon and lindacon Inactive
    Viator: There is one group of Americans who have secured a special carve out from FDA regulations:

    “HIV also has special status among regulators. America’s Food and Drug Administration (FDA) created a faster way to review HIV drugs, allowing them on the market before the most expensive stage of clinical trials.”

    further:

    “More than 60% of HIV drugs in America are bought with public money. Insurers give HIV special treatment: patients are rarely pressed to buy the cheapest pills, as they might be if they had another disease.”

    Which makes one ask, why don’t the rest of Americans have this special status?

    More people die horribly of pancreatic cancer annually (35,628 – 2009) than HIV (9,406 – 2009). Shouldn’t pancreatic cancer patients get equal treatment under the law?

    http://www.economist.com/node/21556275

    http://www.cdc.gov/nchs/data/dvs/deaths_2009_release.pdf · 5 hours ago

    Need I make the obvious comment, or will our libertarain cohort be offended?

    • #17
    • June 27, 2012, at 12:50 PM PDT
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