Mar 03 2017

Trump’s Dangerous Plan to Deregulate Pharmaceuticals

Trump-FDADuring 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.”

and

“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.”

The Reality

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.

17 responses so far

17 Responses to “Trump’s Dangerous Plan to Deregulate Pharmaceuticals”

  1. DS1000on 03 Mar 2017 at 8:28 am

    Most of Trumps ideas are traceable back to extreme conservative, liberatarian, or white nationalist type groups, but this particular crusade seemed to come out of nowhere. Does anyone know how destroying the FDA became a top priority in his mind?

  2. MWSlettenon 03 Mar 2017 at 8:29 am

    The incentive to invest in careful clinical research is now largely gone.

    Except for tort.

    …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.

    Once a regulatory regime is in place it is in the interest of established players to keep it in place. I’m not saying that the pharmaceutical industry isn’t concerned with patient safety, but there is an incentive beyond patient safety for “big pharma” to argue against deregulation. The more regulation in place in a given industry the costlier it is to do business in that industry, thus making it harder for startups and potential competition. A heavily regulated industry is also more open to market manipulation via governmental lobby, which is mostly out of reach to anyone trying to break into the market.

  3. Steven Novellaon 03 Mar 2017 at 9:03 am

    MWS – your points are valid. However, we have the parallel supplement and homeopathic industries to provide guidance. These also sell substances with medical claims. They clearly show that tort can be just a minor nuisance. Further, the supplement industry had no problem adapting to deregulation. They, in fact, exploded (with worthless products).

    Drugs are different in that you need physician buy-in (unless they also weaken those regulations, which is very plausible given Trump’s philosophy).

    I did not say that pharmaceutical CEOs were concerned about patients (I’m not saying they aren’t concerned). They are concerned about doctors prescribing their drugs and insurance companies paying for them. But they also acknowledge that the current balance is pretty good. They do not feel over-burdened.

    I agree with the regulation as a barrier to competition argument, but I do wonder how much that applies to pharmaceuticals because even with weaker regulation is takes a lot of infrastructure and investment to develop new drugs.

  4. tb29607on 03 Mar 2017 at 9:47 am

    I take care of kids so the FDA remains largely irrelevant to mine and most other areas of pediatrics (two notable exceptions being antiviral drugs for HIV and chemotherapy agents). I can not practice good medicine without prescribing off-label (using things without FDA approval). Take the flu vaccine, CDC recommends it for anyone 6 months of age or older. FDA only approves it for those 6 years of age and older. Or asthma attacks. Standard of care includes intravenous or enteral steroids. Not approved. Even a basic treatment like inhaled albuterol is not approved under the age of 4 years old. One of the consequences of this is that no one will make available a pediatric formulary for computerized order entry. So if the individual hospital has not gotten around to pediatric dosing on a drug I am on my own when prescribing. Computer guard rales prevent me from typing in my dose so orders often end up being pharmacy communications. This is no different from the old written orders of the past except that it takes longer. And with standardized resuscitation drug concentrations the FDA is actually contributing to the number of deaths in kids less than a year old. These are being actively reported for the past few years and to date the only response has been to send out a letter with instructions on how to properly set up an infusion of vasoactive drugs. I found this response insulting at best.

    Pediatrics is a glaring example of an area where the FDA is failing and I find changes, like this one, that do not address this deficiency rather frustrating. Sorry for the length. The FDA is a sore spot for me.

  5. Kabboron 03 Mar 2017 at 9:50 am

    Once these changes are in effect, I’ll be able to get my ‘safe’ Heart Drugs (cinnamon hearts) approved by the FDA, and they’ll sell for a fraction of the existing price for heart medications! People would be insane not to enjoy the cost savings, let alone that spicy cinnamon flavour.

    Safety first!

  6. Steven Novellaon 03 Mar 2017 at 10:53 am

    tb – you are correct in that pediatric dosing and approval is sorely lacking. I here this complaint from my pediatric neurology colleagues also.

    But, to clarify for our readers, when you say “unapproved” you mean in the way you are using it (so “off-label” is really a better term). This would affect you, though. Imagine if drugs on the market did not require evidence for efficacy, or had significantly watered down requirements. That would adversely affect the pool of off-label drugs you would have to choose from.

  7. tb29607on 03 Mar 2017 at 12:26 pm

    SN,
    I agree with your point about the FDA controlling what is available. That is fair.

  8. Mexicanon 03 Mar 2017 at 12:42 pm

    Another key point to remember is that, there are developing countries markets such as mine (México) that depend on the approval of the FDA for Medical Devices or Pharmaceuticals. What I mean is that whenever a company gets its product approved in Canada, US or Europe (agencies with a lot of monetary resources and enforced regulations), they can apply for registration in other countries with less monetary power and this approval or registration process can be “fast tracked”, approving drugs or medical devices within days since these have met regulations elsewhere.

    So this de-regulation, while it will have consequences in the US, might have consequences around the globe.

  9. Atlantean Idolon 03 Mar 2017 at 12:57 pm

    I’m not sure whether the FDA is optimal in terms of counteracting bias against Type I errors, but being less inefficient than Canadian and European agencies is hardly compelling evidence of an ideal regulatory regime (if there is such a thing).

    From what I can tell, current state “Right to Try” laws don’t in practice expand access to experimental drugs beyond the FDA’s Compassionate Use policy – Right to Try is voluntary and most pharmaceutical companies choose not to participate under threat of litigation:

    https://www.druganddevicelawblog.com/2016/06/developments-in-compassionate-use-and-right-to-try-laws.html

  10. Xplodyncowon 03 Mar 2017 at 2:26 pm

    One of the lengthiest parts of the approval process is not the actual FDA review but allowing sufficient time to elapse in order to determine a drug’s efficacy-safety profile.

    Some drugs treat conditions that can take years to develop (heart disease, diabetes, etc), and I’ve yet to hear of a medication that can completely reverse a disease process in just one dose. Drugs for terminal illnesses like certain cancers require tracking patients to see how long they live after beginning treatment—which is hopefully years. Plus, side effects may take months or even years before they become apparent.

    If this administration can figure out a way to speed up reality, then that would certainly streamline the approval process.

  11. BillyJoe7on 03 Mar 2017 at 3:34 pm

    Xplodyncow: “I’ve yet to hear of a medication that can completely reverse a disease process in just one dose”

    Azithromycin 1 gm single dose for chlamydia infection.

  12. BillyJoe7on 03 Mar 2017 at 3:37 pm

    Xplodyncow: “I’ve yet to hear of a medication that can completely reverse a disease process in just one dose”

    Diflucan one capsule for vaginal thrush.

  13. BillyJoe7on 03 Mar 2017 at 3:44 pm

    Xplodyncow: “I’ve yet to hear of a medication that can completely reverse a disease process in just one dose”

    Zithromycin 1 gm for gonorrhoea infections

  14. bendon 03 Mar 2017 at 7:19 pm

    I agree with Atlantean Idol in that most present pharma companies would be reluctant to participate in “right to try” activity. My concern would be the proliferation of predatory companies that create worthless drugs based on flimsy theory, get them past phase I, then market them to the desperate as miracle cures. It seems like streamlining a business model for other Burzynskis. We don’t need more quacks to strip the terminally ill of all their remaining time and energy, to say nothing of their bank accounts. Hasn’t life been unfair enough?

  15. Steven Novellaon 04 Mar 2017 at 8:57 am

    Bend – exactly. that is what right to try laws are about too – fast tracking quackery.

  16. RJHon 04 Mar 2017 at 12:55 pm

    Deregulation of the drug approval process would be a great win for patients and prescribers. Some of the fears listed in this article seem to replace deregulation with the idea of “no regulation”. Milton Friedman proposed the idea of limiting the FDA to safety only back in the 50s or 60s so this is not a new idea. Also, it’s inaccurate to compare this idea to the current supplement or homeopathic industries because they have almost no regulation. This proposal would keep new drugs as prescription products so patients would still have the safety net of having a doctor recommend the drug. Doctors don’t need complete efficacy info to make this judgment call. Why would a doctor prescribe a new drug of unknown efficacy unless all other proven remedies have failed a patient?

    It’s also quite easy to keep an incentive for proving efficacy. We could shorten the length of patents when companies only seek approval for safety. Less risk, less reward. But it’s not even necessary because doctors and patients will realize which drugs work and which don’t after small local trials.

    We can compare ourselves to Canada and Europe but that doesn’t give us much insight into how we can improve our choices when we only look at countries who are essentially using the same political systems we use. Our parents said it best when they warned us, “if all your friends jumped off a bridge, would you do that too?”.

    Finally, your logic regarding the pharm companies desire for regulations is completely wrong. Every economist will tell us that large companies benefit most from regulation because it keeps out competition. Of course they don’t use those words, they always claim regulation is good because of safety (think of the children!!). Loosening regulation would allow more companies into pharmaceutical market since costs to bring a drug to market would be lower. More choices is better since drug therapy is largely a game of trial and error.

  17. superdaveon 07 Mar 2017 at 11:17 am

    Trump’s comments seem to indicate he has absolutely no concept of side effects. If drugs either worked or did nothing this plan might be OK. Coming to a decision about wither or not a drug is worth a potential risk requires the most information possible to make the best decision possible. This system literally ensures doctors or patients would be harmed by side effects.

Trackback URI | Comments RSS

Leave a Reply

You must be logged in to post a comment.