Will Trump Appoint a Great FDA Commissioner?

by on January 23, 2017 at 7:14 am in Current Affairs, Economics, Law, Medicine | Permalink

As someone who has written about FDA reform for many years it’s gratifying that all of the people whose names have been floated for FDA Commissioner would be excellent, including Balaji Srinivasan, Jim O’Neill, Joseph Gulfo, and Scott Gottlieb. Each of these candidates understands two important facts about the FDA. First, that there is fundamental tradeoff–longer and larger clinical trials mean that the drugs that are approved are safer but at the price of increased drug lag and drug loss. Unsafe drugs create concrete deaths and palpable fear but drug lag and drug loss fill invisible graveyards. We need an FDA commissioner who sees the invisible graveyard.

Each of the leading candidates also understands that we are entering a new world of personalized medicine that will require changes in how the FDA approves medical devices and drugs. Today almost everyone carries in their pocket the processing power of a 1990s supercomputer. Smartphones equipped with sensors can monitor blood pressure, perform ECGs and even analyze DNA. Other devices being developed or available include contact lens that can track glucose levels and eye pressure, devices for monitoring and analyzing gait in real time and head bands that monitor and even adjust your brain waves.

The FDA has an inconsistent even schizophrenic attitude towards these new devices—some have been approved and yet at the same time the FDA has banned 23andMe and other direct-to-consumer genetic testing companies from offering some DNA tests because of “the risk that a test result may be used by a patient to self-manage”. To be sure, the FDA and other agencies have a role in ensuring that a device or test does what it says it does (the Theranos debacle shows the utility of that oversight). But the FDA should not be limiting the information that patients may discover about their own bodies or the advice that may be given based on that information. Interference of this kind violates the first amendment and the long-standing doctrine that the FDA does not control the practice of medicine.

Srinivisan is a computer scientist and electrical engineer who has also published in the New England Journal of Medicine, Nature Biotechnology, and Nature Reviews Genetics. He’s a co-founder of Counsyl, a genetic testing firm that now tests ~4% of all US births, so he understands the importance of the new world of personalized medicine.

The world of personalized medicine also impacts how new drugs and devices should be evaluated. The more we look at people and diseases the more we learn that both are radically heterogeneous. In the past, patients have been classified and drugs prescribed according to a handful of phenomenological characteristics such as age and gender and occasionally race or ethnic background. Today, however, genetic testing and on-the-fly examination of RNA transcripts, proteins, antibodies and metabolites can provide a more precise guide to the effect of pharmaceuticals in a particular person at a particular time.

Greater targeting is beneficial but as Peter Huber has emphasized it means that drug development becomes much less a question of does this drug work for the average patient and much more about, can we identify in this large group of people the subset who will benefit from the drug? If we stick to standard methods that means even larger and more expensive clinical trials and more drug lag and drug delay. Instead, personalized medicine suggests that we allow for more liberal approval decisions and improve our techniques for monitoring individual patients so that physicians can adjust prescribing in response to the body’s reaction. Give physicians a larger armory and let them decide which weapon is best for the task.

I also agree with Joseph Gulfo (writing with Briggeman and Roberts) that in an effort to be scientific the FDA has sometimes fallen victim to the fatal conceit. In particular, the ultimate goal of medical knowledge is increased life expectancy (and reducing morbidity) but that doesn’t mean that every drug should be evaluated on this basis. If a drug or device is safe and it shows activity against the disease as measured by symptoms, surrogate endpoints, biomarkers and so forth then it ought to be approved. It often happens, for example, that no single drug is a silver bullet but that combination therapies work well. But you don’t really discover combination therapies in FDA approved clinical trials–this requires the discovery process of medical practice. This is why Vincent DeVita, former director of the National Cancer Institute, writes in his excellent book, The Death of Cancer:

When you combine multidrug resistance and the Norton-Simon effect , the deck is stacked against any new drug. If the crude end point we look for is survival, it is not surprising that many new drugs seem ineffective. We need new ways to test new drugs in cancer patients, ways that allow testing at earlier stages of disease….

DeVita is correct. One of the reasons we see lots of trials for end-stage cancer, for example, is that you don’t have to wait long to count the dead. But no drug has ever been approved to prevent lung cancer (and only six have ever been approved to prevent any cancer) because the costs of running a clinical trial for long enough to count the dead are just too high to justify the expense. Preventing cancer would be better than trying to deal with it when it’s ravaging a body but we won’t get prevention trials without changing our standards of evaluation.

Jim O’Neill, managing director at Mithril Capital Management and a former HHS official, is an interesting candidate precisely because he also has an interest in regenerative medicine. With a greater understanding of how the body works we should be able to improve health and avoid disease rather than just treating disease but this will require new ways of thinking about drugs and evaluating them. A new and non-traditional head of the FDA could be just the thing to bring about the necessary change in mindset.

In addition, to these big ticket items there’s also a lot of simple changes that could be made at the FDA. Scott Alexander at Slate Star Codex has a superb post discussing reciprocity with Europe and Canada so we can get (at the very least) decent sunscreen and medicine for traveler’s diarrhea. Also, allowing any major pharmaceutical firm to produce any generic drug without going through a expensive approval process would be a relatively simply change that would shut down people like Martin Shkreli who exploit the regulatory morass for private gain.

The head of the FDA has tremendous power, literally the power of life and death. It’s exciting that we may get a new head of the FDA who understands both the peril and the promise of the position.

1 Alan January 23, 2017 at 7:28 am

All well and good …… but, what political tripwire did these candidates hit? Seems to matter more these days.

2 mobile January 23, 2017 at 12:04 pm

All friends of Peter Thiel.

3 So Much For Subtlety January 23, 2017 at 7:29 am

Have I hit the trifecta – First, beaten Ray and the spambot?

Preventing cancer would be better than trying to deal with it when it’s ravaging a body but we won’t get prevention trials without changing our standards of evaluation.

There are endless people claiming to be able to prevent cancer. It is hard to see what is the point of any study that has to wait forty years. A lot of this looks like trying to rationalize away medicine’s failure to innovate. Instead of wanting to cure cancer, they now want to spend a fortune to tailor a solution to one, presumably extremely rich, man. The narrowing of ambition is sad.

4 So Much For Subtlety January 23, 2017 at 7:30 am

Damn! Only one of the three.

5 marris January 23, 2017 at 9:51 am

This criticism is totally off the mark. Most medical care is pattern matching. Measure these symptoms, apply this rule to cure.

Personalized medicine can be a terrific public good if doctors are incentivized to discover and publish more patterns. What are the odds that you’re the only person in the world with a particular type of cancer?

6 Mark Abrams January 23, 2017 at 12:17 pm

personalized medicine can also mean using your own cells to regenrate or replace damage, or to fight cancers or infections . Because the immune system attacks non-self cells any such biologic must be custom made from the patients own cells . Thus the pattern is quite clear but the FDA trials requirement is completely unsuited for such treatments and so they are unavailable .

7 Alan January 23, 2017 at 7:30 am

Must burn you guys up when the first comment gets in ahead of you by chance.

8 rayward January 23, 2017 at 7:53 am

Medicine is highly vulnerable to snake-oil salesmen because sick people are often desperate people. If the FDA collaborates with snake-oil salesmen, it will risk more than the lives of patients, it will risk the FDA’s credibility; and that credibility, once lost, will be difficult to regain. I appreciate Tabarrok’s desire to facilitate advancements in drugs. I’ve lost many family members to cancer, and shared their desperation for a cure to a terminal illness; indeed, I’ve shared their disappointment when when they were rejected for a drug trial, believing as they must that a cure is there if only they could be chosen for the trial. It’s heartbreaking. But the FDA’s mandate is based on efficacy: only drugs that are proven efficacious are allowed to be sold. That’s different from the SEC’s mandate, which is based on disclosure: as long as the issuing company discloses the risks, it’s up to the investor to evaluate the risk. Sure, sophisticated investors might be able to evaluate the risk of an investment, but people can’t evaluate the risk of a drug and must rely on the FDA. Do we really want to turn the FDA into the SEC?

9 kevin January 23, 2017 at 10:03 am

What is the purpose of doctor’s then? An individual may be suckered into buying “snake-oil”, but doctors shouldn’t be. If they are, maybe we need a better way of evaluating them. The FDA should be responsible for making sure you aren’t poisoned–not for your financial well-being

10 Thiago Ribeiro January 23, 2017 at 10:46 am

“The FDA should be responsible for making sure you aren’t poisoned–not for your financial well-being.”

Why? There are doctors. If they are not preventing you from being poisoned, what are they doing?

11 kevin January 23, 2017 at 11:22 am

I guess I could agree with prescriptions (and that’s partly why doctor’s have such high insurance premiums), but the vast majority of things FDA regulates are over the counter. which doctors have nothing to do with.

12 kevin January 23, 2017 at 11:37 am

Edit: I also think you’d need a better screening/certification for doctors then. Its one thing if a doctor recommends something that doesn’t work, its another if the recomend something that kills you. But to your point, if you somehow managed to put in this better screening/certification, the FDA would be redundant.

13 mavery January 23, 2017 at 4:44 pm

There are plenty of poisonous things that do wonders for your well-being, such as chemotherapy drugs. To evaluate these things, the FDA has to look at efficacy as well as toxicity.

Doctors are fallible just like regular consumers except doctors go in with a strong prior that their medical-related judgements are right. This despite the fact that they don’t work in a very empirical field. In fact, the FDA is the only reason that our current understanding of drugs and devices is as firmly rooted in empirical data as it is. (Note: That’s not meant to imply these areas are heavily empirical; they’re not. They only appear so in relation to the rest of medicine.)

None of this is to dismiss the obvious problems costs associated with having a large, bureaucratic body in charge of regulating this market. I guess to me it just comes down to the fact that whole medical realm is fraught with conditions associated with market failure (moral hazard, asymmetric information, monopolies, decisions made in emotionally charged environments). Given that, any step away from “gold standard” clinical trials is a step towards “preventative” stents and radical mastectomies.

14 JWatts January 23, 2017 at 10:43 am

I don’t see any genuine objections there. Just a vague prediction that one of these people would be a snake oil salesman. A lot of words to say very little.

15 Mark Abrams January 23, 2017 at 12:23 pm

Free men choose their own medical care (usually with expert advice). Slaves and animals get whatever treatment their owners disctate .

The FDA should offer advice only. government nutritional advice has been pitifully bad and I suspect the FDA’s is worse . Their positions on stem cells, biologics and antibiotic trials is truly terrible.

16 Millian January 23, 2017 at 12:58 pm

Gold heals chemtrails wounds caused by jet fuel, paper money causes them to worsen.

17 mavery January 23, 2017 at 4:54 pm

They’re horrible. Only marginally better than the state we’d find ourselves if anyone could claim anything about anything. That gives you Dr. Spaceman prescribing Chatterton’s cigarettes for women “in the family way.”

18 Mark Abrams January 25, 2017 at 10:57 am

anyone can claim anything about anything , its due to that out-dated first amendment . But dont worry in your totalitarian utopia not only will the state determine your medical care it will control all speech as well .

19 Alex January 23, 2017 at 10:10 pm

“people can’t evaluate the risk of a drug and must rely on the FDA“

If people are so stupid then they shouldn’t vote either. I don’t need to rely on the FDA. FDA approval should be voluntary.

20 Axa January 23, 2017 at 8:10 am

Let’s hope the new FDA chief back the ban on antibiotics for cattle feed http://europe.newsweek.com/after-years-debate-fda-curtails-antibiotic-use-livestock-542428?rm=eu

21 Axa January 23, 2017 at 9:13 am

Curious fact: the evil FDA that keeps the population in the dark has not shutdown Mr. Srinivasan’s genetic testing startup (Counsyl) http://www.forbes.com/sites/brucerogers/2016/06/15/counsyl-reinvents-the-clinical-laboratory-to-provide-better-genetic-testing-services/#2e8c4ca48c2a

The difference is that Counsyl offers the test results to be explained by a doctor. In dollar terms the difference is $349 (Counsyl) to $99(23andme). So, both companies do more or less the same. One is thriving, the other declared war at bureaucracy.

From the consumer’s side: is there a large difference between $349 and $99 for a novelty service? From the investor’s side: where put the money? In the business that grows or in the business that got political?

22 Jack January 23, 2017 at 9:24 am

1) Not offering to have a doctor explain your product is “declaring war on the bureaucracy”? Even for someone who is generally OK with declaring war on bureaucracy, that seems a bit hyperbolic

2) Yes, there is a big difference between $349 and $99. To get an idea, picture someone who earns $350/week and calculate how much they would have left of there weekly salary after the purchase.

23 Alain January 23, 2017 at 10:47 am

Counsyl is a testing service. Their screens are very selective. They are only like 23andme in that they work in genomics.

What Obama did to 23andme was a true demonstration of his character. They had the audacity not to knell before the throne and in retribution he smashed the company to little bits.

Disgusting.

24 mulp January 23, 2017 at 3:08 pm

What did “Obama” do to 23andme?

Stop them from telling people they have diseases they don’t have? Why would you want to be told you have a disease you don’t actually have?

If 23andme was presenting useful data, then the former UK spy compiled document for hire by a Republican opponent of Trump from data obtained from sources in Moscow handed by McCain to the CIA must be considered truth that the public needs to know.

After all, things said by Russians unnamed must be considered absolutely useful data without any context, just like tentative linkages between genetic markers and medical conditions are useful without any context.

Nothing prevents you from obtaining the same linkage info from an open source database or two on the Web. The difference is you must read the context in order to draw your conclusion on the meaning of your DNA marker. It’s ironic that Alex argues oversight by experts charged by Congress to be gatekeepers is wrong because experts without any liability or oversight should be free to diagnose you without seeing you as a patient because they are experts you can validate because every individual is expert at validating experts.

Earlier I heard a discussion about anti-vaxers who have gotten broad permission to diminish herd immunity based on individuals being more expert than experts in viral pandemics. And before that, a BBC discussion and “debate” with a guy who claims zero children were killed at Sandy Hook because it was just an Obama hoax perpetrated for $25 million in profits to the fake parents and for Obama to disarm Patriots so he could stage a coup and become president for life.

25 mulp January 23, 2017 at 3:15 pm

For $199, 23andme:

“The first and only genetic service available directly to you that includes reports that meet FDA standards for clinical and scientific validity.

Carrier Status reports*
35+ reports

Ancestry reports
3 reports

Wellness reports
5+ reports

Traits reports
19+ reports

Be part of something bigger.

You can make a difference by participating in a new kind of research—online, from anywhere.

As a customer, you can answer online survey questions, which researchers can link to your genetic data to study topics from ancestry to traits to disease. Your contribution helps drive scientific discoveries.

You can always choose to opt into or out of research.”

In other words, for $100 extra, you can be a lab rat!

26 Lord January 23, 2017 at 8:40 am

An argument for regulatory capture. I would be more persuaded if the profit motive were taken out of early approvals, such as free provision in exchange for more complete data collection.

27 AlanG January 23, 2017 at 9:26 am

it already exists in the Treatment IND regulations. Companies can charge for investigational drugs to recoup full costs of the clinical trial.

28 Turkey Vulture January 23, 2017 at 8:52 am

23andme does offer its health-related analyses at this point, after coming to some kind of understanding with the FDA. Maybe there was a broader package that was once offered, but right now there are a bunch of diseases (mostly very rare) that it will provide carrier-status information on.

29 Alain January 23, 2017 at 10:48 am

For all intents and purposes 23andme no longer exists.

30 mulp January 23, 2017 at 11:52 am

For something that does not exist, 23andme does a lot of gene testing at $99 and $199 a pop.

If you are arguing they are not delivering lots of fake news for profit, why do you think fake news is a good thing?

31 Scoop January 23, 2017 at 8:59 am

Does anyone know enough about the FDA to know whether the commissioner could institute reciprocal approval on his own or whether it would require new legislation?

32 AlanG January 23, 2017 at 9:35 am

It’s really a false issue these days. FDA and the European Medicines Agency collaborate closely. The pharma industry and regulatory agencies throughout the world meet under the auspices of the International Conference on Harmonization (ICH: http://www.ich.org/home.html) whose goal is to harmonize regulatory submissions and standard throughout all the member agencies. Over the recent past the majority of new drugs receive first approvals in the United States. Important new drugs get designated for priority reviews at the FDA with a six month review clock.

The issues of reciprocal approval are complicated. Would the drug given approval based on a EMA decision have to be in the review queue at the FDA? Would FDA have special authorities to revoke the approval? What happens if FDA makes a decision that the drug is not approvable? Would insurers reimburse for drugs granted such an approval? It’s not as easy an issue as one would think.

33 Mark Abrams January 23, 2017 at 12:30 pm

It is not a false issue at all . The FDA does not accept approvals from the EU, Japan or Canade . Drugs approved in those countries need to be go through the FDA approval process , which does not accept foreign trials data, before they can be prescribed or sold in the US . That process can cost billions and take years .

34 mulp January 23, 2017 at 2:44 pm

The FDA does accept foriegn trial data. The trials just need to meet standards for honesty in all dimensions.

“Criterium India: Poised to Excel in Clinical Trials:

Criterium India has created a database of investigators who excel in their respective Indications/Therapeutic areas. This represents the best and most experienced team of medical professionals throughout India.

These investigators are well versed in the laws and regulations affecting clinical trials in India. The study teams include:

Principal Investigators who have proven experience with Clinical Trials, and are specialists in their field;
Medical and non-medical staff to ensure smooth running trials; and
Social workers that are appointed on trials as needed.
Clinical trials in India have also occasionally undergone regulatory or sponsor audits. When this occurs, the Indian Investigators have proven to be superior in meeting International and Regulatory standards. Indian clinical trial data have been successfully transferred to the US for several FDA approvals.”
http://www.criteriuminc.com/clinical_research_india.php

Unless Trump has already issued an executive order prohibiting outsourcing of human lab rat jobs to India, et al.

“A notice was posted on the FDA web site one day after the agency sent a letter to Semler Research Center, which is based in Bangalore, India, saying that inspections found “significant instances of misconduct and violations of federal regulations, including the substitution and manipulation of study subject samples.” The FDA inspected Semler facilities last fall.

The move comes two weeks after the World Health Organization issued a “Notice of Concern” to Semler for the same reasons. Specifically, the WHO examined company computer servers and found a spreadsheet file containing detailed instructions for manipulating drug samples that were used in clinical trials for its clients. The WHO inspections conducted inspections early and late last year.”
https://www.statnews.com/pharmalot/2016/04/25/fda-warn-clinical-trials-run-indian-company/

So, why would for profit corporations fake data to prove drugs were effective and safe???

Isn’t greed good as long as it’s for private profit off private individuals in the billions of dollars, but not for scientists looking for money from the government on the order of a hundred thousands?

35 Mark Abrams January 25, 2017 at 11:05 am

trials data are not approvals, lots of FDA trials use foreign sites (and so accept their honesty) however trials design is a BIG deal and generally the FDA wants to design the trials they use for dRug approval. For years there have been alternatives to FDA trial design , in particular regarding Bayesian probabilities. It should be noted that the FDA is hardly immune from political pressure in altering trials endpoints, one need only look at the complete abandonment of trials endpoints when the FDA approved AIDS drugs .

36 towson January 23, 2017 at 9:04 am

” To be sure, the FDA and other agencies have a role in ensuring that a device or test does what it says it does …”

well, to be sure, there must be some Constitutional clause granting FDA police authority over such things (?)
Many people somehow perceive such an original legal authority, but are completely unable to state any specific limits to that supposed authority. What are the Constitutional limits of FDA authority… and how did you determine those limits?

37 Mark Abrams January 23, 2017 at 12:35 pm

The FDA, indeed the federal government has no right to interfere in anyone’s medical treatment . It’s sole power derives from the ridiculously broad interpretation of the commerce clause . This is the basis of the FDA’s power. You can use any medicine or device made in your own state , the FDA only regulates drugs and medical devices which are part of inter-state commerce.

38 Just Another Right Wing Economist January 23, 2017 at 9:07 am

” In particular, the ultimate goal of medical knowledge is increased life expectancy (and reducing morbidity) but that doesn’t mean that every drug should be evaluated on this basis. If a drug or device is safe and it shows activity against the disease as measured by symptoms, surrogate endpoints, biomarkers and so forth then it ought to be approved. ” Hmmm, i don’t think this is a good idea, sounds like opening the door, to even more profit-driven medicine and overtreatment (and as if there wasnt enough already…).

39 jdm January 23, 2017 at 12:07 pm

Exactly. While I am sympathetic to Alex’s position, “If a drug or device is safe and it shows activity against the disease as measured by symptoms, surrogate endpoints, biomarkers and so forth then it ought to be approved”, this would only produce good outcomes if patients had to pay for their own treatments.

If – as is likely – insurance (i.e. everyone else) is picking up the tab, sick people will demand all kinds of useless but expensive treatments. Medical costs will go up even more rapidly but people won’t be healthier.

40 ttt January 23, 2017 at 1:38 pm

so Death Panels then ?

41 jdm January 23, 2017 at 9:11 pm

It depends. If a patient wants to pay for some unproven treatment out of their own pocket, let them. If, as is more likely, the patient expects everyone else to pay for their treatment, it is reasonable for everyone else to demand that the treatment be efficacious and cost effective.

It is a bit disappointing for to see Alex cheerleading for greater freedom choice when it comes to treatment (which I also favor) while at the same time ignoring the obvious problems with this approach when medical costs are not primarily borne by the patient.

42 Mark Abrams January 23, 2017 at 12:44 pm

Maybe if you get a chronic condition , or long term pain you might be a bit more sympathetic to drugs, devices and treatments to alleviate those conditions even though they have no provable effect upon your morbidity . Also the reflexive hatred of rewarding those who make goods or services which benefit you is a facet of the socialist mindset which tends to destroy civilizations, causes misery and ends in mass murder (try reading the black book of communism some day). Capital is just tools and knowledge, and that is how mankind progresses . The labor theory of value is idiotic (a gusher and a dry hole require the same labor) and consequently so is marxism.

43 dearieme January 23, 2017 at 9:18 am

What about the vital information? Which of the candidates is a woman, or a black, or a BLTPDQNBG?

44 The Other Jim January 23, 2017 at 10:12 am

Four days ago, that indeed would have been the primary criteria.

Not so much any more.

45 JWatts January 23, 2017 at 10:49 am

Well the first name on the list is ” Balaji Srinivasan”. Obviously not an old white guy. But to your point, it’s doubtful that there is much of an affirmative action hiring stance in the Trump Administration.

46 dearieme January 23, 2017 at 10:59 am

Uninterested in matters of race? Just proves he’s a Nazi, eh?

And I noticed that when he did appoint a black man to his cabinet, the black man chosen gives an impression of being intelligent, calm, and honest. What sort of example is that for vibrant youth?

47 Mark Abrams January 23, 2017 at 12:49 pm

Indeed, we must insist that people who chop up their genitals and insist that mammals reproduce by fission are placed into positions of power and responsibility otherwise we may be forced to live in relative comfort and affluence rather than roasting rats around a fire of burning dung (the way Gia wants us to live ).

48 AlanG January 23, 2017 at 9:24 am

Where to start criticizing this disjointed post? Of course I expected it from Professor Tabbarok given his past writings. Unfortunately, he appears to be falling into the trap of “alternate facts” that is becoming all the rage these days. Are we really entering into a new world of personalized medicine? Of course not, we have been in that world for lots of years now. It’s the primary reason that some patients do well on a particular drug and others do not from both a safety and efficacy point of view. We have not just discovered genetic disparities in the last 5-10 years. Most of the drug lag that is regularly complained about is the result of poor clinical trial design or inept regulatory filings. Prior to retirement, I spent just over 30 years working on regulatory affairs and drug safety within the biopharma industry and have been part of countless discussions with FDA over this time period. FDA wants to approve safe and efficacious drugs and does so. The companies that I worked with that had good trial design and regulatory packages all got their drugs approved in a timely manner (usually before the first approval in Europe). Now if we want to change this standard let’s have that debate. FDA recently approved a drug for Duchenne’s Muscular Distrophy that may not be either safe or effective (the trial size was just too small to show either). Now this drug enters the market at a huge cost to families who pressed for it ($300K per year). If anything we need a new paradigm for how to handle these drugs. I don’t know why the Treatment IND regulations are not used in these instances. Companies will have the opportunity of recovering clinical trial costs while the necessary studies are completed and it will relieve the health care system of having to pay for “snake oil” cures. Of course we can also move to a broad “informed consent” regime and let anyone purchase such products and others understanding that they are not been shown to be safe or effective. Also remember, no drug is ever proven to be entirely safe (just ask your doctor the next time a drug has been prescribed whether he/she has read the drug label; better yet, read it yourself at: https://dailymed.nlm.nih.gov/dailymed/index.cfm (maybe ask the doc whether he/she knows of that resource)).

Yes, FDA does have a role in the oversight of new test methodolgies. 23andMe did get approval for some of their testing once they understood what was required. IIRC, lots of people were apoplectic about the way Theranos was treated early on. The FDA role in this case was appropriate and it was good of Alex to acknowledge that.

Before we get too carried away with personalized medicine, one needs to acknowledge the risk of small numbers. Ray Lopez commented the other day about a new NIH treatment for cancer. this was the subject of a New England Journal of Medicine article in December where 12 patients were treated and ONE had a response. Unfortunately, after seven months one of the lesions had developed a genetic resistance to the treatment.

I’ll not address the regenerative medicine issue other than to note that our bodies eventually break down (as most of us in our 60s and 70s find out). Experts in the oncology area are better prepared to address cancer preventative drugs/supplements.

Regarding surrogate endpoints, FDA approves drugs based on them and has for a long time (blood pressure regulation a classic example here). I and others spent a considerable amount of time establishing a consortium that studies biomarkers and their roll in drug development and approval (http://www.fnih.org/what-we-do/biomarkers-consortium) Similarly we worked long and hard to better understand how observational medical data can be used to better address drug safety and efficacy in the post-approval environment (http://omop.org/) and this effort has been transitioned on to the Reagan/Udall Foundation for the FDA. there are a lot of other efforts that are going on as well that collectively are improving the science behind drug development. There is no free lunch here.

Regarding the comment about FDA’s not approving generic drugs quickly that has resulted in several high profile cases of price gouging, it’s not the FDA that is at fault here. All of the drugs in the Shkreli and Valeant examples are small market items produced by a single manufacturer. Because of this, prices could be raised with impunity, a true example of market forces at work. At some point the price level becomes appealing for a 2nd manufacturer to enter the market. If you don’t want price controls, things like this will happen.

49 anon January 23, 2017 at 11:20 am

+1

50 byomtov January 23, 2017 at 1:02 pm

Nice to have someone speak up who has actual long-term experience with the issue, and is not just making things up.

Thanks.

51 Mark Abrams January 23, 2017 at 1:00 pm

The FDA does NOT currently have any economically possible way of approving treatments which are made from a patients own cells . The FDA has a 1930s mass production mindset that one drug will treat all people, however with biologics that is obviously false . One cell line will be rejected by everyone except the patient from who the cells were derived . Thus the effort to cure diabetes by injecting appropriate stem cells into the pancreas is upon how to protect a single line of stem cells from the patients own immune system . if the FDA were favorably inclined towards personalized medicine the effort would be upon standardizing the production PROCESS so that the patients own stem cells could be used . But the FDA is not favorably inclined towards personalized medicine . They want a single product or cell line to treat everyone and because of immune rejection that cannot possibly work for biologics.

52 mulp January 23, 2017 at 2:04 pm

FDA executes the laws under the Constitution, those passed by Congress.

Perhaps you should run for Congress and get a law passed that says anyone can contract with their own money with anyone to develop anything they inject in a person’s body with no legal liability for any outcome as long as the patient paid for it in advance with their money, not with borrowed money.

That would be unregulated free market drug development, testing, and treatment.

I think it likely people will be exploited, but they will likely be “rich” people.

53 AlanG January 23, 2017 at 3:44 pm

@Mark Abrams – Have you even read the FDA guidance documents on cell and tissue therapy? All of this is discussed and dealt with. Spend some time doing research.

54 Mark Abrams January 25, 2017 at 11:37 am

I am currently using transgenically derived human antibodies under an experimental single patient IND so yes I am familiar with FDA guidelines, you smug elitist totalitarian. It has taken 2+ years to get that IND (2+ years I spent crippled and in pain)
as well as collaboration with a major teaching hospital to get this far and only in conjunction with a research program that has been ongoing for 10 years. And before that it took a year to get an IND to use an experimental antibiotic available in the EU. I repeat that the FDA currently requires any commercially available biologic to undergo clinical trials , consequently only a single cell line can be used and because of immune non-self rejection many such treatments will fail or cause graft vs host disease, similarly stem cell based tissue replacement cannot proceed under such a regimen either , The FDA does not even allow in vitro expansion of your own cells (see Regenerex) . The entire biologs program currently in place is seriously flawed and outdated . Explain please why bone marrow transplants are just fine but expanding ones own cells and reinfusing them is not . Fortunately the FDA’s position is so ridiculous that it will have to be abandoned , CAR-T cell therapies are an example of a working therapy that cannot be mass produced and so is not possible to test with current clinical trial designs. Just because you benefitted financially from the existence of the FDA doesn’t mean that many others dont suffer because of it . Does that make you unethical ? evil ? corrupt ? or just a dutiful statist drone ?

55 Bill Kilgore January 23, 2017 at 2:05 pm

— Because of this, prices could be raised with impunity, a true example of market forces at work. —

Lots of dubious commentary in that post, but this comment is particularly bizarre. Market forces would have other manufacturers jumping in to manufacturer the products once the price starting rising. And, of course, the original manufacturer would know this and would need to price accordingly.

The market cannot function this way because the FDA will not allow other parties to enter the market in a reasonable time-frame and actually allow market forces to work to keep the price in line.

In short, as your comment makes clear, the current system allows the FDA to pick the entities who can participate in a given market and essentially preclude those who do not have massive piles of resources and/or political connections from participating- even when they have a superior product. The FDA is essentially a crony based system that protects its friends (massive corporations) – and very little besides.

Glad to know it worked out well for you though. Must be exciting to know one has earned his income by preventing reasonable medicines from hitting the market.

56 Mark Abrams January 25, 2017 at 11:46 am

All quite true. Epipens are an excellent example . the FDA keeps rejecting competitors products and not because the epinephrine is any way inferior or dangerous , competitor products are in use elsewhere but not here in the USA . The FDA is a monopoly protection racket because monopoly profits are necessary to jump through FDA mandated hoops , a symbiotic relationship that both impoverishes and kills the sick .

57 Bill January 23, 2017 at 3:53 pm

Alan,

+1 Thank you for your substantive and fact based comment.

Evidently you are not an economist.

58 Thanatos Savehn January 23, 2017 at 9:36 am

The FDA filled the vast unseen graveyards in one especially efficient way. It defined marketable innovation as shifting the population survival mean slightly to the right. Doing so collectivized the sick, de-personalized medicine and corrupted biomedical science by requiring drug companies to engage in the “null hypothesis racket.” And it is responsible for last week’s news about the disappointing results of efforts to reproduce 5 of the most exciting recent peer-reviewed cancer discoveries published in prestigious journals.

Incentives matter but with cancer research and treatment many of the current incentives aren’t aligned with either individual patient outcomes or even scientific rigor. The fact that so few researchers pause to validate reagents and cell lines is scandalous. The fact that so many patients are being misdiagnosed and thus mis-treated because “oops, somebody long ago got the mouse antigens mixed up with the human ones, nobody thought to check it out and so the immunohistochemical staining we did to specify your disease is fubar and we gave you the wrong chemo, sorry”, ought to be criminal.

If the people marching under pink banners in common cause against a common enemy ever realize how many of their sisters have been sacrificed for the sake of a lie there’ll be hell to pay.

59 Let's be correct January 23, 2017 at 9:42 am

FDA should be headed by a Latina or woman of color, just because.

60 ClickByCommenter January 23, 2017 at 11:54 am

You mean Trump should bring back Margaret Hamburg?

61 collin January 23, 2017 at 9:42 am

The problem with a lot of Trump’s picks is the candidates don’t appear to be experts in the agency they lead. (Think Perry, Carson, Tillerson, Devos. But some do Sessions, Ross and Mnuchin.) So for conservatives they should be careful to ensure the right candidate

1) They need to prove how to fast track some of these medicines.
2) They show the importance of oversight. This is dangerous stuff and recklessly lowering regulation will hurt your cause in the long term.
3) They think about ways to increase competition to decrease patent drug prices. It is ridiculous that the US pays 2 or 3 times as much as other nations.

62 AlanG January 23, 2017 at 10:00 am

Scott Gottlieb spent time at the FDA when Mark McClellan was commissioner. I know him pretty well than believe that he would be a good choice to lead the agency.

63 Mark Abrams January 23, 2017 at 1:07 pm

FDA approves of chloramphenicol which can cause pernicious anemia (kills people) but not thoramphenicol , which has the same efficacy as chloramphenicol but kills no-one . No patent on thoramphenicol is possible so it will never be submitted for approval in the USA . So a useful drug is not available and a couple of people a year die because what is available is dangerous but who cares so long as we can preserve the power of the state and prevent people and their doctors from making their own choices . Hooray for the all powerful state .

64 mulp January 23, 2017 at 1:54 pm

How do you know the safety and efficacy of thoramphenicol without it being possible to gain exclusivity on it?

If it is an old existing drug, it can be produced and sold as a “grandfathered” drug. Further, any manufacturer can file evidence of safety and efficacy and gain exclusivity for a period of time for documenting the drug, and then competing makers must follow the ANDA process to resume producing a generic.

Your claims of its advantages are based on it being used in other nations in uncontrolled use, and if kills people, that is not reported. Even in the US, reporting of adverse reactions to drugs is inconsistent, and that is with a reporting system. Are you relying on the Brazilian government health care system for your assessment? Many drugs in use but grandfathered in the US (pre-1965) were developed with NHS and treatment standardized by the NHS in the UK. Or by the US military. For a government health care system, funding drug development and trials makes a lot of sense because once an effective drug is found, it’s cost is simply manufacturing cost. Developing an injector would be the engineering and testing, and then it’s cost would be manufacturing and the cost of filling it with the drug manufactured at cost.

The money paid in monopoly profits and rents by the government for drugs is probably high enough to fund a lot of drug trials and to do the work to meet ANDA requirements for drugs manufactured and sold at cost under government contract.

Vaccine manufacturing is almost entirely government funded, and the development of new production methods is almost entirely government funded. Congress and the Bush administration started the latter, and in the past 8 years, the Obama administration pushed significant advances in vaccine production.

The idea that monopoly profits is the only way to produce new drugs and production methods and get them on the market is absurd. It makes as much sense as calling for every road and street to your house be a toll road so the private sector can get monopoly profits for building streets to homes.

65 AlanG January 23, 2017 at 3:54 pm

@mulp – “Vaccine manufacturing is almost entirely government funded, and the development of new production methods is almost entirely government funded. Congress and the Bush administration started the latter, and in the past 8 years, the Obama administration pushed significant advances in vaccine production.”

Your statement would certainly come as a big surprise to Merck, Glaxo SmithKline, Novartis, and Sanofi, all of whom fund their own vaccine manufacturing.

66 AlanG January 23, 2017 at 3:52 pm

I did a google search on thoramphenicol and came up with nothing. Please let me know if there is an alternate spelling for the compound in question. Perhaps you are thinking of Thiamphenicol which is approved for human use in a couple of foreign countries. This class of antibiotics has been around for a long time and maybe it was thought that Thiamphenicol is just not as good as a number of other antibiotics with the same spectrum of action. there would be nothing to stop one of the foreign manufacturers from seeking approval in the US.

67 Mark Abrams January 25, 2017 at 12:05 pm

My mistake , my spelling recall is far from perfect . It is thiophenicol also known as dextrosulphenidol . It is available for human use in Italy and China . It has the same uses as chloramphenicol, is more potent and has NEVER been associated with aplastic anemia . Why would anyone seek to approve thioamphenicol and spend a billion dollars for FDA mandated trials on a drug which can not be patented ? If the FDA were interested in saving lives they would accept decades of safe use rather than rigidly require trials . People do die of aplastic anemia and chloramphenicol (or thioamphenicol were it available) is the best treatment for staphylococcal brain abscesses, some forms of bacterial meningitis and various pediatric infections . These are not enough sick people to spend a billion dollars on a trials for a drug which would very soon have generic competitors due to lack of patent protection , or to cause the FDA to potentially damage the monopoly profits of the entities that captured it a long time ago.

68 Turkey Vulture January 23, 2017 at 9:42 am

“Also, allowing any major pharmaceutical firm to produce any generic drug without going through a expensive approval process would be a relatively simply change that would shut down people like Martin Shkreli who exploit the regulatory morass for private gain.”

Even with instant regulatory approval, there is still typically a substantial up-front financial and time investment to produce a new generic drug. This means that any time there is a single producer remaining for an old drug, there will be the possibility of pricing far above cost for an extended period of time because it will take so long for a new competitor to respond and enter the market. Moreover, there is always the risk that the incumbent firm will move right back to pricing at marginal cost once a new firm enters (or, say, is halfway through making investments to enter), making entry unprofitable and thus discouraging attempted entry in the first place.

Of course, reducing the regulatory barrier to entry would reduce total barriers to entry and make entry more likely. I just don’t think you can say that it is entirely (or even necessarily mostly) the “regulatory morass” that is “exploited.”

69 AlanG January 23, 2017 at 10:03 am

There is enough manufacturing capacity available to address the issue you raise and most of that is already FDA-approved. The issue is the small patient population sizes for the drugs in question. We see the same thing in the drug shortage area. The problem is with single sourced products (most injectable sterile products) where there is a manufacturing problem. These take time to address.

70 Turkey Vulture January 23, 2017 at 10:22 am

Do you mean the manufacturing capacity is FDA-approved in a general sense, or that there is spare capacity that has been specifically approved to manufacture specific generic drugs? I have just started to scratch the surface in this area in some of my work, but I had thought a firm ultimately needed FDA approval for the specific drug/product it proposed to produce with given manufacturing capacity.

Even with spare, FDA-approved manufacturing capacity, wouldn’t a generic still need to tailor that capacity to the specific generic it wants to produce, acquire the relevant raw materials, and run enough test batches to be sure it is actually producing an equivalent drug? The latter creates a bit of an issue for the instant approval idea as well, since presumably the FDA would need to require at least some kind of validation that the generic is chemically equivalent to the brand drug. This validation process could be much shorter than the current ANDA process.

If the issue is “small patient population sizes,” that would still seem to be a barriers to entry/expansion problem, but one where minimum efficient scale is such that only one firm can reach (or approach) minimum efficient scale in a market, which leads to a single manufacturer.

71 AlanG January 23, 2017 at 10:55 am

If the chemistry is straight forward, manufacturing is not much of an issue if the facility has already been approved for Good Manufacturing Practices (GMP).. there is excess capacity as I noted. Bioequivalence studies are straight forward to conduct as well which is all that one needs to do. You are still going to have a time lapse between when the prices were jacked up and the competitors come onto the market. this is what Valeant and Shkrbeli were doing with their business model. the difficulty is that a competitor cannot anticipate which drugs will be the subject of price gouging.

72 mulp January 23, 2017 at 1:11 pm

“This validation process could be much shorter than the current ANDA process.”

If Congress required drug makers to reveal full details of every approved drugs chemistry including binding, coatings, buffering, and other active inactive ingredients, generics would be easy to manufacture after developing the required process and QA documents. If the approved drug documents for process and QA were also fully published so a competing manufacture could simply edit the firm, factory, address, contact info to have the required documents for FDA approved manufacturing, that would really cut the barrier to entry.

But Congress has generally gone in the direction of erecting barriers to entry.

73 Stuart Buck January 23, 2017 at 10:22 am

The problem with approving “safe” drugs based on biomarkers is twofold:

1. No drug is totally safe, and many drugs are not safe by any description. (Chemo drugs are notoriously toxic, with side effects that can include heart failure.)

2. There are many examples of biomarkers that aren’t really validated, and even examples of drugs or treatments that moved allegedly well-validated biomarkers (such as cholesterol) in the “right” direction but actually made hard outcomes (such as disease and death) worse. Giving vulnerable people drugs that are costly and have toxic side effects, but either don’t change hard outcomes or even make hard outcomes worse — this is a substantial risk that Alex seems unfamiliar with. Someone with medical expertise would at least try to address this problem.

74 AlanG January 23, 2017 at 11:02 am

Yes, your point #2 is the huge issue here. We are hoping that the Biomarkers Consortium can make progress in this area. The classic example here is Alzheimer’s Disease whose financial and emotional impact on society is well documented. We are no where close to have a good enough understanding of the etiology of the disease and progress towards a good pharmaceutical approach is fraught with complications (look at the Eli Lilly experience who have probably spent more money in R&D here than any other pharma company). Lots of things are being looked at but nothing much has worked. In addition, there is not a good end point for clinical trials other than cognition which is pretty soft to being with. There is a huge effort to look at neural imaging but will that work?

Also look at the history of drugs for mental illness of various sorts. We know that that there are not only issues of efficacy but difficult safety issues as well. Most anti-depressants work in only just over half the cases and there appears to be patient dependent selectivity in terms of efficacy which is not fully understood. In a lot of cases it’s not the FDA that is at fault but the science is just not fully developed.

75 Mark Abrams January 23, 2017 at 1:18 pm

Treating a “disease” which has no somatic basis and can only be determined from behavior is not perhaps a valid field of medicine, much as we wish it were .

76 AlanG January 23, 2017 at 1:48 pm

So we should cease all Alzheimer’s research immediately?

77 Mark Abrams January 25, 2017 at 12:11 pm

Alzhiemer’s has definite somatic indications ranging from plaques to decreased tissue volume. Depression and unhappiness have no somatic indicators at all , indeed they are not often distinguishable .

78 mulp January 23, 2017 at 1:01 pm

The PSA bio marker is a great example of an FDA approved test with lots of data behind its approval, but it turns out far too little data to understand how to interpret and act on the PSA numbers, and subsequent exam and subsequent tests, and worse, subsequent treatments.

On diet, we have the “food pyramid” and other dietary recommendations proposed by experts that get attacked, with thousands of competing dietary recommendations sold for the price of a book or for monthly fees against the free food pyramid recommendations. Not to mention all the dietary advice offered by industry.

For the “free market” dietary recommendation market, the result is rapidly rising obesity.

Likewise, lots of free advice on being active, plus a lot of free market competition on how active and what activity you should do, with the result being the military rejecting volunteers for poor fitness, plus having to devote lots more time to getting recruits fit in basic training.

Where researchers actually try to assess diet and exercise recommendations, they look at bio markers and find the bio markers are influenced by both the recommended regime AND (epi)GENETICS, with specific genes unknown. Ie, controlling for all factors except genetics which are determined by the test subject by definition, the regime may or may not change select bio markers.

79 Mark Abrams January 23, 2017 at 1:25 pm

It is the government propogated dietary recommendations which have resulted in obesity .

The government recommendations concerning sugar, carbohydrates , salt etc were harmful to many .and so they have been changing them, again .

We are all different , we must all make our own choices in life and that includes our diet and health care . Sadly delegating our choices to an omnipotent state (becoming slaves) results in nothing but misery and death although if one believes in the state with all one’s might it seems one can be oblivious to the corpses.

80 anon January 23, 2017 at 11:22 am

Heard this morning – Trump doesn’t like China, but he wants us to have products with Chinese level regulation.

81 anon January 23, 2017 at 11:53 am

I was scolded that this was not clear. The question was “does Donald Trump want melamine in our milk?”

With 75% less, “maybe more,” regulation do we start to get that?

Perhaps Alex has this fear as he places more emphasis than ever before on trade-off and benefits of regulation.

82 Mark Abrams January 23, 2017 at 1:28 pm

Are you incapable of buying locally produced milk ? Does the FDA prevent many locally produced dairy products from being sold ?

83 anon January 23, 2017 at 2:02 pm

As I am sure you know, locally produced dairy products are watched carefully, and the subject of periodic health recalls.

The federal agencies are certainly involved in that, both with regulations and inspections.

84 Mark Abrams January 25, 2017 at 12:21 pm

Your faith in state power is touching but you might read some public choice theory . Bureaucrats are not angels , they have their own interests and those interests are unlikely to be yours . Humans have been drinking milk for long before the FDA and yet here we are . How is that possible ? surely we must all have been poisoned long ago . If a farmer’s family drinks the milk he produces and wants me for a repeat customer that is more protection that any regulation will provide.

85 D. F. Linton January 23, 2017 at 11:45 am

Why not simply have two tracks for drug approval: the current safe and effective standard or just safety tested. Put the approval standard on the label: FDA Safe and Effective Tested or FDA Safety Tested Only. Doctors would use the lower category drugs just as they use off label drugs today.

86 AlanG January 23, 2017 at 11:57 am

Liability issues are too great. Companies can use FDA Approval in court and are routinely successful in those efforts. Would you want to put your company at risk by bypassing the FDA? Also, insurers want FDA approvals before they will pay. Who is going to buy a drug if the costs are huge and the benefits are unknown. Off label use is a red herring in that there is usually significant amount of published medical literature on the drug. Quite frankly off label use stays with us because many of the drugs are off patent and there is no the sponsor has no monetary reasons to get the follow on approval for the drug as only the generic companies end up benefiting.

87 Mark Thorson January 23, 2017 at 12:18 pm

That would open up a Pandora’s box of quack medicines like laetrile, antineoplastons, krebiozen, etc. You might argue that the market would sort out the wheat from the chaff — and you’d be dead wrong. If that were the case, homeopathic medicines would not exist, but it’s a thriving industry. Quack medicines like Prevagen are a bad enough problem today without making it much worse.

88 Mark Abrams January 23, 2017 at 1:35 pm

Placebos can make many people with minor problems feel better , there are plenty of studies showing this. Homeopathics and other “quack ” medicines can help those who believe in them so provided they are safe who cares ? Psych meds are NOT safe, cost much more and dont work any better . Why should you care how I spend my money ? Why should the FDA . The FDA initial mission was safety only it was only expanded to include efficacy relatively recently , they need to have their mission pruned back to safety ONLY . Let free people make their own choices.

89 AlanG January 23, 2017 at 1:47 pm

@Mark Abrams – yes let the people decide. What evidence do you propose to utilize for these decisions? Are you capable of reading a regulatory dossier and making a determination that a drug is effective? Do you rely on Internet chat groups for information in this regard? Have you ever read a drug label? Do you realize that drug safety is not assured at the time of approval but that it is an ongoing concern of the pharma industry and the FDA?

Please let me know the answers to those questions so I can be a better consumer and make my own informed choices.

90 robz January 23, 2017 at 4:12 pm

Libertarian philosophy assumes that everyone worth their salt(ie. not a slave or animal) will always perfectly informed.

You’re clearly not perfectly informed.

Me neither, though I do sometimes try.

Not sure if that makes me a slave or an animal.

91 Mark Abrams January 25, 2017 at 12:38 pm

I tend to hire an expert when making decisions about issues I dont feel competent to decide about . Since the FDA has no remit to interfere in the practice of medicine, they dont influence me or my advisors decisions as to what my health care decisions are . All they can do , and do do , is to limit my possible choices and I dont thank them for that . I have suffered and been crippled for years waiting for FDA experimental IND approvals . The FDA doesnt know if the treatments I fought for are safe but they have eventually approved them , often with ridiculous addendums such as the schedule of infusions and so forth (which cannot be deviated from ) . I and my doctors think these treatments will work, the alternative is an unpleasant and painful existence . Neither I nor the FDA have perfect knowledge and that isn’t the question . I would welcome their advice but not their control (which will be and is abused). The question is who decides . Being sick is similar to being in prison , one’s freedom is constrained . I have little tolerance for those who want others to decide as to whether I should be free or try to be free . I do think these are people who in essence have no problems with totalitarianism or slavery provided it is administered by credentialed “experts” or themselves .

92 Li Zhi January 23, 2017 at 11:49 am

1.”the ultimate goal of medical knowledge is increased life expectancy (and reducing morbidity)”
Uh, no it isn’t. First because there is no ultimate goal. Second because quality of life is as important as longevity. And third because other factors, such as the pursuit of happiness may collide with longevity and/or quality (for instance reproduction vs longevity or reproduction vs quality). Medical knowledge doesn’t have ANY goal, people have goals. Plural, multifaceted and often contradictory.
2. “If a drug or device is safe and it shows activity against the disease as measured by symptoms, surrogate endpoints, biomarkers and so forth then it ought to be approved.” AT is serious? Wow. So, since caffeine will (undoubtedly) show “activity” as measured by Alzheimer’s symptoms, then it should be an approved treatment? What drivel. FDA has a difficult job. One size doesn’t fit all. Incidentally, I was just reading about the child who died in Belgium because the anti-diphtheria medication wasn’t available. Seems the EU has the same problems we do with drugs which are no longer profitable to manufacture. The obvious answer is monopoly.

93 Mark Abrams January 23, 2017 at 1:42 pm

Caffeine is approved for anyone including Alzheimer’s patients and it is cheap too , you can drink as much as you want . Should the FDA ban it ? If a drug isnt profitable in the EU it is because the EU , or national health services wont pay enough to make it profitable . Monopoly wont help that at all .

94 mulp January 23, 2017 at 12:03 pm

“But the FDA should not be limiting the information that patients may discover about their own bodies or the advice that may be given based on that information. Interference of this kind violates the first amendment and the long-standing doctrine that the FDA does not control the practice of medicine.”

Everyone can pay to get gene testing done and get the data and dump it in an open database that likes to research reports related to each marker.

And given Alex view that experts are not good at determining the meaning, you as an individual are in full control of interesting the data as to the best possible medical diagnosis by reading the original research without the filtering of incompetent experts.

You as patient can the tell your doctors what diseases you have because you are smarter than all experts, including your doctor.

95 Mark Abrams January 23, 2017 at 1:51 pm

You should be able to hire anyone at all to analyze your DNA and inform you of any genetic risks you may have . The FDA doesnt think so , fearing that you may undertake unwise actions based upon potential risks . Consequently you can get your dna analyzed but no risk information can be provided . Thanks goodness an all powerful state is available to protect the stupid plebes from making their own undoubtably stupid decision about their health in partnership with medical experts they have engaged to assist them

96 Stuart Buck January 23, 2017 at 12:37 pm

Check out Derek Lowe’s blog at Science to hear from someone who is familiar enough with medicine and drug development to know that the FDA isn’t the real issue.

97 AlanG January 23, 2017 at 1:43 pm

You should realize that these are libertarians. they want to be able to make the choices themselves and don’t particularly care about science. Any of us who have spent time in the pharma industry know full well that what you write is correct. It’s faulty clinical data and poor regulatory submissions.

98 carlospln January 23, 2017 at 10:56 pm

Another dizzyingly naive post by Tabarrok, whose naivety over our lacunae of knowledge @ the molecular scale is apparently infinite.

“It’s easier for a company to blame Evil Regulatory Obstruction, but a big problem with that strategy is that people get a distorted view of the world. Many people (and many politicians among them) seem to imagine that there’s this big logjam of wonder drugs that’s having to work its way slowly through a thin hallway full of persnickity bureaucrats – if only we could open those floodgates! But this isn’t actually the case.” Derek Lowe, ‘In the Pipeline’

99 Mark Abrams January 25, 2017 at 12:58 pm

In my own case I would be interested in using omadacycline, ervacylcine, and brilicidin . However I cant, an experimental IND isnt possible, because until a drug is approved only an idiot would risk a null or adverse result . So I get to wait another 2 to 5 years and knowledge is lost or deferred. I would happily be a phase 1 or 2 data point , but because my infection is so rare I dont fit any likely trial. I get to suffer and it is indeed because of evil regulatory obstruction . I can only wish it were you .

100 anon January 23, 2017 at 12:57 pm

It occurs to me that “regulation” has given foes an easy argument. Because Americans, and citizens of advanced countries in general, are so fully protected, many do not just ignore trade-offs, they never feel them.

You can say “regulation is bad” easier when amoebic dysentery has never been delivered via your supermarket lettuce, or city water supply. That is when lack of regulation has never put you at risk. My Asian friends have a different experience.

It is possible that Trump and cronies will try to roll back too far, and this becomes a little less theoretical, even for libertarians like Alex.

The argument might cease to be without risk.

101 Mark Abrams January 23, 2017 at 1:59 pm

Because somehow the FDA cares more about amoebic dysentery than you, your grocer, your community and state do . You all want to be horribly sick but only the wonderfully beneficent FDA , staffed entirely by angels, wants you to be hale and well. No-one at the FDA ever ever leaves to take a high paying job in pharma , medical device companies or agriculture . No FDA personal have ever engaged in insider trading or influenced clinical trial interpretation for personal gain because they angels and above that sort of thing . And of course the FDA has been a huge proponent of phage therapy and phage anti-bacterial agricultural methods (which are effective and cheap) hasnt it ?

102 anon January 23, 2017 at 2:03 pm

Just wow.

103 Mark Abrams January 25, 2017 at 1:01 pm

I am prostrated by your lucid rebuttal

104 rayward January 23, 2017 at 1:00 pm

At least there’s a method to Tabarrok’s madness: he believes markets will sort all of this out and by some magic (the magic of markets) only safe and efficacious drugs will reach consumers. It’s madness nevertheless, even if it serves only to prove Tabarrok’s undying faith in the magic of markets. Uranium was at one time dispensed as a miracle drug, until the poor saps’ body parts stated falling off. Americans just elected a con man to be president, and Tabarrok believes those same folks have the good judgment to pick safe and efficacious drugs.

105 Mark Abrams January 23, 2017 at 2:07 pm

markets are people , if you want to buy defective poisonous products then you can and people will make those . People ate and prospered before the 1930s and federal regulation and even the blind can see that the size and power of the state seems to be negatively correlated with health and prosperity . Your choices are free markets or state control. You would have to be a fool to chose state control , but it is available. Move to cuba where you are allowed to earn $20 a month and have to being your own medicienes and linens to the hospital, or venezuela where there are no medicines , toilet paper or food, or North Korea where you can eat grass (when it is available) . If you want to stay in the USA then learn how free markets work, behave like a free man and stop extolling state slavery .

106 Just Another Right Wing Economist January 24, 2017 at 7:18 am

Talk about mood affilitation…. Economists applying their logic to fields they have no idea about, may give rise to lunatic ideas, but libertarians are supposed to be lunatics anyway, so nobody finds it troublesome.

107 Mark Abrams January 25, 2017 at 1:18 pm

Libertarians tend towards austrian economics which is a form of logic. As such it is domain independent (just as all logics are ) . By the way your writing is remarkably close to jibberish . Are you using a psychoactive substance ?

108 Mark Abrams January 25, 2017 at 1:19 pm

Clinical studies show that low levels of radiation are beneficial

109 Jon January 23, 2017 at 1:34 pm

Alex writes as if the people currently at the FDA have no clue concerning the trade offs between better evidence and faster approvals. In the past when I assumed people made rules because they were unaware of something, I found myself wrong.

I suspect that it is similar here. The people who lead the FDA have come to different conclusions from Alex either because 1) They have more detailed and relevant information he lacks, 2) They weigh various risks and rewards differently, and 3) they are constrained by laws and political pressure.

An example of (1)—a favorite libertarian complaint is that the FDA is too insistent on demonstrating efficacy by achievement of “end point” goals, as opposed to using intermediate bio-markers. The FDA certainly allows the usage of intermediate biomarkers in many cases. Furthermore there are significant cases where the drug appeared to work based on some physiological effect, was approved, and turned out to be ineffective or even worsen outcomes such as mortality. Examples of this are a blood pressure drug that lowered measured blood pressure, but did not impact the damage from high blood pressure; a diabetes drug that lowered blood sugar but the patients had more heart attacks; and an anti-arrhythmia drug that appeared successful in trials, but later it was found that it caused an increase in arrhythmia death rate in the patients.

110 Mark Abrams January 23, 2017 at 4:19 pm

The people at the FDA come to conclusions that benefit them (as per public choice theory). They would prefer to not ever be wrong and if that means you have to suffer or die while they make absolutely positively certain a product or service wont kill ANYONE well that doesn’t hurt them at all . Moreover if the product or service will cure YOU but might harm someone else well that cant be allowed either . And thy wont just offer their advice , no they insist on the right to make your decision for you .What can you do , one sick or dying person ? NOTHING .

111 carlospln January 23, 2017 at 10:41 pm

‘What can you do, one sick or dying person?’

Move to India, fuckwit.

112 Mark Abrams January 25, 2017 at 1:11 pm

Your advice is asinine, moving to India wont change the FDA in any way , wont improve health care in this country , wont help me at all and would entail many problems . You obviously arent worth much time or effort but you should learn to move your rather odd choice of identification, namely fuckwit down a line on your missives

113 Gomez January 24, 2017 at 8:45 am

Alex writes:

“If a drug or device is safe and it shows activity against the disease as measured by symptoms, surrogate endpoints, biomarkers and so forth then it ought to be approved. It often happens, for example, that no single drug is a silver bullet but that combination therapies work well. … Give physicians a larger armory and let them decide which weapon is best for the task.”

How does this type of thinking apply to the debate over whether or not insurance shows a mortality benefit? The Oregon Medicaid experiment did not show a clear mortality benefit but did show a large mental health benefit. It stands to reason that this is a reasonable surrogate marker for health and even mortality given a long enough trial. And what about comWhat if you replaced the words “drug or device” with “wage subsidy”, “clean air standards”, or “health insurance”? Should Congress give physicians a larger armory and let them decide which weapon is best for the task.?

114 MSimon January 26, 2017 at 10:59 am

Donald Tashkin found that cannabis smoked is mildly protective against lung cancer. The statistical significance of his study was 90%. No larger study has been done to confirm or deny his results. It has been 15 years.

BTW Tashkin was at one time an ardent prohibitionist. He is not quite so ardent these days.

115 Ryan T January 27, 2017 at 7:21 pm

Just a theory and I don’t have time to measure it, but I’ll try to take notice of this in the future. Here it is:

I’ve begun to suspect that posts like these — they’re often tagged ‘current affairs,’ but ‘policy recommendations’ might be more accurate — appeal to Republican vanity (the tone: you could be great!) while those targeted at Democrats appeal to conscience (the tone: you could have done better…).

In this one, the title may be a bit tongue in cheek, but it nevertheless seems to fit. I’m also aware that one comment on one post is a small sample size.

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