The FDA Stifles the Advance of Modern Molecular Medicine

Wall Street Journal review of the new book, The Cure in the Code, by Peter Huber

The Cure in the Code: How 20th Century Law Is Undermining 21st Century Medicine, by Peter Huber, Basic Books, 277 pages, $28.99

We are at a turning point in medicine. Knowledge of the individual's genetic makeup will soon allow molecular medicine to reach deep inside each of us to cure most of the maladies that afflict us—and perhaps even slow the rate at which we age. First we will learn to understand each person's genome; then we will learn to craft treatments tailored to his or her genetic constitution.

But it may not be so easy—and not for purely scientific reasons. Consider 23andMe, a commercial enterprise launched in 2006 that was merely looking to inform Americans about their potential genetic vulnerability to certain diseases. Regulators from the Food and Drug Administration have dropped the hammer on the company, citing baseless fears that its customers will do something dangerously stupid in reaction to the information that the tests provide. The FDA's regulatory labyrinth is not only slow to digest the science behind the genetic testing involved in 23andMe. It also can't quite figure out what to do with the proliferation of molecular biomarkers that can predict treatment efficacy more quickly than the conventional clinical trials the agency relies upon.

All this is just the tip of the iceberg, Peter Huber argues in The Cure in the Code, his urgent, compelling account of how 21st-century medicine is being hampered by a regulatory regime built for the science of the 20th century.

CureintheCodeBasic BooksMr. Huber begins by taking us back to the rise of public health and modern medicine in the late 19th and early 20th centuries. The advances made in this era were enormous: Infectious diseases were tamed by the construction of sewers, the provision of chlorinated water, widespread vaccination and the birth of antibiotics. These magic-bullet successes enabled us to live longer than humans had hitherto—long enough, in fact, that we now suffer more from a qualitatively different set of illnesses, diseases that emerge from the biochemical combinations of our genes and our lifestyles. Today's prominent maladies—cancer, diabetes, heart disease and dementia—take decades to develop, and successful treatments must begin decades before patients become clinically ill.

Our conception of illness has thus been transformed. Sequencing entire human genomes has revealed the tremendous biochemical diversity of human beings. Our susceptibilities are particular to each one of us, and the time when blockbuster drugs, such as penicillin, could treat nearly every patient is over. The biochemical roots of heart disease in two patients sharing a hospital room are very different and won't respond to the same drugs in the same way.

"Health care once mainly depended on killing germs," writes Mr. Huber. "Now most drugs are used to tune people." Lipitor tunes our artery-clogging cholesterol; Plavix our blood-clotting platelets; Prozac our depressed dopamine receptors; Cardizem our irregular heartbeats; and Gleevec toggles down our leukemia. Tomorrow's regimens, as Mr. Huber shows, will be guided and adjusted using relevant biomarkers specific to individual patients.

One can get a glimpse of the future of radically individualized medicine by considering how each woman's breast cancer is now treated in light of her tumor's genetic susceptibility to estrogen, progesterone and the HER2 protein. Breast cancer isn't a single illness. Rather, each woman's cancer may be its own distinct case of malignant disease. Oncologists now input a breast-cancer patient's genetic data into diagnostic algorithms to devise drug regimens just for her. Such therapy is less miss and a lot more hit.

Yet the FDA still operates according to the requirements of the age of mass drugs. Founded in 1906, when quack remedies were common, the FDA has evolved into a bureaucracy more concerned with avoiding risk than speeding the benefits of innovation to patients. Drug approvals still depend on large-scale trials that focus on statistical correlations to determine clinical efficacy. Regulators demand ever larger trials as a way to avoid approving drugs that will turn out to have significant side effects in some patients.

This obsession with side effects is misplaced. Side effects should more often be regarded as information indicating that a treatment that might be effective for many patients doesn't work for some. Too often, the FDA refuses to approve a new drug because it doesn't work for everyone, when the goal should be to find those patients for whom it does work. "The broader lesson here," says Mr. Huber, "is that every new, precisely targeted drug serves, in part, as a diagnostic instrument for exposing and classifying the molecular structure of diseases and the bodies that host them. As the drugs are developed and prove their worth, they redefine the diseases."

So what would a 21st-century drug system look like? The author argues that the current clinical-trial model must be replaced by adaptive trials in which both patients and physicians would continually learn and modify treatments along the way. Information about the molecular processes being targeted and the outcomes of each patient's therapeutic regimen would be reported to a broadly accessible digital knowledge network, which other doctors and patients could query to guide their choice of therapies.

Mr. Huber outlines how to reform the FDA, intellectual-property rights and our tort system so as to unleash "market forces to develop reasonable schemes that integrate the development of drug science with the sale of drugs for treatment." Among other recommendations, he suggests that the FDA permit patients and physicians access to treatments formulated by adaptive trials, whose regimens are constantly fine-tuned in light of molecular findings. In addition, stronger intellectual-property rights should be granted to discoverers of biomarkers and aggregators of clinical databases and predictive-knowledge tools. Such incentives will "reward those who develop new information faster and come up with new ways to distribute it widely at economically efficient prices," writes Mr. Huber.

These reforms would also cut health-care costs. Drugs and diagnostics are in essence distilled information with high upfront costs, but as with information technologies, their costs fall rapidly. Ever cheaper advanced molecular medicine will displace the increasingly expensive and often ineffective hands-on care of doctors and hospitals for cancer and heart disease—just as antibiotics and vaccines supplanted costly iron lungs and tubercular sanatariums.

This review first appeared in the Wall Street Journal on December 3.

See also reasontv's excellent interview with Peter Huber about The Cure Is In The Code below:

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  • Fist of Etiquette||

    FDA bureaucrats have probably harmed more people than they've helped at this point. Any percentage of people dying from untreated conditions is acceptable for the greater good.

    The FDA, like all government certifications, should be voluntary. As a patient I should be able to tell my doctor I want only FDA approved treatments, or be able to ask for the best option from the entire pool of possible remedies for my sucking chest wound.

  • Nazdrakke||

    FDA bureaucrats have probably harmed more people than they've helped at this point

    Without a doubt in my mind, and not just in America.

  • SQRLSY One||

    Y’all are hurtin’ my most Deeply Felt Religious Convictions, making fun of the FDA, of all things… Don’t y’all know, the FDA doesn’t just deserve our Deepest Respects, it actually deserves our all our flat-out WORSHIP! See www.churchofSQRLS.com for details …

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  • Irish||

  • The Rt. Hon. Serious Man, Visc||

    You're really enjoying that, aren't you?

  • ||

    I'm enjoying it, too. Not the blessing by the priest (which is just voodoo), but the crushing of the statue's head.

    (Continental) Marxists can't be harassed anymore by mentioning Stalin or Mao -- or the millions of their victims --; they claim that those dictators weren't following the one true path of Marx, Engels and Lenin. Lenin -- who instituted the Red Terror -- is still held up as the true Communist.

    This statue-toppling has probably more to do with anti-Russian sentiments in the Ukraine than with anti-Communism ... but I still like it.

  • dinkster||

    What, that idiot savant bus driver in Venezuela isn't a good example?

  • John Galt||

    Not sure why BuzzFeed has blocked my accounts. Apparently, either they're a little too sensitive, or too dull-witted to recognize sarcasm.

  • John Galt||

    Lefty hates hard science, he-she vastly prefers politically charged pseudoscience...

    Come on proglodytes, you control the bureaucracies! What is it that makes you fear real science? You've certainly had no problem at all using nonsensical pseudoscientific garbage to great harm many times throughout history. Is it really so difficult to stop thinking with your emotions long enough to allow hard science an opportunity for once?

  • mtrueman||

    "Lefty hates hard science, he-she vastly prefers politically charged pseudoscience..."

    An interesting charge, and perhaps true. But it misses out on a larger truth that our health problems are best solved not through supposedly politically neutral hard science, but through a mix of these coupled with politically charged pseudoscience. Take dehydration, for example, the number one cause of death among infants in the world today. More than a million die of it every year, according to figures gleaned from sources such as wikipedia.

    Hard sciences have done just about all they can do to combat dehydration. Their answer is simple and effective. It comes in the form of supplemental zinc and potassium tablets. Trouble is, this is not enough. To prevent the deaths, leftish beloved pseudoscientific measures like improving mother's education and public sanitation are also called for.

    Whether leftists fear hard sciences or not is a trivial issue I leave for identity political warriors like yourself to debate. The fact remains that health issues are resolved, or not, by a mix of hard science and pseudoscience.

  • David Wall||

    If and when the Liberty movement sweeps enough folks into office to dismantle America's state behemoth, the FDA should be at the top of the list. I would like to live to be 150 years of age. It might become possible if the FDA & the monstrous regulations that go with it are abolished.

    A guy can hope.

  • mtrueman||

    " It might become possible if the FDA & the monstrous regulations that go with it are abolished."

    You forgot to mention that the monstrous regulations of the state IP protection bureaucracy should be beefed up. You won't see your 150th birthday unless this happens.

  • dinkster||

    Could they just be a Ted Turner level of crazy and believe that mass death is the only way to save Gaea?

  • Danno||

    DNA only shows the basic landscape. RNA which is much more plentiful determines what genes are expressed and can influenced by environment and the RNA state can be inherited. .

  • Car Scanner||

    I think so that we are at a turning point in medicine.

  • doc30||

    Don't forget that the Affordable Care Act will further inhibit this type of specialized medicine. One function of the independent Review Board is to contain costs by approving treatments that do the most good for the least cost. This philosophy, while sounding reasonable at face value is the exact "magic bullet" philosophy mentioned by the author that the FDA uses in approving new pharmacological products. Now, under ObamaCare, it has become a legislated reality. Individualized treatment requires more time and overhead than prescribing an existing, far cheaper medicine. The law will never allow such medicine to be practiced.

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