A Great New FDA Commissioner
n an article published online on January 23, 2017,* author Alex Taborrak, PhD, who has written about FDA reform for many years, applauds potential nominees Balaji Srinivasan, Jim O’Neill, Joseph Gulfo, and Scott Gottlieb, the first two of which are Peter Thiel associates and recommendations.
According to Taborrak, each of these understands two important facts about the FDA that there is a fundamental tradeoff. “… [L]onger 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.”
It is also Taborrak’s understanding that each of the leading candidates likewise understands that we are entering a new world of personalized medicine that will require changes in how the FDA approves medical devices and drugs. That’s because the smartphones that we carry in our pockets already embrace the future of medicine or soon will be.
Schizophrenic Attitude Towards These New Devices
The FDA has an inconsistent abnormal attitude towards these new devices. Its tendency is to ban them just as the FDA has done with many new biomedical technologies, including 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.”
While Taborrak thinks that the FDA has a role in ensuring that a device or test does what it says it does, he also believes that 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 (2 undergraduate degrees plus a PhD from Stanford) who has also published in the New England Journal of Medicine, Nature Biotechnology, and Nature Reviews Genetics. More to his power, 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 coming world of personalized medicine.
Moreover, Taborrack believes that world of personalized medicine mandates a different approach for how new drugs and devices should be evaluated. Traditionally, 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. But that has all changed, however, with “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.”
Personalized medicine advocates that we permit more liberal approval decisions and improve our techniques for monitoring individual patients. This will enable physicians to adjust prescribing in response to an individual’s reaction. Give physicians a larger armory and let them decide which weapon is best for the task.
In his article Taborrak also agrees with Joseph Gulfo that in an effort to be scientific, the FDA has sometimes fallen victim to the fatal conceit, the idea that man is able to shape the world around him according to his wishes.
Taborrak further believes that the ultimate goal of medical knowledge is increased life expectancy (and reduced morbidity). Yet that doesn’t mean that every drug should be evaluated on this basis. Drug or device safety showing activity against disease as measured by biomarkers and so forth ought to be approved.
As frequently is the case, no single drug is a silver bullet but that combination therapies work well. These therapies in FDA approved clinical trials frequently requires the discovery of process in 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….
Taborrak conjectures that DeVita is correct. Shockingly, one of the reasons we see lots of trials for end-stage cancer is that you don’t have to wait long to count the dead. This is not so for other cancers and to date, no drug has ever been approved to prevent lung cancer because the costs of running a clinical trial for long enough to count the dead are just too high to justify the expense.
Jim O’Neill, managing director at Mithril Capital Management and a former HHS official, is also a Peter Thiel associate and recommendation. Of further significance is his 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.” This is the kind of thinking that could be just the thing to bring about the necessary change in the FDA’s archaic mindset.
And beyond these big ticket items there ae some simple changes that could be made at the FDA and lead for example to decent sunscreen and medicine for traveler’s diarrhea.
Will a similar consciousness be applied to supplements? We think so, and it may be sooner than you think.
“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.”
Live long and prosper,