The 21st Century Cures Act includes funding for many meritorious public health initiatives. It’s also a gigantic boondoggle for Big Pharma. And it eases up evidentiary standards for the FDA’s drug and device approval process in the interest of innovation and competition.
What if Trump came to cut some red tape, but Obama had already done it?
By signing the 21st Century Cures Act, the sitting president established as law a provision that will “accelerate delivery of medical treatments to patients by advancing the drug approval process,” according to a statement from Virginia Sen. Mark Warner on the act’s passage.
That’s in addition to establishing a mechanism for Congress to allocate $4.8 billion in biomedical research funding for the National Institutes of Health. The “Cancer Moonshot” is a go, as is the BRAIN initiative to take down Alzheimer’s.
The 21st Century Cures Act also includes $1 billion to fund the fight against a nationwide opioid abuse epidemic and measures to make mental health coverage equal with physical health coverage when it comes to insurance policies.
There are also updates that will help Medicare beneficiaries with specific ailments get more targeted care and measures to facilitate access to care for patients in rural areas of the country.
That’s all just policy stuff, though.
Harnessing the Food and Drug Administration (FDA) is about “freedom” and “hope.”
A codicil to President-elect Trump’s promise to “repeal and replace Obamacare” — expressed in his “Contract With the American Voter” — is a commitment to reform the FDA, where “there are over 4,000 drugs awaiting approval.” Trump is particularly keen “to speed the approval of lifesaving medications.”
The FDA is an easy target, a big government bureaucracy with a vast mandate that probably affects you and me every day, with the equivalent of nearly 15,000 full-time employees. (That represents about 0.7% of total executive branch civilian employment.)
Harnessing the Food and Drug Administration (FDA) is about “freedom” and “hope.”
This agency of the U.S. Department of Health and Human Services regulates human and veterinary drugs, vaccines and other biological products, medical devices, most of our food, all cosmetics, dietary supplements, products that give off radiation, and tobacco. (That’s just about all of it, though the Department of Agriculture regulates meat from livestock, poultry and some egg products.)
It does all that via the Office of the Commissioner and four directorates: Medical Products and Tobacco, Foods and Veterinary Medicine, Global Regulatory Operations and Policy, and Operations. (Soviet Russia had many “directorates.”)
The FDA’s budget for fiscal 2016 was $4.9 billion, a 9% ($424.8 million) increase compared with fiscal 2015. (That’s about 0.1% of the total U.S. budget for fiscal 2016 of $3.45 trillion.)
Approximately 45% of that budget comes from “user fees,” or what drug and device makers pay to fund the approval process. (“User fee” is a euphemism for “tax.”)
So when Trump talks about “cutting red tape” at the FDA — when Obama signs legislation ostensibly accomplishing same — the ultimate result is to save pharmaceutical and medical device companies a lot of money. (Surprise: Big Pharma loves the 21st Century Cures Act.)
Trump is very likely to push for even greater clipping of the FDA’s wings, in favor of innovators, entrepreneurs, and investors champing at the bit to unleash their revolutionary magic, save lives, and realize rapid returns.
So when Trump talks about “cutting red tape” at the FDA — when Obama signs legislation ostensibly accomplishing same — the ultimate result is to save pharmaceutical and medical device companies a lot of money.
And at the end of the day, as Veronique de Rugy put it in National Review, aren’t we adults “often better suited to know what is best for [us] than bureaucrats in Washington”?
Celine Ryan certainly stood up for herself. At first rejected due to participation in an experimental study, the 50-year-old engineer offered an effective appeal and was eventually accepted.
And now she’s cancer-free, though not necessarily “cured,” thanks to a novel immunotherapy that worked against her colon cancer.
It didn’t hurt that Ryan “has an unusual genetic makeup that allowed the treatment to work.”
Still, that’s one helluva anecdote — an experience that validates the view that the FDA’s 4,000-strong backlog includes many more such miraculous cures.
It’s the kind of story that supports legislation that eases FDA evidentiary standards for drug approval.
Now thanks to the 21st Century Cures Act, the agency’s experts can evaluate “real-world evidence” and “patient-experience data.” That ought to accelerate patient access to cures.
And it might just stimulate competition by reducing development costs. The current process — based on a series of increasingly large randomized placebo-controlled trials — is extremely expensive.
But let’s stipulate on a matter of common sense: The most important thing for a new drug is that it be effective against the illness, disease, or condition it’s designed to treat.
The priority should not be to get “miracle” drugs to market faster. The priority should be to get effective drugs to patients in an efficient manner.
“Health policy experts, on the other hand,” writes Dr. Alan Levinovitz, an assistant professor at James Madison University and author of The Gluten Lie: And Other Myths About What You Eat, “are rightfully nervous about the unintended results of laxer standards: a market flooded with potentially unsafe drugs that are no more effective than placebo.”
As Ameet Sarpatwari and Michael Sinha write for Health Affairs, explaining the shortcomings of “real-world evidence” and “patient-experience data”:
Defined as information on drug outcomes that are derived from sources other than clinical trials, real-world data are less likely to have been uniformly collected and therefore risk being less reliable. Attempted analysis of real-world information is potentially further plagued by systematic differences between populations of interest that may not be captured and, thus, cannot be adequately controlled, which can distort the true benefits and risks of a drug.
Rachel Sachs, writing for Harvard University’s Bill of Health blog, notes the real-world complications of debased evidentiary standards:
To be sure, this provision gives enormous discretion to the secretary to limit (and maybe even reject) the use of such evidence. But in light of recent high-profile clinical trial failures, most notably just two days ago, we ought to be concerned about claims that the FDA is too slow and imposes too stringent requirements on drug approvals.
Indeed, the FDA is open to scrutiny after it allowed a Phase II clinical trial of Juno Therapeutics Inc.’s (JUNO) “Rocket”/JCAR015 CAR-T cancer immunotherapy to resume following the deaths of three patients earlier this year.
The priority should not be to get “miracle” drugs to market faster. The priority should be to get effective drugs to patients in an efficient manner.
Two more patients died in November of cerebral edema, the same cause as the prior three deaths. Perhaps the FDA should have taken more time to understand the causes of the brain swelling.
Codifying “real-world evidence” and “patient-experience data” is a response to compelling arguments rooted in self-determination and wish fulfilment, anecdotal experience versus empirical evidence.
HealthNewsReview frames the problem well:
“Ten years from now, someone with a cancer diagnosis will be worse off with this bill,” says Dr. Vinay Prasad, an oncologist at Oregon Health Sciences University whose research has demonstrated that many new therapies ultimately fail to cure or are harmful because they are based on inadequate studies. “People will be exposed to more things that don’t work.”
We do like to think of ourselves as a nation of rugged individuals. (“KEEP GOVERNMENT OUT OF MY MEDICARE!”)
And it’s nice that a legion of more than 1,400 lobbyists ensured we the people get to take more responsibility for personal medical decisions by advocating on behalf of the 21st Century Cures Act in the halls of Congress, a bill of more than 1,000 pages that rivals the Affordable Care Act in complexity.
We’re also a litigious people, and no doubt many of those for whom miraculous cures don’t work will resort to those great equalizers, the plaintiff’s attorney and the civil complaint, for redress.
“Freedom” and “hope” are powerful marketing tools.
“Efficacy” has too many syllables.
Money Quote
By exiling human judgment in the last few decades, modern law changed its role from useful tool to brainless tyrant. This legal regime will never be up to the job, any more than the Soviet system of central planning was, because it can’t think. The comedy of law’s sterile logic — large POISON signs warning against common sand, spending 22 years on pesticide review and deciding next to nothing, allowing 50-year-old white men to sue for discrimination — is all too reminiscent of the old jokes we used to hear about life in the Eastern bloc.
Judgement is to law as water is to crops. It should not be surprising that law has become brittle, and society along with it.
— Philip K. Howard, The Death of Common Sense: How Law Is Suffocating America
Smart Investing,
David Dittman
Editorial Director, Wall Street Daily
The post The FDA: Useful Tool or Brainless Tyrant? appeared first on Wall Street Daily.
By David Dittman
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