FDA

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If You Want New Drugs Faster, Get the FDA Out

Monday, March 25, 2013 by

Ogco_fda_1006Megan McArdle of the Daily Beast reports on the FDA’s recent decision to make the approval process faster for Alzheimer’s drugs. This is great news for patients, who may now see new Alzheimer’s drugs on the market years sooner than they otherwise would, and who may now have a more diverse selection of therapies to choose from to address their particular symptoms and stage of the disease.

But McArdle has a worry associated with speeding up the approval process: “This does elevate the risk that we’ll end up with a few drugs of dubious efficacy, or that don’t prevent the most troublesome ravages of the disease.”

But why is efficacy even a concern of government? If a drug doesn’t end up alleviating my particular symptoms, I can always toss it aside and try a new one.

Even under the new standards, there will likely be certain Alzheimer’s drugs that don’t get approved. For example, the FDA might deem a drug “ineffective” because it doesn’t improve cognitive function in 99% of the Alzheimer’s population and thereby ban it from sale. But if I’m in the 1% that benefits, that drug might be a godsend.

Why should the FDA get to decide whether it’s okay for me to take a drug? Shouldn’t I have the freedom to consult with my physician and decide for myself?

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Daily Caller: Will FDA choke off promising adult stem cell research?

Friday, August 10, 2012 by

ARC fellow Keith Lockitch has an editorial published in The Daily Caller today about the recent U.S. District Court decision to grant the FDA control over stem cell therapies (we’ve written about this on the blog here).

“There is no question,” Dr. Lockitch writes, ”that the government must spare no effort in defining and prosecuting real cases of medical fraud, malpractice and criminal negligence — and there is no question that such cases exist in the stem cell market. But  instead of serving as our protector against charlatans who prey on the sick and desperate, the FDA has itself become an agent of coercion, forcibly denying us the freedom to use treatments that could save our lives.”

Read the rest of the article here.

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Posted in ARI/ARC news, Government & Policy, Science & Environmentalism

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The FDA Is the Problem, Not Its Budget

Wednesday, May 2, 2012 by

The Wall Street Journal recently ran an op-ed by a former commissioner of the FDA on the agency’s impact on medical innovations such as stem cell therapy (a subject we’ve blogged about at length). The author, Andrew von Eschenbach, boils down the FDA’s dousing of innovation to one major problem: lack of funding.

But the problem is the FDA itself, not its current budget.

The FDA has no business coercively keeping drugs, devices and other therapies off the market before it deems them worthy. By what right does the FDA force innovative companies like Regenerative Sciences to take a “time out” on the procedure they spent years developing and testing so that the FDA can play catch-up to the new technology? By what right does the FDA make it illegal for Americans to use their own cells until bureaucrats give permission?

The FDA, by its nature, treats innovators as guilty until proven innocent—what’s a bigger extinguisher of innovation, in terms of the costs and time involved for gaining regulatory approval, than that? And the FDA forces the rest of us to abide by its collective edicts, rather than our own doctors’ assessment of risks and benefits in our own, individual medical circumstances.

According to Eschenbach, “Regulatory approval is the only bridge between miracles in the laboratory and lifesaving treatments.” It’s time to dismantle that bridge and establish a system by which the government puts on trial only cases of demonstrable fraud, not all promising therapies.

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Health Care Roundup

Friday, April 13, 2012 by

Here are some interesting and informative articles I’ve come across in following the debate on health care reform:

  • Health savings accounts (HSAs) have become increasingly popular since they were created in 2003. ObamaCare regulations may render HSAs illegal.
  • Employers are increasingly encouraging and incentivizing their employees to practice healthy behavior to cut down on health care costs.
  • Throughout the years, White House pressure has influenced FDA decisions.
  • Two doctors advocate for patients to decide in consultation with their doctors about the best course of care to pursue, versus bureaucrats or insurance companies. They highlight the crucial role a patient’s values and preferences play in medical decisions. (I agree that the government should definitely not encourage or dictate the medical practices patients adopt, but there is nothing inherently wrong with an insurance company on a free market [which is far from what we have today] encouraging what it sees as best practices. If you’re not happy with a company’s service, you can cancel your policy and find another better suited to your needs.)
  • William McGurn of the Wall Street Journal highlights a lesser known consequence of tax code laws in health care: homosexual couples don’t receive the same tax relief for purchasing insurance through their employers that heterosexual couples do.

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FDA Versus Stem Cell Therapies

Friday, March 23, 2012 by

Who owns your cells? The FDA seems to think it does, given its lawsuit against Regenerative Sciences, a company that treats orthopedic injuries by extracting, culturing and reinjecting adult stem cells derived from a patient’s bone marrow.

The case is precedent-setting in that FDA is claiming authority to regulate a patient’s own cells as though they were chemical drugs. As one researcher describes it:

If you start to look at this product as being the patient’s own stem cell, how can the FDA claim Regenerative is manufacturing [cells] – they’re culturing them. . . . They seem to have lost perspective on using autologous stem cells. There’s just no way you could apply manufacturing standards. . . . The FDA does not come into a cardiology practice and tell doctors how to do their surgeries or how to do heart replacements. And yet they feel they can come into a stem cell clinic.

The problem with FDA “coming into a stem cell clinic” is that this could have a significantly chilling effect on this whole field of medical research. Under the burden of FDA’s regulatory intervention, the costs of developing adult stem cell treatments would explode and treatments that might have otherwise been profitable might never even make it to market—as has happened with drug development in the U.S. And while stem cell therapies are under FDA review, patients will be denied government permission to use treatments derived from their own cells.

Christopher Centeno, the director of Regenerative Sciences, described to Forbes his company’s perspective on the case:

We see this lawsuit as a 21st century civil rights issue that will define what control you have about the use of your own cells and tissue. If a loved one is dying in intensive care and a well done study shows that the patient’s own cells can be used to help, does the patient get to decide to use those cells, or is that a decision for the FDA? Will the patient still be alive while we wait on Washington to issue this decision?

The prospect of patients dying waiting for FDA to act is, unfortunately, all-too real.

Because the agency has the power to keep drugs and medical devices off the market while its regulators plod through their labyrinthine review process, doctors and patients are forbidden by law from using treatments that lack the FDA’s stamp of approval, even with informed consent. In cases where the patient has little time left and no other options, this can lead to tragic outcomes.

Consider a device designed to replace a brittle aortic valve that was recently approved by the FDA. During a four-year period of FDA’s review, the device was used safely in Europe while being denied to American patients. According to AEI’s Scott Gottlieb:

more than 15,000 patients world-wide will receive the device by the time it’s slated for approval in the U.S. Some Americans healthy enough to fly have sought the procedure in Europe. Tens of thousands of Americans unable to travel, and too sick to undergo open-heart surgery, have died during the intervening four years.

How would you feel watching a loved one die, knowing that the only thing standing between him and a possible life-saving treatment is a group of government bureaucrats who think they have the right to deny it to him?

Or consider the beta-blocker class of drugs. In the mid-70’s, these had been shown to reduce the risk of secondary heart attack and some were adopted for this purpose overseas. “But in the U.S.,” writes CEI’s Sam Kazman:

FDA imposed a moratorium on beta-blocker approvals due to the drugs’ carcinogenicity in animals. . . . In effect, FDA was denying needed cardiac drugs to people at high risk of heart attacks because of the unproven possibility that those drugs might cause cancer years in the future. Finally, in 1981 FDA approved the first such drug, boasting that it might save up to 17,000 lives per year. That meant, of course, that as many as 100,000 people may have died waiting for FDA to act.

Kazman calls these many thousands of individuals—in this and other similar cases—the FDA’s “invisible victims.”

The field of adult stem cell therapy has been slow to develop and treatments that show signs of real promise are only just beginning to emerge. It would be tragic if regulatory intervention by FDA were to stifle this field in its infancy.

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Do Stem Cell Therapies Stand a Chance Against the FDA?

Tuesday, March 13, 2012 by

There is currently a debate underway about the FDA’s authority to regulate stem cell therapies. The FDA is suing a Colorado-based company, Regenerative Sciences, which has developed a procedure to treat a person’s damaged joints by extracting and re-injecting his own stem cells. The FDA is arguing that the procedure involves more than “minimal manipulation” of the stem cells and therefore falls under the FDA’s authority, requiring a lengthy review and approval process—as other drugs and devices do—before being made available to patients.

There has been controversy over the idea of the FDA asserting the power to regulate a person’s own stem cells—and for good reason. If the FDA loses this case, says one scientist, it “would not be in a position to regulate the production and delivery of patients’ stem cells into themselves. That would remove massive commercial barriers to the implementation of this science and technology, because it would remove a lot of steps that cost a lot of time and money.”

People are right to be concerned about this. Typically when the FDA gets involved, it means more expensive treatments that take years longer to hit the market. For a field that is only just beginning to realize some of its promise, FDA regulatory delays could nip it in the bud.

The FDA’s intervention in the field of gene therapy provides a precedent. According to Christopher Scott of Stanford University’s Center for Biomedical Ethics, “there were concerns that the science was moving too quickly, so the FDA stopped all trials and the field was effectively shut down. There was such a severe reaction on the part of regulatory agencies that the science was effectively moribund, and now it is slowly coming back to viability.”

It would be a shame if a burgeoning field such as stem cell therapy was similarly stifled by the FDA’s intrusion.

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In a mixed economy, regulations always grow

Wednesday, March 7, 2012 by

In my last post, I shared an observation that government regulation always seems to grow in scope and, once established, almost never shrinks. How does that pattern become established? There are many factors that lead to the entrenchment of regulatory power and it’s ever-growing reach.  Over the next few posts, I’ll discuss some of these underlying causes.

Ayn Rand viewed the pattern of increasing regulation as inevitable in the kind of political system that we have, which she described as a “mixed economy.” Because we have a combination of freedoms in the form of individual rights and controls in the form of ad-hoc, piecemeal regulations curtailing those rights, Rand viewed our system as inherently unstable. In her words:

“A mixed economy has no principles to define its policies, its goals, its laws—no principles to limit the power of its government. The only principle of a mixed economy—which, necessarily has to go unnamed and unacknowledged—is that no one’s interests are safe, everyone’s interests are on the public auction block, and anything goes for anyone who can get away with it. Such a system—or, more precisely, anti-system—breaks up a country into an ever-growing  number of enemy camps, into economic groups fighting one another for self preservation in an indeterminate mixture of defense and offense, as the nature of such a jungle demands. While politically a mixed economy preserves the semblance of law and order, economically it is the equivalent of the chaos that had ruled China for centuries: a chaos or robber gangs looting –and draining the productive elements of the country.”

When a question arises about whether stem cell research of the kind that Regenerative Sciences is doing should be regulated, think about all of the lobbying groups with their varying agendas and motivations that claim to have a stake in what the outcome is. Doctors, private corporations, researchers, patients, relatives of patients, insurance companies, pharmaceutical companies, religious opponents of scientific research, universities, political parties, and so on. Each group will immediately feel the urgent need to clamor for regulations and actions that will protect what they claim are the interests of some deserving group. Each group claims to have its own need for protection or concessions–which entail new restrictions or controls on others. No wonder lobbying is a multi-billion dollar industry! No wonder massive institutions like the FDA have expanded, in the face of these alleged high-priority claims! The upshot is a continual erosion of freedom in favor of regulation.

In all of this clamoring, legitimate questions get lost. Questions such as whether there is really a safety threat presented by these particular stem cell technologies (unclear), whether existing laws around fraud and product liability are already sufficient to protect patients’ rights (they are), whether there might be private groups or companies that can offer an independent evaluation of stem cell technology providers (try Googling it).

Instead, in a mixed economy, the pattern is that every problem looks like a nail and the hammer is always to increase regulation.

But surely there’s a limit to all the claims that might be raised and therefore some limit to the growth of regulation? Stay tuned for my next installment.

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Posted in Ayn Rand, Business & Economics, Government & Policy

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Shortage of Drugs, Abundance of Regulations

Monday, February 27, 2012 by

There is currently a shortage of more than 200 drugs in the United States. Among the drugs critically in short supply are anesthetics, blood pressure medications and a whole host of cancer-fighting treatments, the shortage of which has tragically left many patients with the grim prospect that they may not survive ailments that are treatable.

Many explanations have been put forth as to what is causing this drug shortage. The New York Times, for example, cites “contamination problems at some manufacturing plants, forcing unexpected production shutdowns, [and] difficulties in getting pharmaceutical ingredients from suppliers, especially those abroad,” as some of the factors that have played a role.

These factors might help explain why a particular manufacturer stopped producing a particular drug, but they are not sufficient in explaining why such a wide-scale and long-standing shortage of drugs continues to occur in this country.

This sort of lasting shortage is unusual in a free market economy because if the supply of a product falls, prices rise to signal producers to increase production.

So why didn’t we see these market mechanisms operate and resolve the shortages in the drug market? Because that market is anything but free.

It’s a complex task to trace all the effects of the myriad of government controls on a particular market. But to get started, here are a few examples of government interference in the drug market that are most certainly distorting the mechanisms that normally keep supply and demand in check:

  • Drug manufactures are not allowed to ramp up production of a drug without undergoing a lengthy approval process from the FDA. John Goodman of the National Center for Policy Analysis explains how this affects the available supply of a drug:

    “[A] drug manufacturer must get approval for how much of a drug it plans to produce, as well as the timeframe. If a shortage develops…, a drug manufacturer cannot increase its output of that drug without another round of approvals. Nor can it alter its timetable production (producing a shortage drug earlier than planned) without FDA approval.”

  • New suppliers wanting to enter the market are also delayed by the FDA. A Wall Street Journal editorial on the issue notes:

    “It takes as long as two and a half years to receive FDA manufacturing approval for a generic.”

  • Many of the drugs in short supply, particularly the cancer-fighting ones, can only be administered by doctors, who purchase the drugs from manufacturers. If a patient has Medicare, the doctor is paid a fixed price by the government to cover the cost of the drug, even if purchasing the drug cost the doctor more than that amount. Scott Gottlieb of the American Enterprise Institute explains why this is a problem:

    “A 2003 law fixes the price Medicare will pay for injected drugs to an ‘average sales price’ that is at least six months old at any given time. This flawed concept means even if a generic firm raises its price to reflect increased production costs, the new price won’t get paid by Medicare—meaning purchasers would be losing money for months at a time. The result is that generic prices can’t rise to reflect changing demand or the need for bigger investments in manufacturing.”

Why are certain drugs in drastically short supply? Any politician who’s serious about finding an answer to that question should first look at the regulatory road blocks and price controls that are standing in the way of drug producers.

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The Expanding FDA

Thursday, February 16, 2012 by

A lawsuit between the FDA and a medical technology company, Regenerative Sciences, has been getting attention recently.  The company has pioneered a procedure (Regenexx-C) whereby a person’s own stem cells can be extracted and reinjected into his or her body to help repair damaged joints. The FDA argues that they have jurisdiction in this case because the stem cells are being extracted and manipulated via a technological process. They claim that as a result the stem cells themselves need to be regulated the way other medications are.

People are reacting with outrage, and rightly so, that this is a new expansion of governmental power that puts the FDA in control of what individuals do with parts of their own bodies.

While it’s encouraging to know that some Americans find the FDA’s arguments in this case to be ludicrous and to be a particularly offensive increase in the government’s intrusion into our lives, it’s dismaying to observe that the direction in cases like these always seems to be one of increasing regulatory power, never decreasing.

In the current case, the discussion is focused on whether and where the lines of FDA’s authority should be expanded (not shrunk). Should it be able to regulate drugs but not stem cells used in medical therapies? Should a drug be defined as something chemical and manufactured as opposed to something chemical and natural (such as stem cells)? How much manipulation is required for something to be considered a drug? And so on and so forth.

The answers always seem to push us in the direction of increasing levels of regulation. Once a precedent is set for regulating a sphere of human activity, the default becomes a growth in regulation. The pattern seems to be: if you’re not sure where the line should be drawn, better err on the side of regulation . . . just in case.

How does such a pattern become established? That’ll be the topic of my next post.

image: wiki commons

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“Large enough to be meaningful”—who decides?

Wednesday, November 30, 2011 by

As expected (and dreaded, by some), the U.S. Food and Drug Administration has revoked its approval of Avastin for treatment of advanced breast cancer. The Wall Street Journal has reported and editorialized on the event, which came in the form of a 69-page decision by Food and Drug Commissioner Margaret A. Hamburg, M.D.

If you wade through the legalese and medical technicalities all the way to page 38, you will encounter Dr. Hamburg’s crucial conclusion about Avastin’s effects on “PFS.” What’s PFS? It’s an acronym for “progression free survival,” a period of time during which a patient’s disease fails to worsen. In other words, for a patient in a fatal stage of breast cancer, an extra day of PFS = an extra day of no tumor growth.

Back to page 38, where Dr. Hamburg concludes that “the evidence does not show that Avastin has had an effect on PFS large enough to constitute clinical benefit.” The italics are mine—to stress the kind of value judgment Dr. Hamburg is making. What does that mean, “large enough”? According to Dr. Hamburg, it means “large enough to be meaningful to a patient.”

Do you see what’s going on here? Dr. Hamburg, a federal bureaucrat, is claiming a right to decide whether a particular period of delayed tumor growth is “meaningful” to individual patients whom she will never meet, much less examine and treat. In the particular case of Avastin, the latest statistics show that median PFS ranges from 0.8 months to 2.9 months, depending on which study you look at. By putting “median” in italics, I’m stressing that half the women in these studies experienced even longer periods of delayed tumor growth than those deemed not “large enough” to matter. Yet our law permits an FDA bureaucrat to limit these women’s access to medication, simply because an equal number of other women are predicted to benefit less, or not at all.

This is the practical meaning of collectivized medicine, about which I wrote at greater length last year, in a PJMedia op-ed called “The Avastin Travesty”:

But can risks and benefits really be weighed at the level of society as a whole? A society is only a collection of individuals. A society doesn’t enjoy life, or suffer — only individuals do. Metaphors aside, a society doesn’t get sick and die — only individuals do. To appreciate the difference, consider how a rational patient with breast cancer decides whether to undergo drug treatment.

Such a patient weighs (among other things) the statistical likelihood of a favorable result against the statistical likelihood of painful side effects. At all times, her judgment is individual and personal: How will my life improve if these tumors temporarily stop growing? How might side-effects interfere with my enjoyment of life? How much better will I feel if the results are above average — or how much worse, if the results are below average? How much is an additional year, month, or week of relatively normal life worth to me?

The FDA’s experts take professional pride in refusing to allow such individual considerations to influence their decisions. Instead, they float among the statistical clouds, observing that Avastin delays tumor growth by only 3 to 12 weeks on average and that some patients actually get worse after taking the drug. From behind a veneer of scientific respectability supplied by charts and graphs that ignore the individual patient, these experts then ask a question to which no rational answer can be given: What is the meaning to society of one month in an individual’s life?

In the ongoing debate over American medicine, it is crucial to understand how often we are asked to sacrifice the individual’s welfare for the alleged needs of society.

Image: WikiMedia Commons

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