Category Archives: Ethics

It’s in vogue in medicine to market drugs offering slight benefit to desperately ill patients at cost of between $50,000 and $100,000/year.

What’s the real cost of these “advances.”

In a hypothetical (but typical) situation lets say the drug is used in 100 patients and compared with another group of 100 patients receiving placebo (or standard therapy).

As one would hope the group of patients receiving the study drug appears to have some response. In the distant past a response was defined as a decrease in tumor bulk of 50%. In recent years that definition has been broadened, some would say undermined, to mean that there was no evidence of tumor progression during treatment. Since we are in “modern” times lets use the definition of response being no progression.

We do our study and find in the group of patients receiving placebo (or standard therapy) 25% showed no progression. While in the study group receiving the new drug of 100 patients 40% showed no progression.

Drug companies take such data as evidence of the drug’s efficacy and try to get FDA approval. Note there is no evidence at this point of extension of life.

The real cost of this advance is interesting to contemplate. Consider treating 100 patients with a $100,000 drug–that’s 10 million dollars. Consider that just 40% get a “benefit” with the drug and 25% had the same “benefit” with placebo (or standard therapy)–and a marginal “benefit” at that. That means that 6 million dollars was spent on patient who got no “benefit” at all from the drug (the 60 of 100 patients without “benefit”). 2.5 million dollars on the 25 patients who would have shown no progression with placebo (or standard therapy). The incremental “benefit” of the drug over placebo or conventional therapy was therefore a total of 15 patients.

That means in our hypothetical but somewhat typical new drug situation 85 out of 100 patients will be treated with the $100,000/year drug and get nothing from it. 15 patients get a limited improvements. The cost/patient that improved is $100,000 X 100 patients treated/15 patients who get the improvement=$666,666 expended for each patient who improved. It should be noted that this is a cost per year figure for a single drug.

How much is a reasonable amount for the system to spend for an improvement. It’s been thought in the literature that between $50,000 and $150,000 per year of life gained was an affordable sum. It’s never been clear to me where these numbers came from but in the cost efficacy literature they seem to be the most used figures.

In our hypothetical situation our outcome was no progression of disease during the treatment period. But for the sake of our discussion lets say the study continues and the study patients (the 15% with benefit) lived a median of 2,3,4,5, or 6 months longer. For what it’s worth except for the very rare super effective new agent (Gleevec for example) most new biological agent’s improvement of survival is in the 2-6 month range.

If the median improvement is 2 months then the cost of a 12 month survival would be 6 X $666,666 (the cost/patient who benefitted) or about $4,000,000 to buy a total of a year’s survival time for patients who benefit from the treatment.

If the improvement is 6 months then the cost of 12 months survival would be 2 X $666,666 or in the range of 1.33 million dollars.

Neither would be considered cost effective by any of our current measures.

Medicynical Note: You won’t find the drug companies funding cost efficacy studies nor touting their results as cost efficient. Instead our hypothetical study would be touted as showing a 60% improvement in response rates between placebo and study group. (40% response rate with drug/25% with placebo or conventional Rx X 100=160%) It won’t be easy to find out that the difference between a “response” to placebo and the study drug was 15% (40% vs 25%) or that the benefit was just 2-6 months, if that. That the American way of drug marketing.

In case you think the above is exaggerated consider these from real life NEJM 355:2542-2550

The median survival was 12.3 months in the group assigned to chemotherapy plus bevacizumab, as compared with 10.3 months in the chemotherapy-alone group (hazard ratio for death, 0.79; P=0.003). The median progression-free survival in the two groups was 6.2 and 4.5 months, respectively (hazard ratio for disease progression, 0.66; P<0.001), with corresponding response rates of 35% and 15% (P<0.001). Rates of clinically significant bleeding were 4.4% and 0.7%, respectively (P<0.001). There were 15 treatment-related deaths in the chemotherapy-plus-bevacizumab group, including 5 from pulmonary hemorrhage.

Or this from today’s (April 25, 2010) Seattle Times:

Dendreon’s case rests largely on a study of more than 500 men with an advanced form of prostate cancer that spread to other parts of their bodies. Of the men who got Provenge, nearly a third were still alive in three years, compared with less than a quarter of those who got placebos. (medicynical emphasis) The vaccine boosted median survival time by 4 months, from 22 months in the placebo group to 26 months in the Provenge group.

How much difference is there between nearly a third and less than a quarter? I wonder who provided this verbiage? I figure an 8% benefit. Affordable?

Elective surgery, particularly when it has a cosmetic or convenience rationale, should be completely safe and effective.

It now appears that LASIK (laser assisted in situ keratomileusis) has problems and the industry is being criticized for not be fully honest about risks. This in Salon notes:

Last spring, the FDA inspected about 50 Lasik facilities and found that many had no system in place for collecting and transmitting data to the FDA on patients’ reports of post-surgical “adverse events.”

And in August, Consumer Reports Health released the results of a survey, which found that 55 percent of Americans who’ve had laser vision correction surgeries are still wearing glasses or contacts some of the time. Fifty-three percent experienced at least one side effect within the first four weeks of the surgery; 22 percent of patients experienced them six months after surgery, especially dry eyes, halos, glare and starbursts around lights.

Lasik surgery, which can cost up to $5,000, has a 95.4 percent patient satisfaction rate, based on an analysis of research worldwide from 1996 to 2008,

Medicynical note; There are two problems here. The first is the push for revenue by LASIK providers. The more they do, the more they make. With such incentives providers tend to minimize risk and extend their indications for the procedure. The second is the patient wanting (for some reason desperately wanting) surgery assuming they have no to minimal risk or that the other person is more likely to get the complications.

This powerful drive for medical services and the tendency to ignor risk (and cost) is part of our system wide problem.

That’s a deceptive title because when talking about a “free market” for products, there are many things “different” about the health care product.

I touched on a few in yesterday’s entry.

Today I’d like to explore the notion of effectiveness. In my specialty medical oncology, the only way we gauge effectiveness is through large studies of patients with the problem and comparing the outcomes (length of survival, rate of recurrence, etc). In any given patient it’s often difficult, if not impossible, to ascertain whether the treatment has actually worked or not.

For example, consider the patient with breast cancer who has had surgery with the tumor completely removed. If nothing else is done we will know in time whether the surgery cured the patient or not.

But, we often treat these patients with some type hormonal blocker (tamoxifen or another) with or without chemotherapy (called adjuvant therapy in doc talk). This can get quite expensive and is associated with frequent severe side effects.

If treated in this way and the tumor doesn’t recur, in any individual case we cannot know whether the surgery removed it all or the ensuing hormone blocking and/or chemo had some effect. Furthermore, if the tumor does recur later we are unable to determine whether the treatments delayed the recurrence.

The same is true for all other cancer types. In an individual case we simply cannot know whether the therapy given was worth the pain, cost and side-effects, unless large comparison studies of treatments and their outcomes are done.

You can of course say well, if it doesn’t return who cares. But given that tumors often recur and that there are extreme side-effects and greatly decreased quality of life from treatment and yes extreme costs, it’s important to understand whether a therapy works and just how effective it is.

Now to the difference between health care and other products. We spend literally thousands of dollars on treatments for cancer. Single drugs can cost as much as $100,000/year–that’s more than the great majority of people earn in year. In most instances, in cases of advanced disease, the improvement in survival from these agents (take Senator Kennedy’s case for example) is limited, measured in days to a few months. We, our health care non-system spend literally hundreds of billions of dollars for treatment of just these cases.

Can you name another product in this cost range, bought in the free market, that may not work? or may work for only a few days or months? or if it does work costs hundreds of thousands of dollars?

Well, that’s the situation in health care. Our costs are untenable; Our outcomes difficult to measure; and the purveyors of these products don’t want to do comparison studies to figure out what works and how it actually does work.

We do need reform!

Fascinating article in The New Yorker (June 1, 2009) on the variations in health care costs. Atul Gawande visits one of the the most expensive Health Care Markets, McAllen Texas, and looks at the reasons.

No surprises here, but nice documentation and explanation of the issues.

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Medicine’s costs confound economists. Classical economic theory (I’m not an economist, correct me if I’m misinterpreting) would have you believe that as supply increases demand is met and prices stabilize. In health care as supply increases, demand increases and prices increase. Health care is simply not a reasoned rational system.

Consider Miami: (Time, May 20,2009)

“the 2008 average private-provider costs for a Miami family of four – $20,282 – as the highest among the 14 major U.S. cities it studied, adding that more than 40% of that amount came out of Miamians’ own pockets.”

“Miami’s inordinate health-care outlay – 20% more than the national average – “is not a pretty picture,””

“That’s especially true since Miami-Dade County also has one of the country’s lowest median incomes ($43,495).”

“”South Florida has an “excess capacity of health-care providers and institutions,” Quick notes. And to make sure they all get a piece of the action, they’ve created a wasteful and ill-coordinated system of health-care redundancies, from unnecessary MRIs to inpatient treatment that too often could have been cheaper outpatient treatment. Miami-Dade, for example, has one of the nation’s highest hospital readmission rates – and more MRI machines than Canada.” (Medicynical emphasis)

Medicynical Note: For-profit medicine is simply for profit. In Miami, efficiency and even the patient’s welfare comes second. Can we learn from Miami?

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Gifts from drug companies to doctors continue despite reports to the contrary. Companies claim that such gifts are ethical and necessary. The NY Times notes:

“Drug companies spend billions (medicynical emphasis) of dollars wooing doctors – more than they spend on research or consumer advertising. Much of this money is spent on giving doctors free drug samples, free food, free medical refresher courses and payments for marketing lectures. The institute’s report recommends that nearly all of these efforts end.”

Imagine the cost savings to consumers if drug companies spent less on marketing. These companies claim it takes $800,000,000 to bring a new drug to market. How much of this inflated figure is gifts to doctors aimed an influencing their treatment decisions? If we presume the NY Times is correct and more is spent on gifts and advertising then research then the real cost of new drugs is significantly less than companies claim. Guess who ultimately pays for all this?

With drug prices increasing faster than any other part of health care expenditures is it too much to ask, as the IOM (Institute of Medicine) does, that companies and docs forgo this unethical practice?

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