Random Thoughts from a Restless Mind

Dr. Darrell White's Personal Blog

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Is Truly Better Care Truly Worth It?

A recent study looked at survival statistics for colon cancer. While >90% of patients with early stages of colon cancer survive at least 5 years, the 5 year survival rate for advanced cancer is only 12%. One of the conclusions from that study is that the increased survival rate of cancer detected early justifies wider scale screening for this deadly disease. Makes sense, right? Find the cancer early and you live longer. The problem here, of course, is that the incidence of colon cancer in a broadly chosen group of individuals is actually rather small making both the cost of making the diagnosis as well as the “cost” of any complications from the screening test prohibitive. Whether or not to screen is a very complex question. This is a version of “better care” but it is probably not worth the cost unless we can find some way to identify an at-risk population.

Let’s say there’s a brand new test that allows you to identify another, different, important disease years, perhaps decades earlier than is now feasible. It is possible to narrowly define a population of patients at high risk of having the disease. Utilizing this test will provide physicians the opportunity to treat this disease before it causes irreversible destruction of vital organs, destruction which renders present day care of this disease when it is presently diagnosed little more than palliation.  The effects of this disease in later stages cause dramatic negative changes in the lifestyle of affected patients. There appear to be ways to treat the disease in its earliest stages in order to prevent the ravages of its late stages. Do you initiate testing in the at-risk population?

Let’s think about it.

What if the test is relatively easy, relatively painless, but relatively pricey? Remember, you can identify a population for this test that is at risk to have the disease based on patient history and physical exam criteria, making the percentage of people afflicted (incidence of disease) relatively high in the tested group. The weakness of many screening programs (routine colonoscopy for colon cancer for example) as noted above is that the disease is either relatively uncommon, or it is impossible to identify a truly at-risk group. This is not a problem with our hypothetical test. Let’s make the test very accurate, too, say 95% sensitive and 95% specific in any study group; the number of both false positives and false negatives would be very low. This would make it more useful than serum lipids when screening for cardiac risk, for example. While we’re at it, this test is extremely safe with no real risks. It sounds like a pretty strong case to roll it out, right?

Now would be a really good time to go back to one of the earliest things every medical student is (was?) taught about ordering a test: the result of your test should in some way influence your care of the patient. A positive test result should prompt you to do something differently from a negative result. If your course of action would be identical with each/every possible result it is entirely reasonable to ask why you are doing the test in the first place. Our new test meets this threshold. A negative result means continue with “standard operating procedure”, treating a patient with the constellation of symptoms and signs you have identified as you have been. A positive test, on the other hand, obligates you to enlist the assistance of another specialist, and furthermore to insist that your patient receive treatment that is not yet in any way considered standard. Therein lie the problems.

Many, if not most complex medical problems require the engagement of a specialist in order that the patient receive the most up to date and effective  treatment. Contrary to the popular notion that we only have a shortage of Primary Care doctors in the U.S., every family doc will tell you that it is nearly impossible to find a rheumatologist, dermatologist, neurologist, endocrinologist or various other medical specialists to take care of a new, complex patient. Imagine if you have a new test that identifies 1 million or 2 million or MORE new patients whose disease requires one of these specialists to run the show? What if the effective treatment that will be proposed is off-label (FDA approved for something else) and extremely expensive? What now? Remember, failure to identify these patients early and treat them before irreversible damage occurs dooms them to progressive misery as they age. What’s the call?

This would be truly better care. Is it truly worth it? Who should make the call?

In the abstract it’s a different question, and without the context of knowing what disease is involved also makes it somewhat more difficult to analyze this in any meaningful way. Let’s face it, some diseases simply carry more emotional weight than others, and this would likely increase the amount of money that anyone faced with this question would be willing to spend. The question itself is hardly abstract at all, however. Just today I read about tests for Alzheimer’s Disease and Sjögren’s Syndrome that will allow for extremely accurate diagnoses made dramatically earlier than we are now capable. Unaware of the diagnosis physicians and patients have no opportunity to consider treatment; if you can’t take a temperature you can’t find a fever. Without the ability to make an early diagnosis pharmaceutical companies have no incentive to evaluate treatments that will prevent the scourge of late-stage disease. This is the situation on the ground today.

The challenge will come as these tests are put into routine use and we identify large numbers of patients whose eventual course in the absence of treatment is well-known. Who will care for them if we already face a shortage of specialists in these fields? Uncertain of a return on the investment necessary to prove the efficacy of treatments for these diseases, what pharmaceutical company will do the studies that will show the benefit of treatment? With these new treatments likely to be very expensive, will insurance companies and the government pay the costs of the care? In the abstract these tests and the treatments that will follow certainly constitute better care. Will it be worth the cost?

Who makes that call?

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