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What Does it Mean When We Say a Treatment “Works?”

flying cadeuciiMaking a decision requires you to compare tests/treatments that have been contrasted in researh studies to see if one over another results in improved chances of good outcomes. In a sense, medical decision making is a competition. To assess the competition, you compare the chances of outcomes, or results from groups of people taking different options. The comparison is a simple subtraction in the amounts of outcomes that occur in each studied group.

Subtracting results in a difference that is either a benefit (if better for you) or a harm (if worse for you). For nearly all decisions, however, the test/treatment that is better for disease outcomes (benefit) is worse for complications (harm). Comparing, then, results in the following possibilities:

The chances of outcomes associated with the condition you have and the tests/treatments available will be the same for all options. In this case, chose the cheapest option.

The chance of outcomes associated with the condition you have will be less with one option. That option provides added benefit

The chance of a complication caused by the test/treatment that adds benefit for the disease outcomes will be greater (harm).

Since the test/treatment that is better for you in terms of the disease you have will be, simultaneously, worse for you in terms of complications caused by that test/treatment, a trade-off of benefit and harm is required.

Hence, the definition of “works” is that:

A test/treatment works when you feel there is more to gain from the greater chance of better disease associated outcomes than there would be to lose from suffering the complications caused by your chosen treatment.

So, medical-decision-making is a competition between options and there is always some good to be balanced against some bad.

The balance of good and bad from your perspective is what makes one treatment work over another.

Robert McNutt, MD is a board certified internist in Clarendon Hills, Illinois. He is a Professor at Rush Medical College of Rush University.

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  1. Dear Dr Palmer: I am sorry I did not see your comment. I did not even know this was posted. I think we are at our best when we inform patients and then we help them decide on their own. I just had a patient who decided against surgery for prostate cancer because of his wife’s concerns for side-effects. When people weigh and balance harm and benefit, they are responsible to themselves and those that share their lives. It seems to me that the way we stay responsible to all is making sure that people know their data and then can figure out what it means to them. Perhaps this is too simple of an idea, but the secret to better medicine is allowing patients the right to decide. Medical care is produced via decisions and perhaps patients are better at being responsible decision makers. I have a book coming out soon; Your health; Your decisions. I hope you get a chance to read it and critique. I always learn something from your comments. Thanks. Bob

  2. This actually becomes complicated if you keep thinking: You could have a drug that works in every one of your criteria above but leaves some epigenetic methyl or acetyl groups on your germ cell DNA or nucleosomes that could affect your offspring in a harmful way. Or, more elementary, you could find that a drug works fine, increases your qalys, makes you feel better, etc. but harms your wife’s well being because it lowers your libido or exacerbates her asthma. A question: does a doc have a duty to people other than his patients?

    Overall, however, a fine article.