Monday, April 9, 2012

Five ways to tame the risk-benefit-uncertainty troika

There was a story on NPR this morning that sent me in a radical direction. It discussed the increase in use of brachytherapy for localized breast cancer. The idea is that this is a concentrated dose delivered much more locally and rapidly (over 5 days) than the conventional external beam radiation (over 6 weeks). The issue is that it has not been tested rigorously in a randomized controlled trial yet, and some oncologists are concerned about its outcomes as they compare to the conventional approach. One of the concerns stems from an increase in the rates of subsequent mastectomies, which are double in brachytherapy relative to conventional. At the same time, there are clear benefits, not the least of which is the period of exposure and the hassle associated with daily trips for radiation for 6 weeks.

Several points popped up in my head in response to the story:
1. When discussing this predominantly women's disease, the expert voices mostly heard from were male (5 of the 6 doctors quoted). Does this matter? Not sure.
2. The priorities addressed by the experts were the traditional outcomes -- survival, recurrence, metastasis. The priorities described by the patient were about her time and convenience today.
3. The concern about the procedure stems from the observed doubling of the need for mastectomy within 5 years among patients treated with brachytherapy, an event "rare no matter what what kind of radiation women got."

So what's my point? Do I think that more rigorous testing is not indicated? Not at all; we need a more rigorous evaluation of the technique. No, what I am wondering is at what point should a procedure like this (or any intervention, for that matter) become available to patients as an option to be considered? Should its availability be determined in a dark room by bespectacled men around a conference table, or should it be put on the menu of choices, along with its risks, benefits and uncertainties, as soon as it looks safe enough, whatever that looks like?

The larger question this raises is what is the degree of uncertainty that we are willing to accept around interventions that become available, be it a drug or a procedure or a device? How do we incorporate patients' priorities for outcomes that are important to them into these decisions? Remember ACT-UP and how they moved the FDA to make more rapid decisions about treatments for HIV/AIDS? Have we swung too far in the opposite direction today, whereby we want a virtual guarantee of safety before a technology is approved?

I offer these 5 potential question to help with making these decisions:
1. How severe/deadly is the disease in question?
2. What is (are) the known potential benefit(s) of the intervention?
3. What is (are) the known potential risk(s) of the intervention?
4. What uncertainties bracket this risk-benefit equation?
5. How does the patient feel about the extent of this risk-benefit-uncertainty balance in the context of her condition?

I think that the first four are the questions that the FDA struggles with every day. They are the gatekeepers for the availability of new technologies, and, therefore, for the relevance of the fifth question. What I am wondering is whether it is not better to start bringing a lot more of the public perspective to the discussion much earlier, so that the patient can have the option of evaluating more choices sooner. I know I may be treading on thin ice here, but I am ignoring any market forces or special interests for the moment. The question I am asking is "In the best of all possible worlds, where no one is trying to sell you anything, when is the best time to give the patient an opportunity to accept or reject an intervention, given the risk-benefit-uncertainty profile?"

Bottom line: There are no guarantees. Just because something is available on the market does not mean that it is completely safe or completely effective. Most importantly, it does not mean that we come even close to being certain about these attributes. As a corollary, just because there are uncertainties about the risks and benefits of an intervention, does it mean that it should not be available as an option for a patient? My guess is that there is a balance of this troika of properties that may be optimal on average, but I am also guessing that that this average balance will miss a substantial volume of outliers. Just as some people thrive on the thrill of bungee jumping while others clamp down just at the mere thought of it, so some patients may surprise us with their position on this risk-benefit-uncertainty continuum.

I apologize if my argument is not clear -- I am definitely thinking about this stuff actively. The one thing I am absolutely sure of is this: Unless the public and clinicians are educated about how to have these conversations, we will always have to rely on and, consequently, blame someone else for making decisions for us.

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