Tuesday, January 5, 2010

The FDA and drug costs: A health services researcher's epiphany

Happy New Year to everyone, and apologies for not posting for some time. You probably thought I was on vacation, but in fact a family illness has kept me from writing. For reasons of confidentiality, I will not be disclosing the details of it for the moment, but will share certain salient points this experience is clarifying for me.

Take expensive medications, for example. In the last few weeks, the anonymous cost-effectiveness equation of certain interventions has taken on a uniquely personal dimension. Thinking about using a prohibitively expensive medication with limited effectiveness in the abstract derives a much clearer answer than in the case of a loved one. What is a couple of extra months of life and functionality worth to you? And what if it is indeed more than a couple of months, since there are many individual exceptions to average values? Who am I to dissuade my family member from accepting this treatment course, even though I am keenly aware of its fiscal and clinical ramifications?

Here is what I am beginning to think. If even someone as close to health economics as I am cannot say "no", when put in the situation personally, to a therapy that I would academically consider cost-ineffective, how can a lay person faced with such a choice say "no"? If they cannot be expected to say "no", who should? In the spirit of patient empowerment I would certainly not advocate substituted judgment; that is, I would not advise the physician to make this decision unilaterally. And truly, although everyone is interested these days in having MDs take economic aspects into consideration, there is a potential conflict of this stance with the "do no harm" priority.

So, humanistically speaking, who should make this judgment? As you know, I do not believe that we can let the market drive these choices, unless down the road we are willing to spend 100% of our GDP on healthcare! This means that someone does have to say "no". Following the chain of development and marketing of technologies, since neither the MDs nor the patients (or their surrogates) are in a great position to do this, the decision must reside upstream. The most proximate upstream entity is the payor, but look at the political maelstrom that a discussion of limiting payment for existing interventions has precipitated in the US. The FDA is the next stop in this space-time continuum, second only to the manufacturer. This to me seems to be the logical final stop for the decision bus. Beyond the manufacturers themselves, who would be well-served not to invest untold dollars into bringing to market marginally effective exorbitantly priced technologies, the agency is probably the most sensible point in the pathway to close the door to products with questionable value. Today, the FDA is not empowered to consider the value proposition; this role is left to the payor. The FDA can only make judgments on efficacy and safety of technologies under evaluation. Could we change that? Would we want to? Is this the right solution?

In the meantime, we will do the trial of this expensive therapy and see what happens. Maybe it will buy more time than we think. Then again, maybe not.


  1. Marya, I am moved by your questions. I, too, am a health economist, and was faced with certain choices that had economic and clinical ramifications with the birth of my premature twins. Just to keep them alive, with only a 30% chance that they would have no long-term major disabilities was the issue. No parent should have to make a choice about whether their newborn infant should be allowed to live based on cost. But, that was my job before and it is my job now...but as a parent, it was abhorrent. Well, they are now 10 years old, with absolutely no health, physical, or emotional issues. They are 100% perfect. With hospital bills to the tune of about $2M per child, was it worth it? Yep.

  2. Patti, thanks so much for sharing your story. I am so happy for the twins! I have family that have been in the same position as you, and even with some disabilities the decisions were more than worth it!

    Forgive me, as, of course, the health economist in me went to the $$/QALY, which would work out to ~$25K/year, assuming a 78-year life expectancy and no detriment in quality (and this is without applying inflation or discounting). So, the choice is correct on all levels.

    This gets murkier on the other end of the life spectrum, though, don't you think? Anyhow, these decisions, so easy in the impersonal medium of peer-reviewed literature, are so much more fraught in real life...