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.

Voodoo Medicine

An excellent post the other day on the Science-Based Medicine blog addressed "Incorporating Placebos into Mainstream Medicine". In it the author, Harriet Hall, rails against complementary and alternative medicine as inherently leveraging the placebo effect. She goes on to ask "What if scientific medicine were to co-opt the CAM movement? We could take these treatments out of the hands of the less ethical practitioners and outright scam artists and place it in the hands of those who are more likely to be altruistic". This mouthful grabbed my attention.

Here is what bothered me. The traditional healthcare providers believe that they are practicing scientific medicine. And why wouldn't they? The clinical research establishment (of which I am a part, mind you) is constantly touting new breakthrough results, and the FDA after all only approves therapies that are proven to be effective! Well, not so fast; there are an awful lot of assumptions in this statement. First, how much of the research out there is of high quality and how much is bovine excrement? Next, even the best of studies that find statistical advantages to one course of action over another show minute, potentially inconsequential differences that a lot of the time translate into zero benefit outside the laboratory of clinical trials. And as for the FDA, they are paid by the manufacturers to review and approve drugs and devices. And even though I trust in their earnestness, most of the time they require only statistically significant differences (microscopic ones can still emerge given a large enough study size) in outcomes that are not all that meaningful to one's well-being (e.g., drop in cholesterol as a surrogate for a reduced risk of death from cardiovascular disease, a less straight-forward relationship than you might suspect).

So, there is the science bit. As for ethics, I will give Dr. Hall that for the most part MDs do try to practice what is commonly accepted as scientific medicine. The key here is "for the most part". Remember Gawande's story of McAllen, TX? And lo' and behold, just a few hours ago Reuters reported a bust of a large Medicare fraud scheme, where, believe it or not, docs were charging $3,000 to $4,000 for simple knee and shoulder braces and heating pads, calling them "arthritis kits". And while I do not question the ethics of the majority of my brethren, this incident sure underscores that, just as CAM practitioners, the house we live in is also made out of glass.

We have a long way to go to achieve good health in this country. Our culture has become over-reliant on experts in everything, including healthcare and evidence, to slap our wrists when we have been "bad" and to give us marginally useful advice on how to cure our ills. We must question our assumptions. I agree with Dr. Hall, nothing replaces a combination of evidence and experience. Or the placebo effect.