Monday, September 22, 2008

Magical Thinking

Margaret Anderson, COO, FasterCures
I appreciated the message of Carol Diamond and Clay Shirky’s recent piece in the August 2008 Health Affairs titled “Health Information Technology: A Few Years of Magical Thinking?” In it they say that “proponents of health IT must resist “magical thinking,” such as the notion that isolated work on technology will transform our broken system.” It’s interesting to think about systems change at the front end, and how easy it is to get stars in our eyes about how things like health IT or personalized medicine will transform the world as we know it, and how all of our problems will then magically go away.

I was speaking recently to a colleague here Kristin Schneeman about her life working in Vice President Gore’s office in the early days of the Clinton Administration and how new and novel email was at that time. I recall getting my first work email account and not having anyone to really email anything to, except my colleagues on the same floor. Greg Simon, President of FasterCures has talked about how he had a cell phone in the early days and was embarrassed to be talking on the street with it so he’d take it into a pay phone booth to look more “normal.”

Cell phones, email, and the Internet have certainly transformed things in ways we couldn’t have imagined, but they’ve introduced problems we couldn’t have imagined. Technologies such as FAX machines have been leapfrogged over. Problems such as the overabundance of information, and the speed of information flow are here to stay it seems. In the case of health IT, FasterCures sees it as a vital bridge to the future of more rapid information collection, characterization, and analysis which could speed our time to cures.

We are working on a white paper for the U.S. Department of Health and Human Services about educating and building awareness among consumers about personalized healthcare. This is another area where we must resist “magical thinking” and get down to brass tacks. Too often, the discussion about personalized medicine has been at a 30,000 foot level. For this paper, we’ve talked to many patient advocacy and disease research groups and everyone holds their breath about the potential power that these technologies may hold for their disease areas. They all want more targeted therapies with fewer side effects, which is ultimately the promise of personalized medicine. But they also recognize its complexities. It needs to take into account the world of co-morbidities we all live in; even if baby boomers are out running marathons and eating their greens and blueberries, the reality is that many of us are living with many conditions and diseases, not just one. It will probably raise costs before it can lower them. It's unlikely many diseases will yield to the relatively easy HER2-Herceptin gene-to-drug relationship. Patients are likely to get much more information about their genetic makeup than they can act on in the near-term. So, with all these complexities, is it magical thinking to ponder how scientific advancements in genomics and proteomics may change things for the better? Is it magical thinking to imagine a health IT system that can propel these advancements forward even faster? Or can we make it real? You tell me.


Steven Walker said...

Of course we can do it, but not just by talking about it, which is what we have so far gotten from Critical Path, for example. Change in applied science comes at the nuts and bolts level.

How are we going to run clinical trials of "one?" Personalized medicine requires it, and we could already be doing it, using the patient's baseline molecular and macro characteristics as the control, and then observing very closely (through sequential sampling and characterization of the patient's affected and unaffected tissues and detailed monitoring of clinical outcomes) what happens as we treat the patient, then adjusting to changes in the patient as they occur. This would also be a lot closer to the ethical practice of medicine than what we are doing now, and would be more attractive to patients.

In fact, it would be more scientific, more ethical and more effective than what we do now (e.g., forcing dying patients into blinded, no cross-over, placebo-only control arms so they will die on the schedule of an untreated patient to provide a column of "survival" data to FDA statisticians).

We would of course have to conduct many clinical trials of "one" to learn what we need to know about variability. Pooling the results into populations for statistical analysis after characterizing the cause and effect relationships of drug/disease interaction would then be of very little use in making progress, because it would be applicable only to some calculated "average" patient - which we would know did not exist from the indivdualized data we collected - or we would know the population did exist - based on the cause and effect observations - also rendering the statistics meaningless.

We can move to the pursuit of first-principle science because we now have the technology to do it. But first we have to get past the seeming unshakeable belief by the clinical research and regulatory communities that only outcomes in "populations" matter. The science is telling us, in fact screaming at us, that population-based medicine is wrong for many of the diseases that still kill us. The right direction, however, treads perilously close to the statistically-disdained world of "anecdotal" observations. Nonetheless, that is where we have to go, because the populations we have been relying on for 50 years simply don't exist at the molecular level.

We actually don't have a science problem. We can do the science. We have a cultural problem. Our clinical researchers and regulators don't believe in first-principal science - they believe only in statistics derived from population-based outcomes.


The real challenge is breaking the cultural resistance to change that permeates medical/clinical research and government regulation of that research.

A good place to start would be excising the term "well-controlled" from the FD&C Act, which mandates that all clinical research must be designed to produce a statistical result. What other field of science has managed to move forward using that comnvention? The answer is - none.

Imagine Congress mandating in any other field of science (as they have in clinical research) that there is only one way to design an experiment, and that way is to call a statistician?

Let's let the scientists do some science for a change. Science moves forward by itself if we give it enough room to move. The current crop of status quo defenders sense the truth in that, and their efforts to make sure that our system doesn't change in any fundamental way is a reflection, in part, of that sense. No one wants to be made obsolete, but that is the inevitable effect of progress. Keep up, or become irrelevant. But when the powerful institutions responsible for fostering change become the opponents of change (the current situation), progress can't happen.

The answer to this question posed in this article is quite simple. We need to get out of our own way.

asnet said...

how does "personalized" medicine meet the requirements of "evidence-based" medicine that are a precondition for coverage by insurers -- both private and public?