Comparative Effectiveness Research Update: Greater Flexibility, More Outcomes

For an update on how the folks who are in charge of the $1.1 billion's worth of "comparative effectiveness research" (CER) are viewing the world, the Disease Management Care Blog checked out this just-published online Perspective piece in the latest New England Journal of Medicine. 

Study Subjects With Combinations of Conditions

While the focus will be on persons with chronic physical and mental health conditions, the real challenge for the "Patient-Centered Outcomes Research Institute" (PCORI) will be to find the right combinations of conditions (for example, persons with coronary artery disease with advanced heart failure plus depression) in sufficient numbers of individuals that will allow statistically valid inferences to be made. 

Alternative Research Methodologies

While PCORI would have preferred to use prospective randomized clinical trials to conduct their research, they must have been listening to critics like the Disease Management Care Blog.  There aren't enough patients, enough time or enough money within reach.  As a result, PCORI now recognizes that "observational studies" on heterogeneous patients outside the rarefied world of academia - despite the twin threats of confounding and bias - have to be included in the mix of funded study designs.  It turns out that it's possible if statistical controls are carefully and judiciously applied.  This will be considerably easier if large "registries" (or databases) are developed that contain the longitudinal outcomes data of large cohorts of patients.

What Outcomes?

The article also points out that untangling various outcomes involving multiple co-morbid disease states promises to be thorny, especially since treatments that help one condition (high blood pressure) may exacerbate another (low blood pressure and falls in a person with osteoporosis).  The answer will be to find outcomes that can be universally applied across multiple disease states such as "shortness of breath" or overall "quality of life."

The DMCB Reaction

"Hear hear!" says the DMCB.  This latest update indicates the folks in PCORI have recognized that good research methodologies shouldn't be the enemy of the perfect.  They have come to realize what the population health management community realized years ago: that it's possible to simultaneously provide care and intertwine robust research methods that simultaneously render insights about the population under management.

And... Uh Oh!

Unfortunately, the PCORI is widely viewed by supporters and critics as emblematic of "Obamacare" and there are threats by the ACA-hostile Republicans to defund it.  It remains to be seen how likely this is to succeed, but in the meantime the PCORI could help its cause by supporting the location of CER outside the usual insular academic institutions.  For example, it could be done in a commercial disease management setting, where huge amounts of data on millions with chronic conditions are already having their longitudinal data followed in huge terrabye servers (for example is here).