Two cheers for PCORI

In the mid-2000s, when Pharmaceutical Commerce was founded, the editors and writers spent a lot of time talking about how state-of-the-art computing and data analysis would revolutionize medicine. The thought was that as more patient data was collected and analyzed faster, clear conclusions would be drawn about what therapies work best for which patients. There was also an implication that this could be big trouble for some pharma companies, whose products might be found to
be clearly less effective than others.

I’m perfectly comfortable with calling this exercise the arrogance of ignorance. If nothing else has been learned in the intervening years, it’s that getting a clear “signal” (to use a data-analytics term) from patient data is enormously challenging. The number of confounding factors can be as vast as the biology and chemistry of the human organism.

When the Affordable Care Act was passed, enormous attention was paid to the health insurance financing aspects (as it has been to this day). Less noticed was the founding of the Patient Centered Outcomes Research Institute (PCORI), a federal program funded by appropriations and fees from insurance plans. The main focus of PCORI was to be comparative effectiveness research (CER). Funds have been distributed to a wide variety of researchers in studies; there has also been the establishment of PCORnet, a “network of networks” that brings together health records at many research centers.

In its early days, there was some criticism of PCORI that it could, in some fashion, come between physicians and their patients, in effect saying that certain treatments shouldn’t be carried out because of a lack of benefit. To be crude, this criticism sounded like a good thing—if a treatment is not effective, public and private dollars shouldn’t be spent on it. However, the pattern was set early on that the CER studies PCORI funded would be long-term, and tended to be either “soft science” (having to do with how to communicate risks and benefits to patients), or foundational—how to analyze for effectiveness, rather than how to make determinations of actual effectiveness of specific treatments.

Still, some $2.4 billion has been spent by PCORI, and the question can be asked, are US taxpayers getting their value? I would say, sort of; but I wish that the promise of CER could be fulfilled more directly. PCORI’s own fact sheet (available at its website) concludes that its work has “generated particularly promising evidence for improving care and patient outcomes”—how’s that for a ringing endorsement?

So, two cheers for PCORI. Its original funding was for 2010-2020, and there is legislation to renew it for the next 10 years. Let’s hope that the foundational work done in the first decade will shine in the next.