The surprisingly small benefit of some very (expensive) Big Ideas

Last week, JAMA published online a Viewpoint provocatively titled, “What Happens When Underperforming Big Ideas in Research Become Entrenched?” The overarching Big Idea to which the article refers is the “narrative positing that a combination of ever-deeper knowledge of subcellular biology, especially genetics, coupled with information technology will lead to transformative improvements in health care and human health.”

The article highlights three technologies that are integral to the Big Idea but that have not lived up to their promise. The first is genetics/genomics; as an example of unfulfilled promise, the authors trenchantly observe, “Sixty years after the discovery of the genetic defect, no targeted therapy has emerged for sickle cell anemia” — one of the simplest genetic diseases, caused by a single gene. The second is stem-cell therapies; the authors point out one analysis of studies of stem cell therapies, in which the supposed effectiveness of the therapy was directly proportional to the number of factual discrepancies in the published study. The third is electronic health records (EHRs), which have cost billions, but have not realized either the...