The grand promise of precision medicine can only be delivered if the science underpinning it is itself precise. As researchers Spencer Phillips Hey, PhD, and Aaron S. Kesselheim, MD, wrote in a recent article for STAT, there are still a number of factors standing in the way of precision medicine realizing its potential. Only when they're removed will healthcare be changed to a model that emphasizes preventive care and unique treatments for individual patients.
"But all too often the science underpinning these targeted therapies has not been up to snuff and the result has been greater uncertainty about optimal treatment — just the opposite of what precision medicine intends to do," the authors wrote.
The three primary barriers to "truly transformation precision medicine" are, according to the authors:
- Accurate biological theories. One component of delivering precision medicine is identifying what clinicians believe to be root causes of conditions or diseases, such as a particular gene or protein, and applying a treatment to modify it. But such theories about how human biology functions can be wrong. The authors point to the example of statin drugs, which have been widely prescribed for decades to prevent heart attacks. But recently, clinicians have called into question the mechanism that makes statins protective. Without a solid understanding of the underlying way a drug or treatment works, precision medicine solutions would be in effect, imprecise.
- Treatment uncertainty. When treating disease on the individual level, where genetics and other factors vary greatly between patients, the playing field is very different from conventional disease research, where a treatment is applied to groups of patients to determine its effectiveness. In precision medicine, clinicians are trying to influence individual biomarkers in individual patients, and it can be much less clear what effect treatments will have.
- New ground. Because so much of the groundwork being laid for precision medicine is new, there's little understanding of whether the results from different research teams are standard. What's more, these outcomes aren't actively being compared to one another. Without oversight and with some uncertainty about how treatments will ultimately impact patients, providers and companies developing treatments could end up offering solutions that in reality don't offer much help.
"The goal of precision medicine is admirable. But it will take longer to get there and cost far more than anticipated given the current research inefficiencies," the authors conclude. "Multiple research groups are exploring the same targets with little communication and oversight. Instead of leading to better, more reliable biomarker assays and treatments, these uncoordinated efforts can sow confusion and undermine interventions intended to enhance patients' health."