Top health officials will visit Congress Tuesday to share promises and challenges of the cutting-edge — but little-understood — field of precision medicine.
Viewed as the next big frontier for curing diseases, precision medicine has received much attention lately from policymakers including President Obama, who asked Congress earlier this year to put hundreds of millions of dollars into its research.
The approach involves targeted cures based on a patient’s genetic makeup, meaning, for example, that two people with the same variety of cancer would get different, but more effective, treatments.
It’s a field that excites medical researchers, who hope it could open the door to better treatment and cures not just for cancer but also for other serious illnesses such as diabetes or cystic fibrosis. Some patients with certain cancers are already undergoing molecular testing to determine which drug they should be given.
Yet many barriers remain before the precision medicine approach can be applied widely — and members of Congress are trying to highlight those challenges with a Tuesday hearing on the subject, where National Institutes of Health Director Francis Collins will testify, with two other top health officials.
Here are some of the precision medicine challenges likely to be addressed:
1. Genetic data needs to be gathered from lots of patients.
It used to be that doctors could study the effects of a cure on a smaller cohort. But each person has the possibility for about 3 million genetic variants, which means precision medicine researchers must study huge populations to gather any meaningful information.
The president has proposed creating a database with the genetic information of at least one million individuals, which presents its own logistical challenge.
“Biomedical research requires thousands or tens of thousands of patients,” said Richard Weinshilboum, chairman of the clinical pharmacology at the Mayo Clinic College of Medicine. “We can’t run medical research like a mom and pop store.”
2. It’s hard to organize all this genetic information into any usable format.
The huge pool of genetic variations means researchers must collect and store a huge amount of data to discover the connections between different variations and treatments.
And that requires better use of electronic health records, which many providers have struggled with incorporating into their practices. More doctors are using them, but progress has been slow.
“It’s a question of being able to have the systems to put it all together,” said Marc Grodman, chief executive of New Jersey-based BioReference Labs. “That’s something we don’t really have right now.”
Physicians, scientists and researchers use the term “interoperability” to describe the need to easily transmit genetic and clinical information while still protecting patient privacy. Without usable electronic records, they won’t be able to propel precision medicine forward.
3. Targeted therapies can be really expensive.
The cost of genetic sequencing has dropped dramatically. It now costs about $1,000 to sequence a human genome, compared with about $100 million just 15 years ago.
But it’s likely to be expensive to actually develop drugs that target a person’s genetic or molecular characteristics. Further augmenting the issue, such targeted therapies will help a smaller segment of the population than more traditional ones, leading to a high per-patient cost.
Whether insurance companies would be willing to pay for such expensive targeted therapies is also an outstanding question. Yet experts say targeted therapies are what’s needed to move medicine away from “one-size-fits-all” approaches and into the next phase of innovation.
“This revolution needs a lot of care because it’s not based on one size fits all, but on the realization that we are all different,” Grodman said. “We need the funding, the cooperation … to allow us to continue the work.”
