Comparative effectiveness research could threaten personalized medicine, NIH director warns

The federal government’s push to control health costs through comparative effectiveness research could threaten strides in personalized medicine, in which medicines are tailored to an individual’s genetic makeup, said the National Institutes of Health (NIH) chief at a recent forum sponsored by the American Association for the Advancement of Science.

Reuters reported that Francis Collins, MD, PhD, a genetics pioneer tapped by President Obama in July to head the NIH, said studies that lump together large groups of people to test the effectiveness of treatment A versus treatment B run the risk of overlooking clusters of people for whom a drug might have a dramatic effect.

“That’s going to get lost in the wash by considering everybody equivalent, which we know they are not,” said Dr. Collins, who helped lead the Human Genome Project that in 2003 produced a sequence of all DNA in people. Studies need to include genetic information that allows researchers to find such responses, he said.

Backers of comparative effectiveness research, who include insurers and large employers, see the government-funded studies as a way to learn which treatments work best. But Dr. Collins said, the studies should be well crafted.

“We need to be mindful of the goal of comparative effectiveness research and not lose all that we have gained in understanding how individuals differ and how that could be factored into better diagnostics and preventive strategies,” Collins said.

There is already evidence that personalized medicine can help reduce health costs, he added, pointing to Genomic Health’s (GHDX.O) Oncotype DX, a genetic test that can predict the recurrence of breast cancer.

He said the test costs $3,500, and most women who get tested and discover they are at low risk decide to forego chemotherapy, saving an average of $2,000 per patient in additional costs from chemotherapy treatment.

Margaret Hamburg, MD, commissioner of FDA, told the meeting that many clinical trials are structured to determine if a drug is safe and effective in a large group of patients, but the drugs often leave out the why – why certain patients benefit while others do not.

FDA increasingly is approving drugs with companion diagnostic tests using biomarkers – such as specific proteins or genes – that improve the odds that a new, high-cost biotechnology drug will work.

She said studies that look at the genetic profile of patients and its role in how drugs work could strengthen a drug’s application, lending more scientific certainty about why a new drug works.