Innovation and Quality: How Long Should We Wait?

At a Council for American Medical Innovation meeting held earlier this week in Washington, Marc Boutin, executive vice president and chief operating officer of the National Health Council (NHC), recalled how his father died of lung cancer several years ago.

"He spent a year-and-a-half on chemotherapy treatment that did absolutely nothing for him except diminish the quality of his life. We only learned after he died that just 10% of the population responds to that treatment—and yet almost everybody with lung cancer gets that same chemotherapy regime," Boutin said. "If we had known that, I'm not sure that we would have continued through with that treatment."

But changes are afoot for the millions with chronic diseases and disabilities, including those represented by the NHC, in the form of biomarkers, which can be used to diagnose disease risks, assess the presence of disease in an individual, or tailor treatments for the disease in an individual—using drug treatments or administration regimes.

Citing a recent report by PriceWaterhouse Coopers (PWC) on "Moving Toward Personalized Medicine," Boutin said that "we expect that almost all specialty products will have a companion biomarker diagnostic."

This means that it will become easier to tell who "a product will work for and who it will not work for, and we'll be able to pinpoint the treatment right to the person," Boutin said. The big question, though, is: How long will it take?

In the PWC report, the researchers looked at the year 2020 as when biomarkers will be readily available and used. But to Boutin, that's 11 years too late—especially because much of the technology exists now.

Recently, the NHC did some research to look at what are some of the barriers to quicker adoption. He cited several that are contributing to the uncertainly on developing biomarkers:

• Very little guidance is available as to how a biomarker would be approved by the Food and Drug Administration as a diagnostic, Boutin said. "There is a lack of coordination between the approval arm of the FDA and the safety arm of the FDA."
• There are very low reimbursement rates for these groups. "We need to address how the Centers for Medicare and Medicaid Services (CMS) looks at reimbursement of diagnostics. And, we also have to look at how the private sector addresses reimbursement issues in this market," he said. "There are really not a lot of incentives when you start to look at the private sector to address this."

A number of types of biomarkers do exist. Some of them will indicate, for instance, "that if you take this medicine, you're going to have really bad adverse reactions," Boutin said. "That's a great biomarker to have. Other kinds of biomarkers actually tell you how who this will not work for.

"Quite honestly, that would have been very helpful in my father's situation. Had he known that chemotherapy would not work for him, he would have spent that last 18 months of his life not going through the trials and tribulations of chemotherapy," Boutin said.

"But the reality is that negatively impacts your business market. If you're going to determine who your product does not work for, you have less of a market share. So we have to look at some other incentives for industry," he said.

"We need to create a new business model. We need to figure out how we can develop trials...that give conditional approval to these kinds of biomarkers, with the subpopulations that have been identified where it works," he said.

"We need to speed up the development of these new diagnostic mechanisms and put them out into the market and test them," he said. "I can tell you from this perspective that people with chronic conditions . . . are much more willing to accept risk than we give them credit."

Many of these individual are "very interested" in having diagnostics developed—if not for themselves but for their children who may have the same conditions down the road, he said. "There are real opportunities here to create incentives to get these into the marketplace far quicker than 11 years from now."