Medical Device Daily Washington Editor

WASHINGTON – The impetus behind personalized medicine (PM) may well qualify as the latest medical stampede, but don’t tell that to the members of the Personalized Medicine Coalition (PMC; Washington). While hopes are high in some quarters for PM, its proponents are said to be wary of outlandish claims that could provoke a serious drubbing in the court of public opinion.

Still, at the organization’s luncheon last Friday at the National Press Club (Washington) – titled “The Case for Personalized Medicine” – the keynote speaker dangled the case of Singapore to provoke hope for healthcare reform, noting that the island nation of less than 5 million devotes less than 4% of its GDP to healthcare.

In his introductory remarks, Edward Abrahams, PhD, executive director of PMC, said the coalition, launched three years ago, now boasts more than 120 members, up sharply from the original 15.

Abrahams said that PMC members “believe that by focusing on the science ... we can fashion a new paradigm that moves from discovery to development to delivery,” and he listed three prerequisites to moving PM into the mainstream of medical care.

“First, we must develop and showcase the economic model” for PM, one that will work for the drug and device industries and for payers. Second on the list is that modern medicine must “develop reasonable pathways to develop acceptable clinical evidence” in support of PM. And the third is that given that most doctors were trained before the genome was mapped, the drive toward PM must include educational outreach.

Abrahams also noted that the journal Personalized Medicine is in its fourth year of publication.

The guest speaker, Harvey Fineberg, MD, president of the Institute of Medicine (IOM; Washington), said “among the motives for personalized medicine is the performance of healthcare” in terms of quality and outcomes. He said the U.S. “no longer ranks in the very top tier” in measures of life expectancy, and in fact, the U.S. no longer places “even in the top 20.”

“We have the most costly healthcare system in the world,” Fineberg said, lampooning U.S. healthcare as “a little like that football prospect who might not be big, but he’s slow.”

Fineberg described the case of Singapore, “a very small country of about 4.5 million people” that was “a generation ago, a very poor country.” He said Singapore has moved “from eight years behind the U.S. in life expectancy at birth” to a place where life expectancy “is now three years longer.”

“They lavish about 3.8% of their GDP” on healthcare, he quipped, adding that per-capita income in Singapore “is very similar to that in the U.S.” The role of PM, he said, “is to help redress this imbalance.

“Let’s be clear: the Singapores in this world are not ahead of the U.S. because they’ve used molecular biomarkers” more cleverly or because they’ve invented new molecular entities. “They’ve got where they are ... because they’ve used what we’ve learned” better than we have used those things.

Without overtly acknowledging that part of the reason healthcare is so costly in the U.S. is that the U.S. bears the cost of being the world’s primary biomedical research lab, Fineberg said that many observers will question “whether it is possible to have both an innovative health system” and “a system that delivers all the basics.”

“Not only will the two be compatible, they will be interdependent,” he answered.

One of the stumbling blocks is that “[w]e are still relying on a paradigm of diagnosis” that came into being 200 years ago, Fineberg said. “The premise of PM is that the old paradigm is no longer sufficient.”

Fineberg said biomarkers and genomic predictors that are essential to PM will help assess the patient’s disease risk, enable diagnoses, clarify prognoses, and identify the risks associated with available treatments.

“But fundamentally, this is not a difference in kind, but one of degree,” Fineberg said. He mentioned a recent article that evaluated the use of the AIDS drug Abacovir in clinical practice to determine whether patient foreknowledge of their susceptibility to the drug’s side effects would influence their decision to take the drug. He said most people would deal with the side effects in the case of a deadly disease, but he pointed out that patients who were worried about the side effects – but who turned out not to be genetically susceptible to those side effects – were relieved of any worries.

Fineberg exhibited an awareness of the backlash on drug-eluting stents in addressing any appetites for PM hyperbole. “I think it is very important to under-promise, under-hype and over-deliver” in order to keep expectations from blowing past reality, he said. “The risk is that when you look at the enormity of the challenges of healthcare,” it is easy to promise too much.

“Paradoxically, we need a stronger FDA ... to enhance the speed and effectiveness of personalized medicine,” Fineberg said, adding “we will never reach the point at which the agency will be able to deal with” the variety of therapeutic products “if it fails to have the resources and quality of personnel to undertake those assessments.”

FDA’s vigor, he said, is “crucial if this is to succeed.” However, Fineberg also argued for payment reform, stating that we as a society must “transform our reimbursement system into a value-based system” not units of service.

“CMS needs very different authorities” and should be able to be “much more aggressive” in innovating payment policy, he said.

On the question of the additional cost to make PM the operative paradigm, Fineberg told Medical Device Daily that to sell the idea, the best proof of the concept will be “evidence of actual success” with demonstration projects. “Then we have the case and can argue this deserves funding.”

“I think we’re past the point where we can ask the public and policymakers to take on faith that what we say is going to happen will happen,” Fineberg said.