Precision Medicine Initiative: Medical revolution or regulatory headache?

From the moment that President Barack Obama introduced his Precision Medicine Initiative in his State of the Union address, there's been a flurry of debate over the possibilities--and possible pitfalls--associated with using patients' genes to design targeted disease treatments.

The initiative also has reignited a debate in the insurance industry over whether to cover genomic sequencing, with Priority Health announcing this week that it will cover comprehensive genetic testing for patients diagnosed with aggressive forms of cancer, FierceHealthPayer reported.

But while much of the discussion has centered on the feasibility of carrying out such an ambitious initiative, others have begun to question how the government will regulate the whole process. Indeed, "the knotty problem is how to promote rapid innovation while ensuring safety and efficacy," writes Eric S. Lander, Ph.D., co-chair of Obama's Council of Advisors on Science and Technology, in a recent opinion piece published in the New England Journal of Medicine.

Lander expresses concern that some companies have already started to market genotype-based claims about patients' risk for of acquiring certain conditions or about which treatments will most benefit certain patients. Noting that such claims lack meaningful scientific support, Lander adds that they also "may be quite dangerous," and that even laboratories that adhere to high genetic testing standards may struggle to provide clinically meaningful results.

The Food and Drug Administration (FDA), however, has shown it may be up to the regulatory challenges posed by precision medicine, Lander writes, pointing to a recent discussion paper produced by the agency that outlines its planned approach and solicits public comment.  

Before she announced she will step down from her post in March, FDA Commissioner Margaret Hamburg was the first to indicate the agency's role in the Precision Medicine Initiative, saying it will have to modernize regulations that have always supported a "one-test, one-disease paradigm," FierceHealthcare previously reported. To achieve the FDA's regulatory goals, the Obama administration has proposed the agency receive $10 million. 

Not everyone is so confident in the government's ability to provide adequate regulation while still encouraging innovation. "The president failed to acknowledge that the risk-averse posture and policies of his administration's FDA have created significant obstacles to the ultimate success of personalized medicine," Forbes contributor Henry I. Miller writes in a recent article. The problem lies in the FDA's response to accusations that it inadequately tests drugs for safety, Miller writes, which translates to the agency's insistence on large-scale, expensive and time-consuming clinical trials that can stifle progress.

Still, Lander argues the FDA has already taken promising steps with the 2013 approval of a sequencing platform and its diagnostic application to cystic fibrosis, long before precision medicine made its way to the headlines. The agency also has a meeting planned later this month to gather input on its regulatory plans, Lander writes, adding "for the first time, there is cause for optimism that a new framework can unleash creativity in the marketplace without compromising safety."

To learn more:
- read the opinion piece
- access the discussion paper (.pdf)
- here's the Forbes article

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