Peter Huber Explains How 20th Century Law is Undermining 21st Century Medicine
20th Century Regulatory Paradigm Must Be Updated
02.20.14 | By Jeff Francer
A new book, the Cure in the Code: How 20th Century Law is Undermining 21st Century Medicine, provides a fascinating overview of advances in biomedical science with the potential to revolutionize how diseases are treated by providing patients with more targeted therapies.
But before we can recognize the potential of this science, we need to think about updating our 20th century regulatory paradigm. We need a regulatory framework that recognizes the incredible growth of healthcare data now available, from early-stage development to validating potential alternative uses of approved medicines.
In the guest blog below, Peter Huber, author of the Cure in the Code and senior fellow at the Manhattan Institute for Policy Research, shares his perspective on what advances in technology could mean for the practice of medicine.
In at least one area the cost of health care is falling fast. Chip-sized, micro-electromechanical laboratories can, for a few dollars a whiff, search a cheek swab or drop of blood for hundreds—and soon thousands—of genes, proteins, and other biomarkers. By sharply lowering the cost of acquiring the information on which much of the rest of molecular medicine hinges, these technologies will have a profound impact on almost every aspect of pharmacology.
For drug designers and doctors, they reveal how diseases are spawned and propelled down at the molecular level, and thus reveal what drugs should target and provide precise criteria for prescribing drugs to the right patients. And these technologies will soon become cheap enough to be widely and routinely used by almost anyone, healthy or sick.
A first look at where that can lead was provided in early 2012, when scientists at Stanford University described how they had spent the previous two years tracking DNA, RNA, cell proteins, antibodies, metabolites, and molecular signals—some forty thousand biomarkers—in the body of geneticist Michael Snyder to create the first-ever “integrative Personal ‘Omics’ Profile”: an “iPOP.”
Though Snyder had no family history or conventional risk factors, the data revealed a genetic predisposition to type 2 diabetes, and then, later in the study, tracked the onset of the disease in what has been described as “the first eyewitness account—viewed on a molecular level—of the birth of a disease that affects millions of Americans.” Further analysis pointed to an unexpected cause: a viral infection twelve days earlier. The iPOP team then watched the diabetes markers revert to their normal state in response to treatment with aspirin, ibuprofen, exercise, and a low-sugar diet. “In a study like this, you are your own best control,” says Snyder. “You compare your altered, or infected, states with the values you see when you are healthy.” Snyder anticipates a home-use test kit that allows people at risk of diabetes to track 5,000 biomarkers in a single drop of blood.
Medical science has only just begun to pin down the clinical implications of much of the data that molecular diagnoses can supply. As the Stanford study demonstrates, however, we now have tools to integrate the study of a disease’s biology with the study of drugs that might be used to treat it. Once we have identified potential molecular risk factors we can monitor for the transition from dormant risks to active processes that foreshadow clinical disorders, and then track the molecular effects of drugs that target them.
Comprehensive molecular monitoring of this kind should also be an integral part of the drug approval process. It will provide the information needed to conduct adaptive trials that quickly home in on the factors that determine which patients will respond well to a drug. It can also provide rapid feedback on the efficacy of off-label prescriptions of approved drugs. And it will promote and accelerate the development of drugs that intervene early, when treatments are most likely to succeed.
Peter W. Huber is a senior fellow at the Manhattan Institute for Policy Research.