Personalized medicine is perceived by many to be the future of medicine. Discoveries of the human genome paired with big data analysis of what treatments are most effective for which biomarkers hold great potential to significantly change how the medical community diagnoses and treats disease.
Partners Health Care's Center for Personalized and Genetic Medicine defines personalized medicine as follows:
"Personalized medicine is the ability to determine an individual's unique molecular characteristics and to use those genetic distinctions to diagnose more finely an individual's disease, select treatments that increase the chances of a successful outcome and reduce possible adverse reactions. Personalized medicine also is the ability to predict an individual's susceptibility to diseases and thus to try to shape steps that may help avoid or reduce the extent to which an individual will experience a disease."
Many leading academic medical centers already boast personalized medicine centers, including the University of Chicago, Duke, Mayo Clinic and MD Anderson.
For most people who hear of the potential of personalized medicine, there seem to be few drawbacks, save cost, which is expected to go down over time.
However, a new Forbes op-ed by contributor Henry Miller, a biomedical scientist and former FDA drug regulator, explains how personalized medine, while good for patients and providers, could hurt drugmakers — especially if regulators don't adjust approval requirements to account for smaller target populations.
According to Miller:
"Improvements in efficacy and fewer side effects of drug therapy are a boon to doctors, patients and insurance companies, to be sure, but the benefits to drug companies – and therefore, their willingness to embrace personalized medicine in the long term – are less certain."
Specifically, Miller explains that as the target market for a drug narrows (i.e., the drug targets only patients with a specific biomarker), "the medication's approved uses on the label might be narrower, or more restrictive, thereby reducing the size of the patient population for whom the drug is intended – and the revenue potential."
Additionally, in the past few years, Miller argues regulators have required "huge, expensive and time-consuming clinical trials designed to detect even very rare possible side effects," calling the large number of patients required for the studies as "excessive." Continuing to require such large safety studies in the midst of narrower drugs, he says, will limit drugmakers' incentives to develop new treatments.
"Regulators’ demands for vast clinical studies to demonstrate the safety of new drugs, along with the need to develop the diagnostic (biomarker) tests to accompany the drug and also a clinical algorithm to guide the use of the drug/diagnostic combination, could impose huge development costs that might never be recovered by the manufacturers."
So while personalized medicine is clearly a boon for patients, let's hope regulators and drug makers can find a balance between ensuring safety and efficacy and managing regulatory burden so that biomarker-targeted drugs are actually available to patients.