The Case for Personalized Medicine

— Executive Summary —

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The sequencing of the human genome is yielding exciting new tools to help physicians tailor treatments to individuals and their disease. This powerful new capability, called personalized medicine, holds great potential to improve patient health. But significant hurdles must still be overcome for its full potential to be realized.

In many ways, physicians already practice "personalized medicine." Recognizing that patients respond differently to treatments, they routinely use diagnostic tests to learn more about the patient's disease, and choose specific treatment options and drug dosages based on the results of those tests, as well as the patient's family medical history, comorbidities (other medical conditions at the time of diagnosis), and lifestyle factors. However, it is only recently that science has begun to give physicians understanding of individual patient or disease differences at the molecular or genetic level, enabling them to tailor treatment even more effectively. For example, knowledge of genetic variations can now help physicians optimize breast cancer therapy or better manage dosing of blood thinning drugs.

Today, personalized medicine may include testing for variations in genes, gene expression, proteins, and metabolites. Test results are correlated with drug response, disease state, or treatment prognosis, and help physicians individualize treatment for each patient with greater precision. But the use of molecular and genetic information, and its ability to predict susceptibility to disease or guide proactive care, means that personalized medicine has the potential to transform our healthcare system. For personalized medicine to work, the system will require new regulatory approaches, revamped medical education curricula, integrated health information technology, strong patient privacy protections, and an insurance reimbursement system that encourages preventive medicine and supports access to the tests and treatments that enable physicians and patients to personalize care. Because of the many hurdles before it, some experts have questioned whether personalized medicine will become a dominant trend in healthcare, or just a passing phase.

Advocates of personalized medicine have stressed its potential to:

• Detect disease at an earlier stage, when it is easier to treat effectively
• Enable the selection of optimal therapy and reduce trial-and-error prescribing
• Reduce adverse drug reactions
• Increase patient compliance with therapy
• Improve the selection of targets for drug discovery
• Reduce the time, cost, and failure rate of clinical trials
• Revive drugs that failed clinical trials or were withdrawn from the market
• Avoid withdrawal of marketed drugs
• Shift the emphasis in medicine from reaction to prevention
• Reduce the overall cost of healthcare

To date, much of the promise and pitfalls of personalized medicine remain untested. This document presents the evidence that personalized medicine has proven its value, that it will continue to grow in importance, while at the same time acknowledging that uncertainties remain concerning its full impact.

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