In Health Care, One Size Does Not Fit All


The history of pharmaceutical discovery could be summarized with one simple rule: “Treat as many patients as possible.” After all, drug development is a business, and a drug with broad applicability to patient populations means higher sales volume and more profits. Take Lipitor, Pfizer’s mega-blockbuster cholesterol-busting drug, which reigned for several years as the greatest-selling drug of all time. Like other statins in its class, Lipitor is approved to reduce risk of stroke or heart attack in patients with a handful of risk factors for disease. A 2010 report from the National Center for Health and Statistics suggested that a whopping 25% of all Americans 45 and older take Lipitor or a related statin. That pie is big enough that everyone wanted a piece, and the statin market was flooded with name-brand competitors like Crestor and Zocor.

But that one-size-fits-all mentality that has dominated health care is floundering, giving way instead to an explosion in personalized medicine. Now, more than ever before, doctors have the tools to treat patients as individuals, and not as a member of a loosely characterized population. The revolution really came to light with the success of the Human Genome Project, and the realization that genetics give each patient a unique molecular signature. With advancements in in vitro diagnostic tools, wearable technology, and the plummeting price of whole genome sequencing, the future of health care is emerging as a data driven, patient-tailored industry.

So where in the industry are the early signs of a personalized medicine revolution showing up? Pharma has been first grasping at the low-hanging fruit – oncology. With a massive unmet medical need and a growing understanding of the molecular underpinnings of cancer, researchers are beginning to apply a more targeted and molecularly focused approach to drug development. In breast cancer, for example, patients who superficially present with a common illness actually have divergent diseases with unique molecular signatures. Herceptin, from Genentech, was one of the first treatments to target a specific biomarker in a subset of breast cancer patients, and is now a hallmark of personalized medicine.

Now, new therapies for lung cancer, melanoma, and leukemia are slowing disease progression and increasing overall survival, simply by targeting the specific molecular signatures of disease. In what is likely to become standard practice, doctors will have the ability to sequence specific genes present in isolated tumor cells, and navigate the growing landscape of oncology drugs using predefined biomarkers. The real challenge moving forward, then, will be to identify new biomarkers for disease that both differentiate patient subgroups and present a meaningful disease-modifying drug target. That’s no small task, and suggests that basic research will play a critical role in future clinical development.

As new medicines become more selective for specific personalized biomarkers, it’s interesting to note that these drugs will actually treat fewer patients. The natural question for hospitals and insurance payers, then, is how will this new paradigm change the way we think about health care costs? Will drug companies boost the price of more personalized drugs to reap the kind of exorbitant profits they’re used to?

The answer is ‘yes’. But that’s okay. Personalized medicine represents a shift away from volume and toward value. Payers are coming to realize that personalized medicine presents an opportunity to cut long-term health care costs by investing in more effective short-term therapies. Doctors will collect mountains of data to describe an individual patient, and use that data to predict drug action and sensitivity. Perhaps more importantly, a personalized approach may allow doctors to preempt disease by tailoring primary care to the patients’ specific needs, further reducing the burden of elevated drug pricing and reactionary care.

The transition from one-size-fits-all medicine to personalized medicine will take time, but the seeds of change have already been planted. Using oncology as a launching pad, it will soon be a reality that biomarker analysis will differentiate patients and guide prevention and treatment in a clinically meaningful way. By embracing that change, it may be possible to improve patient care while bringing down long term health care costs – a win-win for a society in the midst of a chronic debate over health care reform.

To read more about Personalized Medicine and the future of health care, check out Seth’s work for and follow him on Twitter @sethrobey.