Personalized medicine, also known as precision medicine, is a revolutionary new way of treating disease that could permanently change how medicine is practiced.
A traditional medical approach treats diseases based on the average patient’s experience or needs, as well as the most “typical” form of the disease. In this approach, treatments are developed from research that shows the most likely outcomes for the largest group of people. It is sometimes considered a “one-size-fits-all” approach.
Personalized medicine, by contrast, targets the very specific needs of an individual. These needs are determined by examining a person’s genetic makeup, environmental factors, and lifestyle choices, as well as the specific traits of their disease.
In a traditional approach, a 58-year-old male who smokes and has two parents with lung disease would receive nearly the same treatment for lung cancer as a 28-year-old female who runs marathons and has no history of cancer or lung problems in her family. In a personalized approach, the two treatments would be different, tailored to the genetic makeup and lifestyle of each individual.
The field of precision medicine developed out of the Human Genome Project, which in 2003 successfully developed a way to map a person’s DNA and determine their individual genetic makeup. With this tool available for understanding the genetic code, scientists have been able to delve into the genetic causes of illness, including the mutations that make diseases different from one person to the next.
Since the successful completion of the Human Genome Project, precision medicine has developed rapidly. Beginning in 2011, the Food and Drug Administration (FDA) has approved treatments for certain forms of cystic fibrosis, as well as for numerous cancer patients with specific genetic mutations.
The nature of personalized medicine means each group that could benefit from these treatments is small. When the FDA approved the new drug Zykadia in 2014, for example, it was designed to treat the 5 percent of lung cancer patients with a certain mutation of late-stage metastatic lung cancer.
But because these treatments are so targeted to the specific needs of each group — and often to the genetic mutations of the diseases themselves — they can be much more effective than the options previously available.
Personalized treatments are still new and, as a result, are not yet the standard used by all doctors. However, there are many groups that advocate and fund their development, including The Personalized Medicine Coalition. For patients who may benefit from tailored treatment, there are centers throughout the country and world.
But, like any new developments in the medical field, precision treatments raise as many questions as they provide answers.
New drugs are expensive to develop and can take decades to go from the research stage to a pill that is available to everyday people. Because they target such small portions of the population, they are expensive to buy and use. Government regulators, doctors, and insurance companies all struggle with deciding who should pay for these specialized treatments and how.
For patients with extremely rare diseases, there may not be enough public attention to prompt the development of personalized treatments at accessible prices. And while insurance companies have so far been willing to pay for expensive personalized treatments for small portions of the population, patients with more common diseases affecting many other people may find that their insurers will not cover the costs of treating so many people.
For now, many researchers and health professionals remain optimistic about the lifesaving potential of personalized medicine. “It’s a new world,” Wendy Selig, the president of the Melanoma Research Alliance, told The Washington Post. “The goal is to match the right therapy to the right patient, and to do it with minimal collateral damage.”
July 27, 2016
Christopher Nystuen, MD, MBA