A New Personalized Cancer Treatment Shows What’s Possible in the New Era of Medicine

In recent years, researchers have expanded the limits of science so quickly that methods of treatment thought to be impossible a decade ago are now a reality. Once-in-a-generation medical advances, driven by big data and genetic research, have ushered in a new era of medicine that is changing how we treat disease.

This innovation is on display with the recent U.S. Food and Drug Administration (FDA) approval of a new tumor-agnostic cancer treatment designed to counteract a specific genetic mutation as opposed to a specific type of tumor, such as breast or colon cancer. In the announcement of the approval, the FDA recognizes how it represents a changing trajectory in how we treat disease.

“Today’s approval marks another step in an important shift toward treating cancers based on their tumor genetics rather than their site of origin in the body,” says FDA Commissioner Scott Gottlieb in the release. “We now have the ability to make sure that the right patients get the right treatment at the right time. This type of drug development program … wouldn’t have been possible a decade ago because we knew a lot less about such cancer mutations.”

Commissioner Gottlieb’s comments about getting the right patients the right treatment at the right time represent continued progress in the field of personalized medicine. Many of today’s most devastating diseases—particularly cancer—are heterogeneous, meaning they express themselves uniquely in every patient, based on genetics and a several other factors. In fact, scientists today understand the term “cancer” represents more than 200 different diseases, each with a slightly different cause and thus the need for a different approach to treatment.

Personalized medicine recognizes these differences and aims to tailor treatments specific to a patient’s unique needs. Personalized medicine can also help save precious time and produce cost savings on treatments so patients are getting the right treatment at the right time much faster.

Ten years ago, scientists viewed the level of targeting we can achieve today as science fiction, but thanks to the hard work, drive and immense investment by America’s biopharmaceutical researchers into pushing the boundaries of human knowledge, they are becoming a reality.

Of course, the battle is yet not over. The fight against disease is relentless, and more than 600,000 Americans will die from cancer this year alone. More than 1,000 neurological disorders affect nearly a third of the U.S. population and collectively, 30 million Americans suffer from more than 7,000 rare diseases, including cystic fibrosis, muscular atrophy and juvenile arthritis, among others. Every single one of these patients can benefit from innovation, and it’s why the thousands of biopharmaceutical researchers across the U.S. wake up and go to work—to fight, and one day eliminate disease.

Unfortunately, these researchers’ work is itself fragile. Historically, the United States has been a powerhouse of biopharmaceutical innovation due to supportive policies, such as strong intellectual property protection and a vast array of collaborative partnerships between government, industry and academia, which protect and even encourage our ability to expand the frontier of science. It is little surprise that the recently-approved cancer treatment, brought to patients and providers in a partnership with Bayer, was both developed in and first approved for use in the United States.

Yet despite this and other incredible break-throughs, the Department of Health and Human Services (HHS) recently put forth a proposal that could discourage continued innovation here in the U.S. by replicating flawed price setting practices of foreign governments. History shows that when governments have implemented price setting policies, they’ve driven innovation away. This proposal is a step in the wrong direction for research and development.

Because with that support, the possibilities that we can achieve in the new era of medicine will be limitless.