A number of health care industry leaders are making big bets that the future of health care lies in precision medicine. This July, Dr. Priscilla Chan and Mark Zuckerberg donated $10 million to the University of California-San Francisco’s precision medicine lab. Back in 2016, President Obama launched the Precision Medicine Institute with a $215 million investment from the federal government. In addition, the All of Us program is a National Institute of Health-funded initiative to collect genetic and environmental information to create precision medicines.
All of this money flowing into precision medicine may leave you with only one question left to answer: What is precision medicine?
[See: 5 Rare Diseases You’ve Never Heard of Until Now.]
Defining Precision Medicine
Before we move to a definition of precision medicine, let’s consider an example of a conventional, one-size-fits-all medication: vaccines. The Centers for Disease Control and Prevention recommends that all children receive the same schedule of vaccines. Vaccines have been shown to be both universally and highly effective, so this approach has worked well. They have succeeded in essentially eradicating diseases like polio and smallpox.
In other cases, however, not all patients may respond to a given therapy. Consider the case of treatments for chronic diseases like rheumatoid arthritis. The most commonly used treatments — known as disease-modifying antirheumatic drugs, or DMARDs — work well for many patients. A number of patients, however, still find that they need to switch medications one, two or even more times to find the treatment that adequately addresses their individual disease.
This treatment cycling may become a thing of the past with the introduction of precision medicines. The National Institutes of Health defines precision medicine as “an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment and lifestyle for each person.” Lung cancer treatment provides an instructive example. Doctors treating lung cancer patients now conduct a series of diagnostic tests to identify cancer cells’ genetic mutations in order to identify the precision medicine that would work best for each patient. Some lung cancer treatments are known to work best for patients with an ALK mutation, while other treatments work better when a patient’s tumor has an EGFR mutation. Still others work best when a patient’s tumor has a BRAF mutation, and so on. In the world of precision medicine for lung cancer, there is no single “best” treatment, but rather treatments are selected based on an individual patient’s needs.
Whereas the precision medicines described above are still mass-produced therapies that happen to target specific genetic mutations, other precision medicines are made one at a time for an individual patient. An example of this type of “n of 1” treatment is the emerging chimeric antigen receptors T-cell (CAR-T) therapies. To create these therapies, physicians extract T-cells from each patient’s blood, then genetically engineer them to attack the patient’s specific tumor. Thus, each CAR-T therapy is individually designed to best attack the unique disease of a single patient.
[See: 9 Things You Didn’t Know About Sickle Cell Disease.]
The Unique Challenges of Precision Medicine
Although precision medicine offers the hope of dramatically improving treatment for many diseases, it also raises a number of unique issues that could be barriers for getting these medicines to patients. First, just conducting the necessary clinical trial research to get precision medicines approved by the Food and Drug Administration is a challenge. For therapies where a treatment is individualized to each patient, standard approaches to conducting clinical trials may not be feasible. Fortunately, the FDA is well-aware of these challenges and has developed a Precision Medicine Initiative to streamline the treatment approval process.
Second, patient privacy is a concern. In order for researchers to develop precision medicines, and for physicians to identify relevant treatments, they need access to data on a patient’s genetics. But who owns the data? Who can access the data? Who ensures that data on individuals’ genetics are secure? These questions need to be answered to ensure that patients’ genetic data don’t fall into the wrong hands.
Finally, treatment cost may become a barrier to access. Precision medicines that require manufacturing processes targeted to individual patients — like CAR-T — will not be inexpensive to create. Further, while the research and development costs to develop precision medicines will likely be similar or higher than standard medications, fewer patients will be eligible for treatment since precision — by definition — means that the treatments will be narrowly targeted. When the potential market for any individual treatment is small, prices tend to be higher to compensate innovators for their research and development cost. Groups such as the Innovation and Value Initiative — where I serve as the director of research — can help by creating tools for a variety of stakeholders to measure the treatment value of these precision and other medicines.
[See: Do’s and Don’ts of Home Medical Devices.]
The Future
All signs are pointing toward the U.S. moving head-first into a world of precision medicines. Consumer-facing organizations like 23andMe already offer patients insight into their genetic makeup. Forward-looking health systems — such as Geisinger’s Genomic Medicine Institute — are already prospectively collecting genetic data from their patients. Despite a number of challenges, I expect that patient genetic data will be increasingly shareable, allowing more and more precision medicines to be available in our lifetimes.
More from U.S. News
How Hospitals Are Using Technology to Become More Patient-Centered
10 Lessons From Empowered Patients
How to Be a Good Patient Wingman
What’s All the Fuss About Precision Medicine? originally appeared on usnews.com
