A focus on cost instead of value threatens the future of personalized medicine

One size fits all never worked as a strategy for the fashion industry. Or the auto, furniture, and most other industries. It shouldn’t be the strategy for medicine either, but unfortunately more often than not it is. And it may stay that way if the focus on cost rather than value precludes the improved health care strategies made possible by personalized medicine.

According to the World Health Organization, an effective health system requires “reliable information on which to base decisions and policies.” The evolving field of personalized medicine delivers just that kind of information in the form of data about the unique biological characteristics of each patient. Doctors can often use that information to make medical decisions based on an understanding of how a patient will respond at a molecular level to a specific therapy. This helps ensure that therapies are prescribed only to those who will benefit from them, sparing side effects and expenses to those who will not.

Although the majority of patients say they aren’t yet aware of personalized medicine, they also say they don’t want to miss out on this opportunity.

Survey results released Wednesday by the Personalized Medicine Coalition, a nonprofit education and advocacy organization, and GenomeWeb, an online news service, show that although two-thirds of Americans have never heard of personalized medicine, two-thirds of them also react positively to a description of the approach and only 1 percent react negatively to it. The overwhelming majority agree that replacing one-size-fits-all, trial-and-error medicine with an approach that proactively identifies the right treatment for the right patient is worth pursuing and paying for.

One of every four new drugs approved by the Food and Drug Administration over the last four years was designed to become a personalized (or “targeted”) therapy that zeros in on the subset of patients likely to respond positively to it. That’s a sea change from the way drugs were developed and marketed 10 years ago.

Some of these new treatments have extraordinarily high list prices. But focusing solely on the cost of these therapies rather than on the value they provide threatens the future of personalized medicine.

Most Americans are unfamiliar with the principles and benefits of personalized medicine, with only 13 percent of survey respondents indicating that they feel “very informed” about the field. So they are not yet asking policymakers to make the changes that would ensure continued investment in and adoption of personalized medicine. Patients, like health systems, are still locked into a one-treatment-fits-all world.

At the same time, most policymakers are not asking the right questions about the benefits of these treatments for patients and society. Influenced by cost concerns, they assume that prices for personalized tests and treatments cannot be justified even if they make the health system more efficient and effective by delivering superior, longer-lasting clinical outcomes and increasing the percentage of patients who benefit from prescribed treatments.

Goldman Sachs, for example, issued a report titled “The Genome Revolution.” It argues that while “genome medicine” offers “tremendous value for patients and society,” curing patients may not be “a sustainable business model.” The analysis underlines that the health system is not set up to reap the benefits of new scientific discoveries and technologies. Just as we are on the precipice of an era in which gene therapies, gene-editing, and immunotherapies promise to address the root causes of disease, Goldman Sachs says that these therapies have a “very different outlook with regard to recurring revenue versus chronic therapies.”

In other words, Goldman Sachs suggests that if the pharmaceutical industry wants to make money, it should be cautious about developing one-shot therapies that cure patients and lower costs over time, because decision-makers are more focused on the price of a product than its value to a particular patient or to the health system itself.

If ongoing concerns among policymakers and the public add to the validity of this logic by discouraging payers from covering personalized “cures,” Goldman Sachs and others could choose not to make investments in innovative medicines that patients clearly want and need.

It falls on Congress, the FDA, and the Centers for Medicare and Medicaid Services to put in place an infrastructure that supports personalized medicine by:

• Spearheading the efficient approval of personalized tests and treatments
• Developing innovative reimbursement models that provide incentives for targeting treatment to only those patients who will benefit
• Implementing programs that encourage the clinical adoption of personalized medicine by health systems around the country.

With such efforts, policymakers can accelerate the pace of medical progress upon which patients and the health system depend.

As Tango Therapeutics President and CEO Dr. Barbara Weber summarized at the 12th Annual Personalized Medicine Conference at Harvard Medical School in 2016, “It’s not really ‘should we do this.’ We have to do this. We don’t get to decide what the biology of these diseases are, we just have to work with it.”

Edward Abrahams, Ph.D., is president of the Personalized Medicine Coalition.