Trump administration proposals could stymie personalized medicine

“It is more important to know what kind of patient the disease has than to know what kind of disease the patient has.”

Although Hippocrates made this keen observation more than 2,400 years ago, physicians did not have the tools to decipher the biological and environmental factors influencing an individual’s health and well-being until recently.

Since the human genome was finally mapped in 2003, scientists have made tremendous progress in advancing personalized medicine. By tailoring health care to an individual’s biological characteristics, circumstances, and values, personalized medicine can bring unprecedented benefits to patients with rare genetic disorders, cancer, and other diseases.

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The widely variable effects of the novel coronavirus serve as a painful reminder of the importance of understanding how and why people respond differently to the same disease.

But two recent moves by the Trump administration threaten to turn back the clock on biomedical progress in personalized medicine, which most Americans want, by locking us into a one-size-fits-all world.

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In an executive order issued in mid-September, President Trump proclaimed his intention to cut drug prices by promising to tie what Medicare pays for prescription medications to their generally lower costs in other countries. These costs are most often based on crude one-size-fits-all health technology assessments.

The order would have a devastating effect on the future of personalized medicine and on patients’ access to lifesaving new drugs. By limiting the return on high-risk investment, it will make it more difficult for biopharmaceutical companies to bet on developing paradigm-changing products, including promising gene and cell-based therapies that may one day be able to cure, not just treat, sickle cell anemia, rare genetic disorders, some cancers, and other diseases.

The executive order was issued less than a month after the Department of Health and Human Services proposed eliminating the Food and Drug Administration’s authority to regulate laboratory-developed tests. That move may unfortunately decrease public confidence in groundbreaking diagnostic tests that have not undergone governmental review just when they are on the precipice of being able to predict cancer and Alzheimer’s disease in advance of the appearance of symptoms, when prevention and treatment plans may be more effective.

Drug pricing could and should encourage drug developers to focus on developing products that will deliver the most benefits to patients and society. Instead, in an effort to end what the president calls “global freeloading,” his executive order on pharmaceutical pricing would link the rates the Centers for Medicare and Medicaid Services pays for drugs covered under Medicare Parts B and D to the lower rates established by other developed countries. These rates are set using health technology assessments that typically do not acknowledge the heterogeneity of treatment effects, despite a rapidly expanding body of scientific evidence demonstrating that the effects of a drug often vary considerably among different subpopulations of patients.

Thus, at a moment when advanced data analytics, artificial intelligence, and real-world evidence are yielding unprecedented insights about which patients should receive which therapies, international reference pricing will rob the

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