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 United States of an opportunity to stay ahead of its peers by establishing value-based payment rates for therapies that move us away from one-size-fits-all, trial-and-error medicine and toward personalized medicine.

The HHS announcement states that the FDA cannot require regulatory review of diagnostic tests developed by laboratories unless the agency allows for a rigorous regulatory process known as notice-and-comment rulemaking. Although the policy was designed to ensure that tests for Covid-19 come to market as quickly as possible, it appears to apply to the regulation of all laboratory-developed tests, including those that report genetic variants that may affect the risk of developing a disease or guide the selection of a treatment.

The 252-word statement — which comes during an as-yet unresolved bipartisan debate in Congress regarding the regulation of laboratory-developed tests — has heightened the concerns of some who contend that the success of high-risk genetic- and genomic-based tests depends on FDA review.

The potential unintended consequences of the president’s executive order and HHS’s announcement underline the importance of making careful decisions even during times of crisis. To avoid locking the United States into a one-size-fits-all world, officials at HHS would do well to support ongoing efforts by Congress to develop a clearly defined and adequately vetted regulatory framework for laboratory-developed tests. And any change in pharmaceutical pricing policies should be implemented through a transparent process that engages leaders from every sector of the health care system to ensure that, in addition to addressing cost considerations, it actually meets the needs of patients, particularly in addressing unmet medical needs.

Taking these prudent steps will help ensure that we do not foreclose a brighter future for patients and health systems in which treatments are matched to the patients who will benefit from them.

Edward Abrahams is the president of the Personalized Medicine Coalition, an education and advocacy organization in Washington, D.C.