The implosion of the once-high-flying medical testing startup Theranos was an unmitigated disaster: a fraudulent health tech company led by a charismatic, articulate figurehead, disseminating deceitful diagnostic claims about its blood tests.
Such laboratory developed tests (LDTs), which are created and utilized within individual laboratories and meant for use in patients, have never required any approval from the Food and Drug Administration (FDA). In what I think is a direct result of the Theranos failure, the FDA is proposing a new rule that would require agency approval prior to the implementation of any LDT. Some proponents of the rule think such approval would have prevented Theranos from fooling investors and medical experts alike with its unprecedented blood test that its founder Elizabeth Holmes said could diagnose multiple diseases with a single drop. Given the magnitude of Theranos’s fraud and the devastation left in its wake, I understand why the federal government would want to ensure that it could never happen again.
As a Harvard Medical School psychiatrist who has taught medical ethics for over two decades, I know the answer to medical fraud is not so simple. Although well-intended, this proposal is an overreaction that would ultimately lead to more harm than good and shouldn’t be approved as written. Rather, a toothier and more intensive version of the current program meant to validate LDTs would better serve the public.
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For one, the FDA rule could, in practice, violate one of the fundamental medical ethical principles—justice, which concerns itself with issues such as access to care and health equity. Seeking and obtaining FDA approval for anything is universally acknowledged as a time-consuming and costly process. Consequently, if the proposal is green-lit, the majority of LDTs seeking FDA approval likely would be confined to those targeting mainstream, majority populations.
Why? The substantial costs associated with FDA approval may dissuade companies from proposing laboratory developed tests unless they are likely to be profitable. This profitability hinges on a large market for the tests. Consequently, minority populations might be neglected on a basis of financial impracticality. Children, who constitute 22 percent of the U.S. health care market, could also suffer for the same reason. The outcome could be greater stratification in LDT recipients, reduced access to innovative diagnostic tools tailored to minority needs, and heightened health disparities.
Additionally, if this proposal is passed, many smaller labs would almost definitely stop creating LDTs entirely. Once that happened, much of the test creation along with actual testing would be handled by large labs, which would effectively have a monopoly on the market. In such a market, companies set pricing however they wish, possibly pricing out poor and uninsured individuals. The end result would be a further degradation of health equity.
The rule also might break the medical ethics principles of beneficence (promoting patient well-being) and nonmaleficence, or preventing harms to patients. Both principles require being able to provide timely diagnostic interventions. The FDA’s rigorous approval process can be prolonged and thereby impede the timely dissemination of LDTs, and delays in test availability could deprive patients of important diagnostic information, potentially compromising treatment decisions and prognoses.
Or it might cause labs to decide it’s just not worth creating a particular test.
Consider this hypothetical scenario: a hospital identifies an unknown pattern of illness and wishes to develop a test to investigate; the hospital researchers consider the cost and time associated with FDA approval and decide not to proceed, hoping the mystery illness will simply go away.
The costs associated with FDA oversight pose ethical dilemmas. The principle of distributive justice emphasizes the equitable allocation of resources. However, the financial burdens imposed by protracted FDA approval processes could strain health care budgets, limiting access to essential diagnostic services and potentially widening health care disparities.
In the end, the ethical landscape of diagnostic regulation necessitates a delicate balance between regulatory rigor and practical utility. The FDA plays an indispensable role in safeguarding public health, but its oversight would likely not easily align with the nuanced intricacies of LDT development and implementation.
There is a better solution, and it involves the Clinical Laboratory Improvement Amendments (CLIA), a federal program that currently regulates LDTs. The CLIA provides a tiered regulatory approach that allows for tailored oversight based on the complexity of laboratory tests. In that way, it offers more flexibility and scalability than the FDA’s one-size-fits-all approach, which may be less adaptable to the rapid pace of innovation and technological advancements in diagnostic medicine.
But, in order to oversee the creation and use of laboratory developed tests effectively, the CLIA would need to be beefed up. First, the CLIA ought to offer fewer waivers to labs. Waivers are granted to laboratories that only perform tests considered to be simple and with low risk for erroneous results; currently, regulators don’t inspect such labs every two years like other labs. Second, the CLIA needs to create its own guidelines for the development, validation and use of LDTs (currently set by the labs themselves), including setting standards for analytical and clinical validity. And third, the CLIA should collaborate with professional organizations, such as the American Clinical Laboratory Association, to establish best practices, guidelines and standards for LDTs. Right now, the Centers for Medicare and Medicaid Services draws up this guidance.
Unlike requiring FDA approval and oversight, these measures would not impede the ability of labs to be agile in responding to novel disease outbreaks or make it financially prohibitive to create LDTs for niche markets or minority populations.
For all of these reasons, the potential benefits of preventing another Theranos are far outweighed by the potential harms resulting from subjecting every LDT to costly, time-consuming FDA oversight.
This is an opinion and analysis article, and the views expressed by the author or authors are not necessarily those of Scientific American.