September 29, 2023
A coalition of consumer advocacy groups is welcoming a proposed rule released today by the Food and Drug Administration to regulate laboratory-developed tests (LDTs), a category of diagnostic tests developed and used in a single lab. LDTs are a subset of In Vitro Diagnostics (IVDs) which are FDA regulated, despite the fact that they have been regulated differently for decades. The proposal clarifies that LDTs are medical devices, meaning FDA will ensure they are safe and effective before they are sold to consumers. The tests have long been under “enforcement discretion,” meaning FDA has not enforced premarket approval and other requirements, but stakeholders and regulators alike have been calling for increased scrutiny of these tests for years.
The proposed regulation will take effect 60 days after a final rule is published and contemplates a phase-in over the subsequent four years. As these tests have become more complex and more important to patient care, ensuring their accuracy has become more crucial, according to the coalition, which includes the Center for Science in the Public Interest, the National Center for Health Research, Strathmore Health Strategy, and U.S. PIRG.
False-positive test results could lead patients to believe they have a serious medical condition that they do not have, and false-negative results may cause a patient’s life-threatening condition to be missed. Some tests have falsely diagnosed people with cancer or inaccurately provided results that lead directly to chemotherapy selection. Many of the tests have been found to be inaccurate, including some COVID-19 diagnostic tests, genetic non-invasive prenatal screening tests, and blood tests manufactured by the biotech company Theranos.
The group applauds many provisions of the proposed rule. These include:
- Including Academic Medical Centers in the regulatory scheme. There is no reason that people treated or tested at one facility should be more at risk for inaccurate results than those tested at another facility. All tests should be evaluated based on the benefits and risks of the tests, not the building in which the test is run. The proposed rule notes that “[r]eview of the underlying science behind an [in vitro diagnostic] is based on what the IVD does and is in no way related to where the IVD is made.” The FDA requests additional information on this topic, but the coalition hopes the agency sticks to its guns.
- Including tests for rare diseases in the regulatory scheme. Patients with rare diseases should be equally protected from inaccurate tests.
- Registration and listing requirements for all tests, which exist for all other medical devices. This will allow FDA and the public to know which tests are available. Further, manufacturers are required to publish performance data on IVDs, which will provide much-needed transparency about the clinical and analytical validity of these tests, according to the coalition.
“This rule is a critical step forward for clinical medicine,” said Dr. Peter G. Lurie, President of Center for Science in the Public Interest and a former Associate Commissioner at the Food and Drug Administration where he worked on LDTs, including on a report demonstrating their potential dangers. “It will help ensure that when a patient receives a test, they can rely on the results to make essential decisions for their health. This rule will close a gaping hole in FDA’s current regulatory reach.”
In the absence of FDA oversight, LDTs have been regulated only by the Centers for Medicare and Medicaid Services, which does not require documentation that the test results accurately inform the diagnosis of patients, a concept known as “clinical validity.” CMS only requires laboratories to document the “analytical validity” of their tests, or their ability to reliably detect a biomarker.
Oversight under FDA would be much more comprehensive and would ensure that healthcare providers and patients can rely on results to make medical decisions, particularly the riskiest medical decisions, where inaccurate test results can cause harm to patients. A modern regulatory framework for LDTs will improve patient access to accurate tests and promote innovation in the diagnostic testing industry.
“We strongly support the decision by the FDA to do what is necessary to rectify a situation that has been so harmful to patients,” said Dr. Diana Zuckerman, President of the National Center for Health Research. “We understand the need for a transitional period but urge the FDA to address problems with existing high-risk LDTs as quickly as possible. We welcome the opportunity to work with FDA and other interested parties to ensure that FDA has the resources it needs to robustly regulate LDTs so that patients can make informed decisions based on test results.”
This comprehensive approach will require resources, including both user fees and Congressional appropriations. The coalition will continue working with all stakeholders, including Congress and the Agency, to ensure that the agency is adequately resourced to fulfill this critical function.
For more information, contact Dr. Zuckerman at email@example.com