SILVER
SPRING, Md., April 29,
2024 /PRNewswire/ -- Today, the U.S. Food and
Drug Administration took action aimed at helping to ensure the
safety and effectiveness of laboratory developed tests, or LDTs,
which are used in a growing number of health care decisions and
about which concerns have been raised for many years.
LDTs are in vitro diagnostic products (IVDs) that the FDA has
described as intended for clinical use and designed, manufactured
and used within a single clinical laboratory that meets certain
regulatory requirements. IVDs can play an important role in health
care; they are used in the collection, preparation and examination
of specimens taken from the human body, such as blood, saliva or
tissue. They can be used to measure or detect substances or
analytes, such as proteins, glucose, cholesterol or DNA, to provide
information about a patient's health, including to identify,
monitor or determine treatment for diseases and conditions.
The FDA announced a final rule today amending the FDA's
regulations to make explicit that IVDs are devices under the
Federal Food, Drug, and Cosmetic Act (FD&C Act) including when
the manufacturer of the IVD is a laboratory. Along with this
amendment, the FDA issued a policy to phase out, over the course of
four years, its general enforcement discretion approach for LDTs.
The agency also issued targeted enforcement discretion policies for
certain categories of IVDs manufactured by laboratories.
"LDTs are being used more widely than ever before – for use
in newborn screening, to help predict a person's risk of cancer, or
aid in diagnosing heart disease and Alzheimer's. The agency cannot
stand by while Americans continue to rely on results of these tests
without assurance that they work," said FDA Commissioner
Robert M. Califf, M.D. "The final
rule announced today aims to provide crucial oversight of these
tests to help ensure that important health care decisions are made
based on test results that patients and health care providers can
trust."
Although historically the FDA has generally exercised
enforcement discretion for most LDTs, meaning that the agency
generally has not enforced applicable requirements with respect to
most LDTs, the risks associated with most modern LDTs are much
greater than the risks associated with LDTs used when the FDA's
enforcement discretion approach was adopted many decades ago. At
that time, many LDTs were lower risk, small volume and used for
specialized needs of a local patient population. Now, many LDTs are
used more widely, for a larger and more diverse population, with
large laboratories accepting specimens from across the country.
LDTs also increasingly rely on high-tech instrumentation and
software, are performed in large volumes and are used more
frequently to help guide critical health care decisions.
Moreover, there is a growing body of evidence that demonstrates
that some IVDs offered as LDTs raise public health concerns; for
example, they do not provide accurate test results or do not
perform as well as FDA-authorized tests, including from published
studies in the scientific literature, the FDA's own experience in
reviewing IVDs offered as LDTs, news articles and class-action
lawsuits.
The FDA is aware of numerous examples of potentially inaccurate,
unsafe, ineffective or poor quality IVDs offered as LDTs that
caused or may have caused patient harm, including tests used to
select cancer treatment, aid in the diagnosis of COVID-19, aid in
the management of patients with rare diseases and identify a
patient's risk of cancer.
Without greater oversight of the safety and effectiveness of
LDTs, patients may be more likely to initiate unnecessary
treatment, or delay or forego proper treatment based on inaccurate
test results or tests promoted with false or misleading claims.
This could result in harm, including worsening illness or death, as
well as unnecessarily increase health care costs.
Increased compliance with device requirements under the FD&C
Act (such as premarket review, quality system (QS) requirements,
adverse event reporting, establishment registration and device
listing, labeling requirements and investigational use
requirements) will put patients and health care providers in a
better position to have confidence in IVDs regardless of where they
are manufactured.
With increased oversight, the FDA will also be able to help
promote adequate representation in validation studies, as well as
transparency regarding potential differential performance and
unknown performance in certain patient populations, which may
ultimately help advance health equity.
"Today's action is a critical step toward helping to ensure
the safety and effectiveness of LDTs, while also taking into
account other public health considerations, including continued
access to critical tests patients rely upon," said Jeff Shuren, M.D., J.D., director of the FDA's
Center for Devices and Radiological Health. "Through targeted
enforcement discretion policies for certain categories of
tests manufactured by a laboratory, we expect patients and
health care professionals will continue to have access to the tests
they need while having greater confidence that the tests they rely
on are accurate."
The phaseout of the FDA's general enforcement discretion
approach for LDTs over a period of four years will protect the
public health by helping to assure the safety and effectiveness of
these tests, while avoiding undue disruption to patient care.
Better assuring the safety and effectiveness of LDTs may also
foster test innovation and facilitate the collective efforts of the
scientific and medical communities to identify promising
technologies, new therapies or areas worthy of future research.
Importantly, the FDA considered the large volume of comments
received on the notice of proposed rulemaking, and in light of that
input, has adjusted the phaseout policy in a manner that better
serves the public health. After this phaseout, the FDA generally
will expect IVDs made by either a non-laboratory or laboratory to
meet the same requirements, though certain IVDs manufactured by
laboratories may fall within one of the agency's targeted
enforcement discretion policies.
The FDA intends to exercise enforcement discretion with regard
to premarket review and most quality system requirements for
certain categories of IVDs, including but not limited to:
- Currently marketed IVDs offered as LDTs that were first
marketed prior to the date of issuance of the final rule. This
enforcement discretion policy is intended to address the risk that
the perceived costs of compliance with such requirements could lead
to the widespread loss of access to beneficial IVDs on which
patients currently rely.
- LDTs manufactured and performed by a laboratory integrated
within a health care system to meet an unmet need of patients
receiving care within the same health care system when an
FDA-authorized test is not available. This enforcement discretion
policy is intended to help avoid patients being deprived of
critically needed LDTs where certain risk mitigations exist that
may help laboratories to identify any problems with their LDT and
may help inform appropriate use and interpretation of such
LDTs.
The FDA has also included additional enforcement discretion
policies, such as for LDTs approved by the New York State's Clinical Laboratory
Evaluation Program (CLEP), as described in the preamble to the
final rule, where that program's review of analytical and clinical
validity helps to mitigate the risk of harm from inaccurate and
unreliable LDTs.
Draft Guidance Documents
The agency also issued two draft guidances today. One provides
the agency's thinking about an enforcement discretion policy for
certain laboratories offering certain unauthorized IVDs for
immediate response to an emergent situation, such as an outbreak of
an infectious disease, in the absence of a declaration applicable
to IVDs under section 564 of the FD&C Act. The other provides
insight into the FDA's thinking about the factors the agency
intends to consider when developing a policy regarding enforcement
discretion for certain IVDs during a public health emergency
declared under section 564 of the FD&C Act.
Additional Resources:
- Federal Register, Final Rule
- FDA: Laboratory Developed Tests
- FDA: LDT Frequently Asked Questions
Media Contact: Jim
McKinney, 240-328-7305
Consumer Inquiries: 855-543-3784
The FDA, an agency within the U.S. Department
of Health and Human Services, protects the public health by
assuring the safety, effectiveness, and security of human and
veterinary drugs, vaccines and other biological products for human
use, and medical devices. The agency also is responsible for the
safety and security of our nation's food supply, cosmetics, dietary
supplements, radiation-emitting electronic products, and for
regulating tobacco products.
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SOURCE U.S. Food and Drug Administration