FDA OVERSIGHT OF LABORATORY-DEVELOPED TESTS

To advocate that the Food and Drug Administration be granted increased authority to regulate laboratory-developed tests as medical devices, including tests used for pharmacogenetic testing; further,

To support development of a risk-based framework for regulatory oversight of laboratory-developed tests that promotes innovation while providing a mechanism to ensure that test results are reliable, reproducible, and clinically relevant; further,

To encourage expanded availability of commercially marketed pharmacogenetic tests that would be available for use by laboratory and health care professionals to guide drug therapy.

Rationale

The use of in vitro pharmacogenetic tests has become increasingly common as efforts continue to achieve the promise of personalized medicine. However, the current system of regulatory oversight of these and other laboratory tests used to guide drug therapy is complex and inconsistent. Some laboratory tests (e.g., companion diagnostics devices) receive premarket review and approval by the Food and Drug Administration (FDA) when the test is either developed in tandem with drug development or following the drug’s approval. Other tests, commonly called laboratory-developed tests (LDTs), are proprietary tests that are developed and validated for use at specific laboratory facilities. These tests do not undergo premarket review and approval by the FDA. LDTs currently fall under a mixed system of oversight by the FDA and Centers for Medicare & Medicaid Services (CMS), which regulates these tests based on facilities’ compliance to the Clinical Laboratory Improvement Amendments (CLIA). CLIA compliance serves as the primary mechanism for oversight, as the FDA has traditionally practiced discretionary authority, meaning that only a few of the most complex tests are scrutinized by that agency. While an LDT is monitored for validity and reliability at the laboratory where it is conducted, results may not be reproducible if the test is conducted at a different laboratory site. This variability complicates the interpretation and application of this information in patient care. Therefore, ASHP advocates for the FDA to have increased authority to regulate these LDTs as medical devices to ensure that results are reliable, reproducible, and clinically relevant to patient care.

Development of a risk-based framework represents the ideal model to provide sufficient oversight while creating conditions that support continued innovation in this field. Further, the development of nationally validated and marketed tests that are available for use by laboratory and health care professionals is desirable. ASHP believes that this scenario would provide the most assurance to pharmacists and other health care professionals that the results of these tests are reliable, reproducible, and clinically relevant to patient care.