The Food and Drug Administration (FDA) recently announced its intention to begin to regulate a class of medical laboratory tests called laboratory developed tests (LDTs). But before it embarks on new regulatory initiatives, the FDA should fix the significant deficiencies in its oversight of the laboratory tests that are already within its jurisdiction.

Precision medicine is often defined as providing "the right drug, for the right patient, at the right dose, at the right time." That typically means performing genetic analysis on a patient's blood or a tumor, and linking therapy to the identified genetic profile. Diagnostic laboratory tests are at the center of this process.

In the U.S., diagnostic laboratory tests are either LDTs or in vitro diagnostic test kits (IVDs). IVDs, manufactured and sold to laboratories, are regulated by the FDA. LDTs, on the other hand, are developed and performed within laboratories by skilled professionals for use with their own patients. They are overseen directly by the Centers for Medicare & Medicaid Services' Clinical Laboratory Improvement Amendments program; by accrediting agencies like the College of American Pathologists; or by individual states.

But now the FDA plans to directly regulate LDTs. That would be a mistake.

The current regulatory framework has ushered in bona fide medical miracles through the creation of LDTs for well over 1,000 genetic and genomic biomarkers.

Consider chronic myelogenous leukemia, a once deadly form of blood cancer. LDTs for this disease have helped many patients achieve a life expectancy approaching that of the general population and until last year, there was no IVD test available for it.

LDTs have also played a profound role in turning AIDS into a manageable chronic condition, and in converting acute promyelocytic leukemia, which was historically the most malignant human leukemia, into the most curable one.

They have spared thousands of patients with colon cancer the side effects of expensive medications from which they are unlikely to have benefited, and have allowed thousands of women cured of early stage breast cancer to avoid noxious and unnecessary chemotherapy. These examples only scratch the surface.

What about IVDs? The FDA approval process for these tests is lengthy and expensive. Obtaining approval for updates that keep tests current with rapidly advancing medical and scientific knowledge can be difficult and costly. Unsurprisingly, there are few genetic and genomic IVDs.

Consider the example of next generation sequencing (NGS), a revolutionary new technology that allows physicians to examine thousands of genes at high resolution with great accuracy. NGS-based LDTs are now the tests of choice for the diagnosis of inherited disorders and the detection of mutations used to select therapy in cancer patients.

This is the essence of precision medicine. Yet there still are no FDA-approved NGS oncology tests and only one FDA-sanctioned NGS genetic test. This latter test examines only a single gene.

There are no data showing systematic harms from LDTs, and some evidence of equivalent or superior performance compared to IVDs. LDTs provide safe, useful and analytically sound tests that can be quickly modified in response to advances in medical understanding. Moreover, many LDTs are legally available in Europe and elsewhere in the world. In contrast, the FDA review process can make IVDs less functional, less user-friendly and less safe.

What should the FDA do?

First, rather than expanding its reach, the agency should work to repair the deficiencies in its current oversight of IVDs. The agency should focus on whether IVDs analytically perform as the vendors say they do, with minimal clinical review.

Second, the FDA should examine products for operational safety issues.

Finally, the agency should stop requiring modifications to IVDs that make them more difficult to use.

In short, the FDA should focus on the mechanics of diagnostic tests and leave the medicine to the doctors. By repairing the broken regulatory system for IVDs, the FDA will greatly increase the available choices for laboratories and the number of high-quality tests available for patients.

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Fixing How FDA Regulates Diagnostic Lab Tests - Investor's Business Daily