The US Food and Drug Administration (FDA) has proposed draft regulations that could limit patient access to potentially life-saving therapies. 

What Do We Want?

Like the FDA, we want LDTs to be as validated, accurate, and clinically relevant as possible.  However, we also want the laboratories where these clinical testing services are performed to be able to exercise the flexibility, innovation and medical judgment necessary for good outcomes in thousands of patients.  This isn’t possible with the proposed FDA regulations.

We want the FDA to withdraw its proposed “Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs).”

To the extent there are concerns with the current oversight of these laboratories and their testing services, we strongly urge the agency charged with their oversight--the Centers for Medicare & Medicaid Services--to engage in notice and comment rule-making to modernize the CLIA regulations. 

Please sign our petition so we can get the word out. The FDA, as well as President Obama and Congress, need to know how patients and those who love them feel about this issue.

Why Care About LDTs?

Let's use cancer patients as an example.

Changes over time in certain genes (oncogenes) make normal cells become cancer cells.  Medical science can identify whether some patients’ cells are cancerous using a variety of techniques.  Some patients whose cancer cells test positive for certain oncogenes respond well to drugs that target those oncogenes, and can live months or years longer than patients who receive traditional therapies, often with fewer side effects.

Some drug manufacturers have obtained FDA approval of “companion test kits” along with approval of their new cancer drugs.  Examples are FDA-approved kits for BRAF-driven metastatic melanoma (approved along with vemurafenib) and ALK-driven metastatic non-small cell lung cancer (approved with crizotinib).  Each kit specifies the chemicals, equipment, procedures and patient specimens that may be used to detect one oncogene that responds to their specific drug. 

However, a kit does not detect every patient whose has the targeted oncogene.  Our knowledge of cancer-related genes and proteins continues to evolve after a kit is approved.  Trained medical and scientific professionals such as pathologists or molecular pathologists in Clinical Laboratory Improvement Amendment (CLIA) certified labs create new ways to detect the oncogene with laboratory developed tests (LDTs). Using validated scientific research, their medical savvy has found a new BRAF melanoma mutation that wasn’t detected by the approved companion test, and allowed ALK lung cancer patients with limited tumor tissue to be tested using cells obtained from fluid around the lungs.  Such innovation allows more patients to receive life-saving targeted therapies.

The Issue

The FDA’s proposed “Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs)” attempts to regulate LDTs as if they were self-contained devices (such as stents) or commercial test kits.  This won’t work.  Getting the best diagnostic and treatment outcomes from available specimens relies of the practice of medicine, which the FDA is not supposed to regulate.

The proposed regulations state that once a companion test kit obtains FDA approval, any modification to the test components or any change in specimen type requires separate FDA approval before the LDT can be offered to patients.  If no companion test exists for an oncogene, an LDT may offered to patients without obtaining FDA approval IF it is offered ONLY to patients in the same healthcare system as the laboratory that performs the test.

The proposed regulations require laboratories to submit all cancer-related LDTs for registration and listing.  In addition, LDTs for which a cleared or approved companion diagnostic exists (like BRAF-driven melanoma and ALK-driven NSCLC) must be submitted for financially prohibitive and lengthy premarket review.  Many labs, including those in major cancer centers (which currently offer hundreds of LDTs), might not have the deep pockets or other resources to seek FDA approval for their LDTs and would have to pull LDTs from their list of patient services, or even have to close.  This would impede not only access to potentially life-saving diagnostics, but impede innovation as well. 

For those LDTs that are submitted, FDA approval can take years, even with an accelerated approval process.  Many metastatic cancer patients could die waiting for already-validated LDTs to be approved by a regulatory agency.

These proposed FDA regulations could block patient access to an LDT whose analytical and clinical validity has been documented and demonstrated by qualified medical professionals in clinical laboratories – even when the patient’s physician has determined the test would be useful in guiding treatment decisions.

Molecular and genomic profiling offer hope that patients can get the right drug at the right time.  Metastatic cancer patients have waited for decades to access to such breakthroughs in treatment.  Knowledge of genomic variations, as well as ways of detecting them, is evolving faster than federal regulatory agencies can respond.  We must not throttle these breakthroughs with cumbersome regulations such as the proposed FDA framework.