Call to Action: Proposed FDA Regulations Could Limit Cancer Patient Access to Life-Saving Therapies


The FDA's framework for regulating lab developed tests could keep some patients from receiving validated diagnostic tests and targeted treatments like mine.

As posted in a recent CURE article, the US Food and Drug Administration (FDA) has proposed draft regulations titled “Framework for Regulatory Oversight of Laboratory Developed Tests (LDTs).” The FDA should withdraw this proposed framework because it could limit cancer patient access to potentially life-saving therapies.

Metastatic cancer patients have waited years for the hope that targeted therapies and genomic testing are now giving us. Don't let the FDA throttle our hope. Knowledge of cancer genomics and proteins is evolving faster than government regulation can move.

Of course, we all 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 cancer patients. This isn’t possible with the proposed FDA regulations.

Please sign the petition at, and ask your friends and family to sign. If you or a loved one has benefited from molecular or genomic testing, please say so in the comments. The petition and its comments will be submitted to the FDA as an official comment. The more signatures we have, the stronger our voice will be.

Here is a specific example of patient harm these proposed regulations might cause, taken from my own journey with metastatic ROS1-positive non-small lung cancer (NSCLC).

I live near Seattle. Because I was able to send my slides to University of Colorado for ROS1 testing, and my slides tested positive for ROS1, I was able to take crizotinib and achieve two years (and counting) of No Evidence of Disease. LDTs for ROS1 have been validated by medical research and have given many patients months or years of extra time.

Under the proposed regulations, some patients might have to travel to a distant or out-of-network medical facility to get the existing ROS1 test and receive treatment for their ROS1 cancer. In addition, some labs might stop offering the test because of the lengthy and cost-prohibitive process to obtain FDA approval. A medically validated test that is currently saving lives may become inaccessible to future lung cancer patients. The proposed FDA regulations would have effectively interfered with the practice of medicine.

Getting the best diagnostic and treatment outcomes from available cancer specimens relies of the practice of medicine, particularly the judgment and skill of pathologists, molecular pathologists and other molecular laboratory professionals. The use and safety of LDTs can’t be regulated in the same manner as self-contained medical devices such as stents, or commercial test kits that come with pre-defined instructions. Yet that is how the proposed regulations treat LDTs.

All cancer patients should have access to clinically validated tests that can help decide the best course of treatment. Please sign the petition to tell the FDA to withdraw its proposed regulations.


A more detailed version of my ROS1 example:

ROS1-rearranged non-small cell lung cancer (NSCLC) testing and treatment with crizotinib are recommended in National Comprehensive Cancer Network guidelines because crizotinib, an FDA-approved drug for ALK-positive NSCLC, “showed marked antitumor activity” against ROS1 NSCLC, with a 72 percent response rate in clinical trials—one of the highest response rates of any lung cancer drug—and a duration of response that exceeds 17.6 months. Crizotinib is currently available off-label for ROS1 NSCLC through most insurance companies because of its strong clinical evidence of effectiveness. Since most cancer treatment facilities do not offer an LDT for ROS1, they send slides to one of several CLIA-approved laboratories for testing, and treat ROS1-positive patients at home with crizotinib. Because no FDA-approved companion test exists for ROS1, any non-FDA approved LDT used to detect the ROS1 rearrangement would fall under the “LDT for Unmet Needs” exemption -- IF the LDT is ONLY used for patients within the laboratory’s healthcare system. Therefore, any lung cancer patient whose home clinic does not offer ROS1 testing would have to either send specimens to a laboratory with an FDA-approved ROS1 LDT (none are available at present), or travel for diagnosis and treatment to a facility that offers a ROS1 LDT within its healthcare system--even if the patient’s insurance doesn’t cover that system. Many patients may not be able to travel to another healthcare facility for ROS1 testing and treatment because they might be too sick, or face work, family, or financial constraints (insurance doesn’t cover travel expenses). Thus, under the proposed regulations, some future ROS1 patients would not be able to get crizotinib treatment, even though that treatment is available today at their home cancer clinic.

Another example from lung cancer:

Under the proposed regulations, when using an FDA-approved companion test kit (currently available for EGFR and ALK NSCLC), any change in equipment, reagents, or patient specimen type must be submitted to the FDA and obtain the FDA's approval before it can be offered to patients. Currently patients who have little tumor tissue (a common problem in lung cancer) can sometimes be tested for ALK based on cells obtained from pleural fluid or lymph nodes. Under the proposed regulations, only tumor tissue could be tested for ALK (that's what the FDA-approved test kit requires) unless laboratories submit their modified ALK LDT for FDA approval. Also, published medical research has demonstrated that sometimes patients test negative for ALK using the FDA-approved test, but those patients may test positive using alternative, validated testing methods (such as genomic sequencing) and respond well to crizotinib. Under the new regulations, those alternative testing methods won't be allowed unless they obtain FDA approval. Lung cancer patients who don’t have enough tumor tissue would either go without testing, or undergo risky biopsies in hopes of obtaining enough tumor tissue.

Image credit: "Researcher looks through microscope (2)" by Rhoda Baer. Licensed under Public Domain via Wikimedia Commons.

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