Lab Testing in the Era of Precision Medicine

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Regulations that work for the benefit of patients

LEGAL/REGULATORY ISSUES

[Editor’s note: Dr. Kaul served as a witness at the U.S. Senate Committee on Health, Education, Labor & Pensions hearing on Laboratory Testing in the Era of Precision Medicine on Sept. 20, 2016; her complete written testimony can be read here.]

As precision medicine rapidly evolves, new opportunities to utilize this information and advance our approaches to patient care are frequent, and in order to continue improving outcomes for patients, our laboratories must be able to keep pace.

Recently, I had the opportunity to testify before the U.S. Senate Committee on Health, Education, Labor & Pensions on the topic of pathology and laboratory medicine, specifically how tests used in the lab support precision medicine. Our discussion focused on the current state of regulatory oversight for laboratory developed testing procedures (known as LDPs or LDTs), and the extent to which they should be further regulated by the FDA.

Current LDP oversight is provided by regulations from the Clinical Laboratory Improvement Amendments (CLIA). Although LDPs regulated by CLIA have a long history of success, the rapid growth and use of these tests has raised visibility, prompting concerns that additional oversight from the FDA is needed to ensure all new LDPs meet certain standards. While the FDA’s role is valuable in specific areas of laboratory testing, such as the approval of manufactured test kits, its lengthy review process could prove detrimental to patients.  Specifically, patients need personalized test results in compliance with new knowledge and nationally accepted treatment guidelines. Tests must be developed, and validated and offered clinically in a timely manner to meet these needs.

Patient Safety

For laboratory professionals, accuracy and patient safety are our highest concerns. The patient is the focus of what we do every day in the lab, and for us, CLIA is a way of life. Years of results have shown that lab testing under CLIA regulations has been extremely beneficial to patients, because these regulations extensively define the details for laboratory operation, test validation, staff requirements, training, and more.

The patient is the focus of what we do every day in the lab, and for us, CLIA is a way of life.

Under CLIA, labs participate in ongoing quality activities, such as testing of unknown samples to ensure that test results are correct and match those of other laboratories.  Many tests developed in the 1990s are still successful today under CLIA-based programs, which ensure that results are accurate and reproducible. LDPs regulated by CLIA also grant labs flexibility to meet the evolving needs for new tests to improve patient care.  In fact, many FDA approved tests are based on successful LDTs established and used under CLIA.

Receiving FDA approval for a test kit begins with providing detailed clinical trial data followed by a lengthy review process. As a former FDA panel expert, I understand there is high value in the FDA process, but such a complex approval process is not well suited for precision medicine that requires more rapid approval of new tests, and innovation.

It is often assumed that all lab tests are performed with a kit or on a machine.  In reality, most procedures are performed with the direct involvement of a laboratory professional or physician, such as myself.  In general, our work in the lab is not limited by a “test kit.” Rather, we begin by examining the tissue or sample ourselves, and then determine what additional tools are needed to provide complete information for clinicians to treat their patient appropriately.  These lab-based procedures must be validated and optimized within each laboratory and this is done under the regulatory oversight of CLIA.  Using only FDA-approved test kits would not fully serve the needs of labs, physicians and patients.  Cancer patients tested with these kits often do not receive the full set of results they need to map their personalized treatment plan, in accordance with national consensus treatment guidelines.

Additionally, labs are constantly faced with new diseases and infectious agents threatening our public health, such as we have seen in past years with influenza, or the current situation with Zika. Community and academic hospital labs are often the first responders during these public health outbreaks.  Labs need to be part of a coordinated public health effort, with access to timely test methods and results.  Rapid diagnosis will help to manage hospital resources, and limit infection spread via appropriate isolation efforts.  Further, it is unacceptable for patients facing urgent health implications to wait weeks for their test results.

However, to ensure patient safety, the expansion of CLIA regulation and compliance programs should be considered. As CLIA would not require a redundant new submission and reporting mechanism, CLIA oversight is better suited than the FDA to support the rapid evolution of precision medicine and is a framework within which labs currently operate. Providing a defined set of standards and reference materials could also be useful for labs under CLIA to demonstrate their performance and quality before offering a new clinical test. The expansion of CLIA would not aim to diminish FDA’s role in the lab, but the FDA’s role should be specific to in vitro diagnostics (IVDs) test kits, which are manufactured to work in various labs and settings across the country.

Despite differences in methodology and practice, the overarching goal for all of us, physicians and regulators alike, is to ensure the efficacy and safety of lab tests and procedures for patients. Our shared duty is to focus on the patient at all times.

We’ve heard so much about the “promise of personalized medicine,” and what that means for our current and future generations. To meet this promise, lab test results must be complete, cost effective, timely and accurate, but also keep up with the pace of changes in medicine today. Stakeholders need to collaborate to ensure that the regulations governing labs and lab professionals are the most appropriate and beneficial for patients.

 

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About Author

Karen Kaul, MD, PhD
Karen Kaul, MD, PhD

Karen Kaul is chair of the Department of Pathology and Laboratory Medicine and Duckworth Family chair at NorthShore University HealthSystem, and a clinical professor at the University of Chicago Pritzker School of Medicine.

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