FDA scrutinizes LDTs

Sept. 1, 2010

At this meeting, FDA officials — and those in attendance, for that matter — got quite an earful about what is happening in the world of clinical lab testing. Whether it was the story of a lab in Texas developing a new test to diagnose H1N1 or the constantly improving statistics on survival rates for leukemia, HIV, and other diseases, one could not help but see the bright, exciting future for diagnostic medicine and the patients who benefit from it.

This creates a quandary for the regulators. With the current progress in medical testing and the dramatic leaps on the horizon with advancements in genetic testing, how will the federal government walk the fine line of ensuring public safety without discouraging lifesaving innovation?

As the FDA considers the type of oversight necessary for laboratory-developed tests, it should be apparent that LDTs cannot and should not be wedged into the same regulatory template used for pharmaceutical products or medical devices. Rather, a unique and innovative approach is necessary for this particular brand of medical innovation. The FDA does not decide a drug is safe for patient use until it has undergone different phases of clinical trials that can take years to complete. And when a manufacturer makes a significant improvement in a device, it may have to go through an arduous approval process once again. In the clinical laboratory, tests are constantly being adjusted and refined to reflect new medical and scientific information. Adjustments are made in the way samples are collected, measured, and evaluated to yield the most accurate and relevant information. An approval process similar to that used for drugs and devices will slow the healthcare system's ability to diagnose illness.

Clinical laboratories are not, in fact, “traditional” medical-device manufacturers. Applying a strict paradigm based on traditional medical devices to all LDTs will have major consequences to clinical laboratories and will have the unintended consequence of slowing the availability of these important tests and negatively affecting patient care. Also, a number of tests are used for diagnosing rare diseases like Tay-Sachs and Gaucher, making clinical trials virtually impossible.

LDTs cannot and should not be wedged into the same regulatory template used for pharmaceutical products or medical devices.

Developing an effective, innovative approach to oversight of LDTs does not have to mean creating a large, cumbersome new regulatory apparatus. Instead, we should look at what we currently have in government oversight, weigh it against what the public needs, and then fill in the gaps. Right now, FDA regulation primarily concerns genetic tests manufactured and sold as packaged test kits, while the Centers for Medicare and Medicaid Services (CMS) oversees the work done in labs through the Clinical Laboratory Improvement Amendments. The immediate task at hand is to determine, between these two agencies, where oversight needs to be extended to cover the rapidly evolving world of LDTs. Categorizing different LDTs based on risk, and then determining the appropriate evidence requirements for each category — rather than painting them all with same broad regulatory brush — is worth consideration.

There is so much at stake for patients in the regulation of LDTs that the development of this oversight needs to evolve from a constructive and ongoing dialogue between the FDA, CMS, Congress, and other stakeholders, as there are responsibilities on all sides. Regulators need to realize that the clinical laboratories are the experts on how tests are developed and validated, and how quality is ensured. Laboratories must always be conscious of the need to provide information about ongoing developments in diagnostic medicine to the public sector.

We all have the same priorities: to ensure patient safety without causing disruption in the practice of medicine, or delaying patient care, or slowing the pace of healthcare innovation. Working together, we can fill in the current gaps in regulation and create an effective approach that achieves all of these objectives.

As new rules are being considered, we also need to communicate the human element involved. Regulators need to know about HIV patients whose life spans are longer because of genetic tests that can measure their resistance to drugs, about mutation testing that can mitigate the side effects of colon-cancer treatment, or — if we want to look at the financial aspects of healthcare —about the billions of dollars that are saved through early diagnosis of chronic illnesses like diabetes and heart disease.

In his remarks at the FDA meeting, Sharfstein referenced Daniel Carpenter's book, Reputation and Power: Organizational Image and Pharmaceutical Regulation at the FDA. Carpenter points out that the FDA has become the most influential regulatory agency in the world, with a reputation — developed over decades — for competence and vigilance. Today, the agency is facing perhaps its most important challenge. We stand on the verge of a new era defined by unparalleled success in conquering disease. To usher that era forward will require a light regulatory touch that is creative, innovative, and forward thinking. I have no doubt that, with our participation, the FDA will rise to the occasion.

Alan Mertz, president of the American Clinical Laboratory Association, represents association members' interests before Congress. In 2007, the organization launched “Results for Life” (www.labresultsforlife.org) to promote the value of laboratory services.