In Diagnostic Innovation, Something Incredible Is Waiting to Be Known | Commentary

Posted September 12, 2014 at 2:09pm

Astronomer, scientist and writer Carl Sagan is thought to have said, “Somewhere, something incredible is waiting to be known.” I know what that statement means because I live and breathe the reality of discovery every day, as do my colleagues practicing at nonprofit clinical laboratories in academic medical centers across the nation. We are engaged in scientific innovation that has improved the lives of countless patients. Congress has shown admirable leadership in this area by launching the 21st Century Cures Initiative with the objective of ensuring regulation does not stand in the way of scientific achievements that are changing how we practice medicine.

Those of us in the field of scientific discovery understand that innovation impacting human lives can only flourish when balanced within a process that promotes safety and effectiveness. Thankfully, the world of laboratory developed testing (LDTs) currently has that balance, with a rigorous regulatory framework in place that allows profound discoveries and medical advancements to be brought to patients waiting for accurate treatments and medical solutions.

But recent action by the Food and Drug Administration threatens an existing regulatory process that has worked for decades and has facilitated breakthroughs in testing for HIV, SARS and countless common and rare diseases impacting both larger and smaller populations of people who are suffering and in need of answers. The FDA recently notified Congress of its intent to subject LDTs to its regulatory process, which currently oversees medical devices as defined by the Federal Food, Drug and Cosmetic Act — triggering numerous questions about statutory authority, how a physician, scientist or lab technician can be considered a manufacturer and how the performance and analysis of a lab test can be considered a device. LDTs are essential laboratory services and are the practice of medicine, not the manufacturing of medical devices. The FDA is not authorized to regulate the practice of medicine.

In the nonprofit world of laboratory research conducted in a controlled lab environment, our collective mission is to serve the best interest of the patient, patient care, the public health and health care innovation. Yet we fear movement from the FDA that would hinder our ability to continue ground-breaking discoveries. The FDA’s regulatory oversight would impose on LDTs a notoriously protracted review and approval process. This would slow the development and use of new tests for patients and their physicians, delaying life-changing information that will guide the next steps for treatment.

The current reins over safety and effectiveness of LDTs are strict and historically, very effective. LDTs are highly regulated under a three part framework consisting of federal regulations contained in the Clinical Laboratory Improvement Amendments, state laws, and accreditation by deemed authorities such as the College of American Pathologists. As a CAP inspector myself, I am part of this oversight of clinical laboratory physicians and scientists, performing careful inspections of lab facilities, exhaustive reviews of test protocols and validation, and continual monitoring laboratory performance. This regulatory framework requires both extensive validation and continuous monitoring to ensure the performance, quality and reliability of diagnostic services, and allows laboratories the flexibility to develop and validate lab tests quickly and rapidly respond to un-met public health needs.

Twenty-two academic laboratory leaders and I recently sent a letter to the Office of Management and Budget requesting the agency refrain from releasing FDA guidance that would add another regulatory layer to LDTs. We represent a non-profit community that bases our objectives on serving the public good through exemplary health care services and unencumbered diagnostic research that targets the greatest ills of humankind. While there has been significant pressure from ancillary interests to subjugate LDTs under lengthy and complex FDA regulation, surely the quest for innovation and an improved level of patient care should prevail.

Congress is hearing from stakeholders right now and it is crucial that in this policymaking age of correctly seeking to streamline regulatory processes that prioritize safety and effectiveness, we not take steps backward that impose new layers of requirements and place unnecessary obstacles in the way of innovative diagnostic advances.

It is important for lawmakers to know that nearly all diagnostic tests and FDA-approved test kits begin as LDTs, and in many cases, LDTs represent the standard of care. In fact, LDTs have been instrumental in defining and advancing personalized medicine, particularly in the cancer space. For some of the most widely characterized cancer biomarkers, including KRAS, and others, such testing was available as an LDT long before an FDA-approved or cleared test ever reached the market, in some cases, by almost a decade. It was the validation and demonstrated viability of these vital cancer diagnostics as LDTs which made the subsequent development and commercialization of an FDA-approved and cleared diagnostic possible.

We ask Congress to recognize the importance of a regulatory framework that provides for the development of safe, effective diagnostic tests without slowing the pace of innovative discoveries. Regulatory policy should be smart, diligent and leave room for something incredible to be known.

Edward Ashwood, M.D., is president and CEO of ARUP Laboratories and a professor of pathology at University of Utah.