Ensuring Quality Pediatric Care Through Laboratory-Developed Tests

Laboratory-developed tests (LDTs) play an essential role in diagnosing and treating many of the conditions that affect pediatric patients. On February 20, the Association for Diagnostics & Laboratory Medicine (formerly AACC) and the Children’s Hospital Association hosted a congressional briefing where leading experts in laboratory medicine and pediatric health discussed the necessity of LDTs to timely and personalized pediatric care, and a duplicative rule issued by the Food and Drug Administration (FDA) that jeopardizes access to these vital tests.

The briefing was moderated by Dennis J. Dietzen, PhD, DABCC, FAACC, Megan Dishop Chair of Pathology and Laboratory Medicine, Phoenix Children’s Hospital, who began with an overview of laboratory testing experts’ main priority: the health and safety of patients across the globe.

“Laboratory developed tests are a key way we work toward this goal,” Dietzen said. “Most laboratory developed tests fill gaps where there’s no FDA-approved test available. Physicians depend on these tests to care for the most vulnerable patients—for example, to diagnose treatable genetic abnormalities in newborns … or decide when to perform surgery for cancer patients.”

Dietzen was joined at the briefing by two speakers, Meghan Delaney, DO, MPH, who is chief of the pathology & laboratory medicine division at the Children’s National Hospital, and Stephen R. Master, MD, PhD, who serves as the division chief and director of metabolic and advanced diagnostics at the Children’s Hospital of Philadelphia.

Delaney explained to the audience of congressional staffers that the final rule (Docket No. FDA-2023-N-2177), promulgated by the FDA on May 6, will regulate laboratories that develop LDTs as if they are medical device manufacturers. While manufacturers develop and sell their tests to labs across the country, laboratories develop LDTs specifically for their local patient populations, and only at the request of an ordering physician.

“The [FDA] rule will jeopardize patients’ access to these critical tests by creating unnecessary regulatory burdens and increasing costs for clinical labs,” Delaney said.

Master spoke next, detailing the potential local, regional, and national impacts of the FDA final rule on children’s health. Childhood cancer, child sexual abuse, and rare disease emergencies are a few areas where LDTs are integral to children getting the treatment they need and that showcase the importance of LDTs in a local pediatric care setting. In these scenarios, LDTs enable children to get faster diagnoses, potentially leading to improved patient care and outcomes.

Access to regional expertise is also critical, particularly for the timely treatment of patients with health emergencies at local pediatric hospitals. However, under the new FDA final rule, local pediatric and adult hospitals cannot send samples to each other for testing with a new LDT unless the LDT has undergone the full FDA submission and approval process. Because of this, rural and underserved communities may miss out on the expertise from regional hospitals. “This is a tremendously inefficient system that compromises patient care due to increased testing time,” Master said.

A recent survey of clinical labs from ADLM found that, when asked about whether they would seek FDA authorization for their LDTs or discontinue them if the new rule went into effect, 48 percent of respondents said that they would discontinue their tests. In the US, approximately 12,000 laboratories are qualified to perform LDTs, which means that almost 6,000 will stop offering these important tests. Ultimately, the FDA’s oversight will limit patient access to LDTs without improving pediatric care.

ADLM’s congressional briefing concluded with the speakers asking Congress to revoke the FDA’s rule and to collaborate with the laboratory medicine and healthcare communities to modernize regulation of LDTs.

In the meantime, the FDA’s rule is also being challenged in court. On February 19, Judge Sean Jordan in the Eastern District of Texas heard oral arguments from the American Clinical Laboratory Association (ACLA) and the Association for Molecular Pathology’s (AMP) lawsuit against the Department of Health and Human Services. The associations are challenging the FDA’s authority to regulate medical practice, and have pointed out that, according to CLIA, the authority to regulate LDTs already falls under the Centers for Medicare & Medicaid Services.

In October 2024, ADLM filed an amicus brief supporting ACLA’s position that the FDA’s rule exceeds the agency’s statutory authority and urging the court to strike down the rule before it causes irreparable patient harm.

Topics of the February 19 court hearing included the role of clinical validity and the history of the Federal Food, Drug, and Cosmetic Act and CLIA. ACLA and AMP requested an expedient ruling from Judge Jordan.

- This press release was originally published by ADLM