Concerns, Support Weighed for FDA’s Proposed Regulation of Laboratory Developed Tests

Energetic debate surrounding the U.S. Food and Drug Administration’s (FDA) proposed regulatory framework for oversight of laboratory developed tests (LDTs) is coming to a head in the cancer research community. The official comment period on the FDA’s proposed guidance ended in early February, while the House Energy and Commerce Committee’s 21st Century Cures initiative discussion draft, released in late January, includes a placeholder for LDT language.

2014-10_lab Although a number of organizations have taken formal positions on the matter, the Association of American Cancer Institutes (AACI) continues to weigh the pros and cons and gather input from its members. Two AACI cancer centers—Memorial Sloan Kettering Cancer Center (MSK) and the Mayo Clinic—have publicly filed their reactions. If your institution has submitted comments to the FDA or the House Energy and Commerce Committee on the draft LDT guidance, please share your comments with AACI Government Relations Manager Jennifer W. Pegher,

In a letter to FDA Commissioner Dr. Margaret A. Hamburg, MSK’s David S. Klimstra, MD, and Marc Ladanyi, MD, expressed concerns about patient access to tests, innovation, and duplicative regulation:

“We support a thoughtful, phased-in approach to regulation of the highest risk LDTs, but urge the FDA to revise the draft framework to better safeguard both innovation and patients’ access to the LDTs developed in academic non-profit clinical laboratories that have been such a key element of recent advances in precision medicine for cancer patients. As currently written, MSK anticipates that a significant proportion of the LDTs offered by our Molecular Diagnostics service could be subject to premarket review requirements.”

Drs. Klimstra and Ladanyi made seven recommendations, including that FDA should:

  • Better define the term “LDT”
  • Clarify that a panel test will be treated as one LDT under the new framework
  • Clarify both the standard for and objective of demonstrating clinical validity for intended use for LDTs that are not subject to continuing enforcement discretion under this framework
  • Maximize reliance on existing evidence, consensus statements, clinical practice guidelines, and third-party review for LDTs subject to pre-market review

In a statement submitted to the U.S. House of Representatives Committee on Energy and Commerce Subcommittee on Health, Mayo Clinic’s Department of Laboratory Medicine and Pathology rejected FDA’s plan in favor of updated CLIA regulations.

Commenting on the section of the 21st Century Cures legislation headed, “A Modernized Framework for Innovative Diagnostic Tests”, Mayo wrote:

“[A]dditional layers of regulatory structure, including FDA approval, are not the answer to perceived problems with LDTs. We believe that the appropriate response is to update CLIA and add necessary requirements such as clinical validity, adverse event reporting, quality systems, post-market surveillance, etc. The 21st century modernization of the regulatory framework for LDTs needs to occur through updates to CLIA regulations rather than through additional FDA regulation of LDTs.”

Last fall, the American Association for Cancer Research (AACR) issued a statement supporting FDA regulation of molecular diagnostic tests, especially tests used to make high-risk treatment decisions, arguing that it will ensure patient safety while promoting medical product innovation.

AACR also made the following recommendations:

  1. Ensuring the safety, reliability and accuracy of diagnostic tests is vital to safeguard patients, advanced personalized medicine and promote innovation.
  2. Implementing a single, strict regulatory pathway through the FDA will help reassure clinicians, patients and the public that the tests used to make treatment decisions are safe, accurate and effective.
  3. Having a predictable and reliable regulatory environment is important to encourage an innovative biomedical ecosystem in the United States.

AACI hopes that the ongoing debate will lead to a regulatory policy that encourages and supports the innovation that is taking place at the nation’s cancer centers related to LDTs, while also assuring that patients are protected from premature incorporation of such testing in non-research settings.

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