FDA Seeks Public Comment on Framework for Regulation of Laboratory-Derived Tests (LDTs)

The FDA is now seeking public comment on a guidance document, Framework for Oversight of Laboratory Tests, that will launch the agency’s entry into a new role regulating a sector of laboratory testing, known as laboratory-derived tests, or LDTs. These tests are offered as services from commercial laboratories, and are developed by the company itself. With respect to the field of genetic testing, LTDs are the predominant form of testing available to the public, where a consumer can send a biological sample to a lab for testing (direct to consumer tests), or provide a sample to a health care provider who mediates the testing process (see here). For years, the FDA has declared that while it has the legal authority to regulated such tests, it has exercised “enforcement discretion" – deciding not to require premarket approval from labs seeking to offer such services. As this marketplace has grown, the agency has moved closer to establishing a more formal role over the quality of these tests, and now has done so with its announcement of how it will require the industry to engage with the FDA in order to offer these tests (see here for the agency's new notification requirements for laboratories). The new proposed framework contains the following observations from the FDA as to why it needs to step up now: 

LDT’s are important to the continued development of personalized medicine, but it is important that in vitro diagnostics are accurate so that patients and health care providers do not seek unnecessary treatments, delay needed treatments, or become exposed to inappropriate therapies. 

The FDA has generally not enforced premarket review and other applicable FDA requirements because LDTs were relatively simple lab tests and generally available on a limited basis. But, due to advances in technology and business models, LDTs have evolved and proliferated significantly since the FDA first obtained comprehensive authority to regulate all in vitro diagnostics as devices in 1976. Some LDTs are now more complex, have a nation-wide reach and present higher risks, such as detection of risk for breast cancer and Alzheimer’s disease, which are similar to those of other IVDs that have undergone premarket review. 

The FDA has identified problems with several high-risk LDTs including: claims that are not adequately supported with evidence; lack of appropriate controls yielding erroneous results; and falsification of data. The FDA is concerned that people could initiate unnecessary treatment or delay or forego treatment altogether for a health condition, which could result in illness or death. The FDA is aware of faulty LDTs that could have led to: patients being over- or undertreated for heart disease; cancer patients being exposed to inappropriate therapies or not getting effective therapies; incorrect diagnosis of autism; unnecessary antibiotic treatments; and exposure to unnecessary, harmful treatments for certain diseases such as Lyme disease. 

The House Energy and Commerce Committee Subcommittee on Health held a hearing on the proposed framework last month. The agency has the guidance document open for 120-day public comment period, which will last until February 2, 2015.