FDA Seeks to Enable Patient Access to Diagnostic Tests While Ensuring Reliability

At a recent event hosted by Friends of Cancer Research, Scott Gottlieb, the head of the Food and Drug Administration, laid out several key principles for updating how medical diagnostic tests are overseen. He said the agency hopes to modernize the regulatory framework—which has not changed significantly in more than 40 years despite major changes in the diagnostics market—in a way that enables patient access to innovative new tests while ensuring quality and reliability. The agency’s principles align closely with those that The Pew Charitable Trusts recently published.

A single regulatory pathway for tests involving human samples

Clinical tests involving human blood or tissue currently come to market via one of two regulatory pathways: In the first, tests categorized as “in vitro diagnostics” are developed by manufacturers and must undergo FDA review before they can be used on patients in order to ensure accuracy and reliability. In the second pathway, tests classified as “laboratory developed tests”—which fall into a separate regulatory category because they are not developed by manufacturers but rather designed, assembled, and used in a single clinical lab—are not subject to this level of regulatory scrutiny. Technological advances and market evolution since the current regulatory regime was implemented have blurred the distinction between these two categories of test. Gottlieb said at the Friends of Cancer Research event that the agency “should have a consistent approach for all in vitro clinical tests… whether the test developer is a traditional manufacturer or a clinical laboratory.”

A risk-based approach

Gottlieb also emphasized that the new regulatory regime should be based on risk. In cases where an inaccurate or otherwise unreliable test would pose significant harm to a patient, the agency should review the test before it hits the market. Otherwise, the agency proposes to use a range of mechanisms to allow tests to reach the market without prior review. This includes exemptions for low-risk tests and tests already being sold. The agency would also be able to exempt certain tests under a new “precertification” program aimed at developers.

A risk-based approach is in line with long-standing FDA policy, but as Gottlieb noted, “To balance the change in premarket review, [it is] critical the agency be able to protect patient safety with strong postmarket oversight and enforcement authorities.”

Consistency with Pew’s principles

Gottlieb’s comments are closely aligned with the principles Pew believes should be part of any reform:

• Tests should be accurate and provide meaningful information. 
• Regulatory oversight should be tailored to risk.
• The regulator overseeing tests needs access to information and the scientific and regulatory expertise to review that data.
• To promote compliance and protect patients, enforcement authority must be adequate and clear. 
• Regulators need sufficient resources to ensure effective review without unnecessary delay.

Getting the details of a new oversight scheme right will require cooperation and dialogue among the FDA, the device industry, clinical laboratories, and other public health stakeholders. A system that effectively incorporates the priorities that Gottlieb described—a single regulatory pathway for all diagnostics using human samples and a risk-based oversight framework—would benefit patients by balancing access to diagnostic tests with mechanisms to ensure that they are trustworthy enough to be used as the basis for important medical decisions.

Liz Richardson directs The Pew Charitable Trusts’ health care products project.