Posted 03 September 2014
By Alexander Gaffney, RAC
Last month, the US Food and Drug Administration (FDA) announced it would soon seek to regulate lab-developed tests more similarly to in vitro diagnostics (IVDs), citing the devices' increasing complexity and role in critical clinical decision-making.
And while the plan has received some pushback from legal groups, including the Washington Legal Foundation, it might soon have another, more prominent opponent: Members of Congress.
LDTs are, as their name implies, diagnostic tests developed and used exclusively within a single laboratory.
Unlike their IVD counterparts, which are regulated either through the 510(k) or premarket approval (PMA) process, LDTs have generally been exempted from regulation.
"Initially, laboratories manufactured LDTs that were generally relatively simple, well-understood pathology tests or that diagnosed rare diseases and conditions that were intended to be used by physicians and pathologists within a single institution in which both were actively part of patient care," FDA explained in a 2010 meeting notice. "These tests were ordinarily either well-characterized, low-risk diagnostics or for rare diseases for which adequate validation would not be feasible and the tests were being used to serve the needs of the local patient population."
But in recent years, FDA has noticed LDTs becoming increasingly complex and in some cases nearly indistinguishable from their FDA-cleared or -approved counterparts. Of particular concern to FDA is that many LDTs play critical roles in clinical decision-making in the context of personalized medicine (e.g. genetic testing), which it said raises the risk of incorrect or missed diagnoses, resulting in untimely or improper treatment.
The agency has long asserted its right to regulate the devices, but since 2011 has begun to move strongly toward a system of stricter regulation of LDTs, albeit not quite as strict as the current system for in vitro diagnostic devices. That's because FDA said it "recognizes that while the absence of FDA oversight may make it easier for laboratories to develop and offer tests on a rapid timeline, the absence of a level playing field creates a disincentive to innovation by other manufacturers whose tests are approved or cleared by the agency."
On 1 August 2014, FDA released a new draft guidance document, Framework for Regulatory Oversight of LDTs, that proposes regulating LDTs more or less like IVDs.
Under FDA's proposed regulatory framework, moderate-risk LDTs will be required to be registered and listed with FDA, meet adverse event reporting standards and undergo premarket review starting five years after the guidance is implemented.
High-risk LDTs will need to meet all those requirements listed above, but will instead be required to submit to a premarket approval process within one year of the guidance document's implementation.
Some LDTs will be subject to far fewer requirements. Low-risk devices and LDTs intended to treat rare diseases will only be subject to registration, device listing and adverse event reporting requirements. Devices used solely for forensic purposes and devices intended to facilitate organ transplants in CLIA settings will also be exempt, FDA said.
For more, please see our 1 August 2014 story on FDA's LDT guidance document.
Guidance on Hold
But the draft guidance was unusual in one notable way: FDA wasn't actually formally releasing it for comment. Instead, the agency was announcing that it planned to release the guidance in the coming weeks.
That's because under Section 1143 of the Food and Drug Administration Safety and Innovation Act (FDASIA), FDA is required to notify Congress at least 60 days prior to the release of "any draft or final guidance." Specifically, FDA is required to notify both the House Energy and Commerce (E&C) Committee and Senate Health, Education, Labor and Pensions (HELP) about the pending release of the guidance.
And perhaps unfortunately for FDA, the House Energy and Commerce Committee is in the midst of its 21st Century Cures Initiative, which is tasked with determining how medical advances and cures can be brought to patients more quickly, with a specific focus on re-tailoring regulations to be more flexible.
That could be problematic for FDA. At least one prominent member of the committee, Rep. Michael Burgess (R-TX), has called FDA's regulatory approach to LDT's "redundant," and claimed it will "stifle innovation." And at a time when Congress is trying to figure out how FDA can facilitate faster access of proven treatments to patients, the agency's plan to regulate LDTs could get a chilly reception at a just-announced meeting.
In a notice posted to its website on 2 September 2014, the House E&C Committee said it plans to hold a hearing on 9 September 2014 entitles, "21st Century Cures: Examining the Regulation of Laboratory Developed Tests."
Ominously for FDA, which will be represented at the committee hearing by Jeffery Shuren, director of its Center for Devices and Radiological Health (CDRH), the committee's meeting notice contains a thin veil of hostility.
"Two central goals of [the 21st Century Cures] initiative are encouraging innovation and embracing the rise of personalized medicine," the notice reads. "The conversation will continue next week with a hearing to discuss recent guidance offered by the Food and Drug Administration regarding the regulation of Lab Developed Tests (LDTs). Members will hear testimony from various witnesses on the guidance and its impact on innovation and the practice of precision medicine."
In other words, legislators will be focusing on the things FDA isn't emphasizing about its new LDT policy, which is the safety and efficacy of those devices. Instead, legislators will be focusing on whether it could stifle innovation and whether the policy could affect "precision medicine."
While FDA's proposal may well survive the meeting intact—legislators have said they don't expect any legislation that could challenge the LDT guidance prior to the end of 2014—the meeting might still signal the start of some serious trouble for FDA's approach to LDTs.
Committee Hearing Notice