Personalized Medicine Education and Advocacy

Thought leadership in personalized medicine

Think the Diagnostics Community Doesn’t Agree on Anything When It Comes to LDT Regulation? Think Again.

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by Amy M. Miller, Ph.D., Executive Vice President, Personalized Medicine Coalition


Amy M. Miller, Ph.D.

As we enter a new round of discussions about laboratory-developed test (LDT) regulation, it is helpful to review where we’ve been. Many stakeholders have weighed in on the topic, and GenomeWeb’s comprehensive summary of the different proposals offers a useful comparison of them. At first glance, there appears to be little or no consensus. But listening to the community reveals more.

At the beginning of this year, I moderated a series of discussions on potential legislative solutions with representatives from the entire LDT community, including but not limited to those with an interest in personalized medicine. In short, the community agrees that a legislative solution to LDT regulation should take a risk-based approach and:

  1. Protect public health labs. Public health labs should be protected by any regulatory paradigm, which means that sentinel labs must be able to develop, deploy and use rapidly developed diagnostics to address critical public health needs.
  2. Allow flexibility and efficiency when managing modifications. As diagnostic device developers have long argued, the way modifications are managed by a regulatory system should be flexible and efficient to allow diagnostic tests to evolve with the clinical science that underpins them.
  3. Mitigate regulatory burdens for government and industry. To reduce regulatory burdens on government and industry, regulatory agencies should, when appropriate, recognize when certain safeguards are already in place. These mitigation strategies can help regulatory bodies keep pace with the rapidly evolving pace of personalized medicine diagnostic testing.
  4. Design a grandfathering system for tests already on the market. When FDA published its draft framework for regulating LDTs, we had no clear appreciation of the number of tests that might be captured by it. While we still do not have an exact count, tech firm NextGxDx estimates that there are nearly 70,000 personalized medicine diagnostics offered by about 300 labs with another eight to 10 coming to market each business day. To manage such an enormous workload, a regulatory agency must design a grandfathering system that will allow most tests to remain on the market unless there is a compelling reason to remove them.
  5. Ensure regulatory burdens reflect testing volumes. Regulatory burden must be reflective of testing volume.  For example, diagnostics designed for rare and un-met needs should be given careful and different consideration by any regulatory agency to ensure that tests are developed for micro-markets.
  6. Accept valid scientific evidence for regulatory purposes — even if that evidence does not include data from a randomized control trial. Personalized medicine has challenged how health care products and services are conceived, developed, regulated, covered, paid for and used by physicians.  Evidentiary requirements for regulatory review must also evolve. The community agrees that for diagnostics, valid scientific evidence should be acceptable for regulatory review, even when that evidence does not include data from randomized control trials.

Understanding these points and the logic behind them is essential to progress on this topic. Fortunately, we are not starting from scratch.

While there is no consensus about which regulatory agency should manage LDT regulation, the House Energy and Commerce Committee has released a draft legislative solution designed to address the community concerns outlined above. That proposal builds on a rich dialogue that began when the first genetic tests entered the market and continued in 2007 when Senators Ted Kennedy (D-MA) and Gordon Smith (R-OR) released a bi-partisan proposal for FDA to actively regulate lab tests. Soon after, then-Senator Obama and Senator Richard Burr (R-NC) released a draft legislative proposal that was not quite as burdensome. Finally, in 2010, Senator Hatch outlined a novel path at FDA for diagnostics, which opened up the conversation about the true difference between diagnostics and the medical devices that their regulatory structure mirrored.

These historical efforts stimulated conversation, and what was learned has influenced how we consider the topic. The Committee’s next draft will have been improved by stakeholder input, and we can expect the Senate to continue improving on the next draft.

Addressing this duel path to market and the difficulties inherent in such a regulatory paradigm is essential to the field.  Once this debate is settled, we can all concentrate on the biggest issue in personalized medicine: coverage, payment and use of personalized medicine diagnostics to dramatically improve the care patients receive. Guided by these areas of agreement and rich historical dialogue, we may be able to focus on those conversations sooner than we think.

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