By Diagnostics World Staff
September 13, 2018 | During a speech today given to the Friends of Cancer Research’s 7th Annual Blueprint for Breakthrough Forum, US Food and Drug Administration (FDA) Commissioner Scott Gottlieb announced that the FDA is assisting Congress in crafting new legislation that will help shape a modern framework whereby diagnostics can be relied on as accurate and safe.
“Narrowing this [diagnosis] information gap is a key to making sure patients get the right treatment and timely care,” Gottlieb said during his speech. “It’s also key to helping resolve some of the irksome ambiguities of serious illness.”
Gottlieb said that recent advances have made the treatment of disease, as well as the opportunities of predicting disease more effective than ever, making the point of diagnosis critical.
“As clinical testing gets more and more sophisticated, we need to make certain that we have the right regulatory framework in place to ensure that these new clinical tests—including ones that we cannot even envision today—are accurate, reliable, and safe,” he said. “And that these tests are made accessible to patients as soon as possible.”
The right framework, Gottlieb said, must give doctors and patients more of the information they need to pursue the right treatment at the right time, and for the right patient. This framework must work well across the spectrum of tests and the patients who need them, as well as recognize the significant benefits to a diagnostic test, especially for certain serious conditions.
“Such a framework is readily achievable and it’s something we should all expect,” said Gottlieb. “And the FDA is working closely with our partners to make this framework a reality and has taken new steps to bring it about.”
The FDA has provided the outline for the aforementioned framework to Congress, Gottlieb said, adding that three core principles are fundamental to modernizing diagnostics oversight.
First, there should be a consistent approach to in vitro clinical tests.
Second, the FDA must focus its premarket review on the analytical and clinical validity of a subset of tests either higher risk or highly novel.
And finally, the FDA must have an efficient approach the current framework that’s in place. This includes, according to Gottlieb, the requirements that apply to clinical laboratories under the Clinical Laboratory Improvement Amendments, and leverages the laboratory test expertise of third-party organizations.
“The approach we proposed would grandfather most laboratory-developed tests currently being offered to patients,” Gottlieb said. “Under this framework, all in vitro clinical tests would fall under a unified set of requirements that would also exempt from pre-market review many categories of new or subsequently-modified tests.”
To help facilitate this, Gottlieb said part of the FDA’s feedback to Congress includes a proposed precertification pathway, which builds on an approach already applied by the FDA to direct-to-consumer genetic health risk tests.
In what Gottlieb calls a “compelling new paradigm,” the FDA is also seeking to establish a collaborative community of scientists, clinicians, test developers, and patients to support the agency’s decision making. For example, said Gottlieb, the FDA would work on an on-going basis with oncologists, pathologists, and other outside experts in real time to develop standards for analytical validity for different types of tests. This would help “develop agile regulatory expectations for the most novel technologies, like NGS,” Gottlieb argued.
The FDA is working to keep pace with advances in diagnostics, the commissioner said, but their efforts are constrained by a framework that’s existed since the mid-70’s.
“The decision to exercise enforcement discretion over laboratory developed tests was made in a different time and under different circumstances,” said Gottlieb (FDA most recently set out to regulate laboratory-developed tests in 2014). “We must ask whether that approach still makes sense?... If the opportunity exists to modernize our approach to diagnostics regulation through legislation, FDA is a willing partner in those efforts.”
By modernizing the framework, Gottlieb envisions a scenario where fewer than 10% of all tests would require individual premarket review, about 40% of tests would be eligible for a precertification pathway, and more than 50% of tests would be exempt from premarket review.
“The FDA has an obligation to regulate diagnostics, and I believe we should do so in a way that advances opportunities for innovation, while maximizing protections for patients,” Gottlieb concluded. “We must be flexible and risk-based about where we focus our resources and expertise. We must modernize our approach to keep pace with the technological change that’s underway. I want to assure you that the FDA is committed to working with Congress to move forward on all these goals.”