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Speech | In Person

Event Title
Remarks to the Precision Medicine World Conference
September 24, 2018

Remarks by Lauren Silvis

FDA Chief of Staff
The Precision Medicine World Conference 2018

FDA Commissioner Scott Gottlieb recently described FDA’s proposal for diagnostics reform as “a more modern, flexible approach to promote the extraordinary innovation that’s already well underway in this space, while ensuring patient protections.” 

I’d like to talk to you today about the thinking behind FDA’s proposal, and walk you through how we got here and why we think this approach could improve patient outcomes. 

You might recall that last year, shortly after joining FDA, Commissioner Gottlieb remarked that: “Some of the most creative, and forward leaning advances in regulatory policy involve what we’re doing on the device side of the house.”  He highlighted the device center’s embrace of real world evidence to support product approvals and its willingness to advance new policies to streamline premarket review and protect patient safety.

Before becoming Chief of Staff to Commissioner Gottlieb, I was fortunate to serve as a Deputy in FDA’s device center.  I know that many of the creative ideas that the device center put forward in recent years across the range of medical technologies started in the office that regulates in vitro diagnostics.

That office was its own little incubator for ideas.  For instance, it was the first to adopt a total product lifecycle approach to regulation.  It also found new ways to keep up with the iterative nature of devices, for example, by advancing policies that let manufacturers validate certain changes to diagnostics without making unnecessary premarket submissions.

The experience of piloting novel regulatory approaches balanced with advancing patient safety paved the way for the diagnostics proposal FDA recently put forward—a proposal that reflects real regulatory reform.

Given the how significant the potential impact of new legislation in this space could be, FDA recognized the importance of staying true to certain guiding principles in our feedback to Congress.

We began with the most fundamental premise–that it’s all about improving the health and the quality of life of patients. Patients rely on diagnostic test information to make decisions about their health regardless of who develops the test or where it comes from. This means we should not subject similar tests made by different developers to different standards.

We also put forward the concept that, because different tests present different benefits and risks, FDA should apply a flexible, risk-based approach.  One that would tailor the level of review and oversight to what’s appropriate for that type of test. We shouldn’t apply a one-size-fits-all pathway to the incredible range of in vitro clinical tests that are offered to patients.

Once the appropriate level of scientific review has been determined, we can efficiently select the right expertise and resources that should be applied.  As part of this plan for smart regulation, we also need to make sure we have the right pathway for tests that represent breakthrough technologies –  those that could significantly improve the lives of patients.

In taking a hard look at what a new world of in vitro clinical test regulation could look like, we considered the long-standing premise, rooted in our 40-year old statutory framework, that we had to start by looking at each individual test, regardless of the developer.

And that brings us to pre-cert—a critical alternative to premarket review for individual tests and certain modifications to tests.  I’d like to walk through how pre-cert might work for a range of in vitro clinical tests, from traditionally manufactured in vitro diagnostics, to many laboratory-developed tests.

Pre-cert is about evaluating the developer and its ability to design and validate a product.  And realizing FDA can leverage this evaluation so that a review of each product itself is not needed, or can be streamlined.

There’s a lot of interest in pre-cert, which our device center is piloting in digital health.  We’re doing this to make sure we keep up with technology that by its very nature is rapidly changing and improving.  And to make sure that we are maximizing opportunities for innovation.

But we’ve already employed a pre-cert like approach for certain tests.

Last year, FDA announced a framework to promote the rapid advancement of genetic health risk tests, while deploying FDA expertise efficiently and effectively to protect patients.  After authorizing the 23andMe Personal Genome Service Genetic Health Risk test for ten diseases or conditions, FDA proposed to exempt additional tests that provide information on an individual’s predisposition to other diseases or conditions.  This helps patients continue to get additional information about their health status, to share that information with their doctors and families, and to make better informed decisions about their own health.

At the same time, we set up the pathway to make sure that when the test is intended to provide a diagnosis and be used as the sole basis for major treatment decisions, like variants in the BRCA1 and BRCA2 genes, we’d still review the analytical and clinical validity of the test.  A balanced pre-cert approach such as this one could work for a range of tests that provide critical information to patients and their doctors.

Earlier this year, we announced a major step forward for a pre-cert like approach for certain tests for infectious disease.  FDA plans to propose to exempt certain mass spectrometry microorganism identification system processes from an additional premarket review after a system process receives a first-time FDA marketing authorization.

This could allow more rapid updates to device-specific organism databases and enable these mass spectrometry devices to expand their microorganism identification capabilities more efficiently, without the need for individual premarket submissions.

What does it mean for patients and their doctors?  This approach to diagnostics review could improve our ability to identify outbreaks from emerging infectious pathogens.

FDA announced these plans for future exemptions when it authorized the first test to identify the emerging pathogen Candida auris, which can cause serious bloodstream infections in hospitalized patients and is frequently resistant to multiple antifungal drugs used to treat Candida infections.  When FDA authorized the BRUKER MALDI Biotyper CA system for the identification of C. auris, it added to the system’s already cleared uses for the identification of 333 species or species groups, covering 424 clinically relevant bacteria and yeast species.

The ability to timely identify a wide-range of pathogens helps us address emerging outbreaks.  We are better able to direct the right treatment to the right patient. Overall, this approach to test regulation promotes innovation and improves patient care at the same time.  That’s exactly where we want to go.

These precursors to pre-cert formed the basis of our recent proposal to better manage the wide variety of in vitro clinical tests that are offered to patients today.  For developers that qualify for this path, pre-certification would allow for review of a single test, and the validation procedures associated with that test, to serve as an umbrella for clearance of a suite of related tests.

The pre-cert program would complement other reform proposals to form a more cohesive framework to promote innovation.

For example, FDA has suggested a streamlined process for review of modifications to diagnostics.  One that would better match the iterative nature of test development.  And which would fit within the life cycle of diagnostics in a way that helps foster continuous improvement.

However, to successfully apply an approach like pre-cert, and even more so if we are to grandfather non-FDA approved tests already on the market, we must include robust postmarket controls to protect patients and appropriately balance the more efficient focus of our premarket review resources.

And a final, yet critical core principle in FDA’s proposed framework is the need to for an efficient approach that leverages – rather than duplicates – the current oversight of clinical laboratories.  For example, we believe that incorporating a robust third-party review system is essential to managing FDA’s resources.  FDA recently issued a draft guidance that provides a comprehensive outline of FDA’s approach to improving the third-party review program for certain well-understood, lower-risk medical devices.

For eligible submissions, FDA will review the expert recommendations of the third party to make its final decision and generally will not re-review the submission itself if the third party has demonstrated its proficiency in making well-informed recommendations. Leveraging high quality, FDA-equivalent outside reviews for lower-risk products can improve the FDA’s overall productivity and enable the agency to focus more of its time and resources on evaluating applications for higher-risk and more complex devices.  This approach shows how we are making our evaluation more efficient, while maintaining the FDA’s gold standard for product review.

As we’ve engaged in discussions about the feedback FDA provided to Congress, I’ve heard the questions “Can FDA really do this?  Can FDA fundamentally change the way it regulates an existing product category?  Can FDA expand the scope of its active regulation in a way that promotes innovation in a space that’s rapidly evolving?”   
The answer is yes.

The ideas in FDA’s feedback come from a thoughtful exercise where we carefully considered what works well for diagnostics, and what new ideas could advance patient care.  And we took seriously the stakeholder input over the years as we put together a description of a potential framework that’s more ideally suited to meet the needs of today’s technologies.

And the reason I know FDA will be able to truly embrace new ideas, and be a willing partner in diagnostics reform, is that many of these approaches come from the center that’s been putting forward some of our most creative regulatory solutions.  A center that last year approved a record number of novel technologies, building off a series of changes that has increased the annual number of novel technologies approved in the US by four-fold.

We look forward to continuing to work with Congress and stakeholders on moving this approach forward – an approach that we believe would be a transformative driver of innovation and patient benefit in this space.

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