Statement from FDA Commissioner Scott Gottlieb, M.D. as prepared for oral testimony before the U.S. House Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies, Committee on Appropriations

FDA Statement

• For Immediate Release: February 27, 2019

Chairman Bishop, Ranking Member Fortenberry, and members of the subcommittee. I want to thank you today on behalf of the FDA for your continued support of our public health mission. We’re witness to a period of historic scientific advance. The opportunities we have, across our entire portfolio, to advance health and wellbeing, far exceed any comparable period of rapid technological change. Simply put, we’re in a period of unparalleled medical progress.

Your support is helping us seize these opportunities to improve the lives of Americans. The FDA’s Fiscal Year 2019 budget includes one of the most substantial increases for the FDA in recent years. The initiatives that Congress supported will help uncover new treatment options for patients suffering from debilitating conditions, improve competition to help lower drug costs and improve patient access to medicines, and advance development of treatments for rare diseases. The new funding will also help us better secure consumer safety by improving our oversight of food products; and modernizing our approach to medical product safety and manufacturing. These are just some of the opportunities enabled by the new funding that Congress provided to the agency.

The same rapid pace of science requires us to also modernize our own policies, to make sure we can fully leverage these opportunities.  To complement the support, you’ve given us to secure these scientific advances, we’re undertaking one the most significant modernization effort in decades for our Office of New Drugs. This new effort encompasses steps to update our approach to drug review across every stage in the life cycle of a new innovation, from the time an investigator first asks the FDA for permission to begin the clinical testing of a new drug to how we continue to assess the safety and effectiveness of new medicines after they’re approved by the agency.

I’d like to take this opportunity to tell you about some elements of this undertaking, and how I believe our policy efforts will extend the opportunities from the resources you’ve provided us.

These steps begin at the outset of the new drug development process, when a sponsor files an Investigational New Drug Application (IND) seeking permission from the FDA to administer an investigational drug or biological product to patients for the first time. Beginning in the next few months, our Center for Drug Evaluation and Research (CDER) will adopt new standard templates for our 30-day IND safety reviews and protocol reviews. This will better integrate the work of clinical and scientific reviewers, improve the consistency of the IND review process, reduce duplicative paperwork, provide greater efficiency and predictability to product developers, and enable the FDA to be more effective and strategic in premarket safety discussions with sponsors during its review of these INDs.

By using standardized templates for the submission and review of these INDs, we believe the consistency will help investigators more efficiently advance new products into development, while making sure that the FDA has the information it needs to help safeguard patients.

As part of our pursuit of more standardized methods for the assessment of information across all aspects of the drug review process – and to improve the rigor and predictability of these methods – we’re also extending these same approaches to how we assess product safety. We’re launching a “safety signal tracker” to serve as a repository of important potential safety issues throughout a drug’s lifecycle, from early in IND development through application review and into the post-market setting. This consolidates information about safety concerns during drug development in a single location. In this way, safety questions can be more consistently tracked, annotated, and continuously evaluated through every stage in the life cycle of a new medical product. Reviewers don’t need to hunt for information across multiple databases and documents. All team members share a common view of key information. This helps cement a more systematic approach to how we continue to evaluate certain safety questions throughout the new drug or biological product application review and could measurably improve the drug development process.

As part of this process, the Office of New Drugs has also started conducting meetings early in the drug review cycle to scope-out potential and known safety issues. This helps make sure potential safety questions can be more thoroughly addressed at mid-review-cycle meetings. This, in turn, facilitates more proactive planning to evaluate or mitigate known or perceived risks. Finally, we’re making the process more effective by adding clinical data scientists to our new drug and biological product safety review process, and incorporating standard tables and figures supporting the team’s analysis of safety data. Once again, the aim is to make the entire process more structured and predictable.

This same commitment to using modern tools and policies to achieve a more structured approach to drug development extends into our review of clinical evidence in new drug applications. For the first time in more than 20 years, we’re undertaking the first update of our guidance outlining how we assess the clinical effectiveness for drugs. A lot has changed since we first recommended in guidance in 1998 how investigators should conduct trials to support a drug’s effectiveness. Our approval standard remains unchanged. But the science and data that we evaluate as part of drug review, and the diseases that are more often being targeted by new medicines, has changed a lot. We have much more opportunity to use a broader array of data as confirmatory evidence to help support product review. This includes real world evidence and real world data.

At the same time more, new medicines target rare diseases, where the approaches to the design of clinical trials are carefully tailored to the kinds of clinical circumstances under which new drugs are being studied. Our new guidance on evidence for establishing clinical effectiveness seeks to accommodate these changes. It describes many of the more modern approaches we’ve been recommending when it comes to the design of trials and the generation of evidence to support product review.

These same general approaches extend to our efforts to modernize how we assess post-market drug safety. As part of the New Drugs Regulatory Program Modernization, we’ve launched a new effort to analyze more safety data more efficiently across the entire drug lifecycle, including in the post-market setting.  We’re implementing changes to capture more types of safety data, enhance our data analytics, and grow Sentinel’s ability to detect potential new safety problems.

We’ll be working to link claims data in Sentinel to electronic health records, to improve our ability to conduct active surveillance and use real world data to improve patient outcomes.  We’ll be using the new resources as part of our 2019 budget to help advance these efforts. By linking information from electronic health records to the data we have on medical claims through our existing Sentinel system, we’ll be able to get a much more complete picture of potential safety issues post-approval and to advance the use of real world data to study drug effectiveness.

As another part of these efforts, I’m pleased to announce that the FDA’s Adverse Event Reporting System for drugs will be expanded to cover more types of safety data. For the first time, this system will also include pre-market drug safety data. Specifically, FAERS will include IND safety reports, which are information on serious, unexpected adverse reactions in clinical trials. It will also include product quality defect reports, and generic bioequivalence trial safety reports. These reports are now stored and reviewed individually. Entering them into FAERS and enhancing our analytics will make it easier to see the big picture of potential safety issues related to a drug across its lifecycle. It will also make this information more accessible to the public.

The support of this Committee is key to these efforts. And it’s a key to our goal to help leverage the tools of science and innovation to advance the public health. We have new and better opportunities to help improve the expectation every patient should have that the medical products they use have been more fully evaluated for their safety. And we have new and better methods for more efficiently confirming clinical evidence of benefit for drugs targeted to unmet medical needs.

These methods can help reduce the cost of drug development, hopefully translating into more affordable medicines. They can advance more products to patients and make the entire process safer and more efficient. We have the tools to unlock new opportunities to address disease and disability. I look forward to continuing to work with you on these efforts and answering your questions today.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.