Washington, DC - Chairman Aderholt, Ranking Member Bishop, and Members of the Subcommittee. I’d like to thank the Committee for your commitment to FDA. We’ve been fortunate to receive strong bipartisan support in recent years. And the 2018 budget was no different.

I know this support reflects our shared obligation to FDA’s vital role protecting and promoting the public health.

Last year was a record year for medical products at FDA; with the most ever approvals of generic drugs, novel drugs and medical devices. This reflects the remarkable opportunities we have from science and technology.

At the same time, we aren’t losing sight of our commitment to the public health basics: reducing smoking rates, preventing kids from initiating on tobacco products and supporting proper nutrition and food advances that can lead to more healthful diets and reduce the risk of chronic disease.
FDA is committed to these and other public health goals.

With your continued support, we have more opportunity to deliver on our public health mission than at any other time.

The President’s 2019 Budget request for FDA builds on these goals. Overall, the Budget requests $5.8 billion in total resources for FDA – which is an increase of $663 million or 13 percent above the FY 2018 Annualized Continuing Resolution.

At this total level, the Budget includes an increase of $473 million in budget authority and $190 million in user fees. 

The Budget requests new resources for the FDA to make significant investments in advancing critical areas of science, domestic technology and public health.

Among these efforts, we’re seeking to advance a new paradigm in the regulation of digital health technology that I believe will allow us to grow this promising field more quickly.

It includes investments in advanced manufacturing that can bring more production back to the U.S. while improving our ability to respond to public health emergencies like flu.

It advances a new effort to help more compounding pharmacies expand and grow their businesses.

It promotes programs to modernize generic drug review as a way to increase competition and address high drug costs.

And it advances new approaches to support the development of treatments for rare pediatric diseases.

But I want to highlight one initiative, in particular: our efforts to build a knowledge management platform as part of our drug and medical device review programs.
 
This platform would enable us to store and manage the collected experience of our medical review staff – to have a way to identify how decisions are made across different functions, the scientific precedents we establish in the course of our review process, and the knowledge we develop.

Right now, if you asked me how we made a particular review decision in the past, I’d begin by asking our review staff if they’ve confronted a similar clinical circumstance, how it was decided and why. We have limited options to query review decisions to extract how we reached certain conclusions. We can’t store and interrogate the scientific precedent we establish every day.

This sort of knowledge management system is essential to how we’re modernizing medical product review programs and establish scientific precedents established every day.

Among other things, our new efforts are aimed at bringing a more team-based approach to medical product review, to facilitate the easier sharing of information across scientific disciplines, and to bringing more consistency to decision making across different disease and product areas.
These changes in how we operate will make our review process more efficient, modern and scientifically rigorous.

Among these new steps, we hope to set up a policy office‎ inside the Office of New Drugs in CDER that will distill and align regulatory, clinical and scientific reasoning of review divisions to promote policy transparency and consistency.

And we’ll be converting more of that information into objective policy documents to help advance drug development.

As part of this effort, we plan to issue many more guidance documents focused on specific diseases. These guidances will outline clear, concise and up-to-date development guidelines as a way to foster innovation.

Among some of the many areas we’re working on right now are new guidance to lay out modern criteria for the development of drugs targeted to ulcerative colitis; rare pediatric cancers; pediatric HIV; and serious, life-threatening and non-cancer blood disorders like aplastic anemia.

These guidance documents will aim to apply modern principles to make drug development more efficient by focusing on the most effective ways to prove safety and efficacy. For example, the guidance on blood disorders will allow drug developers to reduce the use of animal testing and will outline ways to measure benefit that may permit more efficient development programs and earlier approvals.

There’s a common theme here. One aim is to focus more guidance on laying out the pathway for developing drugs targeted to less common and serious conditions where there’s a lack of available therapy and drug development pathways can be more challenging. To give you another example, we’ll soon publish a guidance describing the evidence needed to demonstrate the effectiveness of new drugs intended for certain slowly progressive, low prevalence rare diseases.  Clinical trial design in this area can be particularly hard and guidance in this area will reduce regulatory uncertainty and provide clarity to drug developers. The focus of this policy work mirrors the general direction of drug development, where new science is enabling us to discover novel, safe and effective treatments for previously intractable illnesses.

These guidances will offer FDA’s latest thinking on our expectations in a wide range of disease areas. They’ll help drug developers better manage the pathway to approval.

Our goal is to sharply increase the number of disease-focused guidance we issue – developing hundreds of new disease-specific guidances once these efforts are fully engaged and resourced. And we want to take new steps to make sure that these drug policy documents stay up-to-date.
But bringing more alignment and shared learnings -- and more consistency to how we evaluate scientific principles and make decisions -- requires us to have more than just up-to-date guidance documents. It also means we need to have better tools for capturing and sharing what we learn -- so that we can develop the rigorous principles that inform these guidances.

That’s where the new knowledge management system becomes a critical element of our efforts. Let me give you one tangible example of the connection between the knowledge system and how we can advance better and safer medicines.

In the late 1990s and early 2000s, several drugs were discovered after-approval to carry a risk of sudden cardiac death leading to their withdrawal. To identify drugs with this “pro-arrhythmic” potential, a new clinical study requirement was introduced as a pre-approval obligation for all new drugs.

These study requirements looked at something called QT prolongation, where the heart’s beat becomes elongated.

But doing these QT studies is very costly, and the results are imperfect. The studies can flag drugs as having a risk when none really exists, and can miss drugs that carry the danger.
Recently, we’ve improved on this approach by developing cell-based assays that can better discriminate the medicines that are likely to have this side effect.

We’ve done this using a “data warehouse” that we’ve built by collecting knowledge and information over many years and across many different drug reviews, and evaluating the differences between drugs that do and don’t have this risk.

Through a collaborative group of researchers, led by FDA’s Division of Cardiovascular and Renal Products, we’ve developed a faster, less expensive and more accurate method to screen drugs for this safety issue. This new cellular assay is being introduced across all drug programs, across all FDA divisions, and we plan to have it replace the old QT study requirements.

This is precisely the sort of innovation in how we assess risk and benefit that we can make more routine with a better capability for curating knowledge gleaned across drug reviews.

FDA will recommend the use of this new QT method in a new guidance for drug sponsors. This sort of innovation improves drug development efficiency, lowering development costs while improving our predictive accuracy. This is our goal. Better science that gives us a better assurance of safety, and at a lower cost. But we need the infrastructure to support this kind of modern reform.

With better regulatory tools, better management of our process, and better science, we can achieve these goals.

And with your continued support, we’ll continue to fulfill our critical consumer protection role and advance opportunities for Americans to improve their health.

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.