I am delighted to be with you to help kick off this year’s Small Business and Industry Assistance Regulatory Education for Industry conference. 

One of the strengths we most prize at the FDA, in addition to our commitment to rigorous science and data, is our ongoing communication with businesses like yours during the development process.  You are essential catalysts for scientific innovation and we want to do everything possible to support that. 

Throughout this conference, you’ll have opportunities to participate in sessions designed to support and engage the global medical products industry, with a special emphasis on the work of smaller medical development companies such as yourselves.  We understand the unique challenges smaller businesses can face, which is why we’ve developed this conference, now in its 11th year.

I’m especially pleased that this year all three of our human medical product centers, CDER, CDRH, and for the first time, CBER, are represented at this conference. 

You’ll be hearing from all three center directors in the plenary session a bit later.   

I think it’s especially appropriate that CBER is joining us this year, in light of their extraordinary efforts responding to the pandemic. 

This dedication led, most notably, to the FDA successfully, safely, and speedily issuing three Emergency Use Authorizations (EUAs) for the Pfizer-BioNTech, Moderna, and Janssen COVID-19 vaccines, and then working to expand that EUA access to adolescents.

Thanks to this achievement and President Biden’s leadership, a substantial percentage of the U.S. population has now been fully vaccinated.  And this, in turn, has led to significant drops in the rate of infections and deaths from COVID-19.

You’ll hear more about the process surrounding these and other EUAs during the plenary session, along with discussion of how each Center used EUAs and other tools to support our response to the COVID-19 public health crisis.

One of the FDA’s greatest assets is our ability to respond quickly and effectively to public health emergencies while applying what we learn from these emergencies to future crises.

The COVID-19 pandemic has challenged us, but it also has afforded us an opportunity to develop smarter responses, increase efficiencies, and help us prepare for future challenges. 

We did this by embracing state of the art science and new technologies, as well as through the advancement of methods and methodologies we already had been working on, some of which did not involve COVID-specific challenges. 

For example, CBER and our Office of Tissues and Advanced Therapies are responsible for overseeing cutting- edge therapies such as those involving regenerative medicine, including cell-and tissue-based products, as well as gene therapy, 

Historically, novel product development programs for advanced therapies have been initiated primarily by researchers from academic centers. But in recent years, we’ve seen enormous growth in the development of advanced therapies by commercial sponsors.  And these medical science businesses are increasingly and regularly submitting IND applications to the FDA.

The scientific advances in these areas hold great promise for the development of products with substantial clinical effects in the treatment of serious and life-threatening diseases.  It’s a potential already realized in products such as the chimeric antigen receptor, or CAR-T cell products, which have proved to be lifesaving in various types of relapsed or refractory leukemias and lymphomas. 

Our scientists not only have to be up to date in the scientific methods involved, but also must stay in close conversation with the developers of these products.  This helps facilitate accurate, efficient, and rapid development of safe and effective products.

These CAR-T products are regulated by CBER, with the clinical review coordinated with the Oncology Center of Excellence. CBER has a put in place a number of initiatives and processes to support the development of these innovative products and technologies.

For example, with passage of the 21st Century Cures Act, CBER added the Regenerative Medicine Advanced Therapy (RMAT) designation.  Expedited programs for product development are available to sponsors of RMATs.. 

Our INTERACT program (which stands for INitial Targeted Engagement for Regulatory Advice on CBER producTs) supports communication between FDA staff and sponsors, and facilitates preliminary informal consultation for innovative investigational products at an early stage of development on issues that are not yet at the pre-IND meeting phase.

Another way we are advancing this kind of work is through our CATT team (which stands for CBER Advanced Technologies Team), which promotes dialogue, education, and input among CBER staff and between CBER and prospective developers of advanced manufacturing technologies.

I hope you’ll take advantage of the opportunities during this conference to hear from and speak with a number of CBER representatives about these and other initiatives they are working on.

Our Center for Devices and Radiological Health (CDRH) also has been very focused on our COVID-19 response effort, working to evaluate new medical devices to help screen for, diagnose, treat, and protect against COVID-19.  Working with the Office of the Chief Scientist, CDRH has authorized more than 700 medical device Emergency Use Authorizations to date in response to the pandemic. 

The Center also provided regulatory flexibility through the issuance of guidance documents, many of which included EUA templates with recommendations to help make the EUA request process more efficient for developers. 

CDRH also implemented a coordinated response to help address potential device shortages in light of global demand and supply chain disruptions resulting from COVID-19.  These proactive and collaborative efforts helped ensure that PPE, ventilators, and other medical devices remained available.

While maintaining this extraordinary COVID-related workload, CDRH continued to meet its other mission-critical responsibilities and even strengthen its work in these areas.

For example, in September of last year, the Center launched the Digital Health Center of Excellence to provide centralized expertise and serve as a resource to ensure that the most cutting-edge digital health technologies are rapidly developed and reviewed

CDRH also unveiled the Biocompatibility Assessment Resource Center, designed to help address the most frequent issues underlying the rejection of medical device submissions. The web-based resources help guide submitters through the steps involved with evaluating biocompatibility and documenting the results.

In addition, CDRH released the Catalog of Regulatory Science Tools, which collates a variety of tools to help ensure that device developers have the right test methods to evaluate innovations. The catalog includes more than 100 tools, including laboratory methods, tissue-mimicking phantoms, and computational modelling and simulations.

CDRH also was able to fulfill another strategic priority -- to participate in 10 established collaborative communities. You can learn about these on the dedicated webpage on FDA.gov.

Because of time limitations, I’ll mention just one, the National Evaluation System for health Technology Coordinating Center Collaborative Community, also known as NESTcc.

NEST is a national network intended to help develop methodologies designed to drive down the time and cost of real-world data collection and analysis and increase the value and use of real-world evidence to meet the needs of patients, payers, the medical device industry, health care professionals and regulatory bodies.

CDRH also has continued its regular engagement with the medical device industry in support of bringing a growing number of innovative devices to patients.  Last year, the FDA approved, cleared, or authorized a record high 132 novel medical devices, marking the latest milestone in a decade of growth. 

The devices approved, cleared, or authorized last year represent a wide array of cutting-edge technologies addressing a broad range of conditions. They included the first game-based digital therapeutic to improve attention function in children with attention deficit hyperactivity disorder; an anterior cruciate ligament (ACL) implant as an alternative to ACL reconstruction to treat ACL tears; and the first-of-its-kind automated insulin delivery and monitoring system for use in young pediatric patients.  And that’s just scratching the surface. 

Critical support for much of this work came from the Medical Device User Fee Amendments Program (or MDUFA).  As many of you know, we recently commenced negotiations for the latest reauthorization of this program, which sustains and strengthens the FDA's ability to conduct timely and efficient review of medical device applications.

We look forward to discussions with industry and impacted stakeholders, including representatives of patient and consumer advocacy groups, health care professionals, as well as members of the scientific and academic communities.  

Central to all this work across the FDA is the way we learn from our experiences and build on our strengths, even as we continue to focus on responding to the COVID-19 public health emergency.

To give you one example, we are building on experience gained during the pandemic regarding our inspections.   

One of the most significant decisions we made in our response last year was to pause most foreign and domestic inspections, with the exception of mission-critical inspectional work.

This decision aligned with federal guidelines to mitigate the spread of the COVID-19 virus, while helping ensure the safety of our global workforce. Last July, we resumed certain domestic inspections after developing a COVID-19 Advisory Rating system to help us manage risks to our investigators and others. Nonetheless, the pandemic has had an impact on our inspectional work.

We recently released a “Resiliency Roadmap for FDA Inspectional Oversight” report, which detailed the impact of the COVID-19 pandemic on our inspectional activities, as well as plans for our transition back to a more consistent, post-pandemic state of operations, and other future inspectional priorities.

I’m pleased to say that as of this month we have begun transitioning back to standard operations for domestic inspections, while continuing to prioritize mission-critical work for foreign inspections. 

We also continue to build on our experiences during the pandemic to expand the development of new and existing regulatory approaches, methods, and methodologies.

Earlier this year, for example, we released a report, “Advancing New Alternative Methodologies at FDA,” which highlights a number of the activities the FDA is undertaking to help us identify and keep pace with advances in basic and applied science and technology.

One important aspect of this work is that it may help improve predictivity--and potentially replace, reduce and/or refine animal testing.

For example, CDER is evaluating microphysiological systems—or organ tissue on-a-chip systems—as cell culture platforms to model human-specific physiology of tissues or organs and exploring quality control functional parameters for cells to be used in those platforms. 

CDER researchers are evaluating liver microphysiological systems to test their usefulness for the study of drugs with hepatotoxic effects and for studying drug metabolism, transport, and intracellular accumulation.

And CDER scientists are also testing liver-heart interconnected microphysiological systems and how they can be used to predict cardiac drug effects that depend on liver metabolism or cause dual-organ toxicity.

Master Protocols is another rea where we have made advances during the pandemic by applying approaches already under study and development.

Well-designed and conducted trials using master protocols can accelerate drug development.  The clinical trial design originated within oncology trials and frequently has been implemented to generate evidence for multiple oncologic drugs to treat a variety of  diseases to streamline drug development. 

But master protocols have been leveraged in several other therapeutic areas, most notably to accelerate drug development during the COVID-19 pandemic.

Compared with conducting multiple independent trials, conducting an umbrella or platform trial can increase efficiency through shared infrastructure. For example, in the case of a common control, master protocols can lead to the need for fewer patients randomized to a standard control, allowing patients greater likelihood to receive an investigational  drug.

We also continue to expand our use of real-world evidence and real-world data in our regulatory decision-making, which can strengthen clinical trials and transform the efficiency of product reviews and post-market surveillance.

For example, the FDA and the National Institutes of Health National Center for Advancing Translational Sciences collaborated on the development of the CURE ID app, which gathers information from the clinical community on novel uses of existing drugs for difficult-to-treat infectious diseases via a website, smartphone, or other mobile device. The Critical Path Institute is now convening a public-private partnership for CURE ID in collaboration with FDA and NCATS.

CURE ID enables the crowdsourcing of medical information from health care providers to facilitate the development of new treatments for neglected diseases. It facilitates the sharing of information on potential therapies for diseases which lack available, approved treatments, and enables the exchange of opinions from global communities of experts and helps to identify potential new uses for existing drugs, and limit unhelpful or harmful uses.

I’ve offered just a brief summary of some of the exciting activities and developments we’re pursuing at the FDA in support of the development and innovation many of you are engaged in.  But I hope it makes clear our commitment to expansive and early communication with you during the development process. 

We are dedicated to strengthening our regulatory response, streamlining processes to make it easier for developers and scientists to send inquiries and requests, and responding with appropriate flexibility when necessary and possible to enable us to move as quickly and safely as possible to support medical developments.

One of the hallmarks of being prepared for crises is the continuous evaluation and application of lessons learned from experience.  Our response to the COVID-19 pandemic is no different.  

Going forward, we will continue to diligently apply the best available science and most rigorous data to our work, whether in response to a public health emergency or to meeting our regular and critical responsibilities to protect and promote the public health. 

We look forward to working with you and supporting you in this effort.