Remarks by Acting Commissioner Giroir to the Reagan-Udall Foundation for the FDA 2019 Innovations in Regulatory Science Awards Dinner December 11, 2019

Speech | In Person

• Speech by: Brett P. Giroir, MD ; Acting Commissioner of Food and Drugs - Food and Drug Administration

(Remarks as prepared for delivery) 

Good evening.  My wife Jill and I are thrilled to be with you tonight for the presentation of the Reagan-Udall Foundation Innovations in Regulatory Science Awards.

It is my great honor and privilege to be the Acting Commissioner of the FDA, an opportunity I never expected to receive – but one, even though brief, has made an indelible impression on my outlook on the Agency, and its essential role in protecting and advancing public health over the next decades.

After only 6 short weeks, my commitment to – and appreciation for - the many extraordinary professionals could not be deeper or more sincere. 

And it is clear, for the FDA to be effective, it needs the vital contributions of the Regan Udall Foundation.  This is exactly the type of synergistic relationship that allows the achievement of the FDA mission in ways that a US government organization could not do on its own.

While many Americans have some awareness of the FDA’s work, most view it only from brief media highlights. 

To many, the FDA is synonymous with the imposing façade of our campus at White Oak or the iconic FDA logo. 

Others may have been personally affected by a groundbreaking drug approval or the work to track and trace a food outbreak

Or perhaps they’ve interacted briefly with an FDA investigator, special agent, compliance officer, researcher, public affairs specialist, or other staff located in our many field offices and regional laboratories. 

But each of these scenarios still just scratches the surface in gaining an understanding of what the FDA is truly all about.

Within a few short days on the inside, I came to fully appreciate that the FDA is actually defined by thousands of hard-working, diligent, smart, creative, and passionate committed professionals -- here in the nation’s capital, across the country, and around the globe -- who protect and promote the health of the American public and provide leadership for the world.   

While my appreciation has grown, my own understanding and involvement with the FDA preceded both my current acting appointment and my day job as the ASH. 

Throughout my career as a physician, I’ve had the opportunity to benefit from the Agency’s work

As a pediatric critical care physician, by definition, I cared for children on the edge of life, and their always distraught families.   Early on, 90% of what I prescribed was not indicated for children, or the conditions for which I used them.

And while we are not finished with our work, I’ve seen first-hand the dramatic impact the FDA had had on assuring that children are not forgotten in the drug development process, and speeding the development of new treatments and cures for diseases that previously had little hope.

One timely example is HIV/AIDS.  When I started practicing, I saw many of my child patients – especially those with hemophilia, chronic renal failure, and other conditions – receive a diagnosis of HIV – a death sentence, universally, at that time.

Now, just 3 decades later, due to groundbreaking medical research and the regulatory revolution championed by FDA’s viral disease group,  HIV is today a chronic and manageable illness: An individual diagnosed at age 25 with HIV, if compliant with medications and follow up care can, on average, expect to live to age 75 – a near normal life expectancy.

Now, we have come to a point – a truly historical moment – when our Administration plans to End the HIV Epidemic in America in 10 years – a goal that is almost unbelievable – except that it is very very real, and we are going to achieve it.  We have the right data, thanks to FDA the right tools, and finally the right leadership.

But what has impressed me more than anything, is the velocity and magnitude of impact at the FDA.  In just the few weeks I’ve been here, I’ve witnessed a number of significant developments, including approvals of many important new treatments.

These include two treatments for sickle cell disease,  which has had only one prior drug approval in 4 decades; a system for delivery of ear tympanostomy tubes under local anesthesia – right now there are nearly 700,000 placed per year under general anesthesia – a risk that can now potentially be avoided; key guidance to increase efficiency in the development of biosimilar and interchangeable insulin products, with the promise of making insulins more accessible and affordable; and approval of the first treatment for acute hepatic porphyria, a rare genetic disorder that can lead to severe pain and paralysis, respiratory failure, seizures and mental status changes.

If that were not enough, we have attacked – head on with the CDC and the NIH – the need for new Ebola therapeutics and vaccines, the deadly vaping related lung injury, the epidemic of e-cigarette use in youths, and multiple food borne outbreaks of infectious diseases.

Not only do these actions reaffirm the unique and essential role of the FDA, they also underscore the importance of the Reagan-Udall Foundation in our work to do the kinds of regulatory science we need to do to support innovation. 

Quite simply, there are certain things that a government agency can’t get done as quickly or as efficiently as a foundation working to support it can. 

RUF works synergistically with the FDA, building and supporting collaboration, and facilitating shared learning and enhanced communication between regulators, industries, and the public.

As everyone here understands, we are in an era of enormous progress in science and technology that enables us to both be a witness to, and participant in unprecedented innovation and completely new paradigms of disease and treatment – as just one example, the transformation of cancer care via checkpoint inhibitors.

But make no mistake … the application of transformative technological developments doesn’t happen automatically, quickly, or seamlessly. 

There are significant challenges involved in integrating this evolving science and the accompanying wealth of available medical data into effective regulatory decision-making.  Let me provide examples from my federal career.

I had the great privilege of leading the science office at DARPA between 2004-2008; and one of my first actions was to sponsor a liaison within the office of the Commissioner. 

Why?   Because innovators need to work together with the FDA to assure products are safe and effective, and honestly so that the FDA has a framework for decision making.

What do you think what have happened if we showed up one day and said DARPA had developed a brain chip to control a fully function bionic arm based on thought control ???? 

Or how about a pharmacy in a box to manufacture essential drugs from chemical precursors de novo in austere environments?

Or how about large scale manufacturing of vaccines and therapeutic proteins in transfected hydroponic tobacco plants?

FDA needs to stay ahead of, or at least at pace with, scientific change in order to maintain the FDA’s gold standard of review, and to be able to make the most informed regulatory decisions.  And I would say this is particularly the case during our current era of genetic, cell, and regenerative therapies. 

The Reagan-Udall Foundation fills an essential role by supporting the Agency in the development and application of this knowledge, tools, and methods necessary to address the regulatory science challenges we face.

We’ve seen the benefits from the Foundation’s work in our IMEDS (or Innovation in Medical Evidence Development and Surveillance) Program, which helps us leverage and expand the FDA Sentinel network for use by other stakeholders.

Sentinel has allowed us to develop innovative methods of monitoring FDA-regulated products, answering numerous questions simultaneously and in a matter of weeks and months, rather than years, and more quickly and accurately identifying things like drug-associated adverse events while helping to meet post-market surveillance and reporting requirements.

We’ve also seen the importance of the RUF in its work on our Expanded Access Navigator website and database, which provides patients and their health care providers with information about how to access investigational treatments outside of clinical trials. 

And we’ve seen it on a more basic level, in the many meetings the Foundation has sponsored to bring together FDA’s stakeholders on issues of importance to the Agency and the public. 

These have ranged from smaller gatherings of groups of patients to large meetings of hundreds of stakeholders from across industry, academia, healthcare providers, patients, and consumers. 

They’ve included a conference to explore the potential use of real-world data derived from expanded access treatments, a roundtable to discuss barriers in the expanded access process, and a workshop on repurposing of generic drugs, to name just a few.

It’s an important role that makes a real difference in the lives of patients – my patients, your patients, our families.

Tonight, we’re here to honor the winners of the Foundation’s Innovations in Regulatory Science Awards – celebrating several individuals and groups who are being recognized for their contributions to improving our health and safety.

Each of the honorees exemplifies the kind of leadership, innovation and commitment that is important in spurring the advances in regulatory science that make a meaningful difference in the lives of Americans.

As a pediatrician and former CMO of a children’s health system, I had a great deal of interaction with CF patients and the Cystic Fibrosis Foundation – the foundation was always there – funding research, advocating for patients and their families, and providing hope during very challenging times.

CF is a disease that has seen some real benefits as a result of research and scientific innovation,  with the average life expectancy increasing from only 10 years in the 60s to near 40 years today, and the Foundation continues to be a driving force in new advances, and eventually for a cure.

As a physician who has cared for critically ill children, often with rare diseases, I also have familiarity with the FDA’s compassionate use program, and the work the Compassionate Use Advisory Committee is doing in this area is so important.  I know how critical it is for patients without any other options to have a drug made available to them through this program. So I thank them for their work and innovation.

And finally, regarding the winner of the Leadership Award, the FDA’s Theresa Mullin, I can say that since I joined the Agency I have heard from so many people at the FDA about what an extraordinary employee and asset she is and just how much she deserves this reward.  

She is, I would suggest, emblematic of the best that FDA offers.  So my heartfelt congratulations to her.  And her award also allows me to say again what an honor it is to work with so many accomplished and committed people and organizations.

Thanks again to the Reagan-Udall Foundation for its work in support of the FDA and for honoring tonight’s award recipients, and for allowing me to be here with you.