Remarks by Dr. Robert Califf to the 2023 Food and Drug Law Institute Annual Conference 05/17/2023

level of experience and understanding of the work of the FDA as your group The Food and Drug law Institute while the FDA is a science-based organization with a profound Public Health Mission we're also of course a regulatory agency and I've been reminded over and over in the year since I've been back they're regulators often lawyers such as yourselves play an essential and a much discussed role

in shaping and strengthening the diverse and rapidly changing environment in which we work we have our own group of expert Food and Drug lawyers at the agency who have committed themselves to public service and to both defending the role and work of the FDA and recommending legal Pathways that can improve the public health and these days they seem to be extra busy but we also often

rely on many of you as a sounding board a source of knowledge and understanding of current legal and Regulatory issues including the impact of potential changes as well as in promoting understanding and support for the fundamental structural basis for our work which is essential not only for the public but also for many of your clients your wisdom is particularly important these days as the agency faces

some significant even unprecedented challenges that go to the heart of what we do this morning I want to focus on a few of the underlying challenges we face and hope that you'll redouble your efforts to not only serve your clients but also reaffirm the role of the FDA and the system of government that has served us well overall in terms of channeling Industries into producing and

distributing safe and effective medical products and safe foods while also protecting people particularly youth from the death and disability produced by tobacco products it's now been a little over a year since I returned to the FDA although given how much happens here every day I sometimes think the proper calculation should be similar to how we determine the age of a dog seven days for every regular

human day needless to say it's been a bit busy and productive time for the FDA filled with the privilege of regulating a multitude of amazing medical and scientific achievements truly heroic responses to public health challenges and of course something that I know gives many of you a warm feeling a variety of legal conflicts in short it's just another day at the office and the fda's efforts

to protect and promote the health of the American public in many ways we are like referees at the FDA we don't make the products and we adjudicate by a set of rules that are codified in laws rules and guidances of course we have an influence on interpretation of the World book through guidance and we provide technical assistance as Congress writes the laws much like good referees

in sports we cheer on a competitor who makes a great product and plays by the rules and we have an obligation to penalize poor play and dishonest avoidance of the rules but much like the umpires depicted in author Michael Lewis's podcast quote ref you suck unquote in a series entitled against the rules our society is increasingly suspicious of those who make the calls by the rule

book and often downright antagonistic this audience understands perhaps better than most just how important the mission of the FDA is and the importance of playing by the rules not to mention how exciting and rewarding working at the FDA can be you have a grasp of the real world benefits to patients and consumers as a result of the work we do not to mention an understanding of

the rest for patients and consumers when we make the wrong call or when product manufacturers fail to fulfill their responsibilities and of course you have a unique knowledge of the ways in which FDA policies rules and regulations can be interpreted deciphered and deliberated over the relevance of the FDA not just to your clients but to the nation as a whole is obvious from the plethora of

news stories both positive and negative highlighting our activities from reports of products we've approved and the resulting benefits for patients or products that fail to meet the standard for pre-market approval or cause harm to people under our watch order criticism or compliments for that matter for a decision that we make too quickly or for ones that we took too long to make you get the idea

but that prominence not to mention the middle ground between Advocates and critics is exactly where we should be and the tension reflects how our system is designed to safeguard the process from inappropriate influence of politics politicians and courts by nearly every measure it's been an extraordinary year for the FDA for example even as we continue to respond effectively to the covid-19 pandemic we approved a wide

ride to safe and effective new Therapies in 2022 including 37 novel drugs never before approved or marketed in the U.S and numerous drugs for New Uses in patient populations I'm proud that more than half the novel drug approvals this year were for patients with rare diseases but even with this success we recognize that thousands of rare diseases still do not have approved treatments we're committed to

supporting emergency research engaging patients and caregivers and continuing to enhance our review process and methods used for clinical trials to help Advance Medical Products for rare disease patients in 2022 we approved the 40th biosimilar a significant achievement in biosomer product development we also approved two interchangeable biosemlers biological products that May subject to state law be substituted without the intervention of a prescriber similar to how genetic

generic drugs are substituted for brand name drugs these are now four approved interchangeable biosemors I also call your attention to two areas that are shaping up possibly to be major game changers in our ongoing battle with chronic diseases that are causing the vast majority of death and disability in our country we're awaiting final submissions but if the submitted data matches up with the press releases it's

possible we could see significant reductions in the rate of progression in Alzheimer's disease in addition whole new classes of drugs appear to be having a major impact on Obesity and diabetes and these have major implications for a number of associated chronic diseases I want to stress again that we have to wait on the data but both of these areas will raise major questions about the interface

between approval by FDA for unintended use and broader use for tens of millions of Americans this is a good problem to have and we're having productive discussions with our CMS colleagues for example but we all need to be working on these issues our Center for biologics evaluation and research also had a busy and productive year with eight novel approvals six products receiving breakthrough therapy designation for

receiving orphan designation and three receiving Fast Track designation saber also continued its incredible work in response to several Public Health emergencies it's worth it it's worth special note that of the novel approvals by saber five were for gene therapy products this development underscores that one of the areas offering the greatest promise not just in the rare disease space but across the entire spectrum of humans animals

and plants involves the explosion in genetics and Gene editing this has profound implications for the development of new treatments and cures for human disease as well as for the advance of environment friendly and predictable food in the face of climate change and supply chain stresses Gus's involves work across the entire FDA I've asked Dr Naman Jay Bumpus our chief scientist to coordinate our experts across the

agency to make sure we're sharing knowledge in a way that reinforces regulation that supports Innovation enabling the benefit and preventing avoidable risk as the science continues to grow in the food safety area we've made we had a number of major accomplishments including implementing the food safety modernization act through nine foundational rules in more than 70 guidances that have set the foundation of a modern regulatory framework

one of the last major pieces of fisma the food traceability rule was finalized in 2022 and will facilitate faster identification and Rapid removal of potentially contaminated food from the market resulting in fewer foodborne illnesses and or deaths in the nutrition space this San oversaw the first major update to the iconic nutrition tax label that included labeling of added sugars the implementation of legislation requiring calories to

be listed on restaurant and similar food establishment menus as well as launching initiatives to help industry gradually reduce sodium in the food supply we also issued a new proposed definition of the healthy nutrient content claim that consumers can see in the front of food packages updated to align with current nutrition science and the dietary guidelines for Americans food labeling can be a powerful tool for change

by empowering consumers to help them make informed choices about the products they're eating and by encouraging manufacturers to reformulate their foods to create healthier products and thereby helping to foster a healthier food supply our Center for devices and radiologic Health continues to Foster timely patient access to Safe medical devices with a goal of spurring innovation in device safety and ensuring that new products are able to

address unmet medical needs in 2022 the 135 devices were designated as breakthrough devices more than 19 submissions designated as breakthrough devices received marketing authorization and 84 novel devices receive marketing authorization we're increasingly focused on supporting digital Health Technologies which play an increasingly significant role in today's Health Care system an artificial intelligence and machine learning which are powering important advancements in this field the FDA is already

authorized more than 500 AIML enabled medical devices and more under development in 2022 crhs digital Health Center of Excellence makes significant strives to Advance Health Care by fostering responsible and high quality digital Health innovation and cdrh continues to lead the development of new regulatory Frameworks tailored to digital Health Technologies while promoting consistent application of digital Health policies I probably don't need to tell you that large

language models are evolving exponentially and I personally believe this technological Advance will cause revolutionary disruption that could either yield dramatic benefits or serious risks we'll be working with cdrh on both the regulatory scheme for llms and potential new Pathways for the types of complex integrated device and digital systems that could revolutionize clinical care including bringing much more clinical care into the home well there have been

many other exciting developments across the agency I want to mention just a few starting with our work in the area of food and nutrition which has been an area of particular Focus for me this past year over the past decade FDA has made enormous strides in our efforts to make the American food supply as safe as it's ever been indeed the most recent Global Food security

index published by The Economist which considers food affordability availability quality and safety and sustainability and adaptation across 113 countries noted that the U.S has moved up 25 positions for the food safety indicator since 2012 and it's rank to joined first for the most recent year that is 2022. that doesn't mean we haven't faced and I've got challenges in this work of course the infant formula recall

which happened to occur on the day I was confirmed it's been an enormous challenge our response to this recall and subsequent shortage involved wide-ranging collaboration within FDA and across the federal government coordination with U.S partners and domestic and international manufacturers and retailers and Innovation to increase flexibilities and develop new approaches importantly our response also played a key role in stimulating a water review and reorganization of

the FDA human foods program catalyzing a growing understanding both inside and outside the agency that this area needed more attention resources and support earlier this year we announced our vision for a unified human foods program that will combine previously distinct parts of the FDA into a new organization under the guidance of a Deputy Commissioner for human foods in addition we're creating a center for excellence in

nutrition which will Elevate and strengthen fda's nutrition portfolio we'll finalize The Vision by this fall and will continue to engage with stakeholders throughout this process one other example of some tough Waters to navigate over the past year is a regulation of tobacco products the nation has made notable inroads in this area with current cigarette smoking among U.S adults having declined from 20.6 percent of the population

in 2009 to 11.5 percent in 2021 the lowest levels recorded since 1965. but we continue to face Dawning challenges as we seek to regulate this evolving Marketplace involving both combustible tobacco and the industry shift to vaping for example our Center for tobacco products continues to respond to hundreds of thousands of product applications that are remaining among the over 26 million applications that were submitted as well

as dealing with enormous complexities and enforcement and the growth in youth vaping which is producing another generation plagued with addiction to products with unknown long-term Health consequences remains A continuing challenge but I'm optimistic that the work we're doing including our proposals to eliminate flavored cigarettes and cigars and reduce nicotine and cigarettes offer a real opportunity to play a major role in dramatically reducing the toll of

almost 500 000 Americans who still die every year from cigarette smoking and long-saying disparities in tobacco product use if the current patterns persist during this meeting you'll hear Brian King our new CTP director as well as other Center directors about other areas in which the FDA is demonstrating important achievements innovating and responding to daily responsibilities and challenges there's certainly plenty of kudos to be bestowed across

the agency but we also have a lot of work to do even though the FDA continues to operate at a high level there are a number of serious challenges that we need to respond to that I hope you will play a role in make no mistake yeah U.S continues to be the number one innovator in medical products producing drugs biologics tests and devices that fuel Health

Care around the world but to retain this leadership we need to strengthen our structures methods data collection and science to allow us to be even more efficient flexible and effective and the exercise of our regulatory responsibilities let me offer one small but important example the evolving role of advisory committees the FDA has nearly 50 advisory committees and panels which are convened to provide FDA with independent

scientific medical and public health advice while regulatory decision making ultimately rests with fda's full-time civil servants the breadth and complexity of our responsibilities make the use of advisory committees an important tool for obtaining independent advice regarding scientific technical or policy questions to inform our critical work as a former long-term advisory committee member I believe strongly in the value of interchange among our experts at the FDA

and experts who work outside the agency for certain complex issues seeking the advice of an advisory committee not only makes our decisions better but it also adds an element of transparent societal discussion that's important but I also believe that our advisory committee system can be improved to enable our experts to get the best advice possible stay tuned for developments in this area one of the biggest

challenges we can continue to face does not involve refinement of internal processes but rather how we respond to dangerous and corrosive external factors I'm referring of course to the continuing and growing challenge of misinformation and disinformation since our raised associated with you last year it's been an increasing understanding of the problem across government and the nation but little in the way of real solutions that lack

of traction is due in part to the very nature of misinformation which involves unaccountable or unreliable sources often coming through unattributable social media sources one of the most basic responsibilities of the FDA is to disseminate facts about science and medicine to the public in order to help Americans make informed choices about their health but by undermining confidence in science purveyors of misinformation make this much more

difficult by weakening faith in governmental and other institutions including the FDA and in turn endangering the American public health the American public is not an abstract concept these are our family members and Friends the extraordinary tragedy that the vast majority of Americans died from coven are not up to date on vaccination and or were not treated with an authorized anti-viral is only the tip of a

much larger Iceberg at the core of our decline in life expectancy from drug overdose suicide gun violence and chronic diseases the FDA is working to aggressively combat this but we can't do it alone for instance we recently relaunched a new updated FDA rumor control webpage which provides a place for people to find the Pacs on hot topics and learn more about how to spot an address

misinformation I encourage you to stop at our table at this meeting and take a look at the page so you can help disseminate these and other facts that counter misinformation as members of the Food and Drug community in bar you not only can play a role in doing something about misinformation but I'd suggest you have a special responsibility to do so as you well know the

role and mission of the FDA to protect Public Health was created to fund and delineated and mandated by Congress the agency has made an extraordinary difference in the health and safety of American patients and consumers and today it's under attack like never before indeed our fundamental authority to regulate the safety and advocacy of products is being challenged I'm not going to discuss any specific cases I'm

sure many of you follow them closely but what I want to ask you to do as lawyers intimately familiar with and occasionally advocates for the crucial role the FDA plays is to stand strong for the working principles of FDA to not let them be distilled or destroyed through misinformation disinformation or partisan legal challenges many of you have worked at the FDA often in key positions some

of you have litigated on behalf of the agency I participated in many discussions about the revolving door for biomedical scientists but I've heard a little discussion about the revolving door for lawyers and while the considerations are not identical there are significant similarities that deserve more attention and debate to find the correct position here I'm not asking you not to Advocate be advocates for your clients My

Hope however is that in your current work you go beyond simply abiding by the ethical rules governing lawyers who leave federal government practice for the private sector and seek to strengthen the environment in which we all operate each of you have the ability to help shape the debate and discussion to promote communication and collaboration and to work productively to reinforce the importance of our institutions to

progress no matter which side of the legal proceeding you're on in taking this honorable path you can help make sure that facts went out over misinformation and disinformation that the principles we stand for are not eroded and that our ability to protect patients and consumers is reaffirmed and preserved thanks and I'm sure you'll have a great meeting look forward to discussing these issues whether you're in

more depth when I'm back from the anniversary trip