Stephen Ostroff, M.D.
Acting Commissioner of Food and Drugs
April 20, 2015
Thank you Sheila [Hemeon-Heyer], for that kind introduction. And thank you Amy [Comstock Rick] for your leadership of this great organization and for the opportunity to be here today. My role here is to humbly but very proudly represent the thousands of extraordinarily talented people who work at FDA. These are people who serve the American public each and every day by assuring the safety and quality of the food they eat, our medical products and cosmetics, and through efforts to reduce the harmful consequences of tobacco products.
This meeting brings together many key partners in the world of food and drug law and policy. FDLI is an organization and each of you in the audience understand more than almost any other group the complexity and challenging environment FDA works in, the benefits to society of the products we regulate, and the necessity that we do our work well across our wide-ranging portfolio of responsibilities.
Patients depend on us, consumers depend on us, and a large portion of the economy depends on us. These factors highlight the need for a strong, well-resourced, vibrant FDA for today and for the future.
I can think of no more appropriate place for me to be so early in my tenure as Acting Commissioner (this is day 14, but who’s counting?) than with a group that has been such an enduring and steadfast partner to FDA.
Actions speak louder than words. So it’s noteworthy that the FDLI annual meeting is the one and only event that our former commissioner, Peggy Hamburg, never once during her six years at FDA, missed speaking at this meeting. Although it’s strange for you (though perhaps not as strange for me) that she’s not here this year, I’m sure you’ll agree that she’s earned a very well deserved break and can put her FDLI frequent speaker points to good use for some respite from the exhilaration and exhaustion of life as the FDA Commissioner.
I understand that FDA and FDLI go way back. In its early years, FDA consisted of only a small, but talented, group of chemists and their laboratories. The great Harvey Wiley himself did not believe that he needed lawyers on his staff to be effective.
He was indeed effective. But it was Charles Wesley Dunn -- the attorney who founded FDLI in the 1940s and who trail-blazed the disciplines that would shape the field of food and drug regulatory law – who would have an additional profound effect on FDA. He understood that while science was fundamental to the Agency’s mission, FDA wouldn’t succeed in protecting American consumers without access to sound legal guidance.
One can argue that it’s the combination of science and law that makes FDA the preeminent agency it is today. So let me thank Charles Wesley Dunn for his vision and let me thank FDLI for carrying out that vision through this meeting.
Today, of course, we do have our share of lawyers, in addition to many scientists, at FDA. And although I myself am a physician and epidemiologist whose career has mostly focused on infectious diseases, I feel like I have a foot in both camps. Between you and me (and I hope you keep this a secret), in high school and college I was torn between a career in medicine or going to law school. In order to hedge my bets, I majored in biology but minored in political science (a great asset now that I work in Washington) to keep my options open.
But ultimately I let a higher authority help make my decision. Not the higher authority that many of you are probably thinking of. It was my grandmother. She told me that we already had enough lawyers in the family, and what she really needed in her old age was a doctor. How could anyone argue with such logic? So I took her advice, and I’ve never regretted it. But to this day I have great appreciation, and yes, even fondness, for lawyers – even those that aren’t family members.
Maybe with that background it was inevitable that at some point I’d end up at FDA. I must say it took a while to get here, after a circuitous route through our sister agency CDC and time in the Pennsylvania health department. But eventually I made it to the mecca in Silver Spring.
In this talk, I have the distinct honor to highlight some of the important recent accomplishments and exciting developments at the agency. But I certainly can’t take credit for these achievements. The old saying that “Success has many fathers, but failure is an orphan” certainly holds true here. Those amazingly talented and committed people at FDA I mentioned earlier deserve all the credit and kudos for the agency’s enormous success.
But credit also goes to Peggy Hamburg. She had the vision and the perseverance to strengthen the foundation of FDA. She put systems in place that left us much stronger than when she arrived. FDA is better positioned and resourced than ever to address the responsibilities we have today, and hopefully we’re better positioned to meet the challenges and opportunities of tomorrow.
One aspect of Peggy’s legacy that I’m very grateful for is her commitment to strengthen FDA’s science base and ensure that we have the capacity, resources, and expertise to take on the sophisticated scientific challenges we increasingly confront. That made it easy to accept the position of Chief Scientist last year. This important and often unsung achievement was due to her focus on building regulatory science as a discipline and reinforcing the use of science-based decision-making—and scientific integrity – as the foundation for our regulatory decisions.
We do our best when we go where the science leads us, and base our decisions on thorough, data-driven analyses. This assures our work will benefit patients and consumers. If we cut corners with the science, ultimately patients and consumers are shortchanged. There will be no change with Peggy’s departure regarding this central role of science-based decision making during my watch.
Let me now turn to FDA’s accomplishments since this meeting was held a year ago. I can’t cover all of them because if I did I’d be talking all day and into the night. Instead I’ll simply cover some highlights.
Consider the extraordinary number of new approvals last year in drugs and biologics -- 51 new molecular entities and new biological products (41 by CDER and 10 by CBER). These included major therapeutic advances in treatment of cancer, hepatitis C and type-2 diabetes, along with vaccines for meningococcus type B and more orphan drugs for rare diseases than any previous year in our history.
While the numbers themselves are impressive, more important is the profound impact these products have for patients --- the improved quality of life they provide, and in some instances, the offer of cure for previously incurable diseases.
Seventeen of the new approvals are “first in class” therapies, which translates to new approaches in the treatment of disease. And almost two-thirds were approved in the U.S. first before receiving approval anywhere else in the world. Apropos of the focus on precision medicine, many of these new treatments represent innovative therapies targeted to specific characteristics of patients and the specific features of their disease. These medications are also increasingly linked to diagnostics that predict disease risk or determine the likelihood a therapeutic will work in a particular patient.
To help speed the delivery of products that address unmet needs or treat serious diseases, we have effectively employed expedited review programs including priority review, fast track, accelerated approval, and the much touted breakthrough therapy designation.
Fully two-thirds of the new drugs approved last year took advantage of at least one of these programs. And the breakthrough therapy program has been far more popular than originally envisioned. Through the end of March, FDA had received 246 requests for breakthrough designation and granted 71. That’s almost 30 percent. And the submissions keep flowing.
One of the most difficult – and often heart-rending - issues we face occurs when seriously ill patients seek access to drugs that are still under study. Many of us have family members, friends, or colleagues who have found themselves in such circumstances. FDA has a long history of assisting patients who seek such access in circumstances where companies are willing to make the drug available outside of a clinical trial. Earlier this year, FDA streamlined the application procedures for our expanded access program, which should improve our efforts to support patient access to experimental treatments in the safest and most appropriate way.
Earlier access to promising new devices is also critically important for patients with life-threatening or irreversibly debilitating conditions. For them, delayed access may mean the difference between life and death, or may result in irreversible disability. So just last week our Center for Devices and Radiological Health launched its Expedited Access Program modeled in part on the drug program. Sponsors can seek designation if they are developing a device for life-threatening or irreversibly debilitating conditions that meet an unmet need.
Another accomplishment over the past year is FDA’s continued progress on compounding. The 2012 fungal meningitis outbreak was a wake-up call that demonstrated the serious gaps in the oversight of compounding pharmacies.
Congress responded by enacting the Drug Quality and Security Act in late 2013. Since then, we have made great progress in implementing the provisions of DQSA, establishing a framework to address compounding by state-licensed pharmacies and creating the new category of outsourcing facilities that among other things compound sterile drugs.
More than 50 outsourcing facilities have already registered with FDA. We have also issued three final guidances, ten draft guidances, one proposed rule, and a draft MOU for states that choose to investigate and respond to complaints of compounded drugs distributed outside their borders. On the inspection and enforcement front, FDA has conducted over 140 inspections of compounders, issuing approximately 40 warning letters, and we are working with the Department of Justice on civil and criminal enforcement actions.
We’ve also made progress on the longstanding concern about representation of women and minorities in clinical trials that support marketing applications for medical products. In Section 907 of FDASIA, Congress required FDA to analyze the degree of inclusion of demographic subgroups in clinical trials. Our report, issued in 2013, found that while there was variation among applications, the majority included demographic subset analyses and that these analyses were shared with the public in a variety of ways. But not always…..
So last August we released an action plan to help close gaps in data quality, clinical trial participation and data access and are now working on these action items. We issued a guidance document on the “Evaluation of Sex-Specific Data in Medical Device Clinical Studies,” are working to promote clinical trial participation by women and minorities, and are posting on our website easy-to-understand Drug Snapshots about the age, race, and sex of participants in clinical studies of newly-approved drugs and biologics.
A major highlight of this past year is the recent approval of the first biosimilar of filgrastim. Janet Woodcock aptly described this approval as a game-changing event. Although this is the first, there will certainly be more to come, even as we work to educate the public and providers about this new category of products.
We’re also making important contributions to address the growing problem of antimicrobial resistance, or AMR. The past year has seen a whirlwind of activity culminating in President Obama’s National Strategy on Combating Antibiotic Resistant Bacteria, also known as the CARB, to ensure that the “miracle” drugs developed over the last 80 years will remain available and effective for future generations.
Already we see progress. Last year FDA approved four novel antibiotics for several categories of infections. As a comparison, just five new antibiotics had been approved in the previous ten years. We’re also working collaboratively to improve diagnostics to detect antimicrobial resistance, to improve antimicrobial stewardship, and to reduce inappropriate antibiotic use in both human and veterinary settings.
Over the next two years, FDA will be implementing what is known as Industry Guidance 213 to phase out the use of medically important antimicrobials for growth promotion in food-producing animals and to bring remaining therapeutic uses of these drugs under veterinarian oversight.
Have no doubt. It is an enormous challenge to so rapidly implement such a fundamental change in animal husbandry practices in this country. But all 26 manufacturers of antimicrobials used in food-producing animals have voluntarily agreed to fully adopt FDA’s judicious use approach, and 30 products have already been withdrawn from the market. Labels of the remaining drugs are being revised to remove production indications for antibiotics that are medically important in human medicine.
These changes cannot come a moment too soon in light of recently released data by FDA showing continued increases in antibiotic usage in food producing animals. However, it is important to note these data are from 2013 and reflect practices before the 213 Draft Guidance was issued and well before it will take effect. We will use future data to determine the effectiveness of the 213 guidance.
We’ve also taken measures to address the epidemic of prescription opioid abuse and misuse. This terrible problem was recently highlighted at the annual National Prescription Drug Abuse Summit in Atlanta sponsored by Operation Unite, an organization established by House Appropriations Chairman Hal Rogers.
Recent steps taken by FDA include approval of an auto-injector form of naloxone to treat overdoses in the field, which took the agency a mere 15 weeks from application to approval, revamping the labeling for long-acting and extended release opioids, and, just a few weeks ago, issuing final guidance on development of abuse-deterrent opioid formulations.
Interest in producing abuse-deterrent formulations has risen dramatically: FDA has already received some 30 investigational new drug applications from manufacturers seeking to conduct clinical trials on potential abuse-deterrent formulations. We hope this leads to a day, not so far in the future, when the majority of opioids in the marketplace are in effective, abuse-deterrent forms.
Ebola may have faded somewhat from the headlines, but it remains a front burner issue at FDA. This is a disease I know well from my days at CDC, although never with the scope and scale witnessed in West Africa over the past year.
FDA has played a critical, although somewhat unsung, role in the response – involving more than 200 people across the agency, including several dozen deployed FDA commissioned officers to provide care in Liberia.
We’ve used the Emergency Use Authorization mechanism for rapid availability of nine Ebola diagnostics, and CDER and CBER scientists have worked closely with NIH to develop and implement innovative, adaptive, and scientifically rigorous clinical trials for vaccines and therapeutics. Experimental vaccines have been administered post-exposure and experimental drugs and convalescent plasma from recovered patients have been used therapeutically with FDA assistance. We’ve also monitored and taken action for fraudulent product claims to prevent, diagnose, or treat Ebola.
From my perspective, the Ebola response shows FDA at its best, working across the agency to rise to the occasion in a major public health crisis.
Of course, FDA’s responsibilities extend well beyond medical products. We’ve had an equally productive year working to ensure the safety of the nation’s food supply and to provide better nutritional information to consumers to help them make informed food choices.
Front and center in our food safety efforts is making progress on the Food Safety Modernization Act (FSMA). FSMA represents a once-in-a-generation opportunity, or maybe I should say a once-in-a-lifetime opportunity, since it’s the biggest change to the system in more than 70 years. When implemented, it will transform our food safety efforts from responding to problems after they occur to preventing them from occurring in the first place. We must seize this opportunity and fully actualize this vision. The benefits to public health in disease prevention are enormous.
So far, we’ve issued the seven foundational proposed rules and received a great deal of feedback. We paid careful attention to what people were saying in their comments and we responded with supplemental proposals on four of the proposed rules. We are now ready to move to final rules and are poised to move on to implementing them. Without question, it’s a very exciting time for FDA, for the various stakeholders domestically and internationally, and most importantly for consumers.
On the nutrition side, we continue to work on the first update to the Nutrition Facts Label, or as I like to refer to it, the other NFL, in 20 years. This will assure the NFL reflects current nutrition science and meets the needs of consumers. Another milestone is the issuance of the final rules to ensure that chain restaurant menus and vending machines display calorie information.
The last, but certainly not least, area I’ll touch upon is our work to reduce the harm associated with tobacco products, especially youth tobacco initiation and use. Congress gave us this authority only six years ago, and we’ve been able to build a dynamic, committed and science based center that has already made important advances in tobacco regulation.
Last year we began an unprecedented youth education campaign known as the “Real Cost” to help inform young people about the dangers of tobacco. And we are moving full speed ahead on the proposed deeming rule which sets the stage for expanding the types of tobacco products that we regulate, including e-cigarettes. As you may be aware, we received more than 135,000 comments on the proposed deeming rule, and addressing those comments has been a challenge of the first order. Data released last week from the National Youth Tobacco Survey showing dramatic increases in reported use of e-cigarettes is a cogent reminder of just how important the deeming rule is.
While our achievements over the past 12 months reaffirm the critical and unique role of FDA to the American public, it is vitally important that we, continue, sustain, and build upon this momentum. Our work is of utmost importance to the millions of people who rely on our assurance of safe, effective, and quality products today and tomorrow.
We live in an era of unprecedented innovation in pharmaceuticals, diagnostics, medical devices, and food and nutrition science. Such innovation offers unprecedented opportunities to advance and improve public health. It is essential that we at FDA keep pace with these changes, especially the evolving science that underpins them, in order to fulfill our regulatory responsibilities effectively and efficiently.
Change always comes with challenges. And those challenges are not insignificant. But the rewards are great. As John Gardner, the former secretary of HEW, said, “We are all faced with a series of great opportunities – brilliantly disguised as insoluble problems.” I look forward to overcoming these challenges, sustaining the important work that occurs daily at FDA, and bringing many of the things that are currently incomplete across the finish line – including our FSMA rules and the deeming rule.Fortunately, we’ve got a great team at FDA to achieve this goal. First and foremost our people located across the product centers and in the field around the country and overseas. Your work is appreciated and vital to maintaining the high standards we have for our medical products and foods. I’m also blessed with a great leadership team, many of whom are here in the audience. I’d especially like to recognize two new members of our leadership team, Dr. Rob Califf, Deputy Commissioner for Medical Products and Tobacco, and Dr. Susan Mayne, our new head of the Center for Food Safety and Applied Nutrition. After only a few months, you’ve already made a difference.
Finally, I’d like to thank all of you at FDLI and in the audience for your steadfast support of our mission and our work. I firmly believe that we can meet the challenges before us – especially if we continue the tradition of successfully blending law and science in the service of public health. I know that FDLI will continue to a strong partner in this effort, and I look forward to working with you as we advance further along the path of progress that we’ve embarked upon. Both Harvey Wiley and Charles Wilson Dunn would be proud of the road we’ve taken, what we’ve achieved, and where we’re going.