Full article reprinted from The RPM Report June 30, 2009
Jesse Goodman, FDA's new chief scientist and acting deputy commissioner, is in a pivotal role in the new Administration, as the bridge between the career staff at FDA and the incoming political leadership. His keynote address at the Food & Drug Law Institute Annual meeting is a must read for anyone looking for signs of how the Obama agenda will affect FDA. Read on...
Full article reprinted from The RPM Report June 30, 2009
This year was different.
Goodman, the newly appointed chief scientist at FDA and acting deputy commissioner, delivered the keynote address in the opening session on behalf of the agency, speaking to a standing room only crowd filled with representatives from across the spectrum of FDA regulated industries, eager for any sign of how the Obama Administration will affect the agency—and, hence, their clients or businesses.
What they heard had to be reassuring.
Goodman's talk illustrates that some of the central themes of the Obama Administration—transparency, scientific integrity, and a call to "change"—can be compatible with an approach to regulation that is collaborative and even productive for industry.
Goodman began by stressing that it is a mistake to view issues as FDA vs. industry, or industry vs. academia. Together, FDA and its stakeholders "really are a system," mutually dependent on each other for success.
That is not to say things will stay the way they were in the past; but Goodman sees change as an inherent part of the complex system that binds together FDA and industry. So no one should be too entrenched in the status quo. "We have to not get into a variety of fixed positions, because that doesn't work with a changing world and changing system. We need to adapt to the information. Change needs to be part of our inherent processes."
The type of change outlined by Goodman—in an address filled with many examples of ongoing collaborative work by FDA—sounds less threatening to industry than the worst fears from the shift to a Democratically-controlled Washington. The sheer number of ongoing collaborations named by Goodman in his address (many of which have been edited out of the transcript below in the interest of clarity) underscored a sense of continuity in the overall spirit of FDA as partner rather than adversary.
But Goodman also gently underscored another message—that the FDA "system" cannot be simply bilateral partnerships with industry. Other stakeholders from across the spectrum of health care—and across the globe—are partners too.
Still, you could almost feel the sighs of relief as the FDLI audience heard clear reassurance that FDA will not be redefining its mission in adversarial terms.
For regulated industry, the appointment of Josh Sharfstein as acting commissioner has been a cause for nervousness if not alarm. His background (former bosses include Rep. Henry Waxman and Public Citizen's Sidney Wolfe) and recent role in spearheading the withdrawal of pediatric cough/cold medicines while public health commissioner in Baltimore have plenty of people on edge.
Sharfstein will ultimately be deputy commissioner to Margaret Hamburg, assuming she clears Senate confirmation, but Hamburg is also an unknown quantity for most in industry.
She is not, however, unknown to Goodman: the two served together on Institute of Medicine panels focused on biodefense and pandemic preparedness, and Goodman's elevation to the commissioner's office came—not coincidentally—soon after Hamburg was selected by the White House as the commissioner nominee.
And, though no one in the FDLI audience knew it at the time Goodman spoke, the decision to elevate him at FDA was about to pay an unexpected dividend for the Administration. On April 23, the day after Goodman's FDLI address, the Centers for Disease Control & Prevention confirmed two cases of swine flu in the US, giving FDA's new leadership team what could be a defining challenge.
With Hamburg still awaiting confirmation, FDA's role in the response was overseen by Sharfstein—who immediately put Goodman on point for the agency.
So here, lightly edited for clarity, is what Goodman had to say when he laid out his vision for FDA in the coming years.
Jesse Goodman's Address to FDLI
These are very exciting times. While industry, FDA, all our numerous partners, and the country as a whole have had many challenges, the base message, I think, is a very, very positive one. There is an incredible amount going on in the scientific and innovation community. Almost all the stakeholders have common goals here, and we can work together even in a challenging environment to succeed. And the beneficiary of our success—it certainly makes us all happy to succeed—but the real beneficiary is people and communities that are healthier.
Embryonic stem cell research is an example of something where the public health implications and benefits are potentially huge, but where there are a lot of issues, both scientific and social, that are being addressed and where I really think FDA can make a huge difference, but only by being collaborative and scientific. Because what it takes to bring a therapy to people is not just in academia. It's not just in industry. It's also in the process of evaluating those products. It is so important that it be effective and efficient and get it right.
I do see this as a critical moment in time. I'm sure everyone has always felt their moment in time is the most complex ever, but certainly our technologic and social challenges and opportunities make this very unique.
We have epidemics of chronic disease in this country that result in huge economic impacts, but also human impacts. We have opportunities often missed for prevention. That's not only in the healthcare field but it's much more broadly throughout society. If healthcare tries to do prevention alone it will not succeed. This is part of education. This is part of social policy.
As you all are fully aware, we have many global threats, from the threats of infectious disease to the assuring a safe supply chain of foods and medicines. But I would argue that the opportunities are even greater.
The science is truly astonishing. For the first time, the science that we are looking at is beyond the ability of individuals to work with on their own. Science requires teams and science requires computing. It's been transformed by informatics. But the result of so much science is we have an incredible spate of innovation, whether it's in drugs, devices, biologics, foods, that has the potential to transform health.
We also need to consider what we're doing, all of us, in the context of the economic challenges we're facing right now, and I'm particularly sensitive to those challenges in the innovation community. That means that it's ever more important that FDA try to be collaborative in these processes and get it right.
So my overarching theme is that, together we really are a system. It's not "industry," "FDA," "academia," etc. It's a complex system and it changes. We have to reach for common goals, and we have to not get into a variety of fixed positions, because that doesn't work with a changing world and changing system. We need to adapt to the information. Change needs to be part of our inherent processes.
So, in delivering and developing medical products and foods, this is a complex universe. It's not just medicine: it's public health, and we all play a role.
Common Goals
What do I see as common goals that we at FDA really have to keep to our core?
Protecting and promoting health, that's a core value in our mission. But I think we have to look at how we define health. Thanks to changes in information sciences and biosciences, we can define it in many ways and they're not mutually exclusive.
The health of an individual matters. Certainly if you're that individual, your child is that individual, your loved one is that individual, that matters. And we have a role in that. The health of groups matters. The health of the nation matters. And the health of the world matters. We are part of that world. I think these are all increasingly interdependent, but I think where we sometimes get it wrong is, I don't think they're mutually exclusive. They can be competing at times, but they're not exclusive.
Given that, we have to use resources wisely, whether we're in the government or outside. We can't achieve these outcomes without some commitment to constantly be looking at what we're doing and reassess things based on new information or challenges.
Transparency. I think that FDA has often been seen as a black box. Some of this is about the laws we are held in, but some of this is about culture and some of this is about how we view our role. There's a recognition that we would like to make the regulatory process as transparent as possible.
The idea here is not just to make people happy. With transparency we make better decisions. The more input we can get and the more we can explain our thinking, the more testing and debate there is, the better off we are. So we need to see out to get that input. To the extent that others can see in and understand our processes better, that is better. They will understand our decisions better, and that will build trust.
Communication, or the message, is just absolutely critical. We often are making complex decisions. Most public health messages involve uncertainty, different audiences for whom risks and benefits may be different. We need to pay much more attention to our messages.
We've been talking a lot recently about how we're often seen confined by a regulatory message. A regulatory or legal standard is one thing. Yet if we keep our health outcome goals in mind, we also need to be sure we're giving a public health message that people can understand. Because I can assure you, while lawyers may understand the legal and regulatory message, that's not what the public needs to be able to make wise decisions. Of course, we have to follow rules and laws, whether they're designed to increase transparency or protecting patients.
The complexity of things, in my mind, requires appropriate partnerships. This is just not a world where the scientist in the laboratory alone, even the NIH-funded laboratory, or the FDA reviewer in a room or the inspector in a room or, quite frankly, a company in its headquarters, can afford not to be interacting broadly.
Many of these partners are the ones that you would expect, but I want to mention some others. The social and behavioral sciences in terms of how we affect how health outcomes and health products are used are critical. The investors. We don't think enough about the delivery systems. We sort of make recommendations and make new products, approve them, give safety information, etc., but it's really the public and those delivery systems that determine whether that information is helpful to people and actually used.
Because of the global world, we have global regulatory partners, some of whom are quite innovative; some of whom really need us. We all need each other to function well. We are also part of a global public health community. Many things, whether it's antimicrobial resistance, availability of new drugs and vaccines, affect the whole health of the entire world. If we don't get engaged, we will lose, both in terms of markets but also in terms of our nation's health, and also we will lose an opportunity to help others.
Now, I think something that we really need your engagement in, as well, is the issue of integrity. Integrity clearly is precious. It leads to trust. It is hard to repair. And without it no one can succeed. I don't think this is just an FDA issue. I think this is an issue for all of the partners.
FDA, though, has a particularly challenging role, because we want more transparency and we want partnerships, but we need to do those in a way that protect our integrity and the integrity of our staff.
For those of you who aren't aware, the president issued a memo on scientific integrity, and I want to quote from it:
"The public must be able to trust the science and scientific process informing policy decisions. Political officials should not suppress or alter scientific or technological findings and conclusions. If information is developed and used by the government it should ordinarily be made available to the public. To the extent permitted by law there should be transparency in the preparation, identification and use of scientific and technological information in policymaking."
So you see the recognition here that there is policymaking, and that will always take place. There's not one truth in the world about what should be done. But there's a desire for the information and the processes underlying those policy decisions to have a high degree of integrity and transparency.
The President asked the director of the Office of Science & Technology Policy to provide recommendations. There's a task force, which I'm participating in, and there's going to be opportunities for input on this.
What Is Safety?
I'm going to talk a little bit about safety, because that's certainly something the public really looks to FDA for, and some of the issues around that.
What is safety and how it can be achieved? We have an education job to do here. Safety can't be absolute. Even when we have something that has a very good risk/benefit ratio, there is almost always uncertainty. And, as you know, in many of the drug safety issues that have come up there are often data that is very preliminary or we don't know how to interpret it. So frequently our communications deal with the challenge of uncertainty.
In this area, transparency may be challenging in the short run. Communication may be challenging in the short run. But I believe even in the face of uncertainty, transparency is the right approach. "See no evil" is not really an option. But we need to get much better at communication, and the better we are at communication the more trust there is and the better decisions people will make.
So how do we define safety? I think we need to educate ourselves and others that there are two variables. Safety is really about minimizing risks and maximizing benefits. That's really what we can do. The revolution in informatics offers tremendous opportunities.
Informatics can absolutely transform the quality and safety of product development in clinical trials, whether it's predicting what's effective or whether it's in monitoring product safety. Molecular science can be used to design products, predict toxicity, and link clinical outcome to the genome. And there is value to knowing how these products perform in the real world, not just in the clinical trial. For example, the Sentinel Initiative, which intends to bring together outside sources of data, develop analytic tools to look at healthcare data, etc. Despite limited resources for doing this there are a lot of pilot work ongoing and a lot of interest on our part and that of our partners.
But where I wanted to go with this is the personalized medicine issue, I've thought about this a lot recently. It's really been cast as an either/or. It's, "there's going to be personalized medicine now, and that's it." Or, people looking at comparative effectiveness are ignoring individual data. What we have to reach for is a synthesis here, a new epidemiologic paradigm. Years ago, doctors just said, "Well, my patient got better when I did whatever, and therefore I'll do that to the next 10 people." And we know that's not always effective. So we moved to controlled trials.
Well, now we have the ability within carefully managed studies and within healthcare practice to look at individual and subgroup responses to therapies and identify not just the best therapy for individuals, and not just the best therapy for populations, but the best therapies for groups within those populations. These may not even be single drugs. They may be public health interventions. But I think we need to start to define groups—populations—as the therapeutic targets.
So, where is genomics and all this healthcare data at now in picking people out of the crowd? Most people are focusing on preventing harm or maybe targeting effectiveness. So there's numerous studies like the warfarin collaboration that can make commonly used medicines less harmful by having genomic data. We've begun adding that to the label, and there are paradigms for reviewing that.
But ultimately where we're going to get is more shades of relative benefit, which encompasses safety and effectiveness.
So, for example, you have populations of thousands, hundreds of thousands, with diabetes. We will probably ultimately identify not just that there's type 1 and type 2 diabetes but that there are families of gene expression and maybe even expression of unrelated genes and pathways within there. Maybe we will have 10 approaches to treating diabetes that are based on the genomic information that ultimately we're going to be seeing on everyone, because the genomic information is, like most other informatics, exponentially going to get cheaper and more available. So we need to be prepared for this.
So we can move from "everybody should or shouldn't get this treatment, and some people are going to benefit and some people aren't, and some people are going to get hurt and some people aren't," to knowing maybe that for 90 out of 100 people we're really doing the best therapy there is out of the whole menu.
A Global View
Globalization in public health is, to me, another big threat and challenge. There's humanitarian needs and values. Disease threats are global. They know no boundaries. We have malaria mosquito vectors in this country. There's drug-resistant malaria all over Southeast Asia.
This is a global economic and a national security issue as well as a humanitarian issue. For example, look at the chaos that was brought on when one person flew around the country with their extremely drug-resistant TB. Look at SARS. SARS was definitely a bullet that was dodged.
So we need to pay attention to these things. TB is an endemic disease of humans. There are billions around the world who clearly need an effective TB vaccine. If we had a highly safe and highly effective TB vaccine it would probably make sense to use it in this country.
Again, you all know manufacturing is global, knowledge is global and markets are global. There is a group called BIO Ventures for Global Health that, for example, predicted a $1 billion market for an effective tuberculosis vaccine, not even counting our country.
As a result of these kinds of priorities FDA is increasingly globally engaged. Certainly CBER, where we had the vaccines and blood safety, we were particularly engaged with WHO and others. But throughout the agency we're engaged in regulatory information sharing, harmonization and global public health. And this is something we need to continue to do, and it would be good for FDA, good for science, good for industry and good for public health.
Now, the thing that most attention is focused on is global manufacturing and the supply chain. This is a huge challenge.
The resource challenges are tremendous, so primary strategies have to include global information sharing and regulatory collaboration. FDA has begun to get out there in other countries to build some of these bridges. We need to build our field capacity, including our field investigators' capacities to be overseas when they need to in critical places.
We have numerous state partnerships. The Food Emergency Response Network is a multi-state partnership. Our field folks now have a mobile lab that they can send out to sites where there's a problem, including border sites, to look for microbial contamination.
FDA and Health System Change
Finally, I want to talk about health system change. This shouldn't be medical products versus health system change. This should be medical products and health system change together improving people's health.
That's good for innovators. We just have to get it right. You can't just look at the products, because much of the economic issue is with the delivery system, and many of the opportunities, the most cost-effective opportunities, are in prevention.
We shouldn't forget that when we're talking health system reform, we're not just talking about dealing with the efficiency issues, but we're also talking about giving people access to care. Whether it's generic medicines or an appropriate program of follow-on biologics, that's also about access.
There are currently so many underserved and underinsured people, and this leads to tremendous disparities in our country and globally. This offers an opportunity. It's both a humanitarian opportunity, but it's also an opportunity for those developing and marketing products.
Where does FDA play a role here? We have to be scientifically excellent and objective to engage in this world. There is no question. So we need to build and rebuild the expertise and capacity of the agency. We need to keep our own people engaged in the world, collaborating, benefiting from others coming in to FDA, for example, and working with us.
And the culture is a huge thing that everybody from a company to a law firm to the FDA has to be very attentive to. We are very interested in supporting a culture of science, a culture that sees that public health outcome—not just risk and benefit—but that public health outcome, and a culture of teamwork so we make the best decisions. We can't make the best decisions as individuals.
I decided not to use direct words of the president—I am a career civil servant—but I came close, because I think it's a very important message: together we can. I think we can. Yes, we can.
But I also think that we really must. Emerging science offers these incredible opportunities for health, and for equity. We're a very complex system. We've got to pay attention to each other. We can't just go off everybody doing their own thing. You won't succeed. I won't succeed. FDA's people and their strength and integrity can play an important facilitating role, and I'd like to see that.
We all need to be humble. There's a lot of rhetoric in our society. There's a lot of adversity. We do have to all do our specific jobs. People ask us to make sure their foods and medicines are safe and effective, and at the end of the day we really have to do that. But we have to really think hard how we do that, and we have to hear from you and get your input about how we do that.
I ask the same from you and your partners, to learn and change. I would welcome in the time going forward people contacting me, your input, your questions, and more and more your engagement in a constructive enterprise to improve people's health.
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