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Speech before

National Press Club
on
“Dismantling Barriers to Better Medical Information”

Remarks by

Scott Gottlieb, MD
Deputy Commissioner for Medical and Scientific Affairs

September 28, 2005

Thank you for having me here today. I want to thank BioCentury for organizing this event and the National Press Club for hosting us today.

I’d like to start by telling you about a patient I saw when I was in my third year of residency.

It was a woman, 91 years old, and recently diagnosed with breast cancer. I’ll call her Nancy.

Nancy’s doctors started her on a drug to help treat the cancer, but like many patients as they advance in age, she experienced less of the benefits of the medicine she was taking, and more of its side effects.

One of those side effects was a propensity toward blood clots, and sure enough, she developed one. It went to her lung. And she died.

It is probably fair to say that Nancy died of her drug, not her cancer, and long before her tumor had really progressed.

At 91, she might have still lived a long time. In fact, her weak heart might have claimed her long before her tumor.

If Nancy was 30, surely she would have been treated for her tumor. But at 91, was that medicine really the right choice for her?

In other words, was the drug to blame, or a bad medical decision?

In many cases, in many corners of medicine, it is not a drug that hurts someone, but the wrong decision making. A drug that is lifesaving in one context can be deadly in another.

And separating the two scenarios sometimes turns on the slimmest amounts of hard to glean information and the very hardest and most personal of medical decisions.

That is why all medical decisions to use powerful medicines, all of which have certain side effects, is fraught with a certain amount of risk.

Whether its advanced age, or other co-morbidities -- there are many reasons why the risks sometimes outweigh the benefits of even the best medicines

The challenge for doctors and patients is to make their own right decision. The challenge is to match the right treatment decision, with the right patient, and at the right time

And in some cases, it is easier said than done.

But FDA is right now taking new steps to create an information architecture that will enable more timely and complete medical information right at the point of care.

Through steps I will talk about today, FDA is enabling the availability of all of the latest findings literally at every doctors and patients finger tips.

Doctors and patients need these kinds of knowledge system now. We need a drug IT infrastructure that is as robust as the biological systems that we are tasked with navigating each time we make a medical decision.

There are literally thousands of biological responses and molecular mediators that help determine how a particular person is likely to respond to a particular medicine. And those thousands are just the ones we know of.

It has become almost a cliché to say that genomics and pharmacogenomics is revealing thousands more biological markers every day that help determine how certain people will respond to particular medicines.

This science is going to help us do this matching of the right drug to the right patient much better. So we will no longer have to hang our hats on crude proxies for risk like age, or co-morbidities.

But even when we know everything we want to know about how a particular drug is going to affect a particular patient, having this information at your fingertips, and at the point of care, remains a significant challenge for many doctors and patients.

And that’s not to say that we ever know everything there is about particular drugs.

In fact, we seldom do. Even some of the oldest medicines still harbor certain mysteries.

Even the therapeutic benefits of a medicine as old and as simple as aspirin continues to reveal new truths

And so the challenge for all of us, doctors and patients and especially the Food and Drug Administration, is to tease the most prominent of these truths apart before medicines are approved, and then to quickly learn as new ones are revealed down the road.

And our challenge is to make sure this information gets to patients when they are ready to make choices about the medicines they use.

This is how doctors and patients manage their medical decisions.

This is how they manage the risks that are part of every medicine and every medical decision.

This is how millions of people every day decide whether the potential risks of a drug or treatment regimen are worth the potential benefits.

Walk into any doctors’ office and stand in any exam room, walk into any hospital and stand by any bedside, and you will see these decisions played out.

You will see that these are highly individual choices predicated too often on imperfect information, and they become the kind of choices that only a patient can make in consultation with her family and her friends and her doctor.

What is right for one patient in bed “A” may be wrong for the patient right next door in bed “B,” everything else being equal except for the preferences and the values that they bring to their individual decisions.

And so, at the FDA, our goal is to make sure we approve drugs with reasonable risks relative to their proven benefits, and then keep doctors and patients fully informed as new findings about medicines are revealed over time.

A lot of what we do at FDA involves getting patients the information they need to balance these risks and benefits.

Our job, when you boil it down, is to help turn raw information into practical knowledge that patients and doctors can use to make personal decisions about their health and healthcare products.

Let me tell you a little more about that work at FDA, as I see it.

We receive a lot of data about the performance, the safety, and the benefits of new medical products, and about medical products already on the marketplace.

With the help of our tools, with the energies of our skilled professional staff, and the aid of the guidance we get from outside experts such as those on our advisory committees, we turn this raw data into useful knowledge – knowledge that doctors and patients can use to help guide their decisions about how to most effectively use medical products to improve health.

That knowledge is what you read on our labels. It is what you see in our healthcare advisories.

And it is what I want to discuss with you here today. How we can do a better job of generating the most useful information for informing medical practice, and especially, how we can do a better job of getting this information into the right format and at the right place so that patients and doctors have it when they need it.

But our ability to generate and share this knowledge is only as good as the information we receive -- and it is only as useful as our ability to communicate it efficiently and effectively with the people who need it.

And the fact is that today many decisions involve difficult balancing of risks and benefits because we don’t have very specific information on whether a particular patient will experience a risk or a benefit. And we need to do a better job of that

And that is why we need the help of healthcare professionals and others who collect the kinds of drug safety reports that we use to uncover new potential risks that are associated with medicines.

These reports often include what went right and what went wrong with a particular medicine. We need help from doctors especially to send us more of this raw information.

And we need to do more to learn how we can be communicating all of these findings with patients and healthcare providers, in a way that informs the medical decisions that people make.

To do these things, to do them better than we are right now, we face some fundamental challenges.

Some of the recent high-profile problems with some popular medicines have sparked renewed interest in issues of drug safety, and have appropriately brought many of these challenges into sharper focus.

Most patients don’t expect there medicines to be completely safe. Most people accept that there is a certain amount of risk they assume whenever they use a powerful drug to perform powerful actions inside their bodies.

But most Americans are angry that it sometimes takes so long to find out whether or not a medicine has a particular problem.

People want us to uncover these problems much sooner and they want to learn about them more quickly and more clearly after they are fully revealed through a thorough scientific process.

The good news is that there are some very good ideas, many of which we are working on at the FDA right now, to make sure that we find out about drug side effects much sooner than we did in the past and that we can make this information available to patients and doctors much more quickly, more easily, and more widely.

But the challenge is that doing these things are going to require all of us to come up with new ways of monitoring medicines after they are approved, and of especially of informing patients and doctors of new risks and new benefits after they are definitively revealed.

Right now, too much important medical information is poorly accessible today or not accessible at all because we have failed to adapt for healthcare simple IT innovations that have long permeated the workplace.

At FDA, important medical information too often remains bottled up, shielded behind an over-worked and over-burdened staff, and preventing it from being more quickly redacted of its commercial and confidential content so that bottom line medical results are made public.

And too much important medical information remains shielded behind strict embargo policies and too long peer-review cycles that too often serve the interests of enterprising journal editors rather than patients.

It’s also sometimes hard to believe, if not ironic, that even as journals such as the New England Journal of Medicine and Science enforce these strict embargo policies, that they are pressuring everyone, including the FDA, to release information more quickly.

They are right. Information that could influence clinical medical practice needs to be made available more quickly, and more widely, after it has gone through a deliberative scientific process that firms up its meaning and the magnitude and the veracity of its conclusions.

But the medical journals can’t have it both ways. They can’t pound on everyone else, while they conceal peer-reviewed information behind long publishing cycles and strict embargos only to prop up the value of their own franchise.

Now don’t get me wrong. I support the medical journals and the vital role they play. I have served on the editorial staff of two of the leading medical journals, and have written hundreds of medical news articles for them.

But as a friend to the journals, and as a champion of their vital role, I need to ask why they retain the luxury of bottling up information behind peer review processes that can wind on for weeks or months longer than they have to, and embargo policies that serve no useful purpose in a time when medical writers are often professionally schooled in the sciences that they report on.

That is a question that concerns me because it has an impact on the public health. In its September 15th issue, for example, the New England Journal of Medicine published an article concerning early treatment of acute coronary syndromes – the precursors to heart attacks. Just a week earlier, while rounding at my hospital, I had seen a patient with just such a condition.

I am not sure the article would have directly impacted by thinking on how to treat my patient, but it may have.

Could I have received this information sooner? Many of the journals say they release the most pertinent information as web based articles, sometimes months before they appear in print, in order to get them out more quickly.

Surely this doesn’t mean everything else that appears in the print editions doesn’t impact clinical practice. And surely the fact that they can get the web postings out sometimes months before the print versions appear doesn’t mean that these intervening months of delay need not exist?

But since I don’t work for these journals anymore, I can’t answer these questions. I can only pose them. When it comes to making sure consumers are armed with the most up to date and complete medical information, I want to focus the remaining time on something I can affect.

And so I want to focus on the challenge at FDA of not how we collect raw data and turn it into useful medical information.

That is a significant task with its own unique challenges.

But on how we use that information that we derive to help inform doctors and patients and therefore inform medical practice.

And I want to tell you about some of the steps we are taking at FDA to meet these challenges.

Right now, when you are a doctor practicing medicine, bottom-line information isn’t easily accessible when a medical question comes to mind.

The information that is specific to a doctors’ particular medical specialty isn’t always easy to pick out from the reams of general medical information that may be superfluous to a particular doctor’s clinical practice.

So cardiologists are inundated with warnings and writings about psychiatry while surgeons get deluged with data on diabetes.

And matching up a patient’s particular characteristics which the subtle signals and data nuggets that might alert you to a rare problem isn’t as easy as it should be.

Last night, Amazon.com was able to plumb all of my past buying habits and the demographic data that they have on me to alert me that a new book about drug development may be just what I wanted.

But when I go into the hospital to see patients, I need to keep track of dozens of problems my patients have – and those are just the ones I can readily identify – and dozens of side effects common to their medicines.

Then I have to try and do a matching, all within the span of a few mental minutes, to see if any of those problems are going to coax out one of those side effects.

So what if we had a system where information technology tools were able to keep track of all of those problems and those side effects, and readily integrate new ones, and seamlessly alert doctors when their patients had a particular side effect that was likely to become more common.

It’s true that the bottleneck to these kinds of systems have been the lack of information technology tools, and the widespread adoption of electronic medical records that make archiving and retrieving this kind of data possible.

But the problem has not only been about hardware and software, but also about the absence of data. It’s like asking Amazon.com to predict my book-buying habits without giving its computers the benefit of knowing about all the FDA insider books I bought over the last five years.

This is where FDA can help. We are fundamentally in the business of turning raw medical data into knowledge. For many years, we didn’t do as good of a job as we could have of sharing this knowledge widely.

In some cases, we didn’t have the resources, but in others we just didn’t think we had to. This is another place where information technology tools are also helping.

With advances in the way that doctors and patients retrieve medical information, at FDA we have been able to fundamentally re-think the way we provide it. And we are doing just that. Let me tell you how.

Beginning in November 2005, healthcare professionals, patients, and other consumers will be able to access high-quality, up-to-date information on medications via the Internet free of charge through the Daily Med system, which will receive, distribute and display medication information that has been developed by Federal agencies.

This is a monumental change in the way medical information is made available that would not be possible without the leadership and hard work of the professional staff at FDA’s Drug Center and especially my colleagues Dr. Janet Woodcock and Dr. Steven Galson.

A key element of the Daily Med is the electronic product label, known as the package insert. This is the onion-skin piece of paper folded up inside the tops of your pill bottles.

Right now, the content and format of these paper labels is not conducive to easy accessibility, and they are not easy to search for pertinent information, or even find on the web.

FDA will release soon a new rule that would fundamentally re-make this package insert, or the physician label, making the information that’s provided more readable, more accessible, and more amenable for electronic dissemination.

This is an important step toward creating an electronic environment for drug safety and drug effectiveness information that will make it easier for doctors and patients to get up-to-date information right at the point of care. There are many other steps we are taking.

Next month, FDA is requiring drug makers to start submitting label changes electronically in a standard format that, after verification by the FDA, will make updating labels with new information seamless, and automatic.

Imagine a world where the product labels can be pulled off the web or better yet, off a PDA that that doctors carry in their pocket, that is updating instantly each time new safety warnings are added or new information about a drug’s benefits. Doctors could receive special alerts for the drugs they are most likely to prescribe.

In addition to these things, FDA is taking other steps to help build the systems, infrastructure, data standards, and strategies to support and sustain the management of that information well into the future.

We are modernizing our system for reporting drug safety information, called Med Watch, as well as FDA's other reporting systems by expanding on-line reporting, increasing interoperability, and moving toward a consolidated system.

One of the things we’re seriously discussing is whether we can develop better reporting from the Med Watch surveillance data to help better inform doctors about what we are finding, and to help stimulate additional reports.

Right now, when doctors file reports with us about potential problems they might have observed, we have nothing to offer them back in return.

One idea is to do more to report the public information from our Med Watch database in a timelier fashion, perhaps with some kind of regular report from this surveillance data.

It could be something as simple as a regular report, similar to the Morbidity and Mortality Weekly Report that is published by the C.D.C. FDA already reports on the data in Med Watch, but right now this information comes out quarterly.

If we made this information available more regularly, or even in near real time, doctors might be prompted to report more of what they are finding

Making wider and regular reporting of this kind of surveillance data needs to be done with care, but it is far easier and far less likely to confuse consumers and doctors than is making public interim but still scientifically uncertain findings that have undergone a preliminary scientific review process but one that still remains significantly incomplete.

When it comes to the Med Watch data, it is very clear that these are the earliest of early findings that is still un-scrubbed by scientific rigor.

So what is the value then of more widely and regularly releasing this information?

Well, it is manifold, and it is significant. Making this information more widely and quickly available could be a big step in helping to stimulate more wider reporting of potential safety problems.

For medical devices, we are expanding on a program we call Med Sun, where FDA collaborates with healthcare institutions to proactively go out and stimulate the discovery and collection of information about potential problems with medical devices.

FDA’s new bar-coding rule will require bar codes on labels of prescription drugs and over-the-counter drugs.

And the agency is working closely with standards development organizations such as HL7 and CDISC and other agencies, and industry to develop standards for medication information exchange and management and adverse event reporting.

All of these initiatives, as well as several others, together make up what we are calling Facts@FDA – a new electronic architecture for making timely, reliable information available to doctors and patients when they need, where they need, on demand.

And taken together, this comprises a big step into the future of healthcare information, a future when medical decisions can be guided by the most up-to-date and reliable information possible.

We still need to do more to stimulate research that will help unearth information about subtle side effects as well as new benefits of medicines.

We shouldn’t have to wait many years to discover that a drug is causing some unwanted problem, or that a cancer medicine that is originally approved for a third line or terminal indication can literally save thousands of lives when its used much earlier in the course of disease.

We shouldn’t have to wait many years to answer these questions. But when we do get these answers, we need to make sure they get to patients as quickly as they are fully revealed.

And right now, whether its Byzantine embargos or hard-to-hunt down drug labels or overworked officers charged with redacting public information of their private contents, too much information is not getting to patients when they need it and this is a public health hazard.

This is something especially important for many of you here today. Because even with the best information architectures, reporters are often the front line in informing patients about new findings about medicines

And so at FDA we’re making some new commitments to work more closely with reporters as well.

We’re working on a policy that some of the wire reporters call “lock-ups.” We won’t be locking up reporters, although I should remind you we are a law enforcement agency.

We’ll be inviting you in on very important public health announcements to make sure you are fully briefed for a few hours by our experts.

We’re working on some plans to have more regular conference calls on policy issues, similar to the open door forums that the Centers for Medicaid and Medicare Services has developed with a lot of success.

And we’re also going to be taking other new steps, with limited resources, to get information out to the press sooner.

We should be able to release more bottom line medical data more quickly while protecting commercial confidential information.

All of these things are part of our commitment to make sure more information gets to patients and doctors when they need it.

If we’re going to remain the world’s gold standard for drug safety and drug evaluations, we need to do a better job of telling consumers what we’re finding.

And with your help, that’s exactly what we’re doing. I want to thank the National Press Club for hosting me here today, and thanks again to BioCentury for organizing it. Thank you.