In the past year, the Food and Drug Administration approved 35 new medicines — a near-record for the last decade. After years of slow but steady drug approvals, the FDA is considering speedier review and acceptance of innovative medicines, especially obesity treatments, antibiotics and drugs to fight infectious diseases. But scientists warn expedited approvals come with enhanced risks. We explore the push for new medications and the struggle to balance risks and benefits.

Guests

  • Dr. Robert Temple Deputy Director for Clinical Science, Center for Drug Evaluation and Research, U.S. Food and Drug Administration
  • Thomas J. Moore Senior Scientist, Drug Safety and Policy, The Institute for Safe Medication Practices
  • Allan Coukell Director, Medical Programs, Pew Health Group

Transcript

  • 13:23:23

    MR. KOJO NNAMDIIn the early 1940s, Australian scientist Howard Florey and a team of researchers developed penicillin, an antibiotic that went on to save millions of lives. Florey would live to see the development of 13 new classes of antibiotics. But since his death in 1968, just two classes have made it through the drug pipeline. Now many infections are resistant to standard antibiotics so the Food and Drug Administration is putting priority on fast tracking new antibiotics, as well as medicines for weight loss and other diseases through a traditionally slow approval process.

  • 13:23:59

    MR. KOJO NNAMDIWhile many patients in dire straits may applaud a speedier route for badly needed drugs, some experts wonder if safety is being sacrificed in the process. So how do you balance safety and speed in products where millions of lives might be at stake? Joining us in studio to discuss this is Thomas Moore. He's senior scientist in Drug Safety and Policy at the Institute for Safe Medication Practices. He's also the project director for "Quarter Watch," an independent drug safety publication that examines side effects data reported to the FDA. Tom Moore, thank you for joining us.

  • 13:24:34

    MR. THOMAS MOOREIt's a pleasure to be here.

  • 13:24:36

    NNAMDIAlso with us in studio is Allan Coukell, director of the Pew Health Group's medical programs at the Pew Charitable Trusts. Allan Coukell, thank you for joining us.

  • 13:24:45

    MR. ALLAN COUKELLGood to be here, Kojo.

  • 13:24:46

    NNAMDIAnd Dr. Robert Temple is deputy director for Clinical Science at the U.S. Food and Drug Administration. Bob Temple, thank you for joining us.

  • 13:24:54

    DR. ROBERT TEMPLEGood to be here.

  • 13:24:55

    NNAMDIIf you'd like to join the conversation feel free. Call us at 800-433-8850. Would you take a drug that's gone through faster, smaller clinical trials if it meant alleviating a dire condition? 800-433-8850 is the number to call. You can send us email to kojo@wamu.org or send us a Tweet at kojoshow. Bob Temple, I'll start with you. Usually about 20 to 30 drugs are approved each year by the FDA. Now we're seeing some very public plans by your agency to speed approval of some new medicines. Can you give us some context on why we're seeing this new push?

  • 13:25:34

    TEMPLEWell, I think it starts with the fact that there're certain diseases and conditions that don't have any good treatment. There may be something but it doesn't work very well. And these are usually serious and life-threatening diseases. Almost everybody agrees -- even people who aren't sure about how much safety data we get -- that you want new treatments for diseases that don't have a treatment. So if there's a resistant bacterium and nothing now treats it, there really isn't anybody who doesn't think there ought to be something that'll treat it.

  • 13:26:04

    TEMPLEBad neurological diseases need treatment. Bad diseases like cystic fibrosis need treatment. When we've talked to people -- that includes physicians, patients, actually legislatures -- they pretty much all agree that if you had something that worked when nothing else did for a serious or life-threatening disease you would probably accept somewhat greater risk. And I think everybody agrees with that. I'm sure almost everybody does. There is some people, however, who think we give up too much and that's the question.

  • 13:26:39

    NNAMDIIn the past year, the FDA has approved 35 new drugs and almost half of them received some sort of shortened review or expedited approval. Can you tell us about one or two of those drugs and why they got through faster?

  • 13:26:54

    TEMPLEThe term expedited approval has a lot of different meanings. One thing we do, for example, is if a drug looks very hot we might review it in six months instead of the usual ten months. So one of the drugs that Tom Moore singled out was a drug called Fingolimod. That got a priority review and that was a drug that treated -- I just went blank.

  • 13:27:25

    MOOREMultiple sclerosis.

  • 13:27:26

    TEMPLE...that treated multiple sclerosis, sorry. The first very effective oral drug that did so and it worked almost as well as the drug that people were quite worried about called Tysabri, which causes a rare neurologic disease. So that got a priority review because it worked extremely well and that meant we gave it a six-month review. That doesn't mean we think that we decreased the level of review in any way. It just -- we did it faster, we devoted more resources to it.

  • 13:27:55

    MOOREI'd like to...

  • 13:27:56

    NNAMDIWell, Tom, what he was referring to is the September article that you wrote in the Journal of American Medical Association where you looked at three newly approved drugs that received these speedy reviews. And you found that you had some safety concerns. What is it that left you uneasy?

  • 13:28:10

    MOOREWell, I agree with Bob Temple in part and I disagree with him in part. First, I think the metric of speed of approval is sort of misleading and without value. I mean, the whole idea that the way we should judge the performance of the most important safety regulatory agency in the world is how fast they can move. That seems to me sort of an unhealthy proposition to start with.

  • 13:28:41

    MOOREThe real underlying question is two parts, I think, which is what kind of safety questions remain unanswered. And so in the case of one of the drugs that I described in the article, there were seven different safety questions that we really didn't know the answer to. So my sort of cautionary note here is speeding new drugs with less testing to the public is at best a mixed blessing, and if we're unlucky can lead to a serious drug disaster.

  • 13:29:12

    NNAMDIWell, before I bring Allan Coukell in, just one bit of clarification.

  • 13:29:14

    TEMPLEI'd like to respond...

  • 13:29:15

    NNAMDIBob, I know you'd like to respond to that and as you respond could you just briefly go into the weeds a little bit and talk about the four designations that drugs in the fast lane get at the FDA and what it means about the scientific testing behind them.

  • 13:29:28

    TEMPLEYou mean the four different categories that might...

  • 13:29:30

    NNAMDIFast track, accelerated approval, priority review, breakthrough therapy.

  • 13:29:33

    TEMPLEThey're all quite different. Priority review refers to how soon we have a response. It doesn't mean a yes -- how soon we'll have a response after the drug comes in, six months or ten months. That's all it refers to. Fast track doesn't refer to approving the drug at all. It refers to getting in contact with the company that is developing the drug, trying to help them do the most efficient possible studies, making it clear what the requirements are going to be and things like that. It also does say we will accept what's called a rolling review, which means they're allowed to put their chemistry data in before they put their clinical data.

  • 13:30:12

    NNAMDIAccelerated approval?

  • 13:30:13

    TEMPLENow accelerated approval is something of a misnomer. That refers to basing approval on what's called a surrogate endpoint. A surrogate endpoint -- the usual endpoints of a clinical trial are whether you live longer, function better or have fewer symptoms. A surrogate endpoint is a measure like blood pressure that isn't a measure of that but that you think will predict improved survival or fewer strokes.

  • 13:30:37

    NNAMDIAnd breakthrough therapy?

  • 13:30:38

    TEMPLEWell, hold on. The surrogate endpoints we use in accelerated approval are surrogate endpoints that we're not 100 percent sure are going to be predictive. But we think they're reasonably likely to be predictive. And you can often get an answer on such a surrogate endpoint long before the survival data come in. When we use that for approval it's on condition that the real endpoint be studied in further studies afterward. The major place we use that is in cancer. And the surrogate endpoint we use is shrinking the tumor or delaying the progress of the tumor. A very plausible surrogate endpoint, but it isn't actually survival.

  • 13:31:19

    TEMPLEOkay. What have I done...

  • 13:31:20

    NNAMDIBreakthrough therapy.

  • 13:31:20

    TEMPLEBreakthrough therapy.

  • 13:31:21

    MOOREWell, there's an important element of accelerated approval that he omitted.

  • 13:31:26

    NNAMDIBreakthrough therapy?

  • 13:31:26

    TEMPLEBreakthrough therapy is a new term. It's in the recent law (word?) . And it resembles a number of the other things but it means that early clinical evidence have suggested that the drug treats a serious or life-threatening disease that is not currently well treated. And, once again, what we're going to do in that case is work closely with the companies to try to get studies that will be efficient. We have various ways to make studies more efficient, work with them to do that. It doesn't suggest a change in standards at all.

  • 13:32:01

    TEMPLEThe reality is though if a drug does something wonderful that's never been done before we will often accept the smaller safety database but not always.

  • 13:32:09

    NNAMDIAllan Coukell, I have a question for you, but you go ahead. You wanted to jump into this conversation anyway.

  • 13:32:14

    COUKELLYeah, well, thank you. I want to talk about antibiotics, but first I wanted to respond to something that Tom said, which is that speed of drug approval is without value. And I think if you're a patient with a serious disease and you're lacking an effective treatment, then speed of drug approval is of immense value. It's also of immense value if you're a company and you're burning capital to get a product to market.

  • 13:32:34

    COUKELLSo let's say that over the last decade-and-a-half drug approval times at the FDA have fallen. We commissioned a study that was published earlier this year in the New England Journal that showed that the FDA is now approving drugs faster in the United States than European authorities are, than Canadian authorities. You know, two-thirds of drugs come to market here first.

  • 13:32:55

    COUKELLBut I think the right question is not how fast are they coming to market. It's are we getting the evidence that we need, both for the effectiveness of the drugs and the safety. And as we look about are there better ways to get antibiotics to market, that's what we need to be looking at.

  • 13:33:10

    NNAMDILet's be specific. FDA Commissioner Margaret Hamburg recently mentioned three categories of drugs for accelerated approval, weight-loss treatments, antibiotics and drugs to treat infectious diseases. I'd like to delve into the first category I mentioned, since I think some people may wonder why weight-loss drugs are getting this treatment. Why obesity medications, Allan?

  • 13:33:30

    COUKELLI'll talk more about obesity drugs...

  • 13:33:33

    NNAMDIOkay.

  • 13:33:33

    COUKELL...but obesity drugs have -- some of the recent ones have had some serious cardiac risks. And so, you know, what FDA is talking about there is making sure that if we're going to take drugs that have a certain risk profile we're making sure that the patients who get them are the ones who stand to benefit most, if I can oversimplify. Similarly when we look at antibiotics we, as you know, have emerging infections in this country that can't be treated or often can't be treated by very many drugs.

  • 13:34:05

    COUKELLThere was a big outbreak not long ago at the NIH clinical center not far from where we're sitting now in Washington. And so we have to be sure that when we have these bugs that can't be treated -- patients that can't be treated with existing drugs, we have available therapies for them.

  • 13:34:23

    NNAMDITom...

  • 13:34:23

    MOOREYeah, I'd like to just pick up one point where I think that the public may not understand part of the underlying structure here. And that's starting with accelerated approval.

  • 13:34:34

    NNAMDIOkay.

  • 13:34:35

    MOOREAccelerated approval, as Bob Temple said, is based on a measurement of the drug benefit that might not be a real benefit. But the second important part to remember about accelerated approval -- and it could be well justified in some cases -- is that it is typically based on a single phase II clinical trial, 200 or 300 patients. So we have to understand we are taking risks here when we launch a drug into clinical use that only a few patients have taken in a single trial. Not that it hasn't worked out well for some drugs, not so well for -- occasionally for others, but you have to understand here, we're not getting a free ride.

  • 13:35:12

    MOOREThe other part I would just sort of refocus is I believe that the commissioner was talking about obesity drug sort of as a priority, not that it was going to be used in this legal mechanism, but that this was an area where the agency would welcome new drugs and evaluate them carefully. Because the obesity drugs that we have historically have proven -- have been accounted for a disproportionate number of drug disasters. And the two newest ones we have are marginal at best and are available only after the FDA changed its view of them.

  • 13:35:45

    NNAMDIYeah, but Allan, we have statistics saying that 36 percent of U.S. adults are obese. Is it fair to say that we're at the point where the benefits of reducing our waistlines might outweigh the risks that have delayed these kinds of drugs in the past?

  • 13:35:59

    COUKELLI think any time we look at the value of new drugs, the real question to ask is are we making people's lives better and are we preventing a illness or curing illness? So as we look at obesity drugs, that's the question we have to ask.

  • 13:36:14

    NNAMDIWell, Tom, both of the obesity drugs that were approved this year were rejected in 2010 because of safety risks. As an independent drug safety expert have you looked at the research behind these drugs that now make them marketable?

  • 13:36:28

    MOOREI have looked at them and they do indeed have some serious risks that you'd want to consider. And you would have questions about whether it’s a close call at best as to whether some of these drugs have benefits that outweigh their risks. One of them is a combination of two drugs we've had for 20 or 30 years. One of them is a stimulant that has addictive properties and probably some cardiac adverse affects. The other half of that old combination is a drug for epilepsy.

  • 13:37:00

    MOOREDrugs for epilepsy have warnings about suicidal behaviors, hallucinations and some psychiatric effects in some people. Also it has a very serious risk of birth defects. So we have a drug that is a hard balance between its risks and benefits. And the FDA initially had sort of reversed it. There was really a huge political organizational pressure on the FDA saying we have to have drugs.

  • 13:37:30

    MOORENow my question is, are we better off as a society with a marginal drug whose weight loss was really extremely modest with less than 10 percent of body weight in most patients and a lot of risks? Or do we want to ask our regulatory agency to be, if you will, our better angel and say, you know, we set a standard based on experience and these drugs don't make it.

  • 13:37:53

    NNAMDI800-433-8850 is the number to call if you'd like to join the conversation. What kinds of drugs do you think should get fast track at the FDA, if at all, or do you feel should all drugs get the same rigorous testing before they come to market? Let us know how you feel, 800-433-8850. You can send email to kojo@wamu.org. Bob Temple, I would imagine that the FDA has to be extra careful about approving weight-loss drugs since more than just obese people might be taking them. How is the FDA communicating to doctors and patients that these fast track drugs are for patients with the most dire need?

  • 13:38:30

    TEMPLEWell, we are being careful and one of the things we're talking about is the possibility -- but this is not established yet by any means -- that an initial approval could be for people who are really in trouble, morbidly obese, you know, can't get out of their chairs and things like that. Whether we can find a way to do that I don't think is a settled question yet. But clearly those people with the greater degree of weigh loss are both more needful and potentially at greater risk. So this is a difficult balance.

  • 13:39:01

    TEMPLEI should note that nobody's talking about small databases here. We're looking at big databases and in many cases we're asking for people to do what are called outcome studies, actually give the drug to some people, give the drug to others and look at the rate of heart attack, stroke and other things like that. We have not done that in every case where we were reassured enough not to do it. But that's what's on our mind.

  • 13:39:24

    TEMPLEJust what you said before, morbid obesity is a major health quest in this country. People are dying because of these things. You don't want to make them worse, but you don't want to ignore that. And it's perfectly true that some people who don't have that kind of need use these drugs. And we're worried about that. We don't want them to.

  • 13:39:44

    NNAMDITom Moore.

  • 13:39:45

    MOOREWell, the problem I have is with an obesity drug that had a dramatic effect in a morbidly obese population and caused them to perhaps lose 25 percent of their body weight and come closer to normal. But the fact of the matter is these two drugs had very marginal effects. People who are morbidly obese are going to be very little better off on the average than they were before the treatment, and they will have accepted to risks.

  • 13:40:16

    MOOREThat's the hard judgment call. If we had drugs here that were very effective and were working, it might be that some of the most obese people would be willing to take those risks, although if they lost the roulette game, I don't know how they would feel then, and obesity drugs have featured, if you will, some of the worst things that we've had happened. We had the Fen-Phen drug tragedy in which tens of thousands of people suffered irreversible damage to their heart valves.

  • 13:40:44

    MOOREOne of these two new drugs might or might not share that same risk. We can't tell. We hope it won't, but we can't tell.

  • 13:40:53

    NNAMDIWell, we're tossing...

  • 13:40:53

    MOORESo we're talking about serious risks.

  • 13:40:54

    NNAMDI...numbers around here. What you're saying is that weight loss drugs have been approved if somebody weighs 500 pounds, and that the weight loss is only going to take 10 percent of that weight off, that that drug will only take that person down to 450 pounds.

  • 13:41:09

    MOOREAnd it would be probably less than that.

  • 13:41:09

    TEMPLEThe trouble is you don't know that. All of us do this, and it's a mistake. We tend to look at the average weight loss.

  • 13:41:16

    NNAMDITrue.

  • 13:41:16

    TEMPLEThat's a mistake. There's distribution of weight losses, and you see this in weight loss trials and in every other kind of trial where some people have a considerably greater response than others, and so one of the things we've thought about labeling for these drugs is to say if you don't lose X amount of weight in the first four months, or three months, or whatever it is, stop taking the drug. It's not going to do you any good. And that's not an unreasonable possibility.

  • 13:41:41

    TEMPLEIt's very important to know the distribution of responses, and for all of these drugs. Some people lose a lot more than five percent of their weight.

  • 13:41:47

    NNAMDIWell, Allan...

  • 13:41:48

    TEMPLEAnd many people don't.

  • 13:41:49

    NNAMDIAllan Coukell, this might be the major question. Once the FDA fast tracks a medication and it's approved, what kind of follow up goes on afterwards? Is it hard to put the genie back in the bottle so to speak?

  • 13:42:02

    COUKELLI think your, you know, the question is -- there are two questions there, Kojo. One is, once the drugs are out on the market, are our systems as good as they could be at monitoring safety and detecting adverse events, and I think the answer is we have made progress in recent years. There's a system called Sentinel which is being built to more systematically look at the safety of the drugs when they're on the market, and certainly when things are approved by the FDA in many cases they order the companies to do studies once the drugs are out, and it's really important that we keep looking at long-term outcomes, keep looking at safety so that we know the answer to those questions.

  • 13:42:47

    NNAMDIGot to take a short break. When we come back, we will continue this conversation on the Food and Drug Administration's fast track drug approvals. If you have called, stay on the line, we'll try to get to your calls. The number is 800-433-8850. Have you ever taken a drug that's brand new to the market? How did it work for you? 800-433-8850. I'm Kojo Nnamdi.

  • 13:45:07

    NNAMDIWelcome back to our conversation on the FDA's fast track drug approvals. We're talking with Dr. Robert Temple. He is deputy director for clinical science at the U.S. Food and Drug Administration. Allan Coukell is director of the Pew Health Group's medical programs at the Pew Charitable Trust, and Thomas Moore is senior scientist in drug safety and policy at the Institute for safe medication practices. He's also project director for QuarterWatch, an independent drug safety publication that examines side effects data reported to the FDA.

  • 13:45:37

    NNAMDIAnd Tom Moore, you wanted to continue the conversation about post-market scrutiny of drugs.

  • 13:45:43

    MOOREYes. I believe we are making some progress, but that the public does not understand how primitive our systems are once a drug has been approved and run that first gauntlet of approval. The first point, and this is a study I'm just working on right now, looked at all the drugs in 2008, and the issue in my mind really is not how long it takes the FDA to think about and discuss the scientific results of testing, but how long the testing takes all together.

  • 13:46:13

    MOORESo we studied every drug that was approved by the FDA in 2008, and started the clock going from the first human testing. And we found that drugs we get today, the ones that were in 2008, took about seven years from the very first human testing to enter the market. We also did a study of every major safety decision in post-market made by the FDA, and we found -- in 2009 actually, and we found 181 of these major, major safety issues that the FDA had to address after approval, and the median time it took was 11 years.

  • 13:46:50

    MOORESo we have actually a post-market system that is working much more slowly than our drug-approval system. And so we need to think very carefully about if we lower the risk -- if we lower the amount of testing that we require for new drugs, we don't really have a system for catching up with those risks once that approve that is very good.

  • 13:47:12

    NNAMDII am sure that Bob Temple wants to respond.

  • 13:47:15

    TEMPLEWell, the first thing we have to do is see the data. I assume you mean 11 years after approval.

  • 13:47:20

    MOOREYes.

  • 13:47:21

    TEMPLEYeah. Well, I can tell you, a lot of those are going to be things like when we discovered that whole class of drugs has a problem. For example, we, somewhere around that time, relabeled all antidepressants with the possibility that they could cause suicidality, and I'm sure that was many years after many of them were first approved. We also labeled all anti-epileptic drugs with the same label.

  • 13:47:44

    TEMPLESo to the extent these things are that which took accumulated data for decades, I'm sure you're right, that the number could be large. But if a drug causes something overt, it doesn't take us 11 years to figure it out. The other thing is, a recent experience shows that some of the new systems we have in place may well be of value. One of the drugs that was talked about by Tom was an anti-coagulant called Dabigatran, which is an alternative to Coumadin which has been in the marketplace for decades and decades.

  • 13:48:21

    TEMPLEIt was not approved on the basis of a small data base at all. There were 18,000 people in the clinical trial. There was no difference in bleeding rates between the new drug, Dabigatran, and Coumadin. It was actually slightly less, but there was nothing obviously bad. But shortly after approval we got hundreds of reports of people bleeding on the new drug.

  • 13:48:45

    TEMPLEWe couldn't tell whether that was because people report more on a new drug than on the same old drug, but anyway, we put out a public announcement about it and said we were going to use this mini Sentinel system to look at that. We just on Friday reported the results of that. There was really no evidence at all that bleeding rates were higher in the Dabigatran group. They looked slightly lower, but we don't think that's a reasonable conclusion.

  • 13:49:08

    TEMPLEBut they didn't look higher. Meanwhile, the drug that's available, at least in the way it was used in the large 18,000 trial, had a bigger effect on preventing strokes, both strokes that -- this is in people who have atrial fibrillation, both the strokes that occur because clots form in the atrium and go off to the brain, that kind, and also because, for reasons that aren't quite clear, Coumadin itself causes bleeding strokes in the brain, and these drugs do it -- the new drug does it less.

  • 13:49:41

    TEMPLESo we thought we got a very valuable drug out. Hardly -- I mean, we certainly gave it a priority review, but we didn't think we were skimping on anything, and we were able to take care of this quite, you know, signal that has to be of concern, everybody's bleeding, with the mini Sentinel thing which looks at nine different sites and none of them showed an increased rate of bleeding. So we're pretty excited about the ability of that program to at least detect large effects.

  • 13:50:09

    NNAMDIAllan Coukell, the FDA gets its money to review and approve new drugs from something called user-fee agreements, which the drug industry will pay to the tune of nearly half a billion dollars over the next five years it's my understanding. Can you give us an idea of how this money is used, not only to get drugs to market faster, but also how it's used to follow up on the safety of what's released?

  • 13:50:30

    COUKELLSo depending which part of the FDA you're talking about now. Getting close to half the budget comes from the fees that the industry pays. Most of that money goes to funding the reviewers who look at the new drug applications. In some cases, the user fees can also be used to support some of the other science that the agency does in some of this post-market safety work that we're talking about. But in general, most of it is just for the review, and I think this does point to a real challenge when we're looking at what should the FDA look like into the future and what do we all want.

  • 13:51:09

    COUKELLWe have to recognize that there's lots of new science out there that we need to build an infrastructure both for detecting safety problems, and also so that the scientists at the FDA are up-to-date and answering questions that we'll need to answer to approve or evaluate new drugs and technologies that we haven't even thought of yet.

  • 13:51:30

    COUKELLSo there's going to be an ongoing need from one source or another for this kind of, what's sometimes called regulatory science, the science that supports the decision makers that -- the decisions that the regulator has to make.

  • 13:51:41

    NNAMDIGentlemen, please don your headphones all because I'm going to the phones. We'll start with Lisa who is in Fairfax County, Va. Lisa, you are now on the air. Go ahead, please.

  • 13:51:52

    LISAYeah, hi. I'm not sure, in the late '80s or early '90s, I was I think involved in a study. I had to go to Georgetown Hospital Department of Neurology. They had a special headache center, and was involved with the -- what was the new product at the time, it was one of the Triptan drugs for migraines, and was able to get on it really, really early. And I remember the neurologist telling me that one of the other patients in the study was a woman who had 12 children, and the only reason she had 12 children was because the only time she didn't have migraines was when she was pregnant.

  • 13:52:23

    LISAAnd I remember that that was how desperate we all were to find something for migraine, and we were -- we had to go through a whole bunch of -- almost like MRI-type tests to make sure that the drug was interacting safely with us because it was so new. Absolutely saved me. Without it, I wouldn't have made it into the '90s. It was really bad. I was happy to do it. It worked out really well, and now I think there's almost generics of the Triptans now. There's three or four different companies -- or versions of it, and it was well worth whatever it took to get in early on a drug like that for me.

  • 13:52:56

    NNAMDIWell, thank you very much for your call, Lisa, because I want to go to Jof in Crofton, Md., who apparently did not get in as early as you did on a trial, but we'll hear how it worked out for Jof. Lisa, thank you for your call. Jof, you're now on the air. Go ahead, please.

  • 13:53:13

    JOFHi. My name's Jof. I have MS, and have had MS for seven years. I've been on the drug tysabri you guys were talking about. It got me back to able to run. I couldn't run a mile without falling down. Now I'm trying to run a half marathon. I had question...

  • 13:53:32

    NNAMDIAnd you got in you said on the tail end of the program, right?

  • 13:53:35

    JOFI actually came in right after it was reapproved. It went off the market because people got brain disease or brain infection and died. It came back on the market, and a couple months later I got on the drug, and I'm now beyond any of the studies for length of time on the drug in terms of how safe it is.

  • 13:53:58

    NNAMDIOkay. You had a question?

  • 13:54:00

    JOFMine was on the positive results for the FDA, those go through, but what about all the ones that are rejected? I know that a lot of the drugs that are rejected, the papers aren't ever published on the actual trials. Is there anything they can do to actually try and publish those so that we don't keep retesting things?

  • 13:54:20

    NNAMDIBob Temple?

  • 13:54:21

    TEMPLEWell, under current rules, actually the reports of these studies do have to be put into clintrials.gov. Whether they actually get published or not is another question, but there's a fair amount of detail that goes in there, and we think people are complying with it, but, you know, we don't know for sure. We certainly -- we would all agree that the results of the study ought to be published and made available both to the people in the trial and to everybody else.

  • 13:54:49

    MOOREThere's also another step that the FDA could take that is taken in Europe, which is, it is public information when a new drug approval is rejected. But it's extremely difficult to find it or identify it, and there's several classes of information -- valuable information. Now, the FDA overall I want to say has the best record in the world for transparency. But this one is an exception, and the FDA could make much more readily available and accessible to the public it's decisions to turn down a drug, and also its decision to open up drug testing when IND...

  • 13:55:25

    MOOREAnd this would leave us all better about what drugs were -- better informed about what drugs were in the pipeline, and what drugs had had problems.

  • 13:55:32

    NNAMDIJof, thank you for your call. Glad you brought up the Europe experience, Tom, because, Allan, drug makers have long complained that the FDA is too bureaucratic and demanding in terms of proving the safety of experimental medicines. How does the U.S. to compare to Europe and to Canada in how fast we approve new drugs?

  • 13:55:49

    COUKELLWell, I mentioned this briefly earlier, Kojo, but we commissioned a study that looked at this and it was published in the New England Journal earlier this year. And what we found is that in general drugs come to market faster in the U.S. than they do in Europe and Canada. About two-thirds of the drugs that were approved in, you know, two of those markets came first in the U.S. and in general it was about three months quicker.

  • 13:56:15

    COUKELLSo, you know, there are important questions about whether we can reduce the cost of drug development by getting better and faster at doing clinical trials, but in terms of approval times and who's getting access to the treatments first, right now the U.S. looks pretty good.

  • 13:56:29

    NNAMDITom, let's talk about Canada for a minute, because researchers there recently found some troubling issues with drugs that have been on the Canadian market for as long as 15 years. Can you tell us about that?

  • 13:56:37

    MOOREWell, that was a study of priority reviews, which are somewhat similar, but not identical with the priority reviews of the FDA, and that study simply showed that priority review drugs were more likely to have had an important safety problem overlooked than ones that receive the standard review. But I would like to just return to the speed...

  • 13:56:59

    NNAMDIAnd we only have one minute left, but go ahead, please.

  • 13:57:01

    MOOREOkay. To return to the speed derby. I mean, you can get a drug out quicker by simply lowering your standards, and that that should be our principle metric I think is really a mistake, and if you just simply do sloppier or more rapid reviews you can get drugs out quicker. And so I think we have to look more carefully at what questions there are about the drug that have been answered and not answered.

  • 13:57:20

    TEMPLEIt's very important to say that there is not the slightest evidence that we've compromised the evidence of effectiveness or safety, other than in ways we've been very explicit about. That Canadian study was really quite uninformative. It was two pages that didn't say what things they found that went in the labeling later. Remember, priority drugs are drugs that are less familiar to us, so it wouldn't be surprising if something novel turned up in those cases. So I didn't find that very useful.

  • 13:57:46

    NNAMDIAnd this is obviously a conversation we'll have to continue at another time. But thank you, Dr. Robert Temple for joining us. Bob Temple is deputy director for clinical science at the U.S. Food and Drug Administration. Tom Moore, thank you for joining us. Thomas Moore is senior scientist in drug safety and policy at the Institute for safe medication practices. He's also project director for QuarterWatch, an independent drug safety publication that examines side effects data reported to the FDA. And Allan Coukell, thank you for joining us. Allan Coukell is director of the Pew Health Group's medical programs at the Pew Charitable Trust. Thank you all for listening. I'm Kojo Nnamdi.

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