FDA Drug Approval Comes Too Late for Many Patients For many patients dying of cancer, their last chance at survival may not yet be an option. Experimental drugs often take years to approve, and in the testing phase they are only available to a tiny group of patients. Do patients have a constitutional right to try experimental drugs?
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FDA Drug Approval Comes Too Late for Many Patients

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REBECCA ROBERTS, host:

This is TALK OF THE NATION. I'm Rebecca Roberts in Washington. Neal Conan is away.

Today, a Food and Drug Administration scientist said Avandia, the popular diabetes pill, should be pulled from the market because of heart risks. While drugs like Avandia approved for sale by the FDA have to go through a long process of clinical trials before they're sold, those that are still being developed and studied in clinical trials are more risky. People looking for alternate treatments to what's already on the market often turn to clinical trials for access to developmental drugs.

But what happens to the majority of patients who can't get into a clinical trial? And what if the person is terminally ill and desperate for treatment? Today, we discuss the right of patients to access unapproved drugs.

Later in the hour, we remember filmmaker Ingmar Bergman.

But first, should terminally ill patients have access to developmental drugs? We want to hear from you. Our number here in Washington is 800-989-8255. That's 800-989-TALK. Our e-mail address is talk@npr.org. And you can comment on our blog at npr.org/blogofthenation.

We begin with Frank Burroughs. He's president of the Abigail Alliance for Better Access to Developmental Drugs. He joins us from his office in Fredericksburg, Virginia. Welcome to TALK OF THE NATION.

Mr. FRANK BURROUGHS (President, Abigail Alliance for Better Access to Developmental Drugs): Thank you, Rebecca. It's nice to be here.

ROBERTS: Why did you start the Abigail Alliance?

Mr. BURROUGHS: It started because we couldn't get a cancer drug that - for my daughter, Abigail, that was in clinical trials. She didn't qualify for the strict protocol of the clinical trial.

ROBERTS: And ultimately, were you successful at getting access to the drug?

Mr. BURROUGHS: No, we weren't. Abigail died, and the drug was approved four and half years later for head and neck cancer that she had.

ROBERTS: And what would you have hoped to have been able to do for Abigail? What was your goal?

Mr. BURROUGHS: Well, I think what we're trying to do for Abigail was to get her this cancer drug that had showed early efficacy and no known serious safety issues in clinical trials. As we later found out, it could have extended her life, saved or extended her life. After Abigail died, I continued to work on to help tens of thousands, if not hundreds of thousands of other people by founding the Abigail Alliance.

ROBERTS: And what do you say to people who say these phase one clinical trials are just too risky?

Mr. BURROUGHS: There's a quote on our Web site, which in our Web site is abigail-alliance.org. It's very interesting. Every drug for cancer and other serious life-threatening illnesses that the Abigail Alliance has pushed for earlier access to in our six-year history is now approved by the FDA. There's not one drug we pushed for that was rejected by the FDA.

Currently, we're working with patients and have - actually, have been working with patients and are very disappointed about the FDA decision regarding Provenge for prostate cancer. And very saddened by the FDA decision, mostly Dr. Richard Pastor, regarding Genasense, a drug by Genta Corporation for melanoma and CLL leukemia. What's happening is drugs that show early efficacy and no known serious safety issues are being denied these patients who can't get into clinical trials. It's a tragedy.

ROBERTS: Frank Burroughs, thank you so much for joining us.

Mr. BURROUGHS: Okay. Thank you.

ROBERTS: Frank Burroughs is president of the Abigail Alliance for Better Access to Developmental Drugs. He's been talking to us from his office in Fredericksburg, Virginia.

Joining us now is Fran Miller. She's a professor of law at Boston University School of Law. And she's talking to us from member station WBUR in Boston, Massachusetts. Welcome.

Professor FRANCES MILLER (Law, Boston University School of Law): Thank you. Glad to be here.

ROBERTS: So the Abigail Alliance - we just heard from Mr. Burroughs - is suing the FDA. Tell me what's at contest in that case.

Prof. MILLER: Excuse me. The basic thing they're interested in establishing is a constitutional right of access to get experimental drugs. And…

ROBERTS: What constitutional right? Which part of the Constitution?

Prof. MILLER: It's constitutional due process right. It's one of those things that probably laypeople would find obscure and arcane. But what it really means is for the FDA to restrict access to an experimental drug - if the courts find - and as you know, the Abigail Alliance case has been reargued and decision is pending in the D.C. Circuit on this issue - if they find a constitutional right of access, the FDA has a much stricter standard of scrutiny to meet in order to keep people from getting it.

And it's really a question of how much evidence the FDA has to come up with that a particular drug in the clinical stage is dangerous, and how much they can basically interfere with the right of an individual to have access to it.

ROBERTS: So take us briefly through how this lawsuit progressed. The original three-judge panel sided with the Abigail Alliance. The FDA asked the full panel to hear it, 10 judges.

Prof. MILLER: Right. The district court dismissed it because prior precedent indicated there is no constitutional right of access to therapy. There's a constitutional right to refuse therapy, but not to get it. And the D.C. Circuit took a somewhat unexpected position in a two-to-three decision and found that there was a constitutional due process right. And the FDA petitioned for rehearing and - that was granted. The decision was vacated, and the case was reheard on March 1st, and everyone's watching very carefully to see what the full bank of the D.C. Circuit will have to say on this issue.

ROBERTS: Do you care to lay any odds?

(Soundbite of laughter)

Prof. MILLER: Yes. Yeah, I think they will reverse the prior D.C. Circuit holding on it.

ROBERTS: And rule in favor of the FDA?

Prof. MILLER: Yeah. I hate to put it that way.

ROBERTS: Well, it's hard to understand reversing which level, because it's gone back and forth so many times.

Prof. MILLER: Right. Well, when you raise something to a constitutional level, you are in a whole new ballgame. And people thought this issue had been laid to rest long ago with the Laetril cases and again, with the physician-assisted suicide cases, which were basically about the same kind of thing - getting access to drugs in order to terminate life. And the Supreme Court was quite definitive. In a pair of cases, they're saying no, there is no such right of access.

ROBERTS: Is this case likely to end up in the Supreme Court?

Prof. MILLER: Well, certainly. Both sides have a strong stake in the positions they hold. It all depends on whether the Supreme Court, if it is appealed, whether the Supreme Court decides that this is the time - that this is a right time to go forward and decide the issue.

ROBERTS: Let's hear from Charlotte(ph) in Charles City. Charlotte, welcome to TALK OF THE NATION.

CHARLOTTE (Caller): Hello.

ROBERTS: Hi, Charlotte.

CHARLOTTE: Hi. I just was very interested in this subject because I have a brother-in-law who is - who has soft tissue cancer. He's had it for over five years. The traditional treatments available - he has actually taken as many treatments at the maximum allowed for those. So he did get in an experimental treatment program - and I can't remember right now. I think it was Johnson & Johnson. And unfortunately, there were six out of 800 people taking this drug that had a particularly severe reaction.

It was almost - he was - almost died. And because of that, of course, they had to terminate that particular treatment. Now, he has tried getting treatment around us in Illinois and Minnesota - in the areas around us. And he's been rejected from all the experimental treatments now, either because of his age, or the amount of chemo he's had. There's one last chance that we have in Ohio - Cleveland, Ohio, that he may be able to get into.

If he doesn't, then because these other drugs are inaccessible to him unless he's into treatment - an experimental treatment, his treatments will be done. And at that point in time, they don't really expect him to last a long time. So we would love to see some of these drugs available even though you're not within those trials.

ROBERTS: Charlotte, thank you for your call. Thank you for sharing that story with us. Fran Miller, what sort of recourse does someone like Charlotte's brother have?

Prof. MILLER: Well, I like to just go back a little bit. She's talked about recourse - this is his only recourse to treatment. And I think that's something what - that is quite common, that people think of a drug that is in a clinical trial as being treatment. But the whole point of a clinical trial is to figure out whether something does or doesn't work. And so to call it treatment is really an oxymoron. It's - you're engaging in an experiment to see whether something works or doesn't work. And whether on balance the risks that attend taking it will justify whatever possible positive benefit can come out of it.

So it's a question that's a balancing question as to whether people fully understand. For example, the Abigail Alliance litigation said basically how that a mentally competent - the first, the D.C. Circuit opinion - how that a mentally competent, terminally ill adult patient with no alternative FDA-approved treatment options could claim a substantive due process right of access to possibly life-sustaining experimental medications?

Well, there are a lot of interesting slippery points in that holding. But to call it treatment is perhaps, you know, is very illusionary because when you look at the statistics about what kind of drugs, which are in phase one trials - phase one trials are the basic safety trials to determine the maximum safe doze of a drug before they go forward into any efficacy studies. If you've just talking about a drug that isn't a phase-one trial, 80 percent of products which are tested in phase one trials never go forward to market because they don't pan out on efficacy grounds.

ROBERTS: I want to repeat that point because it's worth repeating. Phase one is more or less to test the dosage level of safety for phase two. It's not to measure whether or not the drug is effective.

Prof. MILLER: That's right. You know, they will not ignore incidental efficacy data if there is any that comes forward. But usually, we're talking about testing on healthy patients just as to find what a maximum safe doze is. When you're talking about cancer therapy, you're usually not - doing that on healthy patients.

But 80 percent of products, which are tested in phase one trials, will never get an FDA. So that means 80 percent of products that the Abigail Alliance litigation says you should be able to have access to are going to - never going to pan out for you terms of efficacy or reasonable efficacy and safety. And they may indeed diminish both the quality and the quantity of remaining life.

ROBERTS: We're talking about experimental drugs and the world of clinical trials with Fran Miller, professor of law at Boston University. And we're taking your calls, 800-989-TALK. Or send us e-mail at - the address is talk@npr.org.

I'm Rebecca Roberts. It's TALK OF THE NATION from NPR News.

(Soundbite of music)

ROBERTS: This is TALK OF THE NATION. I'm Rebecca Roberts in Washington, filling in for Neal Conan.

Getting into a clinical trial can elicit many reactions. Some people mistrust unapproved drugs, while others who are terminally ill are desperate for alternate treatment. Who should get access to these drugs while they are still at the developmental stage? My guest is Fran Miller. She's a law professor at Boston University School of Law.

And of course, you're invited to join the conversation. Have you ever participated in a clinical trial or tried to get a family member some drugs that are not yet approved by the FDA? The number to call, 800-989-TALK. Our e-mail address is talk@npr.org. And check out our blog, npr.org/blogofthenation.

Fran Miller, I wanted to ask you quickly about the so-called compassionate use exception for drugs that are not yet approved. How does that work?

Prof. MILLER: The FDA has a procedure whereby you can - if you have physician who knows about a clinical trial and is willing to sponsor you for it - can petition the FDA for permission to get it, get the medication. You can't call it off label because it hasn't gotten a label - it isn't being marketed yet -but outside the clinical trial, and use it as - and basically collect data.

You won't be in a clinical trial. You won't be matched with controls the way a formal trial is handled. But it basically permits you to get it if you're willing - if you've got a physician who's willing to sponsor you and you can go forward with it. And by and large, the FDA has been pretty liberal in granting that.

ROBERTS: But it requires that the pharmaceutical company provide the medication?

Prof. MILLER: It certainly does. And I'd like to just make one comment about the FDA here. We sort of have a love-hate relationship with the FDA. There are people who call it very paternalistic with respect to its drug approvals. And, you know, in some cases, perhaps it is. But they also fault it for not being more strict with medications like, for example, Vioxx. And so, on the one hand, we're very much individuals when it comes to wanting something for ourselves, but on the other hand, when they don't pull something from the market - and certainly what went off with Vioxx is exhibit A with respect to this, and certainly what's happening today with respect to Avandia - there's certainly plenty of controversy over what the FDA advisory committee is going to do today in its decision.

ROBERTS: But isn't the difference in these cases that terminally ill patients have a lot less to lose?

Ms. MILLER: Certainly so. But do you know your terminal when you're taking Vioxx? I would not - I don't even mean to prejudge the causal effect with respect to Merck and Vioxx. But, you know, we're all terminal in one way or another and - but I do, I take your point. Certainly, if you're willing to assume risks as you are terminally ill, the real question is how much do you understand about those risks? How much you're really told about them?

ROBERTS: I want to bring Dr. Emil Freireich into this conversation. He's a distinguished professor at the University of Texas M.D. Anderson Cancer Center in Houston. He's joining us from his office there. Welcome.

Dr. EMIL FREIREICH (Director, Adult Leukemia Research Program, University of Texas M.D. Anderson Cancer Center): Hi.

ROBERTS: You have sided with the Abigail Alliance on this issue. Why do you think patients should have access to developmental drugs?

Dr. FREIREICH: Well, a patient who has a life-threatening illness is a person who is anxious to do whatever he can to make his remaining life pleasant. And therefore, there's no question in my mind that if a patient with life-threatening illness would like to receive an investigational drug and his physician wants to prescribe it, that he should have access to it.

ROBERTS: So you're position is…

Dr. FREIREICH: No doubt…

ROBERTS: …it's almost one of pity.

Dr. FREIREICH: Is almost what?

ROBERTS: One of pity, that…

Dr. FREIREICH: No. I'd - pity is not the right word. It's hope, I would say. When a patient is given a hopeless prognosis, they have several ways to get into a hopeful frame of mind. One is turn to some quack remedy, which is frequently done, you know, you go see someone in Mexico who has a magic potion. Or, you do something that is scientifically legitimate. You consult your physician and he consults literature, or you get on the Web and read everything that's known and try to participate in some activity, which gives you hope for a better outlook than you would otherwise have.

ROBERTS: So why not just give them placebo?

Dr. FREIREICH: Excuse me?

ROBERTS: So why not just give them placebo and tell them they're getting the drug?

Dr. FREIREICH: Well, because that's deceitful and normally, physicians don't deceive people or they wouldn't have faith in them. I think giving placebos is ridiculous.

ROBERTS: Well, I mean I'm being…

Dr. FREIREICH: Unconstitutional.

ROBERTS: Yeah. I'm not arguing that as concern. But if what you're saying is it helps people feel hope about their disease…

Dr. FREIREICH: Absolutely.

ROBERTS: …rather than necessarily cure their disease…

Dr. FREIREICH: Absolutely.

ROBERTS: …then the content of the pill isn't necessarily what's at issue.

Dr. FREIREICH: Of course it is, because they will get hope from real knowledge. You could - you mentioned the placebo, I mentioned quack drugs - you can get hope from those items. But I'm talking about real hope based on real science and real professional recommendations from a professional, normally a physician. If a physician says drug X is in clinical trial and there have been excellent responses, and there's a very good possibility that this will have an effect which cannot be accomplished with anything now available to you, I would recommend you do it.

ROBERTS: Let's hear from Betty(ph) in San Francisco. Betty, welcome to TALK OF THE NATION.

BETTY (Caller): Yeah. Thanks for taking my call.

ROBERTS: Sure.

BETTY: I don't disagree at all with what's been said. And the only, you know, the only comment that I would make is that are, sometimes, physicians really can't say that there's a very good hope. You know, many times, these patients are patients who've seen lots of chemo drugs and they really are at the end of the line. And as your speaker before pointed out, the vast majority of patients who go on phase one of each phase-due trials do not benefit from the drugs, that they aren't (unintelligible) because they're just not efficacious or because they don't get the right dose or because they have (unintelligible) disease or, you know, some other reason like that.

And I think that as I've, you know, gone along with my practice, a thing that I has come to realize is that my responsibility isn't so much getting people on trial, although I'm very pro-trial. I think that…

ROBERTS: I'm sorry, Betty. Are you an oncologist?

BETTY: Yes, I am.

ROBERTS: Okay.

BETTY: I think that what patients are often missing is an understanding of what the balance of the choices that they're making when they go on trial. I mean, you know, you - well, somebody said before, is when there's nothing to lose. Well that's actually not true, there's actually quite a bit to lose.

Dr. FREIREICH: That's true.

BETTY: A lot to be lost in terms of quality of life; useless drugs that has side effects; there's time, energy, and money.

Dr. FREIREICH: True.

BETTY: Time that could have been spent with loved ones. Getting, you know, things together, spending quality of - quality time, doing things that make people happy. Instead, they are running around from medical center to medical center doing tests that are required for the clinical trial.

Dr. FREIREICH: Sure.

BETTY: And also the, you know, price of hope. It is a gamble and it takes, you know, it's going to occupy your mental energy. You're going to put a lot of hope in that. It's not that hope is a bad thing, it's just that if all of your hope at the end of your life is focused on this one thing and not on something else, you know, that doesn't have to do with the treatment but on, you know, just your quality of life.

Dr. FREIREICH: Do I get (unintelligible)?

BETTY: I think that option is not - it doesn't end up being a very good bargain for a patient.

ROBERTS: Betty, thanks for your call.

Emil Freireich? Dr. Freireich?

Dr. FREIREICH: It's a sad thing that many medical oncologists talk the way this particular physician did. I'm talking about a decision that is made by an informed, intelligent physician, usually a physician scientist, who's recommending a choice, which is better than the available choices in his esteem.

As I've pointed out before many venues, the decision of what a patient is willing to do should be made by a patient and his physician, particularly a physician scientist. I think as this physician pointed out, when you go at medical center, you're dealing real experts in the treatment of the disease and those experts have knowledge about the drugs and their potential. And if they have a recommendation that a drug has potential for that patient and that patient doesn't qualify for the clinical trials for whatever reason, I think it's constitutional right for that person to be able to follow his physician's advice.

And remember, that you want to put the decision in the hands of a person best qualified. At the present time, the people who're deciding who should get this drugs are FDA officials, who are certainly not the best qualified.

ROBERTS: And you don't think that the compassionate use exemption is…

Dr. FREIREICH: Oh, it's rare. It's never used.

ROBERTS: Yeah.

Dr. FREIREICH: When you ask for compassion for the FDA - we had a session with the FDA at our hospital where they told us frankly. We never approved it. I mean, that mechanism is there, but it's never to the FDA official's advantage to approve a compassionate use. I'm quoting - I quoted an articled I wrote from Milton Friedman, that an FDA official has two very different kinds of mistakes. One, approve a drug that turns out they have unanticipated side effects, resulting in death or impairment; or refuse approval that is capable of saving many lives.

With visions of Prolinamide(ph) in their head, the knowledge of the fame, the claim that came to the woman who upheld the approval of Prolinamide is there any doubt which mistake you'll be more anxious to avoid. With the best will in the works, you'll be led to reject or postpone, and that's exactly what happens when the hands - when the decisions in the hands of the FDA, compassionate or other use - everything leaves the FDA official to postpone or reject. And that's what happens.

ROBERTS: Dr. Freireich, thanks so much for joining us. Dr. Emil Freireich is director of the Adult Leukemia Research Program at the University of Texas M.D. Anderson Cancer Center. He's been talking to us from M.D. Anderson Cancer Center Studios in Houston.

We have an e-mail from Stuart(ph) in Salt Lake City who says while I'm no expert on being a cancer survivor, I've been living with HIV for over 14 years. I remember well the fight we had to get experimental HIV medication on the fast-track for public release. We were successful in that fight and the results has been overwhelmingly positive. The argument against releasing those drugs was that we did not know the long-term side effects. I was one of a small group of people who did suffer from serious and unanticipated side effects. I was unable to work for three years as a result. In spite of that awful experience, I certainly think it was better than the alternative - death.

My guest is Fran Miller, professor of law at Boston University School of Law. And I'm also joined now by Dr. Ezekiel Emanuel, chair of the Department of Bioethics at the Clinical Center at the National Institutes of Health.

Welcome to the program.

Dr. EZEKIEL EMANUEL (Chair, Department of Bioethics, The Clinical Center, National Institutes of Health): Thank you for having me.

ROBERTS: And I should make it clear that you are not speaking on behalf of NIH in any way. You are here speaking from your own point of view. And what is that point of view? For these people that are terminally ill, why shouldn't they have a right to these medications?

Dr. EMANUEL: Well, first of all, I think we should separate out the issue of the constitutional right from what good public policy is. I think, as Fran Miller correctly pointed out, the Supreme Court has said in its end-of-life-care cases and its physician-assisted suicide cases that the liberty interest people have, the fundamental constitutional right, is not to be invaded, not to have their body invaded. That's a much different item than be able to demand medications or resources.

As a matter of fact, more than 25 years ago, the president's Commission for the Study of Ethical Problems in Medicine and Biomedical Behavioral Research made that point, that the ability to refuse is one thing, but even autonomy and self-determination does not give a patient rights to demand the use of resources, including drugs. So that's been a long-standing view. And I think as Professor Miller said, the D.C. Appellate Court's decision, which was two to one, was kind of what observers of the court would call the twistification(ph) of the judgment of the Supreme Court in the physician-assisted suicide cases, to try to get it to fit their own ideological position, and it didn't fit.

The second issue is should - once we say that there's no constitutional right, the question is - in any individual case - should a patient get access to drugs? And everyone has said the FDA and others, including myself, that patients should, under some circumstances, be able to get access to unapproved drugs. That's the compassionate use item.

And contrary to Dr. Freireich, who said that it's never used, that's simply untrue. As a matter of fact, probably the biggest case of compassionate use was with the drug Iressa, which was developed by AstraZeneca for lung cancer. And while there were some promising results, it actually made access available to 24,000 people worldwide. But then, it was shown not to have any survival benefit in lung cancer, even after many, many people got access to this drug. So it's simply not true that the FDA doesn't use this mechanism.

ROBERTS: You're listening to TALK OF THE NATION from NPR News.

Prof. MILLER: I'd like to echo what Dr. Emanuel just said. I'm wrecking my brain for the numbers. But I was at a conference, Food and Drug Law Institute Conference on the Abigail Alliance a few months ago. And the FDA official there - and I cannot remember the numbers, but if I remember correctly, they said about 75 percent of their, or to 80 percent of their petitions for compassionate use are granted. And I think, perhaps, Dr. Freireich was sort of conflating the basic approval decision of a drug with the compassionate use a separate issue.

Yes, the FDA does indeed often take its time with respect to being sure before granting an NDA, which basically lets a drug go to market. But if I remember correctly, it's more than 75 percent of their petitions for compassionate use are granted.

ROBERTS: And Dr. Emanuel, if a patient is terminal, understands the risk, is willing to assume them, and either the pharmaceutical company isn't offering the compassionate use or there's some other roadblock to that, why not allow them access?

Dr. EMANUEL: Look, there is a very important balance here. When you have a patient in front of you, terminally ill patient, your heart goes out to them, you're feeling very compassionate. You want to do everything you can for them. You'd be a brute, a robot if you didn't feel for them in their situation and their family's situation. On the other hand, we also have to recognize that there's important social policy here. We have had drugs - you mentioned Avandia. Vioxx has been mentioned. But before the FDA existed, we have lots of drugs that harm people and that hurt people, first of all.

Second of all, the most important thing for cancer patients is not access to experimental drugs. It's access to proven drugs that actually fight cancer. And the only we're going to know if they're proven is if we do clinical trials. And we have a lot of experience that access - early access to drugs delays clinical trial and delays our knowledge of whether drugs work or don't. In the cancer field, we have this information from the bone marrow transplant experience where lots of people thought bone marrow transplant was the cure to breast cancer - to metastatic breast cancer. They advocated that insurance companies cover it. They advocated state legislatures, often mandated that it be paid for. And the data are that about 25 to 40,000 women got bone marrow transplantation off protocol, so no data was collected from them. And we barely - we're able to enroll a thousand women in the trial that showed that it had no benefit at all, that in fact all those women were getting this extra high doze, some of them dying in the bone marrow transplant room for no benefit. And we've had that experience with AIDS drugs also.

ROBERTS: Coming up, more of your calls on unapproved drugs and we'll remember the director of "Fanny Alexander" and "The Seventh Seal." Filmmaker Ingmar Bergman died today at the age of 89.

I'm Rebecca Roberts. It's TALK OF THE NATION from NPR News.

(Soundbite of music)

ROBERTS: This is TALK OF THE NATION. I'm Rebecca Roberts in Washington.

Today, we're talking about access to experimental drugs. My guests are Fran Miller, professor of law at Boston University Law School and Dr. Ezekiel Emanuel, chair of the Department of Bioethics for the Clinical Center at the National Institutes of Health. And we're taking your calls at 800-989-TALK. Our email address is talk@npr.org.

And let's take a phone call. This is Hank in San Francisco. Hank, welcome to TALK OF THE NATION.

HANK (Caller): Hello. I'd like to share my experience. I'm a person with AIDS. I was the first person to get one of the drugs. It was a situation where I was - had exhausted the treatments. They weren't working. I had had bad side effects with the ones that were available. And I petitioned to get in - or I tried to get in into a clinical trial. I wasn't qualified, but I petitioned to still get access to the drug and I got it, and now, it's state-of-the-art medicine.

When I took that drug, we had a lot of risks. We didn't know exactly what was going to happen as it never been in humans before, but it was very important for me personally. I had only 26 T cells, I've been in the hospital with a bad brain infection for a week, my weight was just incredibly, incredibly low and it gave me hope. It also had a benefit for society. When we take these experimental drugs - even though we're not in a clinical trial, we get information about side effects. Those can be collected by the FDA. If the FDA was doing a better job in collecting side effect information, every one would be at a whole different level than we are right now.

So there's an individual benefit, there's also a societal benefit. In terms of lung cancer, which I also have and I just learned that I have that in the last few months, I'm on an Internet site with thousands of people with lung cancer. And it's very, very sad to see people that have exhausted all treatments that are currently available and they're aware of other treatments and they would gladly participate in those clinical trials and they can't get in them because they're filled and they're in a waiting pattern and that waiting pattern can be many, many months and sometimes years.

And to read those stories of about the people that would travel all the way across the country, they'd stay in trailers, they'd stay on the floor, in a sleeping bag and they're aware of drugs that are out there. And other people that are taking those drugs are sharing their information, and we know that those drugs are hopeful, they just haven't gone through the quote process, yet.

ROBERTS: Hank, thank you so much for your call.

Mr. MILLER: Actually, I think Hank's story about his AIDS treatment shows you that the system works, that when someone isn't eligible for a clinical trial and does petition, the FDA is responsive and you can get access. But I think we should also be clear that that Hank's drug turned out to be state-of-the-art and to be successful, is the minority of cases. We know…

ROBERTS: You wanted to clarify just how much of the minority by the way…

Mr. MILLER: Right…

ROBERTS: …your number was 95 percent of…

Dr. EMMANUEL: Well, if you look at the 100 drugs that start in human trials that stays one trials, again, to find out dosage, only five will get to FDA approval. Only out of 70 out of those 100 will even go on to test for efficacy, 30 failed because they're too toxic or for some other reason. And even of those that go on pass phase one, only one in twelve will get approval.

So most of the drugs, 11 of those drugs, are going to fail and only one's going to succeed, and we have to recognize that many of those patients who get those other drugs will not get any benefit and may get harm. And I might add, what you collect, the information you collect on compassionate use, is nowhere near as valuable as the information collected on a clinical trial. And…

ROBERTS: I want to just add something to what Dr. Emmanuel is saying there. The bottleneck here is really not the FDA. It's the manufacturers. They want patients in clinical trials so that they can get the safety and efficacy evidence that they need in order to go to market. They don't want patients who are - have co-morbidities or too sick or otherwise don't qualify for the trial, distorting their data on the safety and efficacy. They're the ones - and certainly, the Penelope case, which was widely read about after the Wall Street Journal front-page article on it - was about a manufacturer that didn't want to take the risk of having its clinical trials torpedoed by using out - and it's just tragic, tragic facts with respect to a little girl who was dying of, I believe, it was leukemia. But the problem…

ROBERTS: Well - but, even if the data from terminal patients who have access to these drugs for compassionate users or some other channel isn't as valuable as a, you know, random double-blind clinical trial, isn't all information helpful, to some degree, Hank's point about side effects? Even if it wasn't efficacy?

Prof. MILLER: Well that's certainly true.

Dr. EMANUEL: It can be valuable, usually not alone, but if collected with others. And, actually, again, this is one of the reasons manufacturers might be hesitant. Mostly, if you get side effects, it tends to rule against the drug, not in favor of the drug. Let me make another point about cancer clinical trials, which is an area I've worked on a lot. Mostly, it's not that clinical trials are full up and people are waiting to enroll in them. Mostly, we have clinical trials open and they're not enrolling patients.

The notion that Hank said that we have hundreds of thousands or tens of thousands of people willing to enroll in clinical trials just - is not the reality out there. About 40,000 to 60,000 cancer patients a year enroll in clinical trials. We have many clinical trials, which have been opened where -which aren't enrolling patients, where it's difficult to enroll patients. And so the idea here that patients are just waiting for clinical trials is, I think, not an accurate picture out there.

The second thing I would say and reemphasize a point I made before is that if you have these all alternative mechanisms, which are easily available and patients don't have to go into a clinical trial - we know from history from the bone marrow transplant cases and others that patients will not go into a clinical trial and go into this alternative mechanism, making it harder to enroll people in a clinical trial, harder to get the data to know whether a drug works or doesn't work, and harder to get proven stuff out to patients.

If we slow up the system, slowing up the enrolment in clinical trials, we're actually going to make it worse for cancer patients not better, because we won't know whether these drugs work. It will take many more years to learn their efficacy and their side effects and be it - then present the data to the FDA about the balance. So in point a fact, cancer patients - and again, contrary to what Dr. Freireich and the Abigail Alliance has suggested, most cancer advocacy groups, or many cancer advocacy groups are actually against wider constitutional right to investigational treatments. They like the process because we learn faster whether drugs work or don't work and we can move on when they don't work.

ROBERTS: Dr. Ezekiel Emanuel is the chair of the Department of Bioethics for the Clinical Center at the National Institutes of Health. He's been joining us here in Studio 3A. Thanks so much.

Dr. EMANUEL: Thank you.

ROBERTS: We're also joined by Fran Miller, professor of law at Boston University School of Law. She's been talking to us from member station WBUR in Boston, Massachusetts. Thank you, too.

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