Last year, Colorado became the first state to pass a law that gives terminally ill patients access to experimental drugs without Food & Drug Administration approval. Now, more than 20 states have passed similar, “Right-to-Try” laws. Supporters say they help patients who can’t wait for an F.D.A. approval process that can take more than 10 years. But critics say Right-to-Try laws put patients at risk and undermine clinical trials. And they say the F.D.A. already has a compassionate-use application process that works. Diane and guests discuss debate over Right-to-Try laws in the states.

Guests

  • Julie Rovner Senior correspondent, Kaiser Health News; author of "Health Care Policy and Politics A-Z"
  • Darcy Olsen President, the Goldwater Institute
  • Arthur Caplan Director of the division of medical ethics, New York University Langone Medical Center
  • Dr. Peter Lurie Associate commissioner for public health strategy and analysis, U.S. Food & Drug Administration (FDA)

Transcript

  • 11:06:53

    MS. DIANE REHMThanks for joining us. I'm Diane Rehm. More than 20 states have passed so-called Right To Try laws, which give terminally ill patients access to experimental drugs without federal approval. Supporters say they give dying patients access to cutting edge treatments that would otherwise take years for FDA approval. Critics argue these laws give patients false hope and endanger public health.

  • 11:07:23

    MS. DIANE REHMHere to discuss momentum behind Right To Try laws in the states, what they can and cannot do, Julie Rovner of Kaiser Health News. Joining us from a studio at member station KJZZ in Phoenix, Arizona, Darcy Olsen of the Goldwater Institute. She's author of a new book titled "The Right To Try." Joining us by phone from his office in New York City, Arthur Caplan of New York University's Langone Medical Center.

  • 11:08:01

    MS. DIANE REHMAnd, of course, you are welcome to join us, 800-433-8850. Send an email to drshow@wamu.org. Follow us on Facebook or send us a tweet. And thank you all for being with us.

  • 11:08:19

    MS. JULIE ROVNERThank you.

  • 11:08:21

    MS. DARCY OLSENGood morning.

  • 11:08:21

    DR. ARTHUR CAPLANThank you. Good morning.

  • 11:08:21

    REHMGood to see you all. And Julie Rovner, so last year, you had Colorado become the first state to pass this Right To Try law and now you've got 20-some states. What's behind the trend?

  • 11:08:39

    ROVNERWell, this is a textbook example of really good, effective lobbying, meeting what sounds like a perfectly logical idea. So we're now up to 24 states. It would've been 25, except that Governor Jerry Brown of California vetoed the bill that passed on California in October.

  • 11:08:59

    REHMWhy?

  • 11:08:59

    ROVNERHe said that the -- and I'm sure that we'll get into this, that the Food And Drug Administration is expediting their already expedited process for making these drugs available to people with no other way to get drugs who have terminal illness and he said that they should let the new FDA process work before they go to legislation.

  • 11:09:21

    REHMBut is that true? I mean, can these terminally ill patients get the drugs they need and want, even without FDA approval?

  • 11:09:33

    ROVNERWell, yes and no. And this is part of the problem. I mean, sort of the argument against the FDA is that they've had a compassionate use policy, which is what this is called, since -- really since AIDS. If anybody saw "The Dallas Buyers Club," that was sort of the fight that lead to the FDA making available, in certain cases, experimental drugs. It had not yet been approved for people with terminal illnesses.

  • 11:09:57

    ROVNERThat's been, you know, massaged and rewritten, but the problem was that it took an enormously long time and a lot of effort to put in the application. FDA could turn the application around, sometimes over the phone, if it was an emergency, but it took -- basically everybody agreed, about 100 hours for a doctor to put together that request.

  • 11:10:15

    REHMI see.

  • 11:10:15

    ROVNERIn February, the FDA announced that they're going to shorten that. They're going to have a new form and that they're gonna -- it will only take about 45 minutes. It'll be much easier for the doctor to ask the FDA for approval to get these drugs. The problem is, I think that form is not yet officially in use. The other problem is that companies don't -- that these are not really Right To Try laws. They're Right To Ask laws. They're a right to go straight to the drug company and ask for the drug.

  • 11:10:39

    ROVNERThe drug company doesn't have to give it to patients. Sometimes they don't. In fact, many times they don't.

  • 11:10:43

    REHMAnd Darcy Olsen, what about clinical trials? Why can't patients just enroll in those?

  • 11:10:53

    OLSENDiane, that's a great question and I'm glad that you asked that. Under the Right To Try, essentially what we're doing is extending to all patients with terminal illness the right to access essentially clinical trials. The current process is very restrictive so even though most patients -- at least, for instance, we know of cancer patients want to get into trials. They want these investigational treatments. They can't get in because the standards are too strict.

  • 11:11:20

    OLSENFor instance, it could be too far from your home, you may not be able to travel there. If you have arthritis, that may be a complicating condition. So all of these things keep you out of clinical trials. And we're basically saying people should have the right to those very same drugs that are being tested. Nothing wilder than that, but for people who can't manage to get into those trials because they are so restricted, let them have access in other ways.

  • 11:11:46

    REHMArt Caplan, from your point of view, what's wrong with these Right To Try laws?

  • 11:11:54

    CAPLANWell, I think they raise public awareness and that's good, but I don't think the focus on the FDA is the roadblock is correct. The companies really hold the key. If a little company or a big company wants to give you a drug, it's up to them to decide. You wouldn't even go to the FDA until the company had said yes or no. Wall Street drives a lot of what companies do because they fear adverse events. If you give a drug to someone who's very sick and they die -- and obviously, many of these drugs are not going to work out so people are going to die -- investors get nervous.

  • 11:12:31

    CAPLANWall Street gets nervous. Boards of directors get nervous. So they wind up saying, don't give it and it's not...

  • 11:12:37

    REHMSo help me understand. So if someone is terminally ill, goes directly to a company and gets the drug that has not yet been approved by the FDA, if that person then has adverse reactions, is that company libel? Art Caplan?

  • 11:13:02

    CAPLANSo the FDA keeps saying no, they'll understand that you could have problems and are likely to in very sick, dying people, businesses say they're still nervous. They still worry about it. It looks bad when word gets out that somebody died on your drug. That's a real roadblock to getting companies to say yes. Other big problem, Diane, with Right To Try as it is now is there's no money so people do need money sometimes to pay for those experimental drugs.

  • 11:13:35

    CAPLANInsurance companies will not. They need travel money. Sometimes they need a babysitter when they go far away to get a drug. This country has not budgeted anything for compassionate use and so that's as big obstacle as well.

  • 11:13:50

    REHMJulie, so a company that has made a drug that is still in experimentation is not willing to allow a dying patient to try that drug for free?

  • 11:14:08

    ROVNERUsually not. I mean, the FDA rule under compassionate use is that they can't charge more than their cost. But their costs, obviously, can vary a lot, particularly with some of these, you know, newer molecular biologic drugs. They can be, you know, tens of thousands or hundreds of thousands of dollars.

  • 11:14:26

    REHMPretty expensive, yeah. Yeah.

  • 11:14:28

    ROVNERThat's right. But it's also, you know, a matter of the company literally may, you know, may not have that much of the drug and all of the drug that it has is being used in the clinical trials that are going on.

  • 11:14:37

    REHMI see.

  • 11:14:37

    ROVNERFrequently, what happens is that people who are literally about to die can't get into clinical trials. They're not eligible because they're would skew the results in all likelihood.

  • 11:14:48

    REHMI see.

  • 11:14:48

    ROVNERSo compassionate use is a way, as Darcy said, to get patients those drugs in some circumstances, but as Art said, they may be worried about liability. Whether or not they should be, they may not have enough. They may not want someone to die on their drug and have it look bad, even though the FDA says they won't necessarily hold that against the drug when they go for the formal approval process.

  • 11:15:09

    REHMDarcy Olsen, you write about an example of someone these Right To Try laws are designed to help. Tell us about Jen McNary and her two boys.

  • 11:15:25

    OLSENSo Jen McNary, is a mother with four children and two of her boys have Duchenne's muscular dystrophy, which ultimately will cripple you and most children won't live past the age of 20. One of her boys she was able to get into a clinical trial and he got to the point in recovery where he actually was out on the soccer field playing with friends back on the playground.

  • 11:15:52

    OLSENThe other brother didn't qualify for the clinical trial...

  • 11:15:56

    REHMBecause?

  • 11:15:56

    OLSEN...because they said that he was already too far gone. They said he was too sick and so in the meantime, the second brother, he deteriorated to the point where he couldn't even hold a cup of water to his lips. So there is Jen, this mother, watching one of her boys recover while the other one isn't just deteriorating. He's dying. And so, you know, because he couldn't get into the clinical trial.

  • 11:16:23

    OLSENNow, I tell this story in great detail, all of the hurdles that they went through. It's been four years. The drug has still not been approved, even under accelerated approval. The second brother was finally able to access this medicine because the FDA relented and opened up an expanded special trial really to be able to squeeze him in, but there was so much publicity around this. And so this is great that Jen's boys are both being treated now, but, of course, you know, you have tens of thousands, even millions of patients with other terminal diseases who have not been able to access medicines that could be saving their lives right now.

  • 11:17:02

    REHMAll right. But now tell me about the second child. Did the drug indeed help him, even though it was said he was too far gone to be helped?

  • 11:17:17

    OLSENYes. In fact, it is helping him greatly. And Jen and I are friends on Facebook and a couple of weeks ago, she posted a video of him turning himself over in bed, getting into bed. He hadn't been able to do that from his wheelchair. It wasn't that he was too far gone to be helped by the medicine, Diane. It was that he was too far gone, according to the clinical trial...

  • 11:17:44

    REHMStandards, yeah.

  • 11:17:44

    OLSEN...standards, which were incredibly strict. They talk about wanting, basically, "athletes with tumors." In other words, they don't want anything complicating at all, which is why so many Americans cannot access clinical trials.

  • 11:17:57

    REHMDarcy Olsen, she's president of the Goldwater Institute, author of a new book titled "The Right To Try." Short break, right back.

  • 11:20:02

    REHMOur topic in this hour is the Right-to-Try laws, that is, giving experimental drugs to terminally ill patients. Joining us now from his office in Silver Spring, Md., Dr. Peter Lurie. He's associate commissioner for public health strategy and analysis at the Food and Drug Administration. Welcome, Dr. Lurie.

  • 11:20:34

    DR. PETER LURIEGood morning, Diane.

  • 11:20:35

    REHMI understand that there is an expedited new process for compassionate use. Can you tell us about that?

  • 11:20:47

    LURIESure. Well, you know, going back on the history of this a little bit, when FDA first put its expanded access or compassionate use process into place back in the late '80s, in the context of AIDS, was noticed. We needed to do it as quickly as possible and we used a form that FDA typically uses when drug companies are applying to administer a drug to any patient for the first time. And that form is estimated to take 100 hours to complete. That's the reference that you've heard. In fact, I don't think it ever took anybody 100 hours to complete that. And to think that any individual doctor would have spent two and a half weeks on a single patient seems to me unlikely.

  • 11:21:30

    LURIEBut nonetheless, I did take a look at the form and it seemed more complicated than it needed to be, primarily because it was serving another purpose. And so we slimmed it down such that it will take only 45 minutes to complete now. The number of attachments has been reduced from seven or eight down to one. Everything will be much simpler than it used to be.

  • 11:21:49

    REHMWhen you say it will be much simpler...

  • 11:21:53

    LURIEMm-hmm.

  • 11:21:54

    REHM...when is that going to be available?

  • 11:21:58

    LURIESo, you know, the way things work in government, as you know, is one has to go and propose a new form, which is what we did back in February. And then we have to wait for people to provide comments to us, which they did. And then we have to digest those comments and then put out a final version of the document. So we are in the process of doing that right now.

  • 11:22:16

    REHMAnd how much longer do you think that's going to take?

  • 11:22:20

    LURIEWell, you know, one can never be certain about these things. But we're hoping to get it done, you know, in the next several months.

  • 11:22:26

    REHMDr. Lurie, what about the process of drug approval in this country? Why does it take so long?

  • 11:22:38

    LURIEWell, you know, it takes time. But the statistic offered earlier in the program of about 14 to 15 years...

  • 11:22:44

    REHMRight.

  • 11:22:44

    LURIE...is really from the very beginning of the drug approval process, which includes animal testing, you know, laboratory testing of various kinds. And it takes a while before that even reaches the first patient. So it's not as if, after the patient -- first patient gets the drug, it's 14 to 15 years before the drug is typically approved. The fact is that the United States is now a leader when it comes to drug approval. Of all the drugs that are approved in the world, more than 50 percent of them are approved here first, which is remarkable for only a single country to half of the first approval. So, yes, we're interested in finding ways in which, if there's needless length to the process, we might be able to trim it back.

  • 11:23:27

    LURIEBut we do need to maintain standards as well. We also need to remember that the majority of drugs that enter early phases of human testing do not get approved by the FDA. They don't even make the second round and many don't make the third round of testing, before we even begin to consider the application. There is a failure rate to drug approvals that people don't appreciate. There is a danger to some of the drugs that are administered in early phases to patients that people don't fully appreciate. And FDA's role is to protect those patients early on, while still providing access to the patients who need them most. And we think that our expanded access program does just that.

  • 11:24:06

    REHMAll right. Well, let's talk about clinical trials for a moment. Why is it that terminally ill patients are so often excluded from these?

  • 11:24:20

    LURIEWell, you know, the drug companies get to make their choices of the way that they design their trials. And they make choices of who to include and who not to include. And in essence, it becomes their choice to do so. You know, it's not, from a clinical-trial point of view, helpful to enroll a lot of people who are very, very ill, because you'll never be able to show a benefit for those patients. And in the end, what we're interested in doing is, if the drug really is effective, we want trials that can show that the drug is effective.

  • 11:24:51

    LURIEIn fact, the very best way to get a drug to patients who are dying from terminal illnesses is not to have a scattershot approach where those who apply or mount social media campaigns obtain the drug through expanded access. The best way is to get the drug approved. And that's -- the integrity of that process is what FDA is trying to maintain.

  • 11:25:13

    REHMOn the other hand, a terminally ill patient could be dead by the time that drug is approved. So what choice to terminally ill patients have? But...

  • 11:25:29

    LURIEWell, that's where the expanded access program comes in. And, you know, we approve those applications often over the telephone, especially if it's an emergency, or typically within several days. Ninety-nine percent of people who apply to us for expanded access, because they have a serious or life-threatening condition, are approved. So the hang-up, as Dr. Caplan said, is emphatically not with the FDA.

  • 11:25:54

    REHMSo you believe the hang-up is with the drug companies?

  • 11:25:59

    LURIEWell, the drug companies, at least in certain cases, have chosen not to provide the drug for many of the reasons already described by others on the program. They also don't necessarily want to divert their attention from the clinical trial process, because they agree with us that the best way to get drugs to dying patients is to get effective drugs approved.

  • 11:26:21

    REHMAre you in favor of expanding the Right-to-Try rules across the country?

  • 11:26:31

    LURIEThe FDA has not and the administration has not taken any stand on those bills.

  • 11:26:35

    REHMAnd does not plan to?

  • 11:26:39

    LURIENot at this moment.

  • 11:26:39

    REHMAll right. Dr. Lurie, thank you so much for joining us. Dr. Peter Lurie is associate commissioner for public health strategy and analysis at the FDA. Thanks, again, Dr. Lurie.

  • 11:26:57

    LURIEThank you for having me.

  • 11:26:59

    REHMAnd Darcy Olsen, what's your reaction to Dr. Lurie's comments?

  • 11:27:05

    OLSENWell, there's very -- there's a lot that I agreed with when he was talking. I do think it's correct that the very best thing that we can do is get drugs to market sooner. And that is something that Congress can help expedite and we all need that. But in the meantime, I would disagree that the expanded access program is a success. In fact, in the book I talk about that -- we call it the mythical unicorn, because you hear about it but no one's actually ever seen it. He cited a, you know, that they approved 99 percent of all of these compassionate use requests. But what was not stated is the fact that less than 1 percent of Americans with terminal illnesses will ever get access to these drugs through that process.

  • 11:27:52

    OLSENThe reasons for that are many. Many of them don't know about the process. Sometimes it's the drug companies saying no. But sometimes it's the process itself is too long. And, in fact, I do tell the story about a young woman named Andrea Sloan in my book, for whom Texas named its Right-to-Try act, and she finally got approval from the FDA for her cancer. But it took them 24 days to turn around the particular drug that she needed in the application. And like her mother said, 24 days for a terminal patient is a lifetime. And I tell the story about how it came, it began to shrink the tumor, but she had become so sick during the wait that ultimately pneumonia claimed her life.

  • 11:28:38

    REHMHmm.

  • 11:28:39

    OLSENSo this idea that they turn it around in 24 hours, you know, they can show you examples of that. But we can show you patients who have lost their lives. The fact is, too many people are dying. And the momentum, you know, to have 24 states adopt this in short order tells you about the incredible demand, left and right, among Americans to have access to the most fundamental right to try to save their lives, not a year from now or five years from now, but right now.

  • 11:29:07

    REHMJulie Rovner, in actuality, what do these laws mean for patients?

  • 11:29:15

    ROVNERWell, I think, as I said before, they are less -- they do not confer a right to try the drugs. They confer a right to ask the drug companies directly, without going through the FDA first. That's effectively what they do. Now I might mention there is a federal bill that's been proposed. It would go considerably further than just that.

  • 11:29:34

    REHMHow much?

  • 11:29:35

    ROVNERIt would require the companies to designate a point person and to, you know, to publish what their policies are on this. It does not require the companies to provide the drug either, however.

  • 11:29:45

    REHMNow, Dr. Caplan, despite what you've cited as the shortcomings of these Right-to-Try laws, do you think that they will urge companies somehow to expand their offerings to terminally ill patients?

  • 11:30:08

    CAPLANWell, you know, Diane, we've seen some push in that direction, because not only is the Right-to-Try movement sort of sweeping through many states, but there are many social media campaigns. We all hear about them, people looking to gain access. They're up on Facebook, they're up on Twitter. They sometimes make it into the mainstream media. And I think companies are aware that pressure is out there to try and expand compassionate use. So I've been involved with Janssen, one of the Johnson & Johnson companies. And we've built a committee to try and answer a request so that it just doesn't fall to the company and they don't have to worry about the impact on their stock or whatever it is.

  • 11:30:55

    CAPLANAnd what we tried to do is make sure the websites work. As much as we've been yelling about FDA websites, not every company's website is easy to use and you can't necessarily get in there. So they need to be beefed up, more information to doctors. And let me yell about one other blockade. If you want to find out what clinical trials are out there, to get into them, there's something called ClinicalTrials.gov. It's not updated. It's not adequate.

  • 11:31:22

    REHMYeah.

  • 11:31:22

    CAPLANThat needs better support because that's the initial starting point.

  • 11:31:26

    REHMTell me what happens -- you say you're involved with Janssen -- so what is it that happens? Take me through the process when a patient, terminally ill, applies for a non-FDA approved drug from Janssen.

  • 11:31:47

    CAPLANSo in this case, the drug we're looking at is for multiple myeloma, a terrible, fatal disease. It causes a lot of suffering before death. And they have a drug that is in clinical trials but not approved yet, that is showing both pretty good safety and pretty good efficacy. If you want that drug, you and your doctor can go to the website and find out pretty quickly how to ask for it. We said, make sure the same information is there so that every doctor knows what to say, no -- not leaving it up to the individual doctor. And we anonymized it. You can't call up the company's CEO. You can't go on -- I mean you can go on social media, but we don't know who you are.

  • 11:32:35

    CAPLANSo there's a group of 10. They are blinded from the request, to make it fair. Three of them vote every week. This is drug, by the way, in very short supply. So you can't give it to everyone. And we promise to get back to you within one week, so that no one is left hanging, which is another big problem with compassionate use. So that's the system.

  • 11:32:57

    REHMAnd who pays for that drug, if it...

  • 11:33:01

    CAPLANJohnson & Johnson agreed to pay all the costs, shipping, getting it out there and everything. So that was great for them. But I think the key feature here, Diane, is a fair system. Everybody stands equal. You can't get preference because you're a senator. You can't get preference because you have a social media campaign. And we get the exact same information on everybody, so everybody gets considered equally. Those are key elements to having a fair distribution system when you just don't have enough drug. And remember, for a lot of compassionate use -- not all, but a lot -- there isn't enough drug. You may remember, Diane, Ebola. We didn't have enough medicines there to give out and hard choices had to be made.

  • 11:33:43

    REHMArthur Caplan, he's director of the division of medical ethics at New York University Langone Medical Center. And you're listening to "The Diane Rehm Show." I'm going to open the phones, 800-433-8850. First, to Dearborn, Mich. Julia, you're on the air.

  • 11:34:10

    JULIAHi. I just wanted to say my own experience in this realm. Whether a drug is approved or not approved for usage by the FDA, I think the most important thing to realize is anything that we ingest can be fatal. In my own faith, I tried a non-FDA approved drug recently which, at least for two months -- I should add that I am not having, at least that I know of, a completely fatal condition. However, it may develop into that at this point. The drug improved my life quality for two and a half months remarkably, to the point where I could function as a human being and live a normal life. I've used my own savings upon it and a friend took care of travel expenses and so forth. I was not able to get into a drug trial because of multiple illnesses, because of it.

  • 11:35:27

    JULIAAlso, in my own experience, I have watched people die -- as my own maternal uncle did -- from an FDA-approved drug for gout, allopurinol, where he was, within 10 dies, he died of aplastic anemia. There is no guarantee in life that we are going to live or die.

  • 11:35:50

    REHMIndeed. Julie.

  • 11:35:52

    ROVNERAnd thank you for your call. I just -- I want to add a little bit of context to this. This has been an argument going on and on for years with the FDA between, you know, speed versus safety. You know, how fast can they get the drugs out, but do they want to allow drugs on to the market that might be dangerous? How much danger is appropriate for what the drug is treating? You would allow, obviously, you know, more side effects would be acceptable for someone with a fatal disease than someone, you know, with the flu. It's -- so it's a balancing act.

  • 11:36:21

    ROVNERAnd Congress, you know, when first -- there were huge complaints about backlogs at the FDA in 1991. Congress instituted the user fee that the drug companies pay so the FDA could more staff so that they could get these things through faster. In fact, the FDA has now very much streamlined its overall drug approval process. Then there were, you know, then there was concern again. You know, we saw the whole Vioxx thing. It turns out that after some of these drugs that get sort of fast approval go to market and you try them rather than in 5,000 people, in, you know, several million people, then there are side effects you might not have known about.

  • 11:36:57

    ROVNERSo it's always a push and pull between getting drugs out quickly and trying to ensure, to the extent possible, the safety and efficacy of those drugs.

  • 11:37:06

    REHMJulie Rovner, she's senior correspondent for Kaiser Health News, author of the book titled, "Health Care Policy and Politics A-Z. Short break here. Your calls, you email, when we come back. Stay with us.

  • 11:40:01

    REHMAnd welcome back. We have an email here from Joan, in Ohio, who reminds us of the very reason this long approval process takes place. She says, "I too want the drug approval process sped up if it can be done safely. But keep in mind the U.S. process resulted in disapproval of thalidomide in the U.S., while many children in Europe were born with disabilities caused by thalidomide." And, Darcy, I know you wanted to jump in on the last call.

  • 11:40:55

    OLSENYes. And I appreciate Joan's email. And I think that that's attention that all Americans really share. I don't think that people want to rush drugs to market.

  • 11:41:05

    REHMRight.

  • 11:41:05

    OLSENAnd of course the right-to-try effort, really, it's about the terminally ill. It's about -- you have Lou Gehrig's disease for which there are no treatments and it's 100 percent fatal. There's an investigational medicine, but it won't be on the market for five years. And you'd like the chance to try to save your life. But I also would like to point out that in Europe compassionate use really is the rule, rather than the exception to the rule.

  • 11:41:29

    OLSENSo we can look there to deal with some of the questions about how do insurance companies kick in to help with the payments. And why do companies participate there when they're more reluctant to do so here? But we can't do that unless the FDA has an attitude of we can make improvements.

  • 11:41:48

    OLSENAnd when you hear things like the process is working quite well, or the FDA commissioner, last time she was on the show, saying this is just checking a few boxes, or in my book I interviewed the head of the Center for New Drug Development and asked the question, would you like to see Americans with terminal illnesses having access to these investigational treatments. And she sighed and said, well, it would be another burden on the health care system.

  • 11:42:17

    OLSENSo we do need the government regulators to have an attitude of hey, where can we change this system, how could we make it better, we know people are dying, instead of saying everything is great just as it is and we're just gonna shorten the form. So I recommend that we look to Europe where there's a lot more compassionate use.

  • 11:42:36

    REHMDr. Caplan?

  • 11:42:38

    CAPLANWell, Europe and the rest of the world are a mixed bag. Japan, Poland don't allow compassionate use at all. Some other countries seem willing to sort of let things go out there. But in general, I think it's up -- I'm gonna go back to where I started. You've gotta get the companies on board. If they're not playing ball, it doesn't matter what the FDA think, it doesn't matter what I think or Darcy thinks or anybody thinks.

  • 11:43:03

    CAPLANSo we need to think about incentives for those companies. Where's the money? If you're a little company with 20 employees and one CEO and somebody says how would you like to handle 100 compassionate use requests, you're probably diving under the desk saying, I can't get my product out. I'll never get it approved. Who's gonna help me? How do I build the website? What do I do?

  • 11:43:25

    CAPLANSo those are huge obstacles, especially when you're dealing with the small, innovative companies that tend to have the exciting, cutting-edge hopeful drugs, but they're often at a loss for resources. So I think you gotta look at this across the board, just pounding on the regulators, they're a piece of the puzzle, no doubt, but there's a lot of buttons that need to be pressed to get access here.

  • 11:43:50

    REHMAll right. To Noreen, in Arlington, Va. Hi there.

  • 11:43:56

    NOREENHi, Diane. Thanks for taking my call.

  • 11:43:58

    REHMSure.

  • 11:44:00

    NOREENI just wanted to say that my son is also in the same drug trial that Jen McNary's children are in. And I used to think the worst of the FDA until we actually went through the process ourselves. And really the worst thing for a parent of a dying child is to automatically be put in the hysterical class. And then you have social media and all these people desperate to get some drug for their child that they flood the FDA. My kid can do this now. My kid can do that, when there really is no data backing up what these parents say is happening to their children.

  • 11:44:41

    NOREENNow, my child has been on this drug for about a year. And I can't really go before the FDA 'cause my son is an outlier. He has muscular dystrophy, 100 percent fatal, but he's 16 and still holding his own completely. So I really don't know if the drug he's on is working for him, but I have sat through years and years of pretty much deluded parents thinking their kids are having some miracle when there is just no data.

  • 11:45:12

    NOREENAnd the FDA has to weed through this. And our drug -- there's another drug that just went through an ad-com with the FDA and the -- it's -- almost seems like the drug -- the other drug company has said, this drug is so good, yes, yes, yes, yes. And then the FDA came out and refuted everything.

  • 11:45:33

    REHMDarcy, do you want to comment?

  • 11:45:37

    OLSENWell, one of the reasons that we have the right to try and have designed this the way that it is is so that people who are selling things that are perhaps fictional or the snake-oil salesmen will not have a lot of luck. We're talking about expanding access to drugs that are in use in clinical trials right now. So the example of Jen with one son where she could see. I mean, he got up out of a wheelchair, she could see that. But the other one was declining. So it's really about giving all patients access to what's available right now only to a few people in clinical trials.

  • 11:46:13

    OLSENWe're trying to prevent people who feel like they need to go to Mexico or south of the border to try something that's not really being studied. And we're hoping to direct people toward things that have true promise for these fatal diseases.

  • 11:46:28

    REHMJulie?

  • 11:46:29

    ROVNERI think it's important to remember the placebo effect, which is a real thing. And this is why we have double-blind studies. And I should add that many of these drug studies are not double-blind. Everybody is getting the drug. They may be getting it in different doses, but there are still double-blind studies. And double-blind meaning the patient doesn't know if the patient's getting the drug and the doctor doesn't know if the patient's getting a drug.

  • 11:46:49

    ROVNERSo as if there is improvement or not improvement, that it is not because the patient, or in this case, the parent says, oh, my kid is getting the drug and look at home much better they're doing. It's so that the data can be trusted.

  • 11:47:00

    REHMJulie, how often do insurance companies step in?

  • 11:47:04

    ROVNERMy impression is not very often in these cases. Insurance companies are stepping in more in clinical trials. For a long time that was a big problem. Medicare, for example, didn't pay for patients who went into clinical trials. So there is more reimbursement for clinical trials. I'm not aware that there's much reimbursement for experimental drugs. There's certainly -- no insurance company would have to. I mean, you know, if every insurance company says you can, you know, they will only pay for treatments that are, you know, approved by the FDA.

  • 11:47:31

    REHMAnd Dr. Caplan, does a patient who is approved for a non-approved drug waive all rights to sue the company if something untoward happens?

  • 11:47:50

    CAPLANSo under right-to-try they're asked to do that. And I'm not sure that that's really gonna hold up. I'm not sure you can waive that right. It's sort of a fundamental right if somebody commits malpractice against you, gives you a drug they know to be dangerous but does it anyway, or something they made in the bathtub and gave to the cat.

  • 11:48:08

    OLSENJust -- and just to help clarify, too, Dr. Caplan, and Diane, it's really -- the waiver is the same as it is under clinical trials. So if someone is negligent, of course you can sue, but what you're really signing is an acknowledgement that the -- that no one is making you take this.

  • 11:48:25

    REHMRight.

  • 11:48:25

    OLSENAnd that it's still under process. So the clinical trials…

  • 11:48:27

    REHMSure.

  • 11:48:28

    OLSEN…really the standard for the waivers.

  • 11:48:29

    CAPLANAnd what I was saying is I still don't think you're gonna make companies sleep well at night by that kind of waiver. They still are very -- for a little company with a nervous board, boy, you look at those things and just say this is gonna get me into trouble. I am not going down this path. One other point, there isn't any insurance company in the world that has ever paid a nickel for compassionate use trials. They're not going there. That pot of money is empty. And if we don't do something to fill it, it's great to say we care and it's great to say we want to show compassion. But if there's no money for the families, they're gonna have a hard time accessing.

  • 11:49:06

    REHMAll right. Let's go to Kevin, in Vienna, Va. You're on the air.

  • 11:49:15

    KEVINThank you, Diane. Hey, I just recently read Dr. Atul Gawande's wonderful book, "Being Mortal." And…

  • 11:49:22

    REHMBeautiful, beautiful book.

  • 11:49:25

    KEVINIt really is. And it makes me wonder if this debate is so narrowly focused that we're just further distorting the role of pharmacology in our health care system. And where I'm going with this is it just strikes me that having this debate without also accounting for having a thoughtful and meaningful quality of life discussion with patients and their families at end of life -- that's an opportunity that's being missed at the same time that we're trying to focus on these experimental drugs that may in fact compromise their quality of life. Shouldn't there be some tie together, that we at least see an equivalent effort in having these discussions in support for these families and their patients?

  • 11:50:06

    REHMDr. Caplan?

  • 11:50:07

    CAPLANSo we definitely need to make sure that those discussions take place. Trying to stay alive is perfectly understandable, of course, but at the same time we all will die. The drugs that we're talking about, let's remind ourselves, rarely work. That's why one of the unfortunate facts about medical research is it's very hard. So the chances are overwhelming, even in the drug that we were looking at in our Johnson & Johnson Janssen project, it's not clear that people really can benefit from this drug. It's good, but none the less, many will die. So they need those discussions.

  • 11:50:44

    REHMSure.

  • 11:50:44

    CAPLANAnd one other sort of addition, we have many people who can't get approved drugs, approved treatments. We still haven't solved our insurance access problem. So while we want to give access to experimental things, there are still Americans out there who can't get proven things.

  • 11:51:01

    REHMDarcy?

  • 11:51:01

    OLSENYes. I, you know, I really appreciate Kevin's point. And I think what it comes down to, from my perspective, is who makes that decision at the end of the life. Is it the government making it or you the individual? And in our country we have adopted right-to-die laws. We've said in many states you have the right at the end of your life to try to hasten your death. What people are saying with these laws is you also have the right to try to save your life.

  • 11:51:27

    OLSENAnd there's a lot of chit chat about some, you know, how these drugs don't work or -- and etcetera. But I'd also like to draw your attention to the fact that about 30 percent of the most innovative life-saving cures are now coming out of Europe first. So I tell the story of a boy named Diego Morris who had osteosarcoma, bone cancer of the leg, and had to move with his family to London for a year to get this treatment that saved his life.

  • 11:51:50

    OLSENIt's been approved there for 20 years. It won Europe's, basically, Nobel Prize for advances in cancer treatments. You still can't get it here. So, you know, this is not just about things that have not been proven safe yet. Sometimes there are drugs that have been approved abroad, but are not accessible to Americans. And so we talk about the importance of adopting a reciprocity policy so that some of these life-saving drugs that have been approved and gone through that process overseas in some of these very strict countries could be available to our terminal patients here.

  • 11:52:24

    REHMWhat do you think, Julie?

  • 11:52:26

    ROVNERI suspect that's probably not going to happen. We had this argument about reimporting drugs, buying drugs to save money from companies where they have price controls. And I think that there is still in the U.S. the feeling that things -- that drugs sold, you know, given to patients in this country should go through the FDA process. Not that the, you know, European Medicines Agency isn't very good, but that there is, you know, there's a reason that the drugs go through processes, you know, their separate approval processes in the countries in which they're sold.

  • 11:52:55

    REHMDr. Caplan?

  • 11:52:57

    CAPLANWell, I think we have a situation where right-to-try tells us that people want others to have a shot at rescue. But still right now we're in a country that is basically allowing a right-to-beg. And we've gotta put resources in there if we're gonna shift that.

  • 11:53:11

    REHMAnd you're listening to "The Diane Rehm Show." Let's go to Laura, in Chapel Hill, N.C. You're on the air.

  • 11:53:25

    LAURAHi, there, Diane.

  • 11:53:28

    REHMHi.

  • 11:53:30

    LAURAI speak as someone who worked in the health care industry for 20 years. I'm now a doctoral candidate at UMC Chapel Hill, studying health communication (unintelligible) drug companies. I want to make that clear. But most of the people who are talking today seem to be assuming that these drugs that are still experimental are always and inevitably effective. The clinical trial process has several levels. First, just to make sure that the drug is safe for use in humans, that's a phase one trial. Actually, I think that may be phase two.

  • 11:54:00

    LAURABut a phase three trial, which is usually the last one, still says we don't know if this drug even works. The point that they randomize blinded controlled clinical trial is that we can rule out the placebo effect. If you have thousands of people who do not know whether or not they're getting a drug, and you know that whatever effect they had -- well, you don't -- you still don't really know, but you can at least draw some conclusion. You can conclude there's some evidence that the drug is responsible for their improvement.

  • 11:54:28

    LAURAAnecdotes are wonderful. I believe in the power of narrative, but telling a single story about a person who got better, you don't really know that the drug did that.

  • 11:54:38

    REHMDarcy, do…

  • 11:54:38

    LAURAYou only know that with empirical evidence.

  • 11:54:40

    REHMDarcy, do you want to comment?

  • 11:54:41

    OLSENWell, sure. I mean, I think that she's right. I don't think anybody on here is arguing that we should do away with clinical trials or the drug approval process. But it takes on average now about 15 years to bring a new drug to market from the beginning to the end. And when you have a fatal disease and you've exhausted all of your options and there is something that is a few years away from getting the FDA's green light, and the only thing standing between you and that medicine is that final approval, you should have the right to try to get that investigational medicine.

  • 11:55:16

    REHMWould you agree, Julie?

  • 11:55:19

    ROVNERWell, one thing I'm -- I'm not in a position to agree or disagree. But one thing I will say is that one, that this debate certainly seems to have made more people aware of this as a potential avenue. You know for years and years there were about 1,000 applications a year to the FDA for compassionate use. And as Dr. Lurie pointed out, the vast majority of them are approved. In 2014, there were almost 2,000. So it was almost double.

  • 11:55:43

    ROVNERIt's still a very, very small number, but as we're pointing out, these are, you know, people with very particular needs, you know, who have a terminal illness for whom there is -- who have nowhere else to turn and for whom there is a treatment that is in experimentation. So it's a…

  • 11:55:58

    REHMAnd as…

  • 11:55:59

    ROVNER…fairly small universe.

  • 11:56:00

    REHMBut as more and more people find out about these experimental drugs and as more and more people become ill with these exotic problems, aren't more and more people going to ask for these drugs?

  • 11:56:19

    ROVNERThey could, but it's still gonna be a fairly limited population. And I think one thing that Dr. Caplan said is that, you know, ClinicalTrials.gov is not -- does not list all the clinical trials that should be there. And that's another issue for another day.

  • 11:56:33

    REHMAnd at this point you do not see a federal law going into effect?

  • 11:56:39

    ROVNERThere is a federal law that has been proposed. I'm not sure. I would be surprised.

  • 11:56:45

    REHMAll right. Julie Rovner, she's with Kaiser Health News. Darcy Olsen's new book is titled, "The Right to Try." Arthur Caplan is with the New York University Langone Medical Center. Thank you all so much. Good to have you with us. Thanks for listening. I'm Diane Rehm.

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