Lawmakers in three states have passed so-called “Right-to-Try” laws, which give terminally ill patients access to experimental drugs without FDA approval. Diane and guests discuss the growing demand for these laws and whether they jeopardize patient safety.

Guests

  • Julie Rovner senior correspondent, Kaiser Health News; author of "Health Care Policy and Politics A-Z."
  • Lucy Caldwell communications director, The Goldwater Institute
  • Sascha Haverfield vice president, scientific and regulatory affairs, Pharmaceutical Research and Manufacturers of America (PhRMA)
  • Dr. Margaret Hamburg commissioner of the Food and Drug Administration.
  • Arthur Caplan director of the division of medical ethics, New York University Langone Medical Center.

Transcript

  • 11:06:53

    MS. DIANE REHMThanks for joining us. I'm Diane Rehm. So far, three states have passed so-called "Right-to-Try" laws giving terminally ill patients access to experimental drugs without federal approval. All these laws have passed unanimously. And Arizona has one on its November ballot. Joining me to talk about the emergence of "Right-to-Try" laws, what they mean for patient safety, drug companies and clinical trials, Sascha Haverfield of the Pharmaceutical Research and Manufacturers of America, Julie Rovner of Kaiser Health news, and Lucy Caldwell of The Goldwater Institute.

  • 11:07:36

    MS. DIANE REHMJoining us from the NPR Bureau in New York City, Arthur Caplan. He's with New York University's Langone Medical Center. I invite you to be part of the program. Give us a call at 800-433-8850. Send us an email to drshow@wamu.org. Follow us on Facebook or send us a tweet. And welcome to you all.

  • 11:08:05

    MS. JULIE ROVNERThank you, nice to be here.

  • 11:08:07

    MR. SASCHA HAVERFIELDGood to be here.

  • 11:08:07

    MS. LUCY CALDWELLThank you.

  • 11:08:08

    REHMGood to have you all. Julie Rovner, explain to us what Colorado did, what the law says and why.

  • 11:08:17

    ROVNERWell, basically this is an expansion of what the FDA already has, something called compassionate use, to allow people who are terminally ill to get easier access to drugs that have yet to be proved safe and effective by the FDA. There's already an ability for people to apply to the FDA to have access to these drugs. These are drugs that have passed what's called Phase I trials. So they've been proved basically that they don't kill you right on the spot. They've been tested in very few people, usually between 20 and 80. This is just to test toxicity and to get a general idea of dosing.

  • 11:08:52

    ROVNERIf they pass that test, they go on to Phase II and III trials to determine whether or not they actually work for what they're being tested for. So they become basically investigational new drugs. And these are people who basically have terminal illnesses, their doctors will vouch for that. It is kind of a cumbersome process to get this compassionate use, to get these drugs from the drug manufacturers if these terminally ill people don't qualify for clinical trials.

  • 11:09:18

    ROVNERSo basically what these states are doing is passing laws that would allow these patients to go directly to the drug companies to try to get these drugs. Drug companies don't have to provide them. And if they do, they don't have to pay for them -- they don't have to offer them for free. Their insurance companies don't have to cover them. In fact they, in almost every case won't, because they haven't been approved yet. So it's not entirely clear, you know that some...

  • 11:09:42

    REHMYeah.

  • 11:09:42

    ROVNER...many of these can be extremely expensive drugs. But the idea is to at least allow another way for these patients to get at these drugs. And as we will explore, there are a lot of concerns about it.

  • 11:09:52

    REHMWell, exactly. And, Julie, you say actually it's an expansion of what the FDA already does. But doesn't it take even a long time for the FDA to grant this kind of use to a non fully-approved drug?

  • 11:10:13

    ROVNERYes. And that's been the biggest concern...

  • 11:10:15

    REHMSure.

  • 11:10:15

    ROVNER...that getting a compassionate-use waiver can become very cumbersome. I mean they are normally approved...

  • 11:10:21

    REHMAnd take how long?

  • 11:10:22

    ROVNERIt can take weeks or months. And obviously if you have exhausted all of your treatment options, you know, you're clearly, you know, in most cases about to die. And people don't want to wait for these drugs, so...

  • 11:10:32

    REHMAnd there was really a big case recently where a gentleman was on the verge of dying and very, very much wanted this kind of compassionate use. And that was why the State of Colorado went for it?

  • 11:10:52

    ROVNERYes. There have been a lot of these cases. But in Colorado, in particular, there was a gentleman named Nick Auden who was very, very good, got a lot of publicity, had Stage IV melanoma, wanted a drug and was unable to get it. He actually did qualify initially for a clinical trial for the drug, and then was -- had a complication and was ruled out and was ultimately unable to get the drug. He passed away last November. But he in fact had an enormous publicity campaign. I think he had a change.org petition through the White House.

  • 11:11:26

    REHMHe was the father of young children.

  • 11:11:28

    ROVNERYes. Yes, a father of young children. Actually was Australian originally. But, you know, very telegenic, had a huge campaign going for this and did indeed bring a lot of publicity to the cause. But he's one of many. There have been several of these types of, you know, they're all very sympathetic patients. And it is, you know, indeed, you know, shines a light on the problems of people who are terminally ill and, you know, have exhausted traditional treatment and want to get other treatments. But as I'm sure we will get in to, it does raise other questions.

  • 11:11:58

    REHMArthur Caplan, what's wrong with these "Right-to-Try" laws, if indeed they could help people who otherwise can't get a hold of these drugs?

  • 11:12:12

    MR. ARTHUR CAPLANWell, I think they're well intended and I think we do want to show compassion to terminally ill people to give them a chance. Although it's a long shot and unfortunately research is tough in most Phase I drugs, those early stages that Julie was talking about. They don't make it to success. Probably one in 100 do. So really a long shot to get benefit. All that said, these laws basically go to war with the FDA. They say, we're going to override your authority and basically open the door to let people get access. Going to war with the FDA, to me, doesn't make sense.

  • 11:12:49

    MR. ARTHUR CAPLANWorking with the FDA to speed up that bureaucratic process that they have in place does. But it doesn't really get to the heart of the problem, Diane, which is, as Julie pointed out, these drug companies -- pharmaceutical companies, and I might add device companies too -- they don't have to give you anything. So even if the FDA says, yes, which it does 99.9 percent of the time, there's no money in these bills to help you pay for these drugs. There's no money in these bills to help you travel to get to where the drug might be administered or to get a babysitter or for any purpose whatsoever.

  • 11:13:26

    MR. ARTHUR CAPLANAnd the drug companies, if they use compassion and make the drug available, they're thinking, if something goes wrong and the person dies, they're going to hold it against my drug. And remember these are terminally ill people. So the odds of them getting into trouble are very, very, very high. You've got to solve those problems. Just saying, FDA, get out of my way, I don't think is the sufficient answer.

  • 11:13:48

    REHMAnd to you, Sascha Haverfield. Is Art Caplan, is Julie Rovner right that indeed the drug companies themselves can provide hindrance or be helpful?

  • 11:14:06

    HAVERFIELDThank you, Diane, for allowing me to be part of this very important discussion. We all share the same goal of bringing safe and effective new medicines to patients as quickly as possible. And I can understand the pain, the frustration, the desperation of a patient with a life-threatening disease or of their loved ones, who want to pursue every possible treatment option, including approved medicines, investigational drugs as part of a clinical trial or when there are no other options, expanded access -- so accessing an investigational medicine outside of a clinical trial that has not been proven safe and -- to be safe and effective.

  • 11:14:46

    HAVERFIELDI'm here as a scientist and as someone who's worked for well over a decade in drug development, drug discovery and regulatory policy. And I can tell you that, just as Art outlined, just as Julie outlined, the pharmaceutical industry is committed to actually helping patients and doctors understand and navigate the clinical trial process and the full range of potential treatment options that are available to patients, including expanded access where that is appropriate.

  • 11:15:14

    REHMWell, but you say where that is appropriate. If a patient in dire need comes to one of the pharmaceutical agencies or companies and begs for a drug that perhaps has gone through one trial, but not the entire process, what kind of a process does the company itself go through?

  • 11:15:45

    HAVERFIELDMm-hmm. Our member companies take expanded access requests extremely seriously. And every case is evaluated on...

  • 11:15:52

    REHMHow?

  • 11:15:52

    HAVERFIELD...a case-by-case basis. So in 2009, the FDA, with input from patients, from the biopharmaceutical industry, from lawmakers, from academia, makes you put a process in place to facilitate expanded access requests. And as part of that process, it begins with a discussion between the physician -- the patient's physician and the medical lead at the company to determine what is the best treatment option for the patient.

  • 11:16:20

    REHMCan you give me, say, a ballpark figure of how many of these requests the -- a drug company might grant within, say, a year's time?

  • 11:16:35

    HAVERFIELDUnfortunately I can't give you a number. But I can tell you that the FDA approved over 300 of those requests of these INDs last year.

  • 11:16:44

    REHMSo the request has to go through the FDA and then on to the drug company, or can a patient come directly to a drug company?

  • 11:16:57

    HAVERFIELDSo how the process works is, it starts between the patient's physician and the medical lead at the company to discuss the specifics of this patient, his medical condition, and then also evaluate the scientific and medical criteria of the investigational drug that the patient is seeking. Once there is agreement that this is the best course of action, that there are no clinical trials that the patient could enroll in or could participate in, there's no other treatment that may have been approved by the FDA previously, then an application is submitted to the FDA.

  • 11:17:32

    HAVERFIELDAnd the FDA then reviews and approves that application, which can be as quick as, in emergency use, overnight or by phone, or it can take up to 30 days.

  • 11:17:43

    REHMSo what happens in the kind of case that Art Caplan mentioned. What if somebody dies from taking a drug that's, say, one of the companies that belongs to PhRMA has provided? Who is legally responsible?

  • 11:18:07

    HAVERFIELDSo you're asking a very good question, Diane. The process of drug discovery and development of clinical trials is very, very complex. And you want to provide certainty to the process to drug developers to understand how data will be used, how safety data, how efficacy data will be used coming out of a clinical trial. The same applies to expanded access. When the FDA put this process in place in 2009, we are still looking for final guidance on how FDA will facilitate the process and use data.

  • 11:18:41

    REHMAll right. We'll have to take a short break here. When we come back, we'll continue our discussion. Stay with us.

  • 11:20:00

    REHMAnd welcome back. We're talking about a new law just recently passed in Colorado that gives terminally ill patients access to experimental drugs without FDA approval. They're called Right-to-Try laws and they passed in Missouri and Louisiana. One is on Arizona's November ballot. And joining us now from her office in Silver Spring, Maryland, Dr. Margaret Hamburg. She's commissioner of the U.S. Food and Drug Administration. Thanks for joining us, Dr. Hamburg.

  • 11:20:47

    DR. MARGARET HAMBURGThank you for having me. It's an important discussion.

  • 11:20:49

    REHMIndeed. Tell me what you think of Colorado's new Right-to-Try law.

  • 11:20:55

    HAMBURGWell, let me just say, you know, that at the FDA we are very, very concerned about patients that are suffering and their lives are at stake from devastating diseases for which they may not have an approved therapy as an option. And they have a very real need to turn to experimental treatment options. We have a long history of supporting access to experimental treatments for patients under those conditions. And we have a very good track record of approving or allowing requests for compassionate or expanded access to proceed.

  • 11:21:33

    HAMBURGSo we certainly understand why these concerns are out there and why patients and their families are seeking every avenue. But I actually don't believe that we are the barrier in these instances. And I think that's important to understand. In fact, in many cases I actually think that the FDA engagement, working with the families, working with the patient's physician and working with the companies have helped to make expanded access actually available.

  • 11:22:06

    REHMBut at the same time, with these states passing these laws, does that signal not only to you but to consumers all over the country that there's something wrong with the existing drug approval process?

  • 11:22:27

    HAMBURGI think it signals perhaps a misunderstanding of the existing process. And of course we want to identify any barriers to making important drugs available to people. Of course the goal is a safe and effective drug that is approved and available for all and reimbursable in our health care system. But in these instances where an individual does not qualify for a clinical trial, where there is no approved drug as a treatment for their disease and when they may need to turn to an experimental treatment option outside of the clinical trial mechanism, as I said, I do not believe that FDA is the barrier.

  • 11:23:09

    HAMBURGI was surprised in the earlier discussion to hear about how cumbersome the process is and how it takes weeks or months at the FDA. I'm looking at the form right now. It's three pages long. A lot of it is checking off boxes. As was mentioned by an earlier speaker, in numerous cases we have proceeded within 24 hours or overnight. We move these applications as expeditiously as possible. The ultimate decision however rests with the company. It is their choice to make the drug available. We cannot compel them to make the drug available. We can give our authorization to proceed.

  • 11:23:52

    REHMBut the question remains how you respond to the criticism that the approval process for access to experimental drugs is so long and mired in paperwork. I know there are people who are terminally ill who simply cannot wait for the approval process to play out.

  • 11:24:18

    HAMBURGWell, and that exactly is...

  • 11:24:20

    REHMThey're willing to take the higher risk and try anything. I mean, this 41-year-old father of three who simply could not get the drug he wanted. If the drug companies offer these experimental drugs outside the FDA approval process, how will your agency react?

  • 11:24:49

    HAMBURGDiane, I want to make clear that in the specific case you raise, the Nick Auden case and many, many other cases, FDA is actually not the impediment to access to the drug. In that case, the Auden case, we worked with the family and the physician for that family. In fact, Amy Auden sent a very nice thank you note to the FDA person who had been working on this case for their -- for the support of that individual and the FDA as an agency in gaining expanded access, in gaining access to the drug.

  • 11:25:26

    HAMBURGWe get, you know, close to a thousand requests a year. If -- for our fiscal year '13, which was October, 2012 to September 30, 2013, for example, we got 977. Of that 977 we allowed -- these are requests for expanded access -- we allowed 974 to proceed.

  • 11:25:52

    REHMSo you feel it's not the FDA that's the impediment. It is simply the companies and it is totally their responsibility?

  • 11:26:05

    HAMBURGYou know, I think it's a shared responsibility and I think we all need to work together to make sure that in individual cases access can be made available when it's a terminal illness, when there are no approved treatments that are available to that individual and when there is an experimental treatment option that might make a difference in their lives. Or it's the only last hope for those individuals and their families.

  • 11:26:34

    HAMBURGWe work very hard under the circumstances to try to make those products available. We, as I said -- and it was noted by another speaker earlier -- the FDA actually allows the application under those circumstances to proceed in the vast majority of the cases, 98 percent or so, 99 percent even. And we work with the companies to try to encourage them in many of these cases to make that drug available to these patients given their dire circumstances.

  • 11:27:11

    REHMSo...

  • 11:27:12

    HAMBURGSo what I'm saying is that I think I understand why these patients and their families are advocating for another look at the system. And I think we all should be open to looking to see if there are better ways to do things. But I think it is a mistake to believe that it's the FDA that is the barrier. I think the data does not prove that out. And I actually worry a bit that if FDA is actually not part of the process, sometimes patients and families will lose our advocacy with companies to make those products available.

  • 11:27:45

    REHMI see. So you've got three states so far that have passed these laws. More are pending. What difference will it make to the entire process if these laws do continue to expand across the country?

  • 11:28:09

    HAMBURGWell, I think that, you know, will remain to be seen. These laws are obviously very new. In one instance in Colorado I know, it's been signed into law and some of these still up on the ballot. But I think it reflects obviously a broader concern about access to new better drugs.

  • 11:28:31

    REHMSure.

  • 11:28:32

    HAMBURGAnd I think the FDA has a couple of critical roles. One is what we've just been talking about in these very devastating situations, when there are not options for patients to get access to an approved drug or to participate in a clinical trial, then we are very supportive of expanded access.

  • 11:28:53

    HAMBURGWe also are working very hard to try to advance new product development to find ways to move breakthroughs in science and technology more quickly through the development process to get important drugs to people more quickly, approved drugs, which of course is, you know, the most desirable endgame and is really what is best for patients and their families. So we also have seen, you know, pretty significant progress in that realm as well.

  • 11:29:27

    REHMWhat about the whole realm of clinical trials? For example, if I were very, very sick and agreed to participate in one of these trials, I wouldn't know whether I was getting a new drug or a placebo. And therefore, I would be, you know, feeling as though my chances were not very good of being on the frontline of trying to get a drug that would help me.

  • 11:30:09

    HAMBURGWell, at the end of the day we need, you know, good strong science to really demonstrate whether or not a drug works and whether it is safe and whether the benefits outweigh the risks in terms of the needs of patients and the treatment of their disease. So our goal is to be able to undertake the kinds of scientific studies that allow those important questions to be asked and answered.

  • 11:30:36

    HAMBURGThat does not mean that you always have clinical trials though where you have a treatment arm and a placebo arm and put patients and their families in that kind of a dilemma of not knowing if they'll get the drug or not. That does happen in some instances but we also approve drugs where every single patient is getting the treatment drug in question.

  • 11:31:03

    HAMBURGWe also make sure that with clinical studies that as soon as there is a demonstrated treatment benefit, that the patients on placebo have access to that drug and are converted over so that, you know, you identify what is a real concern. But it also is one that we try as best as possible to address, obviously working with the scientific research community, both in academia and in industry who are doing these important studies. And the goal is as quickly as possible to be able to get the scientific data to support whether a drug actually works, whether it does have benefits that outweigh the potential risks and make sure that the patients who need those drugs have access.

  • 11:31:54

    REHMAll right. And finally, Dr. Hamburg, let's go back to the 41-year-old father of three Nick Auden who tried to get the so-called anti-PD1 drug from Merck which he hoped was going to prevent him from dying of cancer. Two drug companies, Merck and Bristol-Myers Squibb, make versions but both decline to provide it to him outside of a clinical trial. Now, did the FDA attempt to intervene in that situation?

  • 11:32:36

    HAMBURGOh, we did recommend proceeding with expanded access in that case. And we were in discussions with the wife of the patient, the physician and the companies (unintelligible) ...

  • 11:32:52

    REHMAnd the companies said...

  • 11:32:57

    HAMBURGThe company -- you know, I can't speak to all of the specifics but the company, Bristol-Myers Squibb, I believe, turned down the family's request based on safety concerns.

  • 11:33:11

    REHMAnd that is the question, isn't it? If a drug is provided by a company and the drug has not been fully tested and the patient dies, how do you, Dr. Hamburg, see the liability there? Is it the patient's or is it the company's?

  • 11:33:37

    HAMBURGWell, unfortunately in all clinical studies there are potential risks involved. And there can be complications including death. And, you know, this, you know, is part of that spectrum of concern. With respect to how we the FDA consider adverse events reported under expanded access, which is a concern that does come up sometimes from companies, whether it will harm their overall application and their overall research activity around this drug, obviously we recognize that there are many complexities in these patients that are requesting expanded access at the very end of their lives when they have a lot of medical complications and a long medical history, comorbidities, etcetera.

  • 11:34:34

    HAMBURGAnd, you know, any problems that would occur, safety concerns, even, you know, fatalities would be considered in that broader context. And we certainly don't have any examples of a drug that wasn't approved because it was given under expanded access as far of the overall research effort around that product's development and review. So I think that, you know, the specific question of complications that might occur with the drugs used in the expanded access context may be overblown.

  • 11:35:15

    REHMDr. Margaret Hamburg. She is commissioner of the U.S. Food and Drug Administration. Thank you so much for joining us.

  • 11:35:26

    HAMBURGWell, thank you.

  • 11:35:27

    REHMAnd you're listening to "The Diane Rehm Show." Now I do want to bring in our other guest, Lucy Caldwell. And Lucy is with the Goldwater Institute. That is a Phoenix, Ariz.-based public policy advocacy and research organization. Lucy Caldwell, the FDA does have this compassionate use exemption. What did you hear from Dr. Hamburg that you felt makes sense as far as compassionate use and expansion thereof?

  • 11:36:16

    CALDWELLWell, Diane, first of all, thank you so much for having us on. The Goldwater Institute designed the Right-to-Try initiative because we feel a real need out there. And that's why we've been working with lawmakers all over the country, including in Colorado, Michigan, Louisiana, Missouri and Arizona and many more to bring this reform to their states.

  • 11:36:35

    CALDWELLI must say that I think that we do have a problem in this country. Dr. Caplan said that Right-to-Try is an initiative where we're going to go to war with the FDA. That is not what this is at all. We believe though that the current expanded use program does not adequately serve patients. And I think that some of the numbers are a little bit misleading.

  • 11:37:03

    CALDWELLThe first misleading number would be that the FDA has a 99 percent approval rating of this -- of expanded access applications. That is true. It's a very high application rate but in reality it is incredibly cumbersome to go through this application. It looks like three pages at first glance, in reality...

  • 11:37:25

    REHMAll right. Lucy, we're going to have to take a short break here. We'll continue with your comments when we come back.

  • 11:39:58

    REHMAnd welcome back. We're talking about right-to-try laws passed in Colorado and other states that allow terminally ill patients access to experimental drugs without FDA approval. And just before the break, Lucy Caldwell, you were talking about your efforts in this regard, to get states to pass these laws. But the question remains, how much good can these laws do if companies are reluctant or indeed refuse to make these drugs available? From what Dr. Hamburg, the FDA commissioner said, it's not the FDA that's standing in the way, it's the drug companies themselves.

  • 11:40:59

    CALDWELLWell, there are two issues in that regard. One is whether or not that's really the case of expanded access, that it's simply the drug companies refusing to give patients access. And on that point, I have spoken -- we have met so many folks from the drug industry along the way who have expressed to us that they're very fearful about doing something that might upset the FDA. But I was really gratified to hear Dr. Hamburg's comments, because it sounds like she's really in favor of companies extending these drugs to patients when it's appropriate. And that is -- makes me very hopeful.

  • 11:41:40

    CALDWELLWe have worked with several drug companies who are already making plans to go to Colorado, the first state where this has passed just a week ago and is now in effect, to perform experimental treatments or provide drugs. So this law just passed, it really shifts the paradigm. But I believe that we will see some results very shortly. But more broadly speaking, the process itself of approving a drug in this country, we have, you know, a million people a year dying of terminal illness. And a clinical trial process that takes over a decade and a billion dollars to get these drugs to market.

  • 11:42:18

    CALDWELLSo I think more generally, we need to think about how we can reform this process so that we can get these drugs to people faster. And so I'm glad that we're having this discussion (unintelligible).

  • 11:42:28

    REHMAll right. Julie Rovner, I gather that FDA process has changed over the years. It's gotten a little bit more streamlined.

  • 11:42:39

    ROVNEROh, it's gotten considerably more streamlined. And I think, you know, one of the things you can certainly thank, you know, AIDS activists for was bringing some of this to the FDA. And, you know, there's been a lot of criticism about the "Dallas Buyer's Club."

  • 11:42:52

    REHMYes.

  • 11:42:52

    ROVNERAnd some of the things that were not true about that movie, but one of the things that it did, you know, remind people of is the sort of new realization that AIDS brought to the FDA. And that was reflected in the '80s and particularly in the '90s and particularly in things like the 1997 FDA Modernization Act. And I think the FDA -- I, you know, I've covered the FDA under Republican administrations and under Democratic administrations and there's always been this tension between speeding things along and making sure that drugs and devices are safe.

  • 11:43:25

    ROVNERAnd I think that's been, you know, attention that has been -- FDA has always wanted to make sure that drugs and devices that get out on the market are safe.

  • 11:43:33

    REHMAbsolutely.

  • 11:43:33

    ROVNERAnd, you know, there are, you know, there's this concern about these drugs. I've seen a lot of the coverage of these right-to-try laws that, you know, after a Stage 1 trial the drugs are safe. And, you know, a Stage 1 trial is -- these drugs are tested on fewer than 100 people. We've seen drugs that have come to market and been pulled off the market after they've been approved and tested on thousands of people, but not a large population. So it takes a long time to determine that a drug is not only effective, but safe.

  • 11:44:00

    ROVNERAnd this is the constant tension that the FDA has. And Republican and Democratic FDAs really want to make sure that they are not putting drugs out on the market that turn out not to be safe.

  • 11:44:10

    REHMAnd, Art Caplan, that memory of the Thalidomide is still very fresh in our memories.

  • 11:44:18

    CAPLANAbsolutely. So is Vioxx, so are many, many other drugs that have had to have been withdrawn, many psychiatric medications, due to adverse events. But, Diane, I want to come back to this issue of what difference will these laws make. In the short run for people who might benefit, I don't think they're going to make a huge difference because I still think there needs to be money and resources put in here. I sometimes describe these laws as right-to-beg laws, not right-to-try.

  • 11:44:49

    CAPLANAnd the reason I say that is we could help doctors and patients and patient advocacy groups work with the FDA, put some money in there to educate people about how to find out about what might be out there in Phase 1 trials. A lot of doctors don't know. And, by the way, if you don't have healthcare access, which is why health reform plays a role here, you may not even have a doctor until you're almost dead. So money to pay to educate folks about how to work with the FDA.

  • 11:45:18

    CAPLANMoney to pay for the small company that can't afford to give something away. It may only have one drug and it can't really give it away without destroying itself. Some resources to say, "Look, we'll keep tabs on what happens, but we're not going to hold it against you, the company. If people die, who are terminally ill, on your drug we'll note that that happened, but you can still move your drug along through the process. Resources are what's needed here. And I have to say I'm not convinced that just letting people beg is going to really help those who are at death's door.

  • 11:45:53

    REHMAll right. And, Julie, you had a comment made by Mr. Audin's (sp?) wife, that I'd be interested in hearing.

  • 11:46:06

    ROVNERThat's right. You know, the discussion about, you know, who was at fault here, whether it was the FDA, holding it up and obviously Commissioner Hamburg said that they did try to work with the family to get this. And this is from an article that was in the Denver Post. And she said -- this was Amy Audin (sp?) who said, "We had no idea how difficult it would be to convince the drug companies to give it to him." So she's saying that the hold up in that case…

  • 11:46:28

    REHMAnd Merck is -- yeah.

  • 11:46:28

    ROVNERYes. You know, it was Merck and Bristol-Myers Squibb.

  • 11:46:31

    REHMAnd what about that, Sascha? How reluctant are drug companies to, in an extreme case like this, be willing to allow someone to have that drug?

  • 11:46:46

    HAVERFIELDDiane, our goal is to bring safe and effective new medicines to patients as quickly as possible.

  • 11:46:51

    REHMSafe and effective are clearly the keywords here, but these are experimental drugs and the Audins knew about those experiments and were still willing to take that risk.

  • 11:47:09

    HAVERFIELDSo clinical trials are the best way to show that a drug is safe and effective.

  • 11:47:14

    REHMBut he would not have known whether he was getting a drug or a placebo.

  • 11:47:20

    HAVERFIELDThat's a very good point that you make. And there's a common misperception that in an oncology clinical trial a patient would get a placebo and no treatment, when in fact, it's the best supportive treatment versus the investigational drug and comparison between the two. It would be unethical to give a patient in a life-threatening condition…

  • 11:47:40

    REHMNothing.

  • 11:47:41

    HAVERFIELD…nothing, a placebo, as you say.

  • 11:47:42

    REHMI see. I see.

  • 11:47:43

    HAVERFIELDSo clinical trials are the way to show and to bring safe and effective medicines to patients.

  • 11:47:48

    REHMBut if he didn't have the time to take for a clinical trial…

  • 11:47:57

    HAVERFIELDSo this is a very good example where it's important that the physician, the treating physician of the patient actually has that medical conversation with a medical lead at the bio-pharmaceutical company to determine are there other options, are there still clinical trials, and if not, is expanded access an option that should be pursued.

  • 11:48:17

    REHMAnd…

  • 11:48:18

    ROVNERActually, in his case, he had qualified for a clinical trial, but then he had a complication that had disqualified him.

  • 11:48:22

    REHMRight.

  • 11:48:23

    ROVNERSo he was willing to be in the clinical trial. And he had been accepted and then had a problem that kicked him out of the clinical trial.

  • 11:48:28

    REHMAll right. Let's open the phones now, 800-433-8850. First, to David in Jacksonville, Fla. Hi, you're on the air.

  • 11:48:44

    DAVIDHi. How are you doing, Diane?

  • 11:48:44

    REHMFine, thanks.

  • 11:48:46

    DAVIDI'm so glad to hear it. I just wanted to say, quickly, that I know this has been a long talk about the use of experimental drugs. And my cousin passed away a few years ago from cancer. And his wife was trying to find everything they could to give him. He had tried all the major pharmaceutical things like a lot of people do when they're going through cancer. And there's just a whole lot of natural things that can't go through because the major companies -- the way that a drug gets passes, is it takes tens of thousands of pages of reports, hundreds of millions of dollars, and many, many years to get through all of the stuff the FDA has.

  • 11:49:32

    DAVIDAnd only big companies, big pharma companies life Pfizer and GlaxoSmithKline and companies like that have the money to push through a drug. And the only reason they'd want to do it is for profits. And they can only profit something they can patent. And you can't…

  • 11:49:50

    REHMAll right. Do you want to comment, Sascha?

  • 11:49:53

    HAVERFIELDI can certainly understand the pain, the frustration, the desperation of a patient who faces a life-threatening disease and the need to explore every single treatment option that may be available, if that is an approved drug or an investigational drug. What we must not forget is that these investigational drugs that we are talking about here, have actually not been proven to be safe and effective. And may even cause harm to a patient, or pain. So the FDA plays a critical role in insuring the safety and efficacy of new medicines.

  • 11:50:26

    HAVERFIELDThat said, Diane, this is -- I would say it's a call to action to bring together patients, the providers, industry, academia, the FDA to work together to improve the clinical trial of the drug development process, make it speedier to bring new medicines to patients, and at the same time, optimize the expanded access process for patients.

  • 11:50:49

    REHMLucy, do you want to comment?

  • 11:50:52

    CALDWELLAbsolutely. I am very sorry to hear about your family member. And I do think that this speaks to -- I respect Sascha's position, but all the patients that we're talking about are patients who have exhausted all conventional options. They know what the risks are. And they're pursuing treatment plans under the supervision of a licensed doctor. And you're right, we don't know what all the side effects are, but we know the side effect of not taking an experimental medication and that's that you're going to die.

  • 11:51:24

    CALDWELLAnd so I think we need to be more respectful of patients here. I keep hearing, like, I understand the desperation, I hear -- understand the emotion. I've spent a lot of time with terminally ill patients and their families or people who have recently lost loved ones in the last few months. And they don't seem emotional or desperate or frantic at all to me. They're obviously going through the fight of their lives. But they're very well organized and they're very much in their right minds when they're making these decisions.

  • 11:51:53

    REHMHow many drug companies, Sascha, have set asides for people who are desperately ill, who are looking for any kind of solution?

  • 11:52:09

    HAVERFIELDSo our member companies take this process very seriously. And actually have processes in place to evaluate every single case. So every single case is based on what is the condition of the patient, what are the treatment options, is there a clinical trial? Basically, with the intent of identifying what is the best possible treatment option for a patient?

  • 11:52:31

    REHMAnd you're listening to "The Diane Rehm Show." Art Caplan, what needs to be done?

  • 11:52:40

    CAPLANWell, Diane, I think there are a couple of things that need to be done. First, I think we've got to put resources into the system so that doctors out there know what's in Phase 1 studies, what's in animal studies, what's early? We're presuming they do, and they don't always know. Patient advocacy groups need some money so that they can go through a standard template, a standard algorithm. Here's how to apply to the FDA. If one of your people, one of your terminally ill members needs access to a drug, let's educate them, put them resources there.

  • 11:53:14

    CAPLANLet's let the FDA say it will set aside bad outcomes for the terminally if there are deaths. We'll look at them, but we're not gonna hold it against any large or small company. Let's put some money in. We've had years of health reform debate here. Not one politician has said a word about compassionate use and paying for that. A lot of people can't pay. A lot of people can't afford it. Got to put a pot of money aside to allow them access for travel and to pay the cost of the drug.

  • 11:53:54

    CAPLANWe can work together to make compassionate access much more easy, much more reasonable to do, without harming the drug development process. But we have to put some resources there. And I think that's what's missing.

  • 11:53:57

    REHMLucy Caldwell, give me an example of an experimental drug that's out there right now, that could help terminally ill patients.

  • 11:54:11

    CALDWELLWell, I was just with a family last week. They have a 13-year-old son. His name's Diago. And a couple of years ago he fell in a soccer game and scraped his shin and he just kept complaining about it. And his parents believed that he was just complaining. But knowing that he was a tough kid they eventually took him to the doctor and he was quickly diagnosed with osteosarcoma. And it was a strain of osteosarcoma that was going to be very hard to treat. He had a surgery at St. Jude's, but when it came to finding a medicine that would work for him, his doctors told the family that he would have to go somewhere else outside the U.S.

  • 11:54:47

    CALDWELLThey quickly looked at the expanded use access program and discovered that it was going to take too long. They didn't have the time. They're lucky enough to have a lot of financial resources. They were able to move -- they first went to Mexico. Then they went to Israel. They went to Israel and then Italy and eventually England. He had the treatment and he is now in remission -- he's in recovery, excuse me.

  • 11:55:10

    CALDWELLBut that drug is still not approved in the U.S. And so, unfortunately, right now, our current system -- I totally respect what Dr. Caplan is saying about how we need to do even more. I'm in agreement. But, unfortunately, right now we have a system that really, really privileges people with the resources to pick up and move to other countries.

  • 11:55:31

    REHMDr. Caplan?

  • 11:55:33

    CAPLANWell, I agree with that, but I think we've got to get effective legislation through, not just saying, "Let's expedite or ignore FDA." Remember, there are bad guys out there. There are people willing to exploit the terminally ill and take, not only their lives, but their money. We don't want to have that situation out there, as well. So let's have a comprehensive look at this. Let's put resources behind letting the poor and the rich get access.

  • 11:55:59

    CAPLANLet's help the doctors and the families who don't know how to use social media, who aren't mediagenic. They, too, could use some assistance in trying to get compassionate use. There's room here for a much more comprehensive effort than I think the right-to-try bills exemplify.

  • 11:56:16

    REHMJulie, do you see that happening?

  • 11:56:20

    ROVNERI'm not sure whether there's a lot of stomach right now for anything much happening on Capitol Hill, on a federal level. I think Congress has got its hands full on the health side with the fights over the Affordable Care Act.

  • 11:56:34

    REHMSo it is simply the process. You don't see the state laws changing a great deal. You think that people will still have difficulty getting what they need.

  • 11:56:51

    ROVNERI don't know that these are going to make, as I think the panel has mentioned, an enormous amount of difference, if the companies themselves are not really compelled to do -- to change any of the behavior that they have now.

  • 11:57:02

    REHMJulie Rovner of Kaiser Health News, Sascha Haverfield of the Pharmaceutical Research and Manufacturers of America, Art Caplan, director of the Division of Medical Ethics at New York University Langone Medical Center, and Lucy Caldwell, she's director of communications at the Goldwater Institute. Thank you all so much.

  • 11:57:33

    ROVNERThank you.

  • 11:57:33

    HAVERFIELDThank you.

  • 11:57:34

    CAPLANThank you.

  • 11:57:34

    REHMAnd thanks for listening. I'm Diane Rehm.

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