Senate GOP leaders press ahead on a health care reform bill: What's in it, what's not, and will voters like it any better? Then, lessons learned from the Republican victory in a Georgia special election on Tuesday.
Guest Host: Frank Sesno
Earlier this month, the House of Representatives overwhelmingly passed the 21st Century Cures Act. Its authors say it will modernize the way medical treatments are developed and brought to market, offering hope to those suffering from diseases like cancer, Alzheimer’s and an array of rare illnesses. The bill includes nearly $9 billion for research at the National Institutes of Health, and attempts to streamline the approval process at the U.S. Food and Drug Administration. But critics warn we may be trading speed for safety. As the bill moves to the Senate, we discuss balancing patient safety and innovation.
- Rep. Diana DeGette Member, U.S. House of Delegates (D-Colo); a Chief Deputy Whip; co-sponsor of the 21st Century Cures Act
- Rep. Fred Upton Member, U.S. House of Delegates (R-Mich); Chair, House Energy and Commerce Committee; sponsor, 21st Century Cures Act
- Diana Zuckerman President of the National Center for Health Research
- Carolyn Johnson Business reporter for the Washington Post, focusing on health care
- Dr. Otis Brawley Chief medical officer, American Cancer Society and professor of hematology, oncology, medicine and epidemiology at Emory University
MR. FRANK SESNOThanks for joining us. I'm Frank Sesno of the George Washington University and Planet Forward sitting in for Diane Rehm today. She's on vacation, some well-deserved vacation. The House of Representatives celebrated a rare bipartisan victory earlier this month with the passage of the 21st Century Cures Act. Now, this bill aims to help those suffering from serious, sometimes incurable diseases by speeding up drug development and approval.
MR. FRANK SESNOBut some in the medical field say the proposed changes could result in unsafe and ineffective drugs reaching sick patients in the marketplace. Here to discuss the bill is Carolyn Johnson of The Washington Post, Dr. Otis Brawley of the American Cancer Society and Diana Zuckerman of The National Center for Health Research. Welcome to you all.
MS. CAROLYN JOHNSONThank you.
DR. OTIS BRAWLEYThank you.
DR. DIANA ZUCKERMANThank you.
SESNOAnd welcome, welcome. But before we get to the conversation in the room, I'm happy to tell you that we are joined by two members of Congress on the phone, Representative Fred Upton. He's a Republican from Michigan, chairman of the Energy and Commerce Committee and co-sponsor of the 21st Century Cures Act. And Representative Diana DeGette, she's a Democrat from Colorado, chief deputy Whip and co-sponsor of the 21st Century Cures Act. Congressional members, welcome to you both. Thank you.
REP. FRED UPTONYou bet.
REP. DIANA DEGETTEThank you.
SESNOCongressman Upton, let me start with you and then, Congresswoman DeGette, jump in, please. What's the problem here?
UPTONWell, we -- what we've done is expedite the approval of drugs and devices. The world has changed a lot and we're working very closely with the FDA, Food and Drug Administration, as well as the National Institutes of Health researchers really for the last year and a half. The two of us have hosted roundtables and subcommittee hearings all around the country to figure out what we can do to expedite the approval of drugs and devices which, quite frankly, we've seen a lot of those approvals go overseas. It's delayed getting cures for individuals here.
UPTONIn addition, we came up with a new $2 billion, in essence, $10 billion over five years innovation fund to help research so that we can find cures to things like diabetes and cancer, Alzheimer's, a whole host of things. So we worked very closely with the FDA to make sure that the safety standards are maintained and we had a very, very strong bipartisan vote when it passed a couple weeks ago, 344 votes on the House floor.
SESNODon't see that very often.
UPTONAnd we got -- no, and we passed it unanimously in the committee back in May, 51 to nothing.
SESNOSo Congresswoman, let me ask you the same question. What's the problem?
DEGETTEWell, here's the problem. With the mapping of the human genome, with electronic medical records, with the ability to find targeted cures, precision medicine, if you will, our agencies both had -- are lacking in resources and also sometimes they're lacking in the ability to coordinate and expedite review and approval of new drugs and devices. We still don't have a cure for Alzheimer's, Parkinson's, MS, so many diseases that affect millions of Americans.
DEGETTESo what we did, we worked with people all across -- we worked with research institutions and patient advocacy groups and perhaps most importantly, we worked with the NIH and the FDA themselves to figure out how we could do some restructuring so that we could encourage development of these new devices and new drugs.
SESNOSo explain that. What would the restructuring be, what would it look like? What would change?
DEGETTEWell, there's a couple of different -- I mean, it's a big bill. It's a deep dive based on a year and a half of effort. Let me give you a couple of examples. Modernizing clinical trials. Right now, if somebody wants to do a large, randomized clinical trial with many institutions, they have to go through each separate IRB review process and patient consent forms. This would allow a centralized process where one protocol is developed and it would greatly expedite research.
DEGETTEHere's another one. Digitalization of healthcare so that we can make sure that we can have interoperability so researchers can share genetic information about patients. We put more patient involvement in the approval process and many, many other things that we do in this bill to try to streamline and target biomedical research.
SESNOLet me go back to Congressman Upton for a moment. The bill would put, what, about $8.75 billion over the NIH over five years for this research with a novel funding mechanism that one doesn't normally think about when you think about medical research, selling off some of the countries strategic petroleum reserve. Really?
UPTONWell, that's right. That's within our committee's jurisdiction. And, you know, I'm an old Reagan guy, Frank, as you know and I believe in balanced budgets and this was a new increase in spending, mandatory spending, as we say inside the Beltway and so we said from the start that we were going to find a way to pay for it and we would find a way to pay for it within our own committee's jurisdiction. So as you look at this particular issue, SPR, the Strategic Petroleum Reserve, this is, you know, the reserves primarily down in Louisiana, the big salt domes that we've been socking away oil since the oil embargo back in the late '70s.
UPTONThe law says you have to have a 90-day supply. Well, by 2018, we're going to probably have about three times that in terms of supply so we're going to take a half a day use of the United States out. It's going to -- we had it scored by the congressional budget office and the numbers work so the increases that we have for health research are offset with other cuts or savings within our committee's jurisdiction.
SESNOAll right. Let me ask you both a briefly to comment on some concerns raised by a colleague of yours and then we will bring the conversation into the room with our experts here. Representative Rosa DeLauro of Connecticut said she voted against this bill because she believes it opens the door to what she called -- wider to what she called untested and potentially unsafe medical devices and drugs. And she gave a few examples.
SESNOShe said, for example, only 1 percent of medical devices are required to undergo clinical trials as it is, which has lead to the approval of devices like the power morcellator, she says, which can spread uterine cancer to other parts of the body. She notes a flawed drug approval system that's allowed what she referred to as a number of unsafe or ineffective antibiotics onto the market and she says the following, "these tragic stories should prompt us to strengthen our approval system. Unfortunately, the 21st Century Cures Bill, would do the opposite. I believe it would take us down a dangerous path for patient safety." Congresswoman, you want to go first?
DEGETTEYeah, sure. I have deep respect for Rosa, but we look at this in a very different way. We do not undermine any ability that the FDA has to review either drugs or devices to make sure that they're safe and to approve them. And, in fact, one thing that we do that we don't have under current law, we increase post-market review. So this morcellator that Congresswoman DeLauro talks about, this was approved by the FDA under current standards. But it got to the market, it proved to have problems. They don't have enough post-market review and we allow that in our bill.
DEGETTEWe worked very, very closely with the FDA to make sure that the provisions in our bill, for example, breakthrough therapies for devices. So if you have a device to treat something where we don't have a treatment right now, it can go a long the pathway of the breakthrough therapies on drugs that we have under current law, we put that in the bill, but we made sure that the FDA retains all of its ability, first of all, to even allow something to go through the breakthrough pathways, but also to get all of the evidence and post-market review.
SESNOOkay. Let me flip over to...
DEGETTEWith respect to the antibiotics -- let me just say on the antibiotics, you know, we have people in hospitals who are getting terrible, terrible viruses that cannot be treated right now. We don't have the antibiotics. So we put some very narrow language in the bill to allow development for some antibiotics that could treat that very narrow group of patients and there are strict controls that the FDA agrees with. So actually...
SESNOLet me turn to Congressman Upton. Go ahead.
UPTONWell, I agree with what Diana said. I mean, look, we wanted to take the time to listen for the last year and a half so actually one of the things that we did a few months ago was we took the former director of the Food and Drug Administration and he and his staff and working with other researchers literally went -- poured through our draft legislation page by page offering suggestions, which we took. We worked very closely with secretary of HHS, Sylvia Burwell in precision medicine.
UPTONYou might remember that President Obama talked about this in his State of the Union address, that we needed positive change. They were very helpful, very constructive. And we've laid the groundwork with a number of senators on both sides of the aisle, Republican and Democrat, they understand what we've done and, you know, the proof is in the pudding. 344 votes, I mean, as you said at the beginning, that's really rare.
SESNOWell, I want to...
UPTONParticularly when it's that bipartisan.
SESNOAt a minimum, we congratulate you for getting all those votes at a maximum. The conversation will continue. We're going to continue it here. Thanks to both Representative Fred Upton and Representative Diana DeGette for your time today.
DEGETTEThank you so much.
UPTONYou bet. Thank you.
SESNOReally appreciate it. As we turn to the room here for just a couple minutes, Dr. Brawley, let me start with you. What we've heard here is -- from both congressional members is they're feeling that there's a desperate need to modernize this process, a desperate need to reflect where the research is going and what technology allows and to catch up with all that. Correct prescription?
BRAWLEYAbsolutely correct. In my own field of medical oncology, the definition of cancer is changing.
SESNOWhat do you mean, the definition of cancer?
BRAWLEYWell, we have used a 19th century definition of cancer given to us by pathologists who did autopsies. The 21st century definition of cancer involves genomics. There was -- when I went to medical school 30 years ago, there was one breast cancer. Twenty-five years ago, there were two kinds of breast cancer. Today, there's 80 or more. You see, we can't do these large randomized perspective clinical trials for one drug against one disease because there are just too many diseases.
SESNOSo, from your perspective, the single most important thing that's in this act would be what?
BRAWLEYI recognizes the fact that we're in an era of precision medicine. It recognizes the fact that there is a genomic definition of many of these disease and these disease treatments are going to get personalized.
SESNOWe are talking about the 21st Century Cures Act and the new era of medical research and treatment and this bill that could speed to market these new and sometimes revolutionary drugs and devices. Be right back.
SESNOAnd welcome back to "The Diane Rehm Show." I'm Frank Sesno sitting in for Diane today. We're discussing this hour the 21st Century Cures Act, which is a bill that's gone through the Senate -- or the House, rather, that is meant to accelerate the trials and not the tribulations of some of the really earth-shattering medications and devices that can come to market and help people who are severely ill.
SESNOWe're talking with Carolyn Johnson, she's business reporter at The Washington Post with a focus on health care, Dr. Otis Brawley, chief medical officer of the American Cancer Society and a professor of hematology, oncology and epidemiology at Emory University, and Diana Zuckerman, president of the National Center for Health Research, which is a non-partisan think tank focused on issues of public health. So, Carolyn, I'd like to turn to you, as someone who's been covering this topic among so many others. As we think about the money that this act would carve, the additional research money, billions for National Institutes of Health and others, what might this do for advancing the drug development at issue here?
JOHNSONWell, that's the part of the bill that's basically -- you'd have to search far and wide to find someone who really opposes giving more money to the National Institutes of Health. This is the engine of biomedical research in the United States and the world, really. And it's been -- had a flat budget for over a decade. So people really believe -- people who are critics of the bill believe that giving more money to the National Institutes of Health is going to help make the basic, kind of, research insights into cancer, Alzheimer's disease, you name it, that's going to eventually result in new drugs and new innovations.
SESNODr. Brawley, what would $8.75 billion over five years mean?
BRAWLEYWell, to give you an example, the current budget for the NIH is about $30 billion. So we're going to increase the budget by about, oh, 5 percent or so per year for five years. I think that's really important given that the NIH budget has been flat for the last 10 to 12 years. And given inflation, the NIH budget has actually gone down over the last decade.
SESNOWhere will that money get spent?
BRAWLEYIt's going to go to -- there are 27 or 28 institutes -- it's going to a number of different diseases within NIH. And you can think of all the major diseases being positively affected, be it cancer, AIDS, lung disease, heart disease, especially many pediatric development -- developmental diseases.
SESNOIf you'd like to join our conversation, please do so with a question or comment at 1-800-433-8850. Or you can drop us an email -- we have several of those already -- at email@example.com. Diana, let's move to the FDA part of this bill, which is, I think, where you have some concerns. The idea here is to speed up the process. You don't like the sound of that?
ZUCKERMANI certainly agree with Otis Brawley, who I respect very much, that we have to look at certain kinds of research differently -- both the research done at NIH and the research done at FDA. But it's a different matter when you start looking at studies that are based on, what the bill says, case histories. Case histories means one person -- a lengthy description of one or two patients. That's not the way to really learn about what drugs are most effective and what drugs are safer. It also talks about looking at what are called pre-clinical studies. I don't think most congressional staffers know, that means things like rat studies. So what the bill has, you know, the devil's in the details here. It's a 350-page bill, more than that even.
SESNOHave you read it all?
ZUCKERMANAnd I have read almost all of it, which I think is not true for most people -- most sane people. But it's a very long bill. And so there's a lot of good things in it. And then there are these parts that say, "We can lower the standards so that we no longer have to do scientific studies of diseases."
SESNOYeah, there can be anecdotal evidences.
ZUCKERMANThey can be absolutely anecdotal. And I should say, my training is in epidemiology. I know there are a lot of different kinds of studies, not just clinical trials. But they still should be scientific.
SESNOSo are you worried about this thing?
ZUCKERMANI'm very worried about it. And I can give a couple of examples. One, in cancer, where even under current law and FDA's current regulations, sometimes drugs like Avastin for breast cancer, drugs were studied preliminarily. They looked very promising. And then they were later found that the patients not only didn't live any longer but they lived a little shorter and had a worse quality of life.
SESNOCarolyn Johnson, you wrote a piece the other day. "This bill promises to speed up drug approvals so much that it's making people uncomfortable," is the headline.
JOHNSONYes. Well, people like Diana and people -- really respected people in the medical community have legitimate concerns about this bill. It has evolved many times.
SESNOWhat are their other concerns?
JOHNSONWell, mostly that with -- although it does offer the more -- the increase in NIH funding -- that it does loosen the approval process for drugs and devices, basically, the issues that Diana was just speaking about.
SESNODr. Brawley, are you worried about this?
BRAWLEYNo, I'm not. And I'm actually a medical oncologist and epidemiologist who's done a lot of drug development. I actually think that the rules do need to change and do need to reflect the new science. I think this bill does not lower any standards in terms of efficacy. I think this bill creates a new pathway towards those same standards.
SESNOSo, Diana Zuckerman, let me ask -- invite you to talk to Dr. Brawley here and tell him what he should worry about.
ZUCKERMANOkay, sure. Well, first of all, it's true that there -- that the bill doesn't require FDA to lower the standards. But as somebody who's worked on FDA issues and I've been to over 100 FDA Advisory Committee meetings -- you see how the pressure that comes from Congress on the FDA to change their standards, and in this case lower their standards, has a very large impact, even when it doesn't require it. So that, perhaps, today's leaders of the FDA would not lower the standards.
SESNODr. Brawley, what's your response to that?
BRAWLEYYeah, well, I am -- I'm all in favor of experts creating the standards. And we should leave it to the medical profession to create the standards. I think Dr. Zuckerman and I agree on that. I actually think that this bill allows for FDA administration and FDA bureaucrats to set the standards, but do it in a very open way, where the public can see and have input and be able to criticize if they do end up lowering standards.
SESNOCarolyn, doesn't the FDA already have a fast-track for situations where there's a particularly promising drug and a particularly desperate situation?
JOHNSONYeah. There's a break-through therapy kind of pathway. And, you know, they've touted their success at that, at bringing things to market quicker. I mean, this -- for antibiotics, it kind of creates -- it's very specific -- the bill creates a lot of kind of criteria for, you know, how drugs could get -- be tested in a smaller population and, you know. But, you know, I should bring up, there are a bunch of other kind of like things sprinkled into the bill have raised people's eyebrows.
JOHNSONSuch as, kind of a loosening of the reporting requirements for -- now, doctors that receive money from pharmaceutical companies have to make that public because it could be a conflict of interest. I mean, what if you're prescribing a drug from that pharmaceutical company? That could, you know, skew your interest toward the company, maybe not your patient. And this would allow -- this loosens that up. It allows things that are considered medical education to be, like, concealed from that open requirement.
SESNOWhat? So my doctor who's prescribing medicine to me, I might not know that he's on the take from some pharmaceutical company?
JOHNSONWell, I mean, it just -- it opens the door for that, which was like, you know, kind of...
SESNOIs that right, Dr. Brawley?
BRAWLEYI don't think so.
SESNOI mean, that -- that's -- that would be outrageous, wouldn't it?
BRAWLEYI think that would be terribly outrageous. I don't think the bill actually condones that sort of thing.
SESNOBut even if it opens the door a little bit?
ZUCKERMANIt more than opens the door a little bit. A law passed three years ago saying that all of those kind of amounts of money given to doctors would have to be public. And this law specifically says, "Well, not if it pays for medical journal subscriptions and not if it pays for textbooks and not if it pays for certain other things." It's very specific and it certainly says, certain kinds of thousands of dollars can be given to doctors without it being public.
BRAWLEYI don't think it's thousands of dollars. It allows for paying for educational things, especially books, not trips or anything like that. You know, the pendulum on disclosure has gone so far that I accepted a $1.50 Starbucks coffee from a drug rep not too long ago and I'm -- my name is now on a computer data list as having received something of value from a drug company.
SESNOWas that a tall or a grande? I'm going to go there to see.
BRAWLEYIt was tall. Yes.
SESNO$1.50, I can figure that out. I want to ask Dr. Brawley this question. Let's make it real for a minute. I'm a patient. I'm desperately ill. There is a drug that's in clinical trial but it's not ready to be -- or it's not in clinical trial yet. But it should be or it could be or whatever. How is that going to help me? How is that going to help my life, this bill?
BRAWLEYYeah, I actually would like to get the drugs into clinical trial faster. Right now, we do have...
SESNODoes this -- this allows that?
BRAWLEYYeah. In a scientific way.
SESNOHow much -- how much faster? How much faster?
BRAWLEYWell, it's going to vary from drug to drug. This -- what this bill really does, especially when we talk about -- Congressman DeGette talked about IRB regulation.
BRAWLEYThis removes a lot of the paperwork.
BRAWLEYYeah. These are committees at every university that does research that has to look at the research. If I do a clinical trial -- I've done a clinical trial at 210 centers in the United States and Canada. Under the current law, every university's IRB or this committee that only meets once a month must sit down, look at my research and say, "Yes, this is good research and this should be done." Under the new law, it would be one large IRB that would be made up of people representing a number of those universities doing that. So it's just to accelerate the paperwork. It's not removing safety. It's not removing efficacy standards. It's just making things faster and removing bureaucracy.
ZUCKERMANI agree with that completely. But the other side of it is actually selling drugs that haven't been tested well yet. And I'd like to give the example of Alzheimer's disease drugs.
ZUCKERMANThat's something Diana DeGette mentioned and that we all care about. A few years ago, a very, very promising Alzheimer's drug, called Dimebon, was touted as the next great thing. And in The New York Times, in many places, doctors were quoted saying, "This is a breakthrough, wonderful drug." But it hadn't been approved yet. But based on these preliminary studies, it looked fantastic. The preliminary studies, the first one had about a dozen patients, the second one I think had a couple of hundred. But the FDA has higher standards and they required a controlled clinical trial to compare it to other treatments. And that's when they found it didn't work at all.
ZUCKERMANAnd because they found that this drug didn't work at all, it was never approved by the FDA and it wasn't sold to patients. But under the -- this change, these changes that are proposed in 21st Century Cures, this drug absolutely would have been approved. It would have been sold. It would have been used by many, many patients who could have benefited from other drugs, but would have instead have taken one that didn't work.
BRAWLEYSee, that's exactly where Dr. Zuckerman and I disagree. I believe that this -- the new system would stop a drug like that from going in the trial.
BRAWLEYIt's still -- the new system would still require that there be clinical data. It would still require that there be research submitted to the FDA, and the FDA give approval.
SESNOCarolyn, talk a little bit about clinical trials and just give us a little bit of background on how they work now.
JOHNSONWell, the FDA actually does have already the power to kind of, you know, structure clinical trials, which are trials where they give patients suffering from a disease a drug. And then they give the patients maybe a standard of care or kind of a placebo treatment that's like a sham treatment, just to see whether a drug works. And already, the FDA can allow for clinical trials to be tested in small subpopulations of patients. There's a whole exciting generation of new cancer drugs that are, you know, working on people who have a particular genetic mutation driving their lung cancer or, you know, another kind of -- so they have the ability to do that.
JOHNSONBut they're generally large, you know, lots of thousands of people. And they're expensive and so that's kind of how. And then the idea is you can make it smaller. You can make it kind of infinitesimally smaller, potentially, depending on who you talk to.
SESNOI'm Frank Sesno and you're listening to "The Diane Rehm Show." And if you'd like to join the conversation, excuse me, or ask us a question, please do so. Dial us up at 1-800-433-8850. Send us an email at firstname.lastname@example.org. And we're going to move to your parts of the conversation right now. Here we have a comment from -- off of a site of -- interesting question. And, Carolyn, I'll let you take the first shot at this. "With 330-plus in favor, who voted against this. I'd love to hear their reasons for voting against it.
SESNOI'm not saying I agree with them. Just want to know why they vote against it."
JOHNSONWell, you quoted someone at the beginning of the show who did vote against it.
SESNORosa DeLauro, right.
JOHNSONAnd she -- her reasons were more her concerns with the bill. Most of the people, I believe, who voted against it were from the Republican Party and opposed the way the funding increase was going to happen for NIH. So it was sort of tangential to kind of the core issues we're discussing here right now. But maybe they have a different kind of take on the opposition.
BRAWLEYWell, I'm not exactly sure why folks voted against it. I can tell you that many people don't understand this concept of validated biomarkers -- that is, that we have genomic ways of figuring out that a drug works or a drug meets its target. And with those validated biomarkers, we can do smaller clinical trials involving smaller number of people. And I might be able to do a large number of clinical trials in breast cancer. Maybe, since we've now got 80 different kinds of breast cancer, looking at the various markers, maybe we can do 80 different trials using perhaps 90 to 100 people per trial. Whereas, we used to do a big 6,000-, 7,000-person trial.
ZUCKERMANYeah. Dr. Brawley...
SESNOI'm seeing a furrowed brow here.
ZUCKERMANYeah. I just have to respectfully disagree. If you look at the actual language of the bill, you will see how many loopholes there are in it. And even if the scientists at the FDA now would have higher standards, and even if the leadership at the FDA now would have higher standards, once it's in the law, future administrations and future leadership might have much lower standards.
SESNOWhy are you, you know, excuse me, 300 votes on the Hill, you're saying "Whoa. Hold on here." And you're trying, I think, to be an advocate for consumer safety and patient safety...
SESNO...which is up against patient urgency...
SESNO...when they want to adapt to this new world that Dr. Brawley talked about earlier, where everything's moving so quickly. Why do you feel so strongly about what's in this bill?
ZUCKERMANI think we can have both. I think we can make changes that will certainly give us more money at NIH and make changes that are modernized to a new world and new definitions of cancer, and at the same time, keep standards and, in some cases even strengthen them, to make sure that the drugs and devices that are being used by millions of patients have been tested as well as possible.
SESNOBut are you saying that you think patients are going to be at risk -- at greater risk because of this bill?
ZUCKERMANOh, absolutely. Because we already see there are patients that have been harmed.
SESNOBecause we'll rush things to market.
SESNOAnd what's the motivation behind this bill, then? Is this the pharmaceutical companies driving this? Is that what you're suggesting?
ZUCKERMANWell, there's a lot of support from pharmaceutical companies. And there have been a lot of money being spent on this. And there's no doubt about that. But also, I think a lot of people who support this bill just misunderstand. They don't understand how FDA works. And they think FDA is the enemy. I just want to...
SESNOLet me let Carolyn jump in here quickly.
ZUCKERMANOh, okay. Sure.
SESNOGo ahead, Carolyn.
JOHNSONI guess, just to bring this sort of, to zoom out a little bit. I just wanted to pose the question to both of you, because I've heard it from a lot of physicians and scientists, that the bottleneck in the -- the question of where is the bottleneck in the drug-approval process. Like, a lot of people who do medicine don't see it as the approval process. Is it the science?
SESNOWe're going to put that -- that, is the science is the bottleneck question on the table. Come back to it in just a moment. You're listening to "The Diane Rehm Show."
SESNOWelcome back to "The Diane Rehm Show." I'm Frank Sesno sitting in for Diane today. We're talking about the 21st Century Cures Act, which would bring drugs and medical devices to market more rapidly, put $8.75 billion in additional research money over five years into the National Institutes for Health.
SESNOWe're talking with Carolyn Johnson, business reporter at the Washington Post, she focuses on health care, with Dr. Otis Brawley. He's the chief medical officer at the American Cancer Society, professor of hematology, oncology and epidemiology at Emory University. And Diana Zuckerman, she's president of the National Center for Health Research. It's a nonpartisan think tank focused on issues of public health.
SESNOCarolyn, you put a question on the table before the break, where is the bottleneck here? We'll come back to that because I do want to get to our calls. Several people have been patiently waiting. So let me start with Jordan, who joins us from Pemberville, Ohio. Hi, Jordan.
JORDANHi. Thanks for taking my call.
SESNOThanks for calling.
JORDANThis was briefly questioned right before the break and it's unfortunate I feel like this has to be brought up, but an inquiry is justifiable that, you know, it was stated before that Congress kind of putting some pressure on the FDA to maybe not necessarily lower the standards, but maybe try to expedite the process getting the drugs out there. The pharmaceutical companies -- do we have an idea of how much influence they're putting on Congress to kind of push, you know, this legislation so that just maybe it's -- the science is there, but yet, there's more money. So it's, you know, economics pressure there.
SESNOOkay. All right. All right. Carolyn, let me ask you. You've been covering the story. What's the role the pharmaceuticals have played in this 21st Century Cures Act?
JOHNSONI have to confess, I haven't looked specifically. I mean, I've looked at overall. And lobbying, of course, plays a role. And, you know, industry groups have definitely had some input into this. I mean, I've talked to a Harvard Medical School professor who called this bill a Christmas tree to, like, the kind of drug and device industry. And his, you know, I think that it has improved. A lot of people do think this iteration of it that got passed is better. But there are still some weird things.
JOHNSONLike the White House put out its own statement expressing concern about, like, a provision that extends the time that people -- pharmaceutical companies of this certain kind of drug could have exclusivity from marketing it. And they found it would cost hundreds of millions more dollars for drug costs. They were concerned about that.
SESNODiana, what's the role been for the pharmaceuticals in getting this bill through?
ZUCKERMANWell, I can tell you that every time we've gone to speak with Congressional staff or members of Congress, they've already talked to numerous people funded by pharmaceutical companies. So they were lobbying very heavily for this bill. And there's a lot in here that they like. And, you know, I'm not opposed to them liking parts of the bill. And I think there are parts of the bill we all like. But there are also parts of the bill that absolutely benefit the pharmaceutical companies, not patients.
SESNOBack to the phones, and Lisa joins us now, from Raleigh, N.C. Hi, Lisa.
LISAYes. My question has to do with people who are poor or either on Medicaid or Medicare, being usually or often unable to get access to new medications. And I'm wondering if the Act addressed anything to do with the unaffordability of new medications that may help people.
SESNOHow about that, Dr. Brawley?
BRAWLEYI'm not familiar with any part of the bill that really does address getting the medicines to people. I think it is a weakness and something that needs to be addressed.
SESNOI mean, some of these medications, the new medications, are ferociously expensive. Diana?
ZUCKERMANYes. That's a big problem, that the new medications are almost always more expensive than the old ones. If they're not tested to make sure they're any better than the old ones or even as good as the old ones, that costs a lot of money. It -- and Medicare will go bankrupt sooner.
SESNOJason's on the line from North Carolina. Hi, Jason.
JASONHi. Thanks for taking my call. And you've touched on some of the things I'm curious about. Overall, this is very interesting legislation. However, as your pointing out, drugs aren't cheap. I mean, AIDS and cancer drugs can be very expensive. And the speed of production -- and I'm not sure exactly what speeding up necessarily entails, but as was mentioned earlier, speeding up the process won't necessarily create better drugs. And I can't see how it would necessarily make them cheaper. So it seems that it would technically benefit the drug companies more than patients. So how exactly will approving them faster be better for the patients instead of, say, drug companies?
BRAWLEY…you know, there are -- let's take the -- let's take some real examples. The development of the drug cisplatin for ovarian cancer, it took about six to seven years of research before that drug, which has now been on the market for almost 20 years, it took six to seven years of research and clinical trials to get the drug approved. A goodly part of that six to seven years, because it was multiple institution studies, was going to each institution and getting the paperwork signed. This is not…
BRAWLEY…getting the -- yeah. This is not giving the drug to people to see if worked. A large part of that time was getting the paperwork signed. And one of the reasons why I like this bill is this bill gets away from a lot of that paperwork.
SESNOI have an email here from Phillip, who writes us with the following. And Phillip, thank you for sharing this with us and for allowing us to discuss it. He writes, "I have been diagnosed recently with ALS. Does this bill provide an opportunity referred to as Right To Try?" And he points out, "There's a drug called GM6 that's available, but unaccessible to those of us looking for something to slow down, reverse this incurable, untreatable disease. Right To Try would give us that opportunity." Diana?
ZUCKERMANThere are already is a Right To Try. There already are humanitarian exemptions. The FDA does allow patients to have access to experimental drugs that have not been approved yet. It's actually -- has to be okayed by the FDA, which virtually always does okay it. And it has to be okayed by the pharmaceutical companies, frequently they don't okay it. So patients have…
SESNOAnd why don't they okay it? What's the reason behind that?
ZUCKERMANWell, because they would rather have a clinical trial where they choose the patients, they choose the patients most likely to benefit. If they choose any patient who wants to try it, their drug might not look as good.
BRAWLEYThere are actually drugs where they have done Right To Try, and because the first couple of people to get this drug did badly, the drug got a bad name and its eventual approval was delayed by years. So a drug company actually could easily legitimately say doing Right To Try is bad.
SESNOI think that's…
BRAWLEYNow, this bill would hopefully help us accelerate getting those drugs through the regular mechanism.
SESNOI think I have someone on the phone now, Shannon from California, who has a question or a comment about Right To Try. Shannon, have I got that right?
SHANNONHello, panel, thank you for taking my call. I am a parent of a two-and-a-half-year-old with Spinal Muscular Atrophy Type 1. Her symptoms manifest just like ALS, but for babies. And with regard to Right To Try, there are two very promising drug trials that are happening right now within the Spinal Muscular Atrophy community. However, my child and thousands of others who actually have the symptoms of the disease are not being accepted into these trials.
SHANNONSo as far as the Right To Try, I think you already hit on that point, which the pharmaceutical companies wanting to, I guess for the greater good or for whatever their motivation is, but I feel like the regulatory agencies, you know, FDA in particular, who were designed to protect us and protect the consumer really needs to apply a little bit more pressure.
SHANNONAnd I realize these pharmaceutical companies are billion dollar companies and probably have great lobbyists. But for people like me with a child whose alternative is death, I would be willing to sign away any kind of waiver for anything to possibly get her any kind of medication that might help her because the alternative is death.
SESNOThank you, Shannon, first…
SHANNONAnd today, what I've heard is that the quote for the price of much this is gonna cost when it is finally approved is about $300,000 a year. And what regular family can afford that?
SESNOExactly. Well, first of all, good luck with everything that you're confronting. And, Dr. Brawley, let me let you start with this.
BRAWLEYYeah, I totally…
SESNO'Cause this is where it gets real.
BRAWLEYI totally feel for you. And I totally understand where you're coming from on this. The -- I hope that this bill will get drugs like this approved faster. Get them through an assessment and Dr. Zuckerman and I agree, it does need to have a rigorous assessment. We may disagree on what rigorous is, but it does need to be assessed. And hopefully it will be assessed. The other thing I will say to the last caller is, under the current law and under this law, the FDA has very little ability to demand that drug companies do anything. The FDA has about as much ability to make them get that drug to market as you and I do.
ZUCKERMANYes. And I, my sympathy also for your situation. I do want to point out that experimental drugs are experimental. And the reason why we have clinical trials is to find out if people will benefit. And it may seem like you're completely desperate, and I understand that, but even if your child is going to die eventually, you still don't want your child to die sooner. And so you don't want a drug that might make that happen. And so we do have to look at both sides.
BRAWLEYCan I just say that I agree wholeheartedly with Dr. Zuckerman. Because I have seen people who were harmed because they went on experimental studies.
SESNOAnd made it worse?
SESNOThat makes it worse.
SESNOCarolyn, I want to come back to the question that you had tabled before we went to a break, which is where is the bottleneck here? And you posed that to our guests. Dr. Brawley?
BRAWLEYI think the bottleneck is the new science. The bottleneck is the fact that we can't do a large number of these perspective clinical trials the way we used to. The way we developed drugs in the 1980s…
SESNOJust because there are too many?
BRAWLEYJust because we now understand the diseases better.
SESNOSo you can't do…
BRAWLEYThe molecular biology that we've developed over the last few years -- yeah. I used to do, you know, when I was involved in the development of cisplatin for cervix, I'm sorry, cisplatin for ovarian cancer, that was a 300-person trial on women who had ovarian cancer. Now, ovarian cancer's not just one disease. We're starting to say that it's a disease with this particular mutation or a disease with that particular mutation. In the case of breast cancer, breast cancer is now 80 different diseases, no longer one.
SESNOSo you've got to streamline this process. And until you do that…
SESNO…you've got this bottleneck.
BRAWLEYWe don't have enough money to do these large trials.
SESNOI'm sorry, Carolyn, you want to jump in? Go ahead. It's your question.
JOHNSONWell, I was gonna ask -- buy you think we have the insight, you know, we know how to design the trials, we have the drugs, they're just not getting there or, I mean, I guess I was wondering if it's the discovery process itself that, you know, we don't -- is it that, like, there's this wall that we're slamming into because we can't get them approved, because we can't design the trials small enough or effectively enough or is it we don't understand the biology well enough?
BRAWLEYIt's -- it varies from disease to disease, but there are certain diseases -- I'm thinking of crizotinib and the treatment of non-small cell lung cancer. That drug really helps 1.5 percent of people who have non-small cell lung cancer. We need to do clinical trials with people who have the marker, the biomarker that says crizotinib works and then we ultimately did approve crizotinib.
ZUCKERMANI think this is a good example of how certain things in the bill could be very, very helpful. But the bill doesn't change the process with a scalpel. It changes it with a sledgehammer. We haven't talked at all about devices, such as heart valves, which, under this bill, could be put on the market without any kind of clinical trials at all. Or even without a company having to tell the FDA that they had a type of heart valve, they've changed it, now they're going to sell a new kind. They wouldn't even have to tell the FDA beforehand or ask.
SESNODr. Brawley, doesn't that cause you great pause? That something like that could go on the market without…
BRAWLEYYeah, Dr. Zuckerman and I have a difference in our interpretation of the bill. I don't think a device could get on the market without a clinical trial.
ZUCKERMANI'll be happy to show -- well, first of all, 99 percent of devices do get on the market without clinical trials, but I'm happy to…
BRAWLEYWithout a prospective randomized trial.
ZUCKERMANNo. Any clinical trial. I'm happy to…
SESNOAll right. Well, before…
ZUCKERMAN…show you that legislation.
SESNOWe'll let you have that conversation later and we'll need to come back to that, as well.
SESNOBecause that's a vital question. We'll come back with more of your calls and questions for our panel at 1-800-433-8850. You are listening to "The Diane Rehm Show." I'm Frank Sesno and our guests today, Carolyn Johnson, business reporter at the Washington Post, Dr. Otis Brawley, chief medical officer at the American Cancer Society and Diana Zuckerman, president of the National Center for Health and Research.
SESNOAnd I want to go the calls now -- back to the calls. And I believe we have Maria from Virginia. Hi, Maria.
MARIAYes. Good afternoon. Thanks for having me. I had a quick comment and question. I'm going to be really…
SESNOLet me ask you to be very quick 'cause we're almost out of time here.
SESNOSo the more here that we can get in, the more we can do.
MARIAMy -- okay. My question is about the -- within the bill there is an allowance for the -- utilizing Bayesian statistical analysis for approving future vaccines and medicines. And I'm horrified by that. So if someone wants to explain to the listeners about what Bayesian analysis statistical analysis is.
SESNOAll right. I -- okay. And Dr. Brawley, let me take -- let me let you take that.
BRAWLEYThat's a three-hour conversation.
SESNOWe don't have three hours. Can you reduce it to about 10 seconds?
BRAWLEYYeah, yeah. It -- what the bill is actually trying to do is actually to look at large data and then Bayesian analysis is a way of actually selecting the data that you're actually going to use.
SESNOBut this matters, right? This precision matters. We don't have three hours here, but we have, in clinical trials and other trials, we take three hours, three days, three years…
SESNO…to make sure people are safe.
ZUCKERMANI just want to say I don't think Congress should be telling the FDA scientists what kind of statistics to use, period.
BRAWLEYYeah, I would agree with that. We should allow the experts to decide what the appropriate statistics to use. I would actually go to a statistician and even declare myself not eligible to make that…
SESNOKaren joins us from Lawrenceburg, Ind. Hi, Karen. Go ahead with your question quickly, please.
KARENHi. Yeah, my question has to do with the long-term effects of drugs that are approved. I have epilepsy and I took Depakote for -- to control my seizures during pregnancy. I have two sons that have been diagnosed with fetal valproate syndrome and have autism. So how would this allow for trials to consider future generations if they're cut short? Also, if you're gonna put this great burden on the FDA, is there going to be additional funding for the FDA so they can attend to these things in a timely manner?
SESNOThanks, Karen. Great questions. Diana Zuckerman?
ZUCKERMANTo answer the second part, they get a half billion dollars, which is a lot less than NIH gets. And FDA has said not enough to do what they are asked to do. One of the issues of expedited review, when you're speeding drugs to market, or devices, you study them for a shorter period of time. Then you say, after it's on the market we'll study it for a longer period of time. But if you are pregnant and you have a child with a birth defect due to that exposure, it's too late for you.
SESNODr. Brawley, I want to ask you one last question. This one comes from an email from Pearl. It's a great topic. We haven't talked much about it. She writes, "There's plenty -- there's already a shortage of currently effective chemo drugs, due in part to big Pharma's unwillingness to produce drugs that are of low profit to them." She says, "Will this bill provide further incentive for companies to pursue high-priced, new drugs while neglecting current treatments?"
BRAWLEYThe bill actually does allow for people who have -- or companies that have drugs that are for a small niche to have a longer patent on that drug. That actually, unfortunately, is going to keep the price up. But it gives the company an incentive to produce something for which there's a small market.
SESNOCarolyn, you've gotten probably about five story ideas here today. At least I hope you have. But as you go forward and you think about where this story's heading, what are next steps for the bill? How are these questions, that are still not fully settled, going to be addressed in the route ahead?
JOHNSONWell, the Senate hasn't put out a draft of its version of the bill. And that's -- they've had meetings on it. They are still -- so a lot remains to be seen. And it could be very different. They haven't really kind of been very firm about what they're going to put in, how they're going to approach these questions with the FDA. So a lot depends on that. And I know behind the scenes, probably the people at this table have been talking to them about their concerns and what they think is good. So we shall see.
SESNODiana Zuckerman, what's next?
ZUCKERMANYes. The Senate is -- doesn't have an introduction of a bill yet. So we don't know exactly what they're going to do.
SESNOTiming, Dr. Brawley?
BRAWLEYI'm hoping that there will be something out before the end of this Congress. It's going to be a long time, though.
SESNOTo Carolyn Johnson, Dr. Otis Brawley and Diana Zuckerman, thank you all for a fascinating conversation on the 21st Century Cures Act. I'm Frank Sesno sitting in for Diane Rehm. Have a great day.
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