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Should Patients Have A Voice In FDA Drug Approvals?


People suffering from rare or chronic diseases often have to wait years for new drugs to get through the complicated regulatory process in the US. But, putting prescription drugs on the market too soon can put patients at risk. On our monthly segment on ethics in science and technology, we'll talk about the ethics of giving patients more of a voice in the regulatory decision-making process.

People suffering from rare or chronic diseases often have to wait years for new drugs to get through the complicated regulatory process in the US. But, putting prescription drugs on the market too soon can put patients at risk. On our monthly segment on ethics in science and technology, we'll talk about the ethics of giving patients more of a voice in the regulatory decision-making process.


Michael Kalichman, director of the Center for Ethics in Science and Technology. He is Director of the Research Ethics Program at UC San Diego.

Duane Roth, longtime leader in the biotech industry here in San Diego. Among many other titles. Is he Founder and Chairman of the Board of Alliance Pharmaceutical Corporation and Vice-Chairman of the citizen's oversight board of the California Institute of Regenerative Medicine.

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This is a rush transcript created by a contractor for KPBS to improve accessibility for the deaf and hard-of-hearing. Please refer to the media file as the formal record of this interview. Opinions expressed by guests during interviews reflect the guest’s individual views and do not necessarily represent those of KPBS staff, members or its sponsors.

MAUREEN CAVANAUGH: I'm Maureen Cavanaugh, and you're listening to These Days on KPBS. News about a potential new treatment for a devastating disease is just a nice headliner for most of us. But for patients suffering from incurable illnesses, such news is it a life line, after the breakthrough, patients wait to hear when research will result in a new drug or treatment, however, that wait can stretch into months or years, today on our monthly series of discussions on ethics in science and technology, we'll ask whether patients should be more involved in the process of FDA drug approval. I'd like to introduce my guests. Dr. Michael Calichman is director of the Center for Ethics in Science and technology. And Micah el, welcome back.

CALICHMAN: Good morning.

MAUREEN CAVANAUGH: And Dwayne Roth is the CEO of Connect. He is a long time leader in the biotech industry here in San Diego. And he is vice chairman of the citizens' over sight board of the California institute of regenerative medicine. Dwayne, welcome to the program.

ROTH: Thank you, it's nice to be here.

MAUREEN CAVANAUGH: Now, we are inviting our listeners to join the conversation. If patients with life threatening illnesses are willing to take a risk with new drugs, should they be allowed to do it? Have you or someone in your family waited fiduciary a new drug to be approved we the FDA? Give us a call with your questions or comments. Our number is 1-888-895-5727. That's 1-888-895-KPBS. Dwayne, I know you're very familiar with this, so tell us what the process is now, currently, for a new drug developed in the lab to get FDA approval?

ROTH: So it starts with what we call a data package. It's a preclinical animal studies that show the safety and efficacy of that compound as best we can get it. And at that point, we can apply for phase one or early clinical trials.

MAUREEN CAVANAUGH: And then the clinical trials begin with human beings?

ROTH: They begin in man. And depending on the product, it may be in a patient population or on healthy volunteers. And that progresses then to phase two, which involves more patients and you're starting to look at both safety, which we look at predominantly in phase one, and in phase two, look at efficacy, as well as safety. And if that proves out, then large randomized studies in phase three, which takes a considerable period of time usually to conduct, and if those are successful, then you did through the final process of applying with a full data package to have that drug approved for use.

MAUREEN CAVANAUGH: So you take a full data package after those long series of tests to the FDA. How long does it take for them to actually look at if.

ROTH: ? So from the discovery of a new compound to approval is on the -- on the shorted is, ten years.


ROTH: And on the long side, 15 years or longer.

MAUREEN CAVANAUGH: Aside from patients actually being involved in these clinical trials are they involved in the process of approval benefit the FDA?

ROTH: Currently they have advisory capacity. So the FDA will look to patient groups in a disease area, and talk about how they feel about certain product, ethics, come into that, obviously. But their direct involvement is quite limited.

MAUREEN CAVANAUGH: And so they're not, as you say, at the table?

ROTH: So my bias here is that it's their disease. And when the FDA meets with a company to talk about a new product, a new innovative product, they're not in that room. And it makes it very difficult when the FDA is obviously and should be biassed towards safety. That's their concern. Predominantly. And a company sees all the good things that this product could do. And when that discussion takes place about how much preclinical data, how much phase one, phase two data, what are the right end points? That risk benefit is inherently biased between those two parties. And my idea is that the patient should have more involve because I think they can help both parties, they can help the FDA by saying we would like to move this forward, make all the disclosures so every patient and doctor sees that, and to the company, they say tell us everything. Even the things you're remotely concerned about. Because we want to react to that. Today if that happens, the company by telling the FDA basically passes the responsibility to the FDA, who again has to look at it from a safety standpoint. So I think having them in the room makes both parties more likely to make the right decision because only the patients, in my experience, can truly see risk and benefit.

MAUREEN CAVANAUGH: I'm speaking with Dwayne Roth, he is the CEO of connect. And doctor Michael Calichman, corrector for the it center for ethics and science and technology. We're talking about the center's new question of their discussion panel this week, and that question is, should patients have I voice in FDA drug approval? If you'd like to join this conversation, our number is 1-888-895-5727. That's 1-888-895-KPBS. I think we have heard on this program, from callers who have called in, and other programs that we've done about scientific research, that this is a definite desire among people who are suffering from chronic illnesses, and from illnesses that tonight have any real treatments at this time, to get this process moving faster. I wonder what dangers you might see in speeding up that process, Michael.

CALICHMAN: Yeah, that's a good question. In theory, the idea that we're talking about here is very attractive, that the people who have the disease could become better involved in the process. The fears that people have is that those people might under estimate the risk because of their fear enforce their disease. The FDA has this interesting history of actually having been viewed as going too fast and too slow, almost like a yo-yo over the past 20 years or so. Of in the early 1990s, there was a sense that they were going too slow, they went a bit faster, then there were multiple drugs that had to be drawn. Then they started going slower again, then they started going faster again of it's been back and forth. And we're trying to get perfection where perfection isn't an option. You either risk not having any drugs and not having any treatments at one end, and you could do that easily by simply saying we're gonna stop approving anything. Or you could have drugs that, in fact, won't be safe and might not even be as effective as they might have if we waited longer.

MAUREEN CAVANAUGH: I'm wondering, Dwayne, what problems in the process have you seen that makes you think this change is necessary? Do you have examples of people who have been waiting for certain treatments to become available? Certain drugs to become available?

ROTH: My definition, any patient who has a disease at the time that drug is actually approved, has been waiting for that product of it's a question of when that time frame is gonna occur. Will it occur in time for me to help me or will it happen some years subsequent to that. And that's where I did back to, is there a process where we can share the risk and benefit with the person most affected? As I said, it's their disease. Yet the FDA or the company make decisions about their disease without them being part of that process. And I think it's I new way in this country that we've started to think about having the most involved parties participate in that regulation and approval and the FDA would be a perfect place, the FDA regulation, I think, to exercise that -- to get to what Michael was really talking about. How do you balance it? If it swings from risk as it did in the HIV days of the '80s to benefit? Suddenly the FDA and the companies and the patients in that generation got together and products got approved in six months, with all the proper precautions that these products are not fully tested, and each patient doctor were allowed to make that decision. Whether they go on those therapies or not.

MAUREEN CAVANAUGH: I'm wording, what types of diseases, patients suffering from what types of diseases would be more fully impacted with the kind of danger that you're proposing, Dwayne?

ROTH: So I think cancer, chronic diseases like autoimmune diseases like MS, multiple sclerosis, any diabetes, any of those products -- or diseases where you're going to be taking products for a very long period of time. Time is, you know, it's the quality of their lives. And that's what we're trying to figure out. How could we do that in a way that we could share this risk and bring a third party into the room to actually balance that risk and benefit? In my experience, and this came really out of this regenerative medicine where we have patient advocates sitting on the board, and working with them over the last four years, even though I spent my career in this industry, I could not possibly have looked at things the way they did. And that's what we have to say. Why in the world do we relegate them to this cheer leader role, almost, hoping that these products get there instead of being directory involved. Because I think they would balance that risk and benefit.

MAUREEN CAVANAUGH: Away on the other hand if anyone in our audiences actually heard of a medical break through that might help them, and they have been waiting to hear about the release of I new drug or treatment that they can actually benefit from. If you have, give us a call and let us know. 1-888-895-5727. Ellen is on the line from San Diego, good morning, Ellen, welcome to These Days. Of.

NEW SPEAKER: Morning. How is everybody?

MAUREEN CAVANAUGH: Just great, thank you.

NEW SPEAKER: This is an interesting conversation. I appreciate the break down into the various stake holders in this whole process. And I wanted to address one other group of stake holders, as long as one of your guests is it a specialist in ethics, and that is the stake holders of the whole new group of creatures that are now turned into patients by giving them these kinds of heart breaking diseases like cancer. How does the process, especially in stage one, apply to the care of these new patients to your animals being tested? How do ethics apply to pain management and loneliness and [CHECK AUDIO] and I will take my answer off the line. Thank you.

MAUREEN CAVANAUGH: Ellen, thank you for the call, and I dare say that opens up an entirely different question.

ROTH: Well, it might be worth a show or two. But in the meantime, there is the research that we do in humans, necessarily, by almost every ethical convention is supposed to be based first on studies in animals. The caller's question say good one about what we do. We don't prohibit the use of animals in research. Clearly the research model we have is based on animal use. But we do have expectations about how these animals will be cared for. So the caller mentioned a couple things about pain and suffering, about the environment the animals are in, those things are all key and it depends on the kind of research, what will be done. My experience of animal research is that typically animals are not supposed to be suffering pain or other kinds of suffering unless that is the purpose of the study. And it's a good question for society to decide is pain for humans so bad that we think it's worth allowing models and studies of animals to include looking at pain and suffering?

MAUREEN CAVANAUGH: And as you said, that is another ethics question that would be very interesting to visit.

ROTH: Huge.

MAUREEN CAVANAUGH: Right now, we are talking about whether patients should be at the table, when the process of FDA drug approval is under way. Whether their input and their desire to be able to use these new treatments to treat their ailments and illnesses should be taken into consideration. 1-888-895-5727. Ian is calling from Solana Beach, good morning, Ian, and welcome to These Days.

NEW SPEAKER: Good morning, Maureen, and good morning to your panel. My wife recently passed away from brain cancer. Glioblastoma multiforma, which is the same disease that Ted Kennedy had. That condition is terminal. And I find this whole discussion about safety and ethics to be highly specious because had they presented -- had any drug, test drug or anything, had been presented to us and we would have signed a waiver in a heart beat to let her try that intervention because when you are terminal, you simply cannot wait. Otherwise by the way it's approved, they're dead.

MAUREEN CAVANAUGH: Ian, thank you for your call. And we're very sorry for your loss. Thanks for calling in this morning. This does bring up the question, what would stop a desperate patient from taking on too much risk? Saying, you know, I don't care. I don't care. Just give me the drug. Make the drug available.

CALICHMAN: So I think the patient, ultimately, has to make the decision with her doctor and her family about how much risk they're willing to take. What I would like to see is them have that opportunity to make that decision. And make it as soon as possible, especially in a situation like we just discussed. That oven is not the case because the regiment of the studies that are designed are so carefully constructed that there isn't really room for these compassionate use indications of and getting that in it the hands of the people who can individually make those decision what I would propose, a way to do that that would not interfere with the normal process of the clinical trials, phase one, phase two, phase three. But it's more how do you get that available as soon as possible with an appropriate risk that only the individual can ultimate -- them or their family or their fission together decide?

MAUREEN CAVANAUGH: I wonder though, if you have, let's say, a cadre of patients who suffer from the same disease, [CHECK AUDIO] to get this drug approved for general use, and they say they can accept a certain level of risk, what's to say that the general public suffering from the same disease will agree to accept that level of risk?

CALICHMAN: So I would propose that the informed consent which was referred to as the sign off between the patient and the doctor and the process of get that approved should be made available in what I would call plain English. And not, you know, a book that you have to read through to decide what is the risk and benefit. But a way that it says cheer, here is the risk. [CHECK AUDIO] we know about its side effect potential. We know about all of the things, and you make that available so that individuals can make that risk and benefit decision of the and I think there's a lot of layers of safety built into that, first the FDA, and the company aren't going to put something out there that they don't feel passes the necessary preliminary work to be able to be studied in man. And then secondly, you have the patient and the doctor who will discuss that clinical trial along with their family to make that decision.

MAUREEN CAVANAUGH: We have another caller on the lineup, Patrick is calling in from El Cajon. Good morning, Patrick, and welcome to These Days.

NEW SPEAKER: Hi how are you?

MAUREEN CAVANAUGH: Just fine, thank you.

NEW SPEAKER: I feel, as a medical advocate for previous partners and friends, I think that allowing people to push the FDA [CHECK AUDIO] is just handing them a Pandora's box. A lot of the products that the FDA has pushed through have worse side effects than the problem you're trying to cure. A lot of times, the bottom line is trying to get people these drugs for their fear. San Diego has a problem now with its medical marijuana problem. Medical marijuana does not cure anything, but it alleviates a lot of symptoms that doctors and pharmaceutical companies are willing to pump people full of drugs from. And I'll take the answer off the air.

MAUREEN CAVANAUGH: Okay. Thank you, Patrick. John is calling us from Banker's Hill. Good morning, John, welcome to These Days.

NEW SPEAKER: Good morning, and good morning to your panel. I'm a person that has popped every pill known to man due to HIV. I started in the early '80s. And the very early studies when the first studies came about. And 30 years, almost 30 years later, I find it so almost disheartening to think that the conversation you're having exists today that we are not allowed to have a place at the time. I mean I would have done anything back then, and I did, everything and anything, I had the best, because of the studies, I had the best medical care a person could possibly have in the world available to me because I volunteered. I mean, it was unbelievable the kind of care that I had. And how it followed me right up until the drugs became available. I mean, it was just amazing. And I think that my input, you never get to have an input only with your doctor, and it's kind of sad. Even when I go to volunteer to be part of new study groups I'm told I'm not, you know, a candidate because my viral loads and all these things are in good shape. And I don't want -- so I still don't have a voice.

MAUREEN CAVANAUGH: John, thank you for the call. I appreciate it. Michael, I was just gonna ask very quickly, during the discussion, this ethics forum discussion you're going to have, I would imagine the liability this opens up for drug companies white come up.

CALICHMAN: Well, sure. And I think that's -- that liability of the drug companies is the -- sort of at the root of all of these questions, I think for these last three callers. That the choices we make that might speed things up carry with them risks, and who's going to carry those risks? In looking at the three callers, I think we want to distinguish between the question that I think Ian was asking about for his wife, why couldn't she have an individual choice to use something? And the other calls are focussing on, why can't we systemically create approvals for drugs that could be more widely available? In all cases there is this risk that we're going to move in the wrong direction. As I mentioned to you earlier, you could have -- two of the possibilities are good, fast, and cheap, and you can't get all three. And that balance, that tension is the one that keeps us from continuing to have -- it gives us a situation where the FDA approves drugs that are problematic, and the FDA fails to approve drug that are useful. And we've seen both of those instances multiple times. We will not have perfection.

MAUREEN CAVANAUGH: Dwayne, I know that you just published a paper, presenting basically this as an option, bringing patients to the table during the time that drugs are being approved and given their approval by the FDA. How is your paper, and how is this idea being received?

ROTH: Well, the paper was just published. And obviously I've had quite a bit of e-mail, people reviewing it, and I think the reaction in general is nobody has ever thought about trying to do this. They think about patient advocates, and patient advocacy as a very important role but often it's about the research or about other things that are important but perhaps not as important as their perspective would be on -- as I said, risk and -- benefit, the two parties now, which are -- really, it's almost impossible to write rules that'll change that behavior. The only people can help get that done, I think, are the patient groups. The last caller really hit on something I talked about in my paper. The one time that really worked was during the HIV epidemic. When the companies and the FDA brought together by the patient advocates, worked in harmony, and were able to get things done with appropriate risk and benefit. I think we should institutionalize that process that they created 30 years ago. And we lost that. We do not -- we stopped there. We do not take it the next step. Which is -- this worked, and to this day, I don't care who you talk to, to this day they will talk about what a great experience that was. The sort of marching and screaming that is in those rooms, where recent people came together and said how can we move this forward in a very expedited way because people are dying? And they need this. So that's what -- if I could sum it up, I would say, that's what I'm really going back to and saying, why didn't we institutionalize that?

MAUREEN CAVANAUGH: We are out of time. I want to thank you both, Dwayne Roth and doctor Michael Calichman, and I want our listeners to know that the ethics forum will discuss the question, should patients have a voice in FDA drug approval? It should be a very lively discussion: It takes place this Wednesday at the Ruben H. Fleet science center in Balboa park, and it starts at 5:30. Thank you both for coming in. And if you would leak to comment, please go on-line, Days. Stay with us for hour two, coming up in just a few minutes here on KPBS.

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