Print BCTV: 21st Century Cures Part 1 -- Rep. Upton, Rep. DeGette on 'A Path to 21st Century Cures' Initiative

21st Century Cures Part 1

Transcript of BioCentury This Week TV Episode 193



Rep. Fred Upton (R-Michigan)

Rep. Diana DeGette (D-Colorado)

Kathy Hudson, Deputy Director for Science, Outreach, and Policy, NIH



Centers for Disease Control

Food & Drug Administration

National Center for Advancing Translational Sciences

Dr. Francis Collins



Steve Usdin, Senior Editor




STEVE USDIN: Can Congress smooth the path for faster, better cures? A conservative Republican and a liberal Democrat are trying. And they're asking the public for ideas. I'm Steve Usdin. Welcome to BioCentury This Week.


NARRATOR: Connecting patients, scientists, innovators, and policymakers to the future of medicine. BioCentury This Week.


STEVE USDIN: Fred Upton, chairman of the House Energy and Commerce Committee, has teamed up with Democrat Diana DeGette to launch a Path to 21st Century Cures Initiative. Upton described its goals in a recent conversation with BioCentury.


FRED UPTON: Well, we ought to take what we've learned over the last 40 or 50 years, particularly in gene therapy, molecular structure, all those different things, and use that to benefit the world.


STEVE USDIN: Upton focused on the time and cost of drug development.


FRED UPTON: Why is it that it takes 10 years, a billion dollars, to get a normal drug approved? That ought to change.


STEVE USDIN: He said the Committee will travel the country soliciting public input.


FRED UPTON: So our approach now, for the next number of months, probably six, eight, maybe even 10 months, is to listen. We're going to take this show on the road, I guess you could say.


STEVE USDIN: The Michigan Republican made it clear that his goal is to create legislation.


FRED UPTON: We want to take what we learn and then put it into legislative language.


STEVE USDIN: It's too soon to know what the final bill will include, but Upton said it's likely to provide funding increases for NIH.


FRED UPTON: We've got to work with the appropriators we have to make sure that we authorize the right amounts. But, yeah, I would suspect that at the end of the day, we will see increases for the NIH. And we know that if they do their job well, we're going to -- you know, it was pointed out earlier in the week, for every dollar invested, there's tremendous savings, healthcare savings and everything else at the end of the day. Sometimes as much as 200 or 300 times for every dollar invested.


STEVE USDIN: This week, we'll speak with Upton's Democratic partner, representative Diana DeGette of Colorado.


I'm pleased to be joined on Capitol Hill by representative Diana DeGette. Representative DeGette, at the recent roundtable, you were kind of summarizing things at the end of it. And you noted that everybody who was there, just about, talked about the need for increased and sustainable funding for NIH. How realistic do you think it is? Does Congress have the appetite to spend more money for biomedical research?


DIANA DEGETTE: Well, I think that if there was an appetite to spend any money, it would be for biomedical research. Back in the early 2000s, we doubled the NIH budget. And we did that on a bipartisan basis. And that really helped fuel so many more exciting advances.


And so, then what happened of course, during the Sequester funding was cut for the NIH and for biomedical research, just like it was cut everywhere. And not only did that have a devastating effect on research, but it also had a chilling effect on new researchers coming into the market, and so on. And so, I think realistically, any discussion about restructuring our research institutions in this country has to be accompanied by both how do we give adequate funding, and how do we give certainty that we will have that funding over time.


STEVE USDIN: And do you think that's something that's sustainable? You start out by saying if there is an appetite for increased spending on anything. Is that going to happen in this fiscal environment?


DIANA DEGETTE: I do think it's sustainable over time. Because people realize one of the things the US has is its real leadership in research around the world. And in order to keep that level of leadership against people like China and Singapore, is we really do have to put the money in. And so I do think that there's a recognition that we're going to have to have appropriate funding.


STEVE USDIN: So you mentioned China and Singapore. That's something else that's come up -- that came up in the roundtable, it's come up from one of the white papers that your committee has written. Do you think that we're -- you know, that's the right analogy? Are we at kind of like a Sputnik moment, where there's a competition like in the Cold War that there was to the moon in biomedicine?


DIANA DEGETTE: Well, the United States is clearly the leader in the world on biomedical research. But other countries certainly have realized that, and they're starting to make much larger investments. Because of our education system here, and the way our labs work, and everything else, we will be able to maintain that leadership role, but we have to maintain the funding as well.


And so, that's part of what our 21st century cures is about, is not only increasing funding. But figuring out the ways that we do biomedical research, so that we can do it much more effectively and efficiently. And get those cures from the lab into the clinic as quickly as possible.


STEVE USDIN: So fundamentally, what are some of your other ideas about how Congress can actually speed the creation of new cures?


DIANA DEGETTE: Well, one thing I've been talking a lot about is how the very nature of research has changed. And so, for example, when the National Institutes of Health was first formed, they had all of these different institutes out there. And everybody did separate research. There was the Diabetes Institute --


STEVE USDIN: They're by organ systems basically, yeah.


DIANA DEGETTE: Right. But now what we've learned is the way research works, you might be researching something in diabetes, and find an advance for Parkinson's. Or for obesity, or something.


STEVE USDIN: So do you think that there's an appetite for actually restructuring NIH along modern lines?


DIANA DEGETTE: When we authorized the NIH the last time, we did some restructuring. And so, there is an appetite to really look, not just at the NIH, but also with the FDA, the CDC, other agencies. To see how they're structured, how they interact. And how we can break down barriers, have standardization, do some things really differently.


We don't have the answer yet, we're just at the beginning of the process. But there's a consensus, not just on the elected officials' parts, but on the researchers and on the government officials, that these are really ways that we can redo our system to be at the cutting edge, as always.


STEVE USDIN: So that kind of suggests that you think at the end of the day, when the legislation's ready, that you're going to be making and suggesting really fundamental, big changes. It's not just incremental kind of evolutionary changes that you're looking for.


DIANA DEGETTE: Right. I think we're going to be looking at the whole structure of these agencies. To see how they work, how they can work better, smarter, how we can really keep that edge that we have.


STEVE USDIN: Representative DeGette and Fred Upton have divided their initiative into three interlocking parts: discovery, development, and delivery. We'll talk about the discovery piece with NIH's Kathy Hudson in just a moment.


DIANA DEGETTE: We in Congress are going to take a comprehensive look at what steps we can take to accelerate the pace of cures in America. We're looking at the full arc of this process. From the discovery of clues in basic science, to streamlining the drug and device development process, to unleashing the power of digital medicine and social media at the treatment delivery phase.


NARRATOR: You're watching BioCentury This Week.




STEVE USDIN: To discuss options for improving biomedical research, I'm pleased to be joined by Dr. Kathy Hudson, NIH Deputy Director for Science, Outreach and Policy.


Dr. Hudson, one of the things that Representative DeGette mentioned was the idea that NIH's institutes are organized around organ systems and diseases in a kind of an old fashioned way. And it's intellectually appealing to say, well, let's just reorganize the whole thing in a more modern way. Would it be worth the time and effort and money to do that?


DR. KATHY HUDSON: It's an interesting idea. So we currently have 27 institutes and centers that comprise the NIH. And they are largely organized around diseases or organ systems. They have a long history. More recently, we have institutes and centers that are focused on new kinds of technologies and applications of those technologies.


We recently created a new center at NIH, the National Center for Advancing Translational Sciences that's a good example of a disease-agnostic approach to science. But even though we have these different institutes and centers, the work at NIH gets done in a collaborative way across the institutes and centers. So while it may be attractive to reduce the number of institutes, I think we're able to work around those barriers by collaborative work.


STEVE USDIN: So one of the other things that has come up that's been an ongoing theme -- it was in the round table discussion the Energy and Commerce Committee had and has been in the ongoing discussions -- is NIH budget. And one of the questions I have about that is even if Congress finds the money and has the support to increase the NIH budget and do it in a predictable way, the problem we've got now is that there are too many people chasing with too many ideas chasing too little money.


If you increase the budget, the universities are going to increase their infrastructure, you're going to have more people. You're going to have the same problem over again. You're just pushing it out if there are some structural changes. Or am I missing something?


DR. KATHY HUDSON: Well, I think we do need stable, predictable funding for biomedical research. We've had sort of dry spouts and then the era of funding that came in. $10 billion came into NIH in one fell swoop. Then we had the sequestration where we lost $1.5 billion overnight. So that makes it very difficult to plan and to have a stable, predictable pathway forward.


We do need to look very carefully at our training pipeline and make sure that we are knowledgeable about how many people we are training. Right now, we have people who are on training grants and also people who are on other research grants. We need to know how many trainees we have and be able to train the right number of people for the future of biomedical research.


STEVE USDIN: So does that mean that NIH, you kind of look out there at the broad landscape and say, you know, we've got too many PhDs doing this kind of work, or not enough doing this kind of work, and do things to actually move some levers to change that?


DR. KATHY HUDSON: Well, unfortunately, the lever that's being pulled right now is disillusioned young scientists who don't want to enter biomedical research because it looks so grim out there right now. With the reducing budgets, we've lost 20% of our spending power over the last 10 years. So what that means is that people are spending more time writing grants and less time doing science. The success rates for grants that are being submitted to NIH right now is something around 16% or 17%.


So that's a pretty dismal-looking future. So I don't think we need to pull any levers to reduce the number of trainees right now. What we need to do is have a stable, predictable path for budgets moving forward so that we can make sure that we have enough trainees, but not too many trainees coming along, and that we're bringing the best and brightest young people into science.


STEVE USDIN: One of the things that also came up in the Energy and Commerce Committee roundtable discussion -- it comes up frequently, it came up in their white paper -- is this notion that other countries, particularly China, are doing more than the United States or that they're there on a trajectory of increasing their spending. Is it really is zero-sum game? Or is that trying to create a kind of a false Cold War rivalry?


DR. KATHY HUDSON: Well, I think Americans want to be first. And we have been first in biomedical research and technology development for a very long time. And what we're seeing now is that as percent of GDP, the investment in the United States is going down and the investment in biomedical research around the world as a percentage of GDP is going up. And so we're sort of losing our competitive edge. Dr. Collins likes to talk about the young investigators who are leaving the country in order to pursue more productive, more lucrative, high-paying, high-technology jobs in other countries.


STEVE USDIN: Well, we're going to talk about that and some more right when we come back.


DR. FRANCIS COLLINS: Certainly from NIH's perspective, what is most desperately needed in order to continue what has been the most successful story in biomedical research that the world has ever seen is a steady, predictable trajectory of support. We have not seen that.


NARRATOR: Every month, Biocentury This Week will feature Profiles in Innovation, a special segment highlighting the stories of innovators whose work is improving lives and transforming the world of healthcare.




NARRATOR: Back to BioCentury This Week.


STEVE USDIN: We're discussing the path to 21st-century cures with NIH Deputy Director Dr. Kathy Hudson. Dr. Hudson, one of the ideas in addition to increasing the NIH budget is to try to get more science for the money that you're spending now.


And one of the things that I think has troubled some people is the research that's shown that some of the highest profile research that NIH has done is not reproducible, which kind of begs the question of whether some of that money is not being well spent. What's your response to that?


DR. KATHY HUDSON: So this is a really troubling problem. It's mostly in the pre-clinical arena, experiments using mice, cell lines, other experimental organisms. And what we've found is that because the studies are not well designed, because they are not statistically sufficiently well powered, that those data are not reliable. And so you can't rely on them to move forward.


You can't rely on them to design clinical trials in advance. So those aren't dollars that are well spent. So we are attacking this problem full on. We are trying to train investigators to know how to design a study well, to make sure that it's blinded so that you don't know which group is getting what, and also to have sufficiently statistically powered studies.


We need to change the review process so people are asking questions about the scientific premise of a study walking in the door for a grant application. And then we need to work collaboratively with the other folks who are important in the biomedical research community, including companies, academic institutions, journal publishers, and the like. So this is a community problem, and we really need to attack it together.


STEVE USDIN: And how would you know whether you're making progress?


DR. KATHY HUDSON: I think we'll know we're making progress when we can do replication studies. So we can take some random sample of studies we've already supported and see that in fact they are replicable. We'll also see that when studies move to clinical trials that the scientific premise derived from pre-clinical work holds up in the clinical trials setting.


That's one of the reasons why drug development is such a long and tenuous process is because the clinical, pre-clinical data on which it's based is sometimes flawed. And so we really need to work together to correct that.


STEVE USDIN: And sometimes there are very surprising reasons why science isn't reproducible. One that's come up in the media recently is the sex differences of the people who handle some of the experimental animals, that somehow that influences things. Can you talk about that and what NIH is doing about that?


DR. KATHY HUDSON: So the issue is whether or not you can get scientifically meaningful information if you're only using one sex or another in experimental animals. And so it is believed to -- it was believed to have been easier if you kept just, for example, male mice. It turns out that's not necessarily the case, and the biological variability among male mice is as much or more than female mice.


And you're only learning about what goes on in XY creatures, so you're not really learning about the entire spectrum of responses. So we are taking this very seriously. We're now asking grantees, when they come in to us, to talk to us about how they're going to use, what sex of animals they're going to use, and how they're going to do the analysis based on sex.


STEVE USDIN: So another thing that Dr. Collins mentioned in the round table that I think was surprising was an issue of travel restrictions on NIH scientists and said that that's really impeding science. What's that all about?


DR. KATHY HUDSON: So it's not really about scientists wanting to travel. It's more about science not being a solo activity. It's a group activity. And so scientists need to be able to share ideas, communicate with one another. It's all about the interaction and sharing of ideas.


And so with the recent travel restrictions, we're unable to send our scientists to important scientific meetings to present their work and to learn about the work of others. And importantly just to exchange ideas and get new ideas from the catalysis that happens when people are together. So this has really dampened our ability to send scientists to important meetings.


STEVE USDIN: And where do these restrictions come from?


DR. KATHY HUDSON: They originally came from within the administration, and more recently Congress has followed suit and is now law. These travel restrictions are in law.


STEVE USDIN: And presumably, then, you're trying to get that law changed and the policies changed.


DR. KATHY HUDSON: We need some relief from this.


STEVE USDIN: Does NIH need to -- would it be appropriate for more money to be focused on translational research and less on basic research?


DR. KATHY HUDSON: So, presently we spend about 50% of our extramural research dollars on basic research, and that proportion has been really pretty consistent over many, many years. It's important to have focused translational efforts and disease-specific research that are trying to develop an intervention or a diagnostic. But we also need to protect the basic research portfolio for a number of important reasons.


One is because we need to expand knowledge about how living things work, including ourselves. But we also can never really anticipate where the next great thing is going to come from. There are so many miraculous things that we take for granted today in research and particularly in research tools where you would have never gotten that insight if you hadn't allowed free exploration and asking questions about how does this work.


I'll give you a couple of examples. So right now we're working on the BRAIN Initiative at NIH and across the administration. And one of the most amazing techniques is this technique called optogenetics where you can use light to control gene expression. It turns out that that was derived from understanding how light-sensitive genes in algae work. So the study of algae ended up having this tremendous impact on our ability to understand and study the brain.


Another example is this new technology called CRISPR, which is a genome editing technique that we may see enter into human gene therapy and human therapeutic trials. But right now it allows us to precisely edit the genome of virtually any organism, even those that were previously thought to be sort of genetically inaccessible, unlike fruit flies and other organisms that we know about the genomes of.


So these are techniques that were developed because of basic research in plants, and microbes, and other creatures that was undirected. And really the researchers probably had no disease focus in mind at all. And yet it's had this tremendous impact.


STEVE USDIN: So how do you preserve, on the one hand, this kind of curiosity-driven research and fund people over long enough periods of time to try to make it real and on the other hand respond to the kind of urgent public health needs that we have today?


DR. KATHY HUDSON: Right, so we need to be able to have some flexibility in terms of the length of the awards that we provide to people so that they're not writing grants all the time but able to really do their research. And we're looking at making longer awards now and also focusing not so much on the proposed research but on the person who's proposing the research and whether we can have more people-focused awards rather than project-focused awards.


STEVE USDIN: So backing winners, basically.


DR. KATHY HUDSON: Yeah, backing the winners.


STEVE USDIN: Great. Well, thanks. We'll be right back to talk about the path of 21st-century cures with Dr. Kathy Hudson.


DIANA DEGETTE: But it's really not just the matter of, let's just increase the budget of the NIH and/or the FDA, because it's not linear. It's not like purchasing things. It's not like purchasing warships or something like that. You've spent x amount of money and you get y amount of cures.




STEVE USDIN: We're discussing opportunities to improve scientific research with NIH's Kathy Hudson. Dr. Hudson, another thing on this theme of getting more science for the money that's being spent now are clinical trials. NIH hasn't had a great record when it comes to completing clinical trials that it starts to reporting the results of clinical trials, especially reporting negative clinical trials. What are you going to do about that?


DR. KATHY HUDSON: So the issue with reporting clinical trials is not unique to NIH. It's sort of a community-wide problem. And it's one that we are taking very seriously. So it is, in fact, the case presently that our investigators who are conducting clinical trials that a substantial proportion of them have not published their results or made their data public years after the study has been completed.


So of course that means that we don't learn from that experience. It also means that the agreement between the researcher and the research participant has been violated, because what we tell research participants is, even though you may not benefit, somebody will benefit from this knowledge.


And if the knowledge is never out there, then we're really not meeting that ethical obligation. So there is a database at NIH called We are going to be moving forward requiring that people submit their trials, not just our grantees but all clinical trials.


STEVE USDIN: Isn't that the law now already?


DR. KATHY HUDSON: It is the law, and we're going to be putting out some additional regulations that will give some clarity to people about those expectations. And also to our grantees, we're going to make it very clear that if you don't submit your data into, you can expect that there will be substantial limitations on further funding for your institution moving forward.


So institutions are going to have to bring their researchers into compliance with this law. That data will be available, and that means we can learn from the positive outcomes, and we can also learn when things don't go so well and not repeat our mistakes.


STEVE USDIN: And another thing along these same lines is, moving into the era of genomics, there's going to be a lot more genomic research. There's going to be a lot more concern about privacy and the ability to protect the identity of research subjects. What are you doing along those lines?


DR. KATHY HUDSON: So we're really at an interesting point where we are having two values that we want to advance at the same time. One is data sharing. We want to make sure that data is widely shared and widely used so that we get the most bang for our research buck. At the same time, we want to make sure that we're protecting the privacy and confidentiality of people who participate in research.


So we are in the process of enhancing our rules about human subjects protections, making sure that people are asked to give their permission, especially if their data is going to be widely shared, and we want it to be. And then we're putting in place data sharing policies. We've put out a genome data sharing policy telling people what our expectations are so that we can make the maximum use of those sequences that are being derived out there in the labs.


STEVE USDIN: And do you think, again looking kind of at policy and what Congress might do, what government might do, are there new policies that need to be put in place to really to get the most out of genomic information, out of patients contributing that kind of data?


DR. KATHY HUDSON: There are some modest changes in law that would really help us out a lot. There are things that wouldn't seem necessarily important, but in order to adequately protect the privacy of information of research participants, we probably could use some additional tweaks.


We want to make sure that no one has the ability or could be punished if they try to re-identify research subjects, for example. So there are probably some additional tweaks that we could have with legislation that would help us do our research better and help us protect the participants who are so valuable in our research.


STEVE USDIN: And we've got just a few more seconds left. Any other things, kind of low-hanging fruit, that would make a real difference in biomedical research and progress?


DR. KATHY HUDSON: I think having the confidence moving forward that we're going to have stable, reliable, predictable funding path rather than the sort of roller-coaster funding that we've been having will be really beneficial to the entire enterprise.


STEVE USDIN: Thanks very much. That's this week's show. I'd like to thank my guests, Representative Fred Upton, Representative Diana DeGette, and Dr. Kathy Hudson. Next week, we'll continue the discussion with FDA's top drug regulator, Dr. Janet Woodcock, Deerfield Management Partner Jonathan Leff, and Allan Coukell, senior director at the Pew Charitable Trusts.


Remember to share your thoughts about today's show on Twitter. Join the conversation by using the hashtag #BioCenturyTV. I'm Steve Usdin. Thanks for watching.