Bertagnolli sets out eight “core principles” for NCI: “We’ve got to be nimbler, faster”

Monica Bertagnolli: I am so delighted to be here and see so many friends. I’ve been in this position as NCI director exactly four weeks and two days. It’s been amazing and a little bit like drinking out of the fire hydrant, as I’m sure you know.

I’ve been so grateful for all the colleagues at NCI who are trying to get me up to speed on an incredibly complex and deep organization. So, be patient with me. I’m still very much in the learning phase. But the good thing about that is I’m very much in a listening phase, and that’s a lot of what I want to do here today—share some thoughts with you about some things that I’ve learned, but also hear from you.

I have been a member of the extramural NCI community, and the first thing I thought of on taking on a job like this is to just go back to core principles and think about why we’re all here. Because you can imagine, we all get buffeted by the wind when we’re in our job, and core principles really keep us grounded in what we’re here to do.

So, a couple of thoughts. First of all, right now we have a president, President Biden, who’s made ending cancer as we know it really a signature objective. Wow! That’s an amazing opportunity for all of us who share that goal, and we want to make the very best use of this opportunity. Again, core principles. We’re here to prevent cancer and we’re here to help everyone who has had or has cancer to live full and active lives free from cancer’s harmful effects.

Well, that’s really what everything we do is geared towards. NCI does this by funding research, and also I think critically by partnering with everyone who shares that goal. And see, I can’t do it alone. It has a critical partnering role, and I’m sure you all know this because you are among the biggest partners.

I see NCI as supporting the best people in research and making it possible for them to do this research through the resources that can be provided. And it’s absolutely essential that we engage the brightest minds with the biggest hearts. Because basically, as you all know, ending cancer is a human endeavor as well as a scientific endeavor. And that is also a core principle that I know you share.

So, for many years now, we’ve all heard we’re at an inflection point in cancer research. There’s so much possibility. I’ve had a few people challenge me, “Come on. We’ve been hearing that hype forever. What’s really happened?”

But then I think about when I first started as a brand new surgical oncologist, think back and then fast forward to today, I think all of you can think how dramatically different the world of being a cancer clinician, and hopefully for some being a cancer patient, is today than it was when we first started. I mean, when you think on the long sweep, there’s been amazing progress made.

It’s not enough. We’ve got a lot more to do. But the other thing I believe that has happened is there has been a long-term sustained investment in science, in cancer research, and that has produced some opportunities that we have today that we’ve never had before.

And part of what is before us, and really for CTAC, I think almost more critically for CTAC, is how do we make the very best use of those opportunities that are ahead? 

So, I’ll admit that what we aim to [achieve can be] aspirational, but we need to be. We need to be bold and we really need to go forward with what we know people need us to do. So, a couple of thoughts.

Reinventing clinical trials

First of all, we must modernize clinical trials. We’ve got to be nimbler, faster. I know we’ve been saying this forever, but it is more imperative than ever. And we’ve set a goal to double clinical trial accrual. We can’t leave important work on the table, because we lack the bandwidth to conduct our studies. We have to reinvent how we approach, design, and conduct clinical trials and report our results.

We’ve learned some things from COVID, but there’s a lot more that we can think about. Innovation in clinical trials is absolutely essential. On that note, I really look forward to Dr. [Neal] Meropol and Dr. [Sumithra] Mandrekar’s session at noon today, which focuses on streamlining clinical trials.

I think that’ll be very illuminating for us all and is a wonderful place to start. I think all of you have the knowledge we need to achieve these kinds of goals, and you also know that there are resources needed to make this happen, and that’s why your input is so important. So, I want to highlight some important steps NCI is taking to help realize that goal.

I think one step we’re working toward is launching a survey and evaluation of the representation of underrepresented groups and women across NCI, the NCTN, the NCI Community Oncology Research Program, and the NCI Scientific Steering Committees. Of course, we embrace diversity in our workforce. We embrace diversity in our leadership. This is the best way to serve our patients, and the first step there is making sure we look to ourselves, that we’re adhering to the standards we want to see.

Right now, we really don’t have a lot of information on leadership representation, and this survey will really help us collect the demographic information we need to better understand and make any needed improvements. The plan is to pilot the survey in November, prior to opening the survey to all participants in early January. So, I urge you to participate in that.

Another step is that a week from today, NCI is going to host a summit on increasing diversity, equity, and inclusion in early-phase clinical trials. This has been recognized as an acute need. As you know, early-phase trials can take more resources, can be more difficult, but are so important for patients who can benefit from them. And equity and access to these trials is a very important immediate objective.

The intention is also in this initiative that industry participants will work together to structure partnerships that will improve the representation of medically underserved patients in NCI funded trials. If you want, you can still register for this on cancer.gov and attend virtually. Obviously, we need to continue to invest very heavily in basic and translational research to make sure that we have a steady stream of new approaches to cancer prevention and treatment.

And I also am very happy that I’m speaking to a group focused on clinical trials, as well as translational research. I can’t emphasize enough just how important I personally see basic science as the key to providing the foundational knowledge on which translational science stands. We need this to provide the solutions and build the pipeline for testing. And the gastric and esophageal cancer report you will hear about today also provides, I think, some valuable insights as to why basic science is so important.

Focusing on inequities

NCI can play a very important role in building research resources for basic science and growing the field—for example, in diseases such as gastroesophageal and gastric—that we need to better understand. 

However, as we make these discoveries, we have to also think about equitable delivery of evidence-based cancer prevention and treatment.

And it’s vital that we expand our activity and our research to understand how to overcome factors that cause inequity, and to ensure that our current standards of screening, diagnosis, and treatment are available to everyone.

When we collect and share data, we need to do this in an ethical and responsible manner. Of course, that’s the underpinning of what you all do, but we also need to include efforts to increase collection and use of data, particularly from underrepresented populations.

Research using health data must be conducted with respect to the wishes of the patient, including honoring both patient desired restrictions on data use, or also on a desire on the part of patients to have their data shared as widely as possible. I see these principles in an underlying philosophy for how NCI will approach its support of all data infrastructure and collaboration with our partners to share data broadly.

Our data universe is dramatically different than it was just five years ago. And I will claim to you that we really are at a time when we can begin to truly understand and honor our patients’ wishes.

Finally, we have to increase the diversity of cancer research and care workforce to reflect the communities that we serve. Absolutely essential to building trust, increasing uptake of preventative behaviors, which are so key to reducing mortality, expanding clinical trials participation, and improving the overall quality of patient care, a research area that’s very important to us as well.

Funding for NCI

Of course, we still need funding to achieve the full potential for the cancer moonshot and NCI’s work that extends beyond it. In September, NCI shared its annual plan and budget proposal. 

I don’t know if you saw it. It expressed the funding needed in fiscal year 2024 to begin to realize full use of the opportunities before us. It came out with a pretty big number, $10 billion for funding to the NCI for our work.

NCI has been at cancer research for a long time, since the 1930s. This proposal was based on NCI’s professional judgment of what it will take to best support the National Cancer Program to achieve the current president’s goals to reduce cancer mortality by 50% over the next 25 years, and to end cancer as we know it for everyone. You won’t see a line item in the annual plan specific for moonshot funding.

That’s because these goals are infused into different parts of the entire fiscal year ‘24 budget. And we don’t have this funding yet. In fact, even for 2023, NCI is operating under a continuing resolution that runs through Dec. 16, and that means that our current budget will mirror the fiscal year ‘22 budget, which was $6.9 billion, until Congress can pass a new budget for fiscal year ‘23. In a little bit, MK Holohan will elaborate on the budget and the prospects on Capitol Hill intercession that follows this one.

In the meantime, NCI is doing all that it can, and I’m sure so are you, with the resources, looking to partners to help fully realize the opportunities to advance progress.

Working with ARPA-H

How about another new partner, ARPA-H? I know all of us are wondering what the arrival of this new agency means for our work. I’ve been very grateful to have opportunities to begin working with Renee Wegrzyn, the new director of ARPA-H.

It’s a big job setting up an entire new agency and getting it running, and I can tell you lots of us are giving her many good ideas for how that agency can help us. But just some perspectives, it’s still taking shape, obviously. I can give you some insights into what its intentions are.

First of all, the new agency will provide solutions to issues that impede progress in health care overall. It is intended to be focused, nimble, and very project-oriented. It will engage partners from government, academia, industry, and foundations. And I think most importantly, it is not intended to be a new funder of work that is best achieved in an established organization like NCI.

It’s intended to be able to achieve things that are very difficult for organizations like NCI, but really essential to make progress in health care. So, we already have in NCI and other institutes, in NIH, centers that are well equipped to fund certain types of research vital for progress, and this will not reproduce that.

ARPA-H also intends to fund high-risk activities that really don’t make sense in these other agencies, because they’re just of a different nature.

Think of new tools and technologies that really engage, that are very focused, very milestone driven, and if achieved and deployed, can really help us all deliver care and conduct research in a much more effective way. We are very excited about the prospect of working in ways that complement what ARPA-H achieves, them being a great partner for what we do already, and help provide scientific leadership that will create potential for ARPA-H.

Just so you know the overall approach for this new agency, ARPA-H is following the Defense Projects Research Project Agency, or DARPA, playbook. What this means is that ARPA-H doesn’t want to see 200-page grant proposals with many years worth of preliminary data. That’s not what they’re about.

If you want to see how to write an ARPA-H proposal, it follows the Heilmeier catechism, H-E-I-L-M-E-I-E-R. You can Google it. It’s a set of very specific focused questions that ask you to articulate in a very succinct—just a few pages—manner, what you would like to achieve and what are the core features that define your project. 

Empowering translational science

As we consider our work with partners, another real area of opportunity I see is to work more effectively with our clinical trials partners. You all know this is very near and dear to my heart. 

When you look at the continuum of cancer research, we wouldn’t be anywhere without basic science. That’s the seed bed for innovation.

However, we can’t bring that benefit to patients until someone tests it in ways that it is adequately understood to safely and effectively use in real patients. And I know all of you take on that challenge.

Clinical trials and translational science play a central role in cancer research. Not just because of how it carries the science forward to direct patient benefit, but this is why it’s also the translational community, because of how it powerfully informs basic science and also offers an important guide to public policy. I see in this light the absolutely essential role that the network groups play in the mission of the NCI.

As a former group chair, I’ve seen the combination of scientific excellence, caring for patients, incredible volunteerism, and the spirit of doing whatever it takes with research empowered because it’s being conducted by those who care about the patient sitting in front of them in the clinic. 

I really see it as critical that NCI and the network groups work together closer than ever before to develop solutions that advance progress in key areas. Absolutely critical partnership. 

Together we can leverage the unique skills and resources of all the required partners, and there are many, to fulfill the requirements for efficient research that address the needs of all patients.

Breaking data silos

We can adopt a policy of data sharing to the fullest extent allowed by adherence to ethical principles and the wishes of our patients. 

And we can develop and implement an appropriately resource methods to make data sharing a routine feature of all cancer research. We lock way too much knowledge away in data silos. 

We can establish mutually beneficial collaborations with basic science researchers stronger than ever before to maintain a steady flow of knowledge from the bench to the bedside and back again.

Oh, that’s such an old phrase. But it’s difficult to do. It’s always been challenging, and we really need to redouble those efforts and find the resources and the engaged communities that allow us to have clinical science inform our basic science needs, and vice versa.

Supporting the workforce

Finally, I think this is really important. We need to address the needs of our cancer clinical research workforce. We need to do this by achieving diversity and also by providing adequate support so that our clinicians and researchers aren’t lost to burnout.

We need to support education and reimbursement that make it possible for them to do their work. And finally, by highly valuing the unique contribution of clinicians who support their patients by conducting clinical research. Clinicians who do this on behalf of their patients and everyone else have a very special place in our world.

I’m very certain that this approach will maximize the strengths of the network groups in their central position of translating effective knowledge into better care for patients, and laying the foundation for clinical research that leads to better care overall and informs public policy. 

This puts NCI in the best possible position to support critical work for the people who are counting on us to come through for them, namely all people who are living with cancer today. And the rest, we hope will never have to face that challenge.

So, now I hope to hear from you and I have a few questions for you. Really, the main one, what do you see? What do we need to talk about that are the areas of greatest opportunity, and also your greatest concerns with regard to clinical trials and translational research?

I really would like to start a serious conversation, not ending today, but ongoing for my entire tenure about how we can make the kind of progress that we want against cancer a reality for everyone. So, with that, I’d like to turn this back over to Neal and maybe you can help me moderate some questions. Greatest opportunities, greatest challenges.

Neal Meropol [vice president of research oncology, scientific and clinical lead, clinical research, Flatiron Health]: Sounds great. So, Dr. Bertagnolli, I think I speak on behalf of the entire committee in expressing the excitement for how you have hit the ground running and the focus areas that you’ve expressed. So, I’d like to now open it up to the committee to offer suggestions, discussion topics for Monica.

Patricia Spears [scientific research manager, patient advocate, Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill]: It’s so good to see you in this role, Dr. Bertagnolli. We miss you at the Alliance for sure. But of course I agree with everything you’ve said about your goals and objectives, because I’ve been hearing them a long time and I really think you’re on track. And I think that one of the things, I have several things written down, but a lot of it for us as patients is access.

Access, access, access. So, whether it’s access to clinical trials or access to quality care in general for cancer care, I think that that’s really where it is. And I think the opportunity is what we’ve seen happen with COVID, where a lot of the clinical trials as well as care has been more patient centered rather than institutional centered.

And so, keeping some of those things going that are really advantageous for patients and increase the participation in trials, and the participation in care, and just access to care. So, the equity and care I think is a really huge part of what patients are looking for.

Bertagnolli: I absolutely agree. And COVID taught us a lot because it put pressure on us to think differently. And I don’t think we truly know the implications of the changes that happened with COVID. And like anything, I’m sure some of it’s positive, some of it’s negative. That’s why it’s about research. We just don’t make assumptions. We design research that really tests and understands the risks and benefits of any approach, including some of the new access driven approaches.

I think NCI, I’m proud to say, has an increasing portfolio of implementation science, and really directed toward the healthcare environment and how it can be better. And that’s only going to help clinical trials to do that as well. And then finally, we’ve been saying this forever, the availability of clinical trials and participation in clinical research is a standard of care imperative. The learning health system is what we all need.

So, thanks, Patty. And thanks for your advocacy and all your hard work all these years toward this.

Ruben Mesa [executive director, Mays Cancer Center, UT Health San Antonio MD Anderson]: I think we’re all excited with your expertise in clinical trials and clinical research and what that opportunity brings. As I brainstormed along with Mike List, who is our SWOG PI, as well as leads our CTO for our center, what are some key takeaways we wanted to share?

I think key is we’re at a very challenging point as it relates to both the recruitment and retention of really qualified staff for the conduct of clinical trials. I see many of the faces around the WebEx and know that others are feeling this challenge, as well as the ability to really have our trials be accessible in many areas.

So, a few key things I would really highlight. One, the complexity of our trial designs. I have viewed during my career that the complexity of our trials has exploded to where we now have 10-page-long inclusion criteria for clinical trials.

We must modernize clinical trials. We’ve got to be nimbler, faster. I know we’ve been saying this forever, but it is more imperative than ever. And we’ve set a goal to double clinical trial accrual. We can’t leave important work on the table, because we lack the bandwidth to conduct our studies.

Monica Bertagnolli

This is very counterproductive on many levels. The complexity of the number of correlative studies that are done. I know in terms of federal trials, our team says, again, many of these can really be a tremendous burden in terms of being able to implement these trials. The ability to really use telehealth and tele-consent both for screening and consenting is a huge opportunity. We’re the center for South Texas, and South Texas is the size of Pennsylvania. It’s enormous. So, those remote parts are crucial.

The lack of really qualified coordinators. Again, as we think outside the box as individual institutions, but you think more broadly federally, how do we try to leverage all of these opportunities to create a greater pipeline of people interested in these sort of careers that really help to support our clinical research.

So, I think if we just try to extend our current type of clinical trials more broadly without really thinking about stripping them down to their most basic and crucial elements so that they’re portable, that they’re accessible, that they really are able to be enrolled by the diverse swath of people that really constitute our community, we won’t be successful.

And clearly coming up with ways to support both our staff and as well as our clinical investigators. I think that that is another crucial piece. I’ve seen some new elements that I’ve been involved with the review of the R50s for individuals to be involved in trials. And I think efforts like that to support clinical investigators at academic institutions is key. So, a lot I know on your plate, but certainly [on] standby, ready to roll up our sleeves and help any way we can.

Bertagnolli: Oh, that’s wonderful, Ruben. And let me just respond to this one. We’ve been talking about this for a long time. I mean, certainly as long as I was group chair, we’re saying many of these same things. We got to do something about it. We got to quit talking about it and we got to do something about it.

And I can tell you that one thing for sure is NCI will find a way that we can partner with the clinical trials community, partner with the ones who execute on these studies who not only design them, but then make them happen, partner as never before and solve these problems together. We have to solve this together. Neither side’s going to be able to do it by ourselves and we need to pull in other partners that are going to help us solve this part. Maybe FDA; right? There are other good partners who can help us solve some of these things.

We’re working on a way that we can have a real way of interacting that will allow us to do this problem solving. And I don’t want to forget that a key partner helping to drive this is the advocacy community. The advocates are the ones who are really going to hold our feet to the fire and make sure we start doing better here. And we’ll find the structures, the mechanism to do that because it’s really imperative.

And I don’t see it happening without some serious hard work and also having to make some choices that maybe there’s obviously some choices we’ve just not made and they’re going to be difficult to achieve some of this. We’ll do it together.

Mia Levy [chief medical officer, Foundation Medicine Inc.]: I just wanted to take my one little paragraph, which was I really appreciated the sentiment that you said about volunteering and our passion. I think that represents a lot of the people in this group. And I’ve seen that passion from this group and it really feels wonderful to hear you articulate in such a way. So, I hope you continue to bring that message through in the future when you’re talking to groups.

I’m the chief medical officer of Foundation Medicine and data sharing is a big part of what we are focused on with our collaborations with NCI and others. And we anticipate that the environment for data sharing is going to become a little bit more challenging, more related to social media than to maybe medical.

But one of your goals of being able to learn from the experiences of every patient is hindered by our own lack of clarity around consent and secondary use of data, and how that can move forward. And I hope that in your administration we can continue to make progress in this regard.

It’s very near and dear to both Neal and my hearts. We’re on those subcommittees and I know many others in this group, Adam [Dicker] as well. So, we look forward to working with you on that. But I think we have some regulatory and congressional battles ahead of us that we, as a community, need to stay focused on.

Bertagnolli: Right. We do. We need to partner on this, and Mia, of course, this is a really key feature. Not only here in the budget ask, in the moonshot, and I will take it offline. I’m happy to partner with you on all of this and we know that FMI is currently working with NCI on some really important things, and we’re very grateful for that.

You’re right. Again, we have to do what our patients want us to do, end of story. What our patients want is what we have to do, not all this other stuff. So, we get to work toward that.

Seth Lerner [vice chair for faculty affairs, Beth and Dave Swalm Chair in Urologic Oncology, professor, Scott Department of Urology, Baylor College of Medicine]: I’m Seth Lerner. I’m a urologic oncologist at Baylor in Houston, and I chair the local bladder committee in SWOG, so that’s one of the perspectives I have. But I also chaired the Bladder Task Force and most recently the GU steering committee.

So, as one of the surgeons on this committee, please don’t forget innovation, and surgery, and specific opportunities for surgical trials within the NCTN. I just wanted to share a thought about innovation and translation. And I think that one of the observations that I suspect many of us have are the challenges of doing complex clinical trials within the NCTN.

I think by history, we tend to do sequential trials, building on one to the next. And I think there’s a very rich opportunity for using either multiple disease states within an organ site, cancer, multiple treatments. I guess the model for that is I-SPY, for instance. And really investing and encouraging infrastructure to be able to do these more complex trials that I think are going to tend to move the field forward much quicker.

I’d also like to see, and I guess I just don’t know, having been involved in the TCGA for about a decade and now CPTAC, if there’s specific opportunities to fund translation opportunities into clinical trials from those programs. As you know, it’s just a massive investment, and I think the benefit of those two programs is just off the charts and skyrocketing. And I think in terms of the workforce, you’re probably going to hear this theme over and over again—I’ve been harping on it in that there has to be a financial investment.

We’ve got to be able to compensate people at competitive rates. And every cancer center that I’ve heard from is really struggling with this retention. And obviously, the diversity of the workforce is very critical, but just having a workforce is critical to meet your objective, which we’re all thrilled to hear about and doubling the clinical trials accrual. So, thank you so much for everything that you do and for sharing your time with us this morning.

Michael Knopp [professor of radiology, Department of Radiology, Novartis Chair of Imaging Research, The Ohio State University]: Monica, thank you so much for really bringing a lot of concrete concepts to the overall vision that you are putting on the table. What I just wanted to do is bring three different topics together. Number one, you highlighted really the patient wishes on their participation on the data. And I think we couldn’t agree more, and I think that needs to be reflected better. And it’s really great to hear your passion on this. The second, I think what you are hearing from all of us is the complexity that is becoming a major challenge.

And the third is the opportunity of tying real-world data in addition to clinical trials together. The reason I’m saying it this way, I think there’s a tremendous opportunity that we tap into this potential to tie the patient wishes, the complexity, and also the real-world data together with new concepts, with new approaches that really require the leadership from NCI, also to help us, as Mia pointed out, to get through some of the regulatory, of some of the legal, of some of the privacy pieces.

And I think the more this could be tackled and guided from NCI on a leadership level, that will empower all of us to really deliver on this integration and reduction on complexity.

Adam Dicker [professor and chair, Department of Radiation Oncology, Sidney Kimmel Cancer Center, Thomas Jefferson University]: From a systems perspective, I think there’s a tension between quality of the process and rigor, and working within the silo. And it doesn’t always work well across silos, so if there was an incentivization to work across silos while still maintaining some rigor and quality, I think that would go a long way to reducing some of the energy of activation needed to achieve many of the visions that you talked about.

I think whether it’s a data silo, or for clinical trials, I think there’s concern about, and rightfully so, in terms of that if we’re not as careful, we won’t get the same quality of information, etc. It comes at a risk of trying to do more creative approaches. It also comes at a risk of increasing the time for approvals, etc.

I think that’s a normal tension, and the question is if there was an NCI individual who was designated to reduce barriers, to reduce obstacles and to achieve speed, it’s a different perspective on the various things that we all volunteer on committees, for grants and other types of, especially related to clinical trials.

Carolyn Muller [associate director of clinical research, The Judy Putman Dirks Endowed Professor in Gynecologic Cancer Care, Department of Obstetrics and Gynecology, The University of New Mexico Health Sciences Center]: I’m going to take one additional leap. And just for context, I’m a GYN oncologist and run the minority underserved NCORP in New Mexico. And so, my point is the stakeholders for clinical trials are so many different groups that get pushed out because the culture of clinical trials is not strong. It’s not a priority. Everything else pushes it out.

So, I think one of the things that would be very important moving forward in building that workforce and building the commitments and our communities and our community oncology committees, is to really strengthen the culture of clinical trials, the importance, the career paths for our staffs, the integration of our APPs and other stakeholders, and our hospitals to really recognize that that has to be a priority over billing and revenues and other things generated. I’m not quite sure how to do that, but understanding them for each community I think is going to be critical to really overcoming some of the burdens of doing that. Thank you.

Suresh Ramalingam [executive director, Winship Cancer Institute, Roberto C. Goizueta Chair for Cancer Research, Emory University School of Medicine]: Monica, first of all, congratulations. Very excited and looking forward to working with you. And I’ll bring up this issue about NCTN trials. We all enroll close to 20,000 patients every year and collect tons of bio samples on these patients with all the complexity that comes with that was mentioned earlier.

However, when it comes to using these biospecimens to do biomarker discovery research or other translational work, I believe that there is an opportunity to improve the process. Looking at how quickly can we get these samples to the researchers, how can we lower the bar and not let perfect be the enemy of good when it comes to conducting hypothesis generating work. So, I believe that there are opportunities there and that’s my plea.

Bertagnolli: First of all, thank you so much. Amazing insights. I can really validate everything that was said here today and I could talk a long time. I don’t have time. Let me just end with what I think are the keys to everything we’re doing, and that was the comments about culture. What an incredible treasure we have, the people who are devoted to clinical and translational research. That culture is there because so many of you live it all the time.

I mean, the people are living that all the time. Nurturing that and expanding it and not having it be that valuable culture, be victim of burnout, is a huge priority. Because it really is giving. Everyone who does this work doesn’t do it because it makes life easy for them. Really, it’s this culture of volunteerism. So, we’re very, very keenly aware of that and looking to all of you to help me expand that out. It’s our biggest treasure, frankly.