The Academic Perspective on Real-World Evidence
Flatiron's academic partners share their perspective on partnering with life sciences organizations on health outcomes research, and how RWE can be leveraged to drive greater collaboration.
Neal Meropol: As many of you know, Flatiron shares data freely with some of our academic partners. This enables us to conduct research that is spearheaded by academic investigators, hatched by academic investigators, and allows us to engage in work that might have objectives that are different than the research questions that might be asked by a life science company for example. Through these relationships, we hear from our life science collaborators questions related to the fact that we'd like to work more with academia, we're not quite sure how to find these people. We hear from our academic partners we'd like to work with industry, not sure where the shared goals are and how we might accomplish that.
So, we thought that this panel would be an opportunity to get inside the heads of each other. We're gonna structure this, like I said, as a little bit of a first date kind of exercise, let the academics talk about their goals and aspirations and what makes them tick. Ning is gonna actually give us the reality check, not representing industry at large, but from her perspective, having worked in academia and industry, to keep us honest and engaged in a two-way conversation here.
The ground rules are that I'm gonna start it off by asking some questions, but you guys are empowered to ask each other questions. It doesn't all have to come from me. Challenge each other. Make us squirm, perhaps. Let's do it. We're gonna start off by having everybody introduce who they are, what their roles are, and then we'll get going. Let's do it. Cary.
Cary Gross: I'm Cary Gross. I'm a professor of medicine at Yale School of Medicine. Thank you for inviting me to join you all today. I'm a general internist actually, not an oncologist. However, my main interest is cancer. I lead our cancer outcomes program. My primary interests are around comparative effectiveness. My motivation behind all of this work could probably be summarized very briefly talking about my sister-in-law. Right after we were married about 18 years ago, my sister-in-law Lisa was diagnosed with a glioblastoma. I was down at Hopkins, my wife's family was in the New Haven area. We moved up to New Haven, Yale's a great place, but really to be closer to Lisa and the family. Two observations from that experience. I've really been my true north since then with regard to research.
One, the treatment course that she went through is not the most patient-centered, I'll just summarize it at that. It made me realize that we really have a lot of work to do, not only developing new therapies but also making sure we are being patient centered. Even to the point of thinking about how Lisa would get her MRI scans for surveillance every three months after treatment was a horrifying experience for her because she didn't know heading into these tests, am I going to find out based on this scan that that's it? It's recurred? That's something as a physician I had no idea how terrifying scans were for people.
The other take home point is that she kept getting those scans months, years later. It's now 18 years later. She's still tumor free, with a glioblastoma. So, it can happen. We can actually achieve these miraculous results. So, for me it's all about thinking about how can we be patient centered, how can we have more Lisa's.
Neal Meropol: Ron.
Ronac Mamtani: My name is Ronac Mamtani. A little bit different from what Cary does. I'm a professional basketball player. Just kidding, of course. I'm a genitourinary medical oncologist and a clinical epidemiologist. Our research program tries to use observational data to better understand host factors or patient factors, so medications, comorbidities, smoking, to understand their impact on outcomes and cancer treatment outcomes.
Yu-Ning Wong: My name's Yu-Ning Wong. You'll notice that Ron and I have the same master’s degree. He and I actually have very similar backgrounds. I also was also a GU medical oncologist in practice prior to coming to Janssen, and I also have a degree in clinical epidemiology. In my academic life, like most medical oncologists, I did do some clinical trial work, but I also did a great deal of work looking at treatment decision making and uptake of new treatments. Neal and I did some work studying how patients made choices about their treatments. I also did work with observational data as well. I'm now at Janssen in our internal design and analytics group supporting the U.S. market.
Neal Meropol: Okay, great. One of the things that's pretty interesting about how health outcomes research is done in academia is that this activity resides in a different place, in a different realm in every cancer center, and even to find the experts of people who might be interested in working with you is challenging, so I thought I'd start by asking, this is like the softball question of, where does this expertise reside at Yale and Penn and at Fox Chase, when you were there?
Cary Gross: Our cancer outcomes program is called COPPER. It's all about acronyms. Our COPPER center is actually situated in general medicine, so it would be hard for someone to even know. Why would you look in general medicine? We have collaborators, COPPER members, who are across probably 10 different departments across five schools throughout the University, but our physical home is in general medicine. However, the spiritual home really we try to permeate, for me, throughout the center.
Ronac Mamtani: The really exciting thing about Penn is that it's a matrix campus. So, it's not just a cancer center. We have a center of clinical epidemiology and biostatistics and it's an interdisciplinary and interdepartmental link that really links folks that are interested in population science across the medical school, across the health system, and even the larger community. Just to add to that, in our cancer center, we also have a division of population science, which I'm a part of.
Yu-Ning Wong: Fox Chase also has a division of population science, which is where I sat, but I was also a member of the department of medical oncology. I think, while it sounds like it's just in population science, I think the projects are really driven by who was interested in them, who came up with the research question. So, we worked with medical oncologists, surgeons, the genetics group all drove questions, research projects that you would consider observational research.
Neal Meropol: Outcomes research is obviously evolving in terms of its profile within both the academic world and the regulatory world, the world of research at large. Anybody who has been to the ASCO meeting and attended a session that involved outcomes research or a presentation of real-world evidence knows that they had to leave like a 20 minute gap in order to walk five miles to the farthest reaches of the convention center to find where the health services research session was taking place. And I know, having been in academia for so many years, prospect of clinical trials has a really high profile. Tons of investment, big infrastructure. The health outcomes researchers sort of like in a pocket. Not always a lot of investment, not always a lot of attention, not always a lot of recognition for the importance of the work. My question is, as we've observed as an industry the profile of real-world evidence being raised, has that translated in academia? Do you guys have an office? Administrative support?
Cary Gross: Keep us way in the back.
Neal Meropol: Yeah, right. They feed you.
Cary Gross: It's a good question. For years, decades, there's been this hierarchy of research, this pyramid with randomized trials at the very top. What I've always said is that that's a false hierarchy because the appropriateness of the research question, I don't know if it'd help people in this room, really is what should define your methods. So, a randomized trial is not always the most appropriate study design. I think people are really starting to realize that. They say within academia that change occurs one retirement at a time, like you're not gonna convince people, but I don't think that's true because if you look at the development of new cancer drugs, majority of them nowadays actually don't have a randomized design underlying their pivotal FDA filing. New indications that are added to NCCN guidelines for new treatments or new indications, the vast majority of them not based on randomized evidence. So, I think people are starting to realize that the rest of the world, outside academia, is moving ahead with other, and appropriate, study designs. Frankly, I think academics realize this is the way we're moving and we wanna be part of the solution.
Neal Meropol: Ron, what's been your experience?
Ronac Mamtani: Just to add to what Cary said, it's amazing. As you've heard throughout this summit, observational research, if conducted the right way, can really answer questions that have not and will not be answered by clinical trials. On a personal level, as a medical oncologist treating patients, many of the questions that my patients have and that my colleagues have are not answered by trials. These questions involved comparative effectiveness, treatment safety, treatment sequencing. I think the answer to these questions will bring us closer to what I think is the holy grail in oncology, which is how do you find the right drug for the right patient at the right time. And, I think observational research can deliver that.
Neal Meropol: Are there particular types of research questions that you feel that your perch is ideally suited to answer with regard to observational research, observational data?
Cary Gross: We're all trying to have an impact. I think as far as furthering the acceptance of observational research, not by academics, but just as far as within the scientific community, the real question is how can we have an impact on the way we're caring for patients. I would say that within academia, some of the value added that we can offer is not just around furthering helping to refine research methods or thinking through the ethics of big data, which I think there's a lot that we could collaborate to discuss standards and what are some challenges there, but I think one thing that's really interesting from an academic perspective is that we're not just doing engines of research, we're actually caring for patients every single day. Both patient by patient, but also, at Yale, our cancer center has a distributed network. We care for 40% of patients with cancer in Connecticut. We think of that as a grave responsibility and an honor. That is what is driving our choice of questions, how could we understand what our patients need, but also our choice of studies, to think about how could we innovate around care design as well as innovating around new therapies. Personally, I'm interested in comparative effectiveness, looking at what's the right treatment for the right patient at the right time, but also at understanding how can we define and implement high value care solutions across the whole cancer treatment trajectory.
Neal Meropol: Ron, what do you see as your sweet spot?
Ronac Mamtani: As my mentor would always say, the question is what's the question. So, all of my questions and all of my hypotheses are really generated at the patient level. 10 years ago, my first exposure in oncology was a patient with bladder cancer, who asked could he have developed bladder cancer from a diabetes drug. That launched the question that I tried to answer for the past eight years, do diabetes drugs increase the risk of bladder cancer. It's amazing how far that patient has taken me.
Neal Meropol: Let's move into a discussion of potential opportunities for collaboration and what we might build upon. Ning, as you've observed across the industry, what are the types of questions that either you at Janssen or your colleagues across the industry might seek out academic collaboration to answer? What might be the best scenarios or situations where there are the common objectives?
Yu-Ning Wong: Before I answer that, I actually realized I forgot to say in the beginning that I was asked to say that these are actually my opinions and not necessarily those of Janssen. Just had to say that. I'm gonna say it's a very broad thing. I think anything that involves patient care and how doctors and nurses take care of patients is important, and having academic practicing physician insight is incredibly important. We know a lot about our drugs, as the manufacturers, and we've obviously spent a lot of time studying the diseases that these drugs treat. So, the question should always be obvious. If you have this disease, you should get this drug. But, I think you and I know from our work in studying decision making that there's a lot more that goes into the discussion between a doctor and the patient and then the decision that the patient ultimately makes as to whether or not that patient receives that drug, or the diagnostic test, or the surgery. I think all of us know that we thought we were going into a room to see what we thought would be the easy patient of the day and something derails it and the outcome isn't what we thought it would be. I think that that sort of clinical insight from the ground is really important when designing studies. As Ron said, it was probably the same mentor, the question is what is the question.
Ronac Mamtani: Brian Strom?
Yu-Ning Wong: What?
Ronac Mamtani: Brian Strom.
Yu-Ning Wong: Probably, yes. And Neal taught me that when you're designing your research projects, you should always have your specific aim and your hypothesis and that that's what you work off of. I think the folks in academic medicine can help to shape those so that at the end of the day you design a project that actually answers a question that is important to the disease and the patients. At least, I think that's what we're all trying to do.
Neal Meropol: One of the things that you're alluding to is the timing of involvement. You suggested in the design of the study. From your perspectives, talk a little bit about the interactions you've had with industry support or collaboration around your observational work and what you've found is the optimal time to get involved. What's worked? What hasn't worked?
Ronac Mamtani: To answer the second part of the question first, in terms of timing, I feel like a lot of the students that I work with think that I get most excited about data analysis and getting the results, but actually what I really like is being involved in the study design and the hypothesis and the conceptualization of the cohort, defining the exposures. The devil is in the details for this kind of research, so I wanna be involved really early. And it's nice to have some intellectual ownership. I'll let Cary answer that part and then we can maybe talk about the opportunities.
Cary Gross: Oh, sure. I think when we're trying to develop the question is the key time to get engaged, or really trying to solve a particular problem. We have a wonderful, long standing relationship with Janssen, between Janssen, or Johnson & Johnson, and Yale, called the Yale Open Data Access Project, YODA. I'm a Star Wars fan. That was my big contribution is coming up with the name. Basically, we are facilitating the sharing of clinical trial data. We currently have over 280 Janssen clinical trials available on our website for anyone to submit a proposal, download, use at their will. It's been a really productive collaboration.
Cary Gross: My hope is we can actually foster the same type of approach to sharing observational data in the future, recognizing that all of these data really belong to the patients and to the scientific community and how could we maximize, in the way that Flatiron's doing, ensuring that the scientific community has access to these data. One other thought about...actually, I'll leave it. I'll hold off for the challenges part. But, I think a late in the process type of an approach is less likely to yield a fruitful collaboration, meaning after someone's already designed a whole cost effectiveness study and they approach us and say we wanna look at cost effectiveness of drug X. It's often less interesting to the academic, following up on Richard Pazdur's point yesterday that a dose finding study is not going to get someone tenure. For the academics, they really want to have input with regard to study design, as do all stakeholders.
Ronac Mamtani: Right. In terms of actually working with industry partners, I've had a handful of really exciting experiences, and they've ranged. They've ranged from an investigator initiated real-world study, and example is a comparative effectiveness study of treatment choices in bladder cancer, to even an industry-led study that was a cost effectiveness study looking at treatment choices in bladder cancer. You can see the theme. The most unique experience that I had was actually an FDA mandated tenure cohort study that was funded by PhRMA to look at the relationship between a drug and a cancer.
Yu-Ning Wong: I think, as looking at it from the other perch, we don't know everything about every disease. Clearly, people have expertise in particular aspects. There's a project that I'm working on right now, and it's one that I'm comfortable with clinically, business I don't know the subtleties of the observational research methods, so there was an academic involved early. And I've learned a lot. I've actually enjoyed it. If I read the entire volume of literature, I might have understood the subtleties of the coding, but it was great to hear it from him and get that insight early in the project design so that we didn't have to go backwards and correct things later. I think that's why I would advocate trying to get people engaged early, because you get some interesting and important perspectives that add to your own expertise in the disease.
Neal Meropol: Any challenges? Any uncomfortable aspects about working with industry sponsors/collaborators?
Cary Gross: One partnership has raised issues with regard to timing, because sometimes-
Neal Meropol: Can you name names?
Cary Gross: The challenge is that sometimes universities work on a different timeline than industry with regard to refining protocols, but more importantly the legal issues. In our work with Flatiron, actually I think the EL legal has been pretty good, a month or two turnaround at the most. But, there's been other issues with other partners, where the Yale side is very risk averse.
Their mission is that we have a 300 year-old university, we want it to be there for another 300 years at least, so they're not really worried about whether this project gets done, they're worried about a larger issue. So, sometimes early discussions around some pressure points in the legal agreements can go a long way. But, that's probably been the biggest personal challenge that I've had.
Ronac Mamtani: I have a totally ironic challenge, but before I say it, how many folks here are representing industry, by a show of hands? Okay, pretty much everybody. The challenge that I had was actually identifying you guys. I didn't know how to reach out to an industry collaborator. If I'm interested in a research question and I'm searching for an academic partner, I'll go to PubMed, email them, or call them. Yesterday was the best experience of my life, because we had a...well, I shouldn't say that. Marriage and having a kid first.
Neal Meropol: Number three.
Ronac Mamtani: Yesterday-
Cary Gross: Scroll back 10 seconds.
Ronac Mamtani: We had a real-world strategy breakout session, and for the first time, I see the directors of the centers of observational data for each major pharmaceutical, and it was amazing. I wrote down their names. I tried to speak with them. And, I'd love to reach out. In contrast to clinical trials, I know all the medical liaisons for bladder cancer. If I wanna run a bladder cancer or urologic cancer trial, I can contact them and they make themselves available. I find it's not so much the case with industry partners, except Ning maybe.
Yu-Ning Wong: I think for us, it's also important when we're choosing who's the right person to work with. I think it's understanding people's clinical expertise, their methodological expertise, what they've worked on in the past. I think when we're looking for the advice, we should really try to focus on then who's gonna help us in the most effective way. One thing I've learned since coming to Janssen is timelines are very important. It's not the timelines in academics aren't important, but they're very, very important in industry, so a lot of it is planning. As you said, you can have contracting issues. Being able to anticipate these issues ahead of time as you're trying to identify who you wanna work for and where those pain points may be I think is really important.
Neal Meropol: Back in the day, an industry dollar was worth like 35 cents in the academic esteem promotion how your work was viewed broadly, and even in terms of trying to get it published, in terms of what the sponsorship was. Where are things today? What's the thermostat around different types of funding sources and whether there are barriers?
Cary Gross: I'm on the promotions committee, so I've had a good experience learning how people think within academia. Within the world of research funding, the NIH RO1 grant is still the coin of the realm. When someone's coming up for promotion, they want them to have had at least one, hopefully more than one, but at least one big NIH grant. However, that does not mean that any other type of funding is not valued, because the most important thing, and we talk about this in the committee meetings every two weeks, it's all about impact.
What has this person done to really change the way that we're providing healthcare, to generate new knowledge, or in some cases furthering medical educational approaches, whatever it is, are they having an impact? I would argue that we, as academics, can't have an impact without really fostering successful collaborations with industry because that's where so much of the innovation and brainpower and energy reside. So, the ROI is valued, but I think that this idea of the people turning, stubbing their nose, or whatever the analogy is, at the industry dollar, those days are behind us because we're appreciating the importance of impact.
Neal Meropol: Ron, you're a former journal editor. When you get papers, do you care what the sponsorship is in terms of how your reviewers are reviewing things and how you make your editorial decisions?
Ronac Mamtani: I think that source of funding to signal the virtue of an analysis is lazy and a poor predictor of quality, so we don't pay as much attention to that. From the university perspective, I haven't been promoted yet, but I don't know how our promotions committee views these things. Maybe that's why I'm not promoted.
For me, funding is funding. I need to write grants, and it is exciting but it's totally demoralizing, and I need to get funding to support my research and to limit my clinical responsibility. I love taking care of patients, but I cannot take care of patients every day if I wanna be a successful researcher. There are many kinds of grants. Luckily, I'm on an NIH grant. It's a K23 career development award. But, the cycle doesn't end. So, I think the pendulum is probably swinging towards alternative funding mechanisms, like industry, like private foundation. That's my perspective, but I have to keep writing grants to keep this process going.
Neal Meropol: Ning, let's help Ron get promoted, okay? We don't wanna see him back on the basketball court.
Ronac Mamtani: Right.
Yu-Ning Wong: No, we don't.
Neal Meropol: What are the types of research projects, broad categories, what's the sweet spot where this kind of collaboration could be the most successful?
Yu-Ning Wong: I think understanding how the drugs are used. What populations are getting them. There's questions, I think, that arise in clinical practice, populations that aren't studied in clinical trials. We've touched on some of them today. The elderly, the comorbid. But, decisions you have to make every day in real life. Your creatine isn't right or your LFTs were off. What is the right dose to use? Those are the types of questions that I think have an impact in clinical care, and those are the types of studies that can be very helpful and complementing with clinical trial data. There's a lot of interest in care delivery, which is happening right in front of us now, understanding what is the most effective way to get our drugs to the right patients at the right time, as Ron said. I think those are all issues that are really important to study.
Neal Meropol: Cary, what sorts of work that you do, do you think are best suited for a partnership with an industry sponsor?
Cary Gross: As a generalist, I have a long standing interest in comorbidity in cancer and aging in cancer, especially thinking of someone like Arti Hurria, a national leader in geriatric oncology. One thing that we've been thinking a lot about is this effect modification, age as opposed to treatment effect for all these new, wonderful agents that are coming to market. So, I would love to explore not only patterns of care, but the impact of this heterogeneity of treatment effect on care and value. Also, we're starting a new initiative, this cancer care innovations lab, where we're really trying to understand how to best redesign healthcare to reduce acute care use and improve patient-centered outcomes, and eventually improve informed consent.
How wonderful would it be if we could actually have patients come into the clinic and they could ask the question what does this mean for a patient like me and we can say to them because you're 78 years old, with emphysema and heart failure, this is your likelihood of having a significant adverse effect, this is the potential benefit to you for this treatment, this is what it's gonna cost you out of pocket, these are the care burdens it will impose upon you, all those things. I would love to really radically redesign our informed consent process, and if there's a way that we can use that so it's using up to date evidence and being informed by patients in how we're doing that would be wonderful.
Neal Meropol: Ron?
Ronac Mamtani: In terms of what question is best suited for collaboration, I think Ning's point is really important, and that is we're not trying to replace clinical trials, we're trying to compliment them. Trials are notoriously underpowered to address safety. There's all this talk about efficacy, but there's very little talk about drug safety and adverse events. That might be the sweet spot, long-term safety and post-marketing pharmacovigilance studies. I would be very interested in that.
Neal Meropol: Just to follow through on that, how do you think that those might be best set up? With prospective consent, with passive reading of electronic records? How do you think we can accomplish this?
Ronac Mamtani: I think the most efficient design would probably involve a retrospective analysis. I think your prospective idea is interesting, but I think it might be more timely, assuming you have enough follow-up data, to analyze this retrospectively. I think that's how I would approach that.
Cary Gross: Moving ahead, wouldn't it be exciting if we could, following up on some of the things Amy was discussing yesterday, wouldn't it be great if we could embed randomization into clinical care in a more seamless manner so that we could, if there are two tried and true treatments that are available. We have these treatment pathways, so other products are out there that are telling the docs this is what you should do for this patient. Why not just have these are the two options. We don't know which is better. Offer this patient randomization. And when the doc clicks no, have the box appear again. Are you sure? Because 70% of kids with cancer, roughly, are enrolled in clinical trials, whereas, what is it? 3% or 4%, 5% of adults. I'm an observational study person, so I'm not gonna detract from the idea of doing more observational studies, but I really think we need to ramp up our creativity and our urgency around our approaches to innovating around randomized trial design.
Neal Meropol: Cool. We actually have some time for Q&A. I didn't warn you ahead of time that your juices should be flowing, but if anyone has questions for the panel, to put them on the spot, please signal. We've got somebody. We've got a volunteer.
Anu Kalsekar: Hi. Anu Kalsekar, Bristol-Myers Squibb. My question is regarding data. Both of you mentioned patient care and access to applying your research to patients as a motivator for you. One of the needs on the industry front sometimes is building data infrastructure. While we have large datasets that are like the Flatiron dataset available through EHR for some diseases, that's not the case for all. Have you considered any partnerships with industry around building the data infrastructure?
Ronac Mamtani: I haven't considered that, but I think that's a novel idea. I think that's a unique strength from your perspective. We have many existing datasets, sorry Neal, not just Flatiron. There's many datasets. Flatiron may be a great dataset, but there are strengths and limitations. The National Cancer Database, CMS data. But, I like your idea, and I'd like to hear more.
Cary Gross: One thought was-
Anu Kalsekar: Sure, we'll connect.
Cary Gross: One thought with regard to building local data infrastructures is we've been working on a PRO registry. For years, most academic centers have been collecting tissue, and we're doing a better job of getting consent so that there are these genomic registries. As the generalist, I always wanna know what about comorbidity or what about patient reported outcomes? Integrating PROs into clinical care is really hard. Again, we figured out the technology. We haven't figured out the patient engagement side. Because patients often don't wanna fill out a form every week. So, I think there's a lot of room to innovate there. If we do it right, patients will readily see that it's helping them, and if we do that, then they'll fill out the forms. So, I think that's a key, for us, my perspective, a key to the next step.
Neal Meropol: Let me push you a little bit on that Cary. What do you think it will take? As a company, we passively collect data that are collected for other reasons. What do you think it will take to make patient reported outcomes a component of standard care? In essence, what is the evidence required to make this a necessary part of what doctors do in routine care?
Cary Gross: That's hard, because Ethan Basch has already done these wonderful studies, where they have shown Ethan a randomized group of patients, late stage cancer. Half of them were getting routine symptom assessment and then if they have a symptom, a nurse was told. They called the patient and then tried to manage the symptom. This is a randomized trial. The other people didn't have routine symptom assessment. They found the symptom assessment group had less side effects, better symptom control, as you would expect, more likely to repeat their chemotherapy course, as you would expect because you're controlling their symptoms, and a five month survival benefit. Because, if you assume that their treatments are good for them and they're able to complete them.
Cary Gross: So, all biologically plausible. But, think about that. By measuring PROs, five month survival benefit, and it's still not being adopted. I think what we need is an investment on the clinical side, in the infrastructure, to figure out the IT. Also, it's the same thing. It's the difference in fidelity to an intervention in a clinical trial setting versus the real world. We have to figure out how to let patients access their own data as they fill out their symptoms, and see graphs. We all have these, this is not one, but we all have these Fitbits and exciting watches. People love their own data. If there's a way that we can allow people to see how they're doing and then make that part of their conversation with their doc, that would help a lot.
Yu-Ning Wong: I think that-
Ronac Mamtani: I was gonna say I think PROs need to be embedded in the electronic medical record. Take, for example, the most important prognostic factor in all of oncology, which is performance status, is still missing in the medical record system. That's outrageous. At Penn, sometimes I forget to record performance status. So, I don't know if we're ready yet.
Yu-Ning Wong: I think we also, this is my clinician hat speaking, is that we also need to know how to react. What do you do if your FACT-P score goes up or goes down? How do the nurses and the physicians react to these scores? One thing I think most clinicians will say is that they’re on alert overload. So, we don't want every subtle change, but we have to understand what are the meaningful changes that the clinicians and the patients and their families should be aware of, and how to influence care.
Neal Meropol: Yep? Other questions?
Janice Lopez: Hi, I'm Janice Lopez and I'm from Janssen, and I promise I'm not a plant. I am curious. You mentioned that, in the discussion earlier, disparity in timelines between academic and life sciences organizations, as well as the challenge that you have of finding industry partners to engage and to collaborate with. I promise you, it's not as easy for us either, as looking on PubMed to find who has expertise, but also who has interest as well as bandwidth to engage with us.
What recommendations, or at least what has worked for you with respect to getting closer to a match, as far as timeframes, working with an industry partner, as well as what do you recommend for us to find the best academic collaborator?
Cary Gross: I think the best analogy I would make would be to think about community-based participatory research. There's this trend in research in general where researchers are going out into the communities, building long-standing relationships, building trust, sharing ideas. I think we need to do that with academics and industry in the sense of I think we need to have some kind of an ongoing conversation. A working group. A way for people to share ideas and brainstorm and get to know each other so they can have a space to discuss ideas over time. It is challenging.
Yu-Ning Wong: At academic meetings, do you find yourself, at ASCO, or I don't know if you go to ISPOR, do you find yourself interacting with the industry groups as much as with other academics?
Cary Gross: Just other academics. If there's a way to change that...
Ronac Mamtani: The timeline issue, I have to tell you, it's a blessing to work with a short timeline as long as I have funding, because then I could get other people that help me with the programming. With NIH grants, you propose a grant, then two years later you get it. The research question's already been answered.
Jim: Hi, I'm Jinghua He from Merck. I have a question about how you think the academia relationship with FDA or regulatory authority. From industry, you have close collaboration with both sides. However, we are not clear how you actually contribute or interact with regulatory authorities. We talk a lot about real-world evidence and the regulatory-grade and I don't think there will be any significant breakthrough until FDA really endorses real-world evidence as regulatory-grade. Apparently, all you guys have a very positive attitude towards regulatory adopting the real-world evidence toward their future approval, et cetera. I wonder if there is anything you think you can do or you want regulatory to do to interact with academia to speed up this process?
Cary Gross: I can tell you from our experience at Yale, there is a partnership with the FDA. The FDA put out a request for proposals because, again, I think they're recognizing the need to foster those collaborations. It's CERSI, clinical epi research something. The idea is that there are 10 of these centers, and we're one of them, and the FDA is serving as a matchmaker between the universities and industry to actually do real-world evidence studies. The FDA, in one of their many new, wise, fast moving initiatives, I think they're doing a great job overall, they're trying to solve that exact problem through this CERSI program.
Ronac Mamtani: And definitely there's progress being made. Neal and I were talking, 10 years ago it would've been unfathomable to have a synthetic control arm or real-world control arm in drug approval process, as you heard with the leukemia talk. So, clearly, progress is being made.
November 7, 2018
- Neal Meropol, MD — Vice President, Research Oncology at Flatiron Health
- Cary Gross, MD — Professor of Medicine; Director, National Clinician Scholars Program at Yale
- Ron Mamtani, MD, MSCE — Assistant Professor of Medicine at the Hospital of the University of Pennsylvania
- Yu-Ning Wong, MD, MSCE — Clinical and Analytics Leader at Janssen Scientific Affairs