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Alicia Morgans: Hi, I'm so excited to be here with Dr. Michael Morris of Memorial Sloan Kettering and newly of the NCI, and you're here to talk to me today about a very exciting initiative that's going about between the NCI and the FDA to accelerate drug approvals for cancer patients. Can you tell me a little more about that?

Michael Morris: Sure. I mean, I think that there's recognition in many parts of the whole drug development process, many stakeholders. That drug development for cancer patients can be more efficient and less burdensome and faster as well than the current structures that we have for drug development today. And I think that this is both on the NCI side of things and also on a regulatory side of how to make trials more efficient. We recently have a new NCI director, Dr. Monica Bertagnolli, and she comes with a background as a clinician, as a surgeon, and also as a group chair for Alliance in Clinical Trials in Oncology, one of the cooperative groups under the NCTN. So she brings with her to her new position as the director, a real focus on the clinical side of the NCI's overall mission and on the drug development side.

By the same token, the FDA has a real focus on streamlining the process as well. There are few initiatives to really focus on optimizing the drug development process in cancer.

There's Project Front Runner, which is designed to get drug approvals to the patients who would most benefit from them, often earlier in the disease that many drug approvals happen where it happens now. And also Project Optimist to get a better information on dose and safety and toxicity so that you don't end up moving into advanced trials and not having worked out with the best dose of a drug. Between those initiatives between NCI and FDA, this is really the brainchild of Dr. Bertagnolli to create a new unit in which both groups would work together to really focus on highly innovative, highly impactful trials in which you would have very focused endpoints to really streamline the testing and then regulatory review of drugs in a very accelerated process.

Now, this isn't obviously for every drug or for every type of disease, but there are some scenarios where you really, know what you need to know about a regimen or about a biomarker or some other aspect. You need a focused study to quickly yield the data that is actionable and that is what this unit is really focused on.

So it's a new unit. It's going to be an inter-agency partnership between NCI and FDA with a very open and collaborative spirit for extramural partners. And it's just getting off the ground now, and it's a pretty exciting new component to the national clinical trials infrastructure that we'll have where you really have partnership between the cancer specialists and the regulators who will be designing together or at least facilitating the testing of a really new unit focused on streamlining and a rapid response, rapid yield, for the data that could be actionable.

Alicia Morgans: So I think that's a wonderful thing to conceive of. I'm really excited about it. But I think that as I think about clinical trials, especially in some of the diseases that we commonly treat geo cancers and prostate cancer is one of those that the trials take so long. This unit that you're really developing isn't for all studies as you said.

What kind of studies are the ones that might be accelerated and how do we do this and how do we cut out those things that have become so integral to clinical trials in a way that allows us to answer the question but keep the patient safe. And it just seems like a lot to take on. I'm glad you're in charge.

Michael Morris: I should say I'm a co-director with an NCI colleague, Dr. Sheila Prinival, and the... I think we have to, since we're at the start of this and just formulating what is the perfect study and is the perfect study and what's the definition of innovation and what's the definition of the right kind of trial?

But let me give you an example. First, I would say this is not the right place for first in human studies, early drug development, early biomarker development. But let's say you had approved drugs that are being tested for a new application and you really don't need a re-abrogation of the safety data. These are drugs that you have a vast amount of experience with in other diseases, and you already have some preliminary data that you have efficacy in the question that you're trying to answer.

In that circumstance, you could create a really streamlined clinical trial in which you really focus on the question at hand, and you could forsake or forego a lot of the data that's collected in any study and that frequently in any study is really barely used. There's a lot of data that's collected in most trials that just never gets used, but is collected with the thought that maybe somebody would need them at some point.

CONMED's attribution of clinically insignificant events. I think that that sits wrong with almost every investigator who has to actually complete those. And the research staff that is dedicated to entering those data. But for drugs that you pretty much have efficacy data on, both in the question that's being asked, and in other diseases you have the safety data, the drugs are being used by tens, if not hundreds of thousands of other people to ask a new question of that for a new clinical context, you really just need the answer to that. You don't necessarily need all of the sort of extra data.

There's a SWOG study that's going on that follows that model. It's called... It's the closest thing to a pragmatic trial as an interventional trial could get. It's called Pragmatica. And the whole protocol is around 47 pages. Overall survival is the primary endpoint. It's a lung cancer study of FDA-approved drugs. And they had a very specific question to be asked in a very specific context, but only the context was new. The drugs aren't new. Just the question of whether survival's prolonged in that context is. It's that kind of a study where you could shed a lot of the baggage that usually aggregates. And in that study, there's no lab submission, imaging data submission, CONMED's comorbidities. The control arm is just standard and routine practice. You've done everything that you can to just get at the primary endpoint, which is survival. And that's the idea of the kind of concept that could come through this new unit and the kind of trial that could be developed in that context.

Alicia Morgans: I think that's so exciting. And to your point about these extraneous pieces of data, they can be a huge time sink for all of the staff and all the team members and certainly the patients going through extra assessments. So I'm really glad that you're doing this. Now I wonder, it sounds like the Pragmatica trial is running through SWOG. Is the idea for these trials to run through these cooperative groups that we already have or are they being done elsewhere?

Michael Morris: This is the NCI, and so we do have an existing clinical trials infrastructure. And this is not designed to replace that clinical trial infrastructure, but rather to compliment it. And so you would still have the drug testing mechanisms that we have. They can go through those in accelerated ways, but you wouldn't forsake all of that infrastructure and recreate that. The idea is from a concept standpoint to do that acceleration, an approval and review standpoint, but you do have the existing infrastructure with its expertise. And so you'd want to take the value that's conferred by the existing NCTN and the value that's conferred by this new approach and leverage the strengths of both to deliver a mechanism that's both new and leverages the old as well.

Alicia Morgans: That is very exciting. And so final question, and this is to anyone who's put a trial through Alliance or ECOG or SWOG or any of these groups, it can take a lot of time to go through the review processes within the group, then external groups of experts that review things and then give the okay, and then the protocol is developed after that.

Michael Morris: Mm-hmm.

Alicia Morgans: Does this unit take any of that timeline, which can be years down to a shorter duration.

Michael Morris: That's our intent.

Alicia Morgans: Okay.

Michael Morris: To really streamline that whole review process. And again, this is not for every study. It's not for every question. Most questions that review process and process of buy-in will still have that mechanism to go through. This is for a very specific kind of innovative new non-incremental study in which you want to accelerate that. And then yes, you would accelerate that through the whole review process as well.

Alicia Morgans: Well, that's fantastic. So if you had to sum it up, your new co-director role and the initiative by the NCI and FDA to really bring these trials forward and get drugs to patients, what would your message be?

Michael Morris: I think that across the drug development spectrum between agencies and the government, there is recognition that there is a role for having a really accelerated method of getting drugs to patients who need it and who will most benefit by it as quickly as possible, in which you would say it's not business as usual, and we're going to create this mechanism so that patients get access to the drugs they need as fast as they can.

Alicia Morgans: Well, I think that everyone who's watching, I'm sure, is very excited about this, especially the patients and their loved ones. And I really look forward to hearing updates as time goes on. We'll make this a yearly thing and hear from you how things are going, how trials are completing, and really, I think, encourage everyone that this can be done for those patients who need it most. So thank you so much for your expertise.

Michael Morris: Thank you very much.