Working In Oncology
Working In Oncology

Episode · 1 year ago

Using Expanded Access & “Right to Try” for Experimental Drugs w/ Ajeet Gajra

ABOUT THIS EPISODE

For patients who have run out of approved treatment options, hopes of fighting disease can feel diminished. Fortunately, through existing and new programs, patients have access to alternative drugs. Educating physicians more on the program availability can lead to an increase in options for patients and a more positive outlook to recovery.

Dr. Ajeet Gajra, Chief Medical Officer at Cardinal Health Specialty Solutions, joins the show to discuss experimental oncology drug use through the Expanded Access Program & the Right To Try Law.  

Here are the show highlights: 

- Expanded Access Program vs. The Right To Try Law

- Hesitancy & Barriers to Use Programs

- First Steps & How To Stay Up-To-Date on Programs

- Tips for Office Staff w/ These Programs

- Success & Future Use of Drugs 

Check out these resources we mentioned during the podcast:

- Dr. Gajra’s LinkedIn (https://www.linkedin.com/in/ajeet-gajra-md-mbbs-facp-77059748/

- Dr. Gajra’s Research (https://ascopubs.org/doi/full/10.1200/OP.20.00569)

- Cardinal Health (https://www.cardinalhealth.com/en.html)

 

To hear more interviews like this one, subscribe to the Working In Oncology Podcast on Apple Podcasts, Spotify, or your preferred podcast platform.

You're listening to working in oncology, a podcast and video show that spotlights oncology practice staff and industry influencers who work behind the scenes to shape the future of oncology. The more knowledge the oncology community shares with each other, the more we all grow. Let's get into the show. Welcome to working in oncology. I'm your codes for the day, Alicia Evans. I'm joined today by Dr a Jeep Gaza, chief medical officer at Cardinal Health Specialty Solutions and Co author of a recent study published in JCO oncology exploring request for access to experimental drugs. Dr Gayja, we thank you for coming to the show today and welcome Policia. Thank you so much for having me. I'm happy to be here and very eager to talk about this important, but often ignored at topic. Absolutely so. I've been really looking forward to our conversation. Your study was pretty eye opening and I think you can really help kind of fill in some knowledge gaps today that will help our listeners really expand their patients access, potentially to experimental drugs. Sure. So, before we jump in the conversation, can you tell our listeners and viewers a little bit more about you, your background and what you do. Surely she has. So in my current role I serve as a chief medical officer. He had Cardinal Health specialty solutions and I've been here over two years now, but at heart time a clinical, you know, medical oncologist and hematologist, and the start of the journey was back and there was combating myself back in the late s when I chose the field of hematology oncology to pursue after intermadicine training, because I always thought, you know, oncology is the ultimate barrier, sort of, and you know, other substance written about the cancer being the ember of all maladies and I felt like that that that is so true at or at least was so true to me back in the day. So, you know, this field has been very gratifying because it's afforded to me my three loves, which is clinical research, education and, of course, patient care, which supposed to my heart. And I was able to serve bed rooms by working part time at the vmatical center. I've spent most of my time in academic practice, though, until about two thousand and seventeen, when I made a transition to industry focused on clinical research and then here cardinal continuing that kind of work. That seems like you've had kind of an extensive oncology, kind of range of experiences. Certainly, and you know, at the the clinical practice, even though it was an academic practice, I had the opportunity to work in a community setting, in a large Tertiary Care Cancer Center and, like I said, also in a different system, of which the BEA system is a unique system. So I had the benefit of all three and in you know,...

...being able to compare and contrast almost with the good bad of each of those. So, before we jump into what we want to talk about today, is your recent research on the use of expanded access and right to try requests, and I hope you think, sir, ups in this conception about the two programs and maybe discuss maybe the INS and outs of each short. Yes, so you know, this is interesting because while in practice, and currently I'm not in active practice, I see patients were in a variant the basis. But you know, I had the opportunity to be exposed to and work with the expanded access program myself and in fact, the first time I used it for a patient who had a rare form of lung cancer would run through all of their treatment options but, you know, the patient in the family were still wanted to try something else. And so, looking through the clinical trials website, I found that there was a drug which was in being tested but was not a fe approved, not available. So then I was able to reach out to the manufacturer who was willing to, you know, give out the drug from this patient. And but we had to jump through several hoops. So we had to first and foremost, we get to learns from our Institutional Review Board or a protection of, you know, patients best interest, and then they to submit a request to the FDA. So this in effect sums up the expanded access program where a drug which is available it may also be used in other settings, but can be tried in an indication or in a dislease where it's not currently approved, but you have to get your IRB approval any up to get an FDA sign off. And and then, and of course, the manufacture, the by form. A company has to be willing to to offer this drug to the patient. Of course there's an informed consent process. So this, if fan and NASIS program has been in parlance, has been in use since the late s and one thousand nine hundred and eighty seven, I believe, and has helped literally hundreds of thousands of patients gain access to agents outside of a clinical trial. You know that they might not have had access to but again, you know, if you're a busy provider, as many of our listeners might be, it seems daunting for our singlication and I have to go to my Irb, I have to go to the FDA this, this seems like just don't have the time to do it, but it is a very feasible as doable and of course, once you learn how to do it and the more support you have in your institutional practice, the better you can do it. Now fast forward to two thousand and eighteen. A new methodology or new process, new pathway, was signed into law called the right to try, and the difference between expanded access and right to Trib really is that would write to try. You don't have to go to the IRB, you don't have to go through the FDA sign up, so obviously it's a much abbreviated process. However, however, the manufacturer still has to be willing to provide the drug and also there are very clear...

...and strict criteria for what patient, what individual, can qualify for right to try. So for that, really three things happen to be mad in terms of their disease. So they have to have a life threatening disease. They have to have used up all available and FB approved therapies. They cannot, you know, they should not either qualify or clic or trial or should not have access to one in so and of course they still have to provide written informed consent. So those are sort of the patient essentialities in to qualify for a right to try. But then there's also the other conditions that have to be met, which are for the drug, you know. So number one, phase one studies should have been completed. It's because we want to make sure that we at least have a dose which has been identified and you know, that safety has been assessed in you know, it's proven to be safe for use in humans. So face one testing should have been done. This agent, if anyone's going to apply for right to try, should not, could not have been approved or licensed by the FDA for any use. So it's not like okay, I have this drug works in lung cancer. Can I use in press cancer? Not. If it's FB approved, already when you cannot access it by right to try. And the third is that, you know, the manufacturers should have already filed an I and the application with the FBA, which of course is standard. But out there cannot be a clinical home. So sometimes the FDA will notice that, oh, there's maybe a problem or maybe a safety signal or some concern with an agent as it's being developed, and so they might put a clinical whole on the I and d. So again, you know. So if there's a clinical hole, then that drug cannot be accessed through right to try. And again it's the safeguard the patient's interest, because the FDS put a hold on it for a reason and for safety or some of the concern and so then we don't want to unnoyingly expose a patient to such arms tin. No long answer, but that's an overview of these two expended access and right to try it. There are some other terms that come up sometimes. Compassionate use. Yes, so you know, you bring up a very valid point, police her. So Compassionate use has been long used, quite honestly, to describe the expanded access program because what we were doing through that was helping our and still are is and when I say we, I mean as on college dis as fellow oncologist. It is a method off getting access to a drug which for which is not approved or indicated by the label in in a given patients condition. So if a drug is approved for I'll give you an example. You know when you're on collegeist and Alfa for listeners, know that you know neighbors, friends, family all reach out to you. So you know there's someone that reached out. Unfortunately, they had, they have a brain tumor, they have a brain cancer and you ustill and they have, you know, already used up the two, two, three standard frequent options, and so they really...

...want to try Meuno therapy. So you know. But you mean therapy is expensive and it's not approved for glioblestiment. So so they are obtaining this immuno therapy medication then expanded access programs. So in a way this is compassionate use, but because the manufacturers willing to provide that, but right to try, in a way is also a form of compassion use. So I guess my point is that compassion of use is a broad term utilize for off label, which is another term use of these drugs which are not approved by the FDA for a given disease. But again, they have to be provided by the manufacturer and I think that is a part of the compassion, the Compassionate uses coming from that, you know, the manufactures willing to provide access to these agents. Okay, and so this kind of brings us back to your research. And I mean we've already established that these terms are sometimes use interchangeably mistakenly. In your research. I was very surprised to find that such a small percentage of providers had attempted the right to try pathway. Yes, at least you know. So that was a part of the reason why we conducted this. So it had in my role here at Cardinal, I support also one of the divisions called a cardinal help regulatory sciences. So there's a whole, you know, Great Team of scientists and pharmacists and other regulatory specialists and that division that work with FDA and other agencies to get a drug from the Preiind I ind to you know, final approval stage. So anyway, so, as you pointed out, there's even in people who are, you know, very well worst with this work. They can sometimes be confusion in terms of off label compassionate use and then EP expanded access and right to try. So through our summits, so cardinal conduct summits meetings of providers, physicians, but also sometimes apps like counties and PS. But this story was directed towards positions to assess if they understand the differences between a right to try and expanded access. Now, even though we conducted this late two thousand and nineteen to early two thousand and twenty, you know perhaps we were now even thinking about publishing this. But then, unfortunately, as we all know, the pandemic happened and, as we know, the access to medication, in this case anti viral therapy or what was hoped to be undervit therapy. You know that we heard so much about compassionate use of the area's drugs for covid nineteen and so that made us think like, oh, we're I mean, we had the information already. So in fact that was a reason to for us to go ahead and publish our findings, report our findings, because you know some on Cologists, you know that this is the director towards oncology drugs. But we...

...also, in our work and our research, realize that, you know, this has been done for community based on cologists in the past and really those for the reasons to report this in JC or on college practice. So it's summaries. What you're researching, brew of Research Rather, is kind of an assessment of physicians knowledge of these programs and their experience with each of these programs, exactly the knowledge and utilization. So do they know about this? Have the utilize it? And we also wanted to ask them if they utilize it, what is their success rate? Are they able to gain access with the drug that they requested or not? So and again, you know, in a spiritful discussion, we say that clearly in our paper. This is simply based on a survey or on recall. So it's not like we're asking them for, you know, specific evidence, like I'll show me the number of times you've did this, but you know what was their overall impression? Yeah, and so on the results. I see that about forty six of your physicians, forty six percent, excuse me, had actually attempted the EA program while really surprising, the only fourteen percent tried the right to try program or attempted yes, at LEASTA. So I mean part of it is because expanded acts has been there for so long. You know. It's therefore literally dozens of years to time, eighty seven, whereas right to try is very new. I will say they'll. Right to try has garnered a lot of media attention, even while it was a bill and it was being discussed on the floor of the Congress and to the time that it was signed into law. There's been a lot of media attention on this, but you'll write it. Despite that, it was a little surprising that less than half of the physicians had access for tried to utilize EAP, which isn't therefore, you know whatever, forty years or thirty five years and only fourteen percent, like you said, had attempted to use the right to try, and so you think that some of it is attributed to right to try being just the kind of a newer program correct. And it seemed that the success rates were slightly bigger, of course, for expanded access, since you had more more attempted than the right to try. Zecht yes, you know, remarkable success rate. So for those that used expanded access, almost ninety percent, or eighty nine percent, were successful in gaining in a get gaining access to the drug that they requests, and this is pretty impressive. And then with right to try, was slightly though. It was someone Lord Seventy three percent, but still not not, you know, something to ignore. It wasn't. It's a remarkable that three fourth of the time you're successful. And Mind you, neither off these programs, neither the EAP nor the right to try, mandate the manufacturers to give out, you know, the drug that's requests. It's completely up to the manufacturer whether they want to give out, to give this drug out or not. But you know, if you're getting access to a drug ninety...

...percent of the time, that's quite in a mark of statistic in my mind. It seems remarkable to me. So what do you think are some barriers? Is it just a knowledge, or is the earth these programs difficult to access? I think you're right up both front. So what our takeaway was, you know, so we had some questions varied in our survey to assass if the physicians are able to distinguish between the two programs, and only about a fourth of them. So twenty seven percent of them correctly identify that for right to try, you biopass the IRB and you bypass the FBA. So so you know so the vast majority actually didn't realize that that was a major difference. The other thing that we asked to assess, you know, to gage their knowledge in awareness, was for the expanded access, which requires FDA sign off, I would say, or Fba to view. You know, how how often do you think FTBA signs off or approves and expanded access request? And only three percent came up with the correct answer. So, believe it or not, the FDA has approved ninety nine percent of the requests for expanded access and only three percent hours plans actually, you know, give us the right answer that over, ninety five percent of the time you have the approve. That so, which again tells you that there's just lack of awareness. And then we also ask them, you know how often you know, how many patients do you think? And Mind you, this was only a year into right to try, because the seray started in the fall of two thousand and nineteen. Where's the right to try? Legislation was passed in two thousand and eighteen. So we asked them, you know, how many patients do you think thus far have benefited from right to try? And again, you know, only about eleven percent were able to guess the correct answer. So there's definitely, you know, some lack of understanding and you brought up valuable terms in terms of anthing. It August modeled sometimes in people's heads. But also the process has been daunting for expanded access, and we'd write to Quich mouths. It's much simpler. Oh really, so it's the so expanded access will require more support. Correct, then, COR right to try program okay, because you would have to Irb and you have your own irb sign off now to, you know, facilitate expanded access use and act and utilization. You have to. It actually made it much simpler in the past. Up under two thousand and fifteen, I believe, you know, for expanded access the entire IRB, so the entire board have to approve and expanded access request. But to simplify because a lot of times, you know, these requests are coming in in dire circumstances or in you know, when unfortunately a patient has limited time. Perhaps so. So the FT modify that guidance and said that expanded access going to be granted by one IRB member. So you know, if you were want to access something urgently, even a one single...

IAR. Remember, I can authorize or approve in expanded access request. But still there's multiple staps. There's Irb, then going to the FDA. The FD applications long, I'll tell you. I've done in myself. But but you know, the reassoring thing is they do respond very quickly. The median time to response, I think it's three to four days at the FDA and, like I said, they do a promajority. They have a pro majority of the request. Of course, you know the physician, like all of our listeners, you have to make sure that you know they are trying to do the right thing for the patient. You know, we don't want to just throw something and see if it sticks. But if there's some scientific rash now and it makes sense, then it's definitely worth pursuing. So if I read, how do I read? Sorry, how do our listeners begin the process? So let's start with expanded access. What we would be the first step to take right? So again, even for expanded access, you know, suppose we have a patient who unfortunately is progressing through available treatment options, but you know you and maybe there's an ongoing to incod trial, but the patient may not qualify because of certain online conditions. Or they may just not be able to travel to the far off institution to participate in such and generally, most on collegists are aware of other agents or which are being attested in a given disease. Other way, they can use resources like clinical trials dog out that lists all of the trials I'm going in a dilease, and so if you identify a drug or you know that may potentially benefit your patient. Also, the patient has to be in reasonable condition. If someone's very in very, you know, very frail, or that what we say performance status is very poor, you know that may not be the best candidate because at the end of the day we don't want to harm the patient. But there are some patients work, you know, in reasonable performance status and some of them can be a bust. So it's very simple. I would first start with the making out preacher the manufacturer and saying I have such a such patient and you know, might I have access under expanded access? By the way, there's no legislation that requires manufacturers to list on their website whether they offer expanded access, you know, program or not, and you know by making an outreach to them. So they may say yes. If that is said then that's my green light. I go to my irb with my case history and say here's the circumstance, here's the data for the drug. Would you approve this? Assuming they approved that, simultaneously, I will complete my whatever it is, six page FT application and send that packet off with my irb approval and, you know, redacted patient history and why it's appropriate for that patient. I would send that to the FDA. Expect a response within a week and then with that, with those three things, I will go to the manufact sure and, like I said, I've been through this myself several times, and then you get your shipment. Of course there still has to be an informed consent for the patient because you're giving them an agent that we...

...don't know how they might react to. But that's the job the RB to make sure that you have a proper informed consent of the in place. So and you know, then you start. So that would be the EAP program right to try. And you know, I had sort of left full time practice by the time this legislation came out, so I can't play. Cannot claim to have the first time experience with this. But again here I think very often what I'm noticing is that it's more driven by the patient. But the patient cannot do with themselves because they have the physician and even the FDA guidance as that the physician is the best person to decide, you know, whether this drug maybe beneficial or that. You know, some benefits may outweigh harms, because it's not like every person has the right to try any drug or anything. So so you know, the drug already should be purposed, even though not approved, you know, for their condition and like we went all the criteria before, and really the application on the FEU website is quite simple. But again the manufacturer can be mannated and again my suggestion will be each other manufacturer for us ask if they're willing to share the drug, you know, through a right to try and mechanism. If they deny, then it's not worth while going through the extra stops. And sometimes you'll come across a manufacturers say, you know, we cannot do right to try, but we can do expanded access. So so you know, then you might have to take the longer pathway by you just to have access to the agent. Okay, so for both approaches you would go to the manufacturer first. I would I think that that way. Yeah, that would streamline your efforts and that way you're not trying to, you know, walk on a path that's going to lead to nowhere. And it sounds like right to try, especially because there's been kind of recent press and it's kind of a new, new process. These are often patient initiated requests, very to releasious. So you'll see a lot of stories and some of them, of course, very heartrending. But, you know, advocacy, and especially when there are children involved in such and especially with red diseases. I mean, you know, here we're talking about cancer, but then there are other diseases. There's muscular dystrophy, there's spineless atrophy, all kinds of other neological and other conditions that predominantly affect children or even newborns, and you see a lot of stories, you know, of parents trying to make this kind of outreach. So but yes, you know, a lot of times it is, I find, patient of the family's sort of driving this. But then the physician and the team has to support them also guide them, you know, what may do, what may not be appropriate. The cool of course, of course the physician needs to be as deeply involved in their process and the whole actually the whole care team really and these decisions. So our ecology office staff is very busy. Things are always moving, it's always changing parts. How can they stay up to date on these processes? What do you recommend right? So I...

...feel as though you know somebody easier for practices and physicians and teams and, you know, offices which are already involved in research. I think that makes the process a lot simpler because many of these steps you're going for participation in clinical trials anyway now with right to try. I don't believe that you can do that for every patient every time, but they can be select circumstances where it makes a lot of sense to try and pursue, you know, a treatment option for a given patient. You know, certainly I haven't forgotten my full time practice days and very conpisent of how you know the amount of clinical workload and work for responsibilities that lie on the oncologist, research team office staff. You know it the entire team, and and so this certainly would add more work to what they're trying to do. But I feel it's to the same order as it is to enroll a patient on a clinical trial, because you know, especially, even those activities take a lot of time and effort. So I believe in my mind the widget a work that's involved. It is about the same as enrolling a patient on a treatment trial. So it's not an overwhelming it's a standard. Of course, if there were a lot of patients like that, it will get a will bombing. But you know, that's where you have to pick and choose. You know who the program might actually work for. And then another piece that came up in your research that I was curious about is can these requests be made for a small population of patients? So can we? So I say I have three or four patients in the same situation. Can I make one request for all of them? And is that possible for both programs? Great question, you see. So possible. This is possible for the expanded access program not for the right to try program. The right to try is an individual based program based on individual need. But for expanded access, yes, it is possible to, you know, requested for a small group of patients. Do you have any stays of any helpful tips for office staff as they are filling out this document? Any tips on how to communicate with patients while they're going through this proces which I'm sure it is very stressful for all parties involved. Right. So, you know, I think anyone in oncology and you know, everyone that touches a patient, starting from, you know, the intake folks to the nurses, to the nurse practitioners, research coordinators and the back office folks. You know that may not be meeting the patient, but I think it's we have to be careful. I feel as though you have to temper the patients and families expectations. A lot of times, you know folks, unfortunately they're desperate or are losing hope, they tend to amplify, you know, the possibility of success with anything. And so I think you know, while effort...

...is to be laughed we cannot, obviously, first of all, we can I guarantee that, you know, I medication will become available. Number two, even if it becomes available, it may not work, and a number three not only may not at work, it may cause a serious side effect. So so I think counseling the patient, loved ones family is so critical and I think the messaging should be uniform across the team, because human nature is such that we always tend to believe the you know, one person who gave us the most hope when we're down or when we're saying and what you do want to do is that in this process of unfortunately, you know, oncology care or fighting cancers a roller co emotional roller coaster as it is. So you don't want to get them too high and then let them fall too hard. So it was okay to explain and say that we're going to try this but we don't know what the outcome is going to be, so that you know they're that would be my suggestion to entire team so that again, if we gain access, that's a good day and then, once you know, will have to be another a good day after several weeks to see that, you know, a medication working, that it's so Davin's, an honest conversation and the temper your I mean you're excitement of getting the possibility of a due treatment with the realistic outcome. So for someone who has gained access to a drug and have has had success, so for either of these program how does that success affect the future use of this drug? So does that expand the drugs used like permanently to that population. How does how does it affect the the rollout process? A great points. So releases. So I think in this regard the two programs are so different because with the expanded access program there's a more robust back reporting. So you know they're recording in terms of outcomes, in terms of what happens to any given patient, is captured much more assistantly and much more consistently. Number one, with the right to try. First of all, the programs very new. Also understand who's the watchdog for? You know, all of drug therapy in this country is the FDA. And with the right to try program what did we just say? That you bypass the FD. So so I think you know, many have criticized the right to crib program saying that there wasn't enough oversight. You know, of course, the counterpoint is that oversight slows things down and sometimes these patients don't have that kind of time. So I think both camps have valid arguments, but we have the legislation in the form that we have it, and so with that I think it's still an open question terms of how the follow is being managed. Currently there is Nope, to the best of my allege, there isn't a consistent way of capturing as to how many patients are benefiting from the right to cry program. However, in my reading, as of...

...last year, the FDA has instructed many fractures to report to the FDA. You know, how often are the meeting with the right to try utilization and in so so right now, what will happen is in any kinds of outcomes follow up? They those are laid down for right to try by the manufacturer. You know. So what happened? What was the outcome? The manufacturers capturing it, but there isn't doesn't seem to be a consistent way of them putting it into a large national database or what have you. So so information somewhat fragmented, but I feel like that is much better organized with the EAP program because, you know, it's just been around longer and a lot of the bugs have been work at worked out, and also it has more oversight. Okay, so there's additional reporting hopefully coming in the future that we can hopefully build off of that correct and there's been a lot of requests were you know, by the FDA for transparency. That if the right to try is a good thing and a manufacturers are supporting it, well then let's get that word out and, you know, put that out in an accessible area or database so that everyone knows what's going on. And your earlier point about you know, if there is success, that doesn't automatically, that doesn't automatically obviously spend a labl organ f the approval, but you know, the manufacturer does capture all of those real old examples as case reports and then case series which can help them, you know, as they are developing the drug further or going to the FDA for final approval. So in the case of, you know, the patient that I mentioned at the beginning, I had obtained a drug called or Latinum for them and that drug was in you know, in a developmental stage that I only had a number, not even a real name. But since that time, you know, it's been approved, fe approved for that rare form of possible lung cancer and is now available for use in public. So you know, so there are and even the patient that I mentioned, they gain a lot of benefit from that. Ultimately, you know, the cancer unfortunately progressed, but you know, they did, it did help them by time. So all of those and a rule or case supports. Case series kind of get compiled together to indicate success. Aside from, you know, the work that's going on in a tent for trial excellent. So there is some tracking to Mr Gashua, this has been a really great conversation, so much insight and to the to processes that I think that not everyone is has been exposed to what is actually it's pretty recent. For any of our listeners who want to reach out to you with follow up questions or want to continue the conversation. What's the best way for them to do that? Sure, yeah, at least you. Thank you for having you, but you have books. Anyone can reach me. I'm on Linkedin. Is Achieve Gazra had Linden. The good thing about having a rare name is there aren't too many of me, so find me easily. Also, I will Polish a rral. That ad is more at the cardinal level, but we...

...can certainly you can gain access through that route as well. But again, I greatly appreciate the opportunity and for a listeners here, hopefully this has helped you somewhat a game, some confidence in terms of, you know, the crew programs, because they are talked about quite often so thank you for sharing your insights. I'll be sure to include links to your linkedin and also your cardinal health website in our show notes. Again, I appreciate you being a guest of the show today. Thank you for having me. Are You satisfied with your patients curtsy to therapy, or are you just settling? You can improve patient outcomes with bioplus specialty pharmacies power of to the first ever two hour, two day to gather promise it's faster and easier for you and your patients to learn more. Visit bioplus R xcom. You've been listening to working in oncology. To ensure that you never miss an episode, subscribe to the show in your favorite podcast player. If you're listening in Apple Podcast, we've love for you to leave quick rating of the show. Just have the number of stars you think the podcast deserves. Thank you so much for listening. Until next time,.

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