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Transcript:
In this episode, I talk with Cyndi Verst, PharmD MS Senior Vice President of Global Late Phase at i3 Innovis. We discuss blending scientific and commercial objectives in late phase research, saving time and money by using specialized data assets, the challenges of leveraging secondary data assets and what’s in store for late phase research in the near future.
I’m Dan Limbach, your host and producer of the PharmaVOICE Webcast Network.
Dan: Thank you for participating in the podcast, Dr. Verst. Let’s get right into this fascinating topic. What market pressures are driving the need to strategically blend scientific and commercial objectives?
Cyndi: As we all know, the most recent occurrences within the marketplace, dwindling product pipelines and increasing regulatory pressures certainly relative to safety, diminishing patent lines, generic erosion of brands, and of course, payers demanding that evidence of very strong economic value, and not withstanding, even regulatory authorities as we all know around the globe, in the UK, NICE and that being the fourth hurdle in the Germanic area and even here in the United States, having the ability to differentiate one’s product and giving that value proposition to these pricing and reimbursement authorities and regulatory authorities.
In addition to that, the many new biological products that are being developed, unlike the traditional pharmaceutical lines, are very, very expensive. So for all of these reasons and more I think that’s what’s driving the late phase marketplace. In fact, most recently Jefferies & Company released an evaluation back in January 2007 and they estimated that in 2008, that the phase IIIb IV late phase research is estimated to be about $3.5 dollars and in 2010, to increase to $4.7 billion, about a 17% compound annual growth rate. Staggering. So the bottom line, late phase research must ultimately maximize the return on the investment on behalf of the sponsor and be conducted overall in a very cost effective manner due to all of these price pressures.
Dan: You mentioned some pretty big numbers there and everybody can appreciate saving time and saving money. So how can specialized data assets be used to improve time and cost efficiencies in clinical research?
Cyndi: It’s incumbent upon providers, such as CROs, to provide very cost effective modalities in conducting the late phase studies. In particular, leveraging secondary data is a very large opportunity for sponsors. In fact, being able to leverage the secondary data assets of health claims data, for instance. So those health care databases that are comprised of not only pharmacy claims data but also linked to healthcare itself so in terms of the office visits, having visits to urgent care providers, any diagnostics and in fact, laboratory data, being able to link all those data together and then analyzing those data to provide very novel, very cost effective approaches to late phase research is certainly opportunistic and is on the horizon.
In particular, some of the ways in which we can leverage secondary data is being able to evaluate and assimilate protocols. So for instance, testing a protocol, being able to look at certain inclusion/exclusion criterion and then noting how that might impact the overall patient numbers. All too often, and we’ve all experienced this, that we develop a protocol thinking that it’s incredibly feasible. We implement that protocol and then we find out we’re not recruiting patients at the enrollment rate that we had originally anticipated. Upon further evaluation, we come to realize by querying the sites well in fact what’s limiting the patient enrollment, what’s limiting – or increasing rather the screen failure rate, is a certain inclusion/exclusion criterion and then that results in significant time delays, resubmission to IRBs, reengaging the sites, and overall loss of costs and time. Being able to leverage the secondary data assets then we’re able to assimilate that protocol within the confines, of course, of the claims data and understanding which of these inclusion/exclusion criterion majorly might impact enrollment and in fact, that has been incredibly useful in the study designs. And in addition, we’re able to conduct preliminary feasibility; so being able to get drilled down right to individual physicians and identifying then the ability of their recruitment capabilities, for instance, which is really the second way that we can leverage the secondary data assets, is by way of investigator profiling.
So while the data are de-identified, with the appropriate IRB approval, we can in fact, identify down to the individual investigators, and down to individuals patients, we know which physicians are prescribing which products. Accordingly, that’s very important information when designing a clinical trial and understanding the feasibility.
The third most important way in which we can leverage the secondary data assets, in addition to assimilating protocol design, as well as investigating and patient identification, is overall reducing burden on the site. What I mean by that, relative to late phase research, we all know that this then is the arena where we’re reaching out beyond the academicians into the community based practices. So specifically these physicians have more research naivety, if you will, than the academicians. So we know that what is pinnacle to the successful conduct of late phase research within these settings is not to interfere with the normal, daily clinical practice of a physician. That is absolutely of paramount importance.
Accordingly, when leveraging secondary data assets in certain claims databases, we can go in and identify with the appropriate IRB approvals and with the appropriate consent from the physician, we can provide to that doctor the listing of all of his or her patients that meet the criterion of the protocol. And that then is significantly reducing the site’s burden, as they do not typically have survey or study coordinators that can spend the time delving in and identifying through charts who those viable patients are. So that significantly reduces and lessens the burden.
Moreover, in certain other study designs, that is direct to patient observational programs, by leveraging the database we can obviate physicians involvement altogether, again with the appropriate IRB approval, with the appropriate consent of the health claims company, we can in fact, in a very cost effective modality and time efficient, go directly to the patients to collect patient reported outcomes. So for instance, quality of life, treatment satisfaction, etc., which is significantly lessening the burden on the site.
In summary, there are multiple ways that one can leverage secondary data assets, to simulating protocol design, profiling investigators, identify patients, reducing the burden on the site and the ability to conduct very novel study designs (in this case we call then hybrid study designs).
Dan: We’ve talked about a lot of good stuff, now let’s talk about the challenges to leveraging secondary data assets. What can you tell us about that?
Cyndi: Well certainly with all the pros there comes some cons. So definitely I want to be fair balanced in this approach. And I think that some of the challenges, or limitations if you will, leveraging secondary data base assets, is the consideration of the commercial or privately insured sector. Obviously, the database overall represents a younger population compared to that of the aggregate real world population, so one must bear that in mind. And of course, it then depends on the type of clinical studies and the indication that’s being evaluated because that might play a considerable impact. For instance, if the population of interest is an elderly population i.e., greater than 65 or a Medicaid/Medicare population, that database is going to be somewhat limited for that particular population.
In addition, observational data presents issues. Let’s just be completely fair balanced, that understanding that we’ve got missing or omitted variables, we’ve got measurement errors that might be occurring, and even patient and sample selection bias, but all of which indicates some concerns regarding endogeneity. But of course, this is a constant threat in any studies involving observational data; nonetheless, while these challenges are present, I firmly believe that the benefits certainly outweigh the risks.
Dan: And finally, Cyndi, it’s time to look into your crystal ball. How do you envision the area of late phase research evolving in the next two to three years?
Cyndi: Terrific, terrific question and certainly what’s on all of our minds. And I think it’s really a three-pronged approach. I certainly think that the continuous drive to cost effective late phase research conduct, strategic study design that’s near and dear to my heart, there is going to be even further pressures to get that right study design and lastly, a tremendous focus on safety programs. So late phase studies that involves a safety impetus and that starting off with the cost effectiveness. Late phase research is going to require more and more cost sensitive approaches. Sponsors are demanding it and we CROs are trying to acquiesce and meet those demands. And so leveraging secondary data assets obviously is a very viable lever to accomplish those cost effective approaches, but also very strategic monitoring schematics. So being able to offer remote monitoring, being able to augment the site’s efforts, holding the hand of the physician, if you will, to ensuring successful study execution is of absolute importance, and I think you will be hearing more and more about that.
The other thought here is that technology will continue to come on the horizon, will continue to mount in importance. And I think technology overall in the late phase arena, even more so in my humble opinion, than in the phase 2-3 arena, when dealing with very voluminous sized trials where you have now hundreds of thousands of patients and thousands of sites around the globe, that then provides its own set of complexities in managing those data, ensuring communication and connectiveness around the globe, having flexible data collection, for instance in processing, whereby these community based practitioners can fax in their CRFs, can submit paper CRFs or submit electronically; technology is going to be at the hub.
The second important point is blending the clinical and commercial or health economic outcomes into late phase research study design to maximize the ROI. It must occur in my opinion.
And I think thirdly, late phase research will continue to focus more and more on the safety aspects. And in fact, most recently published in the American Journal of Managed Care, April of this year, it states that as of May 2006, there were in fact, 111 unique biopharmaceuticals coming up the pipeline. That is they are in phase 2 and beyond. And biopharmaceuticals they define as recombinant proteins, monoclonal antibodies, peptides, antisense oligonucleotide, etc. that is basically represents about a 16-30% annual growth rate versus the traditional 4% annual growth rate of pharmaceuticals. Why is that important? Well of the 111 unique biopharmaceutical agents, 87 of which are new molecular entities. So that means regulatory authorities are going to be paying particular interest to the safety profile. It’s unknown. There’s not a category class of products already out there in the marketplace to provide a historical perspective. Since that is not the case, then they are going to be paying very close attention to the safety profiles of these products. And importantly, 50% of these products are in fact for indications that are for chronic, long term conditions, that is defined as greater than one year of use.
So what does that mean? Well, since it’s a new molecular entity in it’s being used for a long term condition, again even further rationalization that safety evaluations are going to be required in long term setting, so long term registry studies. But it doesn’t end there.
Over 70% of the agents in the pipeline will require administration by healthcare providers. That means it will be adding to the preponderance of costs associated with these new products. So again, even further reasoning for the combining of not only the scientific, but those health economic outcome research commercial objectives into the clinical trials to help satisfy the regulatory authorities, as well as the pricing and reimbursement authorities and pairs. It’s going to get even further complicated, I guess, is my summary of the late phase environment, but I think identifying some of these pearls and really looking at some the ingenious ways and revolutionized ways of conducting late phase research in a cost effective modality, it certainly exists. I’m just thrilled to look at the horizon and see how this marketplace is changing and it’s thrilling, thrilling to be a part of it.
Dan:Well thank you so much, Cyndi, for sharing your expertise and thought leadership with us today.
Cyndi: Thank you very much. I appreciate the opportunity.
Dan: And that closes out this episode. Special thanks to Dr. Verse for providing some great insights into this new era of utilizing secondary data assets in late phase research. If you would like to know more about this topic, visit www.i3innovus.com. And don’t forget to check out our other podcasts at www.pharmavoice.com/podcasts.
Until next time, I’m Dan Limbach.
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