Quintiles vouches for alternative approval pathways
In early February 2013, FDA held a hearing titled “Creating an Alternative Approval Pathway for Certain Drugs Intended to Address Unmet Medical Need”. Quintiles Chief Medical and Scientific Officer Dr. Jeffrey Spaeder was the first person to testify at the hearing. Dr. Spaeder shared Quintiles’ support of a proposed pathway to speed the introduction of new therapies to patients with life-threatening illnesses and for patients with unmet medical needs. Quintiles executives say that one way that Quintiles is bringing innovations to patient recruitment strategy is through innovative site relationship management. Prime and Partner sites typically outperform non-partner sites by an average of 50 percent, which enhances productivity, quality and an improved experience for the patient. Specifically, the Prime sites offer transformational relationships involving high levels of strategic commitment and a substantial volume of work. These are the elite global research sites, typically conducting 20 to 60 plus studies per year for Quintiles. The Partner sites provide strategic relationships that are actively managed and involve ongoing work across multiple studies. Associate Web Editor Mia Burns spoke to Dr. Spaeder about the proposed alternative pathway and its implications.
Q: How would the patient be the best to judge as to whether or not a risky medication could improve his or her health?
A: I think that when we talk about risk is that what we are really talking about is uncertainty and potential side effects. Let me first talk about uncertainty. There are some patients who have a desire to take a medicine with very well described benefits and side effects. And then there are other patients that maybe value that degree of certainty about the efficacy and the side effects. They place a lower value on that if they can potentially get benefit from a therapy. Essentially, the culprit with uncertainty is really something that is best answered by a patient. I also think that gets to a second point about how much information is available. There are some patients that want a significant amount of information to be available before they take any medication or there are others who want to be one of the first people who take it. With our point of bringing forward the concept of risk here is not like gambling with a drug. It’s really related to the degree of uncertainty about the benefits of and the risk of the drug. Certainly, from our perspective and what we were talking about in our testimony is that we want to do things to help get therapies to patients much more quickly, but that we want to make sure that the use of the medications are only those indications which are most compelling. I think that the whole concept of the alternative pathway is really to get medications to patients who need it more quickly with the acknowledgment that additional insight is going to be gained with use of that medication over time. I think that one of the impacts of the proposed legislation, the pathway, is that it is going to answer some of those questions that maybe have been answered previously before the drug was marketed, to try and answer those questions afterward in order to get the drug in the hands of the patient.
Q: Aside from Infosario Design, does Quintiles have any other advanced trial design tools that could incorporate real-world data into trial designs?
A: Infosario Design is a very sophisticated tool and it’s not only a database and eight million patient lives, but it’s really a whole process of being able to ask the questions. One of the things is that when we talk to the people at Infosario Design is that it we have to tell them it is a database coupled with a way of asking questions coupled with therapeutic insight and asking the right types of questions. It is the whole suite unto itself that we group under the concept of Infosario Design. I think that when we talk about how do we utilize real-world data, how do we format it in terms of extracting knowledge to either help design a study or help with knowledge in getting an answer to a clinical question or to answer medically relevant questions—the other two large capabilities that we have are an advanced statistical analytics group that helps design adaptive studies and design the statistical components of studies prospectively. And then there’s our Outcome late phase group that does Phase IIIb observational and interventional studies and that is a huge burgeoning area where we’re using real-world data to answer questions that are clinically relevant. Some of that insight can be used in designing prospective, randomized controlled studies and other components are used to answer questions just with real-world data, maybe in the place of some randomized controlled studies.
Q: What would be the criteria for a higher-quality study site?
A: All of the sites that we use meet requirements to be able to produce quality clinical insights in an environment that we identify to be safe and scientifically valid. We also know that sites that have a significant emphasis on conducting clinical research tend to attract healthcare providers and tend to attract other professionals that are particularly interested in conducting high-quality research and are interested research as a focus of what they do. We tend to see that those sites tend to have higher volumes because they are actively involved and focused on doing research as part of their mission to deliver care to patients. We see those sites like anything else, the more you do something, the better you get at it. This was a question that came up several times was that these sites already meet our existing thresholds for conducting research, but there are some that because of their focus on it, their dedication, and the frequency with which they do research, clearly differentiate themselves from other sites. It is those sites that have this higher quality that are the ones we see being important in this alternative pathway where you have smaller populations enrolled in the studies and as a consequence much less tolerance for variability.
Q: How soon would you like to see the alternative pathway embraced?
A: As I mentioned in the testimony, these capabilities are things that we currently deploy now. We would be very supportive of a pathway being introduced immediately. We think that we’re in a position to support the needs of researchers that will be conducting studies with new therapies. We would love to it happen sooner and as soon as possible. I would say that we do many of these things right now. I would say that the things that we don’t do right now, that we have the capabilities to do right now.
Q: Would you be able to estimate how much faster the alternative pathway would be compared to the current pathway?
A: It really depends upon the therapeutic area that is being evaluated and the types of endpoints that are being used. I could imagine that with an indication where the endpoints occur relatively quickly or that there are surrogate endpoints that are accepted by the agency that this type of pathway could significantly reduce the time that it would take to get a drug approved. In other indications of longer duration or maybe without surrogate endpoints, it’s going to take much longer. It really is somewhat dependent upon the indication and the mechanism of action. I think that what it does do, however, is that the adoption of this type of pathway makes it easier for researchers, whether they be biopharma, biotechnology, and even other types of researchers to be able bring drugs and to test them sooner because it gives a way to potentially get those drugs to the patients more rapidly. I think that it lowers the risk and some of the uncertainty that sponsors have when they are making decisions about taking certain drugs into later phase development.
Q: Could you please elaborate on Quintiles’ Prime and Partner Sites program for those who may not be familiar with it?
A: In general, the Prime and Partner Sites are what we look at as a continuum of our investigators and our sites with whom we partner. The Partner sites and then the Prime sites, which are kind of an extreme example, they tend to do a higher volume of research where research at the site tends to be more of a focus of the way in which the organization and institutional investigators deliver care. They typically as a result of this focus tend to attract people that are healthcare providers who are particularly interested in doing research. And they have an infrastructure in place that is compatible with performing research on a more almost industrialized type of scale. As a result of these designations, we tend to have more interactions with these particular sites. We can ensure that the quality is in enhanced even above what was already a high level. We have more regular interactions with the management at the sites to essentially problem solve together about identifying things that work well and try to identify issues that there are problems with conducting research and to work collaboratively. The more dedicated sites become to doing research, the more collaborative we become in problem solving in working as a team with our investigators, but even more collaborative as our site progresses from a Partner to a Prime site.
Q: After learning the outcomes in such patients, what would be the next step?
A: Well, a couple things. The first one is that it gives even greater insight into the efficacy and safety of the drug in real-world use. That allows regulators, prescribing physicians, healthcare organizations to integrate what the real-world experience is and how it compares with the clinical trials. It better informs decision makers about the value of a therapy in the real world. The second thing that it does is that it potentially allows researchers to new indications or different subpopulations that might particularly benefit from a therapy and whether that results in a label expansion or is at least hypothesis-generating in terms of label expansion I think is one potential outcome of that data. What we see is that insights about drugs come from a variety of different sources. Certainly it comes from preclinical research, from prospective randomized controls, but also importantly from real-world experience with the drug. I think that there is a real value in being able to incorporate that real-world data into knowledge about when the drug should be used, potentially impact upon the label and guidelines and those practices about how best to utilize it. What I would see, the data that would come from observational studies would really add to our existing knowledge base and just like any other clinical study and how that potentially effects the use of a drug, we see that same type of potential influence from real-world studies having the same type of target audience for the decision making that would be performed.
Posted: October 2013