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While at DIA’s 50th Annual Meeting, we got the chance to speak with Nancy Dreyer, chief of scientific affairs and senior VP at Quintiles Outcome, the CRO’s real-world and late-phase research division, about the current tide for comparative effectiveness research (CER). Dreyer is the leader of the GRACE Initiative, which has developed good practice principles for observational studies of comparative effectiveness. She also is a contributor to the federal handbook “Registries for Evaluating Patient Outcomes: A User's Guide.”
ACT: What trends do you see emerging in CER at the moment?
Dreyer: The uses of CER are growing dramatically. There have been some tremendous changes in the world. One of them is, we know how the FDA has risk-management programs—if a drug comes on the market and they’re concerned about the safety but they think it’s worth marketing, they require post-authorization safety commitments. In Europe, they’ve changed the dynamic. It’s not just safety, but they’ve taken into account effectiveness. It’s not enough to look and say somebody had a side effect. They’re looking in different subpopulations and saying for these people, the benefits are really strong or the risks are small for this population. Europe is ahead of the U.S. because they’re asking for [data] on benefits as well as risks. They have changed their legislation so they can require benefit-risk data collection after launch.
ACT: How have perceptions of CER evolved among healthcare payers?
Dreyer: Payers thrive on CER, they just won’t tell you how to do it. Because every question they ask is, ‘Why should I pay for this?’ It’s not as simple as, ‘This drug costs more.’ Payers have a surprisingly long view. They want to know over the long-term is this benefit really going to make a difference. Then their decisions are yes/no; ‘I’ll cover it or not. I’ll cover it for this person because of their other comorbidities but not for this person.’ They need a lot of very detailed information.
ACT: What about the sponsor’s responsibility in providing CER and real-world data to payers and regulators?
Dreyer: They used to measure time to market approval, which is pretty important. Now they’re starting to look at time to reimbursement. Time to reimbursement means you had enough data to convince somebody [to cover your product]. There’s some great stories about drug companies that do these head-to-head trials because that’s what they think the payer will want. But then they come out with their study and the payer tells them it wasn’t the right comparison. So they spent how many tons of millions on the wrong evidence? Head-to-head trials aren’t very popular, because you’re not likely to have the comparator that will be important by the time it’s done. The real-life experience is very different from the clinical trial experience. Several new products never made it. They were good but you needed the real-world setting to understand the value.
ACT: What can sponsors do to adjust?
Dreyer: You have to build that evidence outside the clinic setting. It comes down to having a good enough product that people are willing to give it a try, and then watching who uses it and how they use it, and finding similar people. You have to find the right comparators. Companies have to start to understand who are the new [treatment] adopters and find people who are similar to them. We do lots of studies in people who are getting their first treatment, for example. It’s all about the context in those real-world studies. That’s where the value is.
ACT: What tools are supporting the advancement and implementation of CER?
Dreyer: There’s an exponentially growing interest in this area, but we traditionally didn’t know what data to use. The only data that was readily available was insurance claims data. Now that there’s so much more electronic medical record data available, we’re starting to marry those and have the real clinical detail along with, ‘Did you actually fill the prescription?’ There are a lot more tools available to pharma companies and to payers and to regulators that they’re starting to pay attention to. Because you can’t do everything in a very expensive prospective study. Sometimes you need to have early indicators.
ACT: Where do you see the future of CER in helping shape healthcare decisions?
Dreyer: CER has the ability to improve not only access to medicine but the practice of medicine. It’s at the heart of these learning healthcare systems that are talked about. They just don’t have all the tools yet. CER is going to be a fundamental game-changer in how we practice medicine and will help us get access to the right medicines at the right time for the right patient. The technology is enabling these changes, but it’s the training and the experience of the people that implement it and interpret it that’s going to be most important.