Value-Based Tools and Real-World Evidence in Oncology

Bruce A. Feinberg, DO: From a provider’s standpoint, though, what I like about [National Comprehensive Cancer Network (NCCN)] Evidence Blocks is that they’re a great visual. But whether it’s Drug Abacus or the American Society of Clinical Oncology’s (ASCO’s) Value Framework, it’s hard to see a lot of differentiation when you look at some of these diseases and the drugs that are available.

If there was such a situation where one company decided to really make a move and discount dramatically, would that impact the provider? Or if that discount isn’t shared by the patient and the provider, but it’s only absorbed [by] the specialty pharmacy and payer, is that going to be something which is going to influence your behavior?

Bruce J. Gould, MD: First of all, it’s going to be hard for us to know how those monies are shared unless it’s from the patient’s perspective. But, again, I would say that if all things are equivalent in terms of efficacy, and side effects [turn out to] be manageable in that particular patient, then I think cost would become the bigger factor.

John L. Fox, MD, MHA: Yeah. I see a lot of challenges though today in version 1.0 of the ASCO Value Framework and in the NCCN Evidence Blocks. If you look at the NCCN Evidence Blocks for relapsed multiple myeloma, they give the same number of blocks and the same configuration for bortezomib, dexamethasone, Thalomid/dexamethasone, and carfilzomib/dexamethasone. They get 4 blocks across efficacy, safety, consistency, and quality of data, and then they vary on the cost.

Bruce J. Gould, MD: And with luck.

John L. Fox, MD, MHA: Yeah. So, if you look at that, you’d say in relapsed multiple myeloma [where] everything else [would be] equal except the cost, we’d use Thalomid and dexamethasone. But I don’t think that’s the standard of care. So, I think there are some things that have to be worked out there, too.

Likewise, daratumumab (Darzalex) was just approved based on a single-arm phase II trial. They give it 4 blocks across for quality, safety, consistency, and efficacy, based on 1 single-arm study. So, the point is, I don’t think we’re ready to use any of these tools quite yet in making decisions on what’s preferred, or not, on our formularies; and I’m not sure how physicians are looking at those tools.

Bruce J. Gould, MD: I would say that most of them probably aren’t looking at them. [In regard to] the value-based framework from ASCO, I’m not seeing an update from their initial presentation. I did see the NCCN [Evidence] Blocks, and I think the only good thing about these 2 programs is it does at least increase the physician’s awareness of the toxicities and costs, and gets them thinking more about costs and maybe what they would have done in the past. But, I think the information is too vague to really make a huge impact. We need, I think, more specific numbers, and I think that would tell a different story.

Bruce A. Feinberg, DO: Many of these agents are never going to be tested head-to-head. It’s not in manufacturers' interests to do so. So, there seems to be a great call for real-world evidence, and as the statistical validity of such research is becoming more and more proven, do you think providers will be comfortable in real-world evidence-based analyses to try to answer some of these questions?

Bruce J. Gould, MD: Well, obviously that’s the idea behind ASCO’s CancerLinQ or Flatiron’s program: to get the collective experience of doctors from around the country and to be able to make some inferences in terms of what therapies are better and which are less toxic. Then, of course, we’d have the cost data of those programs. Obviously, it looks like a very good strategy. It sounds good on paper, but when really trying to get that information to make those analyses, it is much more complicated.

John L. Fox, MD, MHA: We need a sentinel example of how real-world evidence could influence outcomes. Then, I think everyone would get on board and support that. The question is, who’s going to pay for the collection of that data right now? Bruce, I would agree with you that we would like some real-world evidence, in the absence of head-to-head trials, to show what the relative benefits of these different strategies are.

Bruce A. Feinberg, DO: [There is a] tough and continued conflict between population health, the impact, and direct patient care. You live it every day. I don’t know how, as a nation or society, we come to terms with that. But we’re going to have to. It’s inescapable.