How And When to Leverage Integrated Evidence Plans (Part 1)

[00:09:31] Jenny: So exactly. And I think that’s a really important thing is that there isn’t a global health care environment.

[00:09:37] There are local health care [00:09:40] environments, and they have different evidence needs of different evidence requirements. And sometimes when there’s overlap. They can group together to work on a plan to generate their evidence, and sometimes there’s not, and they need to decide, are we going to support this product?

[00:09:56] Is it worth chasing after? Or, or are they [00:10:00] unlikely to beat the standard of care? And so we’re going to let it be. 

[00:10:04] Alexander: And so and finally, of course, there’s also marketing. Yeah. And my very easy between marketing and sales is sales talks to individuals, marketing talks to groups. Yeah. [00:10:20] So that’s for me the very, very easy and simple kind of way to talk about it.

[00:10:26] And of course, since there’s. Something similar on the medical side very often, yeah, where you have some medical affairs functions that falls into people’s that work more [00:10:40] on the, I would say, let’s say, medical marketing. Yeah. So providing content that can be accessed by many people. Or the medical scientific liaisons that very often work on a one to one basis with Q opinion leaders [00:11:00] or very, very small groups.

[00:11:02] Jenny: So what you see then is that over the course of a product’s life cycle, from development to post marketing, the number of stakeholders, internally and externally, grows. And their ideas about the evidence that they need to [00:11:20] support a product also increase. And as we know from Fisher, the idea of bringing a statistician in after data have been generated is putting them in a position to have to do an autopsy to decide what an experiment died of.

[00:11:37] What I talked about at the effective statistician conference [00:11:40] related to the integrated evidence planning is that the statisticians need to get a seat at the table and need to try to understand what the needs are of these different stakeholders so that they can weigh in on optimal evidence generation plans to support a product’s launch and, and address questions [00:12:00] throughout the life and 

[00:12:01] Alexander: the integrated evidence plan.

[00:12:03] always comes with some kind of communication plan around it. Yeah. So don’t think about it just as a very big Word document or slide set or things like that. [00:12:20] Yes, you have that and, or you have a collection of slide sets or things like that, which are probably living documents. And in addition, you have these kind of communication channels.

[00:12:33] Yeah. So that all these different people can actually, you know, [00:12:40] feed their needs into this plan. Yeah. Distill it. Explain it. Combine it. Prioritize it. And so on. Absolutely. 

[00:12:51] Jenny: And I think that’s a really excellent point is that development plans were the most part [00:13:00] tend to be fixed at a certain point of time, because that’s the definition of providing confirmatory evidence is that you eventually settle on a design, you settle on a profile, you develop your evidence generation based on that plan, and you present it to health authorities, but our products.[00:13:20] 

[00:13:20] Are often on the market for a long time, not just five years. It, of course, depends on the product line, but it might be 10 years, 20 years, 30 years. And during that time, one can imagine that things in the health care environment emerged that require additional [00:13:40] evidence generation. So an integrated evidence plan is not fixed at one point in time.

[00:13:45] It is begun at one point in time, hopefully well before launch. To identify gaps, and then it is up on a regular basis to understand what remaining evidence gaps [00:14:00] exist. These gaps can emerge in response to competitor entry into the marketplace. Some of them can emerge based on competitor exit. Of the marketplace.

[00:14:14] New safety signals can emerge. You can learn new things about your product. You might get new indications that [00:14:20] impact the price typically. Going south, just because companies say fine if more patients, or HTA bodies say fine if more patients are going to get this drug, we need a little bit of a break on the cost.

[00:14:35] Alexander: Yeah, yeah. And of course, if you have multiple indications [00:14:40] for your drug, you know, that subsequently, and actually faster and faster subsequently get to market. Then that also means that you will have more and more complex evidence plans. And you just mentioned the [00:15:00] clinical studies that we all know about and that we work on it inside out and we’re trained on.

[00:15:08] So that is just one source. For the evidence. What are other sources of evidence that we can use for the integrated evidence plan? 

[00:15:19] Jenny: [00:15:20] Certainly a big part of access. Evidence is comparative effectiveness data, and so often this is generated via indirect treatment comparisons and network meta analysis, systematic reviews that are generated specifically for the access evidence dossier.

[00:15:37] Some other sources of evidence are, of course, [00:15:40] real world data as your product hits the market, you can learn a lot about how it is being used in which patients it’s being used and, and what new safety signals that you didn’t detect during the development program are starting to emerge. This is a regulatory requirement to monitor that.

[00:15:59] People [00:16:00] run or, or subscribe to registries for diseases or for. Or for products there can be clinical studies there can be chart reviews, you know, there’s any number of, of sources. Sometimes it’s company sponsored and sometimes it is completely independent and [00:16:20] it is important for the statistician to understand the implications of all of it for the product.

[00:16:27] Alexander: Speaking about real world evidence, of course, we are always interested in real world evidence for, from the product itself. And by definition, you can only get that [00:16:40] after you have launched the product. 

[00:16:43] What very often is also required is evidence about the burden of the disease. Who gets treated? What are the current treatments that are used?

[00:16:54] Especially in terms of if you have a completely new class or something like this. How [00:17:00] long are patients treated? What are their side effects? What are their symptoms over time? There’s a lot about these things as well. Also, patient preferences. Might play an important role. Yeah, if you have a new drug that works differently than the others, and [00:17:20] maybe has a much better efficacy profile, but comes for a cost of a different safety profile, or you have a drug that is similar in efficacy but has a different safety profile, then definitely things about patient preferences will play a role. Also, [00:17:40] administration, all kind of different things can play a big role in this area. 

[00:17:45] Jenny: I think it’s, Also a neglected topic that a lot of the stakeholders questions in the post approval phase are comparative in nature. And so while you need to [00:18:00] wait for real world data on your product to be generated, there is also a need to generate Possible evidence about the effectiveness of your product in the real world, which might all using real world data as an external control arm and [00:18:20] and things of this nature.

[00:18:22] This is an area that is evolving rapidly. Of course, anytime you don’t. Contained experiment. There is room for doubt and there is increased complexity, but these stakeholder questions are not unimportant. And so [00:18:40] it is upon the statistician to get creative and Get it right to at least answer the question.

TO BE CONTINUED…