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AMCP Podcast Series - Listen Up: Charles Makin on RWE
Show Notes
Host Fred Goldstein invites Charles Makin, Global HEOR, RWE and Market Access Advisor to discuss their poster at AMCP Nexus 2023 on RWE adoption by US Payers.
Transcript
Fred Goldstein 00:01
Hello and welcome to the AMCP Podcast Series Listen Up as we take a deep dive into the challenges, trends and opportunities in managed care pharmacy, follow the show's social hashtag AMCPListenUp. And to learn more about AMCP Visit amcp.org. I'm your host Fred Goldstein. On today's show, our guest is Charles Makin, Global Health Economics Outcomes Research, Real World Evidence and Market Access Advisor. Welcome, Charles.
Charles Makin 00:32
Fred. Thank you for having me.
Fred Goldstein 00:34
It's a pleasure to get you on today. So why don't we start with, you recently presented a poster at the AMCP Nexus 2023. Can you tell us a bit about the poster presentation and how that poster came to be?
Charles Makin 00:47
Sure, Fred. So for the past few years, a few of us across different pharma companies meet as a group in a non competitive manner to progress the science of Real World Evidence or RWE in the healthcare landscape. We take on different projects as part of this, whether they be by by advancing methods, or establishing frameworks for assessing ROI on RWE investments, and so on. And as part of that, one of the things we are looking into is how best to improve and accelerate the appropriate use of RWE in order to ,RWE is very, very useful tool that's used across the healthcare spectrum, including regulators, clinicians, policymakers, payers, and so on. And we want to understand how it's being used currently. And for this one, we narrow down our focus to payers.
Fred Goldstein 01:34
And so given all the stakeholders, you mentioned this, why did you focus on payers in this case?
Charles Makin 01:39
That's a great question. As we know, health care costs are a major, very visible issue everywhere. And payer needs for evidence are increasingly complex, but also more nuanced. We have aging populations, we have innovative medicines with uncertain long term effects and tightening budgets all over. And in the US in particular, you have the 800 pound gorilla, the IRA, which brings lots of changes, including granting CMS powers to negotiate drug pricing, Medicare Part D redesign, maximum patient out of pocket cap and prescription drug inflation rebates. These changes, and we know CMS has already released its first list of 10 drugs targeted for negotiation, and increase pricing pressures will be key to driving evidence requirements and evaluation standards. So we expect that as a significant hybrid portion of the cost of drugs falls on commercial payers, more comprehensive evidence and value demonstration will be needed to inform decisions and justify risks. So RWE presents a tool that can be used to truly understand the value of medicines. But as of today, as a community, we don't even have a very good understanding of how well how frequently it is being used by payers. So that's what drove this. And then we chose to focus on US payers for this round, at least, to understand the present and future state of Real World Evidence in the context of its use, exploring existing challenges, opportunities and recommendations for improving the uptake. And then, what we did here was to accomplish this, we started with a little review to understand what's out there on this topic, we realized that there was not a whole lot. And then we conducted interviews with key US payers, pharmacy benefit managers, private health plan execs, HTA leaders and all some leading academics in this space to understand this better. Fred Goldstein 03:36 So when you pulled all this information together, what did you find out about how Real World Evidence or RWE is being used for drug value assessments?
Charles Makin 03:46
I wish I had a very elaborate answer for this. Unfortunately, the short answer for this is that RWE usage for drug value assessments is very low today. But I think Fred, for me, one of the most striking aspects was, you know, we created a graph, as we were thinking through visuals for what we learned during our survey, during our interviews, rather than the survey. 100% of our respondents, 100% of the people we interviewed, including the academic said that RWE is becoming increasingly influential in decision making. But right in contrast to that, you know, how many said out of these routinely used in value assessment? Exactly, zero. So, now again, the key over there was in a third third question that we asked was, do you want to use Real World Evidence? Is there appetite today? And again, the vast majority of them said, Yes, but I think it's that gap between well RWE is very influential, we want to use it. But are we using it today? I think it's that gap that we as an industry need to address.
Fred Goldstein 04:52
So let's talk a little bit about that gap. When you think about it, they want to use it. They're not what are some of the roadblocks that are keeping that from happening,
Charles Makin 05:01
Yes sp we identified five key challenges currently hindering the routine use of Real World Evidence in value assessments or by payers in general. The first one is poor understanding of the best use of Real World Evidence for better decisions. The challenge here is that opportunities to collect real world data prior to product launch are few and far between. and then use of Real World Evidence in initial submissions beyond things like natural history and standard of care data is limited. So outside of Real World Evidence generated from external control arm studies, or, or some kind of an endpoint or endpoints in an RCT, the highest value for Real World Evidenceis likely during reassessments. So the majority of our respondents stated that they don't regularly use our RWE in drug value assessments mainly because they don't understand how to apply Real World Evidence to therapies in clinics. And then, you know, as a result of that, many believe that RWE poses little to no added benefit over RCT data. The second roadblock that we uncovered was specific to outcomes based contracts, or OBAs. or OBCs. Everyone we interviewed, as I said, expressed a high degree of interest in using and specifically for outcomes based contracting or outcomes based agreements. And it's actually one thing if you think about it, that pharma and payers can agree upon right, basic price and value and not volume. But the adoption of these agreements has been very slow. And the main reason is difficulties in defining and measuring relevant real world outcome metrics in a practical manner. And most of these approaches are centered around patient level clinical data for relevant populations. And it will take a lot of time and resources to implement and monitor unless it's already present in routinely collected data. The third reason we had was around lack of common guidelines for RWE evaluation and interpretation during value assessments. You know, we know RWE has its own limitations, bias, confounding data, completeness, or incompleteness, mismatch, competitive cohorts, and so on, and so forth. So while there are some frameworks that do exist for this, such as NICE, there are currently no consensus, no consensus guidelines for US payerws on the interpretation or evaluation of RWE. So what ends up happening is many payers that do evaluate RWE, they ended up using tools designed to evaluate RCTs in their effort, despite what we know are significant methodologic and structural differences between the two. That means that players need to devote significant time and expertise to evaluate RWE, which is a big deterrent. And the fourth thing we found was that when and that's very closely linked to this, this past one is the lack of expertise in data assessment and analysis for RWE among payers. Now, historically, RWE is a fairly new science, payers's understand RCTs, they don't fully understand our RWE just yet. I would argue that most of us don't at this point in time, right, including those of us who have been doing this for a while because we're learning about this as we go along. And new advancements are coming at a very rapid rate. So from the interviews, we did, you know commonly mentioned concerns into the data collection issues, study design flaws and poor analytical approaches. And then some of our respondents also noted the lack of consideration for payer requirements such as poor representativeness or relevance to payers populations of interest. And then the last roadblock that, you know, again, we distilled from interviews was the age old problem of a historic lack of trust by payers in pharma sponsored Real World Evidence or any kind of evidence generation, so to speak. This was consistently cited as a key reason for not including RWE in value assessments. Poor transparency and the processes by pharma was mentioned, specifically the absence of regulatory requirements to publish intentions and protocols prior to conducting RWE studies, right as we do for RCTs today, and these things have compounded concerns over selected publishing of results and potential conflicts of interest in sponsored research. So, you know, again, well, there were a lot of smaller reasons we picked up but I'd said those five are the key ones that we distilled it down into,
Fred Goldstein 09:27
Charles, given those five and you just mentioned some of the issues associated with transparency, etcetera. What are some of the actions that pharmaceutical companies can take to expand current payer use of our RWEs
Charles Makin 09:39
So Fred you probably picked up on the fact that the common theme across the challenges mentioned above is a lack of consistent communication between payers and pharma, whether that be in terms of trust or education or availability of payer relevant data. So in theory, a solution should be simple, right? Engage early with payers in steady consultation and execution, to foster a collaborative relationship, make them a partner, get their advice, get their buy in as early as possible and get their insights on specific study designs. The next one and again, I think a lot of what I will talk about in solution is going to be some sort of derivative of this idea, about better communication, open communication, standardization, and so on. So the next one would be how do we improve payer trust and confidence in RWE. To do this, we in pharma need to improve both transparency and perceived scientific merit. And to prioritize transparency, we need to make study details such as intent, data, provenance, analytical strategy, and intended output even; being made publicly available as early as possible. And then when we provide evidence back in submission to payers, we share detailed information on the data collection, data provenance, data curation, and analyses, and the techniques we use to address data challenges inherent in RWE. And then, as I said a minute ago, right, engaging payers throughout the study design and ensuring their data requirements, their needs are met, both in terms of collection and reporting will also improve in trust in the process. The next thing that we suggest for this would be investing in payer education, and developing a universal framework or universal frameworks for the evaluation and interpretation. Now we know payers's are trained to evaluate drugs based on RCT data for the most part. But as RWE becomes more prevalent, payers need to be trained in this evaluation. But we can't assume that payers can spent a lot of resources in upskilling themselves on this, like all of us, like everyone really their resource constrained as well. So pharma needs to be actively involved in providing the tools the education and the expertise that payers need for evaluating RWE. And I think this will be a big one, I mean, for all of us across the industry, is that we need to invest in some kind of a consensus, cross stakeholder payer focused guidelines and framework on the evaluation of RWE. Whether it's standalone or in combination with RCT data, I think, to again, help payers get more educated on the best use cases, the optimal use cases, we need to engage in dialogue on all these optimal use cases for RWE and its value for payers. But to ensure payers are aware of the value provided by RWE in dossier submissions, for example, we need to ensure that they understand the implications of RWE findings as well, such as the focus on cost effectiveness, preferably on the specific payers population that you're working with. And again, the key here is education. Right emphasize Real World Evidence value after initial submission show the benefit of adopting lifecycle regarding reassessment approach, and then as much as possible for us, push real world data collection upstream and collect before initial submission, such as pre approval use, off label use, accelerated approvals, and so on as and when appropriate. And then finally, as I said, outcomes based contracts are a key focus for payers, for pharma, for all of us in terms of improving patient outcomes. And to make that more attractive to make the adoption of those more attractive pharma will need to address both the lack of the appropriate definition of outcome metrics. Right at the outset before these contracts are developed; the measurement systems as well as data availability. So we need to engage payers early to ensure the outcomes and measurements are relevant and meaningful for them as well as their patient populations. And then finally, a collaborative approach that brings together these stakeholders across the industry, from payers, to healthcare providers to patient advocacy groups, is going to be needed to define some appropriate data collection structures and ensure generation of RWE in a practical and timely manner.
Fred Goldstein 13:57
I really want to thank you for putting out this poster. Because, for me, as a population health person, it's obviously important to do randomized control trials understand that, but the end of the day, it's about what happens in the real world with products and services that we provide. And that's, you know, managing that population and measuring those outcomes. So do you have any final thoughts before we wrap up?
Charles Makin 14:19
Yeah, I mean, first of all Fred thank you and do AMCP for giving the opportunity to talk about this key topic. I think it's very, very near and dear to my heart. I think in terms of final thoughts, all I'd like to say is as the interest in the value and application of Real World Evidence continues to grow, and capability surrounding data collection and analysis become more powerful. There's immense potential for RWE to provide payers and really all stakeholders with the ability to understand the true performance of therapies on the market, by definition in the real world. So with current economic pressures and payers and the need to understand drug performance, long after initial inclusion and formularies, consideration of RWE in value assessments will be necessarily in the future, regardless of whatever the current views may be, or whatever the current capabilities may be. The barriers we have discussed have allowed Real World Evidence to reach a fraction of its potential in the US healthcare system. But by adopting some of these measures, by building trust, investing in education of its application and opening dialogues with payers to guide mutually beneficial studies, efforts from pharma will facilitate better adoption of RWE in drug value assessments. Which, in my opinion, would ultimately improve patient outcomes by allowing decision makers to make data driven treatment decisions and select the best therapy for every patient based on the full body of evidence, which, ultimately, is the goal of every single stakeholder in the healthcare ecosystem.
Fred Goldstein 15:48
Absolutely. What an insightful way to finish the show. And thank you so much for joining us, Charles.
Charles Makin 15:54
Thank you, Fred.
Fred Goldstein 15:56
And thank you for joining us today. If you like this show, you can find all our episodes at amcp.org/podcast on our show page at Healthcare NOW Radio.com or on your favorite listening platform by searching Healthcare NOW Radio. You can follow our show's social hashtag at AMCPListenUp. And don't forget to share, like and follow AMCPorg on LinkedIn, Twitter, Instagram and Facebook. I'm Fred Goldstein for AMCP. Until next time!
About the Hosts
Fred Goldstein is the founder and president of Accountable Health, LLC, a healthcare consulting firm focused on population health, health system redesign, new technologies and analytics. He has over 30 years of experience in population health, disease management, HMO, and hospital operations. Fred is an Instructor at the John D. Bower School of Population Health at the University of Mississippi Medical Center and the editorial Board of the journal Population Health Management.
AMCP Podcast Series - Listen Up: Charles Makin on RWE
Host Fred Goldstein invites Charles Makin, Global HEOR, RWE and Market Access Advisor to discuss their poster at AMCP Nexus 2023 on RWE adoption by US Payers.