ARTICLE SUMMARY:
Belgian health economist Lieven Annemans says Europe has fallen short in giving the most innovative new medical technologies a chance to prove themselves. But specialized innovation pathways are increasingly cropping up in different countries, and Annemans is floating a “Value of Innovation and Partnership” model that he hopes will facilitate a systematic response to the problem. He spoke to Market Pathways about his proposal.
Efforts to launch a potentially transformative medical technology often run into this paradoxical challenge: contentions that the technology can change the value proposition in healthcare need to be proven by putting it to the test in real clinical care, but healthcare systems are wary and often lack the frameworks to adopt a new, high-price-tag offering without that level of proof already in hand.
A possible solution is to find some sort of middle ground that provides runway for promising, truly innovative products, but also includes a clear offramp if the promises aren’t met. Efforts are afoot in many countries around the world to try to achieve this balance for up-and-coming medtech. In the US, for instance, there are programs like coverage with evidence development, new technology add on payments, and, most recently, efforts to get the Medicare Coverage of Innovative Technology program off the ground.
The same is true in Europe. A recent assessment commissioned by MedTech Europe identified 26 so-called “accelerated coverage pathways for innovation” (ACPI) across 10 EU member states featuring specialized arrangements that grant market access to new technologies outside of the country’s general reimbursement/funding framework, often under some sort of restraint, such as evidence requirements, utilization caps, etc.
What’s missing, at least in Europe, says Lieven Annemans, PhD, a Senior Full Professor of Health Economics at the Faculties of Medicine at Ghent University, Belgium, is a more systematic approach to the problem. He aims to help fill the gap with a proposed model called the Value of Innovation and Partnership, or VIP. Annemans laid out the concept in a recent white paper circulated by MedTech Europe, and the proposal was granted an audience during an April 20 panel at MTE’s MedTech Forum. Annemans sat for an interview with Market Pathways just before that meeting.
The VIP model seeks to establish a shared understanding of the distinct market access path that the most innovative new medical technologies should pursue. While new technologies that help sustain or improve current practices should go from the R&D/regulatory phase directly to the routine procurement/usage phase, medtech that meets the definition of “transformative” or “disruptive” innovation will need to rely on an intermediate “accessibility” phase where market access and funding is granted, but with strings attached, Annemans explains. His proposal defines the key elements of a fruitful accessibility phase, including robust real-world data sources and governance; full buy-in to value-based care payment models; and, perhaps most importantly, strong and early dialogue between industry and payors, as well as other parties, to set out exactly what questions need to be answered, when they need to be answered, and how longer-term funding decisions will be made.
Existing ACPIs have some of these components in place, but, Annemans laments, there are too few examples of all of these factors coming together in practice to support decision-making on next-generation medtech. Our full interview, edited for clarity, follows.
Market Pathways: In brief, what is the problem that you're trying to address in terms of access in Europe to the most innovative medical technologies? What are the gaps that you're trying to fill?
Lieven Annemans: We are talking about medtech solutions that claim to provide additional value to patients. I emphasize claim because sometimes it's not yet sufficiently supported, that claim. That is the key issue. I would call it evidence gaps. You're in kind of a Catch-22 situation because, on the one hand, the company says, ‘Yes, this is a fantastic technology. It should be used as soon as possible.’ The payors and the HTA bodies say, ‘Yes, but we need more evidence that your product or your technology is indeed adding value and is also cost-effective and value for money.’
“A key element in there is that we select some parameters that are crucial to document the added value of the new technology. Added value must mean something—fewer hospitalizations, fewer re-hospitalizations, shorter length of stay, etc..”
That's why we have been brainstorming on this additional phase where the product or the solution gets the benefit of the doubt; it can already be used in the market, it's already covered by the system, but it's not a blank check. There will be a point of verification in the future and, by then, based on the experience hands-on with the technology, it should be finally decided whether it's indeed of value and value for money. That's the core element of the proposal.
Right, the novel element here is, as you call it, the accessibility phase—allowing access, but with evidence collection, and other controls. How novel is that approach in Europe?
I couldn't say it's novel because there are already several proposals of temporary reimbursement—not a one-off decision, but a dynamic decision process, etc. I think the benefit of the proposal is that, for the first time, it is presented in a more systematic way, taking into account the lifecycle of health technologies, or the innovation cycle approach. We clearly describe a difference between those technologies of which added value is claimed, those that are disruptive, so to say, compared to other technologies. As you know, medtech is such a wide spectrum. There are many medtech technologies that do not really offer big added value. They are maybe marginally better. They should not undergo that intermediate phase. They could go directly to purchasing. So I think that's the benefit of the paper, that we have provided a systematic overview of what's going on and what we propose.
In Europe, there's, of course, a EU-wide approach to regulation. But when it comes to market access, health technology assessment, reimbursement it’s country by country—although there is a pending proposal for EU-level joint HTAs. Is your VIP model something that would have an EU-wide component or some alignment in how the member states operate, particularly when it comes to the accessibility phase?
Let's say we talk about an innovative solution with the claims of benefits, and there is indeed this issue of evidence gaps. We don't have all the evidence yet on that health technology. At that point in time, there could be a joint clinical assessment on the European level, but that joint clinical assessment should have the goal to come to a conclusion on whether this technology indeed has the potential to add value. It should not be a conclusion like, ‘We did the joint assessment and we find that currently there is not sufficient evidence, so it stops here.’ That would not really be a good outcome of that joint assessment. The joint assessment should rather determine, ‘Is there indeed potential value? And what additional data do we need to confirm that added value?’ With that early joint assessment, the different countries could then organize a real-world application of the technology and then enter this patient accessibility phase. That would be ideal. Europe could play a role in that first kind of assessment. I know that medtech companies are a little bit allergic to the term ‘health technology assessment’ because they are afraid that if you talk about health technology assessment, you mean a complete assessment, which will automatically, at that early stage, come to a negative conclusion. That's not what we want. We want to have more of an assessment of the potential value of that technology. In the paper, I didn't call it an HTA.
Let’s talk about the particulars of the agreements that need to be set up during the accessibility phase, ACPIs and the like. What level of detail needs to be worked out, in terms of the type of evidence, timeline, extent of access, etc?
We have this example in Belgium, where I live and work. In Belgium, typically when there are signs that a new technology is promising, but there are still a lot of doubts about, for instance, the long-term effect or the effect in real life, there are some centers in the country selected to use it, but not everybody can use it. It can be reimbursed, but the centers engage themselves to keep track of what happens to the patients treated with the solution so that, automatically, there is a registry created that then allows payors to evaluate the promises that the company makes—for instance, ‘with our technology, we avoid readmissions to the hospital.’ Well then, if that is the claim, then we should see in the registry that we have fewer admissions compared to historical controls or compared to other centers that do not yet apply the technology. There are different designs possible, of course.
But a key element in there is that we select some parameters that are crucial to document the added value of the new technology. Added value must mean something—fewer hospitalizations, fewer re-hospitalizations, shorter length of stay, etc. There should be a claim of additional benefits. That is then translated into a parameter that is followed in the registry.
And I assume this includes a defined process laid out where over a specific amount time the data will be collected, the outcomes will be assessed, and future access/reimbursement will be considered?
Yeah, exactly. It depends, of course, on the nature of the technology. For instance, for TAVI, the transcatheter aortic valve, the companies claimed that it would reduce mortality. You need two years follow-up to confirm this. Then you could say, ‘Okay, you get the benefit of the doubt for two years, or at least two years, and we will follow your product in a registry and then we can see whether it's as good as you promised.’ But in other cases, when it's about a technology used in the hospital that claims to reduce the number of readmissions, then, after a half a year, you might already see some confirmation of the benefits of that product. The time horizon depends on the nature of the disease and the technology.
What are some of the key challenges in putting these arrangements together?
I could say as a first answer, lack of trust. But, of course, as a recommendation, you cannot say, ‘Please create more trust,’ because you will not create trust like that. Trust can only be created when there is dialogue between companies and HTA bodies, early dialogue, and when the company can openly present their evidence-generation plan.
The second key requirement is good data, and good data governance because if you always have to set up a registry from scratch, we know that this will be more time consuming, more administrative burden for the clinicians and they don't like that. In an ideal system, there is good data governance, there are good databases that can be applied, there are disease registries that are automatically populated by medical record data, and so on.
“TAVI is exactly an example of how it all went wrong in several countries, where at a given point in time, there was what I would call, paralysis through analysis.”
Then, of course, there is the overall willingness of policymakers in healthcare to acknowledge and to reward good value of new technologies. There are still many, many decision-makers in Europe who are still in a cost-containment modus, and not in a value modus. Everybody's talking about value-based healthcare, but in practice in many countries, they don't apply it. They are still into cost containment, and that's not the right attitude. You need to have the right attitude that you want to reward the value. If the value is not convincing, then you don't reward it, of course.
How does value-based payment, or alternative payment models like rewarding a reduction in readmissions, work as part of this VIP model? Or, more generally, how should payment levels be considered in the context of the model and, in particular, the accessibility phase?
For instance, for a new technology a company claims a price of €10,000. They say, ‘Well, this is a unique technology. If we can show impact on mortality, etc., we claim a price of€10,000 to be covered by the healthcare system.’ Then they have to come up with a value story based on which they support that price. Because if we are talking about value-based pricing, then you have to be able to justify why you go for that price.
If it appears in the first assessment that, with the potential benefits of this product, that this €10,000 is justified, that this is indeed a fair price given the expected value, then it could enter the patient accessibility phase. It will already be reimbursed.
Then, when we arrive at the point of verification, you can have different models agreed upon. You could argue if it turns out to be not as good as was promised, then we will revise that price downwards from there—if it's not worth the €10,000. Or you could have an even more impactful model where you say, ‘If it turns out that you were not as good as promised, then the company needs to pay back money to the healthcare payor’—kind of a money-back guarantee. We gave you the benefit of the doubt. You didn't perform accordingly. That has repercussions. Of course, all of that needs to be put in a contract as from the start. You cannot come with that kind of story only two years later. It needs to be all in there.
Right. A key element of this seems to be, as you say, the dialogue and establishing a very clear-cut, formal agreement in the first place.
Yeah, the expectations must be clear. The company says, ‘These are the expectations. This is how we believe that the product or the technology will perform.’ That's the company’ claims. It's their value message. If I were a smart payor, I would say, ‘I give you the benefit of the doubt, because this is potentially really good value for the money. However, I will not give you a blank check. I want to have guarantees. I want you and your company to show in the real world that you keep your promises. If not, it will have consequences.’ All of that should be clearly put into some statements in the agreement that accompanies the accessibility phase.
In your view, what are some near-term steps that could or should be taken to move toward this model in a more comprehensive way?
The best way is to rely on early adopters who follow this way of thinking and can say, ‘Thanks to our approach, this technology is one year earlier on the market than in other countries without any financial risk, or with little financial risk,’ to convince others who are more conservative and have more of an attitude like, ‘the evidence is never enough to make a decision,’ that there are indeed better ways to approach this.
I must admit, currently in Europe, I think there are still a majority of payors and HTA bodies that think more in the conservative way, that think more in terms of, ‘I'm only interested in cutting down the price of that new technology, and I care less about the value of it.’ They're more into budget impact and that's it. You need early adopters and cross-fertilization in order to make it happen.
Are there any particular positive examples of this approach in Europe that you would point to as a good model? You referenced the TAVI experience before. Does that qualify?
TAVI is exactly an example of how it all went wrong in several countries, where at a given point in time, there was what I would call, paralysis through analysis. The payors said, ‘Yeah, we need more evidence, we need more evidence, we need more evidence.’ And, in the meantime, the company already started to convince hospitals to work with it, but they're not covered by the system, so using more creative solutions to find some money. It was a bad example. I think currently there are more examples of how not to do it than examples on how to do it.
