Has ADAPT SMART been successful in changing the way we think about approving new therapies?

Duane Schulthess: One of the opportunities of adaptive pathways, and a key objective of the ADAPT SMART project, has been having earlier stakeholder dialogue and communication between the companies, patients, payers, regulators, and health technology assessment (HTA) bodies. At the Member State level, how successful has ADAPT SMART been in changing the way we think about approving new therapies? 

Ad Schuurman: Wearing my Zorginstituut Nederland (ZIN, The National Health Care Institute) hat, I think we’ve been one of the organisations with a positive opinion of Medicines Adaptive Pathways to Patients (MAPPs), this new way of thinking and working on a new concept. My colleagues and I think we need something like this as our current way of handling reimbursement and assessing products for reimbursement is being challenged now, and will be more so in the future.  We must begin to look for new ways to evaluate new treatments and MAPPs is one of these possible new ways.

I’d say one-third of my colleagues in Europe feel positively about this, one-third is against changing our approach, and the final third simply doesn’t know. As some payer organisations came into MAPPs late, and others were not overly active in the process, the challenge has been aligning them.

Changing process is difficult, as some payers think MAPPs is too hard politically, too complicated, or too time consuming. However, the reality is that we have to think about new ways to handle our work; I think this realisation has increased in most EU countries, but so far the daily practice hasn’t really changed. I’d personally hoped we could go for one or two real pilot projects with real stakeholders around the table in ADAPT SMART.

Right now, the only actual pilot investigating MAPPs is at the European Medicines Agency (EMA) and is primarily between the regulator and the company, with some minor input from the HTA. This was running without ADAPT SMART.

I realise, of course, that it was probably not realistic to expect an actual pilot in an Innovative Medicines Initiative (IMI) project like ADAPT SMART as it was not one of the defined goals. However, I hope that it will help create those kinds of practices in the future.

DS: How much inertia to change do you think is caused by a lack of political will and how much is legislative, i.e. where the laws don’t allow them to change processes?

AS: In the Netherlands, if we want to change something, we can also manage the legislation. I think the situation in Germany is more complicated, however I think in most countries, if you want to change things you can do it, especially when it’s a pilot project. If you’re implementing a wholescale change in regulatory assessments, well, that will be different. But for implementing an experimental pilot, most countries who want to can try things. The barriers have been the willingness, capacity, and drive to do so by the payers, and until now, we’ve not seen any companies willing to try to do MAPPs and discuss money or reimbursement.  I’m hopeful if we do a pilot that there will be companies who are willing to dare to try.

I think in many of the large companies, there is a lack of willingness to experiment. I think it’s easier to keep doing it the old way; you know how to do it, you know the market access requirements and the reimbursement process.

DS: Well, there’s a lot of money at stake. Hundreds of millions in cash has gone into a new asset by the time you reach the stage to discuss MAPPs, and the head of clinical research may not want to risk a new process.

AS: And I can understand that. So, we have to begin to say, “No. You’ll use this new process if you want to be reimbursed.” Then, I think companies will have a better reason to move into these kinds of collaborations. But as long as we don’t force this, why should they change? I can totally understand that. But until now, many payers feel they don’t need it and companies don’t feel they need it. The fact is, with many of the new products, they do.

DS: So, what do you think would be the benefit of MAPPs from a Member State perspective? What do you see as the possible upside and why more countries should be looking at it?

AS: It’s the future. One of our needs is how to handle new challenges.  We have more orphan treatments, and more personalised medicines. Many of these treatments work for a sub-population, but they also come with a lot of uncertainty. Increasingly, we cannot handle these using the old way. We will be saying, “We pay for everything” or “We pay for nothing,” but these will be lousy decisions.

I think for a regulator like the EMA, MAPPs is a very good process, it’s brings a lot of new energy and normalises a process to bring treatments to patients. For a regulator it’s good to experiment, to pilot. But the remaining challenge is to move beyond the regulator and make it a real multi-stakeholder process.  I do think that an adaptive approach is one of the ways we can go, I’m convinced about that.

DS: Ad, you see this process from three different perspectives, and you’re one of the only people that I know of in Europe who does so. You are a payer in the Netherlands, you participate in The European Network for Health Technology Assessment (EUnetHTA), and you’re on secondment to EMA. How do payers, HTAs, and regulators each see MAPPs? Do they see it the same way, or do their perspectives differ?

AS: I think EUnetHTA has a quality of organisation that allows them to facilitate the concept of early dialogue and new ways to view the evidence. They are scientists in general, and are willing to look at new ways to assess treatments and collaborate.

However, not all the members see the issues the same way. The Institute for Quality and Efficiency in Health Care (IQWIG) in Germany is less flexible and experimental, in general, than the Italian Medicines Agency (AIFA) or the UK’s National Institute for Health and Care Excellence (NICE). So within EUnetHTA there are many differences, but as a collective, they are more apt and willing to find new pathways. They also have some capacity to experiment.

If you look at the Scientific Advice partnership of EMA and EUnetHTA, it’s really professional with high-quality processes including many participating HTA organisations. It’s a very impressive result. In this area I would say ADAPT SMART, with the HTAs, has been a quite positive experience.

The payers… well… it’s been a bit more challenging.  There is of course, a difference between payers in large countries such as Germany, France, UK, etc., and the smaller ones. What we now see is more and more collaboration between the smaller countries, not only in pricing negotiations, but also in assessments and horizon scanning. This multi-country collaboration is a growing and fundamental change in Europe. We have to collaborate more, or else we’ll need to say no to our patients. This is being driven by economics.

We need to find small and medium-sized enterprises (SMEs) maybe, perhaps orphan drug manufacturers, who are willing to work with payers and HTAs to find new business models. We need to start bringing pricing negations and the money into these multi-stakeholder discussions in MAPPs. That’s still lacking at the moment.

DS: So, if you had the opportunity to make one change to facilitate adaptive pathways in Europe, what one change would you make to put MAPPs into practice?

AS: I think we need to find realistic and effective ways to incentivise companies to move into these new models. If we could arrange a few pilots, together with EMA, perhaps a small company with a compelling orphan drug, plus HTAs and payers, we could construct a common registry that could be helpful for everyone, and actually try these processes out for real.

If I had the ability to do so, and that’s one of my aims being on secondment at EMA and collaborating with the Mechanism of Coordinated Access to orphan medicinal products (MOCA), I would organise some concrete multi-stakeholder actions and collaborations to show conclusively that MAPPs can be done in practice, and demonstrate the benefits for patients, companies, and healthcare systems.

I think, at this very moment, that’s much better than another series of draft documents and written action plans. They’re good to do, don’t get me wrong, but ultimately, those won’t change anything for real.