2026 Trends: A Year Of Change & Opportunity

Welcome to let's Talk Rare, a monthly podcast from Partners for Access, where we discuss news views and the latest insights from the orphan drug and cell and gene therapy world. Welcome to let's Talk Rare, the podcast where we unpack the biggest market access shifts shaping pharma and biotech in 2026. Today we're diving into two of the most talked about forces this year. The Joint Clinical Assessment, or JCA in Europe and U.S. most favored nation, or MFM pricing and what they really mean for global launch and access strategies. 2026 is the year when JCA moves from theory to impact. And with the first oncology and ATMP reports coming through and HTA bodies starting to implement them, and then at the same time, MFN pricing in the US is reshaping how companies think about global price corridors, launch sequencing, and even to manufacture. I mean, it all sounds pretty complicated to me. I think we need some help, Georgie. I think we really need some help. So let's make sense of it with three amazing guests, all from Partners for Access, joined by IRO Malaku, who has been closely tracking JCA readiness and early implementation. We have Sam Morrison and Angelika Melezi and they lead our thinking on US MFN and its global spillover effects. Welcome to the podcast. Thank you very much for having me. Really excited to talk about how JCA is changing the Access playbook, especially for oncology products and ATMPs. Nice to have you here. And Simon Angeliki, great to have you with us today. Angeliki, how are you doing? Great. Great to be here and to speak about mfn, which has moved from a US Only policy story to a structural global pricing issue. So it's definitely the right time to start to unpack it. Looking forward to diving into it. And Sam, you're a podcast favorite. How are you? Happy New Year. Happy New Year, Aaron. Lovely to be here again. Looking forward to the discussion as always. Yeah. Let's get into these trends, shall we? Absolutely. Let's crack on with today's conversation. So we're going to break it down for you into three parts. So the first part really is what's going on with the jca, what's happening right now. The second is how MFN is playing out in practice. And this is a really, really kind of hot trend at the moment. And thirdly, how these two trends connect and what manufacturers really need to do now, including a really quick look ahead to our P4.8 Access Summit in April again, where we'll be diving deeper into MFN scenarios. It's going to be a busy year. It is, it really is. Should we start with Europe? I think we should. Let's go for it. Yeah. We've been talking about the JCA for quite a few years now. Before, during and now kind of after. But during and 2026 is when the first real world impact is going to show up. That's what we believe. Could you give a refresher on what the JCA is and where we are right now? Absolutely. JCA is a new EU level joint clinical assessment process under which oncology and ATMP products have been in scope since January 2025. In 2026 we see the first wave of reports being published and national HTA bodies starting to integrate these outputs into their own assessments. So this is the point when it stops being this theoretical framework and it starts becoming something manufacturers feel in their timelines and evidence expectations. Ah, so let me get this right there Nero. 2025 was all around starting up the framework and now 2026 moving forward is actually when the reports are actually going to start hitting. We're going to actually see what's going on with the companies that are going through the process. Exactly. So by the end of the year the work plan anticipates assessments for approximately 35 oncology products and 15 ATMPs along with a limited number of joint scientific consultations for companies. That means that more products are going through the harmonized EU clinical assessment and there's less room for the notion of will figure access to my country later. This joint scientific consultations, it sounds powerful. Let's talk about those a bit. They are really powerful or they have the potential to be powerful for the companies involved. So the JSC provide early advice and input into the development plans so that you can align with the JCA requirements. But they do need to be scheduled well and ahead. There are certain time slots and you need to have them before your pivotal trial is locked so that combined can make them challenging to time into the product development life cycle. And there are quite limitations on the number of slots available. Okay, so if I'm a manufacturer, what does good look like in 2026 in terms of JCA readiness? Sam, the very first key thing is to monitor those reports coming out, how the assessors interpret a device, how they handle the picos and the absence of. Second, you need to align your development plans up front, endpoints, comparators, evidence generation so you can be prepared for likely pico requests. And the timelines have shifted. We really need to be thinking about these things much earlier. In the development process. Third, start planning if you've got an orphan or a rare disease asset now because you're going to face more challenges than maybe we've seen already as the data is going to be limited and standard of care fragmented across Europe. So you mentioned orphan drugs there, Sam. So the JCA for orphan drugs comes in in 2028 and really that's not far away if you think about it in developmental terms. How should rare disease companies use 2026? They should really start treating 2026 as their learning year. So key things to look out for is to follow the oncology and AT&P JCAS, look at how evidence gaps are treated and stress test how your own pipeline matches against those expectations. The goal is to turn JCA from a compliance heritage ultimately into a competitive advantage by being the company that shows up with well aligned evidence and clear justification where data are inherently limited beyond compliance. There's a real opportunity here to use the JTA as a leverage to accelerate and broaden access across the eu. I mean that's I guess the main goal behind it. So getting the dossier right first time doesn't just avoid delays, it creates momentum for downstream national assessments and supports more consistent access outcomes across markets. Absolutely. And bringing that submission to life and bringing that to a competitive advantage in launching across the whole of Europe is really what the JCA is all about. So. So we know countries will still do their national assessment in terms of price and reimbursement, but really it puts you in a position to be able to launch in markets that maybe you would have deprioritized because the amount of resources required. You can go in and talk about the price and the reimbursement structure for that country rather than starting the process from scratch each time. Thanks Sam, that was great. Should we dive into mfn? Let's do it. So over to the US market or. We've seen IRA negotiations, but MFN feels like a step change because it explicitly brings international reference pricing into the US context. Can you explain the scene, Angeliki? What does that actually mean? Absolutely. So MFN started as an executive level push to ensure the US pays no more than the lowest price available in a basket of comparably developed countries, which is specifically OCD markets with GDP per capita at least 6 of the US the 2025 executive order crystallized this, requiring manufacturers to offer the lowest price in the basket. And it is now being operationalized through a mix of negotiated agreements with pharma companies, tariff threads and purchasing commitments. And I Hear we we saw a flurry of activity after MFM letters went out in mid2025. Yes, there was a target at 17 manufacturers in July, effectively putting them on notice linking compliance with mfn, given that it was an executive order and not a legislation, and using pharmaceutical tariffs to try and encourage participation in the approach. The end of January 2026 we see 16 of those companies have signed MFN linked agreements, typically meaning that they have price reductions in the us There are commitments to extend MFM pricing to Medicaid and a commitment for domestic manufacturing within the US in return for tariff exemptions. Wow. It all sounds quite complex, Sam. And it's not really just about the list prices anymore. Some of those deals are created visible benchmarks, especially in high profile therapeutic areas. Exactly. Georgie Agreements for GLP1 medicines are a good example. These agreements established visible price points across direct consumer channels and public programs that now act as de facto reference anchors. So the result of this is that once those numbers are out there, they influence expectations not only in the US but also in other high income markets that are watching the US price level. And Sam, am I right in saying that the MFN also connects to the new Generis model for Medicaid in 2026? The generous model launched in January 2026. This lets participating state Medicaid programs access prices align to a basket of European prices. Well, mainly European prices in high income countries. This is a voluntary initiative currently, but there are a number of manufacturers committed to participate already. State involvement is also voluntary. So it sounds like MFN is going to effectively narrow the acceptable price corridor across key markets. Yeah, exactly. Historically the US sat outside international price reference. However, this brings us into a world where the prices in the US become linked to a basket of countries like the uk, France, Germany, Italy, Canada, Spain, Denmark and Sweden. So that means that those prices will directly influence the price we see in the us, which historically has been a lot higher. Wow. A lot going on in 2026 it sounds like. And I think companies are going to have to really start thinking in a different way to make sure that they're released. All of these new initiatives that are coming out, are there any behaviours that we're seeing Sam, from companies? I know it's very early days yet, but are there any behaviours that we're seeing companies early 2026 like funding from now? Yeah, we're starting to see countries talk about that and obviously reactions are still coming, but there's more cautious ex US launch pricing, especially in those basket markets. And this is combined with talk about delayed launches where the prices won't be achievable in that defined price corridor. So whether they be direct or indirect consequences on the US pricing because international price ref differencing is really complex about the interdependencies. That was before we had the US in the equation, but it's still evolving. So I guess we continue to watch closely and I'm sure so will our clients. Yeah, I think that's definitely one to watch guys. So do look out for posts and things coming out for partners for access as we follow this throughout 2026 and beyond. So we've talked about the JCA, we've talked about the MFM for a global market access team. These are all happening at the same time. How do they interact with it all? I mean just thinking about it, it's a lot of work for companies now to really start thinking about. So yeah, how are they going to navigate this going forward? So the way these initiatives interact is that they now push towards the need for a more integrated front loaded access strateg. JCA means that pharma companies need a coherent EU level clinical and evidence story that will feed multiple markets, while MFN means that the pricing in key ex US markets can directly impact US outcomes. Essentially, pharma companies cannot treat Europe and the US as separate sequential decisions anymore. Yeah, really. And both trends increase the value of early cross functional planning. I can't stress that enough. For example, the decisions about EU trial design and parenter choice will influence your JCA outcomes which will in turn influence your pricing in those EU markets which will then feed into the US pricing if they're being referenced. So it becomes a real global issue with a misstep in any region potentially having implications globally. I'm just saying if I was in charge of setting all these things at a pharma company, my head would be going bong because it just sounds so complicated and difficult to get all these things together at one time. It's a real challenge. Good luck to you out there. Iro. If you had to give one 2026 must do for our listeners, what would it be? My 2026 must do for the JCA would be to build a systematic internal process that monitors the JCA reports as they come and extracting those learnings into pipeline planning, making it a standing governance item rather than an ad hoc readout. Love that. What about you Sam? Yeah, for mfn, I think really stress test your global pricing order. Think about the launch sequence against both those MFN considerations and the generous model scenarios. See what happens with the pilots. Watch this space closely. Massively. Absolutely. So guys, if you really are struggling with the new MFN legislation that's come out, it takes in something really, really exciting that I want to tell you all about. So this year Partners for Access is hosting its second Access Summit and this year it will be in April in Zug and we're going to be really focused on MFN and global pricing strategy. We'll be running in live scenario workshops and really how we interact with the EU reforms, including jc. So if you're interested and you want to learn more and you want to come along and be part of that, we'll send out all of the links with the podcast description on how you can register and get involved and come along and learn more. Yes, if today's discussion has raised more questions than answers things, you do that for me. But anyway, how to set your price corridor, whether to delay certain launches, how to interact JCA evidence needs with US policy risks. The P4I Access Summit is where you can sit down with peers and P4I experts to work through these concrete cases. We'll also be sharing our latest white paper trends in 2026 and beyond. So do look out for that, which will also include MFN and JCA articles. Absolutely. So to find out more and register, make sure you visit partnersforaccess.com and look out for the P4.8 Access Summit April edition. Spaces are limited guys, so if you are responsible for global market access pricing launch strategy, you'll really want to secure your place soon. So don't miss out. Thank you so much for your time today. Huge thanks to Sam IRO and Angeliki for joining us. If you found the episode useful, subscribe and share with your teams and join us in April to continue the conversation live. Thanks for listening. Thanks everyone. Thank you for listening to the discussion. You can find more information about this subject and other matters relating to rare disease and cell and gene therapies on the Insights section of our website www.partner us for access.com.