November 4, 2026
This is a lightly edited and condensed description of the podcast episode. It is intended to summarize the substance of the discussion, but it is not a verbatim transcription. Language may have been edited, reorganized, or summarized for clarity and readability. For exact wording and the full context of the discussion, please refer to the original audio recording.
Welcome to Perspectives, a signature podcast series from The Leerink Center for Pharmacoeconomics. Hosted by Dr. Mel Whittington, a health economist and Head of the Center for Pharmacoeconomics, we will be hearing from individuals across the industry to better understand and appreciate the societal impact of healthcare innovations.
Mel Whittington: Welcome back to Perspectives, everyone. I’m Mel Whittington, and this is the podcast where we try to look at healthcare innovation through a health economics lens. I get to talk to really great people, like today’s guest, hear what is happening, and then try to figure out what it all means for those who are building and backing new medicines.
The topic today is a good one. We’re going to get into two major changes in the European pharmaceutical policy landscape. First, we’ll talk about JCA, or joint clinical assessment, and hear how this changes the way clinical evidence is assessed in Europe. Then we’re going to zoom out to the EU pharma package and hear how this changes some of the incentives for drug development. We’ll close by attempting to bring in MFN—most favored nation pricing—and bring the U.S. back into the conversation to figure out how MFN might fit into all of this.
Today’s guest is Professor Graham Cookson, the CEO of the Office of Health Economics, or OHE. Graham, can you kick us off by telling us a little bit about you and the great work of OHE?
Graham Cookson: Sure. Thanks for having me, Mel. I’m an economist who focuses on health, and I like to make that distinction from a health economist. As you said, I have the great pleasure of leading the Office of Health Economics, which is the world’s oldest independent health economics research organization. We’ve been going for—what are we now—64 years?
Mel Whittington: Wow.
Graham Cookson: We’re in the UK, and we’re a registered charity. For people not familiar with that, it essentially means we’re a not-for-profit. But while we’re based in London, we work globally, across most countries, actually.
Personally, my work focuses primarily now on the economics of pharmaceutical innovation. Right now, the big focus is around the global effects of U.S. policy decisions—MFN, as we might get onto at some point. For your listeners, what I’m working on is really where a lot of the commercial risk for biotech exists right now. I’m hoping they’ll find today’s conversation valuable.
Mel Whittington: Without a doubt. I loved how you said you’re an economist working on health. I’ve always had a little bit of an identity crisis: I wouldn’t consider myself an economist, and I wouldn’t consider myself a health economist. I like to say I’m a pharmacoeconomist. At least that’s what I like to say right now. That’s shifted some.
I’ve had the pleasure of working with the OHE team on a few things. It’s a great team, and I’m excited to pick your brain on these topics today. Again, thank you for being here.
Before we get into our topics, why do you think this audience—biotech executives and investors, more broadly—should be paying attention to European pharmaceutical policy right now?
Graham Cookson: I think pharma policy in general sets three things that are critically important for investors: the evidence they’re going to need, the length of protection they’re going to get, and, ultimately, the net price they’re going to earn. Right now in Europe, all three are moving at once. It’s quite an unusual and interesting time.
The EU is adopting some of the biggest changes to medicines policy and law in pretty much 20 years. JCA is now producing real reports about real products. We all know that the U.S. is trying to reference many countries’ prices, including many of those in Europe. Even if your primary launch market is going to be the U.S., acquirers and investors will still value that European tail of revenue. These big changes will therefore impact valuation models.
Mel Whittington: I think we just got our sound bite for this episode. You mentioned this a little bit, but I want to reiterate it: How significant a period of change are we in right now?
Graham Cookson: This is where I get to be an academic and sit on the fence. In many respects, these are the most significant changes in 20 or 25 years. They’re really big, but at the same time, they’re staged. We’ve got to be careful about how quickly they’re going to impact the life sciences sector.
The key thing that makes this unusual is that we’ve got three reforms landing together and pulling in different directions. That’s the real tension. JCA right now is focused on a narrow set of products. By 2028, that widens to orphan drugs; by 2030, everything’s in.
The pharma package, which we’re going to get onto, technically still needs a parliamentary vote. For those who aren’t familiar with the EU wranglings, there’s a period of what we call national transposition, when individual countries translate that into their own laws. That could take up to two years.
Then there’s the big thing: MFN. We know it’s contested. We don’t have a settled position, so the impacts are uncertain. The bottom line is that the direction is clear, but the timing is going to be uncertain.
Mel Whittington: Got it. Let’s dig into each of those things, starting with JCA—joint clinical assessment. For someone investing or working in biotech who has probably heard of JCA but doesn’t live and breathe it like you and your team do, can you simply describe what it is and what it’s intended to do?
Graham Cookson: Sure. It starts with this fundamental question: Is this new treatment worth paying for? Before JCA, every country answered that question on its own, assessing essentially the same clinical evidence but with different questions and different formats. That duplicates work for companies, and it’s inefficient in terms of wasting time for the various health technology assessment bodies in each country.
JCA theoretically moves that clinical part to the European level. There is meant to be one joint report on relative clinical effectiveness and safety. But for your U.S. listeners, I’ll stress that this is not about the regulators. This isn’t the regulator’s job. The EMA—the European Medicines Agency—like the FDA, decides whether a medicine is safe and effective enough to be sold.
JCA asks a slightly different question: How much better is it than what patients get today, against the standard of care? Broadly, we call that health technology assessment, and technically there’s no formal equivalent of that in the U.S. The closest, even in Europe, is Germany’s IQWiG, which assesses added clinical benefit. Many people will have heard of NICE in the UK—technically, in England—which goes a step further because it adds cost-effectiveness.
But JCA is only the clinical part. It doesn’t cover cost-effectiveness, pricing, or reimbursement. Those are all national-level competencies.
Why is it here? What problem is it intended to solve? Honestly, it’s really about duplication. Technically, there are four stated aims: reduce duplication, strengthen cooperation, improve quality and consistency, and support timely and equitable access. But fundamentally, it was about duplication.
The interesting thing for me is that what industry really wanted was for JCA to replace the national clinical step rather than add to it, so it would genuinely remove that duplication. What they wanted, therefore, was for countries to be forced to apply that report—to use it. But when JCA was developed, they lost that argument.
Right now, member states keep their flexibility. The final law only requires countries to give the report “due consideration.” We’ve got shared clinical assessment, but without any guarantee that countries are actually going to rely on it.
Mel Whittington: Interesting. Let me try to summarize this, and tell me what I get wrong. EMA regulatory approval is not what JCA is. JCA is really focused on what the medicine could be worth—comparative effectiveness, things like that.
Health technology assessment—we don’t have a centralized body in the U.S., but it is very common outside the U.S.—looks at comparative effectiveness and can then do cost-effectiveness, things like that. Is it fair to say that JCA is really focused on that clinical evidence and comparative effectiveness piece? It’s not necessarily cost-effectiveness or what price the drug should be, that kind of thing.
Graham Cookson: Exactly right. It comes after the EMA or FDA kind of level. It’s just about clinical effectiveness, and it doesn’t touch the all-important economic evidence, pricing, or reimbursement. None of that.
Mel Whittington: And the economic evidence, pricing, and reimbursement—are those still at the individual country level?
Graham Cookson: Yes, they are.
Mel Whittington: One thing I try to do with guests of this podcast is have them put themselves in the shoes of someone running a development-stage biotech company. If you were the CEO of a development-stage biotech company, when should you start thinking about JCA?
Graham Cookson: Unfortunately, because I don’t think anyone really wants to hear this, it’s earlier than anyone actually does. If there’s one thing to take away, it’s to get ahead of it. You should really be doing this when you’re designing the pivotal program, because by the time the JCA scope is set, your trial is already fixed. You’ve got to get in there early.
If you’re in oncology or ATMPs, JCA is active now for you. By 2028, orphan medicines will be in scope; by 2030, basically everything. A company starting a Phase II program today should assume it’s going to be in JCA, no matter what it is.
The practical route in is a joint scientific consultation, which is a voluntary dialogue that can run alongside EMA scientific advice. You can do it in parallel. We’re already seeing some countries turning off national scientific advice. France no longer does that, focusing on the joint route. I think other countries are going to follow suit over the next couple of years as well. So start early.
Mel Whittington: Start early. It goes to all drugs in 2030, so if you have pipeline products, it’s good to be thinking about it right now, especially before the pivotal program.
How should they be thinking about it? I hear you that it should largely be on the clinical development side. Is this comparator selection, endpoints, subgroup analyses, overall trial design? Are there specific tips for how they should be thinking about it, in addition to when?
Graham Cookson: The direct answer is that you’re designing for many different standards of care, not one. This is the real challenge, and it’s what the evidence from the early reports is showing.
Member states set the comparators because standard of care differs from country to country. You can face several comparators, which is a real problem. You can either run your trial against the comparator that matters most or plan credible indirect comparisons for the rest.
Endpoints need to be the ones assessors see as patient-relevant: survival and quality of life. Surrogates are going to need justifying. Subgroups are going to have to be predefined, not found after the fact, and the trial population has to look like European patients on European care. It largely makes sense to be recruiting from Europe. That’s really more about standards of care and different treatment options than things such as ethnic diversity.
If we think about the first JCA, it was published in June for Ipsen’s product for a brain tumor—low-grade glioma in children. It’s an interesting one to start with. It’s a really hard case because you’ve got a rare disease, a single-arm trial, and biomarker-defined subgroups.
It shows all the challenges, because for that one tumor, member states asked for eight PICOs. I’m sure your audience knows that’s population, intervention, comparator, and outcome. Eight PICOs across three molecular subgroups, and a single-arm pivotal trial managed to get one of them. Then the evidence narrowed again, with fewer than 20% of enrolled patients in the relevant arms considered representative of that patient population. Remember, it’s about standards of care. It becomes a fraction of a fraction. We’ve done some work on this. By the way, for your listeners, everything we do is freely available on our website. I’m sure everyone is already subscribed to our mailing list so they get it.
Mel Whittington: If not, they should be.
Graham Cookson: One of the arguments we make is that precision requires precision on both sides of the comparison. We’ve got to get better evidence generation, not lower standards. We need to think about different trial designs—basket, umbrella, adaptive designs, et cetera. But honestly, structured real-world evidence is going to be key to trying to fix this, because it’s pretty hard.
Mel Whittington: When you first talked about JCA and this duplication piece, in my head I was thinking, “Oh, does this make European market access simpler? Are we reducing duplication and streamlining things?” Then we talked about variable standards of care. So does this make European market access simpler?
Graham Cookson: Probably not—not yet. You’ve probably got to be cautious about promising it ever will. It simplifies the architecture because there’s one clinical dossier. But it doesn’t simplify any of the science. As we started to get into, it’s actually really hard because you’re trying to do all of that but put it into one place.
The first report was over 150 pages for the single PICO it managed to do. They wanted seven others, and the full manufacturer’s submission was longer. Even when you do that, you’ve still got the national processes afterwards. Remember what I said: They have to give it due consideration, but they don’t actually have to rely on that report.
Mel Whittington: So it’s not replacing that national piece. It’s adding in.
Graham Cookson: Right. Let’s give them the benefit of the doubt. It’s early days, and we know this is a very risk-averse sector. But even the agency’s own verdict on the first four reports is that while the system is feasible and the quality is defensible—yes, the foundations are there—we’re still a long way from seeing this as a simpler, faster process.
The first four assessments each took between a year and 16 months. That’s not a definition of speed, right? There was a meeting recently of some of the chiefs of agencies, and they also raised the capacity question. Spain said they would want to do more of these JCAs—they’d like to assess more of them—but it takes too long, and they don’t have the capacity to do it.
A process that’s meant to lighten the load is actually heavy for HTA agencies. It’s quite risky for companies, because you’re going to try to do this once.
Mel Whittington: Yeah. I liked what you said about a simpler architecture, but that doesn’t necessarily translate into a simpler or faster process. That’s really well said.
Shoot, I have so many questions about this, but we have other topics we want to get into, too. Wrapping up on JCA, how does this change the economics of drug development? If we bring this back to investors and biotech CEOs, does this—I’m going to call it an additional process of European market access—change the economics of developing a drug for Europe?
Graham Cookson: I think it does, but probably not in a really positive way right now. It certainly changes costs and risks more than it does revenue. For small biotechs that might be tight on funding, those costs may go up, but they’re also going to hit a lot earlier because you need to start doing this comparative evidence early. That impacts the investment side and how much cash they’re going to need.
The risk also goes up because a gap in your comparative evidence is not just in one document, where you can move on to another country or member state. You can’t learn by doing anymore. If it’s in that document, everyone’s got it. That’s a big problem.
There is an upside: You stop writing the same clinical dossier many times. That’s the duplication point. But essentially, JCA is not interested in reimbursement prices, so it doesn’t even guarantee reimbursement. The net effect on a product is essentially going to be modest next to the big things you care about, like price and exclusivity.
Our latest report argues that uncertainty about the reimbursement population could weaken incentives to launch and invest in precision medicines in Europe. But—and it’s a really big “but”—if JCA speeds up those national decisions, then that time to earlier access could outweigh the extra evidence costs. My big caveat is there is a “but” in massive letters.
Mel Whittington: And we haven’t seen that yet.
Graham Cookson: We haven’t got any evidence there yet. Theoretically, this could help, but right now, at the stage we’re at, where we’re still learning how it’s going to work, I wouldn’t say it’s net positive for small biotechs.
Mel Whittington: Got it. We’re going to switch to the EU pharma package. But if you would be willing to share some of your published resources on JCA, I can link them in the show notes. I feel like we just put out so many great teasers. I know I want to dig in more, and I’m sure the listeners will, too. Hopefully, we can link those.
The EU pharma package: What is it? In my limited research, it seems like there was a lot in there. Could you identify two or three things that biotech executives and investors should really understand about this package?
Graham Cookson: I’m going to take the biggest number you gave me, so let’s focus on three things I think matter most. Again, from a European perspective, this is probably the biggest thing in about 20 or 25 years. We see three big changes.
The first is to the baseline protection structure. We’re getting eight years of data protection plus one year of market protection, with some extensions for various criteria, but they’re not given.
Second, around orphan drugs for rare diseases, there’s going to be lower baseline protection, which is not good news, but then a premium for breakthrough products. We can get into whether that’s realistic in rare diseases.
Third, a new transferable exclusivity voucher for priority antimicrobials.
The good news is that the original proposals were significantly harsher, and they have definitely been softened. But these are not net positive generally for industry. I think there’s also a major tension with MFN that we might get into.
Basically, the big goal of the EU pharma package, from the legislators’ perspective, is to encourage broader access across European countries. It’s going to combine incentives with punishments. We’ve got carrots and sticks going everywhere. But the big problem is that the obvious response to MFN for a lot of companies is to wait and see. That may mean delaying or slowing launches. That fundamental strategy runs against the whole point of the EU pharma package.
Mel Whittington: You talked about this a little in your opening remarks. Where does the EU pharma package stand today? Is this a done deal? Is this for sure happening? Are there still things that have to be decided, or when should we expect this to happen?
Graham Cookson: The Council formally adopted it in September this year. That was following the political agreement on the substance last December. The substance, the content, is fixed. Now we’re into the implementation stage.
As I said, it still needs a vote in the European Parliament, which—I think; someone might correct me—is in November. It then technically gets published in what’s called the Official Journal. That’s the signal to individual member states to transpose that directive into their own national laws.
That can take different amounts of time, depending on how technically difficult it is in the country and how much they want to. But basically, it takes about two years. The key thing is that the content, the law, the substance is settled. But when it actually impacts will vary based on when it’s adopted by countries.
Mel Whittington: That’s really helpful. It’s happening; how and when may still be to be determined.
The word you used when first talking about the EU pharma package was “incentives.” It impacts incentives around things like unmet need and orphan drugs, which are right in the lane of this podcast. Can you spend a little more time on how these incentives are structured in the package and what that might mean for development decisions? I’m particularly stuck on your comment about the rare disease and orphan drugs piece.
Graham Cookson: Two things are changing. The floor falls, and then the extras—the add-on protection you can get—are going to matter more. Baseline market protection drops from two years to one, but the overall ceiling is pretty much where it is today, at 11, depending on whether you earn any extensions. If you don’t, you’ve probably lost about a year.
What really happens is a shift from guaranteed protection toward earned protection. As I said, it’s trying to incentivize and reward particular types of assets. From an investor perspective, arguably, for most assets, patents and SPCs are the binding constraint anyway. It’s going to have a modest effect on average products, but I think there’ll be a wider spread between winners and losers.
Mel Whittington: Got it. The incentives are a little differentiated based on breakthroughs or unmet medical need.
Graham Cookson: For orphan products, the baseline falls from 10 to nine years. Then there’s a premium back up to 11 years for what they call breakthrough products. That’s pretty narrowly defined: No product is already authorized for the condition, and the medicine delivers a clinically relevant reduction in morbidity or mortality.
That can be quite challenging for orphan drugs, because new indications for existing orphan medicines will no longer earn separate exclusivity. A lot of orphan drugs are first developed for one indication and then applied to different areas. It really tilts that incentive toward first-in-condition medicines and away from sequential indication strategies.
The problem is that’s not really what happens in rare diseases and orphan medicines. I know what they’re trying to do here: It’s really trying to incentivize and reward breakthrough innovation. But I wonder, can it have an impact when most of the world is set up to understand what the U.S. values? It’s important, but it’s really secondary to what’s happening in the U.S.
Mel Whittington: Which is why we can’t have this conversation without bringing up MFN. But I have two more questions before we get to MFN. I again want to put you in the shoes of a biotech CEO. What should you be doing differently considering the EU pharma package? The content is going to hold, with implementation and timing still to be determined, but is there anything they can do differently to prepare themselves better?
Graham Cookson: I’m going to talk more broadly about how these interact, because I think it’s helpful. First, as I said, you’ve got to plan evidence generation for the regulator and for JCA from the start. Using the joint scientific consultation, if you’re in scope, is important.
Then, back to what we said before: Design that comparative evidence and plan for recruitment in more than one member state, because that actually counts now toward extra protection. Focus on unmet need, and treat orphan status as something to plan for, not assume. Is it going to apply? Will that help you?
You’re going to have to model European scenarios for different launch orders and net prices. I wouldn’t be building the European case on a single list price because of MFN.
The bottom line, though, is that it’s making things really hard. The interaction of JCA, the EU pharma package, and MFN means the right answer is really asset-specific.
Mel Whittington: Even though it’s asset-specific, which I completely understand, is it fair to say that the European development or launch strategy is different than it was before JCA, the EU pharma package, and MFN?
Graham Cookson: Yeah, because they’re pulling in opposite directions. Because of JCA, you’ve got to think really early about evidence generation and plan to do it effectively at scale from the start, not incrementally, market by market. You’re not just thinking, “Let’s think about Germany now. We’ll do everything else later.”
Similarly, with the EU pharma package, what they really want is fast and broad access across Europe. They’re expecting, incentivizing, and almost demanding that as well. Again, a piecemeal, market-by-market strategy isn’t going to work.
If you want to extract the maximum protection under the EU pharma package, you’ve really got to think about whether you can demonstrate that unmet need and get orphan status. It’s really: Start early and think broad.
Mel Whittington: Let’s bring MFN into this. How does the U.S. interest in MFN—most favored nation pricing—change everything we’ve just discussed?
Graham Cookson: It really raises the stakes for each and every European launch decision, because most European prices feed into U.S. exposure. Even the ones that aren’t in the basket may well be referenced by another country in the basket. It makes that local decision a global decision. Given how important the U.S. market is for many companies, they’re going to have to really think about that.
Generally, my view is that I don’t really see MFN as a pricing policy. I think it’s a stress test of global innovation financing and how we deliver fair shares, rather than some sort of coherent pricing policy. Putting my economics hat on, the pricing model doesn’t really fit with the economics that we use to justify it, which should be based on differential pricing.
The bottom line is that companies essentially have three responses, which I’ve talked about before and call “delay, run, or hide.”
Delay is what most companies are doing, even if they don’t admit it. There’s big option value now in waiting to see what happens. The U.S. is still the big, dominant market, and Europe is still the tail. Let’s see how the policy plays out. Let’s see what the member states do about it.
Second is what I call “run,” which is a more extreme version of that: Don’t launch in many European markets. There’s some evidence, for example, that in rare diseases, we are seeing products not come to market.
The third strategy is what I call “hide.” We’re not clear that it’s going to be an effective strategy, but by that I mean hide the price. What tools can be used to effectively disguise the net price? Is it greater use of outcomes-based contracting, or bundling products and services? What would obfuscate a clear net price? Whether that’s ultimately effective is uncertain, but those are the three strategies.
Mel Whittington: I like that summary. I would say “delay, run, or hide” sharply contrasts with “fast and broad.”
Graham Cookson: They’re pulling in totally opposite directions.
Mel Whittington: Yeah. The EU pharma package is intended to be fast and broad. With MFN, if the strategy is “delay, run, or hide,” this is a tension.
If you put the three things we’ve talked about—JCA, the EU pharma package, and MFN—together, what should biotech executives and investors be paying attention to? What should they be doing?
Graham Cookson: The thing I think we should all be paying attention to is this gap between who sets European prices and who carries the consequences.
Member states still set prices under tight budgets, and they’re all claiming affordability crises of various scales. The EU sets the evidence and protection rules. The EU is the body that negotiates trade deals, and trade is being used as a lever to get higher prices. Then, over in Washington, we’ve got this big impact, allegedly, on net prices through trade policies.
At the heart of all this, many European countries are big life sciences hubs and want to protect that. They’ve got an industrial sector policy. There’s this trilemma.
The most constructive solution, I think, is some level of convergence, but without uniformity. Reference pricing is a really bad idea. We shouldn’t all be paying the same price. There’s no reason for that. But I think a lot of European countries do have to accept that they haven’t been paying their fair share of the innovation costs of medicines.
I’d like to see more thought about broader value in value-based assessments across Europe.
If we don’t see Europe moving on some of this, for biotechs, I’d be planning for Europe to be sequenced much later. I know even some large pharmaceutical companies are thinking about that. They’re also asking whether markets that have perhaps been overlooked historically—those outside the basket—could make up for the losses they may see by not launching in Europe as early. We might even see a global reordering.
The last thing—what’s happening with China? I think the U.S. has gifted China this opportunity. There are a lot of policymakers in Europe saying: If the threat from the U.S. is that if we don’t pay higher prices, we don’t get new drugs, then what about China? Will they be riding in on a horse offering us gifts?
It’s also a really big market for biotech. Again, if Europe is problematic, is launching in China an opportunity as well? Lots to think about. It’s not going to be a quiet period for anybody.
Mel Whittington: Lots to think about. I love how that just closed this episode. I’ve learned so much. This was great. Your perspective was brilliant, and I appreciate you sharing it with us. Please send me some of the resources OHE has put out so I can put them in the show notes and everyone can access them. Thank you so much.
Graham Cookson: You’re welcome. Thanks for having me on.
Show links:
- Overview of JCA: One Europe, one assessment? Unpacking the European Joint Clinical Assessment – OHE
- Lessons learned from the first JCA: What the first EU JCA reveals about assessing precision oncology – OHE
- What JCA might mean for orphan drugs: Global rare‑disease policy shifts: what are the implications for equitable access to orphan medicin…
- A global impact framework on MFN: MFN’s global ripple effects: what they mean for manufacturers, payers, patients and policymakers – Change Initiative
- Access considerations in the shadow of MFN: Global equity in the shadow of US pricing reform: Access by bargaining power? | Insights from a WHS side event – OHE
- US pricing negotiations with Germany: Germany’s drug-pricing dilemma: Why Europe’s pivotal market holds the key to the MFN debate – OHE
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