The European Medicines Agency, ECNP, and New Frontiers

Steffen Thirstrup is the chief medical officer at the European Medicines Agency (EMA) in Amsterdam. He is on the programme committee of the 2024 ECNP New Frontiers Meeting on 17-18 March in Nice and also helped design the 2023 ECNP New Frontiers Meeting on psychedelics. Here he is interviewed by ECNP press officer Tom Parkhill.

TP: Tell me a bit about yourself, where you're from, how you came to your current job, and what your role is?
ST: I'm Danish. I'm a medic, I graduated 30-something years ago, then I did basic training. I did a PhD in pharmacology and went into internal medicine. I became a specialist in clinical pharmacology and therapeutics, spending roughly ten years in clinical work. After that I joined the Danish Medicines Agency where I got involved in the European regulatory system. I then became the Danish representative on one of the committees here at the European Medicines Agency.

After a while I became the head of the Danish Licencing Division, which meant I couldn't spend much time on the international work. Roughly ten or 12 years ago, I left the Danish agency to become a consultant for industry, guiding them on how to navigate the European regulatory system. Eventually, I saw this position at the EMA advertised and thought, “this actually sums up everything I've been doing for most of my career in regulatory science; it would be an interesting position to come back to”, and I was lucky enough to be selected. I’ve been in the position now for two years. I'm based in Amsterdam, although my wife and family are still in Copenhagen. So I commute a lot, and of course there’s an option for teleworking which helps greatly.

At the European Medicines Agency I’m an advisor to the to the executive director. This means giving medical advice on a variety of topics and fundamentally keeping an eye on what is happening, both in-house in terms of products undergoing review and, in particular, on what is happening in science around us. For example, I need to be able to explain our decisions around products but also to create and communicate awareness, like the awareness around psychedelics in the media, where the FDA has taken action and granted fast-track review to psilocybin. But I need to be aware of what is actually happening in Europe and what the EMA should be doing: should we better explain our thinking, should we start considering what could be the regulatory pathway? And so forth.

That's interesting you mention psychedelics because I wanted to talk to you about that, because, of course, they're a good subject for the media.
Psychedelics have a history as well. I think some of the reasons why these products are restricted is not so much based on science, it's maybe more politics and Nixon's war on drugs back in the late 60s that caused them to be classified as restricted drugs. Of course, they have side effects like every other drug, and there’s a potential for dependency or addiction, although as far as I know, this is far, far lower than we see for opioids and other central nervous stimulants.

Generally speaking, I think CNS drugs, in particular drugs for mental disorders, have not been getting that much attention in recent decades. We don't see many new products coming along in in that field. Psychedelics won’t be the solution to everything, but they could potentially offer an option for treatment-resistant depression, PTSD, and maybe other areas. We see a lot of activity going on in Alzheimer's disease and dementia, but there's also a challenge with these products in terms of how big the effect is or whether we are actually able to diagnose patients at a sufficiently early stage so that they can benefit from the treatment, or are we actually intervening too late? So there are a lot of relevant questions. And then of course there are issues on pricing and so forth. But pricing and reimbursement are not one of our key responsibilities; that’s left for the national agencies to deal with. We are aware of this, and we are aware that we sometimes approve products that in the end may not be affordable for all healthcare systems across Europe. But we haven't seen much innovation in a lot of other mental health areas in many years, so there's definitely an unmet medical need and a research need. Mental health doesn’t have the extreme industry interest you get in some areas such as oncology. There, there is constant development, I would say; more than half of the new products that we review for marketing authorisation are in the oncology area. Cardiology also suffers in comparison to oncology. It is the second, if not the primary, cause of death in Europe, but there is not that much development in cardiology either.

As regards mental health, we do not really have a detailed understanding of some of the diseases, and we might not be able to diagnose them at a sufficiently early stage to make a meaningful intervention – dementia, for example. So there is a lot of basic research needed there. There might also be an issue around the return of investment for industry: is society willing to pay for the advancements that that industry is generating? Sometimes you can see this in the struggles around the Alzheimer medications, where the benefits might be marginal, but the prices are definitely not marginal.

Also, there may not be so much public awareness about mental disorders. Oncology is a big thing, cardiology as well. I don't think mental disorders always get the public attention that they deserve. So we have a mix of reasons why mental health seems neglected. Maybe it's a less sexy area in comparison to many other areas, but we definitely need to do something.

When I started working for ECNP ten years ago, the first thing that people said to me was there were very few drugs which have come through recently in mental health. And essentially, aside from the psychedelics, we're still in the same situation. There are few of what you might call “conventionally developed drugs”. Does the EMA see that it actually has a role in taking the lead, in making things easier for drug companies to develop drugs in neglected areas?
We do see ourselves as having a role in identifying underserved areas, and in stimulating innovation – of course within the limits we have in terms of framework and legislation. I think one of my key messages to developers in academia and industry is to always come to us as early as possible. You see that in the USA, many drug developers go to FDA at an early stage; they can have a very fruitful interaction with the FDA. But sometimes they forget Europe and when they come to Europe, they come to us with something that has developed in full collaboration with the FDA, but without thinking about Europe and EMA and how we might have different expectations in terms of how they do the trials. Ultimately, we may have some concerns from a European perspective that you don't see in the US. So my main message is this: we want to engage and we want to engage as early as possible.

Industry is one thing, but of course we also want to engage with academia. Many of the promising products emerge from academia via spin-outs, and not all academics are aware of the regulatory world. So it would be very helpful if they could have an early awareness that what they are doing could potentially become something that will be regulated. We have people who deal specifically with academia, and we have a lot of people focusing on small and medium-sized enterprises, smaller companies that have limited budgets compared to big pharma. We aim for good, early engagement and that's part of our goal in stimulating innovation.

I was looking at the EMA website earlier and I see that you have a list of around 30 partner organisations, academic organisations – like ECNP. How can an organisation like ECNP work with the EMA?
We have a lot of good collaboration with external organisations, and the number of these contacts is increasing. We also have a good collaboration with patient communities. It's important to have this interaction to understand what is going on in the real world. This is an important discussion – making sure we don't get lost in an ivory tower where we make decisions out of sync with what is happening in the surrounding world. It's also a good vehicle for us to engage with societies, to bounce off ideas. So for us it's a listening and learning opportunity, and a chance to include some of these members as experts in our decision-making process.

So there should be a high level of contact between the EMA and the various societies?
Yes, if we just consider ourselves as gatekeepers, then we could get lost in the laws and regulations that regulate our field. But we need to understand the fundamental issues, and we need to be able to engage with experts who have their finger on the pulse in that research area. We spend a lot of time building up engagement with healthcare professionals and patients – we also need to engage with the patient societies.

So how would you do this in mental health?
We try to engage with the patient organisations which take an umbrella approach, since it would be too demanding for us to engage with disease-specific patient organisations. For example, we work with the European Brain Council both to understand the issues, and to include them in our decision-making process. We actually have patient engagement in all our scientific committees, and we have dedicated working parties for patients/consumers as well as healthcare professionals. Increasingly, we see patients as being integral to our processes, because sometimes, both as regulators and as scientists who are involved in developing products, we forget to ask what really matters for a patient with a certain disease. If we haven't engaged with patients, haven't talked with patients, we do not really know what matters to them. So that's equally important for us and we have come to realise that over the past decade or so.

You mentioned disease-specific areas. I see that you have developed new guidance on the development of medicines for depression. You're looking for some feedback by the end of March.
It's an update of on our regulatory guidance for depression.1 These are guidelines for industry on how to develop products for different areas, so they are not treatment guidelines. One of the updates in that depression guideline is to take the psychedelics into consideration. We know the FDA has developed specific guidance on psychedelics. I think we wanted to be more modest and start by including it in our depression guideline. This is also based on what we have learned from approving Spravato (esketamine) for treatment-resistant depression. So it was timely to come forward with an update of that of that guideline.

You are on the programme committee for the ECNP New Frontiers Meeting Nice this weekend. Tell us about that.
I got involved with the organising committee for the psychedelics meeting last year. I think the meeting was a good one, at least I learned a lot, listening to people coming from a totally different background than me and also people who had patient experience with psychedelics. Then ECNP asked me whether I would like to be on the organising committee for this year and I said yes. And to be honest, my engagement has been limited and I’m not able to go to the meeting itself due to other commitments. But I think the idea of looking at the diagnostic framework for psychiatric diseases is the right one. For a while, we've seen in oncology a change in focus from considering site and organ-specific cancers. This is still there, but we have come to understand that the diseases need to be considered at a much more detailed level, even at a molecular level. Now many cancer treatments actually target a specific molecular site that you can find in cancers at many different anatomical sites. So we are already moving from talking about lung cancer, breast cancer, kidney cancer and more to talking about cancers that have a specific molecular target. We have started to have a similar – or at least parallel – discussion for neural conditions and psychiatric diseases, where a better biological understanding could be used for diagnostic criteria. And that of course ultimately would have an impact on how we as regulators would look at potential new products for these diseases. So this is an interesting discussion. I hope the New Frontiers Meeting will help bring this out.

Any final thoughts?
To be honest. I didn't really know much about ECNP before I engaged with them on the psychedelics meeting last year. This was a very productive meeting; we’ve had a good collaboration. In fact, earlier this week a couple of papers came out of this: one looking at the very early development and very basic chemistry and so forth, and then there's a section dealing with the clinical and regulatory challenges.2 I'm one of the authors there. There’s good potential for collaboration; I’m very pleased to get to know the organisation!

Notes
1. EMA consultation on Guideline on clinical investigation of medicinal products in the treatment of depression, open for comments until 31 March 2024. See background here.
2. Papers published subsequent to the 2023 New Frontiers meeting:

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