How do new medicines get approved by the European Union?
Interview by Emma Bishop, RARE Revolution, with Steffen Thirstrup, chief medical officer at the European Medicines Agency (EMA)
Estimated reading time: 1 minute
Steffen Thirstrup has been working in regulatory affairs for almost twenty years, and has been chief medical officer at the European Medicines Agency (EMA) for over three.
The EMA is a decentralised agency of the European Union, responsible for evaluating, supervising, and monitoring the safety of medications.
Steffen’s role within the EMA involves “being the outward facing medical voice of the agency,” having an eye on what’s happening within the organisation, which new medicines are being approved or not approved, as well as the reactions of the surrounding community.
Decisions decisions decisions: The stages of approving new medicines
Steffen’s position at the EMA also means being a part of the Health Technologies Assessment matrix (HTA) —a process for EU member states to evaluate health technologies such as medicines and medical devices.
He explains more about this process, and the various different stages involved:
“EMA decides whether a product should be allowed to the market. This is based solely on the benefits and the risks.
Where the HTA comes in, is looking at comparative effectiveness. So, how does this medicine work in comparison to what’s already out there?
And then, of course, when it comes to the national decision, it comes down more to health economics. There is a limited amount of resources, so if we spend a million on a new cancer treatment, that means they’re going to take that million from somewhere else. It’s asking, how do they get the most health from our health expenditure?
The Joint Clinical Assessment
Introduced in January 2025, theJoint Clinical Assessment (JCA)is a standardised process for evaluating the clinical evidence around new medicines. It forms a single, EU-wide scientific assessment of the new therapy, which national HTA bodies can then use to make decisions.
Forming part of the existing HTA process, Steffen explains how the JCA aims to allow patients across the EU to access new medications quicker.
“The idea of the [JCA] is to agree on a common set of standards to do the technology assessment, which can be taken into the member states as part of the pricing and negotiation.
The aim is, of course, to hopefully reduce the time from approval by EMA to actual access at a national level for patients.”
The JCA will initially relate to new oncology products and advanced therapy medicinal products with a view to all new medicinal products being assessed by 2030.
A step towards equitable healthcare
“Despite us having universal healthcare in Europe, we also know there are some parts of Europe that are less well off than others,” Steffen explains how the JCA is bringing us closer to equity in healthcare.
“The political vision is to try to make it even, so that patients in Europe get access to the best care, independent of whether they live north, south, east or west, and that’s a big challenge. This is a way of trying to move the access and availability as close to a national decision as possible.”
The storm before the calm: How has the JCA helped make things smoother?
Earlier in 2025, Steffen spoke at the ISPOR health economics conference in Glasgow, on a panel titled: “Outcome assessment in rare disease: Is JCA breaking the silos or perpetuating the chaos?”
He talked of the situation prior to the introduction of the JCA, and how it may have been perceived by those on the outside.
“It could have been perecived from the outside to be quite random. Whether you, as a patient, are fortunate and live in a country that has a smooth HTA, you’ll get access quickly, whereas if you live in other parts of Europe, you may never get access or may have to wait years, and if you are suffering from a serious disease, it could be a matter of life or death.”
He explained the difficulties and that existed prior to the JCA and how they affected medicine availability.
“EMA would issue a marketing authorisation, which meant companies could sell that product across Europe, but not until they got HTA , pricing and reimbursement sorted out in each of the 27 member states.”
He explained how this then became “a commercial decision,” as there are “parts of Europe that are more attractive than others in terms of speed, and access to the market willingness to pay,” and that hopefully “the HTA regulation will even some of this out and smooth out some of the process.”
The JCA and rare disease: ‘It’s not acceptable for patients to not be treated’
Steffen revealed how roughly 2/3 of all new medications being reviewed by the EMA are for malignant diseases, but there are 17 therapies currently being reviewed for rare diseases.
“We’re already gaining experience in the rare disease area with those advanced therapies.
We managed to do a lot of background work before the launch in January 2025, and have a lot of the implementing acts and the collaborative agreements in place.”
He talked through the unique challenges posed by rare disease therapies, and how shifting approaches to think on a broader level could help patients and families to access better treatment.
“Some of these diseases are so rare that it doesn’t make sense for every member state to diagnose and treat them. Some member states may be too small, have only a few patients, and may not have the expertise.
Maybe we need to think more around an EU wide centre. I know it will be burdensome for patients to say, ‘I have a rare disease and I live in the Netherlands, but I’ll have to go to Sweden or France or somewhere to get it free.’ But I think patients are open to doing that if they realise they then get the best care.
Steffen goes on to say that: “It is not acceptable for patients to either not be treated, knowing that there is a treatment available in larger member states, or maybe treated sub optimally because [their state] doesn’t have the right medical expertise. When we come to the rare diseases, we need to think in another way.”
Patient experiences allow for better decisions
Steffen and colleagues at EMA have worked on a paper published earlier this year, reflecting onpatient experience data (PED).The paper touches on health-related quality of life and patient reported outcomes. Steffen highlighted how using unprocessed data allowed them to get direct insights, as opposed to those processed through doctors or investigators.
“It’s more or less raw data on the patient experience, so how patients are doing with their disease and with the treatment, and how they experience that.
We want to add this to our assessment of medicines. We’re not talking about replacing the existing endpoints with patient experience data, but we’re talking about supplementing.
We’ve come to realise that the patient voice is an important additional input to regulatory decision making.”
The JCA aims to harmonise the clinical evaluation of new medicines across the EU, improving efficiency, reducing duplication of work, and ultimately, allowing faster, fairer access to new therapies for patients.
To find out more about the JCA, please visit;https://health.ec.europa.eu/health-technology-assessment/implementation-regulation-health-technology-assessment/joint-clinical-assessments_en
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