Trust and inequalities challenge EU’s healthcare digital transformation, says EMA Chief Medical Officer

The EU's digital transformation efforts were at the epicentre of the European Health Forum Gastein. Euractiv spoke to Steffen Thirstrup, the European Medicines Agency's Chief Medical Officer, about the challenges of healthcare digitalisation.

For Thirstrup, digitisation presents important opportunities but also requires overcoming some fundamental obstacles. In the regulatory field, EMA has been building its digital toolkit to ensure its own digital evolution through its Medicines Regulatory Network (EMRN).

As the EU tries to follow rapid and multifaceted digital developments, healthcare's digital transformation is promoted on different levels through regulatory milestones like the European Health Data Space (EHDS) and the AI Act.

Speaking on the sidelines of the European Health Forum Gastein (EHFG), Steffen Thirstrup said implementing the European Health Data Space (EHDS) will require national political will and investment.

"For some member states, it might be easier than for others, depending on where they are at the moment in digitisation and how tech-savvy their population is. Some member states may have only a fraction of the population who will feel comfortable having their health records on a phone," the EMA's Chief Medical Officer (CMO) told Euractiv.

Safety and efficacy

"I think the opportunities are manifold," said Thirstrup, "First of all, the access to real-world evidence [RWE] and real-world data [RWD] will not only mean that we will be able to improve our pharmacovigilance (monitoring for side effects), which is already done via a pan-European database."

"But we will actually get more information about the patients who report side effects that may potentially identify why certain patients are at risk of a certain adverse event," he told Euractiv.

RWE and RWD will also help evaluate the efficacy of any product rolled out, whether medicines, vaccines, etc, taken by patients deliver the promise in the real world.

"Approvals are based on clinical trials, and we do as much as we can to create clinical trials in a naturalistic way, but they are still a 'big test tube' because you need to fulfil certain criteria and conditions," he explained.

But in real life, conditions change, "A patient gets side effects, and they take half the dose instead of completely stopping. That will never be caught in a clinical trial," he points out, adding that through RWD, there could be a better understanding of the medicines put on the market, potentially allowing a re-evaluation of the pharmaceutical product to optimise their use.

Challenges: Trust

Thirstrup also recognises that digital transformation does present challenges and faces obstacles.

The word 'trust' was prominent in all discussions during the EHFG, especially when talking about digitalisation. For the EMA's CMO, trust is key; it all boils down to building trust in society and trust in what institutions and authorities do.

"If we ask the citizens of Europe to share their data with the authorities via the European Health Data Space, it requires that they trust the authorities will handle those data in the right way, for the greater good, will not misuse them, storing them in a secure place," he said.

Challenges: Digital literacy imbalance

"We also need to realise that there are differences in digital literacy across Europe," Thirstrup remarked, pointing out the different levels of digitisation between societies.

"For some societies, it will be easier to implement. For others, it will be an enormous challenge, an investment", he added.

And then it is a matter of which pace you follow, the fast or the slow movers? "Should the fast ones sit and wait for the slow ones, or should [they move ahead] with the risk of having a Europe that develops at different speeds?" Thirstrup asked.

Pharma package opportunity

He sees the use of the EHDS and RWE for the agency's decision-making, mentioned in the proposed pharmaceutical legislation, as a stimulus to the route that the regulators should take.

However, "It doesn't happen by just having it in the legislation. It needs to be implemented," Thirstrup said, adding that a plan is needed.

At the same time, "If we start using or accepting new kinds of evidence for our decision-making, we need to have some level of security that we do this within the framework of the current legislation. We cannot go on a completely different route, making our own rules," he said.

So, for EMA's CMO, development and legislation go hand in hand. "Because legislation will also be the rules that would set restrictions on secondary use, again, to protect the public from misuse," he underscored.

"If we left it without regulation, we could end up in a wild west, a data wild west, which I don't think is in anybody's interest, apart from those who have mal intentions," he warned.

Regulatory sandbox

The introduction of 'regulatory sandboxes', proposed in the revision of the EU's pharmaceutical legislation, could help cope with yet unforeseen situations or "new technologies we haven't dreamt about today," according to Thirstrup.

He explained that if a developer comes with that new technology or unforeseen challenge of any form, the EMA could if there aren't specific regulatory tools at hand, create a sandbox.

"We put in place what we potentially could do that is outside the current legislation. We put in place milestones where we need to assess if what we are 'playing in the sandbox' with works or not. And there needs to be a stop date when the sandbox will come to an end. Hopefully, if it goes right, the sandbox will help us solve the problem," he said.

Not only could it ensure an understanding of the new technology and its safe implementation, but it could also signal the need for amendments to the legislation at the next revision.

Thirstrup clarified that the sandboxes won't be a carte blanche for "us regulators to do whatever we like when we want to do it."

"We do this in a very controlled environment," he said.

"It's an attempt to make the legislation a little bit more future-proof and a little bit more flexible to deal with developments that we didn't know about and didn't think about today," he said.

Overregulation

Asked whether claims of overregulation pushing investment in innovation away from the EU have a basis, Steffen Thirstrup referenced the Draghi report and how Europe can become more innovative and more competitive.

“I think it eludes that we need to look at our regulation. Whether we have areas where we are over regulated or where regulation needs to be more flexible? Definitely,” he added.

He explained that the current pharma legislation has been created over the years, with different legislation, such as on orphan drugs, adding new layers of complexity and then going through a process of critical discussion.

However, he believes that some of the EU's complexity cannot be solved because we are a political union of 27 member states.

"You can get a marketing authorisation via EMA in one go. Now, we are trying to establish a common health technology assessment, but you still have national member states deciding on pricing and reimbursement."

"We need regulation to collaborate, to figure out how do we do things, so we actually make them work in a European way", he added.

Return on investment

"If we succeed, we will be able to keep workplaces in Europe, we will be able to put a hold on the brain drain of educated people moving out of Europe, and of not only the pharma industry but a lot of other science areas being attracted to other parts of the world where the legislation or the world is seen as less complicated to work in," Thirstrup remarked.

He added: "Keeping workplaces and brains here means keeping taxpayers here, keeping society alive. I think it's important for us to survive as a continent," he concluded.

[Edited by Brian Maguire | Euractiv's Advocacy Lab]