What will shape the future of advanced therapies in Europe?

Advanced therapy medicinal products (ATMPs) are reshaping the pharmaceutical landscape, bringing new considerations to the fore for Europe across regulation, manufacturing, commercialisation, investment and artificial intelligence (AI).

In this Q&A, Professor Jacob George explores the challenges and opportunities presented by rapidly advancing fields such as cell & gene therapies and genome editing, highlighting the importance of proportionate regulation, regulatory agility and innovative approaches to evidence generation. He also discusses the role of international collaboration in supporting innovation, attracting investment and ensuring timely patient access to the next generation of transformative therapies.

1. Advanced therapies are developing rapidly and becoming increasingly complex. What makes their regulation particularly challenging compared with more established medicines?

ATMPs present a distinct challenge because they’re often complex biological products made on a patient-specific basis. With these therapies, the manufacturing process is as critical to safety and efficacy as it is to the final product. That’s quite different from traditional medicines, where you can usually characterise the product independently of how it was made.

Many of these therapies are being developed for serious or life-threatening conditions where there are few – if any – existing alternative treatment options available, and the patient population is very small. As a result, regulators, including the MHRA, have to make licencing decisions based on a smaller evidence base than usual. That doesn’t mean lowering standards, but it does mean taking a more proportionate approach to understanding and managing uncertainty.

Manufacturing innovation adds further complexity. We’re seeing growing interest in point-of-care and modular manufacturing, where medical products are produced closer to patients rather than in a centralised facility, and while it’s still relatively early-stage in the UK, momentum is building. The MHRA’s Decentralised Manufacturing Framework is looking to provide a framework and a structured pathway for manufacturing these complex products. We’ve had discussions with the UK Space Agency, the Civil Aviation Authority and companies like BioOrbit, who are doing novel work on low-gravity protein crystallisation. While we haven’t yet had a formal designation request, it’s fair to say interest is growing quickly and we expect that trend to continue.

Essentially, our role is to make sure the regulatory framework is flexible enough to support this pace of innovation, while maintaining confidence that anything reaching patients is safe, effective and of high quality.

2. How do you see regulatory principles shaping the future of advanced therapies?

Proportionate regulation has to be the starting point. A one-size-fits-all approach doesn’t always work, especially for rare diseases or conditions where there are limited treatment options, so we need to look closely at the severity of the condition, the availability of alternatives, and what evidence will genuinely help us understand safety and efficacy – particularly where patient numbers are small.

Our frameworks must evolve alongside the science rather than trying to fit novel therapies into systems originally built for conventional medicines”

Early and ongoing scientific dialogue with developers is also critical, as is regulatory agility. Sound advice early in development means therapies are developed more efficiently, reducing the risk of delays further down the line. In regard to agility, our frameworks must evolve alongside the science rather than trying to fit novel therapies into systems originally built for conventional medicines. At the same time, we need to ensure we maintain public trust.

International alignment remains a priority. We work closely with regulators around the world, including in Europe, because being aligned means reducing costs and duplication for developers, which ultimately benefits patients. Only where we can move faster with a clear benefit to patients, will we diverge.

3. How can regulators balance the need for robust evidence and patient safety with the flexibility needed to support innovation?

Patient safety and innovation can sometimes be presented as being in tension, but I don’t see it that way. Innovation only delivers real benefits if patients and healthcare professionals have confidence that new therapies have been properly evaluated.

That principle applies across all medicines, not just advanced therapies, particularly as we look to give patients faster access to innovative products. Regulatory decisions are made on a risk-proportionate basis, considering the seriousness of the condition, the availability of existing treatments and how well any remaining uncertainty can be mitigated. It’s the balance of these factors that helps determine whether a product is suitable for approval or, in some cases, conditional approval.

The idea that every medicine should meet the same evidence requirements in exactly the same way is no longer how modern regulation works. Where there’s a willingness to accept greater uncertainty, this must be matched by strong post-licencing data collection and robust pharmacovigilance. This means continuing to learn about a product once it’s being used in the real world, monitoring both its safety and effectiveness. Organisations like Health Data Research (HDR UK) are playing an important role in building the infrastructure that makes this increasingly possible.

4. As technologies and approaches to advanced therapy development continue to evolve, how should regulatory frameworks adapt?

Regulatory frameworks need to evolve alongside the science. As technologies such as cell and gene therapies, genome editing and synthetic biology continue to advance, regulation must be flexible enough to accommodate new approaches without compromising standards for safety, quality and efficacy.

One of the biggest shifts we’re seeing is a move away from a linear model, where regulation, health technology assessment and NHS adoption happen separately. Increasingly, these conversations are happening earlier and are more collaborative. Through the MHRA’s Innovative Licensing and Access Pathway (ILAP), developers can work with regulators, NICE and the NHS far earlier in development, helping ensure that the evidence they generate supports both regulatory approval and the needs of the healthcare system.

Regulators also need to be more adaptive in the types of evidence they consider. Randomised controlled trials will remain a cornerstone, but for some advanced therapies it may also be appropriate to draw on real-world evidence, natural history studies, platform technology data and emerging tools such as in-silico modelling and AI-enabled research. These approaches can support more progressive licensing approaches, where evidence is built over time and strengthened as a product moves towards wider use.

The goal is not to lower the regulatory bar, but to modernise how we reach it.”

The goal is not to lower the regulatory bar, but to modernise how we reach it. By combining scientific rigour with greater flexibility and collaboration, we as regulators can help ensure that innovative therapies reach patients more quickly while maintaining public confidence in their safety and effectiveness.

5. What would a successful reset of Europe’s ATMP rulebook look like, and what could it mean for Europe’s position in advanced therapies?

Whatever the jurisdiction, a well-functioning regulatory framework for advanced therapies needs to support the translation of scientific innovation into patient benefit. That requires a system that is agile, predictable and proportionate, makes efficient use of evidence, and maintains robust standards for safety, quality and efficacy.

One reform which may have a positive impact is the planned overhaul of the Hospital Exemption (HE) pathway, and the creation of a central data repository to collect all safety and efficacy data from HE pathways across the EU. This will enable a seamless and comprehensive data collection system and enable better confidence in regulatory decision making.

From a broader competitiveness perspective, Europe is operating in an increasingly challenging global environment, with the US continuing to attract investment and talent, and China rapidly expanding its clinical trial and manufacturing capabilities. For us, in the UK, the question isn’t around the quality of our science – we already have world-leading strengths in areas such as ATMPs and vaccines – but ensuring we create and foster an environment where innovative therapies are developed, manufactured and commercialised domestically, rather than seeing value move elsewhere as technologies mature.

Ultimately, success for Europe and the UK alike will be measured by the ability to attract investment, support innovation and ensure patients have timely access to the next generation of advanced therapies.