Эксперт MHRA: регуляторика должна адаптироваться к сложностям клеточных и генных терапий

Professor Jacob George, Chief Medical and Scientific Officer at the MHRA, delivered the opening keynote speech at the Advanced Therapies Europe conference in Barcelona, running from 7-9 September, on Regulatory principles shaping the future of advanced therapies and is also hosting a roundtable on Europe’s ATMP rulebook reset. Cell and gene therapies are moving from niche innovations towards broader clinical use. What unique regulatory challenges do these treatments present that differ from those associated with conventional medicines? Cell and gene therapies are different from ordinary medicines in some important ways. They’re often highly complex biological products, sometimes made specifically for one patient, and how they’re manufactured can matter just as much as the finished product. These treatments are usually for serious or life-threatening conditions where there aren’t many other options. They’re also often developed for very small numbers of patients. That means regulators sometimes have less evidence to work with when deciding whether to approve them, compared with a typical medicine. That’s why we have to think differently about manufacturing itself. Point-of-care and modular manufacturing, where products can potentially be made much closer to the patient rather than in a traditional manufacturing facility, are still at an early stage in their development in the UK but we’ve had interest and enthusiasm from developers already. We have hosted some meetings, either as the MHRA or as multi-agency, with the UK Space Agency and the Civil Aviation Authority, for example, with BioOrbit, which is a good example of people who are doing amazing research in this space, working on low-gravity crystallisation of protein. We haven’t yet had a formal request to use this approach, but interest from companies developing these treatments is growing fast and we expect that to increase over the next year. Our challenge is to make sure our rules are flexible enough to support this kind of new treatment, while still giving patients and healthcare professionals confidence that the products reaching the market work and are safe. Looking ahead, which regulatory principles will be most important in enabling the next generation of cell and gene therapies to reach patients effectively and responsibly? One of the most important principles is proportionate regulation. Rules that work for everyday medicines don’t always work for rare or severe diseases, so we look closely at the mortality and morbidity associated with the condition, whether other treatments exist, and what endpoints can meaningfully inform regulators about the efficacy and safety of the products, particularly when numbers are small. We also need to talk to companies developing these treatments early on. Good advice given early means treatments get developed more efficiently, and it avoids later delays caused by misunderstandings about what we need to see in terms of evidence generation. Regulatory agility matters too, meaning we need to move with the science while ensuring our rules keep up with new technology without losing the public’s trust. Finally, we work closely with regulators in other countries, including in Europe, wherever that makes sense. Staying aligned avoids extra cost and delay for the companies developing these treatments. But where we can move faster and it clearly benefits patients, we will do things differently. Our approach is to stay aligned wherever we can and only go our own way when there’s a good reason to. As the field continues to advance at speed, how can regulators safeguard patient safety and scientific rigour while avoiding unnecessary barriers to innovation? People sometimes think patient safety and new treatments are in conflict, but they’re not. Patients only benefit from new treatments if they can trust that those treatments have been properly checked. When we decide how much uncertainty is acceptable before approving a treatment, we look at the whole picture. How serious is the condition? Are there other treatments available? And how well can any remaining uncertainty be managed once the treatment is in use? For some serious diseases, where there are few or no other options, we may accept more uncertainty than we would for a routine medicine. But if we do that, we need post-marketing pharmacovigilance, to keep checking the treatment is working and remains safe. Groups like Health Data Research Service UK (HDRS UK) are helping build the systems that make this kind of monitoring possible. By combining sensible flexibility with strong pharmacovigilance, we can help patients get treatments sooner, without lowering our standards. Many advanced therapies are designed for rare diseases and small patient populations. How should regulators and developers approach evidence generation when traditional clinical trial models may not always be feasible? This is one of the hardest challenges in this field. Traditional ways of testing new medicines were designed for large numbers of patients, and that doesn’t always work for rare diseases, where there might only be a handful of patients worldwide. The answer isn’t to lower our standards, but instead to think differently about what counts as good evidence. We want to work with the companies and patients in developing these treatments early on, to agree which endpoints are relevant and meaningful to regulators but also to patients and their families. Part of that means looking at many different aspects including the underlying mechanism of how the treatment works, results from clinical trials, how it’s manufactured and, increasingly, real-world evidence gathered after the treatment is approved and being used by patients. These treatments offer real hope to patients with rare diseases who may have had few options before. The MHRA’s role is to balance maintaining high standards with being pragmatic and flexible about how that evidence is gathered. Patient access to advanced therapies remains a significant challenge. What are the key barriers preventing wider access and how can they be addressed? Access depends on much more than regulatory approval alone. We’re one part of a series of steps, one cog in a wheel, but I think we have a key role to play in how smoothly that whole pathway moves. If we can provide faster scientific advice, quicker clinical trial approvals and timely marketing authorisations, that sends a clear signal to investors and developers about where it makes sense to base their work. We’ve worked hard over the past two to three years to speed this up and it’s paying off. We’ve introduced regulatory flexibility through things like the UK Rare Disease Therapies Regulatory Framework and the Clinical Trials 14-day Pilot Programme. Those measures exist to show developers that we are listening and are there to support their ambitions. But speed on our part only helps if the rest of the system can move at a similar pace. Decisions about funding and rolling out new treatments involve a lot of moving parts, and they matter just as much as our approval. That’s why we’ve built closer links with the National Institute for Health and Care Excellence (NICE), the body that decides which treatments the NHS will fund, so these decisions can happen earlier and alongside our own work, through the new MHRA-NICE aligned pathway rather than only starting once regulatory approval is obtained. None of this happens through regulation alone, which is exactly why conversations between industry, investors and healthcare systems at events like Advanced Therapies Europe 2026 matter as much as anything we do inside the agency. Which emerging technologies or scientific advances do you think have the greatest potential to transform the field of advanced therapies over the next decade? The ability to scale up and simplify CAR-T therapy is one area that has tremendous potential for advanced therapies to significantly impact outcomes in many cancers. CAR-T therapy (chimeric antigen receptor T-cell therapy) works by taking a patient’s own immune cells, modifying them so they can recognise and attack cancer cells, and returning them to the patient. It’s already shown strong results in treating some blood cancers. The challenge now is scale. Each treatment is currently made individually for a single patient, which makes the process complex, slow and expensive. If we can simplify manufacturing and make it more efficient, we could treat far more patients and treat them more quickly. This matters because the science already works. What determines whether patients actually benefit is whether we can manufacture at the scale and speed needed.