Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2024

Considered in Grand Committee

16:37:00

Moved by

That the Grand Committee do consider the Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2024. Relevant document: 13th Report from the Secondary Legislation Scrutiny Committee

My Lords, I am grateful for the opportunity to debate these amendments, which represent the most substantial reform of UK clinical trials regulation in over two decades. Clinical trials are vital for developing safe and effective medicines, especially for those with limited treatment options, such as the estimated 3 million people living with cancer in the UK and the 17.5 million people managing long-term conditions. Last week, I saw for myself how innovative UK researchers are transforming cancer diagnosis and treatment. I joined the Science Minister, my noble friend Lord Vallance, at the Royal Marsden to learn about a research initiative that is using cutting-edge AI tools to improve breast cancer detection. To support innovation, our regulations need to be flexible and proportionate. This legislation will do just that by delivering streamlined and efficient regulations, removing barriers to innovation and creating a patient-focused research environment—something that noble Lords called for during Questions on the Statement repeat that we just dealt with in the Chamber. These reforms will support the development of new life-changing treatments for those in need and strengthen the UK’s position as a global leader in clinical trials. I turn briefly to why this change is necessary and timely. The reason is that the current legislation is based on the now-repealed EU clinical trials directive, so it therefore no longer aligns with the rapid advancements in medicine and technology. We have the opportunity to create a world-class regulatory environment for clinical trials, if we can deliver a modernised framework that supports the safe development of innovative treatments. I will outline, for the benefit of the Committee, the key aspects of the reforms. First, on risk-proportionate regulation, regulatory requirements will align with the risk level of a clinical trial. Low-risk trials will receive faster approval through automatic authorisation, without compromising patient safety. The second aspect is that of future-proofing. We have removed duplicative and granular legal requirements in favour of tailored guidance, ensuring flexibility for future innovations and moving beyond a one-size-fits-all approach. On international alignment, the UK will remain aligned to global standards, ensuring that trial data is recognised internationally and strengthening its position as a preferred site for multinational clinical trials. Then there is the important point of cementing the UK as a destination for international clinical trials. Streamlined processes will simplify applications and deliver globally competitive approval timelines. The final change that these regulations deliver is increased transparency. We want to ensure that trusted information about clinical trials is publicly available for the benefit of all. New legal requirements will thus be introduced to register a clinical trial, and publish a summary of results, including an easy-to-read summary for participants. These changes will build public trust in research by improving access to information about ongoing research and enabling informed decisions. Of course, these reforms will also bring benefits to the National Health Service. Evidence shows that hospitals that undertake research have better patient care outcomes and improved staff retention. Improved efficiency in conducting clinical trials will therefore enhance research efforts and foster innovation in prevention, diagnosis and treatment across various conditions. Those conducting clinical trials will also benefit from a streamlined and risk-proportionate regulatory framework, reducing delays and admin burdens. These reforms, I am glad to say, will stimulate growth in the UK’s life sciences sector and position the UK as a global hub for clinical trials. I beg the Committee’s indulgence as I correct an administrative error made in the Explanatory Memorandum. It incorrectly stated that an impact assessment was produced. However, since the projected costs and benefits to business were below £5 million annually, a de minimis assessment was conducted and published instead. Before I conclude, I re-emphasise that participant safety remains absolutely paramount. While this legislation streamlines processes and removes barriers to innovation, what it does is to prioritise robust oversight of all clinical trials, ensuring that the safety of trial participants is never compromised. By modernising our approach, I believe we can strengthen the UK’s position as that global leader, as well as fostering innovation and having the highest safety standards. These reformed regulations accelerate the delivery of tomorrow’s emerging medicines into today’s reality for patients. I beg to move.

I thank the noble Baroness, Lady Merron, for her clear introduction to this statutory instrument, which I broadly welcome. There are some important factors here. I particularly welcome the requirement to register clinical trials and publish a summary of the results within 12 months. It has been widely and long acknowledged, in the research community broadly, that there is an issue where less successful or failed trials, or those that are not seen to have interesting results, are not published. They can be as important, or more important, than the successful ones. The failure to publish them is driven by academic, publishing and promotion imperatives—and, I am afraid, by the profit motive in healthcare, where companies have very much sought to find the successful stories and bury the less successful ones. That is really positive and, if I would say one thing, it would be to encourage the Government to speak more about that, because it is important that people understand it. Given the issues that we have with trust across the board at the moment, I encourage them to highlight that we are actually strengthening and improving regulation.

16:45:00

With that in mind, I turn to some of the rhetoric that has come from the Government around this, and indeed some of the actions. I understand the argument about trials identified as low risk, particularly those using medicines that have already been approved for other purposes. But let us acknowledge that, historically, the use of some of our longest-serving medicines, medical devices and medical practice is often based on custom and practice rather than a strong foundation of evidence. It is important to make sure that what we are identifying as low risk really is low risk.

I welcome the fact that the Health Research Authority has developed a set of questions around diversity and inclusion. We know that there is a big problem, in our whole medical system, with failing to acknowledge the needs of different groups and populations, particularly disadvantaged ones. That is potentially a useful step towards tackling health inequalities.

I am slightly disappointed not to see a fast-growing and relatively new area here: a lot of work is being done now on the environmental impact of clinical trials. I particularly note the work of the Clinical Trials and Statistics Unit of the Institute of Cancer Research, which has developed a greener trials toolkit that enables managers to identify higher-emissions activities and implement targeted actions to reduce them. This calls for accepting carbon footprinting into the development stage of new trials and including it in funding applications.

I note that, in this space, there are a number of all-voluntary actions, such as the NIHR’s carbon reduction guidelines, the UK CRC monitoring group’s green monitoring guidance and the UKTMN guide to efficient local management, but are the Government considering putting some of these environmental steps on a statutory basis, rather than simply relying on voluntary efforts? I do not think that much work is being done in this area, but the noble Baroness will be aware that I have done a great deal of work on the environmental impacts of human medicines in the environment. That is in the context of antimicrobial resistance and more broadly in terms of environmental impacts. Particularly in Sweden, there is a real drive towards encouraging looking at the environmental impacts of medicines while they are being developed, so that we can look to design medicines, at the clinical trial stage, that have a minimal environmental impact.

Those are a few very specific things related essentially to this instrument, but a broader question that this instrument brings up is, how do we make clinical trials more effective? Of course, what we want is clinical trials that work and deliver effective medicines as soon and safely as possible.

In that context, I note that I raised with the previous Government, but have not yet had the opportunity to raise with this one, the call for a human-specific technologies Act to support the full replacement of animals in medical research with new technologies. If we look at that in the context of clinical trials, the likelihood of a cancer drug being approved and progressing from small phase-run trials to larger clinical trials is less than 6%. Alzheimer’s disease has a clinical trial failure rate of more than 99%. We cannot rely on animal tests to indicate whether a drug is safe or effective in people; 92% of the drugs that show promise in animal tests fail to reach the clinic and benefit patients. Are the Government looking at that more broadly when they think about the whole ecosystem of developing new medicines and pharmaceutical trials? Given that I have just sprung that on the Minister, I will entirely understand if she would like to write to me about it later rather than deal with it now.

My Lords, I start by congratulating the Minister; I hear her sniffles. She has been a champion of her brief in having to deal with two statutory instruments, as well as a Statement in the House. I thank her for being here. As the Minister said, the reforms proposed in this statutory instrument aim to create a more streamlined and flexible regulatory environment for clinical trials in the United Kingdom, while balancing safeguarding the interests of trial participants. The amendments seek to uphold the paramount importance of participant safety, ensuring that their rights and well-being remain central to the regulatory framework. By refining the evaluation and development process for new or improved medicines, these changes aim to expedite the delivery of therapeutic benefits to patients and society at large, and we on these Benches recognise that. The instrument will formalise the combined review process, which has been piloted since 2018 and become the exclusive route for clinical trial applications. This process offers a single application pathway and co-ordinated regulatory and ethics committee review, ending in a unified UK decision for clinical trials. That would be helpful for practitioners and those seeking to innovate, but there are still points that require clarification, and while the objectives of these amendments are commendable, I seek clarification from the Minister on several aspects. What measures are in place to ensure that the Medicines and Healthcare products Regulatory Agency and the ethics committees will be adequately resourced to manage the anticipated increase in workload resulting from the streamlined process? If no impact assessment has been made, what working assumptions are the department and NHS England working to regarding the workload that this new process will bring? Can the Minister provide detailed guidance on how the risk-based approach will be operationalised to ensure consistency across different types of trials? I note that she talked about international alignment, but how do the Government plan to align these regulatory changes with international standards to facilitate seamless multinational trials? What frameworks will be established to monitor the impact of these regulatory changes on trial efficacy and patient safety, which is really important? How will these findings be reported locally within the NHS? Will they be reported to Parliament at any point? Finally, can the Minister explain and elaborate on the consultation process undertaken with key stakeholders regarding these changes, including patient groups, industry representatives and academic researchers, in the development of these amendments? Were there any differences that emerged from the stakeholders and how have the Government dealt with them? In conclusion, while we on these Benches support the intention behind these regulations, it is imperative to ensure that the implementation is robust and effective. I look forward to the Minister’s responses on the matters I have raised—less croaky responses, I hope.

My Lords, I thank the Minister for the laying of this statutory instrument. Like the noble Lord, Lord Scriven, I commend her on her valour and robustness, as well as the speed at which she managed to transport herself from the Chamber to here after the Statement repeat, having prepared for that and having been briefed by her officials. Like many other noble Lords, we welcome these regulations, which are grounded in the review by my noble friend Lord O’Shaughnessy and the subsequent consultation with stakeholders, including the Medicines and Healthcare products Regulatory Agency and Health Research Authority, aimed at modernising the regulatory framework that governs clinical trials in the UK. We know that the landscape for clinical research in the UK faced significant disruption during the Covid-19 pandemic, but we also know that we learned quite a lot from trying to get vaccines out very quickly in terms of ways to speed up trials and to make sure that we get the right balance between efficiency and processes, as well as making sure that people are safe. We need to make sure that we can boost the volume of clinical trials and boost patient recruitment. The O’Shaughnessy review identified the need for a more flexible and risk-proportionate approach to clinical trials. One very important point is that, while we cannot eliminate risk altogether, we can manage it. Where there is low risk, we should maybe not be placing so much emphasis on processes as compared with when there is high risk, but I also know that there may always be unintended consequences. Stakeholders such as the Association of the British Pharmaceutical Industry were vocal in their support for these reforms and had in fact been calling for them. The ABPI 2024 report, The Road to Recovery for UK Industry Clinical Trials, highlighted that the number of pharmaceutical industry trials initiated in the UK increased—it was just over 400—between 2022 and 2023, although that still remained 36% below the 2017 level. We therefore understand the need to bring forward these regulations to make sure that we improve the regulatory environment to further increase clinical trial activity. I turn to the detail of the regulations. One major change that noble Lords have already alluded to is the consolidation of the regulatory and ethics review process, enabling researchers to submit a single application for both regulatory approval and ethics review. While this change is aimed at improving efficiency—which we completely accept and welcome—there is a concern that any changes from consolidating these processes may put additional pressure on the regulators and ethics committees. The noble Lord, Lord Scriven, referred to that. If the single application process becomes overloaded, we risk delays in review times rather than the acceleration that was intended by these measures in the first place. It is essential that we have the resources and infrastructure in place to handle the increased workload effectively. Can the Minister therefore say how the Government will make sure to avoid that sort of overload? We completely understand why they want to consolidate the processes, but will it add extra pressure and will the resource be there to make sure that they are not overloaded and we do not just end up going back to square one, or even make things worse? In addition, for low-risk trials, automatic regulatory authorisation will be granted, which will further streamline the approval process and free up resources for the more complex, higher-risk trials. Once again, while this makes sense, we have to be aware of possible unintended consequences or concerns that will be raised. The automatic approval of low-risk trials could, for example, raise concerns over oversight and monitoring. The intention to expedite the approval process for those with lower-risk profiles is laudable, but will there be clear criteria in place to ensure that the appropriate level of scrutiny is maintained, particularly in ensuring patient safety? As I said, while we support the intention, particularly for low-risk trials, we must always be aware of unintended consequences or unforeseen complications. Nevertheless, we completely understand why this is being done, and it is something to be welcomed.

17:00:00

In introducing the intent of this instrument, the Minister discussed the idea of greater transparency. Like the noble Baroness, Lady Bennett, we welcome the fact that clinical trials will be required to be registered on a public database—and not only the positive results but the negative results too. We can always learn from things that went wrong and not downplay them; when mistakes have been made, we can learn from them. Increased transparency is crucial for public trust. There is, I suspect, a need to ensure that sensitive information, such as proprietary data or details that could affect patient confidentiality, are handled properly. What safeguards will be in place to protect the interests of participants and sponsors while promoting greater transparency?

We know that these measures will introduce greater flexibility and accommodate innovative trial designs. Noble Lords know that a one-size-fits-all approach is not appropriate; it is absolutely right that we are as flexible as possible. We will now see guidance, rather than rigid legislative details, used for specific requirements, which will allow researchers to tailor their trials to suit the needs of the study and the risks involved. Once again, that all makes complete sense on the surface, and we welcome it, but how do we avoid a situation where the lack of clear legislative requirements could lead to inconsistencies in trial design? We want flexibility, of course—these things should not all be the same—but, in having that, will this measure introduce some inconsistencies and perhaps complicate regulatory oversight? It is welcome but, once again, let us look out for unintended consequences. If flexibility is not managed, would it lead to varying standards across trials, thus creating gaps in safety and quality assurance? What is the intention to avoid that?

One of the other points that was raised with me by, I think, the Teenage Cancer Trust was that it was recruiting younger people to take part in trials. Of course, there is always a concern that trials should involve consenting adults as much as possible, but there is also a concern—the Teenage Cancer Trust is concerned about this—that, sometimes, you need to involve more children or younger people in these trials. This immediately raises alarm bells because we should really be focusing on adults. What thought have the Government, the NHS and the research environment given to, where appropriate, involving more young people, children and teenagers in research that is specifically appropriate to them, while not affecting their safety and addressing concerns around recruiting children, who may not always be consenting adults, if that makes sense?

Overall, we welcome these regulations. We see them as a crucial step towards modernising the UK’s clinical trials framework. How we need to improve our clinical trials is one of the things that I hear about from industry all the time. We have great potential here, but we get slowed down by this bottleneck. We agree with the aims to improve efficiency, to foster innovation and to enhance transparency, but at the same time we must be mindful of the concerns that have been raised by others. Do we have the proper resources? Have we looked carefully at the unintended consequences? How do we make sure that the focus is on lower risk, with the appropriate focus on higher risk, so that we can all agree with the Government’s position on clinical research? This is about maintaining essential protections for patients and data integrity, making sure that the UK is at the forefront of clinical trials and encouraging more companies to do them here while, at the same time, reassuring the public about safety. I look forward to the Minister’s response.

My Lords, I am most grateful to noble Lords for their time and their constructive contributions. I feel that we are all moving in the same direction, and I appreciate the welcome for these regulations. I also appreciate the understanding that I am not firing on all cylinders, but be warned: I will be at some point. Noble Lords have heard the details of the amendments, which, as I said in my opening speech, represent the most significant reform of UK clinical trials regulation in more than 20 years. As I said in my opening comments, I am conscious of the fact that I have just come from the Chamber, where we heard questions about this very area. So these regulations do seem very timely. If I miss anything in response, I will of course be very pleased to write to noble Lords. In delivering a more efficient and adaptable regulatory framework, and in accelerating life-saving treatment through streamlined and future-proof processes, the reforms will put patients at the heart of clinical trial processes, as we well as strengthening the UK’s position. I turn to some of the key points that noble Lords have raised. On the matter of safety clinical trials of course carry varying levels of risk. No clinical trial is entirely without risk, but the MHRA maintains a rigorous regulatory oversight to safeguard patient safety in all clinical trials, and this legislation does not change that. There will be no compromise on the protection of participants. However, we are removing requirements from the current legislation that simply offer duplication or no additional value when it comes to identifying safety risks. As I and other noble Lords have acknowledged, this is about removing obstacles but ensuring that safety is paramount to ensure that regulators, researchers and participants are all aware of potential risks and can take action to deal with them as appropriate. I very much welcome the removal of unnecessary administrative burdens; I am sure that all noble Lords do. By increasing the opportunities in the UK to access innovative medicines at an earlier stage, we will expand patient access to new therapies and reinforce the reputation of the NHS as a world-leading platform for health and life sciences. The noble Lord, Lord Kamall, and the noble Baroness, Lady Bennett rightly highlighted the matters of transparency and public trust. I am very glad to see the new transparency requirements because, for the first time, there will be a legal requirement for sponsors to register a clinical trial, publish a summary of results and offer to provide participants with an easy-to-understand summary of what the research has found. Clear guidance is being produced to ensure that the summary for participants is accurate, tailored and appropriate for the audience, which includes translation into different languages and an awareness of suitable formats, as highlighted by the noble Baroness, Lady Bennett. A recent study commissioned by the HRA highlighted the importance of transparency, with 69% of respondents stating that they would have greater confidence in research if participants were informed of the outcomes. These measures will therefore foster greater trust and engagement with clinical research, and I certainly welcome that. The noble Lord, Lord Kamall, asked about the protection of pharmaceutical companies’ legitimate interests in protecting commercially sensitive information, and asked what safeguards are in place. I can assure him that we absolutely respect and understand the need for commercial confidentiality. The new regulations will permit research sponsors to request deferrals for registration and the publication of results, including offering to share these with participants where this is necessary to protect commercially confidential information. Deferrals could be granted for up to 30 months, with the possibility of further deferrals, where justified, up to a maximum of 10 years. I hope that these provisions will safeguard the very legitimate interests of companies, while also maintaining the overall goal of transparency, to which we are all committed. We recognise the scale and the vibrancy of the UK’s life sciences industry, particularly those conducting clinical trials. Throughout the development of the reforms, we have engaged with the clinical trial community and received widespread support across key stakeholders, including businesses, academics and charities. The public consultation generated over 2,000 responses and demonstrated a strong appetite from the research community for updating and improving clinical trial regulations. We will continue working closely with the research community to produce guidance that supports the smooth implementation of these new regulations. Noble Lords were very helpful in raising a number of considerations. The noble Lord, Lord Kamall, asked about the criteria for automatic authorisation and for information about low-risk trials. The criteria have been designed to ensure that sufficient scientific evidence already exists regarding the safety of the product and the methodology that has been used in the clinical trial—essentially, that we can be assured that the medicine is safe. The evidence must have been reviewed previously and approved by the MHRA or, where applicable, by regulatory authorities in the EU, the EEA or the USA. Additionally, the legislation defines the criteria for a clinical trial to be eligible for automatic authorisation. I hope that this is helpful to the noble Lord, as his point is very valid. On the matter of implementation, raised by the noble Lord, Lord Scriven, particularly regarding guidance on risk proportionality, guidance will be published in advance of the regulations coming into force. This will ensure that researchers and those undertaking clinical trials understand the changes and have time to prepare. We are working with stakeholders across the sector and taking views into account to ensure that the guidance is as clear and helpful as possible. The guidance will be promoted by a wide range of channels to ensure that it reaches stakeholders across the research and clinical trial participant community. This is vital as we bring in this legislation. The noble Lord, Lord Scriven, also raised a point relating to the performance of the MHRA. Since September 2023, all regulatory assessments for clinical trial initial applications and substantial amendments to protocols have been completed within the current statutory timescales of 30 days and 35 days, respectively. The latest performance information about the MHRA regarding clinical trials assessment shows strong consistency and, I am glad to say, no backlogs. The updated legislation will introduce key measures to make it easier and faster for applicants to gain approval. Noble Lords have acknowledged the need to ensure that the UK remains a prime destination for clinical trials. The noble Baroness, Lady Bennett, raised questions about automatic authorisation. I understand why noble Lords are raising these matters. This is new territory and noble Lords need to be reassured. There are clear criteria embedded in the legislation to ensure that only appropriate clinical trials can use this automatic authorisation route. The criteria are based upon the MHRA stakeholders who were consulted on their extensive experience of clinical trials and the participant safety risks associated with them. I can give the reassurance that, where there is a significant safety concern with the product, clinical trials will not be eligible for automatic authorisation and must undergo full regulatory assessment. The noble Lord, Lord Scriven, mentioned stakeholder engagement. Following the public consultation, a number of policies were adapted to ensure that the regulations did not have any unintended consequences, as the noble Lord, Lord Kamall, said. Let me give one example. The feedback indicated that patient and public involvement would be best addressed in guidance rather than in legal requirements, in order to give that flexibility and to enable it to be kept up to date. The noble Baroness, Lady Bennett, mentioned the environmental impact at the stage of clinical trials. I will be pleased to write to her on that point. As I believe this debate has shown, we are in agreement that, by improving the clinical trial regulatory framework, these changes will expand patient access to cutting-edge therapies, boost the UK’s life sciences sector and reinforce the reputation of the NHS as a leader in health research. On this basis, I hope that noble Lords will feel able to support these vital regulatory changes.

Motion agreed.