Delivery crucial for UK clinical trials improvement - ABPI annual conference, part three

London, ABPI annual conference, part three

The UK Life Sciences Council, a twice-yearly meeting between ministers and global life sciences industry leaders, met on Thursday 25th May to discuss the sector’s economic potential and how to tackle the recent decline in investment.

Hosted at Downing Street and opened by Chancellor Jeremy Hunt – and co-chaired by Chloe Smith, Secretary of State for the Department for Science, Innovation & Technology (DSIT) and Sir Pascal Soriot, CEO of AstraZeneca – the Council’s main points of discussion centred around Lord James O’Shaughnessy’s Independent Review of Clinical Trials, as well as Dame Angela McLean’s review of the life sciences regulatory system in the UK.

To the news that the government will be investing £650 million in funding life sciences research, Michael Young, co-founder of clinical trials start-up Lindus Health, called it “most welcome” and said: “The UK is a global life sciences superpower, discovering cutting-edge treatments that can save lives. But our country’s excellent R&D sector is being held back by an old-fashioned, analogue drug development industry that wouldn’t look out of place in the 1970s.”

Dr Richard Torbett, chief executive of the ABPI, said of the latest announcement that it shows “that the government recognises the huge opportunity waiting to be grasped if the UK can unlock the economic potential of its life sciences industry – already worth £94.2 billion in 2021.” 

Torbett also stated: “Lord O’Shaughnessy is right that making the UK an attractive destination for industry clinical trials requires regulatory reform, speedier study set-up and approvals, and improved access to data. Implementing his proposals, alongside these other announcements, can be a springboard to delivering on the UK’s ambition to be a science superpower, and we now must press forward with delivery at pace.”

Nonetheless, Torbett did note that such improvement of research is “only one part of the equation” – the commercial environment itself must also be fixed. And that means a VPAS amelioration “as soon as possible”.

Given this, as the government’s set of proposals to improve the UK clinical trials environment moves closer to realising its vision for the life sciences industry – including a new biomanufacturing fund, further investment in UK Biobank, and new Health Innovation Networks – it seems apt to turn consideration back once more to discussions last month, voiced during the ABPI Annual Conference.

Building the UK’s global strengths

The panel, ‘Building the UK’s global strengths’, brought together Dr Amit Aggarwal (executive director of medical affairs and strategic partnerships at the ABPI), Dame June Raine (chief executive of the MHRA), Dr Mark Effingham (deputy CEO at UK Biobank), Dr Nathalie Kingston (director of NIHR BioResource for Translational Research in Common and Rare Diseases), and Roz Campion (director of the Office for Life Sciences).

Aggarwal, acting as moderator, admitted the outlook can seem “pretty gloomy sometimes”, but it should be remembered that the UK possess world-class databases, it guides and shapes knowledge and discovery on a truly global basis and, in short, very few countries do what the UK does as well as it does. Indeed, Dame June Raine stated that making the UK part of a global endeavour is something everyone in attendance was passionate about, reminding everyone – “if it needs saying” – that the MHRA’s huge commitment to delivering on its “Brexit freedoms” remains, striving for the UK to be the go-to for new medicines and research. However, to succeed, “it means transforming processes, working with people to do things differently, working in partnership”, and that with purpose. At this juncture, remember, all were waiting on Lord O’Shaughnessy’s review.

Next to speak was Dr Mark Effingham of the 2006-founded UK Biobank, which now has 22 assessment centres and has recruited an ‘unprecedented’ 500,000 participants in the UK, with 30,000 users registered. A “truly special resource and part of the UK research landscape, built on the altruistic endeavours of the participants, with no expectation of feedback or reward,” Effingham said, the process takes a mere five hours spent, and participants are given a cup of tea and sandwich for their troubles. Aged between 40 and 69 at the time of recruitment, information is gathered on their lifestyle and health, undertaking physical measures, and providing biological samples. 

Transforming practice to ensure patient benefit

What’s important is that consent has been given for their data to be used by the wider research community, a biomedical database. Now, UK Biobank is primed to release half a million genomes, available to the community later this year via open access, to be used by researchers, whether they are commercial or academic. Effingham said industry would ask, ‘Where’s the catch?’ Apparently, there isn’t one.

Nathalie Kingston explained that the mission of NIHR BioResource, in comparison to the more well-known UK Biobank – is to transform medical practice to ensure development and benefit for patients. So far, approximately a quarter of a million individuals have given their consent for inclusion in the resource, including access to medical and health-related records. Currently, NIHR BioResource is pursuing programmes in rare diseases and several common diseases, as well as mental health and eating disorders. Its differentiator, Kingston said, is the recall of participants specific to genotype and phenotype, for the purposes of more personalised medicine. So, working much more with Genomic England.

With over 100 recruiting sites, particularly in the most deprived areas so that everyone can contribute to research, NIHR BioResource has worked with GSK and Pfizer, but also smaller SMEs, including drug development in IBD and gene therapies. And in the recent RESTORE trial - a joint research initiative by NHS Blood and Transplant (NHSBT) and the University of Bristol, working with the University of Cambridge, Guy’s and St Thomas’ NHS Foundation Trust, National Institute for Health and Care Research (NIHR) Cambridge Clinical Research Facility, and Cambridge University Hospitals NHS Foundation Trust – BioResource participants were used.

Life sciences at the heart of the matter, the growth sector of the future

Asked by Aggarwal how the Office for Life Sciences is ensuring the UK remains attractive for investment and leveraging assets, Roz Campion replied with a reminder of the political commitment that has been seen from a succession of ministers over the last year, from two different sets of ministers, from three different departments, and the PM himself. There has, she said, been complete consistency. So, that’s a reason to be cheerful, not to mention the creation of the Department of Science, Innovation & Technology (DSIT), which puts “life sciences at the heart of matters, the growth sector of the future”. In addition, Campion noted, there has been clear NHS commitment, which hasn’t always been the case. Turning the NHS into an engine of innovation is key, and the Office for Life Sciences has been working across the system to draw it together for a “steady drumbeat of delivery”. Nonetheless, Campion also made passing mention of the VPAS negotiations: “the thing that must not be named” (this sparked nervous laughter throughout the audience).

At this juncture, Aggarwal commented that one sovereign regulator should make it relatively easy, as there is the risk of the perception of fragmentation and duplication, a general uncertainty over which assets are of key importance to UK attractiveness, and how to draw on those assets. Campion replied that, on the science face, the “big bets” are made on a country level. However, Dame Raine noted that the regulator has the privileged position of seeing everything, yet, she had heard things from Kingston that she had not previously been aware of. The learning from this was that, if we start to see what does and does not work so well, there is a duty to impart that knowledge: “There shouldn’t be dark alleys and rabbit holes and wasting time,” Dame Raine stated. “Let the hallmark of the UK ecosystem not be a case of sitting back, but doing more signposting […] Everybody wins that way.”

Effingham agreed. Last year, he said, a substantial amount of funding for the next 20 years had gone into the UK Biobank (“Poor little robot is quite tired and new infrastructure is needed!” he quipped). This entails a move from Newport to Manchester, where an innovation hub will drive engagement with SMEs, utilising the “same version of the dragon” being used in NIHR BioResource. Kingston responded that there is “space for everybody” and that “not working together, not having alignment, is only detrimental to the patient”. To this end, she said, a secure data environment, a “trustworthy” data environment, permits different industry partners the ability to access existing delivery and promote further discoveries.

Penultimate remarks: Collaborative working within and across the health system

John Stewart, national director of specialised commissioning of NHS England, and interim commercial medicines director, said that in the NHS’s 75th year, its role during that time has been integral in the UK’s becoming an important centre for the life sciences. Innovation, he said, is in the NHS’ DNA.

Stewart described how, in Sir Hugh Taylor’s words, the NHS works at “the limits of sciences, the highest levels of human knowledge and skill to save lives”. When first established in 1948, penicillin had only been publicly available for two years. Conversely, in 2023, the NHS is now using cutting-edge cell and gene therapies, having begun CAR-T treatments but five years ago. In cancer, the NHS is world-leading, he said, in innovative cancer treatment. At the core, success has come from collaborative working within and across the health system; the last five years have shown the difference made for patient through partnership.

The NHS, Stewart said, presents a unique proposition for life sciences companies, with a market of over 55 million people across the country. Turning to the matter of VPAS, he explained that medicines represent the highest area of spend after workforce, and medicines are the only area of healthcare spending that comes with a legal funding requirement. Questioned by Dr Richard Torbett after his presentation, Stewart explained that “VPAS is a real symbol of the three-way partnership between industry, government, and NHS” and that he looks forward to getting going with negotiations and securing that partnership going forward.

Closing keynote: Government a committed partner to life sciences industry

The closing keynote was given by the Rt Hon Steve Barclay MP, Secretary of State for Health and Social Care. He agreed that so much has happened in only the past five years, including factors that have come together to shape a landscape that looks very different today. This includes, of course, the application of AI into almost every aspect of our lives and the potential of AI in the life sciences, from scanning for cancer to transplanting organs, is something everyone in the room was “alive to”. 

The UK, Barclay said, left the European Union just as the pandemic took hold across the Continent and during that time there were learnings on how to make things happen at pace, “holding great promise for other areas in health”. There needs to be, he said, a “laser-like focus on process, partnership, and people.” The MHRA and NICE aim to provide the most effective regulatory approvals process of anywhere in the world, and are one of the most respected.

Commenting briefly on VPAS, Barclay explained that government and pharma came together to create, saving billions of pounds reinvested into patient care, and he greatly welcomed the appointment of Sir Hugh Taylor as chief advisor in negotiations to agree a successor for VPAS – for the good of patients, the NHS, and industry as well. At the core, he wanted to remind the audience that the government is a committed partner to industry, guided by a life sciences vision.

There was one message he wanted to put across, though, and that was that companies need to invest in UK clinical trials. Barclay insisted that “the NHS should be a research partner like no other on earth”, but that too often that had not been the case. One of the key challenges is ‘levelling up’ and making patients more aware of the opportunities to take part in clinical research trials. The clinical trials landscape should be an environment that is people-centred, digitally enabled, and embedded in the NHS, he said.