Reasons to be cheerful: 4 takeaways from the BIA UK Life Sciences Regulatory Conference 2024

We had an insightful day hearing about the future of UK life sciences regulation at the BIA’s flagship annual Life Sciences Regulatory Conference. The conference brings together speakers from UK and international regulators, key decision-makers, industry and charities to discuss the challenges and opportunities for Biotech companies looking to develop and commercialise their products in the UK, and the UK’s evolving role in the global life sciences ecosystem.

There were four key takeaways from the day.

Reasons to be cheerful

The overall tone of the conference was very upbeat. The keynote speakers were keen to emphasise that, after the turbulence of recent years both politically and in dealing with the aftershocks of the pandemic, there are real reasons for industry to be optimistic about the UK environment for life sciences.

Keynote addresses were delivered by Baroness Merron, Parliamentary Under-Secretary of State, Department of Health and Social Care, and Dr June Raine DBE, outgoing CEO of the MHRA. Baroness Merron set out details of the Labour government’s plan for delivering growth in the life sciences industry. These measures include the life sciences manufacturing fund announced in the recent Autumn Budget, encouraging increased investment of institutional capital (such as pension funds) in the UK life sciences industry, and stabilising the regulatory environment in the UK. Dr Raine expanded on this third aspect, highlighting the recent work of the MHRA in reducing backlogs and making improvements to many regulatory frameworks, such the progress towards updating the UK frameworks for clinical trials and medical devices and the introduction of innovative legislation on point-of-care manufacturing.

UK clinical trials

In May 2023, the independent O’Shaughnessy review into UK commercial clinical trials was published. The review set out 27 recommendations where action should be taken by the government and delivery partners, including the MHRA and the NIHR, to address key challenges and transform the UK commercial clinical trials environment.

Speakers in the morning session, including Lord James O’Shaughnessy himself, discussed the progress towards implementing the recommendations of the review and set out additional work that should be prioritised to get UK clinical trials back on track.

Lord O’Shaughnessy advocated for an increased focus at the site level to solve problems relating to initial recruitment, moving trials out of hospitals and into decentralised locations such as primary care and pharmacies, better harnessing of the plethora of technology available to the clinical trial sector in the UK and creating more time for NHS clinicians to get involved with clinical research. Interestingly, Lord O’Shaughnessy also advocated for international recognition of clinical trial approvals in some capacity.

In addition, the MHRA and Health Research Authority presented on the regulatory reform of the UK framework for clinical trials. The reforms will include streamlining the application process, embedding the current notification system, beefed-up transparency obligations and aligning GCP provisions with ICH guidelines. There will be a 12 month implementation period for the new legislation. In addition, the MHRA is evaluating whether it can streamline and enhance its review of clinical trial applications through the use of AI large language models.

Accelerated regulatory pathways in the UK

The International Recognition Procedure (IRP) for medicines, introduced at the start of the year by the MHRA, is open to applicants that have already received an authorisation for the same product from one of the MHRA’s specified reference regulators, including medicines regulators in the EU/EEA, the United States, Japan and others. The IRP allows the MHRA to take into account decisions of these regulators in order to conduct a targeted, expedited assessment of applications for marketing authorisation.

At the conference the MHRA provided an update on how the IRP is functioning ten months on from its introduction. The MHRA presented data on the number of applications it has received through IRP and its excellent adherence to approval timelines (close to 100%). The MHRA set out plans for improving IRP, noting that it will be publishing further updates to the existing guidance on IRP. We note that at the time of writing IRP exists entirely in guidance and has not been put on a statutory footing through amendments to the Human Medicines Regulations 2012. 

The MHRA also used the conference to announce that it is launching an new and revised version of the Innovative Licensing and Access Pathway (ILAP). ILAP was first introduced in 2021 to reduce the time to market for innovative medicines through early engagement on development plans and scientific advice from the MHRA and other bodies such as NICE. The ILAP is being reworked with the NHS now involved as a core partner. ILAP will also be more selective, focussing on medicines that have not yet entered confirmatory trials, providing more opportunities for applicants to benefit from the toolkit available under ILAP. The MHRA used Amicus Therapeutics’ treatment for adults living with late-onset Pompe disease, Pombiliti and Opfalda, as an example to illustrate the potential of ILAP to accelerate access to innovative medicines, as NICE issued final guidance recommending reimbursement of the therapy for use in the NHS just one day after the MHRA granted an MA for the treatment.

Real world data

Speakers from the MHRA and industry set out the significant potential offered by real world data to increase efficiency in the development of medicines. Indeed, clinical trial data is not perfect and bringing this data together with real world evidence would be ideal. However, some challenges stand in the way of industry being able to harness real world data to realise this potential. In particular, it is challenging to find the right real world data at the right level of granularity and quality to use in support of regulatory submissions, including clinical trials. This means that ensuring the quality, completeness, and interoperability of real world data remains a significant challenge.

In view of the challenges, industry called for harmonisation criteria to be issued so that a rigorous methodology can be applied to real world data. In this regard, a workable framework for handling and incorporating real world evidence and ensuring the predictability of the data will help to ensure the optimal use of real world data and evidence where it can add most value. Improvements in the healthcare data infrastructure and the establishment of robust data governance frameworks would help make advances with the use of real world data. Industry called for collaboration with the key stakeholders to improve current frameworks and make significant progress with the use of real world data after years of stagnation in this field.