Out-Law Analysis 4 min. read
09 Mar 2022, 11:24 am
The UK government and regulators have an opportunity to build a unique ecosystem in which businesses developing innovative new treatments based on human cells or genes can thrive.
Several UK initiatives are already being explored to address the current challenges faced by industry to make transformative cell and gene therapies (CGTs) more affordable and accessible. These challenges are recognised by policymakers globally, including in the EU where changes in EU law and policy that could make it less costly to develop and commercialise transformative new CGTs are in the pipeline.
The legislative and regulatory requirements are dynamic and are evolving
Post-Brexit, the UK can take a different approach to that being taken in the EU and find its own solutions for incentivising investment in the sector and addressing the problems faced by industry. As we examine below, that process is being undertaken from a position of strength.
The UK has several ongoing initiatives aimed specifically at establishing and supporting a thriving CGT industry.
Acting as a facilitator, the Cell and Gene Therapy (CGT) Catapult aims to bridge the gap between scientific research and full-scale commercialisation. A focus area has been to increase the UK’s CGT manufacturing capabilities with a state-of-the-art manufacturing centre in Stevenage.
The new Life Sciences Innovative Manufacturing Fund (LSIMF) will support businesses investing in life sciences manufacturing projects in the UK. The fund will provide £60 million of support to commercial-scale and manufacturing investments by companies at the leading-edge of innovation, including CGTs.
Addressing the challenge of how to deliver CGTs to patients, the UK also has a network of Advanced Therapy Treatment Centres (ATTCs) that work together to establish best practice for the safe and effective delivery of CGTs to patients. The ATTC’s NHS readiness toolkit aims to accelerate clinical adoption of CGTs, and more generally ATMPs, within the NHS.
With the aim of accelerating patient access, the Innovative Medicines Fund was launched in July 2021. It is envisaged that the fund will provide £680 million of support to the development of clinically promising treatments where further data is needed to support the National Institute for Health and Care Excellence (NICE) in making a final recommendation. It builds on the success of the Cancer Drugs Fund. The finalised fund is subject to public consultation.
The ongoing NICE methods review is also relevant to CGTs. It has the potential to provide more flexibility within the health technology assessment (HTA) process for CGTs and other ATMPs designed to meet high clinical unmet needs.
The UK’s Early Access to Medicine Scheme (EAMS) is to be placed on a statutory footing with the aim of maximising the scheme’s impact by accelerating availability of medicines for patients and reducing the burden on manufacturers supplying EAMS medicines. The new regulations are expected to provide a framework for collection of real-world data in certain circumstances which is positive news for developers of cell and gene therapies.
New accelerated assessment procedures and new routes of evaluation, that are applicable to CGTs and other ATMPs, have also been introduced post-Brexit. The ‘Innovative Licensing and Access Pathway’ (ILAP) scheme aims to accelerate the time to develop, market, and ultimately provide patients with access to new medicinal products. ILAP is open to commercial and non-commercial developers in the UK and internationally and can be utilised by developers of CGTs if the product meets the defined criteria.
CGT companies will remain a strong area of interest for biotech investors for some time to come
Steps have also been taken to streamline the provision of regulatory advice relevant to CGTs too, with a single point of access for advice on regenerative medicine rules available from the Innovation Office within the Medicines and Healthcare Regulator Authority (MHRA).
The MHRA has also released draft guidance on the use of real-world data (RWD) in clinical studies to support regulatory decisions. This guidance is applicable to studies regardless of geographical location. The UK has also developed capabilities for delivering Electronic Health Records (EHR)-based studies for the purposes of comparative effectiveness.
A number of agreements have already been reached that provide patients with access to CGTs in the UK. They include a fast-track deal for Zolgensma, a one-off gene therapy for spinal muscular atrophy and a managed access deal through the Cancer Drugs Fund for CAR-T therapy KTE-X19 for mantle-cell lymphoma.
CGTs are already widely recognised as the ‘next big therapy area’ within the pharmaceuticals industry. This is reflected in the wave of mergers and acquisitions over the last 12 months involving major pharmaceuticals manufacturers such as Sanofi, Takeda and Novartis, to name but a few. With this kind of attention it is clear that CGT companies will remain a strong area of interest for biotech investors for some time to come, not least because as the sector matures into its commercialisation phase, the potential for revenue generation is extremely high.
The UK is already a thriving ecosystem for transformative treatments such as cell and gene therapy. According to a report from the Alliance for Regenerative Medicine and the BioIndustry Association there are over 70 companies developing advanced therapy medicinal products (ATMPs) in the UK, and the UK industry is predicted to be worth £10 billion by 2035.
The UK is now a third country to the EU and as such, for companies operating in the UK, there have been regulatory changes that must be considered in the development, manufacture, and supply of CGTs and ATMPs more generally. The CGT Catapult has published guidance on the development and marketing of CGTs and other ATMPs in the UK and EU post-Brexit.
However, the legislative and regulatory requirements are dynamic and are evolving.
In the year ahead alone, we anticipate, and in some cases have already seen, major developments in areas of UK policy and regulation such as funding, tax, use of data, clinical trials, medical devices and the use of artificial intelligence tools.
It is incumbent on industry to make use of the existing UK initiatives that support the development and commercialisation of CGTs. They should also engage with the reforms in the pipeline to not only understand how they will impact their business but to take the opportunities, when they arise, to voice their views on how policy, legislation, regulation and guidance might be shaped to provide the best possible ecosystem in which to operate.
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