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This month's Manufacturing Medicines Industry Partnership (MMIP) conference – Propelling growth: taking the next step in innovative medicines manufacturing – took place at an exciting time for the pharma industry. Advanced therapies (ATs) – such as cell therapies in which patients are treated with living cells – have been under development for the last 20 years as we apply our ever-increasing understanding of the genome. But the field has been catalysed by the recent regulatory approval of several very promising products such as Novartis’s Kymriah and GSK’s Strimvelis.

These flagship products put the pharma industry at an inflection point and present an opportunity to move medicine from palliative care to an entirely new paradigm where cures and personalised medicine are fast becoming reality. These new therapies are, however, very complex – and manufacturing needs are significantly different from existing therapies (e.g. small molecule and traditional biotherapeutics). This complexity presents both challenges and opportunities.

Themes and challenges

Roger Connor from GSK kicked off the conference with an introduction on how moving to a new medical paradigm will not be straightforward and what this future will mean for medicines development and manufacturing – and the implications for the wider supply chain and, of course, therapy delivery.

The complexity of these therapies means collaboration at all stages of the lifecycle will be vital. This is especially relevant at the research and development (R&D) stage. Manufacturing these complex therapies is a significant challenge – Andy Evans from AstraZeneca highlighted the need for very early discussion between R&D and manufacturing to build manufacturing strategies around lead therapy candidates.

Once manufacturing processes have been proposed, then robust quality control methods will also need to be established. Cell and gene therapies have a lower R&D attrition rate due to their high specificity. But the enhanced specificity puts more emphasis on manufacturing, as small process fluctuations can have a large effect on the final product and thus therapy specificity.

Currently ATs with regulatory clearance are for rare disease conditions with very small patient populations and low manufacturing volumes. Discussion at the conference highlighted that new therapies under development will move to larger patient numbers. James Miskin from Oxford BioMedica highlighted a gene therapy under development for Parkinson’s disease as an example. It was also said that a medium to long-term challenge will be the development of strategies, tools and technologies that enable manufacture of such ATs in larger volumes.

The delicate nature of the biological materials used in ATs also presents challenges to the wider supply chain – with therapy packaging and delivery now just as important as manufacturing. Challenges here relate to the implications of handling and shipping for product quality and ensuring the integrity of cryogenic conditions to prevent degradation of the delicate biological material affecting therapy quality.

Cost is also a major consideration – commercially available ATs are currently extremely expensive, with each treatment costing hundreds of thousands of pounds. Implementing solutions to the above challenges will have to occur within a cost-sensitive framework to ensure any new therapies are as affordable as possible.

In addition, the complexity of the genome is likely to result in many different therapies, each with their own different manufacturing and lifecycle challenges. It will not be a case of developing one solution for each technical challenge – platform technologies and configurable solutions will be required to provide solutions for multiple therapies.

Innovations

Despite recent success, ATs are still relatively immature. Whilst it is clear these therapies will require new tools and technologies to reduce cost and mitigate manufacturing complexity, it is not clear to the pharma industry exactly what solutions are required – and requirements will continue to evolve as the field matures. This presents opportunities for potential solutions to be adopted early and influence how the AT field matures.

Different speakers at the conference presented on future enabling technologies. These included Mike Houghton from Siemens presenting on digital technologies and Lionel Clarke from the Synthetic Biology Leadership Council discussing synthetic biology. Digital solutions are particularly attractive as development and manufacture of ATs is an inherently data-rich activity. Collection, aggregation and analysis of data at all stages of the AT lifecycle – from R&D and manufacturing to supply chain and therapy administration – offers significant value.

Supply chain challenges were also discussed – including opportunities to implement strategies from other logistic-intensive industries. Just-in-time (JIT) supply chain approaches – as used by the automotive industry - were cited by Anette Doherty from GSK. JIT approaches are particularly relevant given the limited shelf life of biology-derived therapies and could also potentially enable exciting possibilities in decentralised production closer to the point of care.

It is clear there are significant challenges to overcome in the industrialisation of ATs and there is a risk of forcing new therapies to fit into existing solutions and infrastructure. There is, however, an opportunity to develop new tools, technologies and solutions – and implement new manufacturing strategies fit for purpose, as Dave Tudor from GSK discussed.

The inherent complexity of ATs will require pharma companies to be bold and embrace innovation – looking to adjacent industries in addition to developing new first-of-a-kind technologies. These innovations will require a holistic perspective encompassing the entire AT lifecycle, and apply to manufacturing strategies and the wider supply chain, not just R&D.

Despite the challenges, ATs have huge potential – the market for cancer immunotherapies alone is predicted to be worth more than $100bn by 2021. The players – and indeed countries – that are first to understand the challenges across R&D, manufacturing and supply will win the opportunities and become the market leaders of tomorrow.

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