Pharma USA 2023

Mar 28, 2023 - Mar 29, 2023,

Pioneer unparalleled stakeholder engagement at North America’s leading commercial event

Getting ready for primetime in cell & gene therapy

As thousands of novel cell and gene therapies advance along the development pipeline, pressure mounts for scalable treatments

Cell and gene therapies (CGTs) have a lot going for them from an industry perspective – with a market predicted to reach US$50 billion annually by 2027,   an opportunity to expand the frontier of medical science and the potential to save or improve countless lives. But with logistical, supply chain and manufacturing complexities interwoven into development, bringing CGTs to large patient populations may prove uniquely challenging.

With over 3,633 therapies in development as of mid-2022,  CGTs are two of the fastest-growing areas of healthcare. To date, less than two dozen therapies have made it to market, but this number is expected to grow exponentially.  

So far, therapies have been niche, serving relatively small patient populations and costing hundreds of thousands of dollars. But new treatment possibilities are emerging across a broad range of diseases - including leukaemia, haemophilia and sickle cell disease - that could extend CGTs for use as frontline treatments for hundreds of thousands, even millions, of people. 

Genetic and cell engineering advances are driving this rapid evolution. Regulatory bodies are also becoming increasingly accommodating of CGTs. In the US, several FDA-led CGT initiatives and two new guidance documents for CGTs were released this year, sparking optimism surrounding market growth in 2023 and beyond.  The UK has also released new guidance on the development of advanced therapy medicinal products (ATMPs, a term for cell, gene and tissue therapies used in the UK and Europe). 

So, how can CGT developers best develop CGT products for broader markets?

Develop a portfolio of products to benefit from standardised approaches
One way to scale up is developing a portfolio of products within the same class rather than individual therapies. This facilitates standardised approaches, input materials and analytics, which can help scale up CGTs to treat larger numbers of patients more affordably, says Matthew Durdy, Chief Executive of the Cell and Gene Therapy Catapult in the UK. 

“If you can find a way of making components of a therapy standardised, as well as getting it to fit within the healthcare system, there’ll be a great deal that you can leverage into the next product, reducing the cost of development’, says Durdy.

A collaborative approach will also be key here, he adds. “Collaboration is at the heart of bringing advanced therapy medicinal products or “ATMPs” to larger patient populations’, says Durdy, advising developers to “work with the healthcare system’ and ensure that buyers, users, manufacturers and patients collaborate to reduce complexities within the system as much as possible.”

Focus on creating operational excellence
Any CGT launch must content with a range of complex variables including logistics, medical, manufacturing and supply chain complexities. To deal with these challenges, a core team for clinical development, manufacturing and commercialisation should be brought together as soon as possible.
Successful CGTs can be as much about operationalising treatments as they are about the therapy itself.

A highly effective treatment with restrictive requirements on when and how it can be administered, could be rendered useless if those requirements are not met. This means core capabilities and competencies must be in place before launch.

Robert Sexton, VP of Program and Alliance Leadership at US-based Mustang Bio, emphasises early planning for CGT launches. “There’s tech transfers and manufacturing capabilities that need to be in place for cell handling. You have to open treatment centres, and those contracts take time. There’s also intense patient-by-patient management needed, currently.” 

Many CGTs such as CAR-T therapies, encompass a ‘just-in-time’ (JIT) delivery model involving managing capacity across multiple checkpoints. This requires highly sophisticated planning capabilities, says Sexton. “It’s real-time, multi-dimensional management. Most capacity planners in treatment centres and manufacturers think about ‘How many beds will I need? How much lab space, shipping or couriers will I need?’ Then they create a set of assumptions to simplify the math problem to estimate capacity needs over time,” says Sexton. “The model doesn’t account for the arrival time of the patient, which is very dynamic in CGT and can disrupt the model as it’s not easy to predict.”

Phased scale up 
Meeting the challenge of scaling up treatment capacity for larger markets but avoiding scaling beyond commercial and organisational constraints is going to be key. 

When scaling up treatment capacity, planning can go badly awry by misestimating demand, says Sexton. “Even the biggest and best biotech or pharma organisations in the world can say ‘we’re going to build manufacturing capacity for 10,000 patients in a year’ and still get it wrong if the 10,000 patients don’t arrive distributed according to their planning assumptions,” says Sexton. 

“Despite a large total capacity, the operation can only handle a certain number of people at any given point in time. If your influx of patients exceeds your maximum throughput for that time period, you will still have capacity issues, and that’s your rate limiter’.

Scaling beyond commercial capabilities can damage profitability, or could potentially make a treatment unviable. To avoid this, onboarding and activating new treatment centres in phases can help prevent waste until the optimal number of centres is reached. Additionally, centres with commercialisation experience can be advantageous, particularly those staffed appropriately with supportive senior leadership.

Cross-functional planning 
Cross-functional strategic planning and a thorough understanding of the patient population can allow developers to scale up at the right pace, says Michael Hirschmann, VP of US Commercial Development at Legend Biotech. “To plan when and how to scale, you first need to work out what percentage of patients, based on your profile and your label, you think would be eligible for the target product.”

Market research and claims data can help answer these questions, says Hirschmann. “They’ll give you a good understanding of how many patients may be at a treatment centre at a given time. Claims data can tell you how many patients there are at a location. On top of that, treatment centres know what number of patients have been diagnosed with a certain disease, and there may also be data on referrals to the centre.”

The clinical trial marketplace is another factor to consider, says Hirschmann. “Physicians often route patients into clinical trials based on eligibility and availability. Every piece of data helps you to triangulate market size.” 

He also notes that capacity must be top of mind when marketing treatments. “When you’re not to scale, you don’t want to drive those patients prematurely to a centre. You’re just going to frustrate the centre and frustrate the patient, so a commercial consideration is how much awareness you want to create at any given moment.”

Plan for the monitoring and safety requirements
Scaling CGTs to serve relatively large patient populations also requires watching patients closely to ensure against side effects, which requires centres to have the resources to do so, says Hirschmann. “These are highly intensive therapies that take a lot of handholding.” 

Durdy believes this is one aspect of the industry that is developing quickly, and that the intensity of safety monitoring will reduce with time. “With the vanguard, we keep an eye on everything. We have intensive care beds nearby. We need to protect the patients. That’s at one end of the spectrum. But just one notch in, and you’ve now got CAR-T therapies coming through, for instance, where adverse events are much rarer.” 

According to Durdy, some gene therapies may have the potential to be delivered in outpatient settings in the future, allowing an increased volume of patients to be treated. “We've got a lot of learning to do, and conditioning and working within the hospital systems, but problems will be greatly reduced in the future. Therefore, you’ll be able to treat larger numbers of patients more quickly.”

Pharma USA 2023

Mar 28, 2023 - Mar 29, 2023,

Pioneer unparalleled stakeholder engagement at North America’s leading commercial event