The great debate

Optimising the value of novel treatments is a defining discussion of our times

There are a variety of obstacles to the commercialisation of advanced therapies. Dr Christian K Schneider reports on an industry panel debate that thrashed out strategies for a viable path to market.

With so many exciting novel treatments emerging from the labs, biotechs and pharma companies need to understand the potential unknowns as well as the known considerations if they are to optimise their impact in the real world.

In a second ‘Science Huddle’ industry debate hosted recently by Biopharma Excellence, experts from across the field came together to advise on some of the less obvious barriers to commercialisation, and the most effective strategies to ensure a smooth and viable path to market.

Taking part were Keith Thompson, Chairman of natural killer cell therapy company, NK:IO, Michael May, President & CEO of The Centre for Commercialisation of Regenerative Medicine – a Canadian not-for-profit that catalyses investment in regenerative medicine – and Sven Kili, CEO of Antion Biosciences, a UK-based Multiplex Cell Engineering company.

Getting straight to the heart of the debate, the panelists started by giving their respective views on the main obstacles to the commercialisation of advanced therapies today.

Sven identified the two most prominent barriers as financing, and how to get groundbreaking therapies to the patients that most need them around the world. “There are a lot of very similar therapies addressing the same target, so this is about how we direct our efforts to ensure we address more patients with fewer therapies,” he noted.

He added that other issues include how to keep the costs of treatments down – particularly so that they are accessible by patients in low- and medium-income countries – and how to work across industry to make therapies available to patients globally.

This, in turn, highlights the importance of demonstrating how such therapies generate value – requiring collaboration with health technology assessment organisations.

Other challenges involve talent gaps, particularly now that cell and gene therapy manufacturing is maturing. “Manufacturing is now a key gatekeeper for success in cell and gene therapy – that is, robust, scalable clinical and commercial-scale manufacturing,” Michael explained.

“This means having GMP-trained operators and high-quality process development. Although optimisation and targeted automation can help bring down the cost of goods, talent remains an issue,” he elaborated.

Keith felt that beyond access to funding, a key challenge is how to differentiate the various therapies – whether clinically, through how well it addresses an unmet need, and how deliverable the therapy is – along with the treatment’s affordability. Together, these would be the most critical factors influencing commercialisation potential, he noted.

On differentiation and value, the panel considered the concept of competition among similar gene and cell therapies. Would relative success be determined by speed to market, for instance, or could it be argued that even the umpteenth CAR T-cell product would still add value? And what about more traditional treatments as part of the competitive landscape?

From a biotech perspective, Sven felt this was a multifaceted issue, where once being first to market would have seemed the critical determinant of success.

Although more and more cell and gene therapies are being developed, particularly for rare diseases, issues remain, including access to patients with the particular condition when populations are often small, Sven lamented. In such situations, it probably does pay to be the first – at least to be able to secure the patients for studies.

“If we think particularly about CAR-T, discussions with investors often revolve around whether we really need another CD-19 therapy, or are we getting to the iterative stage of the new one being cheaper, or having a better side-effect profile, etc? And I don’t think that’s what’s going to drive the thawing of the financial markets,” Sven noted.

“Rather, we need the big wins – the really strong clinical results; the hard science that’s going to make a measurable difference to patients’ lives. Competition with non-cell and gene therapies becomes absolutely critical here,” he added.

“Now we’re starting to get into some of these bigger use cases – even if these are rare disease indications – such as haemophilia; diabetes; sickle cell disease – where, from a payer perspective, this is a relatively well-controlled disease with a long, associated lifetime cost due to medical symptoms and complications,” he concluded. “Here, the onus is on us to show the value against these much lower-priced therapies that manage patients’ lives, if not cure them.”

Whatever the potential for enhanced patient convenience or comfort, Sven explained, payers aren’t going to be overly concerned about how far the patient has to travel once a week or once every two weeks for a blood transfusion.

He said: “They’re more concerned about the reimbursement side of things, so we need to be thinking very carefully about how we’re positioning our therapies. The real criterion is how we positively influence a patient’s life and make a positive contribution in a particular country or to the payer or the reimbursement authority.”

The panel acknowledged the criticality of effective communication of value, which is often missing in the whole discipline of new drug commercialisation – for instance, around rapid manufacture, which could present a tangible benefit.

“For very severe, end-stage cancer patients, a 17- or 21-day manufacturing run for a CAR-T can be too long, and sadly patients do expire in the meantime,” Sven noted. “This raises the question of whether we should really be using these therapies (which are very expensive) as a last-ditch effort, or introducing them and using them to treat patients earlier – which, importantly, the data does suggest is more effective. So, all of that needs to be part of the communication. Also, the longer it takes to manufacture something, the higher the costs associated with that product.”

Indeed, some of the benefits of allogeneic therapies could occur here as treatments are scaled and batch sizes increased to achieve a better cost-per-cell ratio, Sven noted: “If you can use that comparison, and you can store the cells – e.g., 20 or 30 doses at a hospital – then the cost model improves. All of these are important considerations. But we’re going to struggle to achieve the comparison unless we learn how to talk more effectively about the entire value chain.”

The panel ended by returning to the challenge of funding the commercialisation journey. Referencing his past experience heading up a UK Cell and Gene Therapy Catapult accelerator, Keith said he used to preach the importance of establishing a target product profile very, very early on, and running a ‘skinny’ health technology assessment.

The aim was to understand the reimbursable prices – across the different markets too – and embed these considerations within the strategy. Sven pointed to major changes now underway in Europe and the US, in those markets’ approaches to health technology assessment.

When planning the target product profile, consideration of costs and benefits of a novel intervention in the context of limited resources is a practical necessity. As advanced therapies begin to be applied to more mainstream use cases, understanding the patient pathway becomes increasingly important to optimising the value of novel therapies. 

Dr Christian K Schneider is Head of Biopharma Excellence at PharmaLex.
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