APR Sept/Oct 2023 - 108

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Sven identified the two most prominent barriers as financing, and how
to get ground-breaking 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.
Other issues include how to keep the costs of treatments down, he said,
particularly so that they are accessible by patients in low- and mediumincome
countries; and how to work cross 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 organizations.
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 highquality
process development. Although optimization and targeted
automation can help bring down the cost of goods, talent remains
an issue.
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 commercialization potential,
he noted.
Moving the Needle: The Measurable
Impact of Advanced Therapies
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 multi-faceted 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
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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 hemophilia; 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. 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. " 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 the
particular country or to the payer or the reimbursement authority. "
Communicating Value Across the
Healthcare Ecosystem
The panel acknowledged the criticality of effective communication
of value which is often missing in the whole discipline of new drug
commercialization - 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. "
APR Sept/Oct 2023

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