PH3_2017 - page 4

It is expected that by 2030 around 80% of therapies
available in the OCSE countries will be based on
biotechnology. This is also a trend seen in the
diagnostic sector, especially with companion
diagnostic tools, key to the sustainability of future
biotech personalised therapies.
Based on these premises, it is clear that my first
comment is that the future is biotech, including
therapeutic vaccines and advanced therapies.
The growing biotech trend will involve R&D industries,
scientists, central and regional institutions, regulatory
bodies, payers and patients.
From an industrial point of view, starting from the well-
known “network innovation” research model, industries
and scientists - ones working in public universities and
private institutions too - should more and more focus
not only on true innovation but also on the sustainability
of the entire system.
If we look at the price - $ 475,000 - gained by the first
CAR-T therapy developed in the US by Novartis for a
niche paediatric indication, clearly such costs can be
sustainable only for ultra-rare diseases or indications.
Whether these breakthrough cell therapies were
authorized for a wider patient population, such prices
would not be affordable.
Innovative industries have therefore work not only in
order to demonstrate efficacy and safety, but to reduce
development and manufacturing costs too.
Regarding manufacturing, bio-pharma industries have
also to significantly increase the production amount
of recombinant proteins, monoclonal antibodies,
vaccines and advanced therapies; the current one is
almost saturated for the next years. This creates an
extraordinary opportunity for countries that will be
able to attract the huge investments involved, whose
allocations will be defined mainly based on market
access opportunities (e.g. China), better tax conditions
(e.g. Ireland, Switzerland), high expertise and quality
(e.g. Germany and Italy).
The public institutions of the single countries will play
an important role and should try to attract investments
creating a favourable environment by setting simple
rules (patent application process, regulatory approvals
for R&D laboratories andmanufacturing sites, pricing &
reimbursement processes), convenient taxation schemes
and high value/fast market access for innovative products.
This will then create qualified jobs and increase theGDP.
As for the regulatory aspects, the regulatory framework
should be critically reviewed to adapt the entire
Pharma: a industry horizon guided by patient centricity
approval process to new therapies. Adaptive licensing
and early interaction are certainly good steps in the
right direction, but it appears fundamental that both
regulatory agencies and payers update rules and
processes that were created to approve and remunerate
traditional drugs and are no more suitable to evaluate
innovative biotech therapies. This is an open issue
for advanced therapy research and development -
laboratories inspections and the registry schemes
for a cell & gene personalized therapies cannot fit
with the traditional model - but also for pricing and
reimbursement schemes for innovation. Let’s make
a couple of examples to clarify this point: a drug that
can radically modify the natural history of a disease,
like C hepatitis products, cannot be evaluated with
a one-year perspective and only taking into account
efficacy and safety characteristics. Costs saved in
patient management should carefully be calculated
and considered in defining the value of an innovative
therapy. This is the case, for instance, with liver surgery
costs for C hepatitis, but also applicable to costs saved
for acute and chronic GvHDmanagement in patients
who underwent an aplo-identical transplantation
procedure treated with the recently authorized cell
therapy offered by the company I represent. In addition,
gene therapies for rare diseases that can completely
heal the disease with just one infusion could be
reimbursed with multiple-year reimbursement schemes,
so to spread the huge costs involved with these
therapies - in the range of millions of euros or dollars per
patient – over a longer and more affordable time.
Finally, yet importantly, we believe that patients and
patient associations should be involved in the therapy
developing process right from the early phases.
With all the due respect for both manufacturers
and physicians, nobody understands the issues of
managing a certain disease and the related therapy
more than a patient. This is certainly true when we talk
about rare and ultra-rare diseases, but also in many
other cases, in which patients’ active participation
could add significant value.
MolMed SpA
(Biotech ItalianTrade Association)
focus on Pharma Packaging and Excipients
vol. 1(3) 2017
1,2,3 5,6,7,8,9,10,11,12,13,14,...68
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