The Fourth
Industrial Revolution and Regenerative medicine is transforming
health and medicine due to the lightning-speed advances in genomics, genetic
engineering, synthetic biology, nanotechnology, data science, AI, robotics, to
name just a few.

It has
been a whirlwind time of new innovative drugs, devices, diagnostics, and others
that, we can only sit back and shake our heads at some of these breakthrough
technologies. Therapies such as CAR-T, GLP-1, PCSK9, JAK inhibitors and the
ever increasing Pharmacogenomics are staggering.

All
told, the FDA agency approved 59 new molecular entities in 2018 and
49 in 2019. While this was taking place the race for Gene therapies such as
Zynteglo and Zolgensma were joined by 23 other “up-and-coming” gene-therapy
candidates that have reached Phase III and/or registrational trials as of the
first quarter of 2019.

Many of
these research projects are aimed at Cancers, Haematological, Orphan diseases,
CV and Depressive disorders. One just has to admire the exceptional research
that is currently underway from many Pharma/Biotech corporations. Just at a
glance seeing new molecules/drugs such as: Imlygic (Amgen), Kymriah,
(Novartis), Yescarta, (Gilead Sciences), Opdivo (BMS), Imfinzi (Astra Zeneca),
Skyrizi (Abbvie) and the Immunotherapy Keytruda (Merck) is just amazing.

“Immunotherapy
is the next big breakthrough in cancer treatment and, according to the report,
“will become the cornerstone of oncology and, impressively, will likely apply
to almost all types of cancers”

I am
certain there will be the immediate “naysayers” who will question the
inevitable issue – PRICE. Yes –Some of these drugs, and new diagnostic aids,
will come at a huge cost running into thousands of US$. Some will question are
these breakthrough drugs, especially for Orphan diseases, really worth it?

“Developing
a gene therapy can cost an estimated $5 billion. This is more than five times
the average cost of developing traditional drugs” (Genomic Institute Dec 2019.)
Well, no one can deny the incredible advancement and achievements that has been
made in the entire spectrum of Pharmaceuticals. But, let us not forget, that the R&D and new products coming from
all these multinational corporations, are to-morrows Generics or
Biosimilars
.

The point
is we cannot stop discovering what lies “out there”. As Steve Jobs put it so
eloquently (edited)–

“Here’s
to the … the ones who see things differently. … You can quote them,
disagree with them, glorify or vilify them, but the only thing you can’t do is
ignore them because they change things. … They push the human race forward,
and while some may see them as the crazy ones, we see genius, because the
people who are crazy enough to think that they can change the world, are the
ones who do.”

Yes, it is
time we stand up and applaud all the corporations who have the vision to
explore, and develop some of the most significant breakthrough treatments
which, many years ago, was never even on the radar. To the thousands of men and
women who dedicate their lives to find just “that one” – keep going- for the
world needs you!

*Francois Hoffman – former
Pharmaceutical Industry Marketing Executive now Industry Marketing and Training
Consultant