Applying the science gets better
A key factor behind the success is the growing ability of the industry to translate academic insights about the origins of
diseases into development pathways that lead to commercialization. "The entire cycle of development for anti-tumor agents
has been reversed, because today we start with only those patients who we know have the abnormal mutation we are seeking to
target," Novartis Oncology president Herve Hoppenot tells Pharm Exec. "The practical result is that time beyond proof of concept is reduced and the probability of success is significantly increased."
Quintiles SVP for early clinical development Oren Cohen notes that today's "holy grail" for success is a better proof of concept,
which is facilitated by reliance on "enabler" technologies: biomarkers, gene expression analysis, and major application breakthroughs
in modeling and simulation. "The renewed focus on proof of concept is no surprise, because it gives us firmer evidentiary
ground for making that essential go/no go decision in product development. There is real reason for optimism about the pace
of the development process, as we see continued improvements in all the enablers that allow you to define and target your
decision-making," he said. "And bad decisions cost money."
Money too is driving the change in how companies manage clinical trials—complex science, tough disease targets, and risk-averse
regulation has sent their cost soaring. The Tufts Center for the Study of Drug Development has been monitoring protocol design
practices among drug companies for more than a decade. "What we are now seeing, for the first time, is widespread adoption
of new internal review committees to critically evaluate these designs and remove procedures that unnecessarily complicate
the execution and cost of trials," said Ken Getz, Center director for sponsor research. This is good news, as failure to wrest
more efficiencies from trial management puts industry squarely at odds with the pressure that regulators are facing to raise
the scope, quality, and objectivity of the evidence on which they base drug approvals.
Manufacturing is the proverbial pumpkin turned princess; a function that was non-strategic, humdrum, and marginal has now
become essential to seamless global integration. It's a proven cost-saver for companies looking for new ways to do old things.
Technology and custom engineering are unleashing a revolution in process called continuous manufacturing, which is reducing
the number of steps and locales required to transform basic raw materials into a safe and effective drug. Such turn-on-a-dime
flexibility is a real asset in an era where regulatory and payer restrictions can lead to steep variations in market demand
for medicines across countries. A big competitive differentiator will be among those companies that succeed first and consistently
do best in crossing the "execution gap," between what's in the standard operating plan (SOP) and actual performance on site,
in the field. This is a matter of necessity for the top generic suppliers, who are seeking leadership in manufacturing excellence,
not only as an add-on business opportunity but also to remove the sector's historic taint of inferior quality against branded
A final word: Give me growth!
In many ways, 2013 marks another year of transition away from the stubborn complacency of an industry used to margins that
other sectors might call nose-bleed high. The industry now embraces the concept of partnership with gusto; the clannishness
of the past has been silenced. "Thinking in systems," is how John Doyle, SVP for Market Access at Quintiles calls it. " Every
company today must begin by framing their market as a network of interconnected products and add on services."
Yes, there is change and there is hope –even if the balance sheet on the business of illness has shifted a bit to the night
side. The best news the Big Pharma giants of the United States and Europe could get this year? Five percent real growth in
GDP. When the tide is running in, you no longer have to see who's been swimming naked.
William Looney is Pharm Exec's Editor-in-Chief. He can be reached at email@example.com