Science Moving Forward
One might conclude that, should Big Pharma stumble, the science of drug innovation will proceed elsewhere. This may be true.
The discovery mantle will shift more decidedly to academia, their derivatives, or for-profit startups, and Big Pharma will
simply revert to its traditional strengths: covering late clinical testing, regulatory, manufacturing, and marketing. To a
large extent this is already happening. However, the long-term effects are still cause for concern. For example, will venture
capitalists and other investors continue to fund startups, given the growing concern over the state of Big Pharma and what
that means for a viable exit strategy? What happens when government seeks to drive disease priorities—does it have the staying
power to be consistent and on point during the long pathway toward commercialization? Is disease target prioritization through
public intervention necessarily better than private incentive? Is it foreordained that industry should just wash its hands
of stimulating the bread-and-butter of basic discovery work?
The traditional model of drug discovery has waned in recent years, due to a focus on bolstering the balance sheet. In order
for Big Pharma to uncover a new "golden age" it must regain its responsibility of being the key driver of R&D. (Getty Images
/ Don Bishop)
Such concerns are valid. To declare defeat and advocate a dismantling of what remains of the industry's extended R&D infrastructure
(as some on Wall Street have done) could cripple the best weapon we have against the ever-changing panorama of chronic disease,
which is biomedical innovation. Until we fully dissect the elements of the so-called "drug drought" and understand what is
happening and why, it is premature to pronounce the traditional model "dead."
During the so-called "golden age" which culminated in the mid-'90s, over 30 new drugs on average were brought to market each
year. Life-saving and enhancing new product classes abound. Statin drugs, prescribed to over 40 million Americans today, are
credited with having a major impact in reducing cardiovascular morbidity and mortality. Drugs that interfere with platelet
function and clotting factors have granted further benefits. The protease inhibitors that have transformed HIV/AIDS from a
quick killer to a chronic disease could not have reached patients as quickly without the industry's innovative expertise in
clinical certification, safety profiling, and efficient manufacturing. Likewise, the major advances in transplant surgery
would not be possible without medicines to control tissue rejection. Pharmacologic cancer cures are now emerging for adults
as they have over the past several decades for children. Finally, unlocking of the human genome promises targeted therapy
and tailored treatments for the most vulnerable populations, as well as better health outcomes at a lower cost.
There are many therapeutic successes, yet the pharmaceutical industry is still within the crosshairs of public skepticism.
The issue stems, in part, from a crisis of confidence within industry—an inability or unwillingness to stand up for a better
social contract, where individual reward is evaluated against the larger benefits to society, even when such benefits cannot
always be quantified. The pharma industry produces an essential good that builds on a legacy of centuries of progress in science,
yet many CEOs fail to articulate the connection between what we died of yesterday and how we live today. Have the industry's
leaders lost their identity as passionate innovators, for the sake of being proficient managers?
To Shape the Debate, Marshal the Evidence
It would be unfortunate to accept a future built around Big Pharma as little more than a scaled-up distribution platform.
Industry should articulate the perils to future patient-centered innovation if the collaborative equipoise between academia
and industry is dismantled. Biomedical innovation is not a contest of methodologies, but rather a composite national resource.
To create this fresh dynamic, the reasons that explain success and failure in industry labs must be dissected, in far greater
detail than it has been to date. A suitably resourced, high-level analysis and explanation is overdue. It is up to industry
to take the lead in defining problems in its own shop, and to then build a global policy consensus on how to fix them. For
example, industry's chief scientists—the company heads of R&D—should state plainly as a group that more help is needed from
payers, patient groups, and other stakeholders to make better decisions on what compounds to bring into development. We suspect
that from this endeavor, the need for new incentive-based policies will become apparent. That need, in turn, will drive a
new level of engagement—with government and other partners—for the one proven engine of innovation, which is the private research
enterprise. Protecting this rich resource is good for patients and society. This opportunity must not be squandered.
Eve E. Slater, M.D., is Associate Clinical Professor of Medicine at Columbia University's College of Physicians and Surgeons. She can be reached
Kenneth Kaitin, Ph.D, is Director of the Tufts Center for the Study of Drug Development and Professor at Tufts University School of Medicine. He
can be reached at firstname.lastname@example.org