Criticus Among Us

Having been witness to, and part of, the evolution of the pharmaceutical industry over the past three decades, my foremost thought is of our remarkable contribution to public health. This was achieved through leading science, an exemplary evolution of translational medicine, and a business model anchored in measured milestones, envious interdisciplinary collaboration, and creative scientific persuasion of end users.

Pharma Put the “Translation” Into Translational Research

It was a celebrated day when industry scientists Sir James Black, Gertrude Elion, and George Hitchings were awarded the 1988 Nobel Prize in Physiology or Medicine for their work in drug development. Drugs stemming from their work ranged from a variety of receptor blocking agents to therapies for such diseases as malaria, cancer, and a host of infections, all having a lasting impact on alleviating disability and disease. The development of the leukotriene blocker montelukast for asthma and rhinitis by Merck – within a decade of the 1982 Nobel Prize for describing the metabolism of substances liberated from cell membranes – is another. Then there is the central role of antiretroviral therapy and the associated decline in HIV/AIDS death rates. Truly remarkable. Finally, the role of education in conjunction with pharmacotherapy, such as the “know your numbers” cholesterol campaign, was a primary societal force in getting the masses knowing and acting on their lipid profile both through modified lifestyle and, when appropriate, statin therapy.

Equally acknowledged is the continued presence of skeptics who fear persuasion when not coming from within their walls, and refuse to recognize that industry commercial practices have demonstrated a remarkable change for the better. Not to debate further – there are tarnishes in every industry, including academics – but the overriding commitment to do good and improve public heath is balanced on the right side.

Have the Laurels Wilted?

Even if one agrees with my take on an industry that has done exceedingly good things, it may also foster the “that was then, and this is now” lament as we face very fresh and uncharted challenges. The evolution of focus from effectiveness to cost-effectiveness, and to an efficient system integration of pharmacotherapy, is putting unique stresses on current models.

Does the industry have the competencies to do the same good for optimal integration of innovation as they have for discovery and development? Will the industry practically recognize that premium costs without harvestable savings to systems are no longer sustainable? Can we handle success rates of less than 10% and development costs surpassing $2.5 billion in some estimates? Can we sustain declining projected peak sales of assets approaching 50%?

Navigating the Treacherous Path Ahead

This is beginning to sound very difficult, even dangerous – two words often associated with crisis. In fact, one does not have to read too long or surf too far to find critical, fact-filled essays on a pharma industry in turmoil: R&D crisis, innovation crisis, strategic crisis, productivity crisis, and so on. Admittedly, there is a bit of drama in the word “crisis,” but difficulty and danger requiring attention is no overstatement.

There is no panacea to address the difficulties of the day, but as long as science prevails, our business will have opportunity. My thought, “if we could do just one thing,” is to somehow capitalize on our mastery in bridging the first “valley of death” from science to drug and building a similar grand bridge over that second valley from drug to health. We need to make it our business to understand heath systems and organizations in such a way as to implement effective and efficient drug technologies. Sometimes, science even gets ahead of human behavior and organizational inertia, as seen in the timely testing of genetic mutations in the cancer patient journey. But integration must become the most critical milestone in the industry. Would some of the crisis mentioned above be less critical if this is addressed earlier and deliberated?

While I believe building a bridge over the second valley of death is crucial in implementing the impressive science that could lead us to better diagnosis and therapy, the first bridge needs its preventive maintenance as well. It would be both a crisis and a pity if we optimized implementation only to find discovery and development in trouble.

While pharma has been somewhat slow to navigate fresh seas, there may be some catalytic conversion from another stakeholder who often sets the rules: the government. The United States Congress has been introduced to The 21st Century Cures Act, which carries the tagline “Help and Hope to Patients Through Biomedical Innovation.” That sounds a lot like the mission statement of most pharma firms. With a formal recognition of the difficulty we are speaking about, the act highlights areas of collaboration and coordination within biomedical ecosystems, patient perspective in drug development, modernizing clinical trials, removing regulatory uncertainties, and a host of mandates that demonstrate willingness to address difficulty before danger.

One caution in streamlining the process to preserve the good: Pharma and regulators will need to work hand-in-hand in evaluating the safety component of the risk-benefit ratio, remembering that the absence of evidence is not the evidence of absence.

The word “crisis” was Latinized from the Greek “criticus,” which defined a turning point in a disease. In other words, cure or death. Let’s hope discovery and development keep beating long enough for integration to come into normal range. ////

Dr. Ted Witek is a senior fellow at the Institute of Health Policy, Management, and Evaluation at the University of Toronto Dalla Lana School of Public Health, and senior vice president of corporate partnerships at Theravance. He is based in Toronto, Canada and San Francisco, United States.

the author

Dr. Ted Witek

Dr. Ted Witek is a professor and senior fellow at the Institute of Health Policy, Management, and Evaluation (IHPME) at the Dalla Lana School of Public Health at the University of Toronto and Chief Scientific Officer at Innoviva in San Francisco. He is an advisor to the Design for Health program at OCAD University.