Bringing New Science, and New Partners, Into the Drug Development Process

Today’s environment for healthcare delivery requires that we find new ways to innovate. We are asked to create therapies and medical devices that can offer measurably better outcomes for patients. However, the market’s demands for better and safer treatments and lower cost healthcare appear to be in conflict with an innovation process that is unpredictable, requires trial and error, and as a result is quite costly. This conflict in turn creates concerns among some of the investors whose capital we rely on.

We could choose to discover and develop products in the same way we always have—and then hit a wall when we can’t price our products to cover our cost of research and development costs. Or alternatively, we could choose to embrace the seeming contradiction. We need to be willing to accept new definitions for success. As we do so, it will be critical that we design a more efficient path to achieve success, and ensure that we make critical decisions as early as possible to reduce the high cost of failure. Today’s market is increasingly interested in value metrics that had not been central to the way that treatments have been developed in the past. Pharmacoeconomics is entering the conversation along with the traditional measures of safety and efficacy.

Recently, the exploding understanding of our genetic makeup, and how that biology interacts with our environment, provides us with greater insight on a variety of disease etiologies. This understanding also allows us to shift from a focus on broad-based disease states—like Type II diabetes or cardiovascular disease—to being able to target medicines to more distinct patient populations with a particular sub-type of the disease—and even to the molecular pathways that may give rise to the symptoms. Our research today also benefits from advances in stem cells, RNAi, gene sequencing and diagnostics that can open up new pathways—allowing us to target therapies more effectively, and develop them more efficiently.

From large pharmaceutical companies to small biotechs and academic centers of excellence we are seeing a dramatic increase in collaboration for discovery and the early stages of development. Increasingly the “Aha!” moments of innovation will occur when experts of different disciplines approach problems together. This change in turn also has implications for the culture of pharmaceutical companies. Going forward, we will be part of a much broader ecosystem, with shared ownership of the drug discovery and development process among a number of players. There will be multiple paths to and through development, and pharma will be a valued participant in a more-open innovation process. Certain core areas of research will continue to benefit from the work of tightly knit internal teams of scientists, but discovery and development now involves a variety of collaborative approaches.

These are just the superficial manifestations of more fundamental change in how we must approach an unmet medical need today. The deeper change involves how we approach the problem to begin with. Today we need to think through how to optimize a treatment’s real and perceived value within its anticipated healthcare setting. We need to approach these potentially more complex development programs with a willingness to forge new paths—working closely, and early on, with academia, regulators, and payers.

As we move forward and test new business models we will need to deal with new sets of risks as well as challenges in the macro environment. Some of the newer approaches to innovation for our industry involve emerging or unproven science, or populations that are difficult to study. So these new approaches can add significantly to risk.

For this journey to be productive, and to meet the needs of all our constituencies, we’ll need to create new ways for stakeholders in medical and pharmaceutical research and development—as well as regulators, payers and patient advocates—to pull together. Professionals in project management can play leadership roles in creating development paths that produce differentiated treatments for better medical outcomes—while incorporating approaches that get these treatments to the bedside in a cost effective manner.