The Value Proposition of Molecular Medicine

Managing the Innovation Supply Chain to Maximize Personalized Medicine

“…Personalized medicine is having a transformative impact on disease risk assessment, prediction, prevention, prognosis, and cure 9 , 11 . The present discussion highlights the operational flow of technology along a supply chain of innovation, from laboratory to end users, which requires management at each individual step to maximize health‐care and economic benefits.…”

“…What may be less appreciated is the complexity of processes leading from laboratory‐based discovery to individualized patient management algorithms in practice. We typically consider the science of translational therapeutics in discrete sequential steps in which the flow of information and technology moves from left to right along a continuum, from fundamental discovery of molecular principles and therapeutic targets to development involving clinical trials that prove efficacy and safety, through regulatory approval that certifies the utility of the management approach, to application in patients and populations—the “DDRU continuum.” 3 , 9 , 10 , 11 , 12 This model broadly categorizes the stages translating invention into practice. However, it obscures the integral contributions by individual practitioners and domain‐specific experts as well as the impact of the external environment, all of which shape the process at every phase, from discovery to application.…”

Molecular Insights Provide the Critical Path to Disease Mitigation

“…The foregoing examples underscore the remarkable impact that decoding specific molecular mechanisms can have on advancing the management of life‐threatening conditions 18 , 19 . These examples also provide a striking contrast to diseases that are emerging as the next generation of global pandemics, on the one hand, but for which we have not yet achieved defining molecular insights that can be translated into novel therapeutic interventions for treatment and prevention, on the other.…”

Systems-Based Discovery Advances Drug Development

“…Accelerated drug discovery that ensures fit‐for‐purpose product development is a cornerstone of state‐of‐the‐art clinical pharmacology and therapeutics. Reflecting the convergence of multidisciplinary breakthroughs, contemporary discovery is propelled by the revolution in the new biology that is providing remarkable insights into the resolution of human pathophysiology, the emergence of enabling technology platforms that facilitate quantification and validation of disease‐specific biomolecules, and the evolution in biomedical informatics that integrates and deconvolutes vast arrays of data sets 1 , 2 , 3 . Yet the cost of drug development continues to rise—exceeding $1.3 billion to bring a single agent successfully through the discovery–development pipeline.…”