Clinical Translational Science 2020: Disruptive Innovation Redefines the Discovery‐Application Enterprise

Abstract

Vaccines, analgesia, and antibiotics embody some of the most enduring therapeutic breakthroughs that have transformed medicine. Building on such fine paradigms of biomedical innovation, the evolution of technologies has increasingly sparked spectacular advances across the continuum of wellness and disease-spanning medical and surgical specialties. Discovery science—fueled by government and private sector resources—has systematically instituted the principles of modern healthcare delivery ensuring that medical practice is based on up-to-date scientific evidence. The harmony between science, technology, and resources has culminated in a golden age of discovery and translation, eradicating infections, curing cancers, and palliating endocrine and metabolic diseases. Indeed, proven therapeutic and preventive approaches have progressively moved into everyday practice. The success of the science-medicine union has spurred public and private sector investments, further fueling the engine of discovery and enabling technology. This integration produced the new biology, enabling advances in technology platforms to offer unprecedented opportunities in disease prediction, prevention, and even cure beyond the reach of traditional healthcare solutions. Modern science offers outstanding opportunities to probe the innermost workings of the human body, and to understand how events at the subcellular and molecular levels influence the functioning and integrity of the individual as a whole.1,2 Remarkable progress is being made in understanding the molecular, genetic, and cellular origins of disease, and opportunities now exist to uncover practical uses for this new knowledge, particularly in the realm of personalized medicine.3,4 Unexpectedly, however, acceleration in enabling technologies and the resultant insights into basic pathophysiological mechanisms has outstripped the capacity of current scientific and clinical structures to efficiently manage their translation into new paradigms to improve the health and quality of life of individuals, communities, and populations.5 In that context, federally funded research programs have produced hundreds, if not thousands, of new disease-related molecular targets that represent potential diagnostic and therapeutic targets for tailored disease management.6 Unfortunately, their applications remain only a distant promise, reflecting a limited capacity in structures, resources, and the specialized workforces required for their translations into new healthcare paradigms. Compounding these challenges is the reality that 95% of promising therapies brought into clinical development in the private sector ultimately fail because of limitations in efficacy or unacceptable toxicities. Further research is needed to identify, measure, and validate targets and pathways that have been detected in basic studies, and then to develop new clinical applications and rigorously evaluate their effectiveness and safety. Indeed, further understanding of the translation process itself must occur to expedite and expand the adoption of biomedical advances into clinical practice and individual health behaviors. Recognition of the need to rebalance the equation, and bring translation into register with the power, progress, and pace of discovery science and associated enabling technologies is reflected in the recent enactment of the Cures Acceleration Network (CAN), legislation that seeks to bridge the chasm between basic scientific discoveries and new health treatments.7 Part of the Patient Protection and Affordable Care Act, CAN mandates that the National Institutes of Health (NIH) advance the development of highly needed cures by reducing barriers between research discovery and translation. The CAN provisions grant NIH flexibility to carry out therapeutic development projects, and underscore the expectations of Congress and the American public that the NIH will have a catalytic role in moving scientific discovery along the continu