Process Improvement for Maximized Therapeutic Innovation Outcome
Clearly, this revolution in biology and molecular discovery is the engine of disruptive innovation that ultimately propels the development of novel paradigms to maintain health and treat disease. However, regardless of the strength of that engine of invention, clinical translation of basic innovation can only advance at the rate of the slowest component of the discovery–development–regulation–utilization (DDRU) continuum.2 Translation and ultimately adoption into the clinic can only be accelerated if we begin to streamline clinical trial processes.6 Greater rates of success in clinical development will be achieved by innovation in the development of biomarkers that can predict responses, outcomes, and adverse events that advantage novel clinical trial designs.7 Regulatory decisions about the relative value of developing therapeutics will reflect new paradigms in assessing relative risk and benefit.9 Increased access to expensive biological medicines, whose associated prices are unsustainable for healthcare systems with finite resources, will be achieved through novel regulatory pathways encouraging the availability of biosimilars.12 Ultimately, innovation in the components of the processes that translate novel molecular discoveries into cutting‐edge therapies are as important, if not more so, than the molecular targets being translated.2
These considerations are underscored by considering the emerging field of regenerative medicine, which is revolutionizing all aspects of therapeutic disease management, with a particular focus on degenerative diseases.13 The paradigm suggests that we can improve the endogenous regenerative capacities of tissues that undergo disruption because of injury, disease, or chronic insult by stimulating tissue‐specific regeneration, and/or amplifying endogenous repair propensity.14 For example, articular cartilage damage ultimately progresses to endstage osteoarthritis, affecting about a million people in the US.15 In that context, autologous chondrocyte implantation (ACI) regimens have become standard‐of‐care in specialized orthopedic clinics focused on osteoarthritis.15 Similarly, myocardial damage from ischemic heart disease has produced an explosion in chronic heart failure with its associated morbidities and mortality. This is another example in which regenerative approaches through the provision of stem cells instructed to repair the damaged myocardium and restore cardiac function is potentially revolutionizing the management of heart failure.