Process Improvement for Maximized Therapeutic Innovation Outcome

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When considering therapeutic innovation, we naturally focus on biological discovery and the associated advances in technology, which have revolutionized clinical management paradigms and the delivery of care to patients and populations.1 This evolution reflects the exponential growth in bioinnovation propelled by public–private partnership investment in generating platforms for solutions to health and disease that benefit communities, now and in the future.2 This scientific revolution drives the development of increasingly precise solutions, leveraging insights in molecular mechanisms within a systems context underlying pathophysiology, which offers biologically based targets for novel therapies, enhance the ability to find cures, and restrict adverse events.3 Indeed, the increasing toolbox of cutting‐edge platforms has produced unprecedented opportunities to individualize and indeed optimize drugs, devices, and their delivery that can be best aligned across the spectrum of diseases, communities, and geographies to reach global populations in need.1 The biotechnology and pharmaceutical industry, in turn, has translated these biological advances into new preventive, diagnostic, and treatment approaches that are evolving health and the care of patients and their diseases in ways that were only imagined a decade earlier.2 The developing framework established by biologically targeted biomarker, device, and therapeutic paradigms alters the one‐size‐fits‐all method to managing patients into individualized health solutions.4 These developments are poised to advance, and that acceleration is reflected in emerging fields like regenerative medicine, which is poised to drive the management of degenerative diseases and wellness through direct manipulation of innate regenerative reserves for tissue and organ renewal.5
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.
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