Process Improvement for Maximized Therapeutic Innovation Outcome

Abstract

Deconvoluting key biological mechanisms forms the framework for therapeutic discovery. Strategies that enable effective translation of those insights along the development and regulatory path ultimately drive validated clinical application in patients and populations. Accordingly, parity in What vs. How we transform novel mechanistic insights into therapeutic paradigms is essential in achieving success. Aligning molecular discovery with innovations in structures and processes along the discovery–development–regulation–utilization continuum maximizes the return on public and private investments for next‐generation solutions in managing health and disease.

Health Care Evolves From Reactive to Proactive

“…In that context, the growing toolkit of enabling omic and informatics platforms has yielded extraordinary opportunities to customize curative therapies that can be deployed to individuals, communities, and global populations . Indeed, molecular and technological innovations have been translated by a burgeoning biopharmaceutical industry into new paradigms that interrupt the lifecycle of disease, whose reach and impact are amplified by international regulatory agencies that ensure safe access to global populations . Moreover, clinical paradigms emerging from these molecularly directed therapies shift the “average patient” model of healthcare management to individualized solutions for disease interventions .…”

“…This, in part, reflects a healthcare delivery system that places a priority on reimbursing for the treatment of chronic illness rather than preventing disease in the first place . In that context, advancing molecular and regenerative approaches, in conjunction with enabling informatics and omic technologies, offers unique opportunities to evolve preventive strategies that promote healthy aging and longitudinal wellness across the continuum from inception to senescence …”

“…These considerations underscore the importance of maintaining a focus on preventive approaches that complement therapeutic interventions, as our mechanistic armamentarium evolves from discoveries of the biological bases of health and disease . Although molecular innovation has been impressive, the translation of those discovery‐based insights into preventive therapies for individual patients, which are scalable to populations, must remain a priority . Emerging clinical paradigms should maximize the impact of discovery innovations by facilitating their translation into novel preventive algorithms to maintain longitudinal health and wellness, at all points along the developmental continuum.…”

Phase 0/microdosing approaches: time for mainstream application in drug development?

“…developed. They will enhance the simulation of tissue exposure and the extrapolation of time-limited and local ITM data to continuous, therapeutic-level systemic exposures 176 .…”

The Regenerative Horizon: Opportunities for Nursing Research and Practice

“…In addressing unmet patient needs, the restorative approaches of regenerative technologies are poised to offer therapies that go beyond symptom mitigation. With a deeper understanding of disease causes, it is progressively within reach to consider a therapeutic toolkit targeted on disease underpinnings, not just on symptom relief (Waldman & Terzic, 2018 ). For example, regenerative therapies have the potential to halt the progression of degenerative joint disease in the knee or the hip.…”