14 Accelerating Development of Therapeutics for Preparedness, Response, and a More Secure World

Abstract

AbstractThe accelerated development of therapeutics is essential to infectious disease preparedness and emergency response, just like vaccines and diagnostics. Vaccine research received more public attention than therapeutics during the COVID-19 pandemic. Still, existing drugs, monoclonal antibody cocktails, the combination drug nirmatrelvir/ritonavir (Paxlovid®), and other life-saving interventions for COVID-19 are the result of accelerated therapeutics research during the health emergency. This chapter outlines scientific, regulatory, and social factors that affect the entire therapeutics development process from “bench to bedside,” including preclinical and clinical research, regulatory review, manufacturing, and delivery of safe, effective therapeutics before and during an infectious disease outbreak with pandemic potential. There are both similarities and differences in the processes for vaccines and diagnostics. Still, in all three cases, emergency research response goals are the same: to save lives and avert suffering, accelerate the end of the outbreak, and develop measures to prevent and mitigate future outbreaks. Therapeutics preparedness and response for pathogens with epidemic and/or pandemic potential require innovation to: Correct market failures in therapeutics development, e.g., lack of commercial investment in therapeutics for diseases that affect few people or predominantly low-income populations. Accelerate preclinical development. Receive approvals from oversight bodies for trials in emergencies. Implement large-scale, well-designed randomized clinical studies following good participatory practice (GPP) guidelines, ideally structured by a master protocol so individual trials contribute to a statistically powerful whole. Obtain regulatory authorization or approval. Ensure that the products resulting from research are available to all those who may benefit. Throughout the process, multitasking, or simultaneously acting on steps normally done in sequence, is a key methodology to accelerate results without compromising ethical and scientific standards. A clear, practical vision of what a therapeutics preparedness state should be is outlined here. It will require political support and human and material resources to make it possible.