Advancing Drug Innovation for Neglected Diseases—Criteria for Lead Progression

TLDR

Neglected disease drug R&D pipelines are weak, limiting novel drug registration, and success depends on access to technology, resources, strong management, and clear progression criteria. The study calls for sustained investment and heightened focus on upstream discovery, examining how these factors have shaped tropical disease drug discovery and highlighting opportunities and challenges through a virtual North‑South network and increased participation of developing‑country institutions. The authors present criteria to streamline progression from screening hits to drug candidate selection, guiding ongoing efforts.

Abstract

The current drug R&D pipeline for most neglected diseases remains weak, and unlikely to support registration of novel drug classes that meet desired target product profiles in the short term. This calls for sustained investment as well as greater emphasis in the risky upstream drug discovery. Access to technologies, resources, and strong management as well as clear compound progression criteria are factors in the successful implementation of any collaborative drug discovery effort. We discuss how some of these factors have impacted drug discovery for tropical diseases within the past four decades, and highlight new opportunities and challenges through the virtual North-South drug discovery network as well as the rationale for greater participation of institutions in developing countries in product innovation. A set of criteria designed to facilitate compound progression from screening hits to drug candidate selection is presented to guide ongoing efforts.