Patient-Centred Drug Development Ecosystem for Rare Diseases: The Rare Disease Consortium Japan (RDCJ)

Abstract

The Rare Disease Consortium Japan (RDCJ) is a co-creation platform founded on the philosophy of “leaving no one behind” and dedicated to advancing drug development for rare diseases. It brings together industry, patients and patient groups, academia, government, and citizens to connect the entire pathway from basic research to clinical application and social implementation. By overcoming institutional and sectoral boundaries, RDCJ accelerates consensus-building and aligns development with the patient’s timeline. The approval of viltolarsen, an exon-skipping antisense oligonucleotide therapy for Duchenne muscular dystrophy, demonstrated that Japan can establish rare disease drug development processes consistent with global standards. Moreover, the expansion of exon skipping illustrates how a foundation built for one disease can be extended to other rare diseases and therapeutic modalities. As a shared platform, RDCJ promotes standardisation through annual meetings and working groups, leverages cross-disease infrastructure, and links Japan’s innovations to the world─delivering new treatments to patients without delay.