The Rare Disease Moonshot: Paradigms Shift, Translational Medicine, and Regulatory Science for the World's Rarest Conditions
Year: 2024
Type: article
Abstract: Despite significant initiatives demonstrating the value of multistakeholders' collaborations there remain gaps and hurdles for the world's rarest conditions that currently have no therapeutic options.New approaches are required to transform the Rare Disease research landscape, with an urgent need for targeted public-private partnerships (PPPs).The Rare Disease Moonshot was initiated in 2022 as an informal coalition of the willing to actively support these PPPs.The complementary expertise of its members ensures that funders of research programs from public and private sectors are well-informed to deliver sustainable research that quickly yields more safe and effective medical products.Such collaborations are critical to address the gaps and challenges in therapeutic development.
DOI: 10.1002/cpt.3428
Institutions: Critical Path Institute, AstraZeneca (France), European Clinical Research Infrastructure Network, European Organisation for Rare Diseases, Biobanking and Biomolecular Resources Research Infrastructure Consortium, European Infrastructure for Translational Medicine, Boehringer Ingelheim (Germany), European Federation of Pharmaceutical Industries and Associations
Language: English
Subfield: Physiology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,200
OpenAlex ID: W4401824300