Response to ‘Non-profits wade in where for-profit life science investment fears to tread’
Barbara Nasto’s insightful feature on the growing importance of non-profit organizations in life-science innovation1 rightly highlights how entities such as the Drugs for Neglected Diseases Initiative (DNDi), Medicines for Malaria Venture or Coalition for Epidemiological Preparedness Innovations (CEPI) have redefined translational models for global health. Yet the article could be viewed through a wider lens — one that also captures the profound transformation occurring in the rare-disease ecosystem, where patient-led and philanthropic organizations have become powerful drivers of biomedical progress.
In recent years, hundreds of disease-specific patient organizations have evolved beyond advocacy to act as de facto R&D entities — funding early discovery, de-risking translational steps and co-developing clinical programs with biotech and pharmaceutical partners2. These groups, often founded by affected families, exemplify what we have termed philanthropic drug development: a model in which philanthropic capital and patient expertise merge to propel therapeutic innovation where traditional incentives fail3.
