title: Crowdfunding for Personalized Medicine Research.
abstract: Given the current funding situation of the National Institutes of Health, getting funding for rare disease research is extremely difficult. In light of the enormous potential for research in the rare diseases and the scarcity of research funding, we provide a case study of a novel successful crowdfunding approach at a non-profit organization called Rare Genomics Institute. We partner with biotechnology companies willing to donate their products, such as mouse models, gene editing software, and sequencing services, for which researchers can apply. First, we find that personal stories can be powerful tools to seek funding from sympathetic donors who do not have the same rational considerations of impact and profit. Second, for foundations facing funding restrictions, company donations can be a valuable tool in addition to crowdfunding. Third, rare disease research is particularly rewarding for scientists as they proceed to be pioneers in the field during their academic careers. Overall, by connecting donors, foundations, researchers, and patients, crowdfunding has become a powerful alternative funding mechanism for personalized medicine.
authors.affiliation: Rare Genomics Institute, BeHEARD Initiative.
authors.forename: Danielle C
authors.initials: DC
authors.lastname: Fumagalli
authors.affiliation: Director of BeHEARD Program, Rare Genomics Institute, Hanover, Maryland, and Division of Oncology, Stanford University School of Medicine, Stanford, California.
authors.forename: Arvin M
authors.initials: AM
authors.lastname: Gouw
mesh_terms: D035843:Biomedical Research / Q000191:economics*; D063045:Crowdsourcing; D005582:Foundations; D006801:Humans; D057285:Precision Medicine; D035583:Rare Diseases / Q000235:genetics; D012109:Research Support as Topic / Q000379:methods*
keywords: Sanfilippo syndrome; crowdfunding; genetic sequencing; personalized medicine; rare diseases; undiagnosed diseases