“…Such is already the case in the field of poverty-related infectious diseases and neglected tropical diseases, in which there is a traditional type of what is often termed “market failure,” and other financing mechanisms, such as crowdfunding [36] and prizes [37], are being piloted. There are recent precedents of drugs being taken through such stages with government or philanthropic money and in the absence of patent protection [38], as well as an increasing number of major new funding mechanisms designed to assist with such efforts [39].…”
“…Such is already the case in the field of poverty-related infectious diseases and neglected tropical diseases, in which there is a traditional type of what is often termed “market failure,” and other financing mechanisms, such as crowdfunding [36] and prizes [37], are being piloted. There are recent precedents of drugs being taken through such stages with government or philanthropic money and in the absence of patent protection [38], as well as an increasing number of major new funding mechanisms designed to assist with such efforts [39].…”
“…This could be done through financing by government or private notfor-profit organizations (e.g. WHO, Bill and Melinda Gates Foundation, Wellcome Trust) [63], public-private partnerships, through crowdfunding [64] or pro-bono work by individuals. As discussed earlier, pro-social motivation can be incited through public recognition of individual contributions.…”
Section: Concluding Remarks and Discussionmentioning
“…Just as internet retailers "opened up" the long tail by making niche products available to interested consumers, crowdfunding platforms connect highly-invested individuals to a particular research project. Dragojlovic and Lynd followed five crowdfunding campaigns in 2013, reporting that five out of six met or exceeded their goal (Dragojlovic & Lynd, 2014). The Rare Genomics Institute also uses crowdfunding as a key source of financial support for research projects.…”
There are an estimated 6000-8000 rare Mendelian diseases that collectively affect 30 million individuals in the United States. The low incidence and prevalence of these diseases present significant challenges to improving diagnostics and treatments. Next-generation sequencing (NGS) technologies have revolutionized research of rare diseases. This article will first comment on the effectiveness of NGS through the lens of long-tailed economics. We then provide an overview of recent developments and challenges of NGS-based research on rare diseases. As the quality of NGS studies improve and the cost of sequencing decreases, NGS will continue to make a significant impact on the study of rare diseases moving forward.
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