Opinion: There’s no ‘Moonshot’ or ‘Warp Speed’ for rare diseases. There should be

The term “rare disease” is both an apt descriptor and a misnomer. Individually, each rare disease affects a relatively small number of people. But taken together, more than 30 million Americans, and 400 million people worldwide, are affected by one of the 10,000-plus rare diseases — 95% of which have no approved treatment. More than half of those with rare diseases are children, one-third of whom won’t live to celebrate their fifth birthdays.

Despite the urgent need for research and finding new treatments, there is little help in the form of large-scale research funding, like the kind that was made available for Covid-19. There is no Operation Warp Speed for rare diseases; no Moonshot; no multinational or multi-billion dollar coordinated treatment development effort. As a result, parents, caregivers, and patient groups are often forced to become citizen-scientists and drug developers to identify and advance therapeutic candidates to the point where an academic researcher, company, or investor might take an interest.

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