‘This is not a sci­ence prob­lem any­more': Paths emerge for scal­ing up rare dis­ease med­i­cine

In 2014, the Na­tion­al In­sti­tutes of Health post­ed an es­ti­mate that at the cur­rent pace of drug de­vel­op­ment and ap­proval, it would take 1,000 years to get treat­ments for the 7,000 known rare dis­eases.

A pa­per at the time sug­gest­ed speed­ing up that work by group­ing to­geth­er sim­i­lar dis­eases, and then treat­ing these pa­tients with the same ther­a­py in a sin­gle clin­i­cal tri­al.

A sim­i­lar idea — and oth­er ways to go faster — are now get­ting clos­er to re­al­i­ty, pushed for­ward by new tech­ni­cal, eco­nom­ic and reg­u­la­to­ry ap­proach­es. The frame­work is be­ing cre­at­ed by com­pa­nies like Mod­er­na, by sci­en­tists who de­vel­oped cus­tom ther­a­pies that have the po­ten­tial to ben­e­fit a larg­er pop­u­la­tion, and by a moth­er who knows the pain when a med­i­cine doesn’t ar­rive in time.

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