Top 20 play­ers: Every­body loves an or­phan — es­pe­cial­ly when they come with block­buster sales and a long run of ex­clu­siv­i­ty

Rare dis­eases are a big busi­ness in the bio­phar­ma world. And they’re get­ting big­ger every year.

Ad­van­taged by reg­u­la­to­ry groups in the US and the world over, at­tract­ed by low­er re­search costs with small­er stud­ies and the prospect of field­ing a block­buster with a small group of reps, bio­phar­ma has re­spond­ed with a burst of new ther­a­pies. And now that the gene ther­a­py era is up­on us, we’re see­ing the big play­ers step up to buy a sig­nif­i­cant po­si­tion for them­selves in this boom­ing mar­ket.

Eval­u­ate’s Karen Pomer­anz just wrapped up a deep dive on the top­ic, and con­clud­ed that we’ll con­tin­ue to see dra­mat­ic growth in the field of or­phan drugs as these trends con­tin­ue to play out with a grow­ing pot for the top play­ers to draw from.

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