The 2018 break­down on a chart-top­ping set of new drug ap­provals — and the 5 big things we learned from it

— Af­ter 5 years of his­toric in­vest­ment in drug R&D and star­tups, we’re look­ing at the trans­for­ma­tion of a new gen­er­a­tion of biotechs in­to com­mer­cial play­ers; no easy task which in it­self is forc­ing some fun­da­men­tal changes in many of these com­pa­nies.

— Over the same pe­ri­od, the FDA has changed from a bu­reau­crat­ic hur­dle to ac­tive helper. For es­tab­lished drug­mak­ers, the pol­i­cy at the agency has been to do what­ev­er it can to speed along new drugs. Un­der Com­mis­sion­er Scott Got­tlieb, that has even meant re­vers­ing three ear­li­er re­jec­tions. But so far there have been no cred­i­ble ac­cu­sa­tions that the open-door, speed-’em-up pol­i­cy has threat­ened pa­tients more than it has helped them.

— The rare/or­phan dis­ease R&D strat­e­gy — the sweet spot for com­pa­nies look­ing to score mar­ket-mov­ing ad­vances with high-priced drugs in record time — has come full cir­cle in biotech. And the dom­i­nant trend will stoke greater de­mands in 2019 for pric­ing re­form while beg­ging ques­tions about ef­fi­cien­cy in mar­ket­ing.

— Any analy­sis of R&D pro­duc­tiv­i­ty has to high­light how each of the top 10 play­ers in the in­dus­try per­formed. And once again we have some stel­lar suc­cess­es, some big no-shows and a lin­ger­ing con­cern that the slid­ing over­all ROI on R&D re­mains un­sus­tain­able for the group.

— While this an­nu­al look at NDAs over the course of the years nec­es­sar­i­ly fo­cused on FDA ap­provals — still the in­dus­try stan­dard for top glob­al per­for­mance — we’ll note that in 2018 the in­dus­try tru­ly be­gan to fo­cus on Asian drug OKs. In par­tic­u­lar, Chi­na has emerged as both a place to de­vel­op new drugs, and al­so strike deals to com­mer­cial­ize the world’s new drugs. Any reg­u­la­to­ry strat­e­gy that doesn’t in­clude Chi­na now is in­com­plete.

Here comes com­mer­cial­iza­tion risk

Sev­er­al of the big ap­provals in 2018 came through for com­pa­nies that will now start com­mer­cial­iza­tion work for the first time. The mar­ket­ing class of 2018 in­cludes GW Phar­ma, with its ground­break­ing an­ti-con­vul­sive meds, the re­mark­able ar­rival of RNAi at Al­ny­lam with big ad­vances in play for Agios, Loxo, Ul­tragenyx, Io­n­is/Akcea and even lit­tle Ve­rastem with its or­phaned PI3K drug du­velis­ib.