Thumbs Up/Thumbs Down: The FDA dam­aged it­self bad­ly with the Sarep­ta OK

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

  Califf’s de­ci­sion to bow to Janet Wood­cock on Sarep­ta hurts the en­tire biotech in­dus­try.

Janet Wood­cock’s de­ci­sion to over­ride the caus­tic in­ter­nal crit­i­cism of Sarep­ta’s weak and at times mis­lead­ing case for its Duchenne drug eteplirsen has dam­aged the FDA in ways that will leave a mark for years to come. Over­rul­ing her col­leagues – in­clud­ing se­nior FDA of­fi­cials — to pro­vide an ac­cel­er­at­ed ap­proval this week for this ap­pli­ca­tion sends a clear mes­sage to biotech: If you can muster enough pub­lic sup­port for a cam­paign – which in­cludes an er­ro­neous study for bait — you should pro­ceed full steam ahead. To his cred­it, FDA com­mis­sion­er Robert Califf post­ed his own re­view of the case as well as the heat­ed ob­jec­tions of two top reg­u­la­tors, in­clud­ing the cur­rent act­ing chief sci­en­tist. Not to his cred­it, Califf chose to stand down in “def­er­ence” to Wood­cock’s in­tran­si­gence, but not be­fore chid­ing Sarep­ta for pro­mot­ing a study that raised false hopes in the pa­tient com­mu­ni­ty, which swal­lowed it all hook, line and sinker. He was al­so none too hap­py that Sarep­ta stayed fo­cused on its lob­by­ing cam­paign rather than the clin­i­cal work that need­ed to be done. I sin­cere­ly hope that eteplirsen does every­thing that ad­vo­cates de­vout­ly be­lieve it does, with­out pre­sent­ing the dan­gers cit­ed for a “sci­en­tif­i­cal­ly el­e­gant place­bo.” And let’s hope pa­tients aren’t left to cov­er any of the $300,000 an­nu­al cost. Pay­ers, and ul­ti­mate­ly so­ci­ety, can and should do that now. And what hap­pens next? Does the FDA now slap back the next com­pa­ny to use sim­i­lar tac­tics, to try and reestab­lish old bound­aries? Or does it low­er its stan­dards per­ma­nent­ly, en­cour­ag­ing more large demon­stra­tions and dodgy da­ta to back weak ap­pli­ca­tions? That’s a po­si­tion the FDA should nev­er have been put in. And that is on Janet Wood­cock and Robert Califf.

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