Thumbs Up/Thumbs Down: Ma­jor in­sur­er de­liv­ers a bad blow to Duchenne fam­i­lies

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

  An­them’s rul­ing on Duchenne drug is a bad blow to fam­i­lies

The big US in­sur­er An­them has de­ter­mined that Sarep­ta’s con­tro­ver­sial Duchenne mus­cu­lar dy­s­tro­phy drug Ex­ondys 51 is “in­ves­ti­ga­tion­al and not med­ical­ly nec­es­sary,” a blow for fam­i­lies look­ing to gain cov­er­age for this drug. The de­ter­mi­na­tion cites a long and trou­bled his­to­ry for this drug, with failed stud­ies and a host of ques­tion­able moves Sarep­ta $SRPT made in ad­vanc­ing this drug on the back of a tiny study. “Ex­ondys 51 failed to show it im­proves health out­comes, and there­fore it is not a cov­ered ben­e­fit for our mem­bers,” An­them spokes­woman Leslie Por­ras told Reuters, con­firm­ing the ob­vi­ous. The dis­cus­sion over this drug turned in­to a bat­tle in­side the FDA, with CDER di­rec­tor Janet Wood­cock win­ning the ar­gu­ment in fa­vor of an ac­cel­er­at­ed ap­proval over the ve­he­ment ob­jec­tions of se­nior-lev­el of­fi­cials. As in­sur­ers refuse to cov­er this drug, which will be sold for $300,000, Wood­cock has set up a sce­nario where fam­i­lies will be ex­pect­ed to fork over huge sums to treat chil­dren with a drug they have vowed works just fine. That would be a trag­ic out­come. The drug is ex­per­i­men­tal, of course. But fam­i­lies shouldn’t be left on the hook for the cost of a rare dis­ease treat­ment like this, which may have no af­fect on the dis­ease at all. And there­in lies the tragedy be­hind this painful sit­u­a­tion. In­sur­ers can af­ford it and should step up and do the right thing, but we doubt they will. Af­ter all, in­sur­ers have in­vestors as well. Sarep­ta’s shares were down 8% to­day on the news.

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