ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarep­ta and the FDA as the US drug price watch­dog con­cludes that as cur­rent­ly priced, their re­spec­tive new treat­ments for Duchenne mus­cu­lar dy­s­tro­phy are de­cid­ed­ly not cost-ef­fec­tive.

The fi­nal re­port — which ce­ments the con­clu­sions of a draft is­sued in May — in­cor­po­rates the opin­ion of a pan­el of 17 ex­perts ICER con­vened in a pub­lic meet­ing last month. It al­so based its analy­sis of Em­flaza (de­flaza­cort) and Ex­ondys 51 (eteplirsen) on up­dat­ed an­nu­al costs of $81,400 and over $1 mil­lion, re­spec­tive­ly, af­ter cit­ing “in­cor­rect” low­er num­bers in the ini­tial cal­cu­la­tions.

As cor­ti­cos­teroids and sup­port­ive care cur­rent­ly form the bedrock of treat­ment for the 6,000 young DMD pa­tients in the US, ICER (more for­mal­ly known as In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view) set out to make two main com­par­isons with costs in mind: Is de­flaza­cort that much more ef­fec­tive than pred­nisone, a much cheap­er cor­ti­cos­teroid? Does eteplirsen bring a big enough im­prove­ment to war­rant adding it to the stan­dard reg­i­men giv­en its high price?

The an­swer to both ques­tions, ac­cord­ing to ICER, is no.

To be sure, 10 out of 17 mem­bers on its ad­vi­so­ry coun­cil agreed that de­flaza­cort con­fers a bet­ter health ben­e­fit than pred­nisone, as pa­tients tak­ing the drug ap­pears to have bet­ter mo­tor func­tion and ex­pe­ri­ence less weight gain. The lat­ter point is al­so im­por­tant to care­givers as it re­duces their bur­den on a day-to-day ba­sis, they ac­knowl­edged.

How­ev­er, they ar­gue that the drug’s price tag is un­jus­ti­fied, with the ma­jor­i­ty agree­ing the long­time val­ue for mon­ey is low.

While US pa­tients once im­port­ed the drug for as lit­tle as $1,000 per year, Marathon jacked the price up to $89,000 af­ter ac­quir­ing it and win­ning an FDA ap­proval based on old da­ta. PTC has sub­se­quent­ly ac­quired the con­tro­ver­sial biotech and pledged to re­duce the price.

“The events around and fol­low­ing the FDA ap­proval of de­flaza­cort is a mod­el of how not to pro­mote in­no­va­tion,” read the re­port.

The pan­elists were less im­pressed by eteplirsen’s re­sults, vot­ing 16 to 1 that there is not ad­e­quate ev­i­dence to claim treat­ment with the drug is su­pe­ri­or to cor­ti­cos­teroid and sup­port­ive care alone. They were al­so unan­i­mous­ly skep­ti­cal of Sarep­ta’s ex­per­i­men­tal treat­ment golodirsen, which was al­so in­clud­ed for re­view but not a sub­stan­tial analy­sis due to the lack of clin­i­cal da­ta and pric­ing in­for­ma­tion.

Giv­en that eteplirsen’s ef­fi­ca­cy has yet to be demon­strat­ed — Sarep­ta has just re­cent­ly sub­mit­ted a plan for a con­fir­ma­to­ry tri­al, with da­ta ex­pect­ed in 2024 — ICER sug­gest­ed it should be priced clos­er to the mar­gin­al cost.

The main take­away:

Ac­claim is right­ly giv­en to in­no­v­a­tive treat­ments that can of­fer ben­e­fits for pa­tients and fam­i­lies grap­pling with se­ri­ous con­di­tions that lead to ear­ly death. How­ev­er, when treat­ments like eteplirsen are in­tro­duced with ev­i­dence that is whol­ly in­ad­e­quate to demon­strate clin­i­cal ben­e­fit, at an ex­treme­ly high price, and with­out an ad­e­quate com­mit­ment from the man­u­fac­tur­er to gen­er­ate and share fur­ther ev­i­dence with­in a rapid time­line, pa­tients, fam­i­lies, oth­er pa­tients in the health sys­tem, and even fu­ture in­no­va­tors suf­fer sig­nif­i­cant loss­es.

Pa­tient ad­vo­cates at the pub­lic meet­ing, how­ev­er, ques­tioned ICER’s method­ol­o­gy. Fleur Chan­dler, a health econ­o­mist with Duchenne UK, called their as­sump­tions “huge, mul­ti-lay­ered and not test­ed.”

“The ICER process sim­ply did not al­low enough time to ad­e­quate­ly re­view a dis­ease as com­plex as DMD; for sys­tem­at­ic lit­er­a­ture re­views, ev­i­dence col­lec­tion or ro­bust mod­el­ling,” she said at the meet­ing, ac­cord­ing to a sum­ma­ry.

A rep­re­sen­ta­tive of the in­flu­en­tial Par­ent Pro­ject Mus­cu­lar Dy­s­tro­phy fur­ther urged ICER to con­sid­er de­lay­ing their re­view of prod­ucts gain­ing ac­cel­er­at­ed ap­proval as “there is no val­ue in con­duct­ing an as­sess­ment at a time point in which it is known in ad­vance that ‘in­suf­fi­cient ev­i­dence’ will be avail­able to con­duct analy­ses.”

She al­so cau­tioned against ICER’s sug­ges­tion to pay­ers about pri­or au­tho­riza­tion, in which in­sur­ers set up cri­te­ria to de­ter­mine who would qual­i­fy for cov­er­age. Fre­quent pri­or au­tho­riza­tions could re­sult in gaps or de­lays in care and un­der­mine ef­fec­tive­ness, she said.

So­cial im­age: The key patho­log­ic change of the Duchenne mus­cu­lar dy­s­tro­phy is the my­onecro­sis. At an ear­ly phase, necrot­ic fibers ap­pear swollen, ho­mo­ge­neous and deeply eosinophilic. Shut­ter­stock

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