ICER up­date on Duchenne drugs push­es up price es­ti­mates for Ex­ondys 51, Em­flaza ahead of Ju­ly pan­el re­view

In­creas­ing­ly in­flu­en­tial drug cost-ef­fec­tive­ness watch­dog ICER still doesn’t think that the drugs ap­proved to treat Duchenne mus­cu­lar dy­s­tro­phy have so far proved their val­ue to pa­tients. On Thurs­day, they used sig­nif­i­cant­ly high­er price es­ti­mates for two ther­a­pies in their mod­els, mak­ing them look even less at­trac­tive — and ac­knowl­edged that the es­ti­mates used in their draft re­port in May were in­cor­rect.

ICER’s up­dat­ed re­port on PTC Ther­a­peu­tics’ $PTCT steroid de­flaza­cort and Sarep­ta’s $SRPT Ex­ondys 51 ar­rives ahead of a meet­ing of ex­perts that will dis­cuss the body of ev­i­dence un­der­ly­ing the land­scape of ex­ist­ing and in­com­ing Duchenne mus­cu­lar dy­s­tro­phy (DMD) ther­a­pies. In their analy­sis, ICER has used a price that ex­ceeds $1 mil­lion a year for Ex­ondys 51 and $81,400 for Em­flaza — an old cor­ti­cos­teroid that many fam­i­lies once im­port­ed from over­seas at a cost of about $1,000 a year.

In the Unit­ed States, rough­ly 6,000 young boys suf­fer from the mus­cle wast­ing dis­ease — caused by the ab­sence of dy­s­trophin, a pro­tein that helps keep mus­cle cells in­tact. Symp­toms tend to kick in and pro­gres­sive­ly wors­en be­tween the ages of 3 to 5, typ­i­cal­ly caus­ing the pa­tient to be­come wheel­chair-bound by their ear­ly teens. Even­tu­al­ly, pa­tients suc­cumb to the dis­ease by their 30s. Cor­ti­cos­teroids, which work by di­min­ish­ing in­flam­ma­tion and lim­it­ing the im­mune sys­tem’s ac­tiv­i­ty, are com­mon­ly used to treat DMD.

ICER eval­u­at­ed the ef­fi­ca­cy, safe­ty and cost-ef­fec­tive­ness of four treat­ments in its ev­i­dence re­port. It looked at the steroids de­flaza­cort (sold as Em­flaza) and pred­nisone — as well as the ex­on-skip­ping drugs: the ap­proved eteplirsen (mar­ket­ed as Ex­ondys 51) and the ex­per­i­men­tal golodirsen (both come from Sarep­ta, and each treat­ment is de­signed to treat a dif­fer­ent sub­set of DMD pa­tients). As it con­clud­ed in its draft re­port in May, ICER re­it­er­at­ed that the ev­i­dence sup­port­ing each of the four treat­ments is lack­ing, and their im­pact on pa­tients un­clear.

In the ev­i­dence re­port pub­lished on Thurs­day — which pre­cedes an ad­vi­so­ry pan­el meet­ing that will make its rec­om­men­da­tions to ICER on Ju­ly 25 — used high­er an­nu­al cost es­ti­mates for Ex­ondys 51 and Em­flaza to con­duct var­i­ous cost-ef­fec­tive­ness cal­cu­la­tions.

DMD drug dos­ing is based on weight, which typ­i­cal­ly varies among pa­tients. Akin to its May re­port, ICER used an­nu­al cost es­ti­mates for a 40 kg pa­tient to cal­cu­late cost-ef­fec­tive­ness in its lat­est up­date, putting a fresh spot­light on the ex­ist­ing con­tro­ver­sy sur­round­ing DMD drugs.

In the ev­i­dence re­port, ICER pre­sent­ed this chart:

Source: ICER, Ju­ly 2019

Click on the im­age to see the full-sized ver­sion

In May, when ICER pub­lished its draft re­port, the non-prof­it pre­sent­ed this chart:

Source: ICER, May 2019

Click on the im­age to see the full-sized ver­sion

“We do not know what fac­tors ICER used in their mod­el­ing. We have not raised the price of the drug since launch and are not plan­ning any price in­creas­es,” a Sarep­ta spokesper­son told End­points News.

PTC con­curred. There has been no change in Em­flaza’s pric­ing or dis­counts, a spokesper­son told End­points News.

Lat­er on Fri­day, a spokesper­son for ICER clar­i­fied that the ta­ble in the ear­li­er draft re­port dis­played in­cor­rect an­nu­al costs for both Em­flaza and Ex­ondys 51. ICER’s fi­nal re­port is ex­pect­ed in Au­gust.

So­cial im­age: Sarep­ta, AP Im­ages

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Scott Gottlieb, AP Images

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