New peanut al­ler­gy ther­a­pies aren’t as ef­fec­tive — or near­ly as in­ex­pen­sive — as ab­sti­nence, says ICER

While peanut al­ler­gy ther­a­pies from Aim­mune and DBV Tech­nolo­gies are locked in a race to the fin­ish line, in­flu­en­tial cost-ef­fec­tive­ness watch­dog ICER has de­ter­mined that nei­ther ther­a­py of­fers su­pe­ri­or net health ben­e­fit com­pared to strict peanut avoid­ance, in a fi­nal re­port pub­lished on Wednes­day.

Tra­di­tion­al­ly, peanut al­ler­gies are man­aged by avoid­ance, but the threat of ac­ci­den­tal ex­po­sure can­not be nul­li­fied.

Some physi­cians have al­so been dos­ing pa­tients with peanut pow­der oral­ly, al­beit off la­bel. Aim­mune’s $AIMT AR101 and DBV’s $DB­VT Vi­askin Peanut are set to be the pi­o­neer­ing peanut al­ler­gy treat­ments ap­proved by the FDA. AR101 is ef­fec­tive­ly peanut flour, which must be mixed in­to pud­ding, ap­ple­sauce, or oth­er foods. Dos­ing is es­ca­lat­ed grad­u­al­ly, and the ther­a­py must be con­tin­ued dai­ly to main­tain de­sen­si­ti­za­tion. Vi­askin Peanut is a patch ap­plied dai­ly to the up­per back that de­liv­ers peanut anti­gen to de­sen­si­tize the pa­tient. The patch must be worn for longer pe­ri­ods as time pass­es and used every day to main­tain de­sen­si­ti­za­tion.

While the ICER pan­el ac­knowl­edged that de­sen­si­ti­za­tion as a sur­ro­gate out­come is promis­ing, they were look­ing for da­ta to demon­strate that de­sen­si­ti­za­tion is linked to im­proved qual­i­ty of life and re­duced re­ac­tions to ac­ci­den­tal ex­po­sure to peanuts.

Based on sur­ro­gate out­comes — oral food chal­lenges — AR101 ap­pears to be more ef­fec­tive than Vi­askin Peanut, but car­ries more ad­verse ef­fects. In ad­di­tion, in stud­ies of AR101, Vi­askin Peanut, and off-la­bel ther­a­py (OIT), ep­i­neph­rine use and sys­temic al­ler­gic re­ac­tions in­creased, ICER found.

Over­all, the body of ev­i­dence is not strong enough to sug­gest that AR101 or Vi­askin Peanut of­fer a su­pe­ri­or net health ben­e­fit ver­sus strict peanut avoid­ance, the pan­el said, adding that there is al­so un­cer­tain­ty about the long-term ef­fects of ei­ther ther­a­py.

“There is hope that the rates of sys­temic al­ler­gic re­ac­tions, ep­i­neph­rine use, and re­ac­tions to ac­ci­den­tal ex­po­sure will de­crease with con­tin­ued ther­a­py, but this re­mains to be demon­strat­ed. The po­ten­tial need for life­long ther­a­py rais­es is­sues about long-term ad­her­ence to treat­ment, par­tic­u­lar­ly dur­ing ado­les­cence and young adult­hood,” ICER said.

ICER did ac­knowl­edge, as it did in its draft re­port, that one of the lim­i­ta­tions of its analy­sis is that it as­sumed the util­i­ty of the two peanut al­ler­gy ther­a­pies on the ba­sis of ex­ist­ing da­ta on food al­ler­gies, but not specif­i­cal­ly the peanut al­ler­gy pa­tient pop­u­la­tion, due to “the pauci­ty of pref­er­ence-weight­ed health-re­lat­ed qual­i­ty of life es­ti­mates in food al­ler­gy pa­tients and their care­givers.”

“With both AR101 and Vi­askin, pa­tients still need to spend the rest of their life avoid­ing peanuts and still wor­ried about ac­ci­den­tal ex­po­sure and won­der­ing if their peanut treat­ment will pro­tect them from ana­phy­lax­is,” said James Wallen, who owns AMW Con­sul­tants, which is fo­cused on as­sist­ing for­eign biotech­nol­o­gy com­pa­nies un­der­stand the US al­ler­gy im­munother­a­py mar­ket.

“With OIT (off-la­bel oral im­munother­a­py), the pa­tient is eat­ing peanuts dai­ly so they no longer have that fear of avoid­ing ac­ci­den­tal ex­po­sure. When you are deal­ing with chil­dren (since both ther­a­pies on­ly looked at or “suc­ceed­ed” in kids), the QOL (qual­i­ty of life) dif­fer­ences al­so have to con­sid­er the par­ent’s lev­el of com­fort with their child’s de­sen­si­ti­za­tion suc­cess. This is where OIT is com­plete­ly dif­fer­ent and vast­ly su­pe­ri­or from both AR 101 and Vi­askin in my opin­ion,” he told End­points News.

Nei­ther com­pa­ny has di­vulged its pric­ing plans, so ICER used an­a­lyst pro­jec­tions to eval­u­ate each ther­a­py’s long-term cost-ef­fec­tive­ness: AR101 at $4,200/year and Vi­askin Peanut at $6,500/year.

One of the ways ICER makes these cal­cu­la­tions is on qual­i­ty-ad­just­ed-life-years (QALYs), a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life.

Treat­ment with AR101 re­sult­ed in 0.75 in­cre­men­tal QALYs, while treat­ment with Vi­askin Peanut came up rel­a­tive­ly short, re­sult­ing in 0.26 in­cre­men­tal QALYs — when both were com­pared to no im­munother­a­py treat­ment over a life­time, ICER’s analy­sis sug­gest­ed.

“These ben­e­fits are due to im­proved sub­jec­tive qual­i­ty of life de­spite the rel­a­tive rar­i­ty with which se­ri­ous events oc­cur. The ul­ti­mate val­ue of these prod­ucts will be de­ter­mined by the prices that are set by the man­u­fac­tur­ers and their long-term ef­fec­tive­ness,” ICER con­clud­ed.

ICER’s analy­sis al­so in­di­cat­ed on­ly 41% of el­i­gi­ble pa­tients could be treat­ed with AR101 and 71% of el­i­gi­ble pa­tients could be treat­ed with Vi­askin Peanut in a giv­en year with­out ex­ceed­ing ICER’s bud­get im­pact thresh­old of $991 mil­lion.

Both Aim­mune and DBV said ICER’s con­clu­sions were pre­ma­ture.

The fi­nal re­port is gen­er­al­ly bi­ased against im­munother­a­py — and fails to cap­ture the full val­ue of AR101, Aim­mune said, un­der­scor­ing that ICER did not in­clude in its analy­sis the long-term ef­fi­ca­cy and qual­i­ty-of-life da­ta from an open-la­bel fol­low-on study, as well as clin­i­cal out­comes da­ta from the com­pa­ny’s Eu­ro­pean late-stage tri­al.

“We be­lieve this fi­nal re­port rais­es more ques­tions than it an­swers and should be viewed as an ear­ly start­ing point for fu­ture con­ver­sa­tions—not the fi­nal word—about the val­ue of AR101,” Aim­mune chief Jayson Dal­las said in a state­ment.

Mean­while, DBV voiced sim­i­lar con­cerns, sug­gest­ing it dis­agreed with ICER’s method­ol­o­gy as well as the tim­ing of the re­port.

Among oth­er points of con­tention, DBV took is­sue with ICER’s in­clu­sion of a Lancet study on oral im­munother­a­py (OIT), which did not in­clude Vi­askin Peanut clin­i­cal tri­als. “DBV has con­cerns with group­ing Vi­askin Peanut’s rat­ing along with this sys­temic OIT analy­sis,” a spokesper­son told End­points News.

Aim­mune ef­fec­tive­ly leapfrogged DBV when the lat­ter re­scind­ed an ap­pli­ca­tion to mar­ket Vi­askin Peanut patch last year in re­sponse to FDA con­cerns about the state of man­u­fac­tur­ing and qual­i­ty con­trol da­ta sub­mit­ted.

An FDA de­ci­sion for AR101 is ex­pect­ed in Jan­u­ary 2020, while DBV is ex­pect­ed to sub­mit its mar­ket­ing ap­pli­ca­tion lat­er in 2019. The so far un­tapped mar­ket is ex­pect­ed to grow to $4.5 bil­lion in 2027 glob­al­ly, ac­cord­ing to Glob­al­Da­ta.

Inside FDA HQ (File photo)

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Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

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