ICER chas­tis­es J&J for over­pric­ing de­pres­sion drug es­ke­t­a­mine ‘where there is such need for treat­ment’

Cog­nizant of the myr­i­ad of ap­proved an­ti­de­pres­sants that of­ten don’t work, reg­u­la­tors en­dorsed J&J’s $JNJ phar­ma­ceu­ti­cal ver­sion of the hal­lu­cino­genic anes­thet­ic ke­t­a­mine — es­ke­t­a­mine — in March for treat­ment-re­sis­tant de­pres­sion, well aware that the orig­i­nal cat tran­quil­iz­er is fre­quent­ly used off-la­bel for se­vere de­pres­sion. On Thurs­day, ICER con­clud­ed that while the drug, sold as Spra­va­to, does con­fer a “promis­ing” clin­i­cal ben­e­fit, its cur­rent list price ex­ceeds a com­mon cost-ef­fec­tive­ness thresh­old by a mod­est mar­gin.

In 2017, an es­ti­mat­ed 17.3 mil­lion adults in the Unit­ed States — rough­ly 7% of all US adults — had at least one ma­jor de­pres­sive episode, ac­cord­ing to the NIH. Most an­ti­de­pres­sants usu­al­ly take a few weeks to work – and half of the pa­tients fail to ful­ly re­spond. The par­ty drug (some­times re­ferred to as Kit Kat or Vi­t­a­min K) and anes­thet­ic ke­t­a­mine which can lift de­pres­sion in many pa­tients with­in hours, must be ad­min­is­tered through in­fu­sion but can have pro­found dis­so­cia­tive side-ef­fects, and pa­tients typ­i­cal­ly re­lapse af­ter treat­ment ends.

Es­ke­t­a­mine is a low-dose, nasal-spray for­mu­la­tion of ke­t­a­mine — but due to its side-ef­fect pro­file, the J&J treat­ment is de­signed to be ad­min­is­tered in the pres­ence of a health­care prac­ti­tion­er.  It was ap­proved on the ba­sis of five piv­otal Phase III stud­ies in pa­tients with treat­ment-re­sis­tant de­pres­sion.

The da­ta used to ap­prove the drug sug­gests it is clin­i­cal­ly ef­fec­tive — but with the ab­sence of long-term safe­ty da­ta, the ev­i­dence is “promis­ing but in­con­clu­sive,” ICER re­searchers said. Since there are no head-to-head tri­als com­par­ing es­ke­t­a­mine with any com­para­tors — such as ke­t­a­mine, elec­tro­con­vul­sive ther­a­py, tran­scra­nial mag­net­ic stim­u­la­tion, oral an­ti­de­pres­sants, or aug­men­ta­tion with an­tipsy­chotics (e.g., olan­za­p­ine) — its rel­a­tive ben­e­fit is al­so hard to judge, they added.

Akin to NICE in the UK, ICER is an in­de­pen­dent body that an­a­lyzes the cost-ef­fec­tive­ness of drugs and oth­er med­ical ser­vices in the Unit­ed States. Un­like NICE, though, ICER is not gov­ern­ment-af­fil­i­at­ed, but its de­ter­mi­na­tions are in­creas­ing­ly be­com­ing in­flu­en­tial with pay­ers.

ICER con­duct­ed its analy­ses us­ing two mea­sures: 1) QALYs, or qual­i­ty-ad­just­ed life-years, 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. Es­sen­tial­ly, one QALY is equal to one year of life in per­fect health. 2) Life years gained (LYG), which ex­press­es the ad­di­tion­al num­ber of years of life that a per­son lives as a re­sult of re­ceiv­ing treat­ment.

Com­pared with no ad­di­tion­al treat­ment be­yond a back­ground an­ti­de­pres­sant, treat­ment with es­ke­t­a­mine plus a back­ground an­ti­de­pres­sant re­sult­ed in im­por­tant QALY gains in pa­tients with treat­ment-re­sis­tant de­pres­sion (TRD), ICER said.

Us­ing the es­ke­t­a­mine list price of $295 per 28 mg in­tranasal de­vice, the treat­ment’s use re­sults in an in­cre­men­tal cost-ef­fec­tive­ness ra­tio of ap­prox­i­mate­ly $198,000 per QALY com­pared to no ad­di­tion­al treat­ment, ex­ceed­ing the com­mon­ly cit­ed cost-ef­fec­tive­ness thresh­olds of be­tween $50,000-$150,000 per QALY. Mean­while, es­ke­t­a­mine is es­ti­mat­ed to cost ap­prox­i­mate­ly $2.6 mil­lion per life year gained, ICER found.

Es­ke­t­a­mine’s ap­proval was al­so meant to en­hance ac­cess to treat­ment — since ke­t­a­mine is not cov­ered by health in­sur­ers — al­though there is a con­cern that there may still be high out-of-pock­et ex­pens­es through de­ductibles or non-cov­er­age poli­cies.

David Rind

“Es­ke­t­a­mine shows some ben­e­fits for such pa­tients and pro­vides an FDA-ap­proved treat­ment for TRD that may be cov­ered by pay­ers; how­ev­er, it is con­cern­ing to have an over­priced ther­a­py where there is such need for treat­ment. Ad­di­tion­al­ly, the sim­i­lar­i­ty of ke­t­a­mine to es­ke­t­a­mine rais­es is­sues for all stake­hold­ers about how to con­sid­er off-la­bel pre­scrip­tion and cov­er­age of a treat­ment that has not been as well stud­ied but is be­ing in­creas­ing­ly used for TRD,” said ICER’s CMO David Rind in a state­ment.

The ICER re­port was pub­lished on Thurs­day hav­ing in­cor­po­rat­ed the feed­back from pa­tient groups, clin­i­cians, drug man­u­fac­tur­ers, and oth­er stake­hold­ers to the draft ver­sion orig­i­nal­ly un­veiled in March. A fi­nal re­port is ex­pect­ed to be pub­lished in June, fol­low­ing a vote lat­er this month.

J&J dis­agrees with this re­port, a Janssen spokesper­son told End­points News. “It un­der­es­ti­mates the proven short- and long-term ben­e­fits that this treat­ment…brings to TRD pa­tients in need. The in­ac­cu­rate as­sump­tions in the draft re­port re­lat­ed to the pos­i­tive ben­e­fit risk pro­file of Spra­va­to and the com­par­i­son be­tween this FDA ap­proved treat­ment and ke­t­a­mine, a treat­ment be­ing used off-la­bel that has not been ad­e­quate­ly stud­ied and is viewed as ex­per­i­men­tal for TRD, are reck­less.”

Due to a lack of com­par­a­tive da­ta be­tween es­ke­t­a­mine and ke­t­a­mine, ICER was not able to ex­am­ine rel­a­tive cost-ef­fec­tive­ness be­tween the two ther­a­pies. In­stead, the in­sti­tute com­pared the in­di­vid­ual one-year costs and found that es­ke­t­a­mine was ten times more ex­pen­sive than ke­t­a­mine in the first year of use — de­spite the ad­min­is­tra­tion costs as­so­ci­at­ed with pro­vid­ing ke­t­a­mine in­tra­venous­ly.

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

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