Ger­many’s IQWiG finds first tu­mor-ag­nos­tic drug pro­vides no added ben­e­fit

Ger­many’s In­sti­tute for Qual­i­ty and Ef­fi­cien­cy in Health Care (IQWiG) this week said that Bay­er’s Vi­t­rakvi (larotrec­tinib), ap­proved in the EU last sum­mer, has not been proven to pro­vide an added ben­e­fit.

Vi­t­rakvi was the first med­i­cine to be ap­proved in the EU as a treat­ment for tu­mors with a spe­cif­ic mu­ta­tion, re­gard­less of where in the body the tu­mor orig­i­nat­ed. But the treat­ment’s safe­ty and ef­fi­ca­cy were eval­u­at­ed in three sin­gle-arm tri­als, and IQWiG took is­sue with the lack of com­para­tor arms in the tri­als.“

In ex­cep­tion­al cas­es, stud­ies with­out com­para­tor da­ta may be suf­fi­cient for an ap­proval if the aim is to show that the sub­stance has an ef­fect on the tu­mor. Ex­plic­it com­para­tor da­ta are need­ed, how­ev­er, if the ques­tion is whether such an ef­fect is as­so­ci­at­ed with a pa­tient-rel­e­vant ben­e­fit,” IQWiG said.

The in­sti­tute al­so found that re­gard­ing over­all sur­vival, the dif­fer­ences be­tween larotrec­tinib and oth­er treat­ments ob­served so far “were not so large in any of the can­cer dis­eases that they could not be caused by sys­tem­at­ic bias alone. Re­gard­ing pa­tient-rel­e­vant out­comes from the cat­e­gories of mor­bid­i­ty, health-re­lat­ed qual­i­ty of life and side ef­fects, there were no suit­able da­ta or no da­ta dif­fer­en­ti­at­ed by tu­mor type ei­ther for the new drug or for the com­para­tor ther­a­pies, so that it was al­so not pos­si­ble to de­rive an added ben­e­fit.”

De­fi­cien­cies in the his­tol­ogy-in­de­pen­dent analy­ses pre­sent­ed by Bay­er were al­so not­ed, as were a lack of pre­spec­i­fied analy­ses and se­lec­tive re­sults re­port­ing.

Bay­er, how­ev­er, told Fo­cus via email that it does not share the view of IQWiG and is con­vinced of the added ben­e­fit “due to the ex­cep­tion­al­ly high ef­fec­tive­ness and good tol­er­a­bil­i­ty shown in clin­i­cal stud­ies for pa­tients with so-called TRK fu­sion tu­mors.”

Bay­er al­so said that IQWiG’s meth­ods “are not yet ap­pro­pri­ate for ap­prais­ing his­tol­ogy in­de­pen­dent prod­ucts, so this rec­om­men­da­tion is not sur­pris­ing.” The com­pa­ny al­so not­ed that the ap­proval by the EMA is con­di­tion­al so “fur­ther clin­i­cal ev­i­dence will be gen­er­at­ed af­ter the EMA ap­proval via reg­istries and non-in­ter­ven­tion­al phase IV stud­ies.”

RAPS: First pub­lished in Reg­u­la­to­ry Fo­cus™ by the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety, the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care prod­ucts. Click here for more in­for­ma­tion.

So­cial im­age: IQWiG lo­go, Ralf Baum­garten

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