Vas Narasimhan. AP Images

No­var­tis makes new da­ta in­tegri­ty com­mit­ment

Look­ing to slow the fall­out from No­var­tis’ da­ta ma­nip­u­la­tion fi­as­co with its bil­lion-dol­lar gene ther­a­py Zol­gens­ma (onasemno­gene abepar­vovec-xioi), CEO Vas­ant Narasimhan told an in­vestor con­fer­ence on Mon­day that the com­pa­ny will be more proac­tive in re­port­ing da­ta in­tegri­ty is­sues to the FDA.

“We are vol­un­tar­i­ly and proac­tive­ly tak­ing a pledge with the FDA to en­sure that we will in­form them with­in five busi­ness days of any cred­i­ble al­le­ga­tion re­lat­ed to da­ta in­tegri­ty,” Narasimhan said.

He al­so said No­var­tis has re­spond­ed to the FDA’s Form 483 and is mak­ing doc­u­ments avail­able as re­quest­ed, while re­it­er­at­ing that the da­ta ma­nip­u­la­tion un­cov­ered at the San Diego site “does not im­pact the safe­ty, ef­fi­ca­cy or qual­i­ty of Zol­gens­ma.”

The pledge fol­lows sharp com­ments made last week by act­ing FDA Com­mis­sion­er Ned Sharp­less, who did not men­tion No­var­tis by name, but clear­ly sin­gled the com­pa­ny out in his re­marks be­fore the Re­search!Amer­i­ca 2019 Na­tion­al Health Fo­rum.

While ad­dress­ing da­ta fraud, Sharp­less said: “So it should not be sur­pris­ing to me now at FDA that if peo­ple will lie to get their pa­per pub­lished or get their grant fund­ed, then peo­ple will al­so lie to get a bil­lion dol­lar med­ical prod­uct ap­proved.”

Five De­mo­c­ra­t­ic sen­a­tors (in­clud­ing pres­i­den­tial hope­fuls Bernie Sanders and Eliz­a­beth War­ren) al­so pounced on the da­ta ma­nip­u­la­tion last month, say­ing this “scan­dal smacks of the phar­ma­ceu­ti­cal in­dus­try’s priv­i­lege and greed.” They al­so called on the FDA to hold No­var­tis ac­count­able and they ques­tioned whether FDA has plans to re-is­sue a fi­nal rule on fal­si­fied da­ta that the agency with­drew last Sep­tem­ber.

No­var­tis’ com­mit­ment Mon­day to re­port al­le­ga­tions of da­ta in­tegri­ty trans­gres­sions with­in five busi­ness days brings up an in­ter­est­ing gray area in FDA’s reg­u­la­tions for when com­pa­nies should re­port such is­sues that do not re­sult in se­ri­ous ad­verse events.

As Green­leaf Health ex­perts and for­mer FDA of­fi­cials wrote on the Food and Drug Law In­sti­tute’s web­site, “While it is clear sus­pen­sion or ter­mi­na­tion of the tri­al re­quires the spon­sor to no­ti­fy FDA, spon­sors are of­ten per­plexed about when to no­ti­fy FDA when the pro­to­col non­com­pli­ance is­sue is sig­nif­i­cant, but yet not so sig­nif­i­cant that it re­quires sus­pen­sion or ter­mi­na­tion of the tri­al.”

In ad­di­tion, they note that in the case of emer­gency de­vi­a­tions made to pro­tect the life or well-be­ing of a sub­ject, the FDA must re­ceive no­tice with­in five work­ing days of the de­vi­a­tion, al­though the FDA’s reg­u­la­tions “are not so clear­ly de­fined con­cern­ing when the spon­sor’s du­ty aris­es to re­port a se­ri­ous breach of pro­to­col that does not re­sult in a se­ri­ous ad­verse event, but may cause FDA to ques­tion the re­li­a­bil­i­ty of the da­ta.”

But the ex­perts note that proac­tive re­port­ing to the agency and work­ing with the FDA can be help­ful. Spon­sors “of­ten will find that it is ad­van­ta­geous to re­port da­ta in­tegri­ty is­sues to FDA soon af­ter dis­cov­ery rather than wait­ing for FDA to dis­cov­er the da­ta in­tegri­ty is­sue dur­ing an in­spec­tion,” they added.

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.

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