FDA Approves First NDA Under Real-Time Oncology Review Pilot

As the US grapples with whether Novartis’ new gene therapy for spinal muscular atrophy should cost $2.1 million, the US Food and Drug Administration (FDA) on Friday also quietly signaled that another Novartis drug would effectively kick off a new way for cancer drugs to be approved more quickly.

The approval for the breast cancer treatment Piqray (alpelisib) in combination with fulvestrant and alongside a companion diagnostic — came more than three months ahead of its PDUFA deadline and a little more than five months after it was submitted thanks to FDA’s Real-Time Oncology Review (RTOR) pilot and Assessment Aid (AAid) programs.

Up until last Friday, FDA had only approved supplemental new drug applications (sNDAs) and supplemental biologics license applications (sBLAS) under the RTOR pilot. FDA had never even publicly said that it was expanding the RTOR pilot to NDAs until announcing that Piqray’s NDA had been approved.

News of the expanding pilot should be well-received by the biopharma industry, which so far has seen significant time shaved off their application approval timelines thanks to the pilot. For instance, last July, an sNDA for Novartis' breast cancer drug Kisqali (ribociclib) was approved in less than a month after its formal submission. Similarly, an sNDA for Roche’s Kadcyla (trastuzumab emtansine) earlier this month was approved in just over 12 weeks after submission.

In 2018, two products were approved using the RTOR and AAid, while two products used the RTOR alone and three used the AAid alone.

And although the RTOR frequently asked questions page still says the pilot might be extended to full NDAs or BLAs, an FDA spokesperson told Focus that “other novel drug NDAs may be eligible for the RTOR pilot program and will be evaluated on a case by case basis.”

So how does the RTOR pilot work?

RTOR permits FDA to access key data prior to the official submission of the application, allowing the review team to begin their review earlier and communicate with the applicant prior to the application’s actual submission.

The AAid pilot, meanwhile, uses a multidisciplinary review template divided into two parts: the applicant’s position and FDA’s assessment. The goal is to focus FDA’s written review on critical thinking and consistency and decrease the time spent on administrative tasks, such as formatting, FDA said.

And as far as when additional guidance is coming for sponsors seeking to win approval for new NDAs or BLAs under the pilots, an FDA spokesperson told Focus: "The Real-Time Oncology Review Program is a pilot program and we are continuing to evaluate drug applications on a case by case basis. When we have additional guidance for sponsors, we will share the information at that time."